Pharmacokinetics, efficacy, and safety of arsenic formulations in acute promyelocytic leukemia treatment.

INTRODUCTION: Acute promyelocytic leukemia (APL) is a highly curable subtype of AML, largely due to the introduction of differentiating therapy with all-trans retinoic acid and arsenic trioxide. While intravenous arsenic trioxide (ATO) is considered the standard-of-care in the United States, its prolonged administration and monitoring requirements pose logistical challenges that require high healthcare resource utilization and negatively impact quality of life.

AREAS COVERED: This review summarizes the development and clinical evaluation of intravenous and oral arsenic formulations in APL. We discuss the pharmacokinetics, safety, and efficacy of oral arsenic compared with intravenous ATO across populations, including children and patients with obesity or renal impairment. A PubMed search using the terms 'oral arsenic,' 'arsenic trioxide,' 'arsenic formulations,' 'acute promyelocytic leukemia,' and 'pharmacokinetics' was used to compile data. It draws on current pharmacokinetic and clinical evidence, including data from retrospective publications, phase 3 trials, meta-analyses, and long-term follow-up studies.

EXPERT OPTION: Oral arsenic formulations achieve comparable molecular remission, long-term survival, and toxicity profiles to intravenous ATO. These oral formulations may offer meaningful advantages in quality of life, cost, and overall availability of optimal treatment. Continued clinical trials are expected to further define oral arsenic's role in frontline therapy in the United States.