Kushner B H, LaQuaglia M P, Wollner N, Meyers P A, Lindsley K L, Ghavimi F, Merchant T E, Boulad F, Cheung N K, Bonilla M A, Crouch G, Kelleher J F, Steinherz P G, Gerald W L
Department of Pathology, Memorial Sloan-Kettering Cancer Center, New York, NY 10021, USA.
J Clin Oncol. 1996 May;14(5):1526-31. doi: 10.1200/JCO.1996.14.5.1526.
To test intensive alkylator-based therapy in desmoplastic small round-cell tumor (DSRCT).
Patients received the P6 protocol, which has seven courses of chemotherapy. Courses 1, 2, 3, and 6 included cyclophosphamide 4,200 mg/m2, doxorubicin 75 mg/m2, and vincristine (HD-CAV). Courses 4, 5, and 7 consisted of ifosfamide 9 g/m2 and etoposide 500 mg/m2 for previously untreated patients, or ifosfamide 12 g/m2 and etoposide 1,000 mg/m2 for previously treated patients. Courses started after neutrophil counts reached 500/microL and platelet counts reached 100,000/microL. Tumor resection was attempted. Post-P6 treatment options included radiotherapy and a myeloablative regimen of thiotepa (900 mg/m2) plus carboplatin (1,500 mg/m2), with stem-cell rescue.
Ten previously untreated and two previously treated patients have completed therapy. The male-to-female ratio was 11:1. Ages were 7 to 22 years (median, 14). The largest masses were infradiaphragmatic (n = 11) or intrathoracic (n = 1). Other findings included serosal implants (n = 11), regional lymph node invasion (n = 8), ascites or pleural effusion (n = 7), and metastases to liver (n = 5), lungs (n = 4), distant lymph nodes (n = 3), spleen (n = 2), and skeleton (n = 2). Tumors uniformly responded to HD-CAV, but there were no complete pathologic responses. One patient died at 1 month from tumor-related Budd-Chiari syndrome. Of seven patients who achieved a complete remission (CR), five remain in CR 9, 12, 13, 33, and 38 months from the start of P6, one patient died of infection at 12 months (autopsy-confirmed CR), and one patient relapsed 4 months off therapy. Of four patients who achieved a partial remission (PR), one remains progression-free at 34 months and three developed progressive disease. Five patients received local radiotherapy: three were not assessable for response, but in two patients, antitumor effect was evident. Four patients received thiotepa/carboplatin: two were in CR and remain so, and two patients had measurable disease that did not respond.
For control of DSRCT, our experience supports intensive use of HD-CAV, aggressive surgery to resect visible disease, radiotherapy to high-risk sites, and myeloablative chemotherapy with stem-cell rescue in selected cases.
在促纤维组织增生性小圆细胞肿瘤（DSRCT）中测试基于强化烷化剂的疗法。
患者接受P6方案，该方案有七个化疗疗程。第1、2、3和6疗程包括环磷酰胺4200mg/m²、多柔比星75mg/m²和长春新碱（HD-CAV）。第4、5和7疗程对于既往未治疗的患者为异环磷酰胺9g/m²和依托泊苷500mg/m²，对于既往接受过治疗的患者为异环磷酰胺12g/m²和依托泊苷1000mg/m²。当中性粒细胞计数达到500/μL且血小板计数达到100000/μL后开始疗程。尝试进行肿瘤切除术。P6治疗后的选择包括放疗以及硫替派（900mg/m²）加卡铂（1500mg/m²）的清髓方案，并进行干细胞救援。
10例既往未治疗和2例既往接受过治疗的患者完成了治疗。男女比例为11:1。年龄为7至22岁（中位数为14岁）。最大肿块位于膈下（n = 11）或胸腔内（n = 1）。其他发现包括浆膜种植（n = 11）、区域淋巴结侵犯（n = 8）、腹水或胸腔积液（n = 7）以及肝转移（n = 5）、肺转移（n = 4）、远处淋巴结转移（n = 3）、脾转移（n = 2）和骨骼转移（n = 2）。肿瘤对HD-CAV均有反应，但无完全病理缓解。1例患者在1个月时死于肿瘤相关的布加综合征。在7例达到完全缓解（CR）的患者中，5例自P6开始起分别在9、12、13、33和38个月时仍处于CR状态，1例患者在12个月时死于感染（尸检证实为CR），1例患者在治疗停药4个月后复发。在4例达到部分缓解（PR）的患者中，1例在34个月时无疾病进展，3例出现疾病进展。5例患者接受了局部放疗：3例无法评估反应，但在2例患者中，抗肿瘤效果明显。4例患者接受了硫替派/卡铂治疗：2例处于CR状态且仍维持如此，2例有可测量疾病但无反应。
对于DSRCT的控制，我们的经验支持强化使用HD-CAV、积极手术切除可见病灶、对高危部位进行放疗以及在特定病例中进行清髓化疗并进行干细胞救援。
