Deferasirox in children with transfusion-dependent thalassemia or sickle cell anemia: A large cohort real-life experience from Turkey (REACH-THEM)

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dc.contributor.authorAntmen, Bulent
dc.contributor.authorKarakas, Zeynep
dc.contributor.authorYesilipek, Mehmet Akif
dc.contributor.authorKupesiz, Osman Alphan
dc.contributor.authorSasmaz, Ilgen
dc.contributor.authorUygun, Vedat
dc.contributor.authorKurtoglu, Erdal
dc.date.accessioned2024-02-23T14:24:09Z
dc.date.available2024-02-23T14:24:09Z
dc.date.issued2019
dc.departmentNEÜen_US
dc.description.abstractObjectives To evaluate the long-term efficacy and safety of deferasirox therapy in a large observational cohort of children with transfusion-dependent thalassemia (TDT) and sickle cell anemia (SCA) in Turkey. Methods This was a multicenter, prospective cohort study including TDT and SCA patients aged 2-18 years with iron overload (>= 100 mL/kg of pRBC or a serum ferritin [SF] level >1000 mu g/L) receiving deferasirox. Patients were followed for up to 3 years according to standard practice. Results A total of 439 patients were evaluated (415 [94.5%] TDT, 143 [32.6%] between 2 and 6 years). Serum ferritin levels consistently and significantly decreased across 3 years of deferasirox therapy from a median of 1775.5 to 1250.5 mu g/L (P < 0.001). Serum ferritin decreases were noted in TDT (1804.9 to 1241 mu g/L), SCA (1655.5 to 1260 mu g/L), and across age groups of 2-6 years (1971.5 to 1499 mu g/L), 7-12 years (1688.5 to 1159.8 mu g/L), and 13-18 years (1496.5 to 1107 mu g/L). Serum ferritin decreases were also noted for all deferasirox dose groups but only significant in patients with doses >= 30 mg/kg/d (n = 120, -579.6 median reduction, P < 0.001). Only 9 (2%) patients had adverse events suspected to be related to deferasirox. Serum creatinine slightly increased but remained within the normal range. Conclusions Deferasirox has long-term efficacy and safety in children with TDT and SCA, although higher doses (>= 30 mg/kg/d) may be required to achieve iron balance.en_US
dc.description.sponsorshipNovartisen_US
dc.description.sponsorshipNovartisen_US
dc.identifier.doi10.1111/ejh.13180
dc.identifier.endpage130en_US
dc.identifier.issn0902-4441
dc.identifier.issn1600-0609
dc.identifier.issue2en_US
dc.identifier.pmid30300449en_US
dc.identifier.scopus2-s2.0-85058116788en_US
dc.identifier.scopusqualityQ1en_US
dc.identifier.startpage123en_US
dc.identifier.urihttps://doi.org/10.1111/ejh.13180
dc.identifier.urihttps://hdl.handle.net/20.500.12452/13839
dc.identifier.volume102en_US
dc.identifier.wosWOS:000455499700003en_US
dc.identifier.wosqualityQ3en_US
dc.indekslendigikaynakWeb of Scienceen_US
dc.indekslendigikaynakScopusen_US
dc.indekslendigikaynakPubMeden_US
dc.language.isoenen_US
dc.publisherWileyen_US
dc.relation.ispartofEuropean Journal Of Haematologyen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/closedAccessen_US
dc.subjectHemoglobinopathyen_US
dc.subjectIron Chelationen_US
dc.subjectIron Overloaden_US
dc.subjectPediatricen_US
dc.subjectTransfusionen_US
dc.titleDeferasirox in children with transfusion-dependent thalassemia or sickle cell anemia: A large cohort real-life experience from Turkey (REACH-THEM)en_US
dc.typeArticleen_US

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