Tag Archive for: sickle cell

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Children’s National in the News: 2023

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Explore some of the notable medical advancements and stories of bravery that defined 2023, showcasing the steadfast commitment of healthcare professionals at Children’s National Hospital and the resilient spirit of the children they support. Delve into our 2023 news highlights for more.

1. COVID during pregnancy dramatically increases the risk of complications and maternal death, large new study finds

According to a study published in British Medical Journal Global Health, women who get COVID during pregnancy are nearly eight times more likely to die and face a significantly elevated risk of ICU admission and pneumonia. Sarah Mulkey, M.D., prenatal-neonatologist neurologist, discussed findings based on her work with pregnant women and their babies.
(Fortune)

2. Rest isn’t necessarily best for concussion recovery in children, study says

A study led by Christopher Vaughan, Psy.D., pediatric neuropsychologist, suggests that — despite what many people may presume — getting kids back to school quickly is the best way to boost their chance for a rapid recovery after a concussion.
(CNN)

3. Pediatric hospital beds are in high demand for ailing children. Here’s why

David Wessel, M.D., executive vice president, chief medical officer and physician-in-chief, explained that one reason parents were still having trouble getting their children beds in a pediatric hospital or a pediatric unit after the fall 2022 respiratory surge is that pediatric hospitals are paid less by insurance.
(CNN)

4. Anisha Abraham details impact of social media use on children: ‘True mental health crisis’

Anisha Abraham, M.D., M.P.H., chief of the Division of Adolescent and Young Adult Medicine, joined America’s Newsroom to discuss the impact social media access has had on children’s mental health.
(FOX News)

5. Saving Antonio: Can a renowned hospital keep a boy from being shot again?

After 13-year-old Antonio was nearly killed outside his mom’s apartment, Children’s National Hospital went beyond treating his bullet wounds. Read how our Youth Violence Intervention Program team supported him and his family during his recovery.
(The Washington Post)

6. Formerly conjoined twins reunite with doctors who separated them

Erin and Jade Buckles underwent a successful separation at Children’s National Hospital. Nearly 20 years later they returned to meet with some of the medical staff who helped make it happen.
(Good Morning America)

7. Asthma mortality rates differ by location, race/ethnicity, age

Shilpa Patel, M.D., M.P.H., medical director of the Children’s National IMPACT DC Asthma Clinic, weighed in on a letter published in Annals of Allergy, Asthma & Immunology, asserting that the disparities in mortality due to asthma in the United States vary based on whether they occurred in a hospital, ethnicity or race and age of the patient.
(Healio)

8. How one Afghan family made the perilous journey across the U.S.-Mexico border

After one family embarked on a perilous journey from Afghanistan through Mexico to the U.S.-Mexico border, they eventually secured entry to the U.S. where Karen Smith, M.D., medical director of Global Services, aided the family’s transition and provided their daughter with necessary immediate medical treatment.
(NPR)

9. When a child is shot, doctors must heal more than just bullet holes

With the number of young people shot by guns on the rise in the U.S., providers and staff at Children’s National Hospital are trying to break the cycle of violence. But it’s not just the physical wounds though that need treating: young victims may also need help getting back on the right track — whether that means enrolling in school, finding a new group of friends or getting a job.
(BBC News)

10. This 6-year-old is a pioneer in the quest to treat a deadly brain tumor

Callie, a 6-year-old diagnosed with diffuse intrinsic pontine glioma, was treated with low-intensity focused ultrasound (LIFU) at Children’s National Hospital and is the second child in the world to receive this treatment for a brain tumor. LIFU is an emerging technology that experts like Hasan Syed, M.D., and Adrianna Fonseca, M.D., are trialing to treat this fatal childhood brain tumor.
(The Washington Post)

11. F.D.A. approves sickle cell treatments, including one that uses CRISPR

The FDA approved a new genetic therapy, giving people with sickle cell disease new opportunities to eliminate their symptoms. David Jacobsohn, M.B.A., M.D., confirmed that Children’s National Hospital is one of the authorized treatment centers and talked about giving priority to the sickest patients if they are on Vertex’s list.
(The New York Times)

12. 6-year-old fulfils wish to dance in the Nutcracker

After the potential need for open-heart surgery threatened Caroline’s Nutcracker performance, Manan Desai, M.D., a cardiac surgeon, figured out a less invasive procedure to help reduce her recovery time so she could perform in time for the holidays.
(Good Morning America)

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.”

