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Research priorities will help direct the field and deliver progress for the next five years

(WASHINGTON, September 25, 2024) — The ÎÚÑ»´«Ã½ (ASH) has released new comprehensive sickle cell disease (SCD) research priorities. The priorities highlight seven key areas on which the SCD stakeholder community should focus their efforts to achieve maximum impact and patient benefit, providing a roadmap for advancing SCD research, training health care providers, and improving standards of care over the next five years.

Sickle cell disease (SCD) is an inherited, lifelong chronic disorder affecting nearly 100,000 people in the United States. According to the Centers for Disease Control and Prevention, SCD affects one out of every 365 Black or African American births and one out of every 16,300 Hispanic American births. SCD is a growing global health problem that will touch nearly 30% more people around the world in the next three decades.

Although new approaches to managing SCD have led to improvements in diagnosis and supportive care, individuals living with the disease still have severe complications to overcome. The future of care for those with SCD will depend on advanced and highly targeted approaches to research, discovery, and the implementation of proven and new interventions.

“ASH has been a catalyst for change in sickle cell disease, driving research and improving outcomes for decades,” said 2024 ASH President Mohandas Narla, DSc, distinguished scientist at New York Blood Center Enterprises. “Despite significant advancements, current treatments and care models fall short in addressing the complex challenges faced by individuals with SCD, particularly in marginalized communities. Our new research priorities aim to be a guide for future research and comprehensive care, ensuring better access and equity for all patients. It’s imperative that we continue to move the field forward to deliver more progress for people living with SCD.”

The new research priorities were developed in collaboration with a wide range of researchers and health care providers, with an emphasis on the current realities, challenges, and aspirations of SCD research. The priorities highlight unaddressed questions in the SCD research space, the answers to which would accelerate progress in basic, translational, and clinical research, improving the lives of individuals living with the disease.

The seven key research focus areas are:

I) The assessment, prevention, and treatment of end-organ dysfunction to grow clinical understanding of the risk and progression of organ dysfunction for people living with SCD.
II) Enhance pain research to understand the mechanisms behind pain and develop new treatments to improve patient-reported pain.
III) Optimize use of hydroxyurea, blood transfusions, and currently approved pharmacologic therapies to help providers select the most appropriate medications and doses for individuals living with SCD.
IV) Develop novel drug therapies, such as those which directly inhibit organ damage from SCD or new combination therapeutics.
V) Strengthen curative therapies by continuing investment into different approaches to partial matched transplants and understanding how such therapies may affect organs impacted by SCD.
VI) Impact of the dissemination and implementation of evidence-based guidelines for SCD to expedite the uptake of evidence-based therapies and care delivery across variable settings.
VII) Expansion of sickle cell trait research, including addressing reproductive and genetic counseling for individuals with sickle cell trait through community-based research.

“There have been significant advances in SCD, but research gaps continue,” said Betty Pace, MD, professor of pediatrics at Augusta University and co-chair of the SCD research priorities working group. “There are too many unanswered questions, from how to prevent and treat organ damage to how clinicians understand and treat pain. I urge the SCD community and funders to use these priorities to direct their research so we can work toward our shared goal of providing excellent individualized care for people living with SCD.”

These new research priorities update ASH’s 2014 priorities, which sought answers to unaddressed questions and identified key research topics to advance the understanding of and treatment for SCD. ASH plans to review and update the priorities again in 2029.

The full list of research priorities and other SCD programs led by ASH can be viewed at hematology.org/SCD.

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The ÎÚÑ»´«Ã½ (ASH) (hematology.org) is the world’s largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment, and prevention of disorders affecting the blood. Since 1958, the Society has led the development of hematology as a discipline by promoting research, patient care, education, training, and advocacy in hematology.

The Blood journals () are the premier source for basic, translational, and clinical hematological research. The Blood journals publish more peer-reviewed hematology research than any other academic journals worldwide.

Contact:
Melissa McGue, ÎÚÑ»´«Ã½
[email protected]; 202-552-4927