All-trans retinoic acid (ATRA), an oral retinoic acid derivative, revolutionized the treatment and prognosis of acute promyelocytic leukemia (APL) and anticipated the current age of targeted cancer therapy. The past 25 years have seen an international effort to design multicenter clinical trials that identify the optimal use of ATRA in patients with APL. Indeed, since ATRA is so effective, contemporary clinical trials aim to de-escalate conventional cytotoxic chemotherapy, as has been the case with other highly curable malignancies such as childhood acute lymphoblastic leukemia and Hodgkin’s lymphoma. In this chapter, we review and reflect upon the seminal clinical trials that have led to the current adoption of ATRA as the standard of care for treating APL.
- Acute promyelocytic leukemia
- All-trans retinoic acid
ASJC Scopus subject areas
- Pharmacology, Toxicology and Pharmaceutics(all)
- Biochemistry, Genetics and Molecular Biology(all)
- Immunology and Microbiology(all)