Origa Raffaella, Cinus Monia, Pilia Maria Paola, Gianesin Barbara, Zappu Antonietta, Orecchia Valeria, Clemente Maria Grazia, Pitturru Carla, Denotti Anna Rita, Corongiu Francesco, Piras Simona, Barella Susanna
SSD Talassemia, Ospedale Pediatrico Microcitemico 'Antonio Cao', Via Jenner s.n., 09121 Cagliari, Italy.
Dipartimento di Scienze Mediche e Sanità Pubblica, Università di Cagliari, Cittadella Universitaria di Monserrato-Blocco I, SS 554 Bivio Sestu, 09042 Monserrato, Italy.
J Clin Med. 2022 Apr 3;11(7):2010. doi: 10.3390/jcm11072010.
The aim of this study is the evaluation of the safety and the efficacy of long-term combination therapy deferasirox plus desferrioxamine and deferasirox plus deferiprone in a large group of transfusion-dependent thalassemia patients with high values of serum ferritin and/or magnetic resonance, indicative of severe liver and cardiac iron accumulation. Sixteen adults with transfusion-dependent thalassemia were treated simultaneously with deferasirox plus desferrioxamine, while another 42 patients (seven children) were treated with deferasirox plus deferiprone. The hepatic and cardiac iron overload was assessed prior to treatment and then annually with magnetic resonance imaging, and the serum ferritin was measured monthly. Adverse events were checked at each transfusion visit. The safety of both the combinations was consistent with established monotherapies. Both treatments were able to decrease the serum ferritin and liver iron concentration over time, depending on the level of compliance with therapy. Cardiac iron measured as R2* did not significantly change in patients treated with deferasirox plus desferrioxamine. Most patients with MRI indicative of myocardial siderosis at the beginning of treatment reached normal values of cardiac iron at the last determination if treated with deferasirox plus desferrioxamine. The greatest limitation of these therapies was low patient adherence to the two drugs, which is not surprising considering that the need for an intensive chelation is generally linked to previous issues of compliance.
本研究的目的是评估长期联合使用地拉罗司加去铁胺以及地拉罗司加去铁酮对一大组依赖输血的地中海贫血患者的安全性和有效性，这些患者血清铁蛋白值高和/或磁共振检查显示严重的肝脏和心脏铁过载。16名依赖输血的成年地中海贫血患者同时接受地拉罗司加去铁胺治疗，另外42名患者（7名儿童）接受地拉罗司加去铁酮治疗。在治疗前通过磁共振成像评估肝脏和心脏的铁过载情况，随后每年进行一次评估，并每月测量血清铁蛋白。在每次输血就诊时检查不良事件。两种联合治疗的安全性与既定的单一疗法一致。两种治疗均能够随着时间的推移降低血清铁蛋白和肝脏铁浓度，这取决于治疗的依从程度。接受地拉罗司加去铁胺治疗的患者中，以R2*测量的心脏铁含量没有显著变化。如果接受地拉罗司加去铁胺治疗，大多数在治疗开始时磁共振成像显示心肌铁沉积的患者在最后一次测定时心脏铁含量达到正常水平。这些疗法最大的局限性是患者对这两种药物的依从性低，考虑到强化螯合治疗的需求通常与先前的依从性问题有关，这并不奇怪。
