A prospective non-interventional post-authorization safety study (PASS), designed as a disease registry of patients with transfusion dependent IPSS low or intermediate-1-risk myelodysplastic syndromes (MDS) and isolated del(5q) (CC-5013-MDS-010)

Study type

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

  Safety study (incl. comparative)
• If ‘other’, further details on the scope of the study:
  Patient-based data collection
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Prospective, post-authorisation safety study (PASS)

Study drug and medical condition

• Medical condition to be studied:
  Myelodysplastic syndrome

Population studied

• Short description of the study population:
  The study population included 18 years or older aged patients with transfusion-dependent International Prognostic Scoring System (IPSS) low or intermediate-1-risk myelodysplastic syndromes (MDS) and isolated del(5q) cytogenetic abnormality.
• Age groups:
  • 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)
• Special population of interest:
  Other
• Special population of interest, other:
  Myelodysplastic syndrome patients
• Estimated number of subjects:
  375

Study design details

• Main study objective:
  Whether the 2-year cumulative incidence of acute myeloid leukemia (AML) progression and mortality among transfusion-dependent, IPSS low or intermediate-1-risk MDS and isolated del(5q) patients treated with lenalidomide in a routine-care setting will differ from the incidence observed in Studies MDS-003 and MDS-004 combined.
• Outcomes:
  To ascertain the progression to AML and survival (through calculation of product-limit estimators and incidence rates, as well as the attributable risk AR and AR percentage AR%) among 603 patients (primary population) with transfusion dependent IPSS low- or int-1-risk MDS with del (5q) as an isolated cytogenetic abnormality who have been treated with lenalidomide, To describe the progression to AML and survival among patients with MDS who have never been exposed to lenalidomide. This will include explorative analyses of progression to AML and survival in patients receiving treatments or treatment modalities other than lenalidomide whenever possible. To further characterise the safety profile of lenalidomide among MDS patients treated with lenalidomide
• Data analysis plan:
  The formal multivariate analysis of AML progression risk and OS will be conducted using the primary population of 603 MDS patients with a single del (5q) aberration who have received at least 1 complete cycle of lenalidomide. -For purposes of estimating the incidence of hematologic and nonhematologic AEs, as well as uncommon events not previously documented, all MDS patients treated with at least 1 dose of lenalidomide will be included. -Safety analyses will be conducted separately for the primary population and the safety populations. In addition, explorative analyses of progression to AML and survival in patients receiving treatments or treatment modalities other than lenalidomide, will be run whenever possible.