Reduced intensity transplantation for congenital amegakaryocytic thrombocytopenia: report of a case and review of the literature.

Abstract

CAMT is a bone marrow failure syndrome that usually presents with isolated thrombocytopenia soon after birth. HSCT is curative, and MAC is associated with increased transplant-related morbidity and mortality, especially in the unrelated setting. We used a RIC regimen with alemtuzumab, fludarabine, and melphalan in a seven-month-old patient with CAMT who underwent a MUD HSCT. The transplant was well tolerated with few complications. Neutrophil and platelet engraftment occurred on day +12 and +29, respectively, and she had 100% donor chimerisms on days +19.

• Authors: Gary Woods, Rajinder P S Bajwa, Melissa J Rose
• Journal: Pediatric transplantation (Pediatr Transplant) Vol. 18 Issue 1 Pg. E31-4 (Feb 2014) ISSN: 1399-3046 [Electronic] Denmark
• PMID: 24119002 (Publication Type: Case Reports, Journal Article, Review)
• Copyright: © 2013 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.

Chemical References

• Antibodies, Monoclonal, Humanized
• Alemtuzumab
• Vidarabine
• fludarabine
• Melphalan
• Methylprednisolone

Topics

• Alemtuzumab
• Antibodies, Monoclonal, Humanized (administration & dosage)
• Blood Platelets (cytology)
• Congenital Bone Marrow Failure Syndromes
• Female
• Hematopoietic Stem Cell Transplantation
• Hepatic Veno-Occlusive Disease (complications)
• Humans
• Infant
• Melphalan (administration & dosage)
• Methylprednisolone (administration & dosage)
• Mucositis (complications)
• Neutrophils (cytology)
• Thrombocytopenia (therapy)
• Transplantation Conditioning (methods)
• Vidarabine (administration & dosage, analogs & derivatives)