Evidence map›Paper›PMID 30453981›Full record

Trial reportHealth and quality of life outcomes2018

Patient-reported outcomes from a randomized phase II study of the deferasirox film-coated tablet in patients with transfusion-dependent anemias.

Ali T Taher, Raffaella Origa, Silverio Perrotta, Alexandra Kouraklis, Giovan Battista Ruffo, Antonis Kattamis, Ai-Sim Goh, Vicky Huang, Aiesha Zia, Raquel Merino Herranz and 1 more

Registry-linked trialOpen access · goldAbstract readClinical Trial, Phase IIMulticenter StudyRandomized Controlled Trial
In one paragraph

Trial report in Health and quality of life outcomes, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02125877 (A Randomized, Open-label, Multicenter, Two Arm, Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation), which is not on this map. Cited by 11 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed, 2 pooled it
1.0field-weighted citation impact, top 22% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT02125877 phase2completednot on this map

A Randomized, Open-label, Multicenter, Two Arm, Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)

TypeinterventionalSponsorNovartis PharmaceuticalsRan2014 to 2016Enrolled173ConditionsChronic Iron Overload Due to Transfusion-dependant AnemiasArmsDeferasirox dispersible tablet, Defearisox film-coated tablet
3 · Its place in the literature

Who cites it

11 citing papers in PubMed, 2 syntheses or guidelines pooled it, 24 citations in OpenAlex.

  1. Burden of myelodysplastic syndromes: a systematic literature review of economic burden.The European journal of health economics : HEPAC : health economics in prevention and care · 2025
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

11 authors at 9 institutions in 7 countries.

Ali T TaherDepartment of Internal Medicine, Director - Fellowship and Residents Research Program, Faculty of Medicine American University of Beirut Medical Center, Beirut, Lebanon. ataher@aub.edu.lb.
Raffaella OrigaOspedale Pediatrico Microcitemico 'A.Cao', A.O. 'G. Brotzu', Cagliari, Italy.
Silverio PerrottaUniversità della Campania "L. Vanvitelli,", Caserta, Italy.
Alexandra KouraklisHematology Division, Department of Internal Medicine, University of Patras Medical School, Patras, Greece.
Giovan Battista RuffoU.O.C. Ematolog. Con Talassemia, A.O. Civico-Di Cristina-Benfratelli, Palermo, Italy.
Antonis KattamisFirst Department of Pediatrics, University of Athens, Athens, Greece.
Ai-Sim GohDepartment of Medicine, Hospital Pulau Pinang, Georgetown, Penang, Malaysia.
Vicky HuangNovartis Pharmaceuticals Corporation, East Hanover, NJ, USA.
Aiesha ZiaNovartis Pharma AG, Basel, Switzerland.
Raquel Merino HerranzNovartis Pharma AG, Basel, Switzerland.
John B PorterUniversity College London, London, UK.
Novartis (Switzerland) · CHAmerican University of Beirut Medical Center · LBHospital Pulau Pinang · MYNational and Kapodistrian University of Athens · GRNovartis (United States) · USOspedale Microcitemico · ITUniversity College London · GBUniversity of Campania "Luigi Vanvitelli" · ITUniversity of Patras · GR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundAdherence to long-term chelation therapy in transfusion-dependent patients is critical to prevent iron overload-related complications. Once-daily deferasirox dispersible tablets (DT) have proven long-term efficacy and safety in patients ≥2 years old with chronic transfusional iron overload. However, barriers to optimal adherence remain, including palatability, preparation time, and requirements for fasting state. A new film-coated tablet (FCT) formulation was developed, swallowed once daily (whole/crushed) with/without a light meal.

methodsThe open-label, Phase II ECLIPSE study evaluated patient-reported outcomes (PROs) in transfusion-dependent thalassemia or lower-risk myelodysplastic syndromes patients randomized 1:1 to receive deferasirox DT or FCT over 24 weeks as a secondary outcome of the study. Three PRO questionnaires were developed to evaluate both deferasirox formulations: 1) Modified Satisfaction with Iron Chelation Therapy Questionnaire; 2) Palatability Questionnaire; 3) Gastrointestinal (GI) Symptom Diary.

resultsOne hundred seventy three patients were enrolled; 87 received the FCT and 86 the DT formulation. FCT recipients consistently reported better adherence (easier to take medication, less bothered by time to prepare medication and waiting time before eating), greater satisfaction/preference (general satisfaction and with administration of medicine), and fewer concerns (less worry about not swallowing enough medication, fewer limitations in daily activities, less concern about side effects). FCT recipients reported no taste or aftertaste and could swallow all their medicine with an acceptable amount of liquid. GI summary scores were low for both formulations.

conclusionsThese findings suggest a preference in favor of the deferasirox FCT formulation regardless of underlying disease or age group. Better patient satisfaction and adherence to chelation therapy may reduce iron overload-related complications.

trial registrationClinicalTrials.gov identifier: NCT02125877; registered April 26, 2014.

Indexed as

Patient Reported Outcome MeasuresPatient SatisfactionAdultBlood TransfusionChelation TherapyDeferoxamineFemaleHumansIron Chelating AgentsIron OverloadMaleMiddle AgedMyelodysplastic SyndromesQuality of LifeThalassemiaDeferoxamineIron Chelating AgentsDeferasiroxIron chelationIron overloadPatient-reported outcomes

Identifiers

PMID30453981
PMCPMC6245526
OpenAlexW2901176603

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.