Tag Archive for: sickle cell disease

Clumps of sickle cell blocking a blood vessel

The future of sickle cell disease treatment through gene therapy

Clumps of sickle cell blocking a blood vessel

In a new review article published in The CRISPR Journal, researchers from Children’s National Hospital discuss the progress of gene therapies for sickle cell disease – from preclinical studies to clinical trials and FDA approval – along with the many challenges of these treatments.

Over the past few years, the advances of gene therapy for sickle cell disease (SCD) offer a potential cure for a condition previously managed only through symptom relief and limited treatments. SCD is a hereditary blood disorder caused by a mutation in the beta globin gene leading to painful symptoms and complications.

In a new review article published in The CRISPR Journal, researchers from Children’s National Hospital discuss the progress of gene therapies for SCD – from preclinical studies to clinical trials and FDA approval – along with the many challenges of these treatments.

“We are hoping to bring attention to the past, present and future of this topic,” says Henna Butt, MD, pediatric hematology oncology fellow at Children’s National and one of the review authors. “It is exciting to see the technology move forward and see how far we have come in a disease where so little progress has been made historically.”

The hold up in the field

Gene therapy for SCD remains a time- and resource-intensive process, often taking several months from the initial patient consultation to treatment.

“Progress in SCD gene therapy has been slowed by high costs, limited accessibility and safety concerns, such as off-target effects,” says Dr. Butt. “Additionally, long-term efficacy data is still needed to confirm the durability of these treatments. Regulatory hurdles and ethical considerations also contribute to the delays.”

Moving the field forward

“By using cutting-edge techniques like CRISPR and base editing to directly correct the genetic mutation responsible for SCD, these therapies have the potential to offer long-term or even permanent relief,” says Dr. Butt. “Success in this area could revolutionize treatment options, improve patient outcomes and reduce the global burden of SCD — especially as therapies become more accessible and affordable.”

“Advancing gene therapy for sickle cell disease requires not just scientific innovation, but also the clinical expertise and systems to deliver it safely and effectively,” says David Jacobsohn, MD, SCM, MBAD, chief of Bone and Marrow Transplantation at Children’s National. “As access expands, we must ensure these therapies reach the patients who need them most.”

The patient benefit

Gene therapy offers a potential cure for SCD, reducing the need for ongoing treatments and significantly improving quality of life. It can lower the risk of complications and infections, and over time, reduce healthcare costs – especially for patients with limited access to traditional care. Raising awareness of current challenges can help drive advocacy for affordability and access.

Children’s National leads the way

Children’s National was the first hospital in the world to collect stem cells for the LYFGENIA™ treatment and one of the few pediatric hospitals in the country that offers both FDA-approved sickle cell disease gene therapies – CASGEVY™ (exagamglogene autotemcel) and LYFGENIA™ (lovotibeglogene autotemcel).

Additional authors from Children’s National include: Mamatha Mandava, MD, and David Jacobsohn, MD, SCM, MBA

Read the full review published in The CRISPR Journal.

Kendric receives care at Children's National.

Rewriting the script for sickle cell disease

More than 100,000 Americans have sickle cell disease, an inherited blood disorder that can cause excruciating pain crises and shorter life expectancies.

Children’s National has one of the largest sickle cell programs in the United States. We are pioneering treatments and provide specialized care to about 1,500 patients each year. We participate in clinical trials to improve outcomes, shorten treatment time, reduce complications and minimize the need for opioids and chemotherapy.

Kendric receives care at Children's National.

Kendric receives care at Children’s National.

In recognition of our clinical and research excellence, Children’s National was one of a few U.S. pediatric hospitals selected to offer two promising new FDA-approved gene therapies.

Hematologist Robert Sheppard Nickel, M.D., leads a study to reduce toxicities in bone marrow transplants. “Years of development led to these curative therapies,” Dr. Nickel says. “I hope in the future we can safely cure more children with sickle cell disease.”

“The future looks promising to revolutionize the lives of our patients and make these therapies accessible worldwide,” says Andrew Campbell, M.D., director of our Comprehensive Sickle Cell Disease Program.

Kendric and Nasir find hope

In May 2024 at Children’s National, 12-year-old Kendric of Clinton, Maryland, became the world’s first patient with sickle cell disease to begin a commercially approved gene therapy that could dramatically reduce or even eliminate his pain. It involved extracting his bone marrow stem cells; genetically modifying them in a specialized lab to reduce the risk of sickling; and then, after chemotherapy, infusing them back into his bloodstream.

Expert, compassionate care empowered Kendric to understand the science behind his treatment and chart a path to recovery. “My care team taught me how to deal with my disease and everything that I need to know for the future,” he says. “They gave me hope that I could be cured.”

Nasir and his care team

Nasir and his care team.

Nasir, age 20, spent his childhood waiting to find a match for a stem cell transplant to address his sickle cell disease. Finally, in 2023, at Children’s National, he found an answer in gene therapy to alter his own cells.

Due to painful episodes and the need for frequent blood transfusions, both Kendric and Nasir missed out on a lot of school, important moments with friends and simply being kids. Now, they can explore a world in which patients like themselves can overcome this disease and reclaim their health.

“I have all of this oxygen and energy that came out of nowhere,” Nasir says. “It’s really a new life. I feel reborn.”

“The network of doctors at Children’s National gave us reassurance and lots of hope,” says Kendric’s mom, Deborah. “They made us feel like family. We are in awe of how quickly things moved and how much compassion they have shown us.”

Read more stories like this one in the latest issue of Believe magazine.

collage of news logos

Children’s National in the News: 2024

collage of news logosIn 2024, Children’s National Hospital continued to make remarkable strides across diverse areas of pediatric medicine, from groundbreaking technological innovations to critical health advocacy. The following compilation showcases ten significant stories that demonstrate the breadth and depth of the hospital’s impact, as featured in major national news outlets including NBC Nightly News, CNN, The Washington Post, The New York Times, NPR, The Today Show, Healio, and POLITICO. Delve into our 2024 news highlights for more.

1. World’s smallest pacemaker gives new hope to babies with heart defects

Charles Berul, M.D., and a patient family talk about the pill-sized pacemaker that saved the life of Abby, an infant born with deadly heart defects. (NBC Nightly News)

2. ‘A $10 death trip’: Fentanyl is killing teens. Meet one fighting for his life

Sivabalaji Kaliamurthy, M.D., addiction psychiatrist and director of the Addictions Program, spoke to CNN about the impact of drug addiction on teen health and the lack of resources available to treat opioid use disorder. (CNN)

3. Health panel urges interventions for children and teens with high BMI

Susma Vaidya, M.D., M.P.H., associate medical director of the IDEAL Clinic, shared her concerns about childhood obesity treatment recommendations issued today by a leading panel of independent U.S. health experts. (The Washington Post)

4. An Rx for food? Doctor’s offices offer groceries to those in need

Shideh Majidi, M.D., M.S.C.S., and Emily Frymark, clinical dietitian, spoke about how the food pharmacy, created in partnership with the Capital Area Food Bank, benefits patients with diabetes and other chronic conditions. (The Washington Post)

