research about sickle cell anemia

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Sickle Cell Disease Research

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The National Institutes of Health (NIH) has supported research on sickle cell disease since before the NHLBI was founded in 1948. With each decade that followed, the NHLBI has kept a sustained focus on advancing the understanding of sickle cell disease and improving clinical care. We lead and support research and programs on sickle cell disease in the United States and around the world. Research and initiatives supported by the NHLBI work to improve treatments and evidence-based clinical care for all individuals living with sickle cell disease. We are committed to building on our legacy of research excellence to find new treatments, cures, and personalized care for the approximately 100,000 Americans and over 20 million people worldwide who have sickle cell disease.

NHLBI research that really made a difference

NHLBI-funded scientists found an effective sickle cell treatment in 1995. Results from the NHLBI Multicenter Study of Hydroxyurea showed that hydroxyurea reduced the number of painful episodes by 50% in severely affected adults with sickle cell disease. Three years later, the U.S. Food and Drug Administration approved hydroxyurea to treat sickle cell disease in adults. Research into optimizing the use of hydroxyurea continued over the next decade. In 2011, results from an NHLBI-funded study called BABY HUG found hydroxyurea to be safe for young children who have sickle cell disease.

Current research funded by the NHLBI

Our Division of Blood Diseases and Resources and the Division of Intramural Research , specifically the Cellular and Molecular Therapeutics Branch and the Sickle Cell Branch , oversee much of the research on sickle cell disease that we fund.

Find funding opportunities and program contacts for sickle cell disease research.

Current research on sickle cell disease treatment

Many current studies are looking at how to use genetic therapies and blood and bone marrow transplants to discover new treatment options for patients.

Advances in genetics over the last decade may make effective gene-based treatments a reality for people with sickle cell disease. Through funding by the NHLBI and other collaborations , researchers are developing easy-to-administer gene-based interventions.

Read about the Cure Sickle Cell Initiative , a collaborative research effort led by the NHLBI that is accelerating the development of genetic therapies to cure sickle cell disease.

Many patients with sickle cell disease receive frequent blood transfusions to treat and prevent certain complications. NHLBI-funded researchers are investigating the beneficial and harmful effects of blood transfusions in patients with this condition. In addition, researchers are developing more effective medicine support for blood transfusions when patients undergo bone marrow transplantation. This research is critical because a blood and bone marrow transplant is currently the only cure for many people living with sickle cell disease.
The NHLBI also funds efforts to improve bone marrow transplantation (BMT) through the BMT Clinical Trials Network . For example, one current trial is investigating treatment of severe sickle cell disease with a BMT procedure that involves either a related or unrelated immune-matched donor.

Find more NHLBI-funded studies on sickle cell disease treatment at NIH RePORTER.

Read more in this news release about NHLBI-funded research on blood transfusions. The study found that using fresh red blood cells — cells that have spent seven days or less in storage — are no more beneficial than older red blood cells in reducing the risk of organ failure or death in critically ill children.

Current research on pain management

Vaso-occlusive crises are painful episodes that affect many people with sickle cell disease. The crises are the leading cause of emergency department visits and hospitalizations. As part of the Sickle Cell Disease Treatment with Arginine Therapy (STArT) Trial , NHLBI-funded researchers found that patients with sickle cell disease and pain also had low levels of an amino acid called arginine in their blood. The study is investigating whether arginine supplements can reduce pain in patients. Other NHLBI-funded research seeks to determine the best way to calculate dosages of pain medicine for patients with vaso-occlusive episodes (severe pain that occurs when the sickle-shaped cells block blood flow to a part of the body).
Pain and sickle cell disease often go hand-in-hand, but the exact cause of this pain is not well understood. NHLBI-funded researchers are using animal models of sickle cell disease to better understand the causes and treatments of chronic pain experienced by patients with sickle cell disease.

Find more NHLBI-funded studies on pain management in sickle cell disease at NIH RePORTER.

Current research on improving care for all people with sickle cell disease

The NHLBI is committed to research that will help reduce the barriers patients face when accessing sickle cell disease treatment. Find more NHLBI-funded studies on health disparities and sickle cell disease at NIH RePORTER.

