Bone marrow transplantation in severe Glanzmann's thrombasthenia with antiplatelet alloimmunization.
Glanzmann's thrombasthenia is an autosomal recessive disorder characterized by a lack of platelet aggregation due to the absence of platelet glycoprotein IIb and IIIa. Usually, the disease leads to mild hemorrhage but sometimes bleeding is severe enough to be life-threatening. We report the case of a 16-year-old girl, presenting with very severe type 1 Glanzmann's thrombasthenia, successfully treated with an HLA-identical sibling bone marrow transplant (BMT). We also update the clinical and laboratory data of her brother, who had received a BMT 16 years ago for the same disease. In the light of these two cases and two others published in the literature, we discuss the indications for BMT from HLA-identical sibling donors in Glanzmann's thrombasthenia. Alloimmunization against the missing platelet GPIIb/IIIa complex and severity of bleeding episodes may constitute sufficient criteria for allogeneic BMT after careful assessment of the risk-benefit of such a procedure, although this remains exceptional in this disease. Bone Marrow Transplantation (2000) 25, 327-330.
Bellucci
S
S
Laboratory of Hematology, Hôpital Lariboisière, France.
Damaj
G
G
Boval
B
B
Rocha
V
V
Devergie
A
A
Yacoub-Agha
I
I
Garderet
L
L
Ribaud
P
P
Traineau
R
R
Socié
G
G
Gluckman
E
E
D000293:Adolescent; D001792:Blood Platelets / Q000276:immunology; D016026:Bone Marrow Transplantation; D002648:Child; D005260:Female; D006680:HLA Antigens / Q000276:immunology; D006470:Hemorrhage; D006801:Humans; D007518:Isoantibodies / Q000097:blood; D008297:Male; D009679:Nuclear Family; D010375:Pedigree; D019039:Platelet Glycoprotein GPIIb-IIIa Complex / Q000276:immunology; D013915:Thrombasthenia / Q000097:blood / Q000276:immunology / Q000628:therapy*
D006680:HLA Antigens; D007518:Isoantibodies; D019039:Platelet Glycoprotein GPIIb-IIIa Complex