Evaluation and Comparison of Characteristics and Outcomes Among Frontline Multiple Myeloma (FLMM) Patients with and without Stem Cell Transplant Treatment
Authors: Chari Ajai , Mehra Maneesha , Slavcev Mary , Lam Annette , Potluri Ravi , Kaufman Jonathan L 
Published: October 2019

Abstract
Background: High-dose therapy with autologous stem cell transplantation (SCT) is a standard treatment option in FLMM patients who are ≤65 years of age. Few studies have examined the real-world patient characteristics and outcomes associated with those who receive an SCT compared with non-SCT patients.
Aims: To use real-world data to characterize patients with FLMM who received SCT compared with those who did not receive SCT, and determine their overall survival (OS).
Methods: Data were extracted from 3 real-world data sources from the United States: Surveillance, Epidemiology, and End Results (SEER)-Medicare Linked (January 2007 to December 2014), OPTUM™ Commercial Claims (January 2000 to March 2017), and OPTUM™ Electronic Medical Records (EMR; January 2007 to March 2016) databases. Patients with 1) an index MM diagnosis on or after 1 January 2007 who 2) had known gender and medical prescription coverage at the time of diagnosis, 3) had a 1-year look-back period prior to index diagnosis, 4) had no prior cancers in the 1-year period prior to index diagnosis, and 5) had at least 1 line of treatment were included. SCT patients were defined as those who received an SCT at any time during their follow-up. Patient characteristics such as age (at index diagnosis), gender, and comorbidities (180 days before start of first line of therapy [LOT1]) were descriptively compared. OS was evaluated from the start of LOT1 using the Kaplan-Meier method and multivariable Cox regression analyses.
Results: A total of 9,323 patients were analyzed, comprising 1,599 SCT (17.2%) and 7,724 (82.8%) non-SCT patients. Patient characteristics and OS are summarized in the Table. Descriptive differences in patient characteristics were observed between SCT and non-SCT patients, including median age at start of frontline treatment, gender, and incidences of baseline comorbidities. In terms of survival outcomes, median OS was not reached (NR; 95% confidence interval [CI], 91.8-not estimable [NE]) for SCT patients compared with 45.1 (95% CI, 43.1-46.8) months for non-SCT patients. Age at index diagnosis, gender, time to and year of treatment initiation, and presence of baseline comorbidities were significantly associated with OS. Accounting for differences in these patient characteristics, the adjusted hazard ratio (HR) for OS in non-SCT versus SCT patients was 2.29 (95% CI, 2.01-2.61; P <0.0001). Among the different age subgroups (<65, 65-74, and ≥75 years of age), the OS benefit for SCT versus non-SCT was maintained across these subgroups (Figure).
Conclusions: In a real-world setting, FLMM patients who received SCT were younger and had lower rates of several comorbidities at baseline compared with non-SCT patients. A significant OS benefit was observed among patients who had received SCT, underlying the need for more effective treatment options in patients who do not receive SCT.

Disclosures
Chari: Takeda: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Array Biopharma: Research Funding; Celgene: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Amgen: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Pharmacyclics: Research Funding; Seattle Genetics: Membership on an entity's Board of Directors or advisory committees; Bristol Myers Squibb: Consultancy; Adaptive Biotechnology: Membership on an entity's Board of Directors or advisory committees; Janssen: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; The Binding Site: Consultancy. Mehra:Janssen Global Services, LLC: Employment. Slavcev:Janssen Global Services, LLC: Employment. Lam:Janssen Global Services, LLC: Employment. Potluri:SmartAnalyst Inc.: Employment. Kaufman:Abbvie: Consultancy; Roche: Consultancy; BMS: Consultancy; Janssen: Consultancy; Karyopharm: Other: data monitoring committee.