| Title | Successful unrelated donor cord blood transplantation for Glanzmann's thrombasthenia. | | Author(s) | Kitko CL, Levine JE, Matthews DC, Carpenter PA | | Institution | Department of Pediatrics and Internal Medicine, Blood and Marrow Transplant Program, University of Michigan, Ann Arbor, MI. | | Source | Pediatr Transplant 2009 Oct 22. | | Abstract | Kitko CL, Levine JE, Matthews DC, Carpenter PA. Successful unrelated donor cord blood transplantation for Glanzmann's thrombasthenia. Pediatr Transplantation 2009. (c) 2009 John Wiley & Sons A/S.Abstract: GT, a rare disorder of platelet function, can lead to life-threatening bleeding, particularly following the development of antiplatelet antibodies. Curative therapy includes HCT but previous reports are limited predominantly to matched siblings and have excluded CBT. Delayed or non-engraftment of platelets because of antiplatelet antibodies might be particularly concerning after CBT for GT. Here, we report two successful unrelated cord blood transplants for GT. Recurrent life-threatening bleeding was the primary indication for HCT, with one patient developing antiplatelet antibodies pre-HCT. Bleeding risks associated with delivery of the conditioning regimen and the toxicity that follows should be carefully considered, including tunneled central venous line catheter placement, inclusion of B cell-specific therapy to potentially decrease antiplatelet antibody production, and targeted busulfan dosing. This is the first report of successful unrelated cord blood HCT for GT and indicates that modifications to supportive care can improve the safety of this potentially curative therapy for patients with severe, life-threatening disease manifestations. | | Language | ENG | | Pub Type(s) | JOURNAL ARTICLE
| | PubMed ID | 19849809 |
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