5. First patient begins newly approved sickle cell gene therapy

Kendric Cromer, a 12-year-old boy being treated at Children’s National Hospital, became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that may cure the condition. “This is a big effort,” says David Jacobsohn, M.D., ScM, M.B.A. (The New York Times)

6. ‘We created this problem’: A pediatric surgeon on how gun violence affects children

Mikael Petrosyan, M.D., associate chief of General and Thoracic Surgery, discusses the stress medical staff face when treating young victims of gun violence. (NPR)

7. 7th grade boy rings bell after final round of chemotherapy

Landon, an 11-year-old patient, rang the bell at Children’s National Hospital with family, friends, doctors and nurses cheering after finishing his final round of chemotherapy. (The Today Show)

8. Study: One in three adolescents experience ‘period poverty’

Monika Goyal, M.D., M.S.C.E., pediatric emergency medicine specialist and co-director of the Center for Translational Research, emphasized the need for awareness in addressing period poverty in teenagers and young adults. (Healio)

9. The AI assurance labs are coming

Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., vice president and chief innovation officer, participates in a panel discussion covering AI data collection, associated risks, reliance and other topics related to artificial intelligence. (POLITICO)

10. First day of a ‘new life’ for a boy with sickle cell

Children’s National patient Kendric Cromer, 12, became one of the first children ever to be treated with a newly approved gene therapy that will free him from the sickle cell disease that has stolen his childhood. (The New York Times)

Dr. Andrew Campbell

Meet Dr. Andrew Campbell: Trailblazing treatment for patients with sickle cell disease

Andrew Campbell, M.D., remembers the first time he met a patient with sickle cell disease in the early years of his medical training. It was a brief interaction that had a profound effect on the trajectory of his career.

Sickle cell disease is an inherited blood disorder that primarily affects African American and Hispanic American children. The disease can cause severe pain events as well as progressive organ damage in patients.

This life-altering disease has affected children for more than a century, yet only one FDA-approved drug for treatment of sickle cell disease was developed in the first 100 years of its existence. Recognizing the health inequities that have contributed to an overall lack of therapies and providers in the field, Dr. Campbell knew he had to act.

“It was an area that needed a lot of resources, but also a lot of research and understanding of what our patients are going through. So, that was my initial launch into the field of Hematology and Oncology,” he explained, and he’s been an instrumental leader ever since.

For the past seven years, Dr. Campbell has served as the director of the Comprehensive Sickle Cell Disease Program at Children’s National Hospital — one of the largest sickle cell programs in the country, treating nearly 1,400 patients a year. Locally, he is Principal Investigator for the American Society of Hematology Sickle Cell Research Collaborative’s DMV Sickle Cell Consortium that includes several area sickle cell clinics including Northern Virginia, Richmond, Washington DC and Maryland.

In December 2023, the FDA approved two new gene therapies (CASGEVY™ and LYFGENIA™) to treat patients with sickle cell disease. Children’s National became one of the few pediatric hospitals in the country to offer these therapies, going on to then treat the first patient in the U.S. using the gene therapy method.

Dr. Campbell, who is treating the 12-year-old patient, says his team is excited about the future of these non-chemotherapy treatments and the curative possibilities they will bring to the field.

Despite being part of this significant milestone in the sickle cell community, Dr. Campbell says the work doesn’t stop there. His passion for making a difference and his impact on patients extends far beyond just Washington, D.C., and even the U.S.

For example, Dr. Campbell directs a research group called the Consortium for the Advancement of Sickle Cell Disease Research (CASiRe), with other sickle cell providers across the world. He says one of their goals is to better understand the ways that sickle cell can present itself in patients based on the country in which they receive treatment. “It’s really showing that based on the geographic difference of patients, it has implication in how they receive care,” he explains, adding that the group hopes to take what they learn in these studies to design more inclusive clinical trials in the future.

When he’s not devoting his time to research, you can find Dr. Campbell taking the fight for patients with sickle cell disease directly to lawmakers.

“I have gone to Capitol Hill a number of times to advocate for access and improvement of treatments for sickle cell disease,” he says, hoping that by using his voice and presenting his research, he can help the current generation, as well as the future generation of patients, get the care they deserve.

Assessing psychosocial risk, patient readiness for sickle cell gene therapy

The CureSCi Patient Readiness and Resilience Working Group brought together behavioral health clinicians and scientists from across the U.S. with expertise in sickle cell disease to develop recommendations for assessing and promoting patient readiness for gene therapy.

Two gene therapies for sickle cell disease were recently approved by the U.S. Food and Drug Administration (FDA) and are now commercially available in the U.S. This marks a historic shift in the treatment of sickle cell disease (SCD) and represents a leap forward more broadly for the medical community, opening a range of exciting possibilities for the development of novel therapeutics for other diseases. However, these new therapies are not without medical and psychological risks; therefore, the Cure Sickle Cell Initiative (CureSCi) of the National Heart, Lung and Blood Institute (NHLBI) convened a Patient Readiness and Resilience Working Group to develop recommendations for the assessment of psychosocial readiness for gene therapy.

What’s been the hold-up in the field?

Clinicians have long recognized that psychological and social issues have the potential to affect treatment outcomes following disease-modifying or transformative treatments, such as hematopoietic stem cell transplants. The same concerns exist for gene therapies, but there has not been clear guidance about the best ways to evaluate patient readiness and psychosocial risk and resilience factors in these contexts.

How does this work move the field forward?

The CureSCi Patient Readiness and Resilience Working Group brought together behavioral health clinicians and scientists from across the U.S. with expertise in SCD, as well as caregivers and patients with the lived experience of having SCD, to develop recommendations for assessing and promoting patient readiness for gene therapy. The resulting consensus statement outlines clear and practical guidance for conducting pre-gene therapy patient readiness assessments.

“This is an exciting time for the sickle cell and medical communities,” says Steven Hardy, Ph.D., director of Behavioral Health Services in the Divisions of Hematology, Oncology and Blood and Marrow Transplantation at Children’s National Hospital and lead author on the consensus statement. “But it is also a time to exercise caution to ensure that, in the cloud of such enthusiasm, we do not lose sight of the complex ways that human psychology, relationships and biology interact to influence health.”

How will this work benefit patients?

This new guidance for evaluating psychosocial readiness will ensure that important issues — such as the degree to which patients have been informed of and understand key treatment details, are interested in and motivated to pursue treatment, and have considered how undergoing gene therapy will affect their activities, relationships and mental health — are considered and patients are provided the necessary supports.

“These recommendations offer a blueprint and a charge to institutions, payors and policymakers around the world to prioritize the psychosocial well-being of patients with SCD undergoing gene therapy,” says Dr. Hardy.

How is Children’s National leading in this space?

Children’s National has participated in gene therapy clinical trials and is the first institution globally to treat a patient with SCD with one of the new commercially available gene therapies. Dr. Hardy chaired the CureSCi Patient Readiness and Resilience Working Group that developed the consensus recommendations. Psychologists in the Divisions of Hematology, Oncology and Blood and Marrow Transplantation have adopted a standard protocol, informed by the consensus recommendations, for conducting pre-gene therapy assessments of patient readiness.

