title: Sustained engraftment post bone marrow transplant despite anti-platelet antibodies in Glanzmann thrombasthenia.
abstract: BACKGROUND
  Patients with Glanzmann thrombasthenia (GT) have normal platelet counts but abnormal platelet aggregation and carry the risk of life-threatening bleeding. We report three patients who received bone marrow transplantation (BMT) for type I GT and discuss the risk and management of anti-platelet antibodies.
  
  
  RESULTS
  Diagnosis of GT was made through abnormal platelet aggregation studies or the absence of GPIIb/IIIa by flow cytometry. All patients had severe bleeding requiring multiple red blood cell transfusions. One patient received an unrelated donor transplant and two received matched sibling donor transplants following conditioning therapy with busulfan, cyclophosphamide, and fludarabine. Two patients developed an anti-platelet antibody, treated in one with intravenous immune globulin (IVIG). Engraftment of white blood cells and platelets was achieved on day +13 to +14 and +17 to +25, respectively. Complete donor chimerism and GPIIb/IIIa+ platelets are sustained at +22 to +30 months post transplant.
  
  
  CONCLUSIONS
  In summary, patients with GT and history of severe hemorrhage can be cured with BMT, but the presence of anti-platelet antibodies should be sought and platelet transfusions minimized prior to transplant. IVIG may be helpful in cases of refractory immune thrombocytopenia related to anti-platelet antibodies. Improvement in transplant-related complications with current transplant regimens allows consideration of BMT for life-threatening non-malignant disorders such as GT.
authors.affiliation: Pediatric Stem cell Transplant Program, Division of Pediatric Hematology/Oncology, Oregon Health & Science University, Portland, Oregon 97239, USA. floodv@ohsu.edu
authors.forename: Veronica H
authors.initials: VH
authors.lastname: Flood
authors.forename: F Leonard
authors.initials: FL
authors.lastname: Johnson
authors.forename: Lynn K
authors.initials: LK
authors.lastname: Boshkov
authors.forename: Gregory A
authors.initials: GA
authors.lastname: Thomas
authors.forename: Diane J
authors.initials: DJ
authors.lastname: Nugent
authors.forename: Antony C
authors.initials: AC
authors.lastname: Bakke
authors.forename: H Stacy
authors.initials: HS
authors.lastname: Nicholson
authors.forename: David
authors.initials: D
authors.lastname: Tilford
authors.forename: Mary P
authors.initials: MP
authors.lastname: Brown
authors.forename: Kamar T
authors.initials: KT
authors.lastname: Godder
mesh_terms: D001323:Autoantibodies / Q000097:blood / Q000276:immunology; D001792:Blood Platelets / Q000276:immunology; D016026:Bone Marrow Transplantation / Q000379:methods; D002648:Child; D002675:Child, Preschool; D005260:Female; D006085:Graft Survival / Q000187:drug effects; D006801:Humans; D016756:Immunoglobulins, Intravenous / Q000008:administration & dosage; D007155:Immunologic Factors / Q000008:administration & dosage; D007223:Infant; D008297:Male; D019653:Myeloablative Agonists / Q000008:administration & dosage; D010974:Platelet Aggregation; D010976:Platelet Count / Q000379:methods; D019039:Platelet Glycoprotein GPIIb-IIIa Complex / Q000378:metabolism; D013915:Thrombasthenia / Q000097:blood / Q000276:immunology / Q000628:therapy*; D018183:Transplantation Chimera / Q000097:blood / Q000276:immunology; D019172:Transplantation Conditioning / Q000379:methods
chemical_list: D001323:Autoantibodies; D016756:Immunoglobulins, Intravenous; D007155:Immunologic Factors; D019653:Myeloablative Agonists; D019039:Platelet Glycoprotein GPIIb-IIIa Complex