Allogeneic hematopoietic stem cell transplantation for sickle cell disease: the time is now.

Abstract:

:Although sickle cell disease (SCD) has a variable clinical course, many patients develop end-organ complications that are associated with significant morbidity and early mortality. Myeloablative allogeneic HSCT (allo-HSCT) is curative but has been historically performed only in children younger than 16 years of age. Modest modifications in the conditioning regimen and supportive care have improved outcome such that the majority of children with a suitable HLA-matched sibling donor can expect a cure from this approach. However, adult patients have been excluded from myeloablative allo-HSCT because of anticipated excess toxicity resulting from accumulated disease burden. Efforts to use nonmyeloablative transplantation strategies in adults logically followed but were initially met with largely disappointing results. Recent results, however, indicate that nonmyeloablative allo-HSCT in adult patients with SCD allows for stable mixed hematopoietic chimerism with associated full-donor erythroid engraftment and normalization of blood counts, and persistence in some without continued immunosuppression suggests immunologic tolerance. The attainment of tolerance should allow extension of these potentially curative approaches to alternative donor sources. Efforts to build on these experiences should increase the use of allo-HSCT in patients with SCD while minimizing morbidity and mortality.

journal_name

Blood

journal_title

Blood

authors

Hsieh MM,Fitzhugh CD,Tisdale JF

doi

10.1182/blood-2011-01-332510

subject

Has Abstract

pub_date

2011-08-04 00:00:00

pages

1197-207

issue

5

eissn

0006-4971

issn

1528-0020

pii

blood-2011-01-332510

journal_volume

118

pub_type

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