Matthew A Kutny1, Susan Geyer2, Kristina M Laumann3, John Gregory4, Cheryl L Willman5, Wendy Stock6, Richard A Larson6, Bayard L Powell7, James H Feusner8. 1. Department of Pediatrics, Division of Hematology/Oncology, University of Alabama at Birmingham, Birmingham, Alabama. 2. Health Informatics Institute, University of South Florida, Tampa, Florida. 3. Alliance Statistics and Data Center, Mayo Clinic, Rochester, Minnesota. 4. Atlantic Health System, Goryeb Children's Hospital, Morristown, New Jersey. 5. Department of Pathology, School of Medicine, University of New Mexico Cancer Center, Albuquerque, New Mexico. 6. Section of Hematology/Oncology, Department of Medicine, University of Chicago, Chicago, Illinois. 7. Section on Hematology and Oncology, Comprehensive Cancer Center of Wake Forest University School of Medicine, Winston-Salem, North Carolina. 8. Division of Hematology/Oncology, Children's Hospital and Research Center Oakland, Oakland, California.
Abstract
BACKGROUND:Acute promyelocytic leukemia (APL) is a unique leukemia subtype requiring specialized treatment including all-trans retinoic acid (ATRA). A prior report demonstrated worse outcome among young children <5 years old compared with older children. METHODS: We evaluated outcomes for pediatric patients (<18 years old; N = 83) with APL treated on North American intergroup study CALGB 9710 at Children's Oncology Group sites. Induction and consolidation included ATRA, cytarabine, and anthracyclines. Patients ≥15 years old were randomized to addition of arsenic trioxide (ATO) consolidation. All patients were randomized to ATRA maintenance with versus without oral chemotherapy. RESULTS: The estimated 5-year overall survival (OS) rate was 82%, and the event-free survival (EFS) rate was 54%. Seven patients (8.4%) died during induction due to coagulopathy. Maintenance randomization demonstrated that addition of oral chemotherapy to ATRA significantly reduced relapse rate, but difference in EFS did not reach statistical significance (P = 0.12; 5-year rates [95% CI]: 41% [17%-64%] ATRA only vs 72% [56%-88%] ATRA plus chemotherapy). There was no difference (P = 0.93) in EFS for age <5 years versus 5-12.99 years versus 13-17.99 years (5-year rates: 56%, 47%, and 45%, respectively). Among adolescents 15-17.99 years old in the ATO randomization, there was a significantly lower relapse risk at 5 years for those receiving ATO (0% ATO vs 44% no ATO; P = 0.02). CONCLUSION: Our data demonstrate that intensified ATRA, cytarabine, and anthracycline chemotherapy is effective for pediatric APL including very young patients, but early deaths and relapses remain barriers to cure. Further improvements are likely with incorporation of ATO into pediatric APL regimens.
RCT Entities:
BACKGROUND:Acute promyelocytic leukemia (APL) is a unique leukemia subtype requiring specialized treatment including all-trans retinoic acid (ATRA). A prior report demonstrated worse outcome among young children <5 years old compared with older children. METHODS: We evaluated outcomes for pediatric patients (<18 years old; N = 83) with APL treated on North American intergroup study CALGB 9710 at Children's Oncology Group sites. Induction and consolidation included ATRA, cytarabine, and anthracyclines. Patients ≥15 years old were randomized to addition of arsenic trioxide (ATO) consolidation. All patients were randomized to ATRA maintenance with versus without oral chemotherapy. RESULTS: The estimated 5-year overall survival (OS) rate was 82%, and the event-free survival (EFS) rate was 54%. Seven patients (8.4%) died during induction due to coagulopathy. Maintenance randomization demonstrated that addition of oral chemotherapy to ATRA significantly reduced relapse rate, but difference in EFS did not reach statistical significance (P = 0.12; 5-year rates [95% CI]: 41% [17%-64%] ATRA only vs 72% [56%-88%] ATRA plus chemotherapy). There was no difference (P = 0.93) in EFS for age <5 years versus 5-12.99 years versus 13-17.99 years (5-year rates: 56%, 47%, and 45%, respectively). Among adolescents 15-17.99 years old in the ATO randomization, there was a significantly lower relapse risk at 5 years for those receiving ATO (0% ATO vs 44% no ATO; P = 0.02). CONCLUSION: Our data demonstrate that intensified ATRA, cytarabine, and anthracycline chemotherapy is effective for pediatric APL including very young patients, but early deaths and relapses remain barriers to cure. Further improvements are likely with incorporation of ATO into pediatric APL regimens.
Authors: Anna Maria Testi; Paolo Musiu; Maria Luisa Moleti; Saveria Capria; Walter Barberi Journal: Mediterr J Hematol Infect Dis Date: 2022-05-01 Impact factor: 3.122
Authors: Francianne G Andrade; Suellen V M Feliciano; Ingrid Sardou-Cezar; Gisele D Brisson; Filipe V Dos Santos-Bueno; Danielle T Vianna; Luísa V C Marques; Eugênia Terra-Granado; Ilana Zalcberg; Marceli de O Santos; Juliana T Costa; Elda P Noronha; Luiz C S Thuler; Joseph L Wiemels; Maria S Pombo-de-Oliveira Journal: Front Oncol Date: 2021-03-19 Impact factor: 6.244