Long-term Outcomes in Patients With Multiple Myeloma: A Retrospective Analysis of the Dutch Population-based HAematological Registry for Observational Studies (PHAROS)

Registry data are important for monitoring the impact of new therapies on treatment algorithms and outcomes, and for guiding clinical decision making in multiple myeloma (MM). This observational study analyzed real-world data from patients in the Population-based HAematological Registry for Observational Studies who were treated for symptomatic MM from 2008 to 2013 in the Netherlands. The primary endpoint was overall survival (OS) from initiation of first-line treatment. Secondary endpoints included OS and progression-free survival per treatment line, treatment patterns, and treatment response. Between 2008 and 2013, 917, 583, 283, and 139 patients had initiated first, second, third, and fourth treatment lines, respectively. Thalidomide-based regimens were the most frequently used first-line treatment (66%); bortezomib- and lenalidomide-based regimens were most often used in the second line (41% and 27%, respectively). The median OS (95% confidence interval) ranged from 37.5 months (34.8–41.8 months) in the first line to 9.2 months (6.2–12.3 months) in the fourth line. Univariate analyses showed that survival benefits were most apparent in younger patients (≤65 vs >65 years). These analyses provide important real-world information on treatment patterns and outcomes in patients with MM.

Study endpoints

The primary endpoint of the study was OS from initiation of the first treatment line. The secondary endpoints included OS from the second, third, and fourth line, PFS, best treatment response, and treatment received, all by treatment line. We also performed analyses of OS and PFS according to age, ISS stage at diagnosis, (previous) SCT status, and treatment type.

Statistical analyses

No formal hypotheses were tested. Patients were grouped according to treatment line. Patients who initiated a treatment line between 2008 and 2013 were included for each treatment line; therefore, individuals could have been included in multiple treatment lines if these were initiated within the time frame of these analyses. Patient characteristics and most commonly used treatment regimens were summarized by line of therapy. A line of therapy was considered as a period during which a patient was treated with a specific antitumor regimen and the period following treatment. A treatment line ended when a new treatment was initiated, the end of the follow-up period was reached, or the patient died. A new treatment line was defined as the initiation of treatment with a new antitumor drug regimen when the patient appeared to be refractory, or after disease progression. Changes in dosing were not considered a new line of therapy. Retreatment with the same antitumor regimen was considered a new line of therapy only if it followed disease progression. Patients may have been diagnosed with MM (or with SMM), but did not immediately start treatment; hence, it was possible that there was a delay between diagnosis and first-line treatment initiation.

All analyses were descriptive, and no formal comparisons were made. OS and PFS were estimated using Kaplan–Meier curves. Treatment responses were described by treatment line and depth of response, and patient characteristics were summarized using descriptive statistics.

Acknowledgments

The authors are grateful to the various registration teams of the Netherlands Cancer Registry for the data collection. PHAROS is a cooperation of HOVON (Dutch–Belgian Cooperative Trial Group for Hematology Oncology), the Institute for Medical Technology Assessment at Erasmus University Rotterdam, and the Comprehensive Cancer Organization, the Netherlands (IKNL).

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