The evolving treatment landscape of multiple myeloma in Portugal: A nation-wide retrospective cohort study of real-world clinical practice.

Objectives: To characterize variations in real-world treatment patterns in multiple myeloma (MM) in Portugal over a 5-year period.
Methods: A retrospective cohort multicenter study using secondary data of national hospital drug consumption database from 11 Portuguese public hospitals between 2017 and 2022.
Results: Number of MM-treated patients increased 53% over 5 years (from 825 to 1266 patients). Constant slight predominance of male patients (55%), 82% over 60 years old (median age, 70 years), and half of newly diagnosed patients were transplant-eligible. The highest growth rate was in second-line treatments, with a sixfold increase in patients in fourth-line or beyond. First-line treatment pattern remained stable both in transplant-eligible (bortezomib, cyclophosphamide and dexamethasone (VCd_, bortezomib, thalidomide and dexamethasone (VTd), and bortezomib, lenalidomide and dexamethasone (VRd)) and noneligible patients (bortezomib, melphalan and prednisolone (VMP), VCd, and lenalidomide, dexamethasone (Rd)). Maintenance therapy increased from 5% to 16%, shifting from thalidomide to lenalidomide. Second and third lines were dominated by daratumumab-based regimens after 5 years. No standard of care in fourth-line treatment. Treatment duration increased in transplant-eligible due to maintenance therapy and in noneligible due to fourth-line treatments. Patients moved from first- to second-line more rapidly over time.
Conclusions: There was an increase in MM patients reaching advanced treatment lines and significant changes in the treatment patterns, driven by access to more effective frontline treatments and longer duration of treatment.
Competing Interests: Rui Bergantim received fees from Janssen, Amgen, BMS, Takeda, Sanofi, and Pfizer for consultancy and/or speaker services, and research funding from Amgen and BMS; Catarina Geraldes received fees from Celgene/BMS, Janssen, Amgen, Takeda, Sanofi, Pfizer, Gilead, and Abbvie for consultancy, speaker services, and/or advisory boards; Cristina João received fees from Janssen, Amgen, BMS, Takeda, Sanofi, Lilly, and Pfizer for consultancy and/or speaker services, and research funding from Janssen, Takeda, and Amgen; Paulo Lúcio received fees from Janssen, Amgen, BMS, Takeda, Sanofi, and MSD for consultancy and/or speaker services; Manuel Neves received fees for consultancy and/or speaker services from Amgen, Janssen, BMS, Sanofi, Pfizer, or Takeda; Susana Santos and Diogo Ramos are employees of Johnson & Johnson Innovative Medicine; Hugo Pedrosa and Miguel Ventura are employees of IQVIA Solutions contracted by Johnson & Johnson Innovative Medicine for developing and implementing the project, including data collection and analysis.
(© 2024 The Author(s). eJHaem published by British Society for Haematology and John Wiley & Sons Ltd.)