You can read the full consensus statement, Assessing Psychosocial Risk and Resilience to Support Readiness for Gene Therapy in Sickle Cell Disease: A Consensus Statement, in JAMA Network Open.

researchers in a lab at Children's National

Pioneering gene therapy as a treatment for sickle cell disease

Gene therapy is a new and exciting treatment option available for patients with sickle cell disease (SCD). Children’s National Hospital is one of the few pediatric hospitals in the country that offers both FDA-approved sickle cell disease gene therapies: CASGEVY™ (exagamglogene autotemcel) and LYFGENIA™ (lovotibeglogene autotemcel).

What this means

Gene therapy involves an autologous transplant, taking the patient’s own stem cells, genetically changing those stem cells and then, after chemotherapy, infusing those stem cells back into the patient to make healthy blood.

“I’m excited about gene therapy for sickle cell disease. I think it has the potential to be a curative option for every single child with sickle cell disease,” said Robert Nickel, M.D., hematologist at Children’s National.

Currently both treatments are only approved for patients 12 years and older with severe disease. Children’s National was the first hospital in the world to collect stem cells for the LYFGENIA™ treatment.

Moving the field forward

Clinical trials hold incredible promise to advance the care of SCD. Children’s National continues to pioneer transplant therapies to cure SCD and is one of the leading centers participating in clinical trials of new treatments for this condition.

Experts at Children’s National are leading a multi-site clinical trial of a chemotherapy-free transplant approach for SCD using a matched sibling donor. This chemotherapy-free approach has less toxicity and side effects for children undergoing transplant.

In addition, Children’s National has been leading the way with innovative approaches to support sickle cell patients. “We’re providing alternative approaches to pain such as healing touch, acupuncture, massage VR technology, physical therapy and exploring other ways of treating pain in an integrated manner,” said Andrew Campbell, M.D., director of the Comprehensive Sickle Cell Disease Program at Children’s National.

The team is also exploring non-opioid treatments, such as intravenous citrulline, a naturally occurring amino acid that has been proven to enhance blood flow and potentially alleviate pain in treated patients in preliminary studies under the direction of Suvankar Majumdar, M.D., chief of Hematology at Children’s National.

illustration of sickled blood cells

Children’s National experts showcase sickle cell disease research

illustration of sickled blood cellsAndrew Campbell, M.D., director of the Comprehensive Sickle Cell Disease program, assessed the lifetime value of cell and gene therapy (CGTS) through a case study at The American Society of Gene and Cell Therapy’s (ASGCT) Annual Meeting.

Dr. Campbell and other Children’s National researchers will be presenting again at the Foundation for Sickle Cell Disease Research’s (FSCDR) Annual Sickle Cell Disease Research and Educational Symposium, June 7-9, 2024. The symposium includes more than 500 leading researchers, physicians, clinicians and social workers from all over the world.

Here’s a look at the presentations from Children’s National:

Day Time Presenter(s) Title
Sunday, June 9, 2024 8:00 AM – 9:15 AM Andrew Campbell, M.D. Update on Sickle Cell Legislation in the US

 
Sunday, June 9, 2024 3:00 PM – 3:15 PM Andrew Campbell, M.D., Deepika Darbari, M.D., and Regine Hyppolite, MSA Diagnostic Potential of Platelet-Neutrophil Ratio (PNR) for Stroke Risk in SCD Children

 
Sunday, June 9, 2024 3:15 PM – 3:30 PM

 

 Andrew Campbell, M.D. A Pilot Study to Increase Naloxone Education and Prescriptions in Sickle Cell Clinics

 
Sunday, June 9, 2024 4:00 PM – 4:15 PM Steven Hardy, Ph.D. Correlation Between VOC and Cognitive Function Using The NIH ToolBox in SCD
Sunday, June 9, 2024 4:15 PM – 4:30 PM

 

 

 Andrew Campbell, M.D., Deepika Darbari, M.D., and Regine Hyppolite, MSA Platelet to neutrophil ratio as a novel marker for monitoring SCD patients on hydroxyurea

 

 

2023 with a lightbulb

The best of 2023 from Innovation District

2023 with a lightbulbAdvanced MRI visualization techniques to follow blood flow in the hearts of cardiac patients. Gene therapy for pediatric patients with Duchenne muscular dystrophy. 3D-printed casts for treating clubfoot. These were among the most popular articles we published on Innovation District in 2023. Read on for our full list.

1. Advanced MRI hopes to improve outcomes for Fontan cardiac patients

Cardiac imaging specialists and cardiac surgeons at Children’s National Hospital are applying advanced magnetic resonance imaging visualization techniques to understand the intricacies of blood flow within the heart chambers of children with single ventricle heart defects like hypoplastic left heart syndrome. The data allows surgeons to make critical corrections to the atrioventricular valve before a child undergoes the single ventricle procedure known as the Fontan.
(3 min. read)

2. Children’s National gives first commercial dose of new FDA-approved gene therapy for Duchenne muscular dystrophy

Children’s National Hospital became the first pediatric hospital to administer a commercial dose of Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy for the treatment of pediatric patients with Duchenne muscular dystrophy (DMD). Elevidys is a one-time intravenous gene therapy that aims to delay or halt the progression of DMD by delivering a modified, functional version of dystrophin to muscle cells.
(2 min. read)

3. New model to treat Becker Muscular Dystrophy

Researchers at Children’s National Hospital developed a pre-clinical model to test drugs and therapies for Becker Muscular Dystrophy (BMD), a debilitating neuromuscular disease that is growing in numbers and lacks treatment options. The work provides scientists with a much-needed method to identify, develop and de-risk drugs for patients with BMD.
(2 min. read)

4. First infants in the U.S. with specially modified pacemakers show excellent early outcomes

In 2022, five newborns with life-threatening congenital heart disease affecting their heart rhythms were the first in the United States to receive a novel modified pacemaker generator to stabilize their heart rhythms within days of birth. Two of the five cases were cared for at Children’s National Hospital. In a follow-up article, the team at Children’s National shared that “early post-operative performance of this device has been excellent.”
(2 min. read)

5. AI: The “single greatest tool” for improving access to pediatric healthcare

Experts from the Food and Drug Administration, Pfizer, Oracle Health, NVIDIA, AWS Health and elsewhere came together to discuss how pediatric specialties can use AI to provide medical care to kids more efficiently, more quickly and more effectively at the inaugural symposium on AI in Pediatric Health and Rare Diseases, hosted by Children’s National Hospital and the Fralin Biomedical Research Institute at Virginia Tech.
(3 min. read)

6. AAP names Children’s National gun violence study one of the most influential articles ever published

The American Academy of Pediatrics (AAP) named a 2019 study led by clinician-researchers at Children’s National Hospital one of the 12 most influential Pediatric Emergency Medicine articles ever published in the journal Pediatrics. The findings showed that states with stricter gun laws and laws requiring universal background checks for gun purchases had lower firearm-related pediatric mortality rates but that more investigation was needed to better understand the impact of firearm legislation on pediatric mortality.
(2 min. read)

