High-dose therapy and autologous bone marrow transplantation for follicular lymphoma in first complete or partial remission: results of a phase II clinical trial

Advanced stage follicular small cleaved and mixed cell lymphoma is characte rized by relapse from remission and survival ranging from 6 to 12 years. Be cause young patients have the greatest compromise in longevity, the efficac y and toxicity of high-dose radiochemotherapy and bone marrow transplantati on after conventional chemotherapy was evaluated in a prospective phase II clinical trial. Thirty-seven patients in a minimal disease state after conv entional chemotherapy received fractionated total body irradiation and high -dose etoposide and cyclophosphamide, followed by purged autologous bone ma rrow. A reference sample of 188 patients of similar age, stage, and histolo gy managed at this institution before 1988 was identified for comparison of patient characteristics and outcomes, Compared with reference patients, tr ansplant recipients had a higher tumor burden at diagnosis. With a median f ollow-up of 6.5 years, the estimated 10-year survival after transplantation was 86%, There was a single lymphoma death yielding a 10-year disease-spec ific survival of 97%, There were 2 early transplant-related deaths and 2 la te acute leukemia deaths. Ten patients relapsed, one with microscopic disea se only. High tumor burden at diagnosis and incomplete response to chemothe rapy adversely influenced survival in the reference but not in the transpla nted patients. The estimated risk of death of 14% and relapse of 30% at 10 years in our transplanted follicular lymphoma patients, the majority of who m had high tumor burdens, compares favorably with our observations in appro priately matched reference patients. (C) 2001 by The American Society of He matology.