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.

 

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

Treating neurocognitive difficulties in children with sickle cell disease

Sickle-Cell-Blood-Cells

An adaptive cognitive training program could help treat attention and working memory difficulties in children with sickle cell disease (SCD), a new study published in the of Journal of Pediatric Psychology shows.

An adaptive cognitive training program could help treat attention and working memory difficulties in children with sickle cell disease (SCD), a new study published in the of Journal of Pediatric Psychology shows.

These neurocognitive difficulties have practical implications for the 100,000 individuals in the U.S. with SCD, as 20-40% of youth with SCD repeat a grade in school and fewer than half of adults with SCD are employed. Interventions to prevent and treat neurocognitive difficulties caused by SCD have the potential to significantly improve academic outcomes, vocational attainment and quality of life.

The study, led by Steven Hardy, Ph.D., director of Psychology and Patient Care Services at the Center for Cancer and Blood Disorders at Children’s National Hospital, examined a promising approach using an adaptive cognitive training program (known as Cogmed Working Memory Training) that patients complete at home on an iPad.

Using a randomized controlled trial design, children were asked to complete Cogmed training sessions 3 to 5 times per week for about 30 minutes at a time until they completed 25 sessions. The Cogmed program involves game-like working memory exercises that adapt to the user’s performance, gradually becoming more challenging over time as performance improves. The team found that patients with sickle cell disease (SCD) who completed the cognitive training intervention showed significant improvement in visual working memory compared to a waitlist group that used Cogmed after the waiting period. Treatment effects were especially notable for patients who completed a training “dose” of 10 sessions.

“Patients who completed at least 10 cognitive training sessions showed improved visual working memory, verbal short-term memory and math fluency,” Dr. Hardy said.

SCD increases risk for neurocognitive difficulties because of cerebrovascular complications (such as overt strokes and silent cerebral infarcts) and underlying disease characteristics (such as chronic anemia). Neurocognitive effects of SCD most commonly involve problems with attention, working memory and other executive functions.

“This study demonstrates that digital working memory training is an effective approach to treating neurocognitive deficits in youth with sickle cell disease,” Dr. Hardy added. “We also found that benefits of the training extend to tasks that involve short-term verbal memory and math performance when patients are able to stick with the program and complete at least 10 training sessions. These benefits could have a real impact on daily living, making it easier to remember and follow directions in school and at home, organize tasks or solve math problems that require remembering information for short periods of time.”

To date, there have been few efforts to test interventions that address the neurocognitive issues experienced by many individuals with SCD. These findings show that abilities are modifiable and that a non-pharmacological treatment exists.

The Comprehensive Sickle Cell Disease Program at Children’s National is a leader in pediatric SCD research and clinical innovation. This study was funded by a grant from the Doris Duke Charitable Foundation, which was the only Innovations in Clinical Research Award ever awarded to a psychologist (out of 31 grants totaling over $15 million), since the award established a focus on sickle cell disease in 2009.

blood cells with sickle cell anemia

Advances in therapy for sickle cell disease and hemophilia

blood cells with sickle cell anemia

Despite having a network of providers and a national database, access to care and treatment burden continue to be issues that affect quality of life in the hemophilia population.

Hemophilia and sickle cell are disorders that are associated with comorbidities and significant treatment burden, discussed Christine Guelcher, PPCNP-BC, lead advanced practice provider for the Center for Cancer and Blood Disorders at Children’s National Hospital, during the virtual 62nd ASH Annual Meeting and Exposition.

During the satellite symposia, Guelcher explained a network of hemophilia treatment centers (HTCs) was developed in the 1970s. The model of multi-disciplinary care in the HTC network has demonstrated improved outcomes. Despite having a network of providers and a national database, access to care and treatment burden continue to be issues that affect quality of life in the hemophilia population.

