Unità Operativa Semplice Dipartimentale Malattie Rare del Globulo Rosso, Azienda Ospedaliera di Rilievo Nazionale 'A. Cardarelli', Napoli, Italy.
Magnetic Resonance Imaging Unit, Fondazione G. Monasterio CNR-Regione Toscana, Pisa, Italy.
Br J Haematol. 2020 Oct;191(1):107-114. doi: 10.1111/bjh.16753. Epub 2020 May 19.
We longitudinally evaluated the effects of regular blood transfusions (BTs), in the real-life context of the Myocardial Iron Overload in Thalassaemia network, in patients with thalassaemia intermedia (TI). We considered 88 patients with TI (52 females) who started regular BTs after the age of 18 years. Magnetic resonance imaging was used to quantify iron overload and biventricular function. For 56·8% of the patients there were more than two indications for the transition to regular BTs, with anaemia present in 94·0% of the cases. A significant decrease in nucleated red blood cells, platelets, lactate dehydrogenase, bilirubin, and uric acid levels was detected 6 months after starting regular BTs. After the transition to the regular BT regimen there was a significant increase only in the frequency of hypothyroidism and osteopenia, and a significant decrease in liver iron and cardiac index. The percentage of chelated patients increased significantly after starting regular BTs. The decision to regularly transfuse patients with TI may represent a way to prevent or slow down the natural progression of the disease, despite the more complex initial management.
我们在铁过载地中海贫血网络的实际环境中对中间型地中海贫血（TI）患者进行了定期输血（BTs）的效果进行了纵向评估。我们考虑了 88 名 TI 患者（52 名女性），他们在 18 岁后开始定期接受 BTs。磁共振成像用于定量铁过载和双心室功能。对于 56.8%的患者，有两个以上的指征需要过渡到定期 BTs，其中 94.0%的病例存在贫血。在开始定期 BTs 后 6 个月，发现有核红细胞、血小板、乳酸脱氢酶、胆红素和尿酸水平显著下降。在过渡到定期 BT 方案后，仅甲状腺功能减退症和骨质疏松症的频率显著增加，而肝铁和心脏指数显著下降。开始定期 BTs 后，螯合治疗的患者比例显著增加。定期为 TI 患者输血的决定可能代表了一种预防或减缓疾病自然进展的方法，尽管初始管理更加复杂。
