Successful haploidentical hematopoietic stem cell transplantation for Glanzmann’s thrombasthenia: First report from northern India

Glanzmann thrombasthenia (GT) is a rare inherited platelet disorder that leads to lifelong mucocutaneous bleeding and transfusion dependence. We report a 15-year-old boy with severe, transfusion-refractory GT who underwent haploidentical hematopoietic stem cell transplantation (HSCT) using his 18-year-old brother as donor. Baseline studies showed absent platelet aggregation with adenosine diphosphate, collagen, and epinephrine; preserved response to ristocetin; and markedly reduced expression of glycoprotein IIb/IIIa. Conditioning consisted of fludarabine, busulfan, and cyclophosphamide, followed by infusion of peripheral blood stem cells. Neutrophils and platelets engrafted on days 14 and 18, respectively, with donor chimerism exceeding 99% by day 30. Early complications included grade 3 mucositis and mild cutaneous graft-versus-host disease, both controlled conservatively. At 7 months, the patient remains well and transfusion-independent, with normalizing platelet aggregation and fibrinogen levels. This case demonstrates that haploidentical HSCT is a feasible and curative option for GT when a matched donor is not available.