“While similar programs were developed in sickle cell with similar improvements in care, the funding was not sustained,” Guelcher said. However, efforts are underway to develop multi-disciplinary care and data infrastructure in the sickle cell community.

“The lack of specialized providers, particularly adult hematologists, continues to be an issue for both non-malignant hematologic disorders,” she added.

Advances in care

While hemophilia is rare, it is an expensive disease. Controlling bleeding with medications is expensive and associated with significant treatment burden. Failure to prevent bleeding due to lack of access or adherence can result in debilitating bleeding that impacts on productivity and quality of life. Additionally, clinical trials with gene therapy are ongoing, though questions remain about sustained levels and durability.

“Recent development of drugs that can reduce the frequency of intravenous infusions (extended half-life factor replacement products or subcutaneous novel non-factor prophylaxis) have improved the treatment burden,” Guelcher said. “But access to care continues to be an issue for up to 30% of the patients with bleeding disorders in the U.S.” Sickle cell disease affects mostly Black/African American and Hispanic patients, many of whom already experience health care disparities. While newborn screening, antibiotic prophylaxis and immunizations have decreased life-threatening infections, vaso-occlusive (pain) crisis continues to be a debilitating complication. Furthermore, stroke, pulmonary, cardiac and renal disease are significant comorbidities.

While advances in therapies for sickle cell have provided new treatment options to decrease the frequency of vaso-occlusive crisis, the pathophysiology that results in all of the sequalae is not fully understood. While Bone marrow transplant is potential treatment of the underlying sickle cell disease process, only 20% of patients have a matched sibling donor. Currently, clinical trials are investigating the safety and efficacy of gene therapy. Despite all of these advances, the life expectancy of somebody with sickle cell is 30 years shorter than the general U.S. population.

Access to care

The multi-disciplinary panel presentation at ASH gave participants an opportunity to hear about the challenges facing these patients and families. The overview of new and emerging treatment options gave providers an understanding of treatment options.

“Hopefully, presentations like this will inspire providers to consider a career in non-malignant hematology (particularly adult providers),” Guelcher added.

As one of the nation’s hemophilia and thrombosis treatment centers, Children’s National Hospital provides comprehensive, multi-disciplinary care. Patients can participate in two national registries in order to collect aggregate data that are used to identify trends that impact bleeding disorder patients. Our sickle cell program also offers multi-disciplinary clinics for infants, integrative care for chronic pain and transition, addressing some of the unmet needs that continue to be an issue nationally.

“We also participate in industry sponsored clinical trials to ensure that new therapies, including gene therapy, are safe and effective,” Guelcher explained. “This gives our patients access to state-of-the-art care. Numerous clinical trials to ensure that recently licensed products and gene therapy are safe for use in a pediatric patient with hemophilia and sickle cell are ongoing.”

Steven Hardy

Steven Hardy, Ph.D., awarded prestigious NIH grant for sickle cell research, career development

Steven Hardy

Steven Hardy, Ph.D., a pediatric psychologist in the Center for Cancer and Blood Disorders at Children’s National, has been awarded a K23 Mentored Patient-Oriented Research Career Development Award by the National Heart, Lung, and Blood Institute (NHLBI) in recognition of his progress toward a productive, independent clinical research career. National Institutes of Health (NIH) Mentored Career Development Awards are designed to provide early career investigators with the time and support needed to focus on research and develop new research capabilities that will propel them to lead innovative studies in the future.

Dr. Hardy, who has worked at Children’s National since 2013, specializes in the emotional, behavioral and cognitive aspects of children’s health, with a particular emphasis on evaluating and treating psychological difficulties among children with cancer or sickle cell disease. With the K23 award, he will receive nearly $700,000 over a five-year period, which will provide him with an intensive, supervised, patient-oriented research experience. The grant will support Dr. Hardy’s time to conduct research, allow him to attend additional trainings to enhance research skills, and fund a research project titled “Trajectory of Cognitive Functioning in Youth with Sickle Cell Disease without Cerebral Infarction.”