7. Why a colorectal transition program matters

Children’s National Hospital recently welcomed pediatric and adult colorectal surgeon Erin Teeple, M.D., to the Division of Colorectal and Pelvic Reconstruction. Dr. Teeple is the only person in the United States who is board-certified as both a pediatric surgeon and adult colorectal surgeon, uniquely positioning her to care for people with both acquired and congenital colorectal disease and help them transition from pediatric care to adult caregivers.
(3 min. read)

8. First-of-its-kind holistic program for managing pain in sickle cell disease

The sickle cell team at Children’s National Hospital received a grant from the Founders Auxiliary Board to launch a first-of-its-kind, personalized holistic transformative program for the management of pain in sickle cell disease. The clinic uses an inter-disciplinary approach of hematology, psychology, psychiatry, anesthesiology/pain medicine, acupuncture, mindfulness, relaxation and aromatherapy services.
(3 min read)

9. Recommendations for management of positive monosomy X on cell-free DNA screening

Non-invasive prenatal testing using cell-free DNA (cfDNA) is currently offered to all pregnant women regardless of the fetal risk. In a study published in the American Journal of Obstetrics and Gynecology, researchers from Children’s National Hospital provided context and expert recommendations for maternal and fetal evaluation and management when cfDNA screening is positive for monosomy X or Turner Syndrome.
(2 min. read)

10. Innovation in clubfoot management using 3D anatomical mapping

While clubfoot is relatively common and the treatment is highly successful, the weekly visits required for Ponseti casting can be a significant burden on families. Researchers at Children’s National Hospital are looking for a way to relieve that burden with a new study that could eliminate the weekly visits with a series of 3D-printed casts that families can switch out at home.
(1 min. read)

11. Gender Self-Report seeks to capture the gender spectrum for broad research applications

A new validated self-report tool provides researchers with a way to characterize the gender of research participants beyond their binary designated sex at birth. The multi-dimensional Gender Self-Report, developed using a community-driven approach and then scientifically validated, was outlined in a peer-reviewed article in the American Psychologist, a journal of the American Psychological Association.
(2 min. read)

12. Cardiovascular and bone diseases in chronic kidney disease

In a study published by Advances in Chronic Kidney Disease, a team at Children’s National Hospital reviewed cardiovascular and bone diseases in chronic kidney disease and end-stage kidney disease patients with a focus on pediatric issues and concerns.
(1 min. read)

Clumps of sickle cell blocking a blood vessel

Neurocognitive and emotional factors predict hospital visits in children with SCD

 

Clumps of sickle cell blocking a blood vessel

Neurocognitive and emotional factors are predictors of visits children with SCD make to the hospital.

Many children with sickle cell disease (SCD) experience significant pain that often results in trips to the emergency department (ED) or hospitalization. Disease characteristics explain some – but not all – of the variability in pain-related healthcare utilization. In a new study published in Pediatric Blood & Cancer, experts examined the role of neurocognitive functioning and emotional factors in predicting future healthcare visits for pain up to three years later.

The big picture

In the 3-year study of 112 youth with SCD between ages 7-16, researchers found that poorer attentional control and greater emotional distress predicted more ED visits and hospitalizations for pain years later.

Past studies that have pointed to the importance of neurocognitive and emotional factors in the experience of pain have only looked at relationships at a single point in time.

“Those types of studies limit our ability to evaluate the directionality of relationships and consider the implications of neurocognitive functioning and emotional well-being for children’s health as they get older,” said lead author Steven Hardy, Ph.D., director of Psychology and Patient Care Services and psychologist in the Center for Cancer and Blood Disorders (CCBD) at Children’s  National Hospital.

Why it matters

This study advances the understanding of the effects of neurocognitive functioning and emotions on the course of pain and disease morbidity in pediatric SCD.

Additionally, the findings will benefit patients by shedding light on potential intervention targets to support effective pain management.

“It also helps to reframe the conversation around pain and healthcare utilization in SCD by broadening our understanding of the factors that precipitate pain to include neurocognitive functioning and mental health,” Dr. Hardy added.

What’s unique

This work demonstrates that factors beyond disease characteristics are important to consider in a comprehensive approach to managing pain and improving quality of life for children with SCD.

“It shows that we also need to be thinking about how neurocognitive functioning and mental health affect one’s ability to manage their disease in terms of taking medications, avoiding pain triggers, coping with pain, communicating about symptoms and advocating for needs,” Dr. Hardy said. “Future work will look to see if we can preserve neurocognitive functioning and support mental health as a way to reduce pain impairment and improve quality of life.”

Children’s National is conducting several studies focused on better understanding the course of neurocognitive functioning and pain in pediatric SCD. Psychologists in the CCBD are also exploring novel interventions to preserve neurocognitive functioning and promote the development of effective strategies for coping with pain in SCD.

First-of-its-kind holistic program for managing pain in sickle cell disease

The new sickle cell clinic prioritizes looking at the whole person and considering multiple factors that promote health.

The sickle cell team at Children’s National Hospital received a grant from the Founders Auxiliary Board to launch a first-of-its-kind, personalized holistic transformative program for the management of pain in sickle cell disease (SCD). The clinic uses an inter-disciplinary approach of hematology, psychology, psychiatry, anesthesiology/pain medicine, acupuncture, mindfulness, relaxation and aromatherapy services.

Focusing on the “whole person health,” this clinic prioritizes looking at the whole person — not as individual organs or body systems — and considering multiple factors that promote health. Strategies taught in the clinic allow patients to manage their pain effectively by improving self-efficacy, coping mechanisms, and encouraging use of non-opioid and non-pharmacological modalities for pain management. Below, Deepika Darbari, M.D., hematologist and lead of the clinic, and Andrew Campbell, M.D., director of the Comprehensive Sickle Cell Disease Program, tell us more about this unique clinic.

Q: What’s been the hold-up in the field to implement a clinic like this?

A: There are many barriers at different levels in establishing a clinic like this. Most commonly it is the lack of provider expertise, which may not be available at many institutions. Furthermore, services may be available but may not be covered by health insurance. Sometimes, access to these services may be difficult because of the limited locations where they may be offered and not in conjunction with a patient’s hematology care – like in our clinic – which adds to the burden for patients and their families.

Q: How does this work move the field forward in the space of SCD?

A: This clinic is a unique concept where patients and their families actively contribute to and are at the center of the management plan. The goal of this clinic is to provide holistic care to our patients and families and positively impact all aspects of their wellbeing.

Instead of treating a specific disease, “whole person health” focuses on restoring health, promoting resilience and preventing diseases across the patient’s lifespan.

This clinic will continue to provide traditional treatment options for management of SCD along with non-opioids and nonpharmacologic therapies for management of pain, which is the most common complication of SCD.

We are not aware of any such multidisciplinary clinic for SCD like ours at Children’s National. Our team has been invited to national and international scientific conferences to share our experience and educate other programs about how to establish and sustain a clinic like ours.

Q: How will this clinic benefit patients?

A: In SCD, the symptom of pain can start as early as in the first 6 months of life and continue to occur through the lifespan of a patient, often turning into a chronic pain condition. This chronic pain is very refractory to traditional treatments including strong medications like opioids, which may not provide relief while contributing to many side effects. Our goal for patients attending this clinic is to improve their pain experience without increasing side effects.

