Patient Characteristics, Treatment Outcomes and Healthcare Resource Utilization across Europe in Multiple Myeloma Patients Ineligible for Stem Cell Transplantation Who Received Lenalidomide- or Bortezomib-Based Regimens

BACKGROUND: ESMO guidelines (Moreau P, et al. Ann Oncol. 2017) recommend lenalidomide- (LEN) and bortezomib-based (BORT) regimens as first line (1L) treatment (tx) options for patients (pts) with multiple myeloma (MM) who are ineligible for stem cell transplantation (NSCT). The aim of this study was to determine tx outcomes and healthcare resource utilization (HCRU) for pts with NSCT MM receiving 1L LEN- or BORT-based regimens in 7 European countries.

METHODS: Physicians from Austria, Belgium, France, Germany, Italy, Spain, and Netherlands abstracted retrospective data from medical records of pts with MM who received 1L LEN- or 1L BORT-based regimens between Jun 1 2015 and Nov 30 2016. Data were abstracted during Q1-2 2019 to allow adequate follow up time to assess outcomes of interest.

Data collected included pt demographics, clinical characteristics, tx patterns, health outcomes (e.g. progression free survival [PFS], time to next tx [TTNT]), and HCRU (e.g. supportive tx, hospitalizations, healthcare professional [HCP] visits, monitoring tests).

Health outcomes were compared for LEN- and BORT-based regimens using the Kaplan-Meier estimator. HCRU was calculated as means per month (mo), per usage type, to account for variation in tx duration. Patients with complete resource use data for an HCRU category were included in the analysis for that category.

RESULTS: 59 physicians provided data on 453 pts. A total of 220 (48.6%) pts received 1L LEN- and 233 (51.4%) received 1L BORT-based regimens. The most common 1L LEN- and BORT-based regimens were LEN + dexamethasone (DEX; n = 194) and LEN + prednisone (PRED; n = 15), and BORT + melphalan + PRED (n = 94) and BORT + DEX (n = 82), respectively. Mean follow up time was similar for both regimens (38.2 vs 39.4 mo, respectively). Demographic profiles of pts receiving 1L LEN- or BORT-based regimens were similar (P > 0.05 for all demographic variables); time from diagnosis to start of 1L, performance status at first tx, comorbidities, CRAB criteria, bone lesions at diagnosis, or cytogenetic testing did not differ significantly.

Within the follow up period, pts treated with 1L LEN-based regimens received significantly fewer lines of tx (mean [SD] 1.55 [0.64] vs 1.75 [0.69] lines; P < 0.01) vs pts treated with 1L BORT-based regimens. Of the 233 pts receiving 1L BORT-based regimens,142 went on to receive 2L tx, of whom 104 (73%) received a second-line (2L) LEN-based regimen. Of the 220 pts receiving 1L LEN-based regimens, 105 went on to receive 2L tx, of whom 50 (48%) received a 2L BORT-based regimen.

Health outcomes: Significant differences in PFS (P < 0.01) were observed; the probability of maintaining PFS was higher for pts receiving 1L LEN- vs 1L BORT-based regimens at 12 (94% vs 85%) and 24 mo (76% vs 63%) post-1L initiation. A significantly longer TTNT (median 45.7 vs 36.5 mo; P < 0.01) was observed for pts receiving 1L LEN vs those receiving 1L BORT. A significantly longer time to third line (3L) tx was estimated for pts receiving 1L LEN- vs 1L BORT-based regimens: approximately 15% of 1L LEN pts had started 3L tx at 48 mo vs 22% of 1L BORT pts (P = 0.01). From a sequencing perspective, a trend toward longer time to 3L tx was observed for pts receiving 1L LEN followed by 2L BORT vs pts receiving 1L BORT followed by 2L LEN (P = 0.11).

HCRU: Across the follow up period, no differences were observed, with low overall HCRU observed for each cohort.

CONCLUSIONS: Following the approval of LEN in the 1L setting, physicians have more options when treating NDMM. The findings of this study suggest that in a real-world setting, health outcomes are significantly better for NSCT pts with NDMM who receive 1L LEN- vs 1L BORT-based regimens. A US claims database study also suggested that LEN-based tx is associated with longer TTNT (Chari A, et al Clin Lymphoma Myeloma Leuk). In this analysis, 1L LEN-based regimens extended the time to 3L tx, potentially delaying progression to use of and associated cost impact of expensive tx in later line settings. Longer duration on 1L tx may also be expected to reduce total per pt per mo (PPPM) costs, as reported by a US study (Arikian SR, et al. Curr Med Res Opin). In our study, routine tx-related HCP visits were not explicitly captured and so the data may not accurately reflect differences in HCRU for pts receiving 1L LEN- vs 1L BORT-based regimens. This study provides real-world evidence to support 1L LEN-based regimens as providing clinically meaningful benefits for pts.