Deferasirox for the treatment of iron overload in thalassemia ：a rapid health technology assessment / 中国药房

ABSTRACT

OBJECTIVE To eval uate the effectiveness ，safety and economy of deferasir ox for the treatment of iron overload in thalassemia with rapid health technology assessment ，and to provide evidence-based basis for rational clinical use. METHODS Retrieved from Chinese and English database/website as PubMed ，Embase，Cochrane Library ，NHS EED ，CADTH，CNKI and Wanfang database ，health technology assessment （HTA），systematic evaluation/meta-analysis and pharmacological studies about deferasirox versus deferoxamine/deferiprone for the treatment of iron overload in thalassemia were collected from the inception to June 2021. Based on literature screening and data extraction ，the quality of literature about HTA reports ，systematic evaluation/ Meta-analysis and pharmacoeconomic research were evaluated with HTA checklist ，A Measurement Tool to As sess Systematic Reviews，standard scale of economic evaluation report. The effectiveness and safety results were described quantitatively ，and the economic evaluation results were described qualitatively. RESULTS One HTA report ，five systematic evaluation/meta-analysis and five pharmacoeconomic studies were selected from 1 569 literature. Included HTA reports ， systematic evaluation/meta-analysis，pharmacoeconomic studies were high in quality. Most studies reported that 30 mg/（kg·d） deferasirox was E-mail：aydgs@126.com better than deferoxamine in reducing the levels of s erum ferritin and liver iron overload ；ADR induced by deferasirox were mainly gastrointestinal irritation symptoms ，skin itching ，joint pain，transaminase elevation ，etc.，which generally did not affect subsequent treatment. There was no statistical significance in severe ADR between deferoxamine group and deferasirox group [RR ＝0.96，95％CI（0.85，1.08），P＝0.52]. Compared with deferoxamine，deferasirox had higher cost-effectiveness ；but deferasirox was less likely to be cost-effective than deferiprone. CONCLUSIONS Deferasirox has good effectiveness and safety for iron overload in thalassemia ，and has good economic advantages in Britain and Iran ，compared with deferoxamine.