Adis, Level 1, 5 The Warehouse Way, Northcote 0627; Private Bag 65901, Mairangi Bay, 0754, Auckland, New Zealand.
Drugs. 2014 Jun;74(9):1017-27. doi: 10.1007/s40265-014-0238-0.
Deferasirox (Exjade(®)) is a once-daily orally administered iron chelator which has been approved for use in the treatment of transfusional-dependent chronic iron overload since 2005. Based primarily on the findings of the THALASSA (Assessment of Exjade(®) in Non-Transfusion-Dependent THALASSemiA) trial, the approval for deferasirox has recently been expanded to include the management of chronic iron overload in patients with non-transfusion-dependent thalassaemia (NTDT) syndromes. Despite the lack of regular blood transfusions, NTDT patients can still develop clinically relevant iron overload, primarily due to increased gastrointestinal absorption secondary to ineffective erythropoiesis, and may require chelation therapy. The THALASSA trial, the first placebo-controlled clinical trial of an iron chelator in NTDT patients, demonstrated that deferasirox was effective in reducing liver iron and serum ferritin levels in this population. Deferasirox has an acceptable tolerability profile, with the most common adverse events reported in the THALASSA trial being related to mild to moderate gastrointestinal disorders. Although further long-term studies will be required to clearly demonstrate the clinical benefit of chelation therapy in NTDT patients, deferasirox presents a useful tool in the management of iron overload in this population.
地拉罗司（Exjade(®)）是一种每日口服一次的铁螯合剂，自 2005 年以来，已被批准用于治疗输血依赖性慢性铁过载。基于 THALASSA（评估 Exjade(®)在非输血依赖型地中海贫血）试验的结果，地拉罗司的批准最近已扩大到包括非输血依赖型地中海贫血（NTDT）综合征患者慢性铁过载的治疗。尽管 NTDT 患者没有接受常规输血，但他们仍可能发生具有临床意义的铁过载，主要是由于无效的红细胞生成导致胃肠道吸收增加，可能需要螯合治疗。THALASSA 试验是 NTDT 患者中进行的第一项铁螯合剂安慰剂对照临床试验，证明地拉罗司可有效降低该人群的肝脏铁和血清铁蛋白水平。地拉罗司具有可接受的耐受性，THALASSA 试验中报告的最常见不良事件与轻度至中度胃肠道疾病有关。尽管还需要进行进一步的长期研究，以明确证明螯合治疗在 NTDT 患者中的临床获益，但地拉罗司为该人群的铁过载管理提供了一种有用的工具。
