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Durable engraftment and correction of hematological abnormalities in children with congenital amegakaryocytic thrombocytopenia following myeloablative umbilical cord blood transplantation
Author(s) -
Mahadeo Kris M.,
Tewari Priti,
Parikh Suhag H.,
Driscoll Timothy A.,
Page Kristin,
Martin Paul L.,
Kurtzberg Joanne,
Prasad Vinod K.
Publication year - 2015
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/petr.12577
Subject(s) - medicine , umbilical cord , curative treatment , umbilical cord blood transplantation , transplantation , surgery , hematopoietic stem cell transplantation , disease , immunology
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 Gv HD . 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 .