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Bone Marrow Transplant. 2014 Jul;49(7):913-20. doi: 10.1038/bmt.2014.84. Epub 2014 May 5.

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

Author information

1
Department of Pediatrics, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA.
2
1] Department of Pediatrics, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA [2] Department of Biostatistics, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA.
3
Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
4
Department of Pediatrics, New York Medical College, Valhalla, NY, USA.
5
Department of Pediatrics, Wyckoff Heights Medical Center, New York, NY, USA.
6
Department of Surgery, University of California San Francisco, San Francisco, CA, USA.
7
Department of Pathology, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA.
8
1] Department of Pediatrics, New York Medical College, Valhalla, NY, USA [2] Department of Medicine, New York Medical College, Valhalla, NY, USA [3] Department of Pathology, New York Medical College, Valhalla, NY, USA [4] Department of Microbiology and Immunology, New York Medical College, Valhalla, NY, USA [5] Department of Cell Biology and Anatomy, New York Medical College, Valhalla, NY, USA.

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.

TRIAL REGISTRATION:

ClinicalTrials.gov NCT00408447.

PMID:
24797180
DOI:
10.1038/bmt.2014.84
[Indexed for MEDLINE]
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