Casale, M; Forni, GL; Cassinerio, E; Pasquali, D; Origa, R; Serra, M; Campisi, S; ... Perrotta, S; + view all Casale, M; Forni, GL; Cassinerio, E; Pasquali, D; Origa, R; Serra, M; Campisi, S; Peluso, A; Renni, R; Cattoni, A; De Michele, E; Allò, M; Poggi, M; Ferrara, F; Di Concilio, R; Sportelli, F; Quarta, A; Putti, MC; Notarangelo, LD; Sau, A; Ladogana, S; Tartaglione, I; Picariello, S; Marcon, A; Sturiale, P; Roberti, D; Lazzarino, AI; Perrotta, S; - view fewer (2021) Risk factors for endocrine complications in transfusion-dependent thalassemia patients on chelation therapy with deferasirox: a risk assessment study from a multicentre nation-wide cohort. Haematologica 10.3324/haematol.2020.272419. (In press).

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Abstract

Transfusion-dependent patients typically develop iron-induced cardiomyopathy, liver disease, and endocrine complications. We aimed to estimate the incidence of endocrine disorders in transfusion-dependent thalassemia (TDT) patients during long-term iron-chelation therapy with deferasirox (DFX).We developed a multicentre follow-up study of 426 TDT patients treated with once-daily DFX for a median duration of 8 years, up to 18.5 years. At baseline, 118, 121, and 187 patients had 0, 1, or ≥2 endocrine diseases respectively. 104 additional endocrine diseases were developed during the follow-up. The overall risk of developing a new endocrine complication within 5 years was 9.7% (95%CI=6.3-13.1). Multiple Cox regression analysis identified 3 key predictors: age showed a positive log-linear effect (adjusted HR for 50% increase=1.2, 95%CI=1.1-1.3, P=0.005), the serum concentration of thyrotropin (TSH) showed a positive linear effect (adjusted HR for 1 mIU/L increase=1.3, 95%CI=1.1-1.4, P.

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