Data from a real-world analysis on the role of autologous stem cell transplantation (ASCT) in Waldenström macroglobulinemia (WM) was presented at the 2021 American Society of Hematology (ASH) Annual Meeting to review response and survival outcomes, and relapse risk factors, in patients with WM who underwent ASCT.

“It remains unclear where ASCT should be placed in the sequence of treatment lines. The debate is stronger in the era of targeted therapies that can achieve prolonged progression free survival (PFS) intervals and expected treatment-free intervals of approximately 4 to 8 years,” explained Suzanne O. Arulogun, MBBS, FRACP, FRCPA, University College London Hospital NHS Foundation Trust, London, United Kingdom, and colleagues.

The primary aims of the retrospective cohort analysis were to determine the depth of response (DOR), overall survival (OS), PFS, transplant related mortality (TRM), and relapse-associated mortality.

Of the 32 patients that received ASCT, the median age was 57 years (range 40-68 years), and the median interval from diagnosis to ASCT was 2.3 years (range 0.5-16.8 years).

“At a median follow up of 8.9 years (range 0.1-18 years), the estimated median PFS was 4.5 years (95% confidence interval [CI] 3.2-5.7 years), with estimated 2-year and 5-year PFS rates of 75 percent and 35.9 percent, respectively. In this small cohort, there was no significant difference in PFS based on age, number of prior lines of treatment, pre-ASCT remission status (CR/VGPR vs PR) or post-ASCT response achieved,” explained Dr Arulogun and colleagues.

Dr Arulogun and colleagues conclude that ASCT is a feasible treatment option for patients with relapsed WM, which produces deeper responses following salvage therapy. Further, it results in PFS intervals comparable to other therapeutic options.

“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; timing of ASCT should therefore be considered on an individual patient basis and in light of other available therapy options for relapsed disease,” concluded Dr Arulogun and colleagues.

Suzanne A, Kyriakou C, Horder J, et al. Autologous Haematopoietic Stem Cell Transplantation in Waldenström Macroglobulinemia: A Single-Centre 18-Year Experience. Presented at: the 2021 ASH Annual Meeting; Dec. 11-14; 2021; Abstract 3548.