Hammond John, Thompson Alexis A, Fogel Mark A, Hammond Katherine, Kokroko Jolene, Kwiatkowski Janet L
Division of Hematology.
Division of Hematology/Oncology, Ann & Robert H. Lurie Children's Hospital of Chicago.
J Pediatr Hematol Oncol. 2019 Jan;41(1):e47-e50. doi: 10.1097/MPH.0000000000001269.
An open-label, pilot study was conducted to evaluate deferasirox/deferiprone combination chelation therapy in adult patients with transfusion-dependent thalassemia and severe iron overload. Enrollment proved difficult. Nine patients (median age, 27.4 y; ferritin, 4965 ng/mL; liver iron concentration, 28.5 mg/g dry weight; cardiac T2*, 13.3 ms) received treatment. Two were withdrawn for treatment-related adverse effects. Arthralgia (4 patients) and gastrointestinal symptoms (5 patients) were common; no episodes of neutropenia/agranulocytosis occurred. Adherence difficulties were common. Of 6 patients with 12 to 18 months follow-up, 3 showed improvement in cardiac T2* and 2 in liver iron. Combination oral chelation may be effective but adverse effects and adherence challenges may limit efficacy.
开展了一项开放标签的试点研究，以评估去铁斯若/去铁酮联合螯合疗法对依赖输血的成年地中海贫血患者和严重铁过载的疗效。结果发现入组困难。9例患者（中位年龄27.4岁；铁蛋白4965 ng/mL；肝脏铁浓度28.5 mg/g干重；心脏T2为13.3 ms）接受了治疗。2例因治疗相关不良反应退出。关节痛（4例患者）和胃肠道症状（5例患者）较为常见；未发生中性粒细胞减少/粒细胞缺乏症。依从性差很常见。在6例随访12至18个月的患者中，3例心脏T2有所改善，2例肝脏铁有所改善。联合口服螯合疗法可能有效，但不良反应和依从性问题可能会限制疗效。
