Complete correction of Glanzmann's thrombasthenia by allogeneic bone-marrow transplantation

Br J Haematol. 1985 Apr;59(4):635-41. doi: 10.1111/j.1365-2141.1985.tb07358.x.

Abstract

Allogeneic bone marrow transplantation (BMT) successfully corrected type I thrombasthenia in a 4-year-old boy. The donor was his HLA-A, B and D identical 14-year-old brother who was heterozygous for thrombasthenia. A first transplant after conditioning with cyclophosphamide and thoracoabdominal irradiation was rejected, but a second transplant using CCNU, cyclophosphamide, procarbazine and horse antihuman thymocyte globulin in the preparative regimen was successful. Engraftment was proven by studies of platelet membrane antigens, PLA1 and glycoprotein IIb/IIIa complex and by platelet function studies. Haemorrhagic manifestations completely disappeared; platelet membrane markers and clot retraction returned promptly to normal values, and platelet aggregation tests more slowly. Twenty-four months after bone-marrow transplant, the patient was well with mild chronic hepatic graft versus host disease. BMT therefore appears to be a possible treatment for severe inherited platelet disorders.

Publication types

  • Case Reports

MeSH terms

  • Blood Platelet Disorders / complications
  • Blood Platelet Disorders / genetics
  • Blood Platelet Disorders / therapy*
  • Bone Marrow Transplantation*
  • Child, Preschool
  • Hemorrhage / etiology
  • Hemorrhage / prevention & control
  • Humans
  • Male
  • Platelet Aggregation
  • Platelet Count