Improved Patient-Reported Outcomes with a Film-Coated Versus Dispersible Tablet Formulation of Deferasirox: Results from the Randomized, Phase II E.C.L.I.P.S.E. Study

• Published in: Blood 2016-12, Vol.128 (22), p.850-850
• Main Authors: Taher, Ali T., Origa, Raffaella, Perrotta, Silverio, Kouraklis, Alexandra, Ruffo, Giovan Battista, Kattamis, Antonis, Goh, Ai-Sim, Cortoos, Annelore, Huang, Vicky, Weill, Marine, Herranz, Raquel Merino, Porter, John B.
• Format: Article
• Language: English
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood.V128.22.850.850

▪ Background: Patients (pts) with hematological disorders such as transfusion-dependent thalassemia (TDT) and myelodysplastic syndromes (MDS) require long-term supportive therapy with iron chelation therapy (ICT) to remove excess iron and prevent organ failure. Adherence to ICT can affect survival, so a well-tolerated and effective chelation regimen is required. The once-daily dispersible tablet (DT) formulation of the iron chelator deferasirox (DFX) has been available since 2005, offering an alternative to parenteral deferoxamine, yet barriers remain to pt adherence including the need to take the drug in a fasting state, palatability and GI tolerability. A DFX film-coated tablet (FCT) was developed that can be taken orally, once-daily with or after a light meal, and is potentially less burdensome to pts than the DT formulation. Here we report pt-reported outcomes, during the 24-week (wk) study. Methods:ICT-naïve or pre-treatedpts aged ≥10 yrs, requiring ICT at DFX DT ≥30 mg/kg/day (TDT) or ≥20 mg/kg/day (MDS), with serum ferritin (SF) >1000 ng/mL, were enrolled. ICT-naïve pts received either DFX DT 20 mg/kg/day or DFX FCT 14 mg/kg/day. ICT pre-treated pts received a DT or FCT dose equivalent to their pre-washout dose. Dose was adjusted based on SF and investigator’s judgment after wk 4 for ICT-naïve pts and after 3 months for ICT pre-treated pts (±5-10 mg/kg/day, maximum 40 mg/kg/day [DT]; ±3.5-7 mg/kg/day, maximum 28 mg/kg/day [FCT]); dose adjustments for safety reasons permitted at any time. The modified satisfaction with iron chelation therapy (modified SICT; 5-point response scales to assess adherence (six questions), satisfaction/preference (two questions), and concern (three questions) domain scores) and palatability (taste, aftertaste, ability to consume medicine, perception of medicine) questionnaires were completed at wks 2, 3, 13, and end of treatment. The GI symptom diary consisted of five items (pain in your belly, nausea, vomiting, constipation, diarrhea) rated on an 11-point scale and was completed daily. Results: In total, 173 pts were enrolled; 87 were treated with FCT and 86 were treated with DT. Completion rates for questionnaires (~80% at the start reducing to ~70% by wk 24) and GI daily diary (~70% at the start reducing to ~35% by wk 24) were similar for each formulation. Throughout the 24-wk study period, FCT pts consistently reported greater adherence (attributable to: easier to remember to take medication, thinking less about stoppi