Julie Kanter1, Robert I Liem2, Françoise Bernaudin3,4, Javier Bolaños-Meade5, Courtney D Fitzhugh6, Jane S Hankins7, M Hassan Murad8, Julie A Panepinto9, Damiano Rondelli10, Shalini Shenoy11, John Wagner12, Mark C Walters13, Teonna Woolford14, Joerg J Meerpohl15,16, John Tisdale6. 1. Department of Hematology-Oncology, University of Alabama Birmingham, Birmingham, AL. 2. Division of Hematology, Oncology and Stem Cell Transplant, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL. 3. French Referral Center for Sickle Cell Disease, Centre Hospitalier Intercommunal Créteil, Université Paris XII, Paris, France. 4. Société Française de Greffe de Moelle et de Thérapie Cellulaire, Lille, France. 5. Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, MD. 6. Cellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD. 7. Department of Hematology, St Jude Children's Research Hospital, Memphis, TN. 8. Division of Preventive Medicine, Mayo Clinic, Rochester, MN. 9. Medical College of Wisconsin/Children's Wisconsin, Pediatric Hematology, Milwaukee, WI. 10. Division of Hematology-Oncology, University of Illinois at Chicago, Chicago, IL. 11. St Louis Children's Hospital, Washington University School of Medicine, St Louis, MO. 12. Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, MN. 13. Benioff Children's Hospital, University of California San Francisco, Oakland, CA. 14. Sickle Cell Reproductive Health Education Directive, Owings Mills, MD. 15. Institute for Evidence in Medicine, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany; and. 16. Cochrane Germany Foundation, Cochrane Germany, Freiburg, Germany.
Abstract
BACKGROUND: Sickle cell disease (SCD) is a life-limiting inherited hemoglobinopathy that results in significant complications and affects quality of life. Hematopoietic stem cell transplantation (HSCT) is currently the only curative intervention for SCD; however, guidelines are needed to inform how to apply HSCT in clinical practice. OBJECTIVE: These evidence-based guidelines of the American Society of Hematology (ASH) are intended to support patients, clinicians, and health professionals in their decisions about HSCT for SCD. METHODS: The multidisciplinary guideline panel formed by ASH included 2 patient representatives and was balanced to minimize potential bias from conflicts of interest. The Mayo Evidence-Based Practice Research Program supported the guideline development process, including performing systematic evidence reviews (through 2019). The panel prioritized clinical questions and outcomes according to their importance for clinicians and patients. The panel used the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach, including GRADE Evidence-to-Decision frameworks, to assess evidence and make recommendations, which were subject to public comment. RESULTS: The panel agreed on 8 recommendations to help patients and providers assess how individuals with SCD should consider the timing and type of HSCT. CONCLUSIONS: The evidence review yielded no randomized controlled clinical trials for HSCT in SCD; therefore, all recommendations are based on very low certainty in the evidence. Key recommendations include considering HSCT for those with neurologic injury or recurrent acute chest syndrome at an early age and to improve nonmyeloablative regimens. Future research should include the development of a robust SCD registry to serve as a comparator for HSCT studies.
BACKGROUND: Sickle cell disease (SCD) is a life-limiting inherited hemoglobinopathy that results in significant complications and affects quality of life. Hematopoietic stem cell transplantation (HSCT) is currently the only curative intervention for SCD; however, guidelines are needed to inform how to apply HSCT in clinical practice. OBJECTIVE: These evidence-based guidelines of the American Society of Hematology (ASH) are intended to support patients, clinicians, and health professionals in their decisions about HSCT for SCD. METHODS: The multidisciplinary guideline panel formed by ASH included 2 patient representatives and was balanced to minimize potential bias from conflicts of interest. The Mayo Evidence-Based Practice Research Program supported the guideline development process, including performing systematic evidence reviews (through 2019). The panel prioritized clinical questions and outcomes according to their importance for clinicians and patients. The panel used the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach, including GRADE Evidence-to-Decision frameworks, to assess evidence and make recommendations, which were subject to public comment. RESULTS: The panel agreed on 8 recommendations to help patients and providers assess how individuals with SCD should consider the timing and type of HSCT. CONCLUSIONS: The evidence review yielded no randomized controlled clinical trials for HSCT in SCD; therefore, all recommendations are based on very low certainty in the evidence. Key recommendations include considering HSCT for those with neurologic injury or recurrent acute chest syndrome at an early age and to improve nonmyeloablative regimens. Future research should include the development of a robust SCD registry to serve as a comparator for HSCT studies.
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