NYMC Faculty Publications

Reduced Toxicity, Myeloablative Conditioning With BU, Fludarabine, Alemtuzumab and SCT From Sibling Donors in Children With Sickle Cell Disease

Author Type(s)

Faculty

DOI

10.1038/bmt.2014.84

Journal Title

Bone Marrow Transplantation

First Page

913

Last Page

920

Document Type

Article

Publication Date

7-1-2014

Department

Pediatrics

Keywords

Adolescent, Adult, Alemtuzumab, Anemia, Sickle Cell, Antibodies, Monoclonal, Humanized, Antineoplastic Combined Chemotherapy Protocols, Busulfan, Child, Child, Preschool, Female, Hematopoietic Stem Cell Transplantation, Humans, Male, Pilot Projects, Prospective Studies, Siblings, Tissue Donors, Transplantation Chimera, Transplantation Conditioning, Vidarabine, Young Adult

Disciplines

Medicine and Health Sciences

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.

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