Reduced toxicity, myeloablative conditioning with BU, fludarabine, alemtuzumab and SCT from sibling donors in children with sickle cell disease.

Abstract:

:BU and CY (BU/CY; 200 mg/kg) before HLA-matched sibling allo-SCT in children with sickle cell disease (SCD) is associated with ~85% EFS but is limited by the acute and late effects of BU/CY myeloablative conditioning. Alternatives include reduced toxicity but more immunosuppressive conditioning. We investigated in a prospective single institutional study, the safety and efficacy of a reduced-toxicity conditioning (RTC) regimen of BU 12.8-16 mg/kg, fludarabine 180 mg/m(2), alemtuzumab 54 mg/m(2) (BFA) before HLA-matched sibling donor transplantation in pediatric recipients with symptomatic SCD. Eighteen patients, median age 8.9 years (2.3-20.2), M/F 15/3, 15 sibling BM and 3 sibling cord blood (CB) were transplanted. Mean whole blood and erythroid donor chimerism was 91% and 88%, at days +100 and +365, respectively. Probability of grade II-IV acute GVHD was 17%. Two-year EFS and OS were both 100%. Neurological, pulmonary and cardiovascular function were stable or improved at 2 years. BFA RTC and HLA-matched sibling BM and CB allo-SCT in pediatric recipients result in excellent EFS, long-term donor chimerism, low incidence of GVHD and stable/improved organ function.

journal_name

Bone Marrow Transplant

authors

Bhatia M,Jin Z,Baker C,Geyer MB,Radhakrishnan K,Morris E,Satwani P,George D,Garvin J,Del Toro G,Zuckerman W,Lee MT,Licursi M,Hawks R,Smilow E,Baxter-Lowe LA,Schwartz J,Cairo MS

doi

10.1038/bmt.2014.84

subject

Has Abstract

pub_date

2014-07-01 00:00:00

pages

913-20

issue

7

eissn

0268-3369

issn

1476-5365

pii

bmt201484

journal_volume

49

pub_type

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