Arndt C A, Nascimento A G, Schroeder G, Schomberg P J, Neglia J P, Sencer S F, Silberman T L, Moertel C L, Tillisch J K, Miser J S
Department of Pediatric Hematology-Oncology, Mayo Clinic, Rochester, Minnesota, USA.
Eur J Cancer. 1998 Jul;34(8):1224-9. doi: 10.1016/s0959-8049(98)00080-x.
Over 50% of patients with newly diagnosed rhabdomyosarcoma (RMS) are in the 'intermediate risk' group with a 3-year progression-free survival of approximately 65%. This group consists of stage 1, group III, non-orbit tumours; stage 2, group II and III; and all stage 3 patients utilising the Intergroup Rhabdomyosarcoma Study (IRS) staging system. The role of doxorubicin in the treatment of RMS has been controversial. Ifosfamide, both alone and in combination with etoposide, has significant activity in patients with RMS. The aim of this pilot study was to examine the efficacy and toxicity of a chemotherapy regimen of alternating cycles of vincristine/doxorubicin/cyclophosphamide and etoposide/ifosfamide for intermediate risk RMS. 30 patients with intermediate risk RMS or undifferentiated sarcoma (US) were treated with alternating cycles of vincristine/doxorubicin/cyclophosphamide (VDC) and etoposide/ifosfamide (EI) at planned intervals of 3 weeks. Local treatment of the tumour in most cases was performed after four cycles of chemotherapy, followed by an additional 10 cycles of chemotherapy. At a median follow-up of 37.5 months, the Kaplan-Meier estimate of 3-year event-free survival was 85% (95% confidence interval 72-99%). The overall survival at 3 years was 91% (95% confidence interval 80-100%). No patient died from toxicity. The most common toxicity was febrile neutropenia in 35% of VDC and 26% of EI cycles. No nephrotoxicity or cardiac toxicity was seen. No patient progressed prior to week 12 local therapy. Alternating cycles of VDC and EI are an effective treatment for patients with intermediate risk RMS and US. Toxicity is tolerable. Delaying local treatment until week 12 does not compromise outcome.
超过50%新诊断的横纹肌肉瘤（RMS）患者属于“中危”组，其3年无进展生存率约为65%。该组包括采用横纹肌肉瘤协作组（IRS）分期系统的1期、III组非眼眶肿瘤；2期、II组和III组；以及所有3期患者。阿霉素在RMS治疗中的作用一直存在争议。异环磷酰胺，无论是单独使用还是与依托泊苷联合使用，在RMS患者中都具有显著活性。这项初步研究的目的是检验长春新碱/阿霉素/环磷酰胺和依托泊苷/异环磷酰胺交替周期化疗方案对中危RMS的疗效和毒性。30例中危RMS或未分化肉瘤（US）患者接受长春新碱/阿霉素/环磷酰胺（VDC）和依托泊苷/异环磷酰胺（EI）交替周期治疗，计划间隔为3周。大多数情况下，在4个周期化疗后进行肿瘤局部治疗，随后再进行10个周期化疗。中位随访37.5个月时，3年无事件生存率的Kaplan-Meier估计值为85%（95%置信区间72 - 99%）。3年总生存率为91%（95%置信区间80 - 100%）。无患者死于毒性反应。最常见的毒性反应是发热性中性粒细胞减少，VDC周期中发生率为35%，EI周期中为26%。未观察到肾毒性或心脏毒性。在第12周局部治疗前无患者病情进展。VDC和EI交替周期是治疗中危RMS和US患者的有效方法。毒性反应可耐受。将局部治疗推迟至第12周不影响治疗结果。
