Second marrow transplants for graft failure in patients with thalassemia

Thirty-two thalassemic patients with a median age of 7.7 years (range 3.4-2 6 years) were given a second HLA-identical related marrow transplant (BMT2) for graft failure. Four patients were in class 1 and 28 patients in classe s 2 and 3, Twenty-one patients had full thalassemia recurrence (first group ) and 11 patients had aplastic marrows (second group) either with or withou t residual donor marrow cells after the first BMT (BMT1), As conditioning r egimen for BMT2 all but five patients received BUCY or CY in association wi th total lymphoid irradiation (TLI) and/or anti-lymphocyte globulin (ALG), whereas nine patients received a new preparative regimen with hydroxyurea, azathioprine, fludarabine before conditioning with BUCY. Twenty one of 31 e valuable patients (67.7%) had initial, and 16 (51.6%) had sustained engraft ment. Ten patients (32.3%) failed to engraft. Overall and event-free surviv al for the entire group of patients were 49% and 33%, respectively, with a median follow-up of 4 years (range 0.6-14 years) for surviving patients. Ev ent-free survival was higher in the second group of patients compared with the first group (41% vs 29%), The second group of patients appeared to have less graft failure compared with the first group (30% vs 63%; P = 0.1). Tr ansplant-related mortality was 28%, A linear stepwise regression analysis r evealed that occurrence of graft failure within 60 days after BMT1 (P = 0.0 4) and absence of residual donor marrow cells (P = 0.009) predicted for gra ft failure following BMT2, whereas the occurrence of graft failure after 60 days (P = 0.03) had a positive influence on survival following BMT2 The in cidence of grade greater than or equal to 2 acute GVHD was low (14%), Eight of nine patients who received the new preparative regimen are alive, four without thalassemia, This study shows that BMT2 can be an effective therapy for a proportion of patients with poor survival expectancies despite conve ntional treatment.