Tag Archive for: SCD

Sickle Cell Anemia 3D Illustration

New telemedicine-based behavioral intervention program eases pain of patients with SCD

Sickle Cell Anemia 3D Illustration

Telemedicine-based behavioral interventions can reduce pain-related functional impairment in youth with SCD.

Sickle cell disease (SCD) pain is often associated with functional impairment and treatment is often limited to pharmacological approaches with unwanted side effects. Behavioral interventions are common for non-SCD pain populations, but interventions designed to address pain-related impairment in SCD are lacking.

In a recent study published in Pediatric Blood & Cancer, researchers conducted a pilot of a 4-week behavioral pain intervention for youth with SCD delivered via telemedicine known as the Balance Program.

Using an innovative combination of cognitive-behavioral therapy and acceptance-based approaches, researchers found that the intervention was feasible, evidenced by youth and caregiver ratings of high acceptability and satisfaction and excellent treatment completion rates. In addition, youth and their caregivers both reported significant reductions in the degree to which SCD pain interfered with daily activities after the treatment.

What’s been the hold-up in the field?

Researchers and clinicians know that there is a strong psychological component to all experiences of pain and there has been growing evidence in recent decades regarding the effectiveness of behavioral therapies for reducing pain and improving functioning.

“However, sickle cell disease presents unique challenges because unlike many pain presentations, it is common for patients with sickle cell disease to experience both acute and chronic pain, making treatment recommendations less clear,” said Megan Connolly, Ph.D., psychologist at Children’s National Hospital and the study’s lead author. “Previous studies have rarely focused on reducing pain-related disability, which is important for optimizing quality of life.”

How does this work move the field forward?

This study demonstrated the feasibility and acceptability of a telemedicine-based behavioral intervention to reduce pain-related functional impairment in youth with SCD. Nearly all youth and their caregivers rated the intervention as moderately or highly acceptable and 90% of patients completed the full treatment program.

“Moreover, the Balance Program resulted in significant reductions in the extent to which sickle cell disease pain interfered with daily activities,” Dr. Connolly added.

What about the findings is exciting?

This research explains what experts can be doing to reduce the impact of pain on the lives of children and adolescents with SCD. Through their findings, researchers learned that a telemedicine-based behavioral pain intervention, which is often more convenient for families than traveling to the hospital for weekly visits, can meaningfully reduce the impact of pain on daily living.

“It is one thing to develop a program that you think will be helpful, but it’s another thing to develop a program that families will be interested in and doesn’t add unnecessary stress to their lives,” Dr. Connolly said. “Although this study had a relatively small sample, I was also excited to see the magnitude of improvements in pain-related impairment, which was quite large. We plan to continue refining this treatment based on patient and caregiver feedback and looking for ways to increase accessibility to these types of treatments for sickle cell disease pain.”

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.

stem cells

How our BMT program is excelling: Q&A with David Jacobsohn, M.D.

David Jacobsohn

Dr. Jacobsohn has led the BMT program at Children’s National as the division chief and talks about their incredible success over the last 5 years.

Over the last five years, the bone marrow transplant (BMT) program at Children’s National Hospital has continuously improved. From decreasing transplant-related mortality to 0%, to increasing the complexity of their transplants, the program continues to succeed in providing the best care to patients and their families.

David Jacobsohn, M.D., Blood and Marrow Transplantation division chief, offers insight on the goals the program has reached, the obstacles it has overcome and the vision for what’s next.

Q: How would you describe the success of the BMT program over the last 5 years?

A: We have progressively seen outcomes improve, marked by improvement in one-year overall survival of allogeneic transplants. Contributing to that is our outstanding day 100 transplant-related mortality (TRM). For the first time ever, the day 100 transplant-related mortality, averaged over allogeneic transplants done in the last 3 years, was 0%. That means that during that time, we have not lost a patient due to transplant complications in the first 100 days. This is a remarkable achievement in the world of transplantation.

Q: How does this work move the field forward?

A: We are particularly interested in continuing BMT in non-malignant conditions, such as beta-thalassemia, immunodeficiency and sickle cell anemia. We have one of the largest programs in the country for transplantation of patients with sickle cell anemia. We have been able to offer BMT to patients with sickle cell disease (SCD) and no prior complications, as a preventative procedure. Whereas in the past, it was mostly reserved for patients that had already been severely affected.

Q: How will this work benefit patients?

A: One of the key benefits that we’re seeing is that complications such as graft-versus-host disease (GVHD) have really decreased over the last few years based on the type of medications we’re using and procedures we’re doing.  Now most of our patients that are about six months out from transplant are off immunosuppression and are living relatively normal lives.

Q: What excites you most about this advancement?

A: We’re very excited about something called the Alpha/beta T cell depletion (A/B TCD) . We’re one of the few hospitals in the country offering this process.

This means we’re able to collect the donor stem cells and remove the T cells in the lab. Particularly the A/B T cells, which cause GVHD. We’re able to do this successfully not needing any medications to suppress the immune system. This is really quite novel. A lot of those medications have different side effects on organs, especially the kidneys. Now we can do transplants, even from half-matched donors, without immunosuppression.

We want to expand to more and more patients in the next three to five years so that no patients will need immunosuppression.

Q: What do you look forward to in the next couple of years?

A: In the next few years, we’re excited to venture more into cellular and gene therapy. With regards to cellular therapy, we’re offering something called CAR T cells to patients with acute leukemia. And it’s possible that this will actually replace transplant in some very high-risk leukemia patients.

We’re also looking forward to offering gene therapy to patient with SCD and beta-thalassemia.

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.