학술논문
Stem Cell Transplantation in Glanzmann's Thrombasthenia: A Report of Two Adult Patients
CASE REPORT
CASE REPORT
Document Type
Clinical report
Author
Source
Journal of the College of Physicians and Surgeons Pakistan. December 2022, Vol. 32 Issue 12, p1626, 3 p.
Subject
Language
English
ISSN
1022-386X
Abstract
INTRODUCTION Glanzmann's Thrombasthenia (GT) is a rare congenital bleeding disorder caused by a defect in the platelet surface receptor glycoprotein (GP) IIb/IIIa complex (ITG [alpha]IIb[beta]IIIa). It is characterised by mucocutaneous [...]
Glanzmann's thrombasthenia (GT) is an autosomal recessive bleeding disorder characterised by mucocutaneous bleeding. At molecular level, defect in platelet receptor glycoprotein (GP) IIb/IIIa leads to defective platelet aggregation. Anti- fibrinolytic agents, platelet transfusions, and factor rVIIa are used for prophylaxis before invasive procedures and treatment of bleeding events. Allogeneic stem cell transplant is the only curative option. Here, we report cases of two adult male patients who underwent matched sibling donor stem cell transplantation for GT with recurrent bleeding requiring platelet and red cell transfusions. Both showed marked improvement in quality of life. To conclude, stem cell transplant is a viable treatment option for severe, difficult-to-control cases of GT. Key Words: Platelet disorders, Hematopoietic stem cell transplantation, Thrombasthenia.
Glanzmann's thrombasthenia (GT) is an autosomal recessive bleeding disorder characterised by mucocutaneous bleeding. At molecular level, defect in platelet receptor glycoprotein (GP) IIb/IIIa leads to defective platelet aggregation. Anti- fibrinolytic agents, platelet transfusions, and factor rVIIa are used for prophylaxis before invasive procedures and treatment of bleeding events. Allogeneic stem cell transplant is the only curative option. Here, we report cases of two adult male patients who underwent matched sibling donor stem cell transplantation for GT with recurrent bleeding requiring platelet and red cell transfusions. Both showed marked improvement in quality of life. To conclude, stem cell transplant is a viable treatment option for severe, difficult-to-control cases of GT. Key Words: Platelet disorders, Hematopoietic stem cell transplantation, Thrombasthenia.