1 Hematology Department, Azienda Ospedaliera Antonio e Biagio e Cesare Arrigo, Alessandria, Italy.
2 Haematology Research Centre, Department of Immunology and Inflammation, Imperial College London, London, UK.
3 Center for the Study of Myelofibrosis, IRCCS Policlinico S. Matteo Foundation, Pavia, Italy.
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data indicate post-transplant lenalidomide prolongs progression-free
survival and probably survival after an autotransplant for multiple
myeloma (MM). However, optimal therapy duration is unknown,
controversial and differs in the EU and US. We compared outcomes
and cost-effectiveness of 3 post-transplant lenalidomide strategies in
EU and US settings: (1) none; (2) until failure; and (3) 2-year fixed
duration. We used a Markov decision model, which included six
health states and informed by published data. The model estimated
the lenalidomide strategy given to failure achieved 1.06
quality-adjusted life years (QALYs) at costs per QALY gained of €29,232 in the EU and $133,401 in the US settings. Two-year fixed-duration lenalidomide averted €7,286 per QALY gained in the EU setting and saved 0.84 QALYs at $60,835 per QALY gained in the US setting. These highly divergent costs per
QALY in the EU and US settings resulted from significant differences in
post-transplant lenalidomide costs and 2nd-line therapies driven by
whether post-transplant failure was on or off-lenalidomide. In
Monte Carlo simulation analyses which allowed us to account for the
variability of inputs, 2-year fixed-duration lenalidomide remained the
preferred strategy for improving healthcare sustainability in the EU
and US settings.
|Figure 1. Markov model. MT = maintenance therapy (post-transplant lenalidomide)
1. Input clinical values of the model.[6,16,18-23,25,26]
|Table 2. Input cost values of the model.
|Table 3. Base-case cost-effectiveness analysis
|Table 4. Scenario analysis.
|Supplementary Table 1. Distribution of therapy choices for second and third line: carlfizomib, lenalidomide, dexamethasone (KRD), daratumumab, lenalidomide, dexamatheasone (DaraRD), daratumumab, bortezomib, dexamethasone (DaraVD), carlfizomib, dexamethasone (KD), pomalidomide, bortezomib, dexamethasone (pomVD), pomalidomide, cyclophosphamide, dexamethasone (PomCD).|
|Supplementary Table 2. Literature search strategy.|
|Supplementary Table 3. Retrieved studies.|
1. Breakdown of costs in the EU setting (panel A) and in the US
setting (panel B). X-axis shows thousand euros in panel A and thousand
dollars in panel B.
Abbreviations: continuous lenalidomide maintenance: “cont”; 2-year fixed-duration lenalidomide maintenance: “fixed”; no post-transplant maintenance: “no maint”.
|Supplementary Figure 2. Monte Carlo simulation of the decision model outputs. Incremental cost and incremental effectiveness (quality-adjusted months) of continuous or fixed-duration lenalidomide maintenance versus no maintenance are reported: each simulation is represented by a dot. Continuous lenalidomide maintenance versus no maintenance is reported in panels A (US setting) and B (EU setting). Two-year fixed duration maintenance versus no maintenance is reported in panels C (US setting) and D (EU setting). Willingness to pay (WTP) for an additional QALY in thousand dollars or thousand euros is plotted. The higher is the number of dots plotted above the WTP line, the less cost-effective was the maintenance strategy assessed.|
|Supplementary Figure 3. PRIMSA flow-chart.|