All-Trans-Retinoic Acid (ATRA): Pediatric Acute Promyelocytic Leukemia

Abstract

Acute promyelocytic leukemia (APL) is a distinct subtype of acute myeloblastic leukemia (AML). In this report, we present the clinical features, management, and outcome of pediatric patients with APL treated with all-trans-retinoic acid (ATRA). Of 52 newly diagnosed cases of APL between February 1992 and December 1996, 15 were in the pediatric age group (younger than 15 years). Four patients were treated with ATRA alone and 11 were allocated to receive ATRA followed by chemotherapy. Eighty-six percent of the patients achieved a complete response. The patients who received ATRA alone as maintenance therapy had relapses with a median duration of remission of 8 months (range 6-12). The patients who received ATRA, followed by consolidation chemotherapy, had a prolonged duration of remission, with a median of 20 months (range 13-28). In addition, rapid correction of coagulopathy was observed in these patients. The median duration for correction of coagulopathy was 7 days (range 5-11) and the median duration for recovery from neutropenia after chemotherapy was 10 days (range 7-20). Two major side effects of ATRA were hyperleukocytosis and retinoic acid syndrome. Significantly prolonged disease-free survival was seen in patients who received ATRA with chemotherapy. APL is not uncommon in the pediatric age group. ATRA was well-tolerated by these patients. Consolidation with chemotherapy helps in prolonging the disease-free survival in patients with APL in comparison to treatment with ATRA alone.

Treatment with all-trans retinoic acid in acute promyelocytic leukemia reduces early deaths in children

“…According to Bapna et al [2] and Fenaux et al [10], the early death rate was significantly reduced by the treatment with ATRA.…”

Pharmacology of all‐trans‐retinoic acid in children with acute promyelocytic leukemia

“…Along with the altogether lower incidence of AML in children compared to adults (AML only accounts for 15% of acute leukemia cases in children compared to 85% of acute leukemia cases in adults), APL is a comparatively rare event in children. Thus, despite the first report on the efficacy of ATRA in APL by Huang et al in 1988 [2] and apart from case reports, only a few studies have been reported supporting the benefits of ATRA in the treatment of childhood APL [29,33,34].…”

Acute promyelocytic leukemia: evolving therapeutic strategies

“…[63][64][65][66][67][68] The results of contemporary treatment of children with APL also appear similar to those reported in adults. The CR rates for combination ATRA and anthracycline-based therapy have been reported to be 79% to 88% [68][69][70][71] and long-term EFS of 64% to 76%. [69][70][71][72] There are, however, some special considerations in treating children with APL.…”

“…The CR rates for combination ATRA and anthracycline-based therapy have been reported to be 79% to 88% [68][69][70][71] and long-term EFS of 64% to 76%. [69][70][71][72] There are, however, some special considerations in treating children with APL. The first special feature concerns the difficulty of some young patients to reliably swallow capsules.…”