Raney R B, Tefft M, Maurer H M, Ragab A H, Hays D M, Soule E H, Foulkes M A, Gehan E A
Division of Pediatric Hematology-Oncology, University of Virginia Children's Medical Center, Charlottesville 22908.
Cancer. 1988 Oct 1;62(7):1257-66. doi: 10.1002/1097-0142(19881001)62:7<1257::aid-cncr2820620703>3.0.co;2-k.
The patterns of disease extent, response to treatment, and survival rates were examined in 129 children registered as metastatic (Group IV) soft-tissue sarcoma patients on the Intergroup Rhabdomyosarcoma Study (IRS)-I protocol from November 1972 through October 1978. The patients' ages at diagnosis ranged from 3 months to 21 years (median, 10.5 years); 72 were male patients and 57 were female patients (male-to-female ratio, 1.3:1). The tumor arose in an extremity in 34 patients, the head and neck (exclusive of the orbit) in 27, the genitourinary tract in 25, the retroperitoneum or pelvis in 14, the trunk in 12, the intrathoracic region in nine, the hepatobiliary system in four, the perineum in two, and the orbit in two patients. The histologic types of soft-tissue sarcoma confirmed by the pathology review committee were embryonal rhabdomyosarcoma in 68 patients, alveolar rhabdomyosarcoma in 41, undifferentiated sarcoma in 14, embryonal-botryoid in two, pleomorphic in one patient, and other sarcoma in three patients. Of 105 patients who were confirmed in Group IV and had complete information concerning sites of metastatic deposits, 53 patients presented with metastases restricted to only one anatomic region or tissue; the other 52 had diffuse metastases. The most common sites of distant tumor deposits were the lungs, bone marrow, and other soft tissues. After treatment with vincristine, dactinomycin, and cyclophosphamide with or without Adriamycin (Adria Laboratories, Inc., Columbus, OH) and radiation therapy, 65 (50%) of the 129 eligible patients achieved complete disappearance of all detectable tumor. The likelihood of achieving and maintaining a complete response for 2 years was highest among patients whose tumors originated in the genitourinary tract, retroperitoneum-pelvis, or the hepatobiliary or perineum regions. Thirteen of these 45 children (29%) are alive and disease-free at a minimum of 2 years after initiation of treatment. Only 10 of the other 84 (8%) are surviving disease-free at 2 years (P = 0.02). The presence of restricted rather than diffuse metastases at diagnosis (P = 0.02) was the only other characteristic significantly related to long-term complete remissions. One hundred and two patients (79%) died at a median of 47 weeks after diagnosis, 92 from sarcoma and ten from other causes.(ABSTRACT TRUNCATED AT 400 WORDS)
对1972年11月至1978年10月期间登记在横纹肌肉瘤协作组研究（IRS）-I方案中的129例转移性（IV组）软组织肉瘤患儿的疾病范围模式、治疗反应和生存率进行了研究。诊断时患者年龄从3个月至21岁不等（中位数为10.5岁）；男性患者72例，女性患者57例（男女比例为1.3:1）。肿瘤发生在四肢的有34例，头颈部（不包括眼眶）27例，泌尿生殖道25例，腹膜后或骨盆14例，躯干12例，胸内区域9例，肝胆系统4例，会阴2例，眼眶2例。经病理审查委员会确认的软组织肉瘤组织学类型为胚胎性横纹肌肉瘤68例，肺泡性横纹肌肉瘤41例，未分化肉瘤14例，胚胎性葡萄状肉瘤2例，多形性肉瘤1例，其他肉瘤3例。在IV组确诊且有关于转移灶部位完整信息的105例患者中，53例患者的转移仅局限于一个解剖区域或组织；另外52例有弥漫性转移。远处肿瘤转移最常见的部位是肺、骨髓和其他软组织。在用长春新碱、放线菌素D和环磷酰胺治疗，加或不加阿霉素（阿德里亚实验室公司，俄亥俄州哥伦布市）及放射治疗后，129例符合条件的患者中有65例（50%）所有可检测到的肿瘤完全消失。肿瘤起源于泌尿生殖道、腹膜后-骨盆或肝胆或会阴区域的患者达到并维持2年完全缓解的可能性最高。这45例患儿中有13例（29%）在开始治疗后至少2年存活且无疾病。其他84例中只有10例（8%）在2年时无病存活（P = 0.02）。诊断时存在局限性而非弥漫性转移（P = 0.02）是与长期完全缓解显著相关的唯一其他特征。102例患者（79%）在诊断后中位47周死亡，92例死于肉瘤，10例死于其他原因。（摘要截短至400字）
