Antmen, BülentKarakaş, ZeynepYeşilipek, Mehmet AkifKüpesiz, Osman AlphanŞaşmaz, IlgenUygun, VedatKurtoğlu, ErdalOktay, GönülAydogan, GönülAkın, MehmetSalcıoğlu, ZaferVergin, CananKazancı, Elif GülerÜnal, SelmaÇalışkan, ÜmranAral, Yusuf ZiyaTürkkan, EmineGüneş, Adalet MeralTunc, BahattinGümrük, FatmaAyhan, Aylin CanbolatSöker, MuratKoç, AhmetOymak, YeşimErtem, MehmetTimur, ÇetinYıldırmak, Yıldızİrken, GülersuApak, HilmiBiner, BetülEren, Tuğba GürleyenBalcı, Yasemin IşikKoçak, UlkerKarasu, GulsunAkkaynak, DiyarPatıroğlu, Türkan2024-07-242024-07-242019-02-010902-4441https://doi.org/10.1111/ejh.13180https://onlinelibrary.wiley.com/doi/10.1111/ejh.13180https://hdl.handle.net/11452/43415Objectives 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.eninfo:eu-repo/semantics/closedAccessMedically treated patientsIron-overloaded patientsPediatric-patientsChelation-therapyDiseaseDeferoxamineEscalatorSurvivalEfficacySafetyHemoglobinopathyIron chelationIron overloadPediatricTransfusionScience & technologyLife sciences & biomedicineHematologyArticle000455499700003123130102210.1111/ejh.131801600-0609