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

Autor:	Diyar Z. Akkaynak, Yeşim Oymak, Gonul Aydogan, Tuğba Gürleyen Eren, Gülsün Karasu, Bahattin Tunç, Fatma Gumruk, Selma Unal, Umran Caliskan, Turkan Patiroglu, Adalet Meral Güneş, Zafer Salcioglu, Ahmet Koç, Yusuf Ziya Aral, Yasemin Isik Balci, Mehmet Akin, Aylin Canbolat Ayhan, Vedat Uygun, Osman Alphan Küpesiz, Gönül Oktay, Canan Vergin, Betül Biner, İlgen Şaşmaz, Mehmet Ertem, Hilmi Apak, Emine Türkkan, Yıldız Yildirmak, Cetin Timur, Elif Güler Kazanci, Gülersu Irken, Ülker Koçak, Murat Söker, Erdal Kurtoğlu, Mehmet Akif Yesilipek, Bülent Antmen, Zeynep Karakas
Přispěvatelé:	Çukurova Üniversitesi
Jazyk:	angličtina
Rok vydání:	2019
Předmět:	Male thalassemia drug safety creatinine blood level Turkey Thalassemia Gastroenterology preschool child Cohort Studies chemistry.chemical_compound 0302 clinical medicine iron Prospective cohort study Child iron chelation chelation therapy adult creatinine kidney tubule disorder clinical trial Hematology General Medicine biological marker cohort analysis Sickle cell anemia Hemoglobinopathy female Treatment Outcome priority journal 030220 oncology & carcinogenesis monotherapy Child Preschool Cohort Female liver enzyme deferasirox medicine.drug medicine.medical_specialty Iron Overload erythrocyte transfusion side effect Adolescent Iron complication Anemia Sickle Cell protein urine level blood transfusion Iron Chelating Agents Anemia Sickle Cell/*complications/therapy Biomarkers Blood Transfusion Deferasirox/administration & dosage/adverse effects/*therapeutic use Ferritins/blood/metabolism Humans Iron/blood/metabolism Iron Chelating Agents/administration & dosage/adverse effects/*therapeutic use Iron Overload/*drug therapy/*etiology/metabolism Thalassemia/*complications/therapy Article enzyme blood level 03 medical and health sciences sickle cell anemia blood turkey (bird) Internal medicine medicine hemoglobinopathy follow up human iron overload drug dose reduction Adverse effect transfusion Creatinine business.industry Deferasirox ferritin abdominal pain iron chelating agent medicine.disease school child major clinical study drug efficacy ferritin blood level pediatric multicenter study chemistry Ferritins observational study proteinuria business metabolism 030215 immunology
Popis:	PubMedID: 30300449 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
Databáze:	OpenAIRE
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