Department of Pediatrics, Faculty of Medicine, Cairo University, Cairo, Egypt.
Department of Pediatric Hematology, Pediatric Hospital of Cairo University, Cairo, Egypt.
Pediatr Blood Cancer. 2024 Jan;71(1):e30711. doi: 10.1002/pbc.30711. Epub 2023 Oct 9.
Children with sickle cell disease (SCD) who are chronically transfused often, require iron chelation therapy. There are limited data that allow for comparison of the efficacy and safety of the iron chelator deferiprone versus deferoxamine in children with SCD.
This post hoc analysis of the phase 3b/4, randomized, open-label FIRST (Ferriprox in Patients with IRon Overload in Sickle Cell Disease Trial) study (NCT02041299) included patients 17 years and younger with SCD or other anemias receiving deferiprone or deferoxamine.
Overall, 142 patients were evaluated; mean ages were 10.5 and 11.7 years in the deferiprone and deferoxamine groups, respectively. At 12 months: mean change from baseline in liver iron concentration was -3.3 mg/g dry weight (dw) with deferiprone and -3.4 mg/g dw with deferoxamine (p = .8216); relative mean change (coefficient of variation %) in log cardiac T2* magnetic resonance imaging was 1.02 (21.8%) with deferiprone and 0.95 (19.5%) with deferoxamine (p = .0717); and the mean (standard error) change in serum ferritin levels was -133.0 (200.3) μg/L with deferiprone and -467.1 (244.1) μg/L with deferoxamine (p = .2924). The most common deferiprone-related adverse events (AEs) were upper abdominal pain (20.2%), vomiting (13.8%), pyrexia (9.6%), decreased neutrophil count (9.6%), increased alanine aminotransferase (ALT; 9.6%), and increased aspartate aminotransferase (AST; 9.6%). All cases of increased ALT, increased AST, and neutropenia resolved, most without intervention.
This post hoc analysis of pediatric patients from FIRST corroborated previous findings in adults that deferiprone is comparable to deferoxamine in reducing iron overload. No new safety concerns were observed. Deferiprone is an oral chelation option that could improve adherence and outcomes in children.
经常接受慢性输血的镰状细胞病 (SCD) 患儿需要铁螯合治疗。目前仅有有限的数据可以比较 SCD 患儿使用去铁酮和去铁胺的疗效和安全性。
这项 3b/4 期、随机、开放性 FIRST（Ferriprox 在患有铁过载的 Sickle Cell 疾病患者中的试验）研究的事后分析（NCT02041299）纳入了接受去铁酮或去铁胺治疗的 17 岁及以下的 SCD 或其他贫血患儿。
总体而言，共有 142 名患儿接受了评估；去铁酮组和去铁胺组的平均年龄分别为 10.5 岁和 11.7 岁。12 个月时：去铁酮组和去铁胺组肝铁浓度自基线的平均变化分别为 -3.3mg/g 干重 (dw) 和 -3.4mg/g dw（p=0.8216）；心脏 T2*磁共振成像的相对平均变化（变异系数%）分别为 1.02（21.8%）和 0.95（19.5%）（p=0.0717）；去铁酮组和去铁胺组血清铁蛋白水平的平均（标准误差）变化分别为 -133.0（200.3）μg/L 和 -467.1（244.1）μg/L（p=0.2924）。最常见的去铁酮相关不良事件（AE）为上腹痛（20.2%）、呕吐（13.8%）、发热（9.6%）、中性粒细胞计数减少（9.6%）、丙氨酸氨基转移酶（ALT；9.6%）升高和天冬氨酸氨基转移酶（AST；9.6%）升高。所有 ALT、AST 升高和中性粒细胞减少的病例均得到解决，大多数无需干预。
这项 FIRST 儿科患者的事后分析结果与先前在成人中观察到的结果一致，即去铁酮在降低铁过载方面与去铁胺相当。未观察到新的安全性问题。去铁酮是一种口服螯合剂，可改善儿童的依从性和治疗结局。
