Evidence map›Paper›PMID 25934475›Full record

Trial reportBlood2015

Effects of deferasirox-deferoxamine on myocardial and liver iron in patients with severe transfusional iron overload.

Yesim Aydinok, Antonis Kattamis, M Domenica Cappellini, Amal El-Beshlawy, Raffaella Origa, Mohsen Elalfy, Yurdanur Kilinç, Silverio Perrotta, Zeynep Karakas, Vip Viprakasit and 5 more

Registry-linked trialOpen access · bronzeAbstract readClinical Trial, Phase IIMulticenter Study
In one paragraph

Trial report in Blood, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01254227 (Phase II, Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety of Deferasirox in Combination With Deferoxamine Followed by Deferasirox Monotherapy in Patients With Severe Cardiac Iron Overload Due to Chronic Blood Transfusion), which is not on this map. Cited by 37 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
37citing papers in PubMed, 1 pooled it
5.9field-weighted citation impact, top 3% 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.

NCT01254227 phase2completednot on this map

Phase II, Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety of Deferasirox in Combination With Deferoxamine Followed by Deferasirox Monotherapy in Patients With Severe Cardiac Iron Overload Due to Chronic Blood Transfusion (HYPERION)

TypeinterventionalSponsorNovartis PharmaceuticalsRan2011 to 2013Enrolled60ConditionsCardiac Iron OverloadArmsDeferasirox and Deferoxamine
3 · Its place in the literature

Who cites it

37 citing papers in PubMed, 1 synthesis or guideline pooled it, 86 citations in OpenAlex.

  1. [Guidelines for iron chelation therapy in thalassemia in China (2025)].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2025
    Guideline
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  13. Liver Iron Loading in Alcohol-Associated Liver Disease.The American journal of pathology · 2023
    Review
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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

15 authors at 12 institutions in 7 countries.

Yesim AydinokDepartment of Pediatric Hematology, Ege University Hospital, Izmir, Turkey;
Antonis KattamisFirst Department of Pediatrics, University of Athens, Athens, Greece;
M Domenica CappelliniUniversitá di Milano, Ca Granda Foundation Istituto Di Ricovero e Cura a Carattere Scientifico (IRCCS), Milan, Italy;
Amal El-BeshlawyDepartment of Hematology, Pediatric Hospital, Cairo University, Cairo, Egypt;
Raffaella OrigaOspedale Regionale per le Microcitemie, University of Cagliari, Cagliari, Italy;
Mohsen ElalfyThalassemia Center, Children's Hospital, Ain Shams University, Cairo, Egypt;
Yurdanur KilinçMedical Facility, Cukurova University, Adana, Turkey;
Silverio PerrottaDepartment of Woman, Child and of General and Specialist Surgery, I°Policlinico II°Università di Napoli, Naples, Italy;
Zeynep KarakasIstanbul Medical Faculty, Istanbul University, Istanbul, Turkey;
Vip ViprakasitDepartment of Pediatrics and Thalassemia Center, Siriraj Hospital, Mahidol University, Bangkok, Thailand;
Dany HabrNovartis Pharmaceuticals, East Hanover, NJ;
Niculae ConstantinoviciNovartis Pharma AG, Basel, Switzerland; and.
Junwu ShenNovartis Pharmaceuticals, East Hanover, NJ;
John B PorterDepartment of Hematology, UCL Cancer Institute, University College London, London, United Kingdom.
HYPERION Investigators
Ain Shams University Hospital · EGCairo University · EGCukurova University · TREge University · TRIstanbul University · TRIstituti di Ricovero e Cura a Carattere Scientifico · ITNational and Kapodistrian University of Athens · GRNovartis (Switzerland) · CHSiriraj Hospital · THUniversity College London · GBUniversity of Cagliari · ITUniversity of Naples Federico II · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Deferasirox (DFX) monotherapy is effective for reducing myocardial and liver iron concentrations (LIC), although some patients may require intensive chelation for a limited duration. HYPERION, an open-label single-arm prospective phase 2 study, evaluated combination DFX-deferoxamine (DFO) in patients with severe transfusional myocardial siderosis (myocardial [m] T2* 5-<10 ms; left ventricular ejection fraction [LVEF] ≥56%) followed by optional switch to DFX monotherapy when achieving mT2* >10 ms. Mean dose was 30.5 mg/kg per day DFX and 36.3 mg/kg per day DFO on a 5-day regimen. Geometric mean mT2* ratios (Gmeanmonth12/24/Gmeanbaseline) were 1.09 and 1.30, respectively, increasing from 7.2 ms at baseline (n = 60) to 7.7 ms at 12 (n = 52) and 9.5 ms at 24 months (n = 36). Patients (17 of 60; 28.3%) achieved mT2* ≥10 ms and ≥10% increase from baseline at month 24; 15 switched to monotherapy during the study based on favorable mT2*. LIC decreased substantially from a baseline of 33.4 to 12.8 mg Fe/g dry weight at month 24 (-52%). LVEF remained stable with no new arrhythmias/cardiac failure. Five patients discontinued with mT2* <5 ms and 1 died (suspected central nervous system infection). Safety was consistent with established monotherapies. Results show clinically meaningful improvements in mT2* in about one-third of patients remaining on treatment at month 24, alongside rapid decreases in LIC in this heavily iron-overloaded, difficult-to-treat population. Combination therapy may be useful when rapid LIC reduction is required, regardless of myocardial iron overload. This trial was registered at www.clinicaltrials.gov as #NCT01254227.

Indexed as

AdolescentAdultBenzoatesChildDeferasiroxDeferoxamineFemaleHeartHumansIron Chelating AgentsIron OverloadLiverMaleMyocardiumSiderophoresTransfusion ReactionBenzoatesDeferasiroxDeferoxamineIron Chelating AgentsSiderophoresTriazoles

Identifiers

PMID25934475
PMCPMC4490296
OpenAlexW2133733540

What OpenQuestion holds

Textmetadata
Read underepoch 390

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.