Oral Realgar-Indigo Naturalis Formula Treatment for Acute Promyelocytic Leukemia in Children: A Randomized, Control Clinical Trial

Abstract

Objective. To analyze the efficacy, safety, and economy of RIF compared with intravenous arsenic trioxide (ATO) for the induction and consolidation therapy of pediatric APL. Materials and Methods. In this randomized control clinical trial (NCT02200978), children with newly diagnosed APL from June 2013 to December 2017 were randomly divided into RIF and ATO groups. The groups were treated with RIF or ATO in combination with all-trans retinoic acid (ARTA) and conventional chemotherapeutic drugs during induction and consolidation therapy. Results. Ninteen patients were enrolled, including eight in the RIF group and 11 in the ATO group. After induction therapy, the bone marrow morphologic complete remission (CR) rate, the median time to CR, and molecular remission (promyelocytic leukemia protein (PML)/retinoic acid receptor α (RARα) conversion) rates showed no significant differences between patients in the RIF versus ATO groups (100% vs. 100%, $p=1.000$ ; 22 vs. 24 days, $p=0.395$ ; 28.5% vs. 54.5%, $p=0.367$ , resp.). After consolidation therapy, the molecular remission rate was 100% in both groups. At the end of more than two years of follow-up, the disease-free survival (DFS) rate was 100% in both groups. Conclusion. Oral RIF can achieve similar efficacy to intravenous ATO for APL in children with good safety, less toxicity, fewer side effects, and fewer inpatient days. Therefore, oral RIF can be used as an alternative to intravenous ATO for the treatment of APL in children.