Department of Internal Medicine, American University of Beirut, Riad El Solh 1107 2020, Beirut, Lebanon.
Expert Rev Hematol. 2013 Oct;6(5):495-509. doi: 10.1586/17474086.2013.827411. Epub 2013 Oct 2.
Non-transfusion-dependent thalassemia (NTDT) defines a group of patients who do not require regular transfusions for survival, but are at significant risk of iron accumulation from underlying disease-related mechanisms distinct from transfusional iron overload. Management of iron overload in NTDT has received little attention compared with that of β-thalassemia major, despite evidence of significant iron-induced complications with advancing age. The efficacy and safety of the iron chelator deferasirox in NTDT has been evaluated in two pilot studies and the first prospective, randomized, placebo-controlled study (THALASSA) of any chelator in NTDT. Treatment with deferasirox for up to 2 years yielded a sustained reduction in iron burden, with a clinically manageable safety profile. Following these trial data, deferasirox is the first iron chelator approved for use in NTDT patients, and with NTDT guidelines now available, physicians are better equipped to achieve effective monitoring and management of iron burden in NTDT.
非输血依赖型地中海贫血症（NTDT）定义了一组患者，他们为了生存不需要定期输血，但由于与输血性铁过载不同的潜在疾病相关机制，存在着明显的铁蓄积风险。与重型β地中海贫血症相比，NTDT 中的铁过载管理受到的关注较少，尽管随着年龄的增长，有大量证据表明铁诱导的并发症显著增加。两种试点研究和第一项前瞻性、随机、安慰剂对照研究（THALASSA）评估了铁螯合剂地拉罗司在 NTDT 中的疗效和安全性。地拉罗司治疗长达 2 年可持续降低铁负荷，且安全性具有临床可管理性。基于这些试验数据，地拉罗司成为首个获准用于 NTDT 患者的铁螯合剂，并且随着 NTDT 指南的发布，医生能够更好地监测和管理 NTDT 中的铁负荷。
