An International, Multi-Centre, Retrospective Study to Describe Treatment Pathways, Outcomes, And Resource Use in Patients with Multiple Myeloma (INTEGRATE)

Study type

• Study topic:
  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology
• Data collection methods:
  Combined primary data collection and secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Review of medical records

Study drug and medical condition

• Medical condition to be studied:
  Plasma cell myeloma

Population studied

• Short description of the study population:
  Adult patients newly diagnosed with MM (NDMM) who have received frontline treatment with chemotherapy (Cohort 1) and patients with a diagnosis of RRMM.; ; Inclusion criteria; • Patients who have newly diagnosed symptomatic MM (Cohort 1) or presented with RRMM (Cohort 2) between 01st January 2010 and 31st December 2011 (inclusive).; • Patients who have completed at least one full line of treatment.; • Age ≥ 18 years at first diagnosis of MM (Cohort 1) or at diagnosis of RRMM (Cohort 2).; • Alive or deceased.; • Patient provides written informed consent for study data collection (as required by local regulations).; ; Exclusion criteria; • Patients for whom the minimum study dataset (Table 3) is unavailable from their hospital medical records.; • Patients with smouldering myeloma.; • Patients with monoclonal gammopathy of unknown significance (MGUS).; • Patients enrolled in a clinical trial of an investigational medicinal product during the observation period.
• 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:
  2600

Study design details

• Main study objective:
  The purpose of this study is to describe time to next treatment at each line of therapy in participants receiving frontline treatment for newly diagnosed Multiple Myeloma (NDMM), Cohort 1, and in participants with relapsed or refractory MM (RRMM), Cohort 2.
• Outcomes:
  The primary outcome measre will be time to next treatment (TTNT) defined for Cohort 1 as the time from initiation of first treatment for MM and for Cohort 2 as the time from initiation of first treatment following presentation with RRMM, to date of next treatment or death, censored at date of data collection. The secondary endpoints will be patient characteristics, treatment pathways, clinical outcomes & adverse events and resource use and costs.
• Data analysis plan:
  Descriptive analyses will be conducted to describe patient demographics and clinical characteristics, treatment patterns, clinical outcomes, healthcare resource use and costs. Key categorical endpoints will be summarised using number and percentage in each category. Key continuous endpoints will be summarised using summary statistics of mean and standard deviation, median and interquartile range. Key time to event endpoints will be summarised in terms of total number of events observed and proportion of participants who have died/progressed at a given milestone after accounting for censoring using Kaplan-Meier curves. In addition to descriptive analyses, multivariate analyses will be conducted if possible to adjust for differences in patient demographic, clinical, and treatment characteristics, as well as risk factors that will be conducted for key time to event analyses, when treatment subgroups will be compared within the RRMM or NDMM cohorts wherever possible.