Evolution of Management and Outcomes in Waldenström Macroglobulinemia: A Population-Based Analysis

Abstract

Most older patients with Waldenström macroglobulinemia currently treated in the U.S. receive rituximab as monotherapy or in combination with bortezomib or bendamustine. Newly designed trials should consider control arms aligned with this prevalent real-life standard. Compared with the 1990s, patients diagnosed according to current criteria are more likely to have anemia or neuropathy, or to receive early chemotherapy, but only 2.5% require plasmapheresis at diagnosis. The incremental clinical value of newly introduced agents needs to be assessed through a combination of clinical and health services research, taking into consideration their associated survival benefits, toxicities, and associated costs of care.

Long survival in patients with Waldenström macroglobulinaemia diagnosed at a young age

“…Approximately, one-third of patients needed therapy at the time of diagnosis and half of the patients needed therapy within 1 year from diagnosis. This finding is similar to the observations from a population-based study in WM patients aged 65 or older (Olszewski et al, 2016). Our study suggests encouraging OS rates in young WM patients.…”

Cost-effectiveness analysis of ibrutinib in patients with Waldenström macroglobulinemia in Italy

“…Olszewski et al in 2016 estimated that novel treatments, adopted as a standard of care, may lead to observable changes in both survival and costs of therapy [40]. In the study, mean total Medicare payments for the care of a patient with WM at 15 years from diagnosis were calculated to be $163,432 (€147,301).…”

“…Only a few economic studies were identified while neither cost-effectiveness analyses nor economic evaluations on ibrutinib for WM, published in full, were found [2,40].…”

Response and survival for primary therapy combination regimens and maintenance rituximab in Waldenström macroglobulinaemia

“…We acknowledge that it is a common practice in the US to offer single agent rituximab as primary therapy in WM patients (Olszewski et al , ). However, we consider single agent rituximab a less effective therapy in WM, which is associated with an ORR of 30–40% and median PFS ranging between 1 and 2 years (Gertz et al , ; Treon et al , ).…”