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De-Escalating Therapy in Pediatric Acute Promyelocytic Leukemia
High cure rates are achieved in adults with acute promyelocytic leukemia (APML) using combined all-trans retinoic acid (ATRA) and arsenic trioxide, with or without anthracycline chemotherapy. Now, the Children's Oncology Group reports a multinational noninferiority trial of de-escalated induction therapy with ATRA plus arsenic trioxide in patients with APML aged 1 to 21. Standard-risk patients (white blood cell count <10,000/μL) received ATRA plus arsenic trioxide, and high-risk patients (WBC ≥10,000/μL) received the same treatment plus four doses of idarubicin. Following induction, all patients received four cycles of consolidation therapy with ATRA plus arsenic trioxide; no maintenance therapy was given.
During a median follow-up of 2 years, standard-risk patients (n=98) had an event-free survival (EFS) rate of 98% and overall survival (OS) rate of 99%; there was one death during induction therapy and one patient relapsed. Among high-risk patients (n=56), EFS was 96% and OS was 100%; there were no deaths and two relapses. These results were noninferior to those in a historical control group of pediatric patients in earlier chemotherapy-containing clinical trials (the primary endpoint). Toxicities included known side effects of ATRA/arsenic therapy, including leukocytosis, differentiation syndrome, and pseudotumor cerebri; 13% of patients had clinically significant bleeding or thrombosis during induction therapy, with no coagulopathy-related deaths.
This trial indicates the safety and efficacy of ATRA plus arsenic trioxide treatment for children, adolescents, and young adults with APML, with limited dosing of an anthracycline in high-risk patients. Sparing longer-term treatment and chemotherapy will minimize late toxic effects in this young population, and late relapse hopefully will not be observed with longer follow up, given that all patients tested after the second cycle of consolidation treatment were in molecular remission.
Kutny MA et al.
Title: Assessment of arsenic trioxide and all-trans retinoic acid for the treatment of pediatric acute promyelocytic leukemia: A report from the Children's Oncology Group AAML1331 trial.
Source: JAMA Oncol 2021 Nov 11; [e-pub]. (Abstract/FREE Full Text)