Real-world treatment outcomes in multiple myeloma: Multicenter registry results from Finland 2009-2013

PLoS One. 2018 Dec 5;13(12):e0208507. doi: 10.1371/journal.pone.0208507. eCollection 2018.

Abstract

Outcomes for patients with multiple myeloma (MM) have improved with the advent of novel therapies, however, real-world evidence of outcomes in clinical practice is scarce. We conducted a multi-center registry study to build a reliable picture of treatment and patient outcomes in Finland. The aim of this study was also to understand any methodological challenges in assessing treatment outcomes using disease registry data.

Methods: We carried out a retrospective, observational study using data from the national Finnish Hematology Registry (FHR) to provide real-world evidence of outcomes for all adult patients diagnosed with and treated for MM between 2009-2013 at one of the six regional hospitals, with at least six months of recorded follow-up. Patients were identified within the FHR by applying eligibility criteria of a diagnosis of MM and verifiable records of medical treatment and lines of treatment during the study period. Patients receiving allogenic stem cell transplantation were excluded from the cohort, as were individuals who only had monoclonal gammopathy of undetermined significance diagnosis and patients who had not initiated treatment during this period. Kaplan Meier curves were used to calculate overall survival and time to next treatment. Stratification was carried out by drug status (conventional/novel) and by autologous stem cell transplant (ASCT) status.

Results: A total of 321 patients met the inclusion criteria and were included in this study. Overall survival (OS) was longest in patients who received first-line novel therapy and ASCT (median not reached during 60-month follow-up) versus 46.2 months for novel first-line therapy without ASCT and 25.6 months for first-line conventional therapy without ASCT. Similarly, median time to next treatment were 33.9 months, 12.6 months and 7.8 months, respectively.

Conclusions: The adoption of novel treatments in MM in Finland has had substantial impact on patient outcomes. Given the reality of complex treatment combinations for MM and relatively low patient numbers, assessing individual treatment effectiveness will require substantial cohort sizes and advanced, collaborative analytics on an international scale.

Publication types

  • Multicenter Study
  • Observational Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aged
  • Aged, 80 and over
  • Antineoplastic Agents / therapeutic use*
  • Female
  • Finland
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Kaplan-Meier Estimate
  • Middle Aged
  • Multiple Myeloma / mortality
  • Multiple Myeloma / therapy*
  • Registries
  • Retrospective Studies
  • Transplantation, Autologous
  • Treatment Outcome

Substances

  • Antineoplastic Agents

Grant support

This research is sponsored by Janssen Pharmaceuticals. The funder provided support in the form of salaries for authors AL, PCK, TMJ, FS, MaP. This was a company sponsored study conducted in collaboration with the Finnish researchers, where both parties were involved in the study design, analysis and publication as per ICMJE standards. Data collection was carried out at the Finnish hospitals, with financial support from the funder. Janssen funded JB Medical Ltd to provide medical writing support, TD is a paid employee of JB Medical and carried out the work. JB Medical did not have any additional role in the study design, data collection and analysis or decision to publish.