BACKGROUND:Preoperative cisplatin alone may be as effective as cisplatin plus doxorubicin in standard-risk hepatoblastoma (a tumor involving three or fewer sectors of the liver that is associated with an alpha-fetoprotein level of >100 ng per milliliter). METHODS:Children with standard-risk hepatoblastoma who were younger than 16 years of age were eligible for inclusion in the study. After they received one cycle of cisplatin (80 mg per square meter of body-surface area per 24 hours), we randomly assigned patients to receive cisplatin (every 14 days) or cisplatin plus doxorubicin administered in three preoperative cycles and two postoperative cycles. The primary outcome was the rate of complete resection, and the trial was powered to test the noninferiority of cisplatin alone (<10% difference in the rate of complete resection). RESULTS:Between June 1998 and December 2006, 126 patients were randomly assigned to receive cisplatin and 129 were randomly assigned to receive cisplatin plus doxorubicin. The rate of complete resection was 95% in the cisplatin-alone group and 93% in the cisplatin-doxorubicin group in the intention-to-treat analysis (difference, 1.4%; 95% confidence interval [CI], -4.1 to 7.0); these rates were 99% and 95%, respectively, in the per-protocol analysis. Three-year event-free survival and overall survival were, respectively, 83% (95% CI, 77 to 90) and 95% (95% CI, 91 to 99) in the cisplatin group, and 85% (95% CI, 79 to 92) and 93% (95% CI, 88 to 98) in the cisplatin-doxorubicin group (median follow-up, 46 months). Acute grade 3 or 4 adverse events were more frequent with combination therapy (74.4% vs. 20.6%). CONCLUSIONS: As compared with cisplatin plus doxorubicin, cisplatin monotherapy achieved similar rates of complete resection and survival among children with standard-risk hepatoblastoma. Doxorubicin can be safely omitted from the treatment of standard-risk hepatoblastoma. (ClinicalTrials.gov number, NCT00003912.) 2009 Massachusetts Medical Society
RCT Entities:
BACKGROUND: Preoperative cisplatin alone may be as effective as cisplatin plus doxorubicin in standard-risk hepatoblastoma (a tumor involving three or fewer sectors of the liver that is associated with an alpha-fetoprotein level of >100 ng per milliliter). METHODS:Children with standard-risk hepatoblastoma who were younger than 16 years of age were eligible for inclusion in the study. After they received one cycle of cisplatin (80 mg per square meter of body-surface area per 24 hours), we randomly assigned patients to receive cisplatin (every 14 days) or cisplatin plus doxorubicin administered in three preoperative cycles and two postoperative cycles. The primary outcome was the rate of complete resection, and the trial was powered to test the noninferiority of cisplatin alone (<10% difference in the rate of complete resection). RESULTS: Between June 1998 and December 2006, 126 patients were randomly assigned to receive cisplatin and 129 were randomly assigned to receive cisplatin plus doxorubicin. The rate of complete resection was 95% in the cisplatin-alone group and 93% in the cisplatin-doxorubicin group in the intention-to-treat analysis (difference, 1.4%; 95% confidence interval [CI], -4.1 to 7.0); these rates were 99% and 95%, respectively, in the per-protocol analysis. Three-year event-free survival and overall survival were, respectively, 83% (95% CI, 77 to 90) and 95% (95% CI, 91 to 99) in the cisplatin group, and 85% (95% CI, 79 to 92) and 93% (95% CI, 88 to 98) in the cisplatin-doxorubicin group (median follow-up, 46 months). Acute grade 3 or 4 adverse events were more frequent with combination therapy (74.4% vs. 20.6%). CONCLUSIONS: As compared with cisplatin plus doxorubicin, cisplatin monotherapy achieved similar rates of complete resection and survival among children with standard-risk hepatoblastoma. Doxorubicin can be safely omitted from the treatment of standard-risk hepatoblastoma. (ClinicalTrials.gov number, NCT00003912.) 2009 Massachusetts Medical Society
Authors: Heng Xu; Giles W Robinson; Jie Huang; Joshua Yew-Suang Lim; Hui Zhang; Johnnie K Bass; Alberto Broniscer; Murali Chintagumpala; Ute Bartels; Sri Gururangan; Tim Hassall; Michael Fisher; Richard Cohn; Tetsuji Yamashita; Tal Teitz; Jian Zuo; Arzu Onar-Thomas; Amar Gajjar; Clinton F Stewart; Jun J Yang Journal: Nat Genet Date: 2015-02-09 Impact factor: 38.330
Authors: Alexander Beck; Corinna Eberherr; Michaela Hagemann; Stefano Cairo; Beate Häberle; Christian Vokuhl; Dietrich von Schweinitz; Roland Kappler Journal: Cancer Biol Ther Date: 2016-09-16 Impact factor: 4.742
Authors: Linde A D Busweiler; Marc H W A Wijnen; Jim C H Wilde; Egbert Sieders; Sheila E J Terwisscha van Scheltinga; L W Ernest van Heurn; Joseph Ziros; Roel Bakx; Hugo A Heij Journal: Pediatr Surg Int Date: 2016-10-11 Impact factor: 1.827
Authors: Howard M Katzenstein; Max R Langham; Marcio H Malogolowkin; Mark D Krailo; Alexander J Towbin; Mary Beth McCarville; Milton J Finegold; Sarangarajan Ranganathan; Stephen Dunn; Eugene D McGahren; Gregory M Tiao; Allison F O'Neill; Muna Qayed; Wayne L Furman; Caihong Xia; Carlos Rodriguez-Galindo; Rebecka L Meyers Journal: Lancet Oncol Date: 2019-04-08 Impact factor: 41.316