A global, prospective, non-interventional, observational study of presentation, treatment patterns, and outcomes in multiple myeloma patients - the INSIGHT-MM study

Study type

• Study topic:
  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Effectiveness study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medical condition to be studied:
  Plasma cell myeloma
• Additional medical condition(s):
  Newly diagnosed multiple myeloma within 3 months from initiation of treatment. Relapsed/refractory multiple myeloma who have received 1-3 prior lines of treatment.

Population studied

• Short description of the study population:
  The study included both newly diagnosed and relapsed/refractory multiple myeloma patients aged 18 years or older between July 2016 and November 2018 at 136 centers in 15 countries.; Inclusion Criteria: ; • Is 18 years of age or older. ; • Is experiencing one of the following:; a) Newly diagnosed multiple myeloma (MM) within 3 months from initiation of treatment with documented month and year of diagnosis, criteria met for diagnosis, stage, and MM-directed treatment history, including duration. ; b) Relapsed/refractory MM who have received 1 to 3 prior lines of therapy with documented data in the medical record regarding diagnosis (month and year), the regimens used in 1st, 2nd, and 3rd line as applicable, whether stem cell transplant was part of 1st, 2nd, and 3rd line of therapy, whether consolidation/maintenance was part of 1st, 2nd, and 3rd line of therapy, also whether investigational therapy/treated on a clinical trial was part of any of these regimens.; • Patients willing and able to complete PROs in accordance with local regulatory and data protection requirements will be enrolled. ; • Is willing and able to sign informed consent to participate.; ; Exclusion Criteria: ; • Patients reporting to a site in this study for a second opinion (consultation only) or patients whose frequency of consult and follow-up are not adequate for quarterly electronic case report form (eCRF) completion. ; • Participation in another study (observational or interventional) that prohibits participation in this study.
• 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

  Renal impaired
• Special population of interest, other:
  Relapsed/refractory multiple myeloma patients
• Estimated number of subjects:
  4200

Study design details

• Main study objective:
  The primary objective of this study is to describe contemporary, real-world disease and patient presentation, therapies, and clinical outcomes in participants with ND MM and participants with R/R MM.
• Outcomes:
  The primary outcomes will assess the number of participants with co-morbidities, diagnosed and with symptoms of ND MM and R/R MM, sites of disease, ECOG performance status, frailty index, participants evaluated for laboratory test, duration for treatment, overall survival, progression status, response to each regimen, time to next therapy and participants with stem cell transplant. Secondary outcomes are treatment combinations,sequencing,rechallenge,clinical outcomes for different strategies,between continuous and intermittent,triggers of treatment initiation at relapse,biochemical progression/symptomatic progression,reasons for treatment modifications,HRQoL,HRU,associations between presentation,disease characteristics,choice of therapy and clinical outcomes,SAE and non-SAE.
• Data analysis plan:
  Population characteristics (including demographics, medical conditions, duration of disease, and types of therapy used at study entry) and all relevant primary and secondary outcomes measures will be summarized as mean, standard deviation, minimum, maximum, median, 25th and 75th percentile, and 95% confidence interval (CI) of the mean for continuous variables, and count and proportion with 95% CI of the proportion for categorical data as appropriate. Descriptive statistics will be used to describe treatment patterns, safety assessments, clinical outcomes, economic outcomes, and HRQoL self-reported outcomes observed during the study period. Outcomes of interest (DOT, PFS, TTNT, OS) will be analyzed using Kaplan Meier methods/estimates and will be adjusted using Cox PH regression models to account for covariantes that could impact treatment regimen assignment and outcomes.