Durable engraftment and correction of hematological abnormalities in children with congenital amegakaryocytic thrombocytopenia following myeloablative umbilical cord blood transplantation.

Abstract

The use of HSCT is the only potentially curative treatment for CAMT, but access is limited by the availability of suitable donors. We report five consecutive patients with CAMT who received MAC and partially HLA-mismatched, UCBT (unrelated, n = 4). Median times to neutrophil (>500/μL) and platelet (≥20 000 and ≥50 000/μL) engraftment were 19, 57, and 70 days, respectively. Acute GvHD, grade II, developed in one patient, who subsequently developed limited chronic GvHD. At median follow-up of 14 yr, all patients are alive with sustained donor cell engraftment. To our knowledge, this is the largest single-center series of UCBT for patients with this disease and suggests that UCBT is a successful curative option for patients with CAMT.

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Citation

Published Version (Please cite this version)

10.1111/petr.12577

Publication Info

Mahadeo, Kris M, Priti Tewari, Suhag H Parikh, Timothy A Driscoll, Kristin Page, Paul L Martin, Joanne Kurtzberg, Vinod K Prasad, et al. (2015). Durable engraftment and correction of hematological abnormalities in children with congenital amegakaryocytic thrombocytopenia following myeloablative umbilical cord blood transplantation. Pediatric transplantation, 19(7). pp. 753–757. 10.1111/petr.12577 Retrieved from https://hdl.handle.net/10161/24646.

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Scholars@Duke

Mahadeo

Kris Mahadeo

Professor of Pediatrics
Driscoll

Timothy Alan Driscoll

Assistant Professor of Pediatrics

Dr. Driscoll participates in multi-institutional studies for the treatment of high risk neuroblastoma patients using high dose chemotherapy with stem cell transplant and the development of new therapies for high risk neuroblastoma patients.


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