Study Reviews Response and Survival Outcomes of Transplant for Waldenström Macroglobulinemia

A study that reviewed response and survival outcomes in Waldenström macroglobulinemia (WM) patients who underwent autologous stem cell transplantation (ASCT) found that ASCT is a viable treatment modality for this population. The study was presented at the 2021 American Society of Hematology Annual Meeting.

The analysis was a retrospective cohort analysis of 32 consecutive WM patients treated with ASCT (median age at time of ASCT = 57 years; 43.7% male) between 2003 and 2020. The researchers collated baseline demographic and biologic data, number and types of prior therapies, and pre- and post-ASCT depth of response using patient records and the WMUK Rory Morrison Registry. The primary outcomes of interest were depth of response, overall survival (OS), progression free survival (PFS), transplant-related mortality (TRM) and relapse-associated mortality.

Disease status pre-ASCT among the study population was complete remission (CR)/very good partial response (VGPR) in 43.7% of patients, and partial response (PR) in 56.2% of patients. Restaging at 100 days post-ASCT showed robust response by ASCT in over half of patients (53.1%). Moreover, CR/VGPR was achieved by most patients (81.2%), and PR by four (12.5%). At a median follow-up of 8.9 years, the estimated median PFS following ASCT was 4.5 years, with estimated two-year and five-year PFS rates of 75% and 35.9%, respectively.

“ASCT is a feasible treatment option for patients with relapsed WM, producing deeper responses following salvage therapy and resulting in PFS intervals comparable to other currently available therapeutic options,” the authors concluded. “With the benefit of a long follow-up period, performing ASCT at later stages in the treatment course (i.e., following 3 or more prior therapy lines) did not appear to result in inferior survival outcomes.” Therefore, they suggest that timing of ASCT should be considered on an individual patient basis and on the basis of other available therapy options for relapsed disease.