Bergantim Rui, Geraldes Catarina, João Cristina, Lúcio Paulo, Neves Manuel, Trigo Fernanda, Pedrosa Hugo, Ventura Miguel, Santos Susana, Ramos Diogo
Hematology Department ULS São João Porto Portugal.
i3S Instituto de Investigação e Inovação em Saúde University of Porto Porto Portugal.
EJHaem. 2024 Oct 28;5(6):1144-1153. doi: 10.1002/jha2.1035. eCollection 2024 Dec.
To characterize variations in real-world treatment patterns in multiple myeloma (MM) in Portugal over a 5-year period.
A retrospective cohort multicenter study using secondary data of national hospital drug consumption database from 11 Portuguese public hospitals between 2017 and 2022.
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.
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.
描述葡萄牙5年间多发性骨髓瘤（MM）实际治疗模式的变化。
一项回顾性队列多中心研究，使用2017年至2022年期间葡萄牙11家公立医院的国家医院药品消费数据库的二次数据。
5年间接受MM治疗的患者数量增加了53%（从825例增至1266例）。男性患者持续略占优势（55%），82%的患者年龄超过60岁（中位年龄70岁），且一半新诊断患者符合移植条件。二线治疗的增长率最高，四线及以上治疗的患者增加了六倍。一线治疗模式在符合移植条件的患者（硼替佐米、环磷酰胺和地塞米松（VCd）、硼替佐米、沙利度胺和地塞米松（VTd）以及硼替佐米、来那度胺和地塞米松（VRd））和不符合移植条件的患者（硼替佐米、美法仑和泼尼松龙（VMP）、VCd以及来那度胺、地塞米松（Rd））中均保持稳定。维持治疗从5%增至16%，从沙利度胺转向来那度胺。5年后二线和三线治疗以达雷妥尤单抗为基础的方案为主。四线治疗没有标准治疗方案。由于维持治疗，符合移植条件的患者治疗持续时间增加，由于四线治疗，不符合移植条件的患者治疗持续时间增加。随着时间的推移，患者从一线治疗转向二线治疗的速度更快。
由于可获得更有效的一线治疗和更长的治疗持续时间，达到晚期治疗线的MM患者数量增加，治疗模式发生了显著变化。
