Survival trends in young patients with Waldenström macroglobulinemia: Over five decades of experience

Abstract

Waldenström macroglobulinemia (WM) is a rare, indolent lymphoma, that predominately affects the elderly. We report the outcomes of young WM patients, evaluated over five decades, compared to their older counterparts, matched for the time of diagnosis. Between January 1, 1960 and October 31, 2013, 140 (11.8%) WM patients were ≤50 years of age at diagnosis in our database, and their estimated 10‐year overall survival (OS) was 74%, with death attributable to WM in a higher proportion of patients compared to their older (≥65 years) counterparts (91% vs. 58%, p = .0001). Young patients were grouped into three cohorts based on the timing of the initiation of therapy: Group 1 (1960–1977, n = 12), Group 2 (1978–1995, n = 48), and Group 3 (1996–2013, n = 74). Among young patients, there was no disease‐specific survival (DSS) difference across the three periods, [median DSS at 13 years (95% CI 5–23), 16 years (95% CI 14–22), and 15 years (95% CI 10‐NR; p = .41), respectively]. However, DSS for the older cohort incrementally improved (Group 1, median 5.2 years, Group 2: 9.6 years, Group 3: 12 years; p = .05) over these periods. The estimated average years‐of‐life lost for the young cohort was 11.2 years from diagnosis, based on the expected survival for a normal age‐ and sex‐matched population. Despite a protracted disease course, nearly all young patients succumb to their disease. In contrast to the improved survival of the elderly patient population, the evolving treatment strategies in WM have not impacted the outcome of young patients; however, the impact of Bruton tyrosine kinase inhibitors on this unique patient population remains to be determined.

Waldenström Macroglobulinemia - A State-of-the-Art Review: Part 1: Epidemiology, pathogenesis, clinicopathologic characteristics, differential diagnosis, risk stratification, and clinical problems.

“…Younger patients with lower mortality and comorbidities may benefit less from these factors and die from WM-related causes. 181 , 182 …”

BeEAM Conditioning including High-Dose Bendamustine before Autologous Stem Cell Transplantation Is Safe and Effective in Patients with Waldenstrom’s Macroglobulinemia

“…However, in this analysis, 63% of patients were considered as IPSS-WM low risk and only a minority (10%) as high risk [ 6 ]. Despite the generally indolent course of the disease and improvement in prognosis, the disease remains a major cause of morbidity and mortality in younger patients with intermediate- and high-risk WM and novel strategies are needed, especially for this patient population [ 7 ].…”

Born in the UK: A registry to improve and broadcast knowledge on Waldenström macroglobulinaemia

“…Indeed, a few studies have addressed this question, mainly in recent years, with conflicting results on the life-expectancy compared to age-and sex-matched general population, probably due to different analysed periods with different therapies and/ or different age cut-offs. 2,3 The other interesting observation of the RMR is the clear evolution of the landscape of WM-directed therapies with the wide use of chemoimmunotherapy (bendamustine-rituximab and rituximabcyclophosphamide-dexamethasone) in first line and single-agent ibrutinib at relapse in the UK. This observation was made possible by the inclusion of patients with 'historic' diagnosis.…”