A retrospective population-based study of long-term treatments and outcomes inwaldenstrom macroglobulinemia in Ontario, Canada
Bibliographic record
Abstract
Abstract Introduction: Waldenstrom Macroglobulinemia (WM) is a rare B-cell malignancy characterized by periods of low burden disease not requiring treatment eventually leading to active disease requiring therapy. Treatment approaches have evolved over time including chemotherapy ± monoclonal antibodies such as rituximab and most recently targeted agents including Bruton Tyrosine Kinase Inhibitors. We sought to characterize treatments and real-world long-term outcomes in a contemporary cohort of Waldenstrom Macroglobulinemia/Lymphoplasmacytic Lymphoma patients. Methodology: This was a retrospective population-based study in Ontario, Canada using linked administrative databases. All eligible patients >18 years diagnosed from 2005 to 2023 identified within the Ontario Cancer Registry were included. All provincially funded treatment regimens were documented; however, treatments funded by compassionate access, private insurance or investigational regimens as part of clinical trials were not captured. In total, n=1,986 patients identified as having either Lymphoplasmacytic Lymphoma or Waldenstrom Macroglobulinemia were included in this study. We evaluated outcomes including types of treatment, time to second line therapy and overall survival using the Kaplan Meier method. Results: Out of the 1,986 patients with Waldenstrom Macroglobulinemia/Lymphoplasmacytic Lymphoma included in the cohort, the median age was 73 (IQR: 64-80) and 39% were female. Patients were followed for a median of 4 years (IQR: 2-8). In total, 55% of patients (n=1,093) received provincially funded treatment for Waldenstrom's during the follow-up period; median Kaplan Meier estimate of time from diagnosis to first-line treatment was 1.9 years. Of those who received frontline therapy, 52% of patients (n=569) went on to receive rituximab maintenance therapy and 23% of patients (n=250) went on to receive second-line treatment; median Kaplan Meier estimate of time to second line therapy was over 13 years. In total, 80 patients (4% of the overall cohort and 7% of those who received frontline therapy) received three or more lines of treatment. In total, 37% (n=727) of patients died within with the follow-up period, with median Kaplan Meier survival estimate of 9.9 years after diagnosis. The most common frontline treatment regimens (each +/- anti-CD20 antibody) were Bendamustine (60%), CVP (21%), Bruton Tyrosine Kinase inhibitor (5%), rituximab monotherapy (5%), Chlorambucil (4%) or CHOP (2%). Comparison of overall survival, time to second line therapy and depth of response based on IgM for different treatment regimens is under active investigation. Conclusion: We characterized real-world long-term outcomes in a large population-based cohort of Waldenstrom's patients. Newly diagnosed patients over the past two decades have received multiple frontline treatment regimens and overall have robust long-term survival outcomes.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.001 | 0.002 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.000 |
| Bibliometrics | 0.001 | 0.005 |
| Science and technology studies | 0.002 | 0.001 |
| Scholarly communication | 0.001 | 0.000 |
| Open science | 0.001 | 0.001 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.001 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".