MARROW TRANSPLANTATION FOR PATIENTS WITH THALASSEMIA - RESULTS IN CLASS 3 PATIENTS

Thalassemia patients can be categorized as class 1 (minimal liver dama ge and iron overload), class 3 (extensive liver damage from iron overl oad), and class 2 (intermediate). These categories are prognostic for treatment outcome after marrow transplantation. Class 3 patients have more transplant-related mortality than other patients. This study exam ines transplantation outcome for class 3 patients, Records were review ed of 215 patients in class 3 who received transplants in Pesaro from HLA-identical related donors between May 1, 1984 and May 1, 1994. The influence of pretransplant, peritransplant, and post-transplant variab les on survival, relapse, and transplant-related mortality was examine d by product-limit and proportional-hazards multivariate analysis, Age and conditioning regimen were influential on survival, and regimens w ith less than 200 mg/kg cyclosporine (CY) were associated with 5-year survival probabilities of .74 and .63 in patients younger than 17 year s and older patients, respectively, Transfusion history and regimen we re influential on rejection, with 5-year probabilities of .53 and .24 in patients who received less than or greater than 100 red blood cell transfusions before transplantation and regimens containing less than 200 mg/kg CY, Results of transplantation for patients with advanced th alassemia treatment have improved with the introduction of conditionin g regimens with less CY, This has been associated with an increase in rejection (particularly in patients who have received <100 red blood c ell transfusions before transplant), Efforts at reducing the rejection rate by modifying the conditioning regimen should be concentrated on younger patients who have received a small number of transfusions. Pat ients with thalassemia who have HLA-identical family members should be transplanted before they are in class 3. (C) 1996 by The American Soc iety of Hematology.