We hope that the approach offered in this clinic will allow us to decrease the incidence and burden of chronic pain in individuals living with SCD. We would like to offer these treatment strategies early in life, which may help reduce the burden of chronic pain in our patients. We also hope that patients who have developed chronic pain can utilize these strategies to manage their pain, enhance function, reduce opioid use and improve health-related quality of life.

Q: What are you most excited about?

A: We are very excited to build upon our previous work in this space. Our pilot program was started by members of our multidisciplinary team who volunteered their time and effort for this important work.

While providing care to our families and patients, we are also looking forward to collecting robust data that can demonstrate the impact of such an approach in reducing burden of pain in SCD. This data will be helpful in supporting future research and expansion of this approach to benefit all individuals living with SCD.

 

boy using spirometer

Hydroxyurea initiation improves spirometry results in children with sickle cell anemia

boy using spirometer

Hydroxyurea therapy may help preserve lung function over time in children with sickle cell anemia.

Children’s National researchers participated in a recent study suggesting that the use of hydroxyurea (HU) therapy in children with sickle cell anemia (SCA) may help preserve lung function over time.

Sickle cell disease is associated with various pulmonary complications, including reactive airways, acute chest syndrome (ACS), pulmonary fibrosis and pulmonary arterial hypertension, which can lead to increased morbidity and mortality. Lung function tends to decline over time in individuals with SCA, and a more irreversible restrictive pathology develops with age and increasing respiratory complications.

Hydroxyurea is a disease-modifying therapy that has been shown to lower the rates of several complications of SCD, such as ACS and painful vaso-occlusive crises. It also might help lessen the severity of airway hyperreactivity in children.

This study provides evidence that hydroxyurea therapy may have a positive impact on pulmonary function in children with SCA, which could be of interest to doctors treating this patient population. By slowing the decline in lung function, hydroxyurea therapy may help reduce the risk of pulmonary complications and improve the overall health outcomes of children with SCA.

You can read the full study, Spirometric Changes After Initiation of Hydroxyurea in Children With Sickle Cell Anemia, in the Journal of Pediatric Hematology/Oncology.

Authors on the study from Children’s National Hospital include Dinesh Pillai, M.D., Deepika Darbari, M.D., and Anastassios Koumbourlis, M.D., M.P.H.

pregnant woman at fertility consultant

Fertility preservation in sickle cell disease patients

pregnant woman at fertility consultant

Fertility is a long-standing concern for patients with sickle cell disease and their families.

In a recent review in the Journal of Clinical Medicine, researchers from Children’s National Hospital look at the current state of fertility preservation in patients with sickle cell disease and make recommendations for longitudinal post-treatment for these individuals.

Fertility is a long-standing concern for patients with sickle cell disease and their families. Current curative therapy for the disease requires gonadotoxic conditioning, which many patients resist because of the resulting risk of infertility. And, while standard fertility preserving interventions exist for pre- and postpubescent females and males, best practices for integrating these interventions into sickle cell disease care have not yet been established.

In their article, Children’s National hematologist Robert Sheppard Nickel, M.D., and co-authors review current fertility assessments, fertility considerations in pre- and post-transplant patients with sickle cell disease and fertility preserving interventions for patients. The authors conclude that in the future, less toxic curative approaches may make fertility preservation unnecessary, but at present, fertility preservation should be offered to patients with sickle cell disease pursing curative therapy.

Additional authors from Children’s National include Michael Hsieh, M.D., Ph.D., and Jacqueline Maher, M.D.

Read the full review article, Fertility after Curative Therapy for Sickle Cell Disease: a Comprehensive Review to Guide Care, in the Journal of Clinical Medicine.

father and son in playground

Using psychological treatments to help pediatric pain management

father and son in playground

Behavioral pain management can utilize approaches to address the experience of co-occurring acute and chronic pain.

There are different types of pain that people experience, such as short-term (acute) pain and longer term (chronic or persistent) pain. Just like there are different pharmacologic treatments depending on the type of pain, there are different psychological or behavioral therapies that seem more effective when tailored to a person’s type of pain. Furthermore, certain psychological treatments that are known to be helpful for managing acute pain may actually be unhelpful for a child dealing with chronic pain. A framework has not previously existed for the provision of psychological treatment when children are experiencing both acute and chronic pain.

In a new article, Megan Connolly, Ph.D., pediatric psychologist, and Steven Hardy, Ph.D., pediatric psychologist and director of Psychology and Patient Care Services for the Center for Cancer and Blood Disorders, both at Children’s National Hospital, outline recommendations for the psychological treatment of acute and chronic pain in children.

The hold-up in the field

Only recently have psychological therapies gained empirical support for their efficacy in treating pain conditions. Very little research, however, has been done on how to effectively tailor psychological treatments for groups experiencing both acute and chronic pain, likely because the pain conditions that involve both acute and chronic pain are rare.

In their new article, Dr. Connolly and Dr. Hardy draw attention to this issue by pulling together empirical evidence and clinical expertise to outline a framework for the psychological and behavioral management of co-occurring acute and chronic pain in children.

“This framework will help to guide the development of novel clinical interventions for individuals who experience co-occurring acute and chronic pain,” Dr. Connolly said.

Why we’re excited

The authors reviewed how behavioral pain management strategies can utilize complementary approaches to address the experience of co-occurring acute and chronic pain.

“It’s challenging that there are not many psychological treatments that were developed with the populations we see in mind,” Dr. Connolly added. “And it’s exciting there is now a more coherent framework for treating patients that have acute and chronic pain.”

Children’s National Hospital leads the way

Many clinicians see the unique challenges of treating co-occurring acute and chronic pain. However, this is the first article to propose a clinical framework for the psychological treatment of patients experiencing both types of pain.

The authors said they hope that their article helps to enhance the current dialogue around pain perception and pain burden to address the needs of co-occurring acute and chronic pain presentations.

You can read the full study, Clinical Considerations for Behavioral Pain Management in Co-occurring Acute and Chronic Pain Presentations, in the journal Clinical Practice in Pediatric Psychology.

Sickle-Cell-Blood-Cells

Children’s National joins ASH RC Sickle Cell Disease Clinical Trials Network

Sickle-Cell-Blood-Cells

The American Society of Hematology Research Collaborative (ASH RC) has announced the first 10 clinical research consortia to join the ASH RC Sickle Cell Disease Clinical Trials Network. Children’s National Hospital will be one of the clinical trials units to serve in the DMV Sickle Cell Disease Consortium (DMVSCDC).

The sites will be able to enroll children and adults living with sickle cell disease (SCD) within their patient populations in clinical trials as part of an unprecedented national effort to streamline operations and facilitate data sharing to expedite the development of new treatments for this disease.

“As part of the ASH RC SCD clinical trials network, we will learn regionally and nationally how sickle cell patients respond differently to therapies, hopefully giving us clues to provide more successful targeted and individualized treatments that will improve the morbidity and mortality in sickle cell disease patients,” said Andrew Campbell, M.D., director of Comprehensive Sickle Cell Disease Program at Children’s National.

