Realgar-Indigo naturalis formula (RIF), an oral traditional Chinese medicine mainly containing Realgar (As4S4), is highly effective in treating adult acute promyelocytic leukemia (APL). However, the treatment efficacy and safety of RIF have not been verified in pediatric patients. SCCLG-APL group conducted a multicenter randomized non-inferiority trial to determine whether intravenous arsenic trioxide (ATO) can be substituted by oral RIF in treating pediatric APL. Of 176 eligible patients enrolled, 91 and 85 were randomized to ATO and RIF groups, respectively. Patients were treated with the risk-adapted protocol. Induction, consolidation, and 96-week maintenance treatment contained all-trans-retinoic acid and low-intensity chemotherapy, and either ATO or RIF. The primary endpoint was 5-year event-free survival (EFS). The secondary endpoints were adverse events and hospital days. After a median 6-year follow-up, the 5-year EFS was 97.6% in both groups. However, the RIF group had significantly shorter hospital stays and lower incidence of infection and tended to have less cardiac toxicity. All 4 relapses occurred within 1.5 years after completion of maintenance therapy. No long-term arsenic retentions were observed in either group. Substituting oral RIF for ATO maintains treatment efficacy while reducing hospitalization and adverse events in treating pediatric APL patients, which may be a future treatment strategy for APL.
雄黄-青黛复方（RIF）是一种以雄黄（四硫化四砷）为主要成分的口服中药制剂，对成人急性早幼粒细胞白血病（APL）具有显著疗效。然而，该治疗方案在儿童患者中的疗效与安全性尚未得到验证。为明确口服RIF能否替代静脉注射三氧化二砷（ATO）用于儿童APL治疗，儿童白血病协作组-急性早幼粒细胞白血病学组开展了一项多中心随机非劣效性试验。研究共纳入176例符合条件患者，其中91例随机分配至ATO组，85例分配至RIF组。所有患者均接受风险分层治疗策略，在诱导治疗、巩固治疗及96周的维持治疗中，均联合使用全反式维甲酸、低强度化疗及相应分组药物（ATO或RIF）。主要研究终点为5年无事件生存率，次要终点包括不良事件发生情况及住院时长。中位随访6年后，两组5年无事件生存率均为97.6%。但RIF组住院时间显著缩短，感染发生率更低，且表现出心脏毒性减轻的趋势。所有4例复发均发生在维持治疗结束后1.5年内。两组均未观察到长期砷蓄积现象。在儿童APL治疗中，以口服RIF替代ATO可在保持疗效的同时减少住院时间及不良事件，这可能成为未来的治疗策略。
肿瘤(癌症)患者之家