恩瑞格（Deferasirox）疗效怎样呢？

It is used to treat chronic iron overload in beta-thalassemia patients older than 6 years old due to frequent blood transfusions (monthly administration of concentrated red blood cells ≥ 7 mL/kg). For children under 6 years old and iron overload caused by other transfusion-dependent diseases, Deferasirox has limited safety and efficacy data for Chinese patients, and it is recommended to use it with caution based on the specific conditions of the patient.

How effective is Deferasirox? 

To study the iron-removing efficacy and safety of Deferasirox in patients with aplastic anemia (AA) associated with iron overload. Methods: The iron-removing efficacy of 64 AA patients with iron overload after 12 months of treatment with Deferasirox was analyzed, and the safety was evaluated. Results The starting dose of Deferasirox in all patients was 20.0 mg·kg-1·d-1, and the average dose was (18.6±3.60) mg·kg-1·d-1. After 12 months of treatment, the median serum ferritin (SF) level dropped from the baseline of 4 924 (2 718 to 6 765) μg/L (64 cases) to 3 036 (1 474 to 5 551) μg/L (23 cases), a decrease of 38%, and the median SF reduction was 651 (126 to 2 125) μg/L; the median SF level of 23 patients who completed 12 months of treatment dropped from the baseline of 5 271 (3 420 ~ 8 278) μg/L to 3 036 (1 474 ~ 5 551) μg/L, a decrease of 42%, and the median SF reduction was 1 167 (580 ~ 4 806) μg/L.

Increased serum creatinine (40.98%) and gastrointestinal discomfort (40.98%) were the most important adverse events during Deferasirox treatment, followed by increased liver aminotransferases (ALT: 21.31%; AST: 13.11%) and proteinuria (24.59%). The increase in serum creatinine is reversible and non-progressive.

For the 38 patients who were concurrently taking cyclosporine, 12 patients (31.8%) had creatinine values >the upper limit of normal (ULN) for 2 consecutive times, 10 patients (26.3%) had creatinine values >1.33 baseline value for 2 consecutive times, and only 1 patient (2.6%) had serum creatinine that increased beyond the 1.33 baseline value and exceeded the ULN. For AST and ALT, no patient throughout the study had two post-baseline values ​​>5×ULN or >10×ULN. For patients with baseline PLT levels below 50 × 109/L, median PLT did not decrease during Deferasirox treatment. Conclusion: Treatment of AA patients with iron overload can achieve good iron removal efficacy, the drug is well tolerated, and there are no clinically uncontrollable serious adverse events.