Walterhouse David O, Barkauskas Donald A, Hall David, Ferrari Andrea, De Salvo Gian Luca, Koscielniak Ewa, Stevens Michael C G, Martelli Hélène, Seitz Guido, Rodeberg David A, Shnorhavorian Margarett, Dasgupta Roshni, Breneman John C, Anderson James R, Bergeron Christophe, Bisogno Gianni, Meyer William H, Hawkins Douglas S, Minard-Colin Veronique
David O. Walterhouse, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Donald A. Barkauskas, University of Southern California, Los Angeles; David Hall, Children's Oncology Group, Monrovia, CA; Andrea Ferrari, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Istituto Nazionale Tumori, Milan; Gian Luca De Salvo, Istituto Oncologico Veneto Istituto di Ricovero e Cura a Carattere Scientifico, Padua; Gianni Bisogno, University of Padua, Padova, Italy; Ewa Koscielniak, Olgahospital, Stuttgart; Guido Seitz, University Hospital, Marburg, Germany; Michael C.G. Stevens, Royal Hospital for Children, Bristol, United Kingdom; Hélène Martelli, Assistance Publique-Hôpitaux de Paris, Hopital Bicetre, Le Kremlin-Bicetre; Christophe Bergeron, Centre Léon Bérard, Lyon; Veronique Minard-Colin, Gustave Roussy, Villejuif, France; David A. Rodeberg, East Carolina University, Greenville, NC; Margarett Shnorhavorian and Douglas S. Hawkins, Seattle Children's Hospital, University of Washington; Douglas S. Hawkins, Fred Hutchinson Cancer Research Center, University of Washington, Seattle, WA; Roshni Dasgupta and John C. Breneman, Cincinnati Children's Hospital Medical Center, Cincinnati, OH; James R. Anderson, Merck Research Laboratories, North Wales, PA; and William H. Meyer, University of Oklahoma School of Medicine, Oklahoma City, OK.
J Clin Oncol. 2018 Oct 23;36(35):JCO2018789388. doi: 10.1200/JCO.2018.78.9388.
Treatment recommendations for localized paratesticular rhabdomyosarcoma (PT RMS) differ in North America and Europe. We conducted a pooled analysis to identify demographic features and treatment choices that affect outcome.
We retrospectively analyzed the effect of nine demographic variables and four treatment choices on event-free survival (EFS) and overall survival (OS) from 12 studies conducted by five cooperative groups.
Eight hundred forty-two patients with localized PT RMS who enrolled from 1988 to 2013 were included. Patients age ≥ 10 years were more likely than younger patients to have tumors that were > 5 cm, enlarged nodes (N1), or pathologically involved nodes ( P ≤ .05 each). With a median follow-up of 7.5 years, Kaplan-Meier estimates for 5-year EFS and OS were 87.7% and 94.8%, respectively. Of demographic variables, cooperative group, era of enrollment, age category, tumor size, Intergroup Rhabdomyosarcoma Study group, and T stage affected EFS ( P ≤ .05 each). Surgical assessment of regional nodes, which was performed in 23.5% of patients-usually in those age ≥ 10 years or with suspicious or N1 nodes-was the only treatment variable associated with EFS by univariable and multivariable analyses ( P ≤ .05 each) in patients age ≥ 1 year. A variable selection procedure on a proportional hazards regression model selected era of enrollment, age, tumor size, and surