Department of Pediatric Hematology, Ankara Dışkapı Child Health and Diseases Hematology Oncology Training and Research Hospital, Ankara, Turkey.
Pediatr Hematol Oncol. 2020 Sep;37(6):455-464. doi: 10.1080/08880018.2020.1734124. Epub 2020 Mar 5.
Endocrine system dysfunctions are the significant complications of excessive iron overload in beta thalassemia patients. The aim of this study was to evaluate the long-term effect of chelation with deferasirox on endocrine complications. The study group consisted of children with beta thalassemia who had been evaluated for the growth and pubertal development, bone metabolism, thyroid/parathyroid functions, glucose metabolism dysfunctions in the department of pediatric hematology of Ankara Dışkapı Child Health and Diseases Hematology Oncology Training And Research Hospital between 2009-2011 and reevaluated after deferasirox chelation therapy in 2018. Thirty-one transfusion dependent beta-thalassemia patients were enrolled for the study. Seventeen (54.8%) patients were male and the mean age was 16.9 ± 3.8 (9-23) years. Splenectomy was performed in 11 patients (35.5%). In the initial evaluation, 26 patients (84%) received deferoxamine and/or deferiprone and five (17%) patients received deferasirox as a chelator; in the final evaluation all patients were receiving deferasirox. The mean duration of deferasirox treatment was 5.9 ± 2.02 years (1-10 years). Of the 26 patients who had endocrine complications between 2009-2011, 18 were recovered. In the final evaluation, eight patients (25%) developed new endocrinopathies. The frequency of endocrine complications seen before the deferasirox treatment (83%) was higher than the frequency of complications while receiving deferasirox treatment (25.8%) (p < 0,05). In this study, it was determined that both existing endocrine abnormalities were reduced and recent developed problems were less likely with long-term deferasirox treatment in thalassemia patients.
内分泌系统功能紊乱是β地中海贫血患者铁过载过多的重要并发症。本研究旨在评估地拉罗司螯合治疗对内分泌并发症的长期影响。研究组由安卡拉 Dışkapı 儿童健康和疾病血液学肿瘤学培训和研究医院儿科血液科于 2009-2011 年评估的生长和青春期发育、骨代谢、甲状腺/甲状旁腺功能、葡萄糖代谢紊乱的β地中海贫血患儿组成，并在 2018 年接受地拉罗司螯合治疗后重新评估。31 例依赖输血的β地中海贫血患者入组本研究。17 例（54.8%）为男性，平均年龄为 16.9±3.8（9-23）岁。11 例（35.5%）患者行脾切除术。在初始评估时，26 例（84%）患者接受去铁胺和/或地拉罗司治疗，5 例（17%）患者接受地拉罗司作为螯合剂；在最终评估时所有患者均接受地拉罗司治疗。地拉罗司治疗的平均时间为 5.9±2.02 年（1-10 年）。在 2009-2011 年间有内分泌并发症的 26 例患者中，18 例恢复。在最终评估时，8 例（25%）患者新出现内分泌疾病。在开始地拉罗司治疗前（83%）出现内分泌并发症的频率高于接受地拉罗司治疗时（25.8%）的频率（p<0.05）。本研究表明，长期接受地拉罗司治疗可降低地中海贫血患者现有的内分泌异常发生率，并减少新发生的问题。