SCD is a chronic, progressive, life-threatening, inherited blood disorder that affects more than 100,000 Americans and an estimated 100 million persons worldwide. Clinical trials hold incredible promise for the development of much-needed new treatments, and possibly even a cure. While there are currently only four U.S. Food and Drug Administration (FDA)-approved drugs to treat the disease, there is a robust SCD drug development pipeline that will drive demand for clinical trials to a new level, providing a prime opportunity to advance treatment and care of those affected by SCD.

“We are proud that the DMV Sickle Cell Disease Consortium will contribute regionally, allowing our patients and families to benefit from new clinical trials investigating new therapies that may improve the clinical course and quality of life of patients living with sickle cell disease in the DMV region,” Dr. Campbell added. “We will also have an integrated Community Advisory Board who will continue to provide guidance and expertise for our consortium including patients, families and caregivers.”

Read the full list of other hospitals joining the network.

Deepika Darbari

Deepika Darbari, M.D., receives the 2021 ASH Award for leadership in promoting diversity

Deepika Darbari

Deepika Darbari, M.D., hematologist at Children’s National Hospital, is being honored by the American Society of Hematology (ASH) for her significant contributions to the mentorship and training of underrepresented minority researchers and for advancing the care for underrepresented patient populations, primarily individuals living with sickle cell disease (SCD). Dr. Darbari started studying and treating SCD at Howard University, where she also saw firsthand the many disparity issues surrounding the condition, such as inadequate funding, limited treatment options and biases and stigma. She also learned about barriers to career development that minority students faced. She worked to address those issues through her mentorship.

Dr. Darbari has mentored many medical students, residents and fellows whose research projects focused on improving care for individuals living with SCD. She has also fostered the careers of junior investigators of underrepresented minorities as well as served as a member of the ASH Minority Medical Student Award Program, the ASH Committee on Promoting Diversity and the ASH Women in Hematology working group, all in her continued efforts to increase diversity, equity and inclusion at ASH and in the health care community at large.

Suvankar Majumdar

Spotlight on Suvankar Majumdar, M.D.

Suvankar Majumdar

As a provider with international experience, Suvankar Majumdar, M.D., joined Children’s National in August 2017 as chief of Children’s Division of Hematology within the Center for Cancer and Blood Disorders. Dr. Majumdar is excited to be at Children’s National because of the opportunities for growth, cutting-edge research and continuing education that our diverse population of patients can provide clinicians.

Born in Zambia, in southern Africa, and educated in the United Kingdom, Dr. Majumdar moved to Zimbabwe to study medicine, which he considers the turning point of his career. While in medical school, Dr. Majumdar oversaw and managed the treatment of patients with HIV and other chronic illnesses and determined that blood disorders, particularly sickle cell, was where he wanted to place his focus. Since then, he has served as the Director of the Comprehensive Pediatric Sickle Cell Program as well as Director of the Hemophilia Treatment Center at the University of Mississippi and is a recognized leader in hematology and sickle cell disease. It is this expertise, as well as his dedication to research studies, that have already made him an asset to Children’s National.

Within the Division of Hematology, Children’s providers focus on treating patients with blood disorders, bleeding and clotting disorders, red blood cell disorders (such as sickle cell) and more. Since coming to Children’s National, Dr. Majumdar has experienced a tremendous amount of dedication and enthusiasm from his colleagues. “I’m excited to build on what our faculty has accomplished so far. We’re already well poised to become a national leader in hematology,” he says. “I have no doubt that we will continue to accomplish our goals through collaboration and working toward a common life-saving cause.”

One of his immediate goals for the division is to focus on bringing improved patient care and accessibility in the surrounding Washington area. Additionally, Dr. Majumdar is currently conducting two research studies for sickle cell disease. As one of his studies enters the second phase, he’s focused on seeing the impact of an intravenous citrulline, a nitric oxide booster, on patients with sickle cell disease. Another study has begun to determine if specific genetic mutations that cause prolonged QT, or irregular heartbeats in patients, cause mortality, as sickle cell patients are predisposed to cardiac episodes.

It is Dr. Majumdar’s hope that the hematology team at Children’s National will also continue training the next generation of providers to advance research, education and clinical aspects of the field. To those looking to join the specialty, Dr. Majumdar suggests keeping an open mind when it comes to collaborating with colleagues. “My dad always said to my siblings and I that ‘to break one stick is easy, but to break three sticks is harder’ and really impressed upon us that we’re stronger together,” he says. “By working together, we’re more likely to produce the results that we’re looking for.”

Being located in the nation’s capital, providers at Children’s National are accustomed to seeing a diverse array of patients. For Dr. Majumdar, this presents a unique opportunity. “Meeting and interacting with different patients and families was really appealing when I decided to come to Children’s National. The variety of cases we see in the Division of Hematology can definitely present new challenges, but it’s also more rewarding,” he says.

Working with the pediatric population is also a passion of his. “Children are resilient and tend to bounce back quickly,” Dr. Majumdar says. “As a parent, I try to empathize with treatment concerns and always treat every child as if they were my own. I’m always going to make sure it’s the best level of care possible.”

Billie Lou Short and Kurt Newman at Research and Education Week

Research and Education Week honors innovative science

Billie Lou Short and Kurt Newman at Research and Education Week

Billie Lou Short, M.D., received the Ninth Annual Mentorship Award in Clinical Science.

People joke that Billie Lou Short, M.D., chief of Children’s Division of Neonatology, invented extracorporeal membrane oxygenation, known as ECMO for short. While Dr. Short did not invent ECMO, under her leadership Children’s National was the first pediatric hospital to use it. And over decades Children’s staff have perfected its use to save the lives of tiny, vulnerable newborns by temporarily taking over for their struggling hearts and lungs. For two consecutive years, Children’s neonatal intensive care unit has been named the nation’s No. 1 for newborns by U.S. News & World Report. “Despite all of these accomplishments, Dr. Short’s best legacy is what she has done as a mentor to countless trainees, nurses and faculty she’s touched during their careers. She touches every type of clinical staff member who has come through our neonatal intensive care unit,” says An Massaro, M.D., director of residency research.

For these achievements, Dr. Short received the Ninth Annual Mentorship Award in Clinical Science.

Anna Penn, M.D., Ph.D., has provided new insights into the central role that the placental hormone allopregnanolone plays in orderly fetal brain development, and her research team has created novel experimental models that mimic some of the brain injuries often seen in very preterm babies – an essential step that informs future neuroprotective strategies. Dr. Penn, a clinical neonatologist and developmental neuroscientist, “has been a primary adviser for 40 mentees throughout their careers and embodies Children’s core values of Compassion, Commitment and Connection,” says Claire-Marie Vacher, Ph.D.

For these achievements, Dr. Penn was selected to receive the Ninth Annual Mentorship Award in Basic and Translational Science.

The mentorship awards for Drs. Short and Penn were among dozens of honors given in conjunction with “Frontiers in Innovation,” the Ninth Annual Research and Education Week (REW) at Children’s National. In addition to seven keynote lectures, more than 350 posters were submitted from researchers – from high-school students to full-time faculty – about basic and translational science, clinical research, community-based research, education, training and quality improvement; five poster presenters were showcased via Facebook Live events hosted by Children’s Hospital Foundation.

