Bone marrow transplantation in thalassemia

Hematol Oncol Clin North Am. 1991 Jun;5(3):549-56.

Abstract

Since 1983, 350 patients aged 1 to 19 years with beta-homozygous thalassemia were given infusions of HLA-identical marrow after high doses of busulphan and cyclophosphamide. Survival and event-free survival leveled off about 1 year after bone marrow transplantation at 82% and 75%, respectively. In 172 consecutive patients who were treated with our current regimen since June 1985, a multivariate analysis demonstrated that portal fibrosis, hepatomegaly, and a history of inadequate chelation therapy were significantly associated with reduced probabilities of survival and event-free survival. The patients were divided into three classes on the basis of the presence of hepatomegaly, portal fibrosis, and inadequate chelation therapy. Class 1 had none of the factors and class 3 had all three factors; class 2 had different associations of two out of the three factors. For class 1 patients, the 3-year probabilities of survival and event-free survival were 97% and 94%, respectively. For class 2 patients, the probabilities were 86% and 83%, and for class 3 patients, 58% and 52%. Bone marrow transplantation from HLA-identical donors is followed by a high probability of event-free survival in thalassemic patients, particularly if they belong to class 1.

MeSH terms

  • Adolescent
  • Adult
  • Bone Marrow Transplantation*
  • Busulfan / therapeutic use
  • Child
  • Child, Preschool
  • Cyclophosphamide / therapeutic use
  • Fibrosis
  • Graft Rejection
  • Graft vs Host Disease / etiology
  • Hepatomegaly
  • Humans
  • Infant
  • Portal Vein / pathology
  • Survival Rate
  • Thalassemia / complications
  • Thalassemia / mortality
  • Thalassemia / surgery*

Substances

  • Cyclophosphamide
  • Busulfan