The role of autologous hematopoietic stem cell transplantation in multiple myeloma

Abstract

In the past 10 years, a number of uncontrolled studies have shown that autologous transplantation is a useful salvage therapy for refractory and relapsed multiple meyloma (MM) and a safe and effective consolidation for patients responding to initial conventional chemotherapy. However, because of selection bias in all these studies, the exact role of autologous transplant in the management of newly diagnosed patients was not known. In 1990, the Intergroupe Français du Myelome (IFM) initiated a randomized study comparing conventional chemotherapy and high-dose therapy (HDT) followed by autologous bone marrow transplantation. Two hundred patients under the age of 65 years were randomly assigned at diagnosis to receive either conventional chemotherapy or autologous bone marrow transplantation. In the conventional chemotherapy arm, 14% of patients achieved complete remission (CR) or very good partial remission (VGPR) as compared with 38% in the transplantation arm. The 5-year event-free survival (EFS) and overall survival (OS) were, respectively, 10% and 12% in the chemotherapy arm and 28% and 52% in the transplantation arm. This intention-to-treat analysis demonstrates that HDT represents a significant improvement and should be proposed as part of front-line therapy to younger patients. However, several issues regarding the indications and modalities of HDT remain and are discussed.