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  • Title: Deferasirox in children with transfusion-dependent thalassemia or sickle cell anemia: A large cohort real-life experience from Turkey (REACH-THEM).
    Author: Antmen B, Karakaş Z, Yeşilipek MA, Küpesiz OA, Şaşmaz İ, Uygun V, Kurtoğlu E, Oktay G, Aydogan G, Akın M, Salcioglu Z, Vergin C, Kazancı EG, Ünal S, Çalışkan Ü, Aral YZ, Türkkan E, Meral Güneş A, Tunç B, Gümrük F, Ayhan AC, Söker M, Koç A, Oymak Y, Ertem M, Timur Ç, Yıldırmak Y, İrken G, Apak H, Biner B, Eren TG, Işık Balcı Y, Koçak Ü, Karasu G, Akkaynak D, Patıroğlu T.
    Journal: Eur J Haematol; 2019 Feb; 102(2):123-130. PubMed ID: 30300449.
    Abstract:
    OBJECTIVES: 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 μg/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 μg/L (P < 0.001). Serum ferritin decreases were noted in TDT (1804.9 to 1241 μg/L), SCA (1655.5 to 1260 μg/L), and across age groups of 2-6 years (1971.5 to 1499 μg/L), 7-12 years (1688.5 to 1159.8 μg/L), and 13-18 years (1496.5 to 1107 μg/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.
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