NEW! ASH Clinical Practice Guidelines on Stem Cell Transplantation in Sickle Cell Disease
The American Society of Hematology (ASH) has published clinical practice guidelines on stem cell transplantation as a treatment for sickle cell disease (SCD). The guidelines, which are the final installment of the Society’s five evidence-based clinical practice guidelines on sickle cell disease (SCD), round out the full collection of recommendations for improving care for individuals living with SCD.
Allogeneic hematopoietic cell transplantation (alloHCT) is currently the only potentially curative therapy for SCD. The guidelines, published in ASH’s journal Blood Advances, advise when to use alloHCT in clinical practice through weighing of risks and benefits of transplantation versus disease-modifying therapies or potential curative therapies still under development, such as gene therapy.
“We have known for decades that transplantation is indeed curative for SCD, but we have never before had a set of comprehensive recommendations about how the therapy fits into the treatment options for this disease,” said John Tisdale, MD, guideline co-chair and Chief of the Cellular and Molecular Therapeutics Branch at the National Heart, Lung, and Blood Institute (NHLBI). “It is important that discussions about transplantation begin at a young age for all individuals with SCD as one possible treatment option available over their lifetime. These guidelines inform the continued discussions and risk-benefit analyses between patients and providers.”
Highlights of the ASH Guideline Recommendations on stem cell transplantation in SCD include:
- alloHCT should be considered over standard of care (transfusion) in individuals with SCD who have experienced a stroke or are at very high risk of stroke. Further, transplantation should be considered for all patients with neurologic injury who have a matched, related sibling donor. Recommendations point to evidence suggesting that children under age 13 who receive alloHCT from a matched sibling donor have better outcomes than those older than age 13.
- For patients with frequent pain, as well as those with recurrent episodes of acute chest syndrome, the ASH guidelines suggest transplantation from a matched sibling donor over the standard of care.
- For individuals with an indication for alloHCT who lack a matched sibling donor, the ASH guidelines suggest transplantation from alternate donors only in the context of a clinical trial.
- In patients with an indication for transplant, the ASH guidelines suggest transplantation with cells from a matched donor earlier in life due to the risk of irreversible SCD-related damage to the body that increases with age.
In partnership with the Evidence-Based Practice Research Program at Mayo Clinic, the ASH Guidelines on SCD were developed using the GRADE methodology to ensure the highest standards for trustworthiness. ASH brought together 61 clinical experts, five methodologists, and 10 patient representatives to identify best practices for the management of acute and chronic complications of SCD and ways to improve the quality of care for patients.
“The culmination of the full set of clinical practice guidelines for sickle cell disease is an important achievement for ASH, since supporting research and improving care for all people living with the disease is one of the Society’s key priorities,” said ASH President Martin S. Tallman, MD, of Memorial Sloan Kettering Cancer Center. “It is of even greater importance to the SCD community. As treatment for individuals living with SCD has continued to evolve and progress, a comprehensive, evidence-based set of treatment guidelines is invaluable to patients, providers, and caregivers.”
Find more information about the ASH Guidelines on SCD, including fact sheets, infographics, clinical teaching slide sets, and patient stories, at hematology.org/SCDguidelines.
ASH Honors Donna S. Neuberg with 2021 Exemplary Service Award
ASH will recognize Donna S. Neuberg, ScD, senior statistician for the Leukemia, Lymphoma, and Benign Hematology Programs at Dana-Farber Cancer Institute, with the 2021 Exemplary Service Award for her years of service and dedication to ASH and to hematology.
The Exemplary Service Award was established in 1998 to recognize an individual whose outstanding service, extending over a period of years, has significantly advanced the interests of the Society. ASH President Martin S. Tallman, MD, will present this award during the 63rd ASH Annual Meeting and Exposition this December that will take place both in-person in Atlanta, Georgia, and virtually.
“Dr. Neuberg is an outstanding colleague and mentor as well as a world-class statistician with an encyclopedic knowledge of hematology,” said Dr. Tallman. “Through her decades of dedicated service in ASH training programs, Dr. Neuberg has empowered innumerable investigators to design clinical trials with the highest standards of scientific rigor and statistical methodology. We value Dr. Neuberg’s contributions to shaping the future of hematology and of ASH.”
After receiving her ScD in Biostatistics from the Harvard School of Public Health, Dr. Neuberg joined the Dana-Farber Cancer Institute as a biostatistician. She currently serves as the senior statistician for the Leukemia, Lymphoma, and Benign Hematology Programs at Dana-Farber, where her research interests focus on the planning and analysis of studies that integrate laboratory outcomes at the genome, transcriptome, or proteome level with clinical and demographic information.
From 2014 to 2017, Dr. Neuberg served on the editorial board of Blood, ASH’s peer-reviewed journal. She has also demonstrated her commitment to ASH through her service as a statistician for the ASH Research Collaborative (ASH RC) Data Hub. Her expertise was integral in the development of the ASH RC COVID-19 Registry for Hematology data summaries, figures, and publications.
Dr. Neuberg has served as a dedicated mentor for the ASH-European Hematology Association (EHA) Translational Research Training in Hematology (TRTH), a year-long training program helping early-stage researchers build successful hematology research careers, since its inception in 2010. Dr. Neuberg is celebrated by TRTH participants for empowering trainees to take steps toward becoming not only rigorous scientists, but also thoughtful citizens of the international hematology community. In addition to celebrating the help they received in bringing their research to exceptional standards, trainees laud Dr. Neuberg for her generosity with her time and energy, and her ability to inspire them personally and professionally.
Dr. Neuberg’s deep passion for training the next generation of scientists is also reflected in her long-standing service as a mentor in the ASH Clinical Research Training Institute (CRTI), a year-long education program for hematology fellows and junior faculty at academic medical centers. She has shared her expertise as CRTI faculty from 2010 to 2016, and has participated in the study sections for the CRTI and the ASH Scholar Award. Her mentees and scholars credit her with imbuing them with her own unmistakable passion for sensible study design, experimental rigor, and enjoyment of a life in biomedical investigation.