Takashi Taga1, Tomoyuki Watanabe2, Daisuke Tomizawa3, Kazuko Kudo4, Kiminori Terui5, Hiroshi Moritake6, Akitoshi Kinoshita7, Shotaro Iwamoto8, Hideki Nakayama9, Hiroyuki Takahashi10, Akira Shimada11, Tomohiko Taki12, Tsutomu Toki5, Etsuro Ito5, Hiroaki Goto13, Katsuyoshi Koh14, Akiko M Saito15, Keizo Horibe16, Tatsutoshi Nakahata17, Akio Tawa18, Souichi Adachi19. 1. Department of Pediatrics, Shiga University of Medical Science, Otsu, Japan. 2. Department of Nutritional Science, Faculty of Psychological and Physical Science, Aichi Gakuin University, Nisshin, Japan. 3. Division of Leukemia and Lymphoma, Children's Cancer Center, National Center for Child Health and Development, Tokyo, Japan. 4. Department of Pediatrics, Fujita Health University, Toyoake, Japan. 5. Department of Pediatrics, Hirosaki University Graduate School of Medicine, Hirosaki, Japan. 6. Division of Pediatrics, Department of Reproductive and Developmental Medicine, Faculty of Medicine, University of Miyazaki, Miyazaki, Japan. 7. Department of Pediatrics, St. Marianna University School of Medicine, Kawasaki, Japan. 8. Department of Pediatrics, Mie University Graduate School of Medicine, Tsu, Japan. 9. Department of Pediatrics, National Hospital Organization, Fukuoka-Higashi Medical Center, Fukuoka, Japan. 10. Department of Pediatrics, Toho University, Tokyo, Japan. 11. Department of Pediatrics, Okayama University Hospital, Okayama, Japan. 12. Department of Molecular Diagnostics and Therapeutics, Kyoto Prefectural University of Medicine, Kyoto, Japan. 13. Division of Hemato-Oncology and Regenerative Medicine, Kanagawa Children's Medical Center, Yokohama, Japan. 14. Department of Hematology/Oncology, Saitama Children's Medical Center, Saitama, Japan. 15. Laboratory of Clinical, Epidemiological and Health Services Research, Clinical Research Center, National Hospital Organization, Nagoya Medical Center, Aichi, Japan. 16. Clinical Research Center, National Hospital Organization Nagoya Medical Center, Nagoya, Japan. 17. Center for iPS Cell Research and Application, Kyoto University, Kyoto, Japan. 18. Department of Pediatrics, National Hospital Organization, Osaka Medical Center, Osaka, Japan. 19. Human Health Sciences, Kyoto University, Kyoto, Japan.
Abstract
BACKGROUND: On the basis of results of previous Japanese trials for myeloid leukemia in Down syndrome (ML-DS), the efficacy of risk-oriented therapy was evaluated in the Japanese Pediatric Leukemia/Lymphoma Study Group AML-D05 study. PROCEDURE: All patients received induction chemotherapy that consisted of pirarubicin, intermediate-dose cytarabine, and etoposide. Patients who achieved complete remission (CR) after initial induction therapy were stratified to the standard risk (SR) group and received four courses of reduced-dose intensification therapy. Patients who did not achieve CR were stratified to the high risk (HR) group and received intensified therapy that consisted of continuous or high-dose cytarabine. RESULTS: A total of 72 patients were eligible and evaluated. One patient died of sepsis during initial induction therapy. Sixty-nine patients were stratified to SR and two patients to HR. No therapy-related deaths were observed during intensification therapy. The 3-year event-free and overall survival rates were 83.3% ± 4.4% and 87.5% ± 3.9%, respectively. Age at diagnosis less than 2 years was a significant favorable prognostic factor for risk of relapse (P = 0.009). CONCLUSIONS: The attempt of risk-oriented prospective study for ML-DS was unsuccessful, but despite the dose reduction of chemotherapeutic agents, the overall outcome was good, and further dose reduction might be possible for specific subgroups.
BACKGROUND: On the basis of results of previous Japanese trials for myeloid leukemia in Down syndrome (ML-DS), the efficacy of risk-oriented therapy was evaluated in the Japanese Pediatric Leukemia/Lymphoma Study Group AML-D05 study. PROCEDURE: All patients received induction chemotherapy that consisted of pirarubicin, intermediate-dose cytarabine, and etoposide. Patients who achieved complete remission (CR) after initial induction therapy were stratified to the standard risk (SR) group and received four courses of reduced-dose intensification therapy. Patients who did not achieve CR were stratified to the high risk (HR) group and received intensified therapy that consisted of continuous or high-dose cytarabine. RESULTS: A total of 72 patients were eligible and evaluated. One patient died of sepsis during initial induction therapy. Sixty-nine patients were stratified to SR and two patients to HR. No therapy-related deaths were observed during intensification therapy. The 3-year event-free and overall survival rates were 83.3% ± 4.4% and 87.5% ± 3.9%, respectively. Age at diagnosis less than 2 years was a significant favorable prognostic factor for risk of relapse (P = 0.009). CONCLUSIONS: The attempt of risk-oriented prospective study for ML-DS was unsuccessful, but despite the dose reduction of chemotherapeutic agents, the overall outcome was good, and further dose reduction might be possible for specific subgroups.
Authors: Carla L Pennella; Tamara Muñoz Cassina; Jorge G Rossi; Edgardo M Baialardo; Patricia Rubio; María A Deu; Luisina Peruzzo; Myriam R Guitter; Cristian G Sanchez de La Rosa; Elizabeth M Alfaro; María S Felice Journal: Cancers (Basel) Date: 2022-07-05 Impact factor: 6.575