Safety and Efficacy of the New Combination Iron Chelation Regimens in Patients with Transfusion-Dependent Thalassemia and Severe Iron Overload.

Autor: Origa R; 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., Cinus M; Dipartimento di Scienze Mediche e Sanità Pubblica, Università di Cagliari, Cittadella Universitaria di Monserrato-Blocco I, SS 554 Bivio Sestu, 09042 Monserrato, Italy., Pilia MP; SSD Talassemia, Ospedale Pediatrico Microcitemico 'Antonio Cao', Via Jenner s.n., 09121 Cagliari, Italy., Gianesin B; Fondazione For Anemia, Via Garibaldi 7 C3, 16124 Genova, Italy., Zappu A; SSD Talassemia, Ospedale Pediatrico Microcitemico 'Antonio Cao', Via Jenner s.n., 09121 Cagliari, Italy., Orecchia V; SSD Talassemia, Ospedale Pediatrico Microcitemico 'Antonio Cao', Via Jenner s.n., 09121 Cagliari, Italy., Clemente MG; SSD Talassemia, Ospedale Pediatrico Microcitemico 'Antonio Cao', Via Jenner s.n., 09121 Cagliari, Italy., Pitturru C; SSD Talassemia, Ospedale Pediatrico Microcitemico 'Antonio Cao', Via Jenner s.n., 09121 Cagliari, Italy., Denotti AR; SSD Talassemia, Ospedale Pediatrico Microcitemico 'Antonio Cao', Via Jenner s.n., 09121 Cagliari, Italy., Corongiu F; SSD Talassemia, Ospedale Pediatrico Microcitemico 'Antonio Cao', Via Jenner s.n., 09121 Cagliari, Italy., Piras S; SSD Talassemia, Ospedale Pediatrico Microcitemico 'Antonio Cao', Via Jenner s.n., 09121 Cagliari, Italy., Barella S; SSD Talassemia, Ospedale Pediatrico Microcitemico 'Antonio Cao', Via Jenner s.n., 09121 Cagliari, Italy.
Jazyk: angličtina
Zdroj: Journal of clinical medicine [J Clin Med] 2022 Apr 03; Vol. 11 (7). Date of Electronic Publication: 2022 Apr 03.
DOI: 10.3390/jcm11072010
Abstrakt: 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.
Databáze: MEDLINE
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