Unrelated bone marrow transplant for congenital amegakaryocytic thrombocytopenia: Report of two cases and review of the literature

Haydar Frangoul, Jennifer Keates-Baleeiro, Cassie Calder, Becky Manes, Christine Crossno, Victoria L. Castaneda, Jennifer Domm

Research output: Contribution to journalArticlepeer-review

Abstract

CAMT is a very rare cause of thrombocytopenia in infants. Most of the patients will progress to marrow failure. Allogeneic stem cell transplant remains the only curative therapy. We present two patients with CAMT who underwent an unrelated donor bone marrow transplant, one after developing marrow failure and another early in the course of the disease. Both patients tolerated the transplant with minimal toxicity and durable engraftment. We also present a comprehensive review of the literature for unrelated donor transplant for this condition.

Original languageEnglish (US)
Pages (from-to)E42-E45
JournalPediatric transplantation
Volume14
Issue number4
DOIs
StatePublished - Jun 2010
Externally publishedYes

Keywords

  • Congenital amegakaryocytic thrombocytopenia
  • Pediatrics
  • Unrelated donor bone marrow transplantation

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Transplantation

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