Objective1
A prospective, randomised, double-blind, placebo-controlled, parallel-group Phase II clinical study evaluating the EFS and safety of EXJADE in iron-overloaded patients with MDS.
Trial cohort1
In total, 225 patients participated and were randomised in a 2:1 ratio where 149 patients received EXJADE and 76 patients received placebo.
Key inclusion criteria1
- ≥18 years of age
- Haematologically stable with an IPSS score of low- or intermediate-1-risk MDS, confirmed by bone marrow within 6 months prior to study entry
- Serum ferritin >1,000 μg/L
- Prior receipt of 15 to 75 pRBC units, with the anticipation to be transfused with at least 8 pRBC units annually during the study
- An Eastern Cooperative Oncology Group status of 0 to 2
- Without cardiac, liver and renal abnormalities
Study design
Patients received 10–40 mg/kg/day based on dosing guidelines
Primary endpoint1
To evaluate EFS with EXJADE vs. placebo, defined as time from date of randomisation to first documented non-fatal event (related to cardiac or liver dysfunction and transformation to acute myeloid leukaemia) or death, whichever occurred first.
Key secondary endpoints1
- To evaluate OS, calculated as the date of death minus date of randomisation plus 1
- Time to first occurrence of serum ferritin >2 times the BL value at 2 consecutive assessments
- Haematologic improvement in terms of erythroid response
- Changes in endocrine function
- The safety and tolerability profile of treated patients
*After study treatment discontinuation, patients without a fatal event were followed up for 28 days for safety. Depending on consent, patients could then be followed for evaluation (every 3 months), survival (every 6 months), or both.1
Abbreviations: BL, baseline; EFS, event-free survival; IPSS, International Prognostic Scoring System; LPFV, last patient first visit; OS, overall survival; pRBC, packed red blood cell; MDS, myelodysplastic syndromes.
Reference
- Angelucci E, et al. Iron chelation in transfusion-dependent patients with Low- to Intermediate-1-risk myelodysplastic syndromes: a randomized trial. Ann Intern Med. 2020 Mar. [Epub ahead of print].
