DARWIN EU® - Multiple myeloma: patient characterisation, treatments, and survival in the period 2012–2024

Study type

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

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Additional medical condition(s):
  Multiple myeloma

Population studied

• Short description of the study population:
  The study population will include all individuals with a first diagnosis of multiple myeloma identified in the database between 01/01/2012 and 31/12/2024. Participants with a diagnosis of cancer (any, excluding non-melanoma skin cancer) any time prior to the diagnosis of multiple myeloma will be excluded.
• Age groups:
  • In utero
  • Paediatric Population (< 18 years)

    • Neonate

      • Preterm newborn infants (0 – 27 days)
      • Term newborn infants (0 – 27 days)
    • Infants and toddlers (28 days – 23 months)
    • Children (2 to < 12 years)
    • Adolescents (12 to < 18 years)
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)

Study design details

• Study design:
  A cohort study will be conducted using routinely collected health data. The study will comprise of:; • A patient-level characterisation will be conducted to address objective 1,2,3,4 to characterise patients with multiple myeloma diagnosed in the period 2012–2024. ;
• Main study objective:
  1. To describe demographic and clinical characteristics of patients with multiple myeloma at the time of diagnosis.; 2. To describe multiple myeloma treatments (including combinations and regimen types, e.g. triplets, etc.).; 3. To describe sequences of treatments and treatment combinations for multiple myeloma.; 4. To estimate the overall survival of incident multiple myeloma patients during the study period (2012–2024).;
• Setting:
  This study was conducted using routinely collected data from 6 data sources in the DARWIN EU® Network of Data Partners from 6 European countries, of which 6 EU member states (Table 3). All data were a priori mapped to the OMOP CDM. Detailed information on the data sources used for the study are described in Annex I.
• Outcomes:
  Two main outcomes of interest were studied: ; • Multiple Myeloma treatments: treatments and treatment classes initiated at index date, 1 to 30, 1 to 90, 1 to 365 days, and 1 day to 5 years post index date (Objective 2); and the sequences of treatments and treatment combinations post index date were described at 1 to 365 days, and 1 to 5 years ; (Objectives 3). ; Multiple Myeloma treatments included chemotherapies (melphalan, bendamustine, doxirubicin, cisplatin, cyclophosphamide, etoposide, vincristine), IMiDs (thalidomide, lenalidomide, pomalidomide), PI (bortezomib, carfilzomib, ixazomib, venetoclax), monoclonal antibodies (daratumumab, isatuximab, denosumab, elotuzumab), nuclear export inhibitor (selinexor), CAR T-cell (lisocabtagene maraleucel, idecabtagene vicleucel, brexucabtagene autoleucel, axicabtagene ciloleucel, tisagenlecleucel), glucocorticoids (dexamethasone, prednisone), bisphosphonates (zoledronate, pamidronate, clodronate, ibandronate, etidronate), others (panobinostat), and autologous/allogenic stem cell transplantation. The combination of the aforementioned treatments in the treatment of multiple myeloma was also investigated. The description of the construction of treatment combinations is described in Section 8.9.4. Please see Table S2 for a list of codes to identify these treatments and ; treatment classes. ; • Time to death: Overall survival in individuals with incident multiple myeloma was calculated based on the registered date of death for the latter. Individuals contributed survival time as per the follow-up described in Section 8.9.4.