Division of Hematology/Oncology, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.
Am J Hematol. 2014 Jul;89(7):684-8. doi: 10.1002/ajh.23715. Epub 2014 Apr 10.
Oral iron chelators and magnetic resonance imaging (MRI) assessment of heart and liver iron burden have become widely available since the mid 2000s, allowing for improved patient compliance with chelation and noninvasive monitoring of iron levels for titration of therapy. We evaluated the impact of these changes in our center for patients with thalassemia major and transfusional iron overload. This single center, retrospective observational study covered the period from 2005 through 2012. Liver iron content (LIC) was estimated both by a T2* method and by R2 (Ferriscan® ) technique. Cardiac iron was assessed as cT2*. Forty-two patients (55% male) with transfused thalassemia and at least two MRIs were included (median age at first MRI, 17.5 y). Over a mean follow-up period of 5.2 ± 1.9 y, 190 MRIs were performed (median 4.5 per patient). Comparing baseline to last MRI, 63% of patients remained within target ranges for cT2* and LIC, and 13% improved from high values to the target range. Both the median LIC and cT2* (cR2* = 1000/cT2*) status improved over time: LIC 7.3 to 4.5 mg/g dry weight, P = 0.0004; cR2* 33.4 to 28.3 Hz, P = 0.01. Individual responses varied widely. Two patients died of heart failure during the study period. Annual MRI iron assessments and availability of oral chelators both facilitate changes in chelation dose and strategies to optimize care.
自 21 世纪中期以来,口服铁螯合剂和磁共振成像(MRI)评估心脏和肝脏铁负荷已广泛应用,这使得患者对螯合治疗的依从性提高,并能进行非侵入性的铁水平监测,以滴定治疗。我们评估了这些变化对我们中心的地中海贫血和输血性铁过载患者的影响。这项单中心回顾性观察研究涵盖了 2005 年至 2012 年期间。肝脏铁含量(LIC)通过 T2方法和 R2(Ferriscan®)技术进行估计。心脏铁通过 cT2进行评估。共纳入 42 名(55%为男性)接受过输血的地中海贫血患者,这些患者至少接受过两次 MRI(首次 MRI 时的中位年龄为 17.5 岁)。在平均 5.2±1.9 年的随访期间,共进行了 190 次 MRI(中位数为每个患者 4.5 次)。与基线相比,最后一次 MRI 时,63%的患者 cT2和 LIC 仍处于目标范围内,13%的患者从高值降至目标范围内。LIC 和 cT2(cR2*=1000/cT2*)的中位数状态均随时间改善:LIC 从 7.3 降至 4.5 mg/g 干重,P=0.0004;cR2*从 33.4 降至 28.3 Hz,P=0.01。个体反应差异很大。在研究期间,有 2 名患者死于心力衰竭。年度 MRI 铁评估和口服螯合剂的可用性均有助于改变螯合剂量和优化治疗策略。