Many children with sickle cell disease (SCD) also have intellectual challenges which stem from two primary pathways – stroke and other disease-related central nervous system effects. While stroke is a major complication of SCD, the majority of children with SCD have no evidence of stroke but may still exhibit cognitive functioning challenges related to their disease. Such cognitive difficulties have practical implications for the 100,000 individuals in the SCD, as 20-40% of youth with SCD repeat a grade in school and fewer than half of adults with SCD are employed. Dr. Hardy’s project will focus on understanding the scope and trajectory of cognitive difficulties in children with SCD without evidence of stroke, as well as the mechanisms that precipitate disease-related cognitive decline. The study will characterize temporal relationships between biomarkers of SCD severity and changes in cognitive functioning to inform future development of risk stratification algorithms to predict cognitive decline. Armed with the ability to predict cognitive decline, families will have additional information to weigh when making decisions and providers will be better able to intervene and tailor treatment.

Steven Hardy presents sickle cell findings at ASPHO annual meeting

Steven Hardy

Steven Hardy, Ph.D.

Steven Hardy, Ph.D. recently joined medical leaders in Montréal for the American Society of Pediatric Hematology/Oncology’s 30th Annual Meeting, where he and his team presented key findings from their cognitive and psychosocial research program involving youth with Sickle Cell Disease (SCD).
The first presentation, “Processing Speed and Academic Fluency in Youth With Sickle Cell Disease,” showed that, on average, children with SCD are less able to quickly and efficiently process information than their healthy counterparts. This weakness negatively impacted their academic performance, particularly in math fluency, and increased the children’s odds of having to repeat a grade in school.

A second presentation, “Quality of Life and School Absences in Children With Sickle Cell Disease With and Without Asthma,” explored the differences in quality of life between children with SCD only and children with both SCD and asthma (a common comorbidity). Dr. Hardy and his team found that children with both diseases tend to experience a greater impact on quality of life. Other factors – such as the child’s IQ and the family’s financial, material and social resources – moderated this risk.

The presentations were met with enthusiasm from renowned medical professionals from around the world, all of whom came together for collaborative and constructive sessions to move the needle on pediatric care.

In Brief- Fetal Medicine

Cognitive training exercises at home help kids with sickle cell boost visuospatial working memory

A team led by Children’s National Health System clinicians and research scientists attempted to identify novel approaches to boost working memory in children who suffer from sickle cell disease.

A team led by Children’s National Health System clinicians and research scientists attempted to identify novel approaches to boost working memory in children who suffer from sickle cell disease.

Youths with sickle cell disease who used hand-held computers to play game-like exercises that get harder as a user’s skill level rises improved their visuospatial working memory (WM). Children with sickle cell disease, however, completed fewer training sessions during an initial study compared with children with other disease-related WM deficits.

A team led by Children’s National Health System clinicians and research scientists attempted to identify novel approaches to boost WM in children who suffer from sickle cell disease. Kids who have this red blood cell disorder inherit abnormal hemoglobin genes from each parent. Rather than slipping through large and small vessels to ferry oxygen throughout the body, their stiff, sickle-shaped red blood cells stick to vessel walls, impeding blood supply and triggering sudden pain. Children with sickle cell disease have more difficulty completing tasks that place demands on one’s WM, the brain function responsible for temporarily remembering information and manipulating that information to facilitate learning and reasoning. As a result, they’re more likely to repeat a grade, require special academic services, and to have difficulty maintaining employment as adults.

Because computerized cognitive training programs have been used with success to boost WM for children with other health conditions, such as childhood cancer, the research team sought to examine the feasibility of using the technique for kids with sickle cell disease. “This small study highlights the challenges and opportunities of implementing a home-based cognitive training intervention with youths who have sickle cell disease,” says Steven J. Hardy, PhD, a pediatric psychologist in the Divisions of Hematology, Oncology, and Blood and Marrow Transplantation at Children’s National. “While a larger, randomized controlled clinical trial is needed to better characterize efficacy, our initial work indicates that Cogmed is acceptable and moderately feasible in this population.”