Sickle cell disease research labs at NHLBI

Researchers from the NHLBI Division of Intramural Research , which includes investigators in our Sickle Cell Branch , are focused on developing new treatments for sickle cell disease.

Intramural NHLBI researchers have developed a new and improved viral vector — a virus-based vehicle that delivers therapeutic genes — for use in genetic therapy for sickle cell disease.
The NHLBI established the Biologic Specimen and Data Repository Information Coordinating Center (BioLINCC) in 2008. Since then, it has grown to include datasets from more than 140 epidemiological studies and clinical trials and about 4 million specimen collections, including from patients with sickle cell disease. Researchers within the NHLBI recently used data from the BioLINCC to reaffirm that hydroxyurea is safe and effective for very young children with sickle cell disease.

Read more about these projects and ongoing clinical trials .

Related sickle cell disease programs

The NHLBI leads and supports many programs and initiatives around the nation and the world as we search for a cure and work to improve the lives of people with sickle cell disease.

The NHLBI’s annual Sickle Cell Disease Research Meeting brings together investigators, practitioners, and healthcare providers to discuss the progress of ongoing clinical trials and hear presentations about new developments in scientific and clinical aspects of sickle cell disease.
The NHLBI supports three major programs in sub-Saharan Africa across 9 countries and 11 cities: The Sickle Pan-African Research Consortium , the Sickle Cell Disease Genomics Network of Africa , and the Realizing Effectiveness Across Continents with Hydroxyurea (REACH) Program . All are working to build research capacity and develop an infrastructure to enhance disease surveillance and delivery of care.
The NHLBI participates in the Stimulating Hematology Investigation: New Endeavors (SHINE) Program, which focuses on basic and early translational hematology research and encourages applications from investigators at all career stages.
The Recipient Epidemiology and Donor Evaluation Study (REDS) Program evaluates and improves the safety and effectiveness of transfusion therapies. The program also works to proactively address potential emerging threats to the United States’ blood supply and serves as a resource for ongoing work in transfusion research.
The NHLBI has taken a lead role in managing the Regenerative Medicine Innovation Project (RMIP) under the 21st Century Cures Act . Beginning in 2017, the Act authorized the investment of $30 million in clinical research with adult stem cells to treat diseases including sickle cell disease.

Explore more NHLBI research on sickle cell disease

The sections above provide you with the highlights of NHLBI-supported research on sickle cell disease. You can explore the full list of NHLBI-funded studies on the NIH RePORTER .

To find more studies:

Type your search words into the Quick Search box and press enter.
Check Active Projects if you want current research.
Select the Agencies arrow, then the NIH arrow, then check NHLBI .

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View all journals

Sickle cell disease articles from across Nature Portfolio

Sickle cell disease is an autosomal recessive blood disorder that can lead to anaemia. It is caused by a mutation in the haemoglobin gene, which leads to deformation of red blood cells. Deformed red blood cells can obstruct small vessels and they are prone to destruction.

Latest Research and Reviews

Brain network hypersensitivity underlies pain crises in sickle cell disease

Minkyung Kim

Risk factors for acute chest syndrome among children with sickle cell anemia hospitalized for vaso-occlusive crises

Faisal A. Alghamdi
Fawaz Al-Kasim
Rehab Alluqmani

Development of pathophysiologically relevant models of sickle cell disease and β-thalassemia for therapeutic studies

Sickle cell disease (SCD) and β-thalassemia (BT) are globally prevalent inherited blood disorders but, despite extensive research, no ex vivo system exists for SCD and BT. Here, the authors generate pathophysiologically relevant erythroid progenitor models of SCD and BT.

Pragya Gupta
Sangam Giri Goswami
Sivaprakash Ramalingam

The value-based price of transformative gene therapy for sickle cell disease: a modeling analysis

George Morgan
Gregory F. Guzauskas

Evaluating sheep hemoglobins with MD simulations as an animal model for sickle cell disease

Caroline E. Kuczynski
Christopher D. Porada
Graça Almeida-Porada

An α-chain modification rivals the effect of fetal hemoglobin in retarding the rate of sickle cell fiber formation

Eli H. Worth
Mark K. Fugate
Frank A. Ferrone

News and Comment

Autologous globin-edited HSCs ameliorate sickle cell disease

Gene correction for sickle cell disease hits its prime

Prime editing can efficiently rewrite the genetic mutation causing sickle cell disease, in haematopoietic stem cells from patients.

