Glanzmann thrombasthenia- Allogeneic hematopoietic stem cell transplantation

Allogeneic hematopoietic stem cell transplantation has been reported in several patients with Glanzmann thrombasthenia.

The first was a 5-year-old male who had several severe gastrointestinal hemorrhages.195 His bleeding diathesis was cured and he was alive and well, but with mild graft-versus-host disease, 16 years after the transplant.196 The second patient, who required multiple hospital admissions to control bleeding, but who only received a platelet transfusion once, was transplanted at age 2.5 years from an HLA-identical sibling who was heterozygous for Glanzmann thrombasthenia.197 She was well 19 months after the transplant. The third patient was transplanted at age 5 years with marrow from a sibling and did well.198 The fourth patient, the sister of the first patient, was age 16 years at the time of transplantation and also had an uneventful course.196 Success has also been reported using an unrelated donor199 and reduced intensity conditioning regimens (three patients),200 and in a recipient with antiplatelet antibodies, including antibodies to IIb3.199,201 Nonmyeloablative marrow transplantation has been performed successfully in a dog model of Glanzmann thrombasthenia.202 In utero transplantation at 16 weeks of gestation of fetal liver cells from a 16-week fetus led to platelet alloantigen chimerism 3 weeks later (at the time of pregnancy termination), supporting the potential of such therapy for treating fetuses with Glanzmann thrombasthenia.203