恩瑞格治疗铁质积聚患者效果如何呢？

It is a new type of oral iron-removing agent, mainly used in 2-year-old patients with chronic iron overload caused by blood transfusion. So, how effective is Enrig in treating patients with iron accumulation?

Therapeutic effects of Enrige in treating iron accumulation:

Study the iron-removing efficacy and safety of Enrig in patients with aplastic anemia (AA) associated with iron overload.

The iron-removing efficacy of 64 AA patients with iron overload after 12 months of treatment with Enrige was analyzed, and the safety was evaluated.

Results: The starting dose of Enriga for 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 main adverse events during treatment with Enriga, 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 50×10^9/L, median PLT did not decrease during treatment with Enriga.

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.