Treatment patterns, clinical outcomes, and cost-effectiveness of cancer care for advanced multiple myeloma patients: Findings from a large nationwide, population-based cohort
This project was divided into three papers with the following objectives: (1) examine if novel agents proteasome inhibitor and immunomodulatory drugs are effective in prolonging overall survival (OS) for patients with newly diagnosed multiple myeloma (MM); (2) evaluate the risks of adverse events associated with front-line (FL) anti-MM therapy use; (3) determine the cost-effectiveness of FL novel agent-based regimen use from a payer’s perspective. The SEER-Medicare database, a large repository of population-based cancer registry data linked to Medicare claims data, was utilized in this study. 8,839 elderly patients with advanced MM in 2000-2009 were identified from the database, including 4,028 cases in 2000-2004 and 4,811 in 2005-2009. Patients were followed from their initial anti-MM therapy date until death or end of study period (31 December, 2011 for survival, 31 December 2010 for costs) for the purpose of addressing each study objective.^ In the first paper, we found that the Kaplan-Meier (KM) estimate for OS was significantly longer for patients in 2005-2009 than those in 2000-2004 (27.9 mo v 20.0 mo, P<.001). The hazard ratio (HR) for OS for cases in 2005-2009 compared with those in 2000-2004 was 0.78 (95% CI: 0.74-0.82). Among the 4,811 cases in 2005-2009, 54% (n=2,587) received anti-MM therapy. The KM estimates for OS was significantly longer in the treated compared with the untreated (41.1 mo v 27.9 m, P<.001), and the HR for the treated compared with the untreated was 0.58 (95% CI: 0.54-0.62).^ In the second paper, the 2,587 patients receiving anti-MM therapy in 2005-2009 were divided into two subcohorts: patients receiving novel agent (n=2,048) and those receiving chemotherapy or steroids (other, n=539). The HRs for anemia, peripheral neuropathy and thromboembolic events for patients receiving novel agents compared with those receiving other therapies were 1.19 (95% CI: 1.06-1.32), 1.57 (95% CI: 1.15-2.15), and 1.31 (95% CI: 1.06-1.67), respectively. The HRs for anemia, neutropenia, and thromboembolic events for patients receiving PI plus IMiD combination therapies compared with those receiving PI- or IMiD-based therapies were 1.31 (95% CI: 1.12-1.54), 1.66 (95% CI: 1.27-2.18, and 1.37 (95% CI: 1.02-1.86), respectively.^ In the third paper, the 12-month total MM-related costs were assessed for the 2,249 patients in 2000-2009 who had received novel agent-based therapy or chemotherapy for ? 30 days. We found that average 12-month total MM-related costs were 2.03 times higher for novel agent-based therapy ($144,665) versus chemotherapy $47,750). 12-month survival rates increased significantly among patients receiving novel agents compared with those receiving chemotherapy. Treating patients with novel agents was only cost-effective compared to chemotherapy when the willingness-to-pay thresholds were very high.^ In conclusion, we demonstrate improved survival outcomes in patients with newly diagnosed MM in 2005-2009 compared with those in the previous five years. The data suggest that novel agents significantly increase the risk for anemia, peripheral neuropathy, and thromboembolic events. PI plus IMiD combination therapies further increase the risk for anemia, neutropenia, and thromboembolic events. Given the most common norms in the US, the use of FL novel agent-based therapy is not cost-effective at their current level of cost and effectiveness.^
Chen, Ying, "Treatment patterns, clinical outcomes, and cost-effectiveness of cancer care for advanced multiple myeloma patients: Findings from a large nationwide, population-based cohort" (2016). Texas Medical Center Dissertations (via ProQuest). AAI10245357.