Abstract
Treatment paradigm of acute promyelocytic leukemia (APL) is by no mean the most remarkable story of cancer therapy. Recently, the advent of oral arsenic formulations (oral-arsenic trioxide and Realgar-Indigo Naturalis formula (RIF)) based regimens may provide a therapeutic advance by curing APL with two oral agents. Indeed, the oral RIF plus all-trans-retinoic acid (ATRA) without chemotherapy display highly efficacy in patients with APL. The safety profile of RIF plus ATRA make possible to treat APL patients in a home-based manner during postremission therapy. To our knowledge, RIF was the first commercially available oral arsenic agent approved in China. The RIF plus ATRA regimens are becoming a preferred frontline care for APL in China. In this review, we will discuss the history, current evidences and challengers of RIF-based strategies in APL. More and more APL patients may enjoy a cure with a normal quality-of-life after induction in the near future.
Highlights
Acute promyelocytic leukemia (APL) was first described in 1923 by Swiss hematologist Dr Albert Alder [1]
Since the oral Realgar-Indigo Naturalis Formula (RIF) plus all-trans-retinoic acid (ATRA) may be easier for pediatric patients to take, the South China Children Leukemia Group (SCCLG) conducted a randomized study to compare the efficacy, safety and the number of hospital days between RIF or f intravenous arsenic trioxide (ATO)-based therapies in pediatric acute promyelocytic leukemia (APL) [20]
Over 8,000 patients with APL have been treated with oral RIF in China
Summary
Acute promyelocytic leukemia (APL) was first described in 1923 by Swiss hematologist Dr Albert Alder [1]. The initial breakthrough was the uncover of the unique chromosomal aberration t(15, 17) (q22;q12) in APL by Dr J Rowley in 1970s [4] It was not until 1990, the landmark molecular fusion of the promyelocytic leukemia (PML) and retinoic acid receptor alpha (RARA) gene was identified [5]. The targeted therapy model assumed to eradicate the APL-initiating cells and reduce the need for chemotherapy [14] In line with this preclinical model, the followup trials demonstrated that the ATO plus ATRA targeted approach could definitively cure APL [14]. The frequent main side effects were epigastria discomfort, abdominal distention, mild diarrhea, skin rash, and liver enzyme increase This is the first study reported the efficacy and safety of RIF-based therapy in APL.
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