Two faculty members won twice: Vicki Freedenberg, Ph.D., APRN, for research about mindfulness-based stress reduction and Adeline (Wei Li) Koay, MBBS, MSc, for research related to HIV. So many women at every stage of their research careers took to the stage to accept honors that Naomi L.C. Luban, M.D., Vice Chair of Academic Affairs, quipped that “this day is power to women.”

Here are the 2019 REW award winners:

2019 Elda Y. Arce Teaching Scholars Award
Barbara Jantausch, M.D.
Lowell Frank, M.D.

Suzanne Feetham, Ph.D., FAA, Nursing Research Support Award
Vicki Freedenberg, Ph.D., APRN, for “Psychosocial and biological effects of mindfulness-based stress reduction intervention in adolescents with CHD/CIEDs: a randomized control trial”
Renee’ Roberts Turner for “Peak and nadir experiences of mid-level nurse leaders”

2019-2020 Global Health Initiative Exploration in Global Health Awards
Nathalie Quion, M.D., for “Latino youth and families need assessment,” conducted in Washington
Sonia Voleti for “Handheld ultrasound machine task shifting,” conducted in Micronesia
Tania Ahluwalia, M.D., for “Simulation curriculum for emergency medicine,” conducted in India
Yvonne Yui for “Designated resuscitation teams in NICUs,” conducted in Ghana
Xiaoyan Song, Ph.D., MBBS, MSc, “Prevention of hospital-onset infections in PICUs,” conducted in China

Ninth Annual Research and Education Week Poster Session Awards

Basic and Translational Science
Faculty: Adeline (Wei Li) Koay, MBBS, MSc, for “Differences in the gut microbiome of HIV-infected versus HIV-exposed, uninfected infants”
Faculty: Hayk Barseghyan, Ph.D., for “Composite de novo Armenian human genome assembly and haplotyping via optical mapping and ultra-long read sequencing”
Staff: Damon K. McCullough, BS, for “Brain slicer: 3D-printed tissue processing tool for pediatric neuroscience research”
Staff: Antonio R. Porras, Ph.D., for “Integrated deep-learning method for genetic syndrome screening using facial photographs”
Post docs/fellows/residents: Lung Lau, M.D., for “A novel, sprayable and bio-absorbable sealant for wound dressings”
Post docs/fellows/residents: Kelsey F. Sugrue, Ph.D., for “HECTD1 is required for growth of the myocardium secondary to placental insufficiency”
Graduate students: Erin R. Bonner, BA, for “Comprehensive mutation profiling of pediatric diffuse midline gliomas using liquid biopsy”
High school/undergraduate students: Ali Sarhan for “Parental somato-gonadal mosaic genetic variants are a source of recurrent risk for de novo disorders and parental health concerns: a systematic review of the literature and meta-analysis”

Clinical Research
Faculty: Amy Hont, M.D., for “Ex vivo expanded multi-tumor antigen specific T-cells for the treatment of solid tumors”
Faculty: Lauren McLaughlin, M.D., for “EBV/LMP-specific T-cells maintain remissions of T- and B-cell EBV lymphomas after allogeneic bone marrow transplantation”

Staff: Iman A. Abdikarim, BA, for “Timing of allergenic food introduction among African American and Caucasian children with food allergy in the FORWARD study”
Staff: Gelina M. Sani, BS, for “Quantifying hematopoietic stem cells towards in utero gene therapy for treatment of sickle cell disease in fetal cord blood”
Post docs/fellows/residents: Amy H. Jones, M.D., for “To trach or not trach: exploration of parental conflict, regret and impacts on quality of life in tracheostomy decision-making”
Graduate students: Alyssa Dewyer, BS, for “Telemedicine support of cardiac care in Northern Uganda: leveraging hand-held echocardiography and task-shifting”
Graduate students: Natalie Pudalov, BA, “Cortical thickness asymmetries in MRI-abnormal pediatric epilepsy patients: a potential metric for surgery outcome”
High school/undergraduate students: Kia Yoshinaga for “Time to rhythm detection during pediatric cardiac arrest in a pediatric emergency department”

Community-Based Research
Faculty: Adeline (Wei Li) Koay, MBBS, MSc, for “Recent trends in the prevention of mother-to-child transmission (PMTCT) of HIV in the Washington, D.C., metropolitan area”
Staff: Gia M. Badolato, MPH, for “STI screening in an urban ED based on chief complaint”
Post docs/fellows/residents: Christina P. Ho, M.D., for “Pediatric urinary tract infection resistance patterns in the Washington, D.C., metropolitan area”
Graduate students: Noushine Sadeghi, BS, “Racial/ethnic disparities in receipt of sexual health services among adolescent females”

Education, Training and Program Development
Faculty: Cara Lichtenstein, M.D., MPH, for “Using a community bus trip to increase knowledge of health disparities”
Staff: Iana Y. Clarence, MPH, for “TEACHing residents to address child poverty: an innovative multimodal curriculum”
Post docs/fellows/residents: Johanna Kaufman, M.D., for “Inpatient consultation in pediatrics: a learning tool to improve communication”
High school/undergraduate students: Brett E. Pearson for “Analysis of unanticipated problems in CNMC human subjects research studies and implications for process improvement”

Quality and Performance Improvement
Faculty: Vicki Freedenberg, Ph.D., APRN, for “Implementing a mindfulness-based stress reduction curriculum in a congenital heart disease program”
Staff: Caleb Griffith, MPH, for “Assessing the sustainability of point-of-care HIV screening of adolescents in pediatric emergency departments”
Post docs/fellows/residents: Rebecca S. Zee, M.D., Ph.D., for “Implementation of the Accelerated Care of Torsion (ACT) pathway: a quality improvement initiative for testicular torsion”
Graduate students: Alysia Wiener, BS, for “Latency period in image-guided needle bone biopsy in children: a single center experience”

View images from the REW2019 award ceremony.

Dr. Andrew Campbell examines a child

Children’s National physicians provide education at 46th Annual Sickle Cell Disease Association of America Convention

Dr. Andrew Campbell examines a child

Andrew Campbell, M.D., presented at the conference on the topics of hydroxyurea (HU) and blood transfusions.

More than 600 researchers, physicians, nurses, social workers and individuals living with sickle cell disease (SCD) and sickle cell trait (SCT) gathered in Baltimore for the 46th Annual National Sickle Cell Disease Association of America (SCDAA) Convention in mid-October. Children’s National physicians Andrew Campbell, M.D., director of the Comprehensive Sickle Cell Program, and Deepika Darbari, M.D., were among the speakers at the four-day convention discussing the latest scientific research and clinical information through seminars, panel discussions and symposiums.

Dr. Campbell presented at the conference on the topics of hydroxyurea (HU) and blood transfusions. He spoke to families about the benefits of HU, explaining how it lowers the percentage of sickle cells in the blood and decreases the overall inflammatory process. He stressed the importance of HU as a medication used in the prevention of SCD and emphasized the potential decrease in organ damage and increased overall survival rate of SCD patients. The importance of minor antigen blood group phenotyping was also discussed, as it can decrease the chance of patients rejecting future blood transfusions by developing new red blood cell antibodies.

