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

Kris M. Mahadeo, Priti Tewari, Suhag H. Parikh, Timothy A. Driscoll, Kristin Page, Paul L. Martin, Joanne Kurtzberg, Vinod K. Prasad

Research output: Contribution to journalArticle

5 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)753-757
Number of pages5
JournalPediatric Transplantation
Issue number7
StatePublished - 2015
Externally publishedYes



  • congenital amegakaryocytic thrombocytopenia
  • cord blood transplantation
  • hematopoietic stem cell transplantation
  • thrombocytopenia

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Transplantation

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