title: Unrelated bone marrow transplant for congenital amegakaryocytic thrombocytopenia: report of two cases and review of the literature.
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.
authors.affiliation: Pediatric Hematology/Oncology, Vanderbilt University, Nashville, TN 37232-6310, USA. haydar.frangoul@vanderbilt.edu
authors.forename: Haydar
authors.initials: H
authors.lastname: Frangoul
authors.forename: Jennifer
authors.initials: J
authors.lastname: Keates-Baleeiro
authors.forename: Cassie
authors.initials: C
authors.lastname: Calder
authors.forename: Becky
authors.initials: B
authors.lastname: Manes
authors.forename: Christine
authors.initials: C
authors.lastname: Crossno
authors.forename: Victoria L
authors.initials: VL
authors.lastname: Castaneda
authors.forename: Jennifer
authors.initials: J
authors.lastname: Domm
mesh_terms: D016026:Bone Marrow Transplantation / Q000009:adverse effects; D005260:Female; D006801:Humans; D007223:Infant; D008533:Megakaryocytes; D013921:Thrombocytopenia / Q000151:congenital* / Q000628:therapy*