Findings from a large observational dataset published in The Lancet Haematology may provide insight to help fine-tune and standardize the currently heterogeneous treatment of patients with Waldenström macroglobulinemia (WM).1
Although the diagnosis and criteria for treatment initiation is well-established, a lack of prospective data has led to differences in treatment and management strategies for patients with WM. A better understanding of current practices and outcomes may lead to improved, standardized care, as well as a better use of health care resources.
For this large, observational review, investigators retrospectively reviewed the electronic records of 454 patients with WM across 10 countries in Europe. Eligible patients were diagnosed or initiated therapy between January 1, 2000, and January 1, 2014, and had complete clinical and biological data and symptomatic disease.
Results showed that of the patients receiving front-line therapy, 193 (43%) patients received monotherapy, 164 (36%) received chemoimmunotherapy, and 95 (21%) received other combination therapies. Patients who received first-line therapy had a median progression-free survival (PFS) of 29 months (95% CI, 25-31) and a 10-year overall survival (OS) rate of 69% (95% CI, 25-31). Median overall survival was not reached.
A multivariate analysis revealed that patients had significantly worse PFS and OS if they were considered high risk based on International Prognostic Scoring System for WM compared with patients who were low-risk.
Chemoimmunotherapy or other combination therapies led to prolonged PFS compared with monotherapy, and receiving treatment in the community versus academic institutions led to similar improvements in PFS.
Anemia was the most frequently cited cause for therapy initiation. Constitutional symptoms (eg, fever, unexplained weight loss), with the exception of fatigue, were independently associated with a worse OS.
The authors concluded that “future surveys might allow investigation of the effect of potential therapies or combinations of novel drugs on the survival of patients with Waldenström macroglobulinemia, as well as their long-term outcomes in the real-world setting.”
- Buske C, Sadullah S, Kastritis E, et al. Treatment and outcome patterns in European patients with Waldenstrom’s macroglobulinemia: a large, observational, retrospective chart review. Lancet Oncol. 2018;5:e299-e309.