Rare lymphomas in routine practice-Treatment and outcome in Waldenström's macroglobulinaemia in the prospective German Tumour Registry Lymphatic Neoplasms

Abstract

Waldenström's macroglobulinaemia (WM) is a rare indolent B-cell lymphoma for which only little prospective phase III evidence exists. Thus, real world data are important to provide insight into treatment and survival. We present here data on choice and outcome of systemic treatment of patients with WM treated in German routine practice. In total, 139 patients with WM who had been documented in the prospective clinical cohort study Tumour Registry Lymphatic Neoplasms (NCT00889798) were included into this analysis. We analysed the most frequently used first-line and second-line treatments between 2009 and 2017 and examined best response, progression-free survival (PFS) and overall survival (OS). Bendamustine plus rituximab, with a median of six cycles, was by far the most frequently used first-line treatment (81%). Second-line treatment was more heterogenous and mainly based on bendamustine, cyclophosphamide/doxorubicin/vincristine/prednisone (CHOP), fludarabine or ibrutinib, the latter approved in 2014. Three-year PFS from start of first-line treatment was 83% (95% confidence interval [CI] 74%-88%), 3-year OS was 87% (95% CI 80%-92%). These prospective data give valuable insights into the management and outcome of non-selected patients with WM treated in German routine practice. In the lack of prospective phase III clinical trials, real world data can help bridging the gap of evidence.

Keywords: Waldenström's macroglobulinaemia; disease management; lymphoma, B-cell; outpatients; prognosis; registries.

Conflict of interest statement

Hans Rainer Slawik, Ute Bückner, Annette Sauer, Mark‐Oliver Zahn, Natalie Wetzel, Anja Kaiser‐Osterhues and Leonora Houet declare no conflict of interest concerning the topic of this publication. Wolfgang Knauf has received honoraria by Mundipharma and Janssen for talks and attendance of conferences. Wolfgang Abenhardt has received financial support from Roche, Amgen, Celgene and Gilead Sciences in the context of a lymphoma symposium. Burkhard Otremba declares stock ownership of iOMEDICO. Norbert Marschner is Chief Executive Officer of iOMEDICO and holds shares of this company.

© 2020 The Authors. Hematological Oncology published by John Wiley & Sons Ltd.

Figures

FIGURE 1

Cohort definition. Number of patients enrolled in the TLN from April 2009 until August 2014, split up according to different types of lymphoid B‐cell neoplasms. Of all evaluable patients with indolent NHL (other than CLL or MM), those patients with WM who had been prospectively enrolled at the start of their first‐line treatment were included into this analysis (n = 139). Data cut‐off for this analysis was 31 August 2018. CLL, chronic lymphocytic leukemia; MM, multiple myeloma; NHL, non‐Hodgkin's lymphoma; TLN, Tumour Registry Lymphatic Neoplasms; WM, Waldenström's macroglobulinaemia

FIGURE 2

Choice of systemic first‐line treatment in Waldenström's macroglobulinaemia. Shown are first‐line treatments/treatment combinations between 2009 and 2014 sorted by relative frequency (n = 139). Bendamustine‐based: bendamustine (mono), bendamustine + rituximab (±prednisone/dexamethasone); chlorambucil‐based: chlorambucil (mono), chlorambucil + rituximab (±prednisone/mitoxantrone); fludarabine‐based: fludarabine (mono), fludarabine + cyclophosphamide (±rituximab); CHOP‐based: variations of CHOP (CHOP, CH, COP, HOP) ± rituximab (±prednisone/dexamethasone). Percentages may not add up to 100% due to rounding. C, cyclophosphamide; H, doxorubicin; O, vincristine; P, prednisone

FIGURE 3

Progression‐free survival of patients with Waldenström's macroglobulinaemia since start of first‐line treatment. Progression‐free survival of patients with WM who had been enrolled at the beginning of their first‐line treatment (n = 139). CI, confidence interval; NA, not applicable; PFS, progression‐free survival, WM, Waldenström's macroglobulinaemia

FIGURE 4

Overall survival of patients with Waldenström's macroglobulinaemia since start of first‐line treatment. Overall survival of patients with WM who had been enrolled at the beginning of their first‐line treatment (n = 139). CI, confidence interval; NA, not applicable; OS, overall survival; WM, Waldenström's macroglobulinaemia