Disease progression and resource utilization in treated relapse/refractory multiple myeloma in Spain (PREMIERE)

Study type

• Study topic:
  Disease /health condition

  Other
• Study topic, other:
  Disease/Epidemiology study
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

  Other
• If ‘other’, further details on the scope of the study:
  Treatment patterns and resource utilisation
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Retrospective chart review

Study drug and medical condition

• Medical condition to be studied:
  Plasma cell myeloma

Population studied

• Short description of the study population:
  Patients treated for a first episode of relapse/refractory multiple myeloma in in a real-world setting in Spain.
• Age groups:
  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Special population of interest:
  Other
• Special population of interest, other:
  Multiple myeloma patients
• Estimated number of subjects:
  375

Study design details

• Main study objective:
  The primary objective is to estimate progression free survival (PFS) in a cohort of patients in Spain treated for a first episode of relapse/refractory multiple myeloma (RRMM).
• Outcomes:
  Progression-free survival (PFS), Overall survival (OS), socio-demographic, clinical and treatment characteristics, healthcare resource utilisation and associated costs, and reasons for change in Line of Therapy (LOT)
• Data analysis plan:
  PFS and OS will be described using Kaplan-Meier plots with estimation of the median PFS and 95% CIs. The number of events and the number of patients at risk by 1 month periods will be reported. If the median level (50%) is not reached, cumulative incidence by 3 month periods will be provided. Analyses will be performed overall and stratified by age, presence of previous treatment with immunomodulatory drugs and prior LOT (1st, ≥ 2). Patient characteristics at the index date will be summarized. Descriptive statistics will be used to describe treatment characteristics by LOT during follow-up. Summary descriptive statistics will report HRU and costs during the follow-up in the pre-/post-progression states. The overall proportion of RRMM patients who undergo a change in LOT during follow-up, proportions by LOT and reasons for change will be presented. To identify patient- and center-level characteristics associated with PFS Cox regression models will be used.