Sébastien Levesque
Daniel E. Bauer

Health-related quality of life in sickle cell disease

Julie A. Panepinto
Gregory J. Kato
Wally R. Smith

Comparing health-related quality of life in chronic diseases: the importance of analyzing references

Christine Maynié-François
Stéphane Burtey

Omega-3 fatty acids are a potential therapy for patients with sickle cell disease

Adrian Rabinowicz
Kebreab Ghebremeskel

Sickle cell solutions in sight

New targets, new drug modalities and new business strategies are drawing long-awaited attention to sickle cell disease.

Katie Kingwell

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Trends in quality of care among children with sickle cell anemia

Affiliations.

1 Susan B Meister Child Health Evaluation and Research Center, Department of Pediatrics, University of Michigan, Ann Arbor, Michigan, USA.
2 Department of Epidemiology, University of Michigan, Ann Arbor, Michigan, USA.
3 New York State Department of Health, Albany, New York, USA.
4 Department of Pediatrics, Nationwide Children's Hospital, Columbus, Ohio, USA.
PMID: 34854548
PMCID: PMC9367210
DOI: 10.1002/pbc.29446

Introduction: For decades, it has been recommended that children with sickle cell anemia (SCA) receive antibiotic prophylaxis to prevent serious infections and undergo transcranial Doppler (TCD) screening to identify those at highest risk of overt stroke. We assessed recent temporal trends in antibiotic prophylaxis prescription fills and TCD screening among children with SCA using validated quality measures.

Procedure: Using validated claims-based definitions, we identified children with SCA who were enrolled in Michigan or New York State (NYS) Medicaid programs (2011-2018). Among recommended age groups, two outcomes were assessed yearly: (a) filling of ≥300 days of antibiotics, and (b) receipt of greater than or equal to one TCD. The proportion of children with each outcome was calculated by state. Temporal trends in each preventive service were assessed using generalized linear models.

Results: A total of 1784 children were eligible for antibiotic prophylaxis (Michigan: 384; NYS: 1400), contributing 3322 person-years. Annual rates of filling ≥300 days of antibiotics ranged from 16% to 22% and were similar by state. There was no change in rates of antibiotic filling over time in Michigan (p-value: .10), but there was a decrease in NYS (p-value: .02). A total of 3439 children with SCA were eligible for TCD screening (Michigan: 710; NYS: 2729), contributing 10,012 person-years. Annual rates of TCD screening ranged from 39% to 45%, were similar by state, and did not change over time (p-values >.05).

Conclusions: Most children with SCA do not receive recommended antibiotic prophylaxis and/or TCD screening. New, sustainable, and coordinated interventions across preventive services are urgently needed.

Keywords: epidemiology; quality of life; sickle cell anemia.

PubMed Disclaimer

Conflict of interest statement

Conflict of interest: The authors have no conflicts of interest to report.

Proportion of children with sickle…

Proportion of children with sickle cell anemia in Medicaid receiving 300+ days of…

Proportion of children with sickle cell anemia in Medicaid receiving annual TCD screen

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Sickle Cell Disease Research | NHLBI, NIH
Current research on improving care for all people with sickle cell disease. The NHLBI is committed to research that will help reduce the barriers patients face when accessing sickle cell disease treatment. Find more NHLBI-funded studies on health disparities and sickle cell disease at NIH RePORTER.
Sickle cell disease - Latest research and news | Nature
Sickle cell disease is an autosomal recessive blood disorder that can lead to anaemia. It is caused by a mutation in the haemoglobin gene, which leads to deformation of red blood cells.
Trends in quality of care among children with sickle cell anemia
Introduction: For decades, it has been recommended that children with sickle cell anemia (SCA) receive antibiotic prophylaxis to prevent serious infections and undergo transcranial Doppler (TCD) screening to identify those at highest risk of overt stroke.