Sustained engraftment post bone marrow transplant despite anti-platelet antibodies in Glanzmann thrombasthenia. Pediatr Blood Cancer 2005 Dec;45(7):971-5
Date
03/16/2005Pubmed ID
15768382DOI
10.1002/pbc.20365Scopus ID
2-s2.0-27644524098 (requires institutional sign-in at Scopus site) 33 CitationsAbstract
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.
PATIENTS AND 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.
Author List
Flood VH, Johnson FL, Boshkov LK, Thomas GA, Nugent DJ, Bakke AC, Nicholson HS, Tilford D, Brown MP, Godder KTAuthor
Veronica H. Flood MD Chief, Professor in the Pediatrics department at Medical College of WisconsinMESH terms used to index this publication - Major topics in bold
AutoantibodiesBlood Platelets
Bone Marrow Transplantation
Child
Child, Preschool
Female
Graft Survival
Humans
Immunoglobulins, Intravenous
Immunologic Factors
Infant
Male
Myeloablative Agonists
Platelet Aggregation
Platelet Count
Platelet Glycoprotein GPIIb-IIIa Complex
Thrombasthenia
Transplantation Chimera
Transplantation Conditioning
