American Society of Hematology 2021 guidelines for sickle cell disease: Stem cell transplantation

Julie Kanter, Robert I. Liem, Françoise Bernaudin, Javier Bolaños-Meade, Courtney D. Fitzhugh, Jane S. Hankins, M. Hassan Murad, Julie A. Panepinto, Damiano Rondelli, Shalini Shenoy, John Wagner, Mark C. Walters, Teonna Woolford, Joerg J. Meerpohl, John Tisdale

Research output: Contribution to journalReview articlepeer-review

6 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)3668-3689
Number of pages22
JournalBlood Advances
Volume5
Issue number18
DOIs
StatePublished - Sep 28 2021

Bibliographical note

Funding Information:
The work of this panel was coordinated with 4 other guideline panels (addressing other aspects of SCD) by ASH and the Mayo Evidence-Based Practice Research Center (funded by ASH under a paid agreement). Project oversight was provided by a coordination panel, which reported to the ASH Guideline Oversight Subcommittee. ASH vetted and appointed individuals to the guideline panel. The Mayo Center vetted and retained researchers to conduct systematic reviews of evidence and coordinated the guideline development process, including the use of the GRADE approach.8 The membership of the panels and the Mayo Center team is described in Supplement 1.

Publisher Copyright:
© 2021 by The American Society of Hematology.

PubMed: MeSH publication types

  • Journal Article
  • Practice Guideline

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