“The indications for blood transfusions in the acute ‘ill’ setting can be life-saving and improve oxygen delivery and overall clinical outcomes within sickle cell complications, including acute chest syndrome, stroke and splenic sequestration. Approximately 10 to 15 percent of patients will need monthly blood transfusions for primary (i.e. stroke, patients with abnormal brain vessel TCD velocities >200cm/s) and secondary (i.e. patients with a previous stroke, multiple splenic sequestrations, recurrent priapism, recurrent acute chest syndromes) sickle cell complications,” explains Dr. Campbell.

Dr. Darbari, a hematologist at Children’s National, educated medical colleagues on chronic pain in SCD and emphasized the increase in pain from adolescence to adulthood.

“During childhood, pain in SCD is considered a consequence of discrete episodes of vaso-occlusion.  Such vaso-occlusion is a complex process in which abnormally shaped (so-called ‘sickled’) red blood cells episodically obstruct the microcirculation thereby causing distal ischemia and resultant pain. As patients get older, mechanisms such as peripheral neuropathic or centralization may play important roles in transition and maintenance of chronic pain. It is important to consider underlying mechanisms contributing to pain when managing a patient with SCD,” states Dr. Darbari. She referenced her coauthored and published Analgesic, Anesthetic and Addiction Clinical Trial Translations Innovations Opportunities and Networks (ACTTION)-American Pain Society Taxonomy (AAPT) criteria for classifying chronic pain in SCD and how useful this tool can be for physicians in the treatment of patients with SCD.

Both Drs. Campbell and Darbari shared their expertise on different facets of SCD with families and medical professionals alike. Their impactful work is paving the way for future treatments and pain management techniques for treating patients living with SCD and their families.

ASCAT Conference Attendees

Children’s National represented at ASCAT conference in London

ASCAT Conference Attendees

From left to right: Lisa Thaniel, Ph.D., Brittany Moffitt, Deepika Darbara, M.D., Steven Hardy, Ph.D., Andrew Campbell, M.D., Barbara Speller-Brown, DNP, Stefanie Margulies and Karen Smith-Wong all represented Children’s National at the ASCAT Conference in London.

Deepika Darbari, M.D., Andrew Campbell, M.D., and Steven Hardy, Ph.D., represented Children’s National at the Annual Sickle Cell Disease and Thalassemia (ASCAT) Conference in London in late October. The theme of this year’s conference was Sickle Cell Disease and Thalassemia: Bridging the Gap in Care and Research.

Dr. Darbari, a Children’s National hematologist, was the featured Grand Rounds speaker and led a pain management symposium. Dr. Darbari studies complications of sickle cell disease with an emphasis on pain. She conducts clinical and translational studies to better understand sickle cell pain and its management. She addressed the topics of pain mechanisms and phenotypes in sickle cell disease during her symposium.

Dr. Campbell, Director of the Comprehensive Sickle Cell Disease Program at Children’s National, has served on the steering committee for this annual international conference for the past two years, working alongside colleagues from across the globe to bring together multiple experts who work with children with blood disorders. Dr. Campbell remarks, “I’m pleased to promote and be a part of [this conference] because it’s one of the best sickle cell/thalassemia conferences in the world pushing the field forward with international representation.” He spoke at the conference during Dr. Darbari’s symposium, discussing sickle cell disease pain around the globe.

Dr. Hardy, a pediatric psychologist in the divisions of Blood and Marrow Transplant, Blood Disorders (Hematology) and Oncology and the Center for Cancer and Blood Disorders at Children’s National, also presented at the conference on his abstract “Computerized Working Memory Training Improves Cognition in Youth with Sickle Cell Disease.” His abstract received the Best Oral Abstract Award at the conference and was awarded a 500 pound prize. In his work at Children’s National, Dr. Hardy provides evidence-based psychological assessments and treatments for children with cancer, sickle cell disease and other blood disorders, as well as those patients undergoing bone marrow transplants.

Poster presentations were also given by Barbara Speller Brown, NP, DNP, Lisa Thaniel, MSW, Ph.D., Brittany Moffitt, MSW, and Stefanie Margulies, senior clinical research coordinator, all representing Children’s National at the ASCAT Conference.

Maureen E Lyon

Maureen E. Lyon, Ph.D., ABPP, lauded for outstanding excellence in patient-centered advance care planning

Maureen E Lyon

Maureen E. Lyon, Ph.D., a principal investigator at Children’s Center for Translational Science, will be honored with a “Recognition Award for Excellence and Innovation in Research” by Respecting Choices for outstanding excellence in patient-centered advance care planning and shared decision-making.

Respecting Choices will present the award on Oct. 26, 2018, during its “National Share the Experience Conference” in Bloomington, Minnesota.

Lyon’s expertise is in advance care planning and shared decision-making for children and adolescents with life-threatening illnesses and their families, a field that has transformed in recent decades in order to pave better paths forward for difficult but necessary conversations.

“It came from my clinical experience,” Lyon says. “In the early days of the human immunodeficiency virus (HIV) epidemic in the U.S., everything, absolutely everything, was done to keep the kids alive in the hopes that some new drug would come around the corner, and we could bring them back from the brink. I remember one of the young boys saying to his case manager that he didn’t want all of these interventions. But he hadn’t told his family.”

That young man’s eye-opening comments – and learning that Children’s National Health System had a policy that teenagers were to be included in conversations about their own advance care planning – inspired Lyon to conduct a series of surveys involving adolescents, families and clinicians.

“I remember sitting down with friends and saying ‘There must be a better way to do this. Everyone is afraid to broach the subject,’ ” Lyon recalls. So, she conducted surveys of all healthy kids coming through Children’s adolescent clinic and kids diagnosed with HIV, cancer and sickle cell disease.

“It turned out the kids did want to talk about it. That was the first thing. Families told us they wanted help breaking the ice. Physicians felt it wasn’t their role – many doctors felt their role was to save people – or, they didn’t have the training,” she says.

Through a series of focus groups with youths living with HIV, families and community members, Lyon adapted the adult-centric Respecting Choices model to create a three-session intervention to better meet the advance care planning needs of youths and adolescents living with HIV.

Lyon’s recent work includes a single-blinded, randomized study published Oct. 19, 2018, in Pediatrics that finds the more families understand the end-of-life treatment preferences expressed by adolescents living with HIV, the less likely these youth are to suffer HIV-related symptoms, compared with youths whose families do not understand their end-of-life care goals.

She also has adapted the Respecting Choices intervention to facilitate its use with children diagnosed with cancer. More recently, she has adapted the model for use by parents of children with rare diseases who cannot communicate on their own.

“For the other life-threatening health conditions, we worked to support adolescents in expressing their advance care planning choices in their own voices. With rare diseases, we’re shifting gears,” she adds.

Published research indicates a sizable proportion of pediatric patients who die in hospitals now have confirmed or suspected rare diseases, she says. During a pilot involving seven families, many parents multitasked during the conversations, taking pauses to attend to various alarms as they sounded, to complete regular feedings and to contend with their child’s petit mal seizures.

“The level of burden of taking care of these children with terminal illnesses was pretty overwhelming,” she says. “Still, families were not too burdened to participate in advance care planning, but first wanted to identify their priority palliative care needs and to develop a support plan to meet those needs. We also had more fathers involved.”