Successful unrelated donor cord blood transplantation for Glanzmann's thrombasthenia.
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.
Department of Pediatrics and Internal Medicine, Blood and Marrow Transplant Program, University of Michigan, Ann Arbor, MI, USA.
Carrie L
CL
Kitko
John E
JE
Levine
Dana C
DC
Matthews
Paul A
PA
Carpenter
D001791:Blood Platelet Disorders / Q000628:therapy; D001792:Blood Platelets / Q000166:cytology; D002405:Catheterization, Central Venous; D057785:Catheters; D002648:Child; D002675:Child, Preschool; D005312:Fetal Blood / Q000166:cytology*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006470:Hemorrhage / Q000517:prevention & control; D006801:Humans; D008297:Male; D019039:Platelet Glycoprotein GPIIb-IIIa Complex / Q000276:immunology; D013915:Thrombasthenia / Q000628:therapy*; D016896:Treatment Outcome
D019039:Platelet Glycoprotein GPIIb-IIIa Complex