Yesilipek MA, et al. - A prospective, phase II, multicenter, single-arm study was performed to assess the efficacy and safety of deferasirox in patients (aged >2 to <18 years) with β-thalassemia major (TM) who have undergone hematopoietic stem cell transplantation (HSCT) and had evidence of iron overload (serum ferritin >1000 μg/L, cardiac MRI T2* <20 ms, or liver iron concentration [LIC; by MRI R2] ≥5 mg/g). Researchers noticed that in these children, deferasirox treatment at doses up to 20 mg/kg/day reduced iron burden, with a manageable safety profile.

• Deferasirox at an initial dose of 10 mg/kg/day, with up-titration to a maximum of 20 mg/kg/day was administered to the patients.
• The study continued for 52 weeks.
• Researchers included a total of 27 patients (mean age 9.1 ± 3.8 years, 70.4% male).
• As a result of loss to follow-up, 1 (3.7%) patient discontinued the study.
• The intended dose of 20 mg/kg/day was achieved by most of the patients (n=20, 74.1%). They noticed no deaths.
• During the study, they noticed 134 adverse events (AEs) in 25 (92.6%) patients.
• Grade 1 or 2 AEs were observed in most of patients; only 8 (29.6%) patients showed grade 3 AEs.
• In 4 (14.8%) patients, only 10 AEs suspected to be related to deferasirox were noticed(ALT/AST increase, n=4; urinary tract infection, n=1).
• For 6 AEs occurring in 4 (14.8%) patients, the deferasirox dose was adjusted or interrupted.
• In 3 (11.1%) patients, a total of 6 serious AEs occurred, which seemed not related to deferasirox.
• From baseline to week 52 , a decrease in median ALT (30.0 to 17.0 IU/L) and AST (35.5 to 26.0 IU/L) was noticed.
• At baseline and week 5, median serum creatinine and cystatin C were similar.
• Following 52 weeks of therapy, a continuous and significant decline was noticed in median serum ferritin level from 1718.0 μg/L at baseline to 845.3 μg/L (P<.001); 9 (33.3%) patients achieved a level of <500 μg/L.
• A marked decrease in median LIC (8.6 to 4.1 mg/g; P < .001) and an increase in median cardiac T2* (26.0 to 28.0 ms; P=.520) were also noticed from baseline to week 52.