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)

Study Objective 

Primary Objective

• 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, International Prognostic Scoring System (IPSS) low- or intermediate-1 (int-1)-risk MDS with deletion 5q as an isolated cytogenetic abnormality who have been treated with lenalidomide

Secondary Objectives

• 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 different treatments or treatment modalities other than lenalidomide, whenever possible.

• To further characterize the safety profile of lenalidomide among MDS patients treated with lenalidomide through calculation of incidence measures for hematologic and nonhematologic adverse events, including infections, bleeding, thromboembolic events, major cardiac events, and second primary malignancies (SPMs) other than AML (considered disease progression)

Participation conditions

Inclusion criteria

Patients may be enrolled if they:
• Understand and voluntarily sign an informed consent form, as appropriate according to local regulations
• Have confirmed diagnosis of IPSS low or intermediate-1-risk MDS with isolated del(5q) (with morphological and cytogenetic information) diagnosed on 15th June 2007 (date of Revlimid® first approval in Europe) or later;
• Are transfusion dependent or have an history of transfusion dependence; for the purpose of the current MDS registry, transfusion-dependence is defined as ≥ 1 RBC units every 8 weeks during a minimum observation period of 16 weeks;
• Have commenced first treatment with lenalidomide in the EU after 13th June 20134 (limited to the primary population)

Exclusion criteria

Patients cannot be enrolled if they:
• Refuse to participate in the MDS registry
• Are currently participating in an interventional therapeutic clinical trial for MDS (except for ESAs and granulocyte colony-stimulating growth factors) or receive any type of investigational agent in combination with lenalidomide for MDS

CHL et CHEM