Bone marrow transplantation for Glanzmann's thrombasthenia.
Allogeneic matched bone marrow transplantation (BMT) was performed in a patient with type I Glanzmann's thrombasthenia, a rare, inherited bleeding disorder caused by a deficiency in the platelet membrane glycoprotein IIb-IIIa complex. The patient was a 2-year-old girl with a history of frequent hospitalisation. She was successfully transplanted with BM from her HLA-identical sibling. Engraftment was monitored by analysis of the platelet GPIIb-IIIa complex and by RFLP analysis using a minisatellite probe. Complete engraftment was seen at day +25. The patient has been clinically stable for 19 months. It is proposed that BMT is a suitable treatment for this condition where a matched, related donor is available and at an early stage, before the development of anti-platelet antibodies as a result of repeated transfusions.
Bone Marrow Transplant Unit, Westminster Children's Hospital, London, UK.
A
A
Johnson
A H
AH
Goodall
C J
CJ
Downie
A
A
Vellodi
D P
DP
Michael
D001792:Blood Platelets / Q000378:metabolism; D016026:Bone Marrow Transplantation; D002675:Child, Preschool; D005260:Female; D006579:Heterozygote; D006720:Homozygote; D006801:Humans; D010641:Phenotype; D010980:Platelet Membrane Glycoproteins / Q000235:genetics / Q000378:metabolism; D012150:Polymorphism, Restriction Fragment Length; D013915:Thrombasthenia / Q000097:blood / Q000235:genetics / Q000628:therapy*; D014019:Tissue Donors; D014184:Transplantation, Homologous
D010980:Platelet Membrane Glycoproteins