Allogeneic stem cell transplantation for Glanzmann thrombasthenia.
Glanzmann thrombasthenia (GT) is a rare autosomal recessive bleeding disorder characterized by normal platelet count, but lack of platelet aggregation. The molecular basis is linked to quantitative and/or qualitative abnormalities of the membrane glycoprotein IIb/IIIa complexes. Usually it is associated with mild bleeding but may lead to severe and potentially fatal hemorrhages. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment. However, because of the risks associated with HSCT, it is generally not recommended unless there are life threatening hemorrhages, or the patient has developed refractoriness to platelet transfusion due to antibody formation. Herein, we report an 11-year-old female from United Arab Emirates (UAE) with severe GT and anti platelet alloimmunization successfully treated with HSCT from her HLA-identical sibling.
Ishaqi
M Kashif
MK
Division of Pediatric Hematology/Oncology, Tawam Hospital in Affiliation with Johns Hopkins Medicine, Al-Ain, United Arab Emirates. kishaqi@hotmail.com
El-Hayek
M
M
Gassas
Adam
A
Khanani
M
M
Trad
Omar
O
Baroudi
M
M
Jamil
A
A
D002648:Child; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D033581:Stem Cell Transplantation; D013915:Thrombasthenia / Q000601:surgery*; D014184:Transplantation, Homologous