¹University of Illinois at Chicago, Chicago, IL, USA

Correspondence: Dr K van Besien, Section of Hematology/Oncology, University of Chicago, Rm I 209, MC 2115, 5841 South Maryland Avenue, Chicago, IL, 60637-1470, USA

Received 21 January 2003; Accepted 25 March 2003.

Abstract

A total of 31 consecutive patients with hematologic malignancies who were considered poor candidates for TBI underwent allogeneic stem cell transplantation after conditioning with fludarabine and melphalan. A total of 25 matched sibling recipients received fludarabine 25 mg/m² 5 days and melphalan 70 mg/m² 2 days. For unrelated and haploidentical donor recipients, fludarabine was increased to 30 mg/m² and ATG 30 mg/kg 4 days was added. Graft-versus-host disease prophylaxis consisted of tacrolimus and mini methotrexate. All patients engrafted. Regimen-related toxicity was considerable and included mainly renal, hepatic and mucosal toxicity. There were seven regimen-related-deaths including two VOD, two pulmonary, one renal, one cardiac and one mucosal toxicity. One case of fatal pulmonary toxicity death could be attributed to pre-existing pulmonary damage. Progression-free survival at 12 months was 44% (90% CI: 30–58%) for recipients of HLA-identical sibling transplants and 33% (90% CI: 21–45%) for all patients. In conclusion, the fludarabine–melphalan regimen leads to consistent engraftment. The regimen-related toxicity is considerable and cannot be explained solely by patient selection. Cardiac toxicity is emerging as a unique toxicity of this regimen. Despite toxicity, fludarabine–melphalan has considerable activity and leads to durable remission in a proportion of patients.