Steven G DuBois1, M Meaghan Granger2, Susan Groshen3, Denice Tsao-Wei3, Lingyun Ji3, Anasheh Shamirian4, Scarlett Czarnecki5, Fariba Goodarzian6, Rachel Berkovich6, Hiroyuki Shimada7, Judith G Villablanca4, Kieuhoa T Vo8, Navin Pinto9, Yael P Mosse10, John M Maris10, Suzanne Shusterman1, Susan L Cohn11, Kelly C Goldsmith12, Brian Weiss13, Gregory A Yanik14, Clare J Twist15, Meredith S Irwin16, Daphne A Haas-Kogan17, Julie R Park9, Araz Marachelian4, Katherine K Matthay8. 1. Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA. 2. Cook Children's Hospital, Fort Worth, TX. 3. Department of Preventive Medicine, Keck School of Medicine, University of Southern California, Los Angeles, CA. 4. Department of Pediatrics, Keck School of Medicine, University of Southern California, Children's Hospital of Los Angeles, Los Angeles, CA. 5. Department of Pediatrics, Loma Linda University Medical Center, Loma Linda, CA. 6. Department of Radiology, Children's Hospital of Los Angeles, Los Angeles, CA. 7. Department of Pathology, Stanford University School of Medicine, Palo Alto, CA. 8. Department of Pediatrics, UCSF Benioff Children's Hospital and UCSF School of Medicine, San Francisco, CA. 9. Department of Pediatrics, Seattle Children's Hospital and University of Washington School of Medicine, Seattle, WA. 10. Department of Pediatrics, Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia, PA. 11. Department of Pediatrics, Comer Children's Hospital and University of Chicago Pritzker School of Medicine, Chicago, IL. 12. Department of Pediatrics, Children's Healthcare of Atlanta and Emory University School of Medicine, Atlanta, GA. 13. Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH. 14. Department of Pediatrics, CS Mott Children's Hospital, University of Michigan Medical School, Ann Arbor, MI. 15. Department of Pediatrics, Roswell Park Comprehensive Cancer Center, Buffalo, NY. 16. Department of Pediatrics, Hospital for Sick Children, Toronto, Ontario, Canada. 17. Department of Radiation Oncology, Dana-Farber Cancer Institute and Brigham and Women's Hospital, Harvard Medical School, Boston, MA.
Abstract
PURPOSE: 131I-metaiodobenzylguanidine (MIBG) is an active radiotherapeutic for neuroblastoma. The primary aim of this trial was to identify which of three MIBG regimens was likely associated with the highest true response rate. PATIENTS AND METHODS: Patients 1-30 years were eligible if they had relapsed or refractory neuroblastoma, at least one MIBG-avid site, and adequate autologous stem cells. Patients received MIBG 18 mCi/kg on day 1 and autologous stem cell on day 15. Patients randomly assigned to arm A received only MIBG; patients randomly assigned to arm B received intravenous vincristine on day 0 and irinotecan daily on days 0-4; patients randomly assigned to arm C received vorinostat (180 mg/m2/dose) orally once daily on days 1 to 12. The primary end point was response after one course by New Approaches to Neuroblastoma Therapy criteria. The trial was designed with 105 patients to ensure an 80% chance that the arm with highest response rate was selected. RESULTS: One hundred fourteen patients were enrolled, with three ineligible and six unevaluable, leaving 105 eligible and evaluable patients (36 in arm A, 35 in arm B, and 34 in arm C; 55 boys; and median age 6.5 years). After one course, the response rates (partial response or better) on arms A, B, and C were 14% (95% CI, 5 to 30), 14% (5 to 31), and 32% (18 to 51). An additional five, five, and four patients met New Approaches to Neuroblastoma Therapy Minor Response criteria on arms A, B, and C, respectively. On arms A, B, and C, rates of any grade 3+ nonhematologic toxicity after first course were 19%, 49%, and 35%. CONCLUSION: Vorinostat and MIBG is likely the arm with the highest true response rate, with manageable toxicity. Vincristine and irinotecan do not appear to improve the response rate to MIBG and are associated with increased toxicity.
PURPOSE: 131I-metaiodobenzylguanidine (MIBG) is an active radiotherapeutic for neuroblastoma. The primary aim of this trial was to identify which of three MIBG regimens was likely associated with the highest true response rate. PATIENTS AND METHODS: Patients 1-30 years were eligible if they had relapsed or refractory neuroblastoma, at least one MIBG-avid site, and adequate autologous stem cells. Patients received MIBG 18 mCi/kg on day 1 and autologous stem cell on day 15. Patients randomly assigned to arm A received only MIBG; patients randomly assigned to arm B received intravenous vincristine on day 0 and irinotecan daily on days 0-4; patients randomly assigned to arm C received vorinostat (180 mg/m2/dose) orally once daily on days 1 to 12. The primary end point was response after one course by New Approaches to Neuroblastoma Therapy criteria. The trial was designed with 105 patients to ensure an 80% chance that the arm with highest response rate was selected. RESULTS: One hundred fourteen patients were enrolled, with three ineligible and six unevaluable, leaving 105 eligible and evaluable patients (36 in arm A, 35 in arm B, and 34 in arm C; 55 boys; and median age 6.5 years). After one course, the response rates (partial response or better) on arms A, B, and C were 14% (95% CI, 5 to 30), 14% (5 to 31), and 32% (18 to 51). An additional five, five, and four patients met New Approaches to Neuroblastoma Therapy Minor Response criteria on arms A, B, and C, respectively. On arms A, B, and C, rates of any grade 3+ nonhematologic toxicity after first course were 19%, 49%, and 35%. CONCLUSION: Vorinostat and MIBG is likely the arm with the highest true response rate, with manageable toxicity. Vincristine and irinotecan do not appear to improve the response rate to MIBG and are associated with increased toxicity.
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Authors: Steven G DuBois; Susan Groshen; Julie R Park; Daphne A Haas-Kogan; Xiaodong Yang; Ethan Geier; Eugene Chen; Kathy Giacomini; Brian Weiss; Susan L Cohn; M Meaghan Granger; Gregory A Yanik; Randall Hawkins; Jesse Courtier; Hollie Jackson; Fariba Goodarzian; Hiroyuki Shimada; Scarlett Czarnecki; Denice Tsao-Wei; Judith G Villablanca; Araz Marachelian; Katherine K Matthay Journal: Clin Cancer Res Date: 2015-02-18 Impact factor: 12.531
Authors: Maryam Fouladi; Julie R Park; Clinton F Stewart; Richard J Gilbertson; Paula Schaiquevich; Junfeng Sun; Joel M Reid; Matthew M Ames; Roseanne Speights; Ashish M Ingle; James Zwiebel; Susan M Blaney; Peter C Adamson Journal: J Clin Oncol Date: 2010-07-06 Impact factor: 44.544
Authors: Wenyin Shi; Yaacov Richard Lawrence; Hak Choy; Maria Werner-Wasik; David W Andrews; James J Evans; Kevin D Judy; Christopher J Farrell; Yaron Moshel; Adam C Berger; Voichita Bar-Ad; Adam P Dicker Journal: J Neurooncol Date: 2014-04-13 Impact factor: 4.130