Artikel
Effectiveness of treatment sequences for elderly patients with multiple myeloma – a comparison of treatment schedules from clinical trials and experience from the Austrian Myeloma Registry
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Autoren
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Ursula Rochau
- UMIT – University for Health Sciences, Medical Informatics and Technology, Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health, Health Services Research and Health Technology Assessment, Österreich
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Durda Vukicevic
- UMIT – University for Health Sciences, Medical Informatics and Technology, Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health, Health Services Research and Health Technology Assessment, Österreich
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Silvia Angerer
- UMIT – University for Health Sciences, Medical Informatics and Technology, Institute for Management and Economics in Healthcare, Department of Public Health, Health Services Research and Health Technology Assessment, Österreich
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Monika Schaffner
- UMIT – University for Health Sciences, Medical Informatics and Technology, Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health, Health Services Research and Health Technology Assessment, Österreich
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Vjollca Qerimi Rushaj
- UMIT – University for Health Sciences, Medical Informatics and Technology, Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health, Health Services Research and Health Technology Assessment, Österreich
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Annette Conrads-Frank
- UMIT – University for Health Sciences, Medical Informatics and Technology, Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health, Health Services Research and Health Technology Assessment, Österreich
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Beate Jahn
- UMIT – University for Health Sciences, Medical Informatics and Technology, Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health, Health Services Research and Health Technology Assessment, Österreich
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Werner O. Hackl
- UMIT – University for Health Sciences, Medical Informatics and Technology, Institute of Medical Informatics, Österreich
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Elske Ammenwerth
- UMIT – University for Health Sciences, Medical Informatics and Technology, Institute of Medical Informatics, Österreich
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Harald Stummer
- UMIT – University for Health Sciences, Medical Informatics and Technology, Institute for Management and Economics in Healthcare, Department of Public Health, Health Services Research and Health Technology Assessment, Österreich; Seeburg Castle University, Institute for Healthcare Management and Innovation, Österreich
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Louis Preston Garrison
- University of Washington, The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, USA
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Roman Weger
- ONCOTYROL – Center for Personalized Cancer Medicine, Österreich; ACMIT – Austrian Center for Medical Innovation & Technology, Österreich
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Ella Willenbacher
- Medical University Innsbruck, Internal Medicine V, Hematology and Oncology, Innsbruck, Österreich
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Wolfgang Willenbacher
- ONCOTYROL – Center for Personalized Cancer Medicine, Österreich; Medical University Innsbruck, Internal Medicine V, Hematology and Oncology, Innsbruck, Österreich
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Uwe Siebert
- UMIT – University for Health Sciences, Medical Informatics and Technology, Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health, Health Services Research and Health Technology Assessment/, ONCOTYROL – Center for Personalized Cancer Medicine, Österreich; Harvard Medical School, Institute for Technology Assessment, Department of Radiology, Massachusetts General Hospital, and Harvard T.H. Chan School of Public Health, Center for Health Decision Science, Department of Health Policy and Management, USA
| Veröffentlicht: | 20. März 2019 |
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Gliederung
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Background/research question: Multiple myeloma (MM) originates from a neoplastic proliferation of plasma cells in the bone marrow. Usually, multiple treatment lines are applied after disease progression [1]. Results from randomized controlled trials evaluating different treatment regimens are available. Trials comparing sequences of treatment strategies over time are lacking. However, clinically relevant, long-term outcomes depend on the optimal treatment sequence rather than on each single treatment line. Decision-analytic modeling is a tool to synthesize evidence from different sources, extrapolating from short-term data and evaluating lifetime outcomes under uncertainty [2]. Our aim was to develop a decision-analytic model to evaluate the likely long-term effectiveness of different sequential treatment regimens for elderly patients with MM not eligible for stem cell transplantation in Austria.
Methods: We developed a state-transition Markov model to assess five different sequential treatment options including four treatment lines and palliative treatment for elderly MM patients. Treatment strategies, labeled based on 1st-line treatment, were as follows: melphalan/prednisone/bortezomib (MPV); melphalan/prednisone/thalidomide (MPT); bortezomib/thalidomide/dexamethasone (VTd); bortezomib/dexamethasone (Vd); and lenalidomide/dexamethasone (Rd). Clinical pathways in the model were based on MM treatment guidelines and validated by Austrian clinical experts. Effectiveness parameters were extracted from clinical trials. Evaluated outcomes were patient life expectancies. In addition to the treatment schedule derived from clinical trials, we evaluated a treatment schedule according to experts’ suggestions with experience from the Austrian Myeloma Registry. Further model assumptions were assessed in deterministic sensitivity analyses.
Results: Based on the treatment schedule derived from clinical studies, the longest life expectancy was achieved on the treatment line starting with MPV (6.39 life-years (LY)). MPT resulted in the lowest life expectancy (5.10 LYs). In the scenario analysis based on experts’ suggested treatment schedules, life expectancy ranged from 5.27 LYs (MPT) to 6.38 LYs (MPV). Sensitivity analyses showed that MPT remained the strategy with the lowest life expectancy and MPV the one with the highest.
Conclusions: Based on our model findings, MPV produces the largest life expectancy and can be recommended as an effective treatment option for elderly MM patients in Austria.
Competing interests: This work was supported by the Tiroler Wissenschaftsfonds. The project has received funding from the Tiroler Wissenschaftsfonds under grant agreement number UNI-0404/1425. This work was supported by the COMET Center ONCOTYROL, which is funded by the Austrian Federal Ministries BMVIT/BMWFJ (via FFG) the Tiroler Zukunftsstiftung/Standortagentur Tirol (SAT) and the Krebsforschungsverein Tirol. This work has been financially supported through Erasmus Mundus Western Balkans (ERAWEB), a project funded by the European Commission.