Children’s National is home to the Sickle Cell Disease (SCD) Program, one of the nation’s largest, most comprehensive pediatric programs that cares for 1,350 patients younger than 21 annually. Over 15 months, the team recruited youths aged 7 to 16 participating in the program who had an intelligence quotient of at least 70 and an absolute or relative memory deficit. Those who lacked access to a tablet computer were loaned an iPad Mini 2 loaded with Cogmed RM, an interactive audiovisual cognitive training program that consists of exercises that get progressively more challenging. A clinical psychologist provided coaching and moral support through weekly telephone calls to review progress and challenges, and to offer tips on how to optimize the youths’ progress.

Six of 12 eligible participants – all girls – completed by finishing at least 20 sessions of the program. The mean number of sessions completed was 15.83, and the kids spent a median of 725 minutes working actively on Cogmed exercises. “Participants who completed Cogmed indicated that they perceived greater levels of social support from teachers,” Hardy and colleagues write in the study, published by Pediatric Blood & Cancer. “[T]here was not a statistical difference in perceived parent support.”

Among those children who completed Cogmed, standard scores increased an average of 5.05 on a measure of visuospatial short-term memory, 19.72 on a measure of verbal WM, 27.53 on a measure of visuospatial short-term memory, and 23.82 on a measure of visuospatial WM. The researchers also observed a normalizing of memory functioning for those who finished Cogmed, as a significant portion of participants scored below the average range before using Cogmed and most scored in the average range or higher on memory tests after finishing the program.

“In this initial feasibility trial, adherence to Cogmed was lower than expected (50 percent completion) compared to adherence rates of other samples of children with medical histories, including patients with symptomatic epilepsy and youth treated for cancer,” Hardy and co-authors write. “Thus, additional modifications may be needed to achieve consistent delivery of the intervention to youth with SCD.”

Related Resources: Research at a Glance

Feasibility of home-based computerized WM training for sickle cell disease

PDF Version

What’s Known
Cognitive deficits are a common complication affecting about one-third of kids who have the higher risk sickle cell disease genotypes, HbSS and HbSβ0 thalassemia. While such deficits have been well-documented, no treatment has been proven to recover cognitive function for kids with sickle cell disease. Sickle cell disease is a group of red blood cell disorders in which abnormal genes that children inherit from parents cause their bodies to make sickle-shaped hemoglobin S. Kids with sickle cell disease are at heightened risk for neurocognitive deficits, which can have practical implications for their ability to learn and to succeed academically.

What’s New
Because home-based computerized cognitive training programs have helped to improve working memory (WM) for children with epilepsy and for childhood cancer survivors, a team led by Children’s National Health System researchers and clinicians sought to gauge the feasibility of using such an approach for a small number of children with sickle cell disease. The pilot study found that girls were more likely to complete the cognitive training exercises than boys. Additionally, children whose teachers showed a high level of support spent more time working on the exercises, completed more sessions, and were more likely to finish the program. The mean number of completed sessions was 15.83. Participants who reported experiencing fewer functional limitations as a result of sickle cell disease-related pain completed more sessions. Overall, children who completed Cogmed exercises showed improvements in verbal WM, and visuospatial short-term memory and WM.

Questions for Future Research
Q: Because sickle cell disease is often accompanied by sudden attacks of severe pain, would concurrent delivery of pain-management interventions impact children’s ability to complete cognitive training exercises?
Q: Would adding functional magnetic resonance imaging help to clarify the association between adaptive cognitive skills training and changes to physiological processes, such as alterations in prefrontal and parietal cortical activity, and functional connectivity?

Source: S.J. Hardy, K.K. Hardy, J.C. Schatz, A.L. Thompson, and E.R. Meier. “Feasibility of Home-Based Computerized Working Memory Training With Children and Adolescents With Sickle Cell Disease.” Published online by Pediatric Blood & Cancer May 26, 2016.

Feasibility of home-based computerized working memory training with sickle cell disease patients

Children with sickle cell disease are at heightened risk for neurocognitive deficits. The research team sought to fill a gap in the research by evaluating the feasibility of using a home-based computerized working memory (WM) training intervention for children aged 7 to 16 years with sickle cell disease. Study participants used loaner iPads and were asked to work on Cogmed five days a week for five weeks – or a maximum of 25 sessions. According to research published by Pediatric Blood and Cancer, girls were more likely to complete the assignments, compared with boys. The mean number of sessions completed was 15.83.