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Conditioning Regimens

A pilot study of reduced toxicity conditioning with BU, fludarabine and alemtuzumab before the allogeneic hematopoietic SCT in children and adolescents

Bone Marrow Transplantation volume 46, pages 790–799 (2011)Cite this article

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Abstract

We report the results of a pilot study of a BU–fludarabine–alemtuzumab (BFA)-reduced toxicity conditioning (RTC) followed by allogeneic hematopoietic SCT (AlloHSCT) in 12 children and adolescents (<21 years) with malignant and non-malignant diseases. Stem cell sources were: two unrelated cord blood, one unrelated BM, two related and seven unrelated PBSC. Positive CD34 selection was performed in five unrelated PBSC grafts. RCT was carried out with BFA, and GVHD prophylaxis was FK506 and mycophenolate mofetil. The median time for neutrophil and platelet engraftment was 16 and 31 days, respectively. The P of developing ⩾grade II, ⩾grade III aGVHD and cGVHD was 41.6, 25 and 9%, respectively. Only 1 out of 12 developed ⩾grade III toxicity. There was one primary and no secondary graft failure. Mixed donor chimerism on day 100 and 1 year was median 99 and 96%, respectively; ⩾90% of recipients achieved ⩾80% donor chimerism. The 3-year overall survival (OS) in all patients was 91.7±8% (100% for malignant vs 80% for non-malignant diseases, ns). In all, 11 (91%) patients remain alive at median 2.8 (0.3–6.8) years. RTC followed by AlloHSCT, based on BFA conditioning, is feasible and tolerable in children and adolescents, and results in prompt achievement of durable mixed donor chimerism and excellent OS.

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Acknowledgements

This work was supported in part by the Pediatric Cancer Research Foundation, Marisa Fund, Sonia Scaramella Fund, Paul Luisi Foundation, Dream for Discovery and Cure Fund, Brittany Barron Fund (MSC), and by a grant from Children's Medical Care Foundation (JSt).

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Authors and Affiliations

  1. Department of Pediatric Hematology and Oncology, Collegium Medicum UMK, Bydgoszcz, Poland

    J Styczynski

  2. Department of Pediatrics, Columbia University, New York, NY, USA

    J Styczynski, B Tallamy, I Waxman, C van de Ven, L Harrison, E Morris, P Satwani, M Bhatia, D George, M B Bradley, J H Garvin & M S Cairo

  3. Department of Pathology and Laboratory Medicine, University of Pennsylvania Medical Center, Philadelphia, PA, USA

    M C Milone & L M Shaw

  4. Department of Pathology and Cell Biology, Columbia University, New York, NY, USA

    J Schwartz & M S Cairo

  5. Department of Surgery, University of California, San Francisco, CA, USA

    L A Baxter-Lowe

  6. Department of Medicine, Columbia University, New York, NY, USA

    M S Cairo

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Correspondence to M S Cairo.

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Competing interests

LMS is a member of the scientific advisory board of Saladax Biomedical Inc. MBB is Associate Director of Global Clinical Research in Oncology at Bristol-Myers Squibb. Rest of the authors declare no conflict of interest.

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Styczynski, J., Tallamy, B., Waxman, I. et al. A pilot study of reduced toxicity conditioning with BU, fludarabine and alemtuzumab before the allogeneic hematopoietic SCT in children and adolescents. Bone Marrow Transplant 46, 790–799 (2011). https://doi.org/10.1038/bmt.2010.209

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