Revisiting the role of doxorubicin in the treatment of rhabdomyosarcoma: An up-front window study in newly diagnosed children with high-risk metastatic disease

被引:27
作者
Bergeron, Christophe [1 ]
Thiesse, Philippe [2 ]
Rey, Annie [3 ]
Orbach, Daniel [4 ]
Boutard, Patrick [5 ]
Thomas, Caroline [6 ]
Schmitt, Claudine [7 ]
Scopinaro, Marcelo J. [8 ]
Bernard, Frederic [9 ]
Stevens, Michael [10 ]
Oberlin, Odile [3 ]
机构
[1] Ctr Leon Berard, Paediat Oncol Unit, F-69373 Lyon 08, France
[2] Ctr Leon Berard, Radiol Unit, F-69373 Lyon, France
[3] Inst Gustave Roussy, F-94805 Villejuif, France
[4] Inst Curie, Paediat Oncol Unit, F-75248 Paris 05, France
[5] CHRU Caen, Paediat Oncol Unit, F-14033 Caen, France
[6] CHRU Nantes, Paediat Oncol Unit, F-44035 Nantes, France
[7] CHRU Nancy, Paediat Oncol Unit, F-54511 Vandoeuvre Les Nancy, France
[8] Hosp Pediat SAMIC JP Garrahan, Buenos Aires, DF, Argentina
[9] CHRU Montpellier, Paediat Oncol Unit, F-34295 Montpellier, France
[10] Univ Bristol, Inst Child Life & Hlth, Bristol, Avon, England
关键词
doxorubicin; rhabdomyosarcoma; children;
D O I
10.1016/j.ejca.2007.12.007
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
Purpose: Many cooperative groups have reported on the chemo-sensitivity of rhabdomyosarcoma (RMS). Doxorubicin has been tested but remains a controversial treatment option. We report here the results of the up-front evaluation of the efficacy of doxorubicin in children and adolescents with high-risk metastatic RMS. Patients and methods: Patients younger than 18 years of age (>6 months) with newly diagnosed, histologically confirmed high-risk metastatic RMS were required to have measurable disease, to have undergone no prior chemotherapy or radiation therapy and to have normal liver, renal and cardiac function before treatment. Doxorubicin was administered intravenously over 48 h to a total dose of 60 mg/m(2). Two courses were given separated by a 21 day interval. Response to therapy was assessed by diagnostic imaging after the second course. The study was designed as a two-stage procedure according to the multistep plan described by Fleming. Results: Twenty patients were eligible for analysis. Median age at diagnosis was 9.8 years (range from 2 to 16). Thirteen of the 20 patients treated in the first step responded to treatment, corresponding to an overall response to doxorubicin of 65% [95% confidence interval (CI), 44-85%]. The rates of CR and PR were 5% [95% CI, 0-14%] and 60% [95% CI, 39-81%], respectively. Four (20%) patients had progressive disease, corresponding to a progression rate of 20% [95% CI, 2-38%]. Conclusion: This window study provides the definitive demonstration of the efficacy of doxorubicin in untreated RMS. Given the inconclusive results obtained from previous using differing schedules chemotherapy incorporating doxorubicin, the next step should be a randomised study testing dose intensity in high-risk localised RMS. This issue is being addressed in a current European study (EpSSG RMS 2005). (c) 2007 Elsevier Ltd. All rights reserved.
引用
收藏
页码:427 / 431
页数:5
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