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


Journal Article

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.

Full Text

Duke Authors

Cited Authors

  • Mahadeo, KM; Tewari, P; Parikh, SH; Driscoll, TA; Page, K; Martin, PL; Kurtzberg, J; Prasad, VK

Published Date

  • November 2015

Published In

Volume / Issue

  • 19 / 7

Start / End Page

  • 753 - 757

PubMed ID

  • 26369627

Pubmed Central ID

  • 26369627

Electronic International Standard Serial Number (EISSN)

  • 1399-3046

Digital Object Identifier (DOI)

  • 10.1111/petr.12577


  • eng

Conference Location

  • Denmark