地拉罗司的治疗效果怎样呢?
It is an oral iron chelator. Its primary use is to reduce chronic iron overload in patients receiving chronic transfusions for conditions such as beta-thalassemia and other chronic anemias. This is the first oral drug approved for this purpose in the United States. Deferasirox was approved by the U.S. Food and Drug Administration (FDA) in November 2005. According to the FDA (May 2007), renal failure and cytopenias have been reported in patients receiving deferasirox oral suspension tablet, which was approved in the EU by the European Medicines Agency (EMA) for patients 6 years of age and older with chronic iron overload from repeated transfusions.
Deferasirox dispersible tablets should be taken on an empty stomach at least 30 minutes before a meal, once a day, preferably at the same time every day. Tablets should not be chewed or swallowed whole. Deferasirox dispersible tablets should not be taken together with aluminum-containing antacids, and the dose (mg/kg) needs to be calculated and rounded to the nearest whole tablet. Completely dissolve the tablets in water, apple juice or orange juice (100-200mL) by stirring until a clear suspension is obtained and then drink it. The remaining medicine must be added with a small amount of water, apple juice or orange juice and mixed well before being taken. It is not recommended to dissolve the tablets in carbonated drinks or milk because it will cause foam and delay the dispersion speed.
[Methods] The iron-removing efficacy of 64 AA patients with iron overload after 12 months of deferasirox treatment was analyzed, and the safety was evaluated. Results The starting dose of deferasirox 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 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] The 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.
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