Prescribing information

 

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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

Diagram summarising the design of the TELESTO study from screening to randomisation to final assessment

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

  1. 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].
HCP20-C005e June 2020.
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Adverse events should be reported. Reporting forms and information can be found at yellowcard.mhra.gov.uk. Adverse events should also be reported to Novartis via [email protected] or online through the patient safety information (PSI) tool at https://psi.novartis.com
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