Welcome to the Sickle Cell Disease Association of America’s Clinical Trial Finder, created in partnership with Forma Therapeutics. Our Clinical Trial Finder is a centralized, simple-to-navigate resource that helps people with sickle cell disease, their families and caregivers find clinical trials. Clinical trials help pharmaceutical companies and researchers develop new sickle cell treatments. Your participation helps guide these breakthroughs and also gives you access to these new treatments before they are available to the public. Click here to learn more about clinical trials and why they are important. Use the tool below to search for trials by keyword, location, study type and phase – now listed in one place.
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As we closed 2023, the Food and Drug Administration (FDA) approved two gene therapies to treat sickle cell disease (SCD). These potential curative therapies include CASGEVY™ (exagamglogene autotemcel [exa-cel]) from Vertex Pharmaceuticals Incorporated and CRISPER Therapeutics, and LYFGENIATM from bluebirdbio. While there is a lot of excitement for these advances, the impacts of these therapies are not currently known. Like with many therapies, the impact on women and their future fertility are among the unknowns and are areas of concern for individuals living with sickle cell disease and their healthcare providers.
Please join 7-2-1 for this special edition episode, How New Gene Therapies for SCD May Impact Women’s Fertility, with WGSCD LAN Chair, Dr. Alecia Nero and special guest, Dr. Kelly Acharya, a Reproductive Endocrinologist, and Infertility Specialist with Duke Health. Together, they discuss the areas of concern for women and girls living with SCD and consider what the future may hold for those who are able to receive these life-changing treatments so that they may stay informed in the decision-making process.
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Two gene therapies were recently approved by the Food and Drug Administration (FDA) to treat sickle cell disease: Casegevy from CRISPR/Vertex and Lyfgenia from bluebird bio. You probably have questions about these new treatment options. Read more below.
Is gene therapy a cure for sickle cell disease?
Gene therapy is a potentially curative therapy. This means that it could act as a cure, but it is too new to say for sure. It causes a big decline in pain episodes, but we need to learn more about long-term impacts and side effects. It is also not a “one-and-done” treatment. The FDA currently recommends 15 years of patient follow up.
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One unifying message in the sickle cell community is that our mental health is just as important as our physical health. Therefore as a community, we have to start recognizing that our mental health matters by prioritizing our emotional well-being, we open ourselves up to cope with the physical demands of sickle cell disease in a healthier way. We develop resilience, an acceptance of what we can and cannot control, and the tools to manage life’s ups and downs.
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Prioritizing Mental Health as a Sickle Cell Warrior, Caregiver, and Healthcare Worker
As an individual living with sickle cell, caregiver, or healthcare worker, you may be accustomed to prioritizing physical health needs above all else. However, mental health is critical for overall well-being and should not be ignored. Although mental health challenges can be stigmatized and overlooked in the Black community, it is important to erase the stigma and seek the care that is needed. In this mental health toolkit, we discuss the importance of prioritizing mental health as a sickle cell warrior, caregiver, or healthcare worker and how therapy can help to achieve that goal. This resource is a sample of what is offered in the Sickle Cell Community.
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Objective: Sickle cell disease is a lifelong illness affecting millions of people globally but predominantly burdensome in sub-Saharan Africa, where most affected children do not live to adulthood, despite available evidence-based interventions that reduce the disease burden in high-income countries.
Method: We reviewed studies evaluating evidence-based interventions that decrease sickle cell disease-related morbidity and mortality among children living in sub-Saharan Africa. We used the Joanna Briggs scoping review methodological framework and grouped identified evidence-based interventions into preventative pharmacotherapeutic agents, newborn screening and comprehensive healthcare, disease-modifying agents, nutritional supplementation, systemic treatment, supportive agents and patient/carer/population education.
Results: We included 36 studies: 18 randomized controlled trials, 11 observational studies, 5 before-and-after studies and 2 economic evaluation studies, with most of the studies performed in West African countries. Included studies suggest evidence-based interventions effectively to reduce the common morbidities associated with sickle cell disease such as stroke, vaso-occlusive crisis, acute chest syndrome, severe anaemia and malaria infection. Evidence-based interventions also improve survival among study participants. Specifically, our review shows hydroxyurea increases haemoglobin and foetal haemoglobin levels, a finding with practical implications given the challenges with blood transfusion in this setting. The feasibility of implementing individual interventions is hampered by challenges such as affordability, accessibility and the availability of financial and human resources.
Conclusion: Our review suggests that regular use of low-dose hydroxyurea therapy, sulphadoxine-pyrimethamine chemoprophylaxis, L-arginine and Omega-3 fatty acid supplementation and establishment of specialist stand-alone sickle cell clinics could reduce the sickle cell disease-associated morbidity and mortality in sub-Saharan Africa countries.
Keywords: evidence-based medicine; scoping review; sickle cell disease; sub-Saharan Africa.
