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Blood. 2019 Oct 28. pii: blood.2019000821. doi: 10.1182/blood.2019000821. [Epub ahead of print]

How I treat sickle cell disease with hematopoietic cell transplantation.

Author information

1
UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States.
2
Washington University School of Medicine, Saint Louis, Missouri, United States.
3
Children's Healthcare of Atlanta - Egleston, Atlanta, Georgia, United States.

Abstract

Sickle cell disease (SCD) leads to significant morbidity and early mortality, and hematopoietic cell transplantation (HCT) is the only widely available cure, with impacts seen on SCD-related organ dysfunction. Outcomes are excellent following matched related donor (MRD) HCT, leading to significantly expanded application of this treatment over the past decade. The majority of SCD patients lack a MRD, but outcomes following alternative donor HCT continue to improve on clinical trials. Within this framework, we aim to provide our perspective on how to apply research findings to clinical practice, for an individual patient. We also emphasize that the preparation of SCD recipients for HCT and supporting them through HCT has special nuances that require awareness and close attention. Through the use of clinical vignettes, we provide our perpsective on the complex decision-making process in HCT for SCD as well as recommendations for the evaluation and support of these patients through HCT.

PMID:
31697818
DOI:
10.1182/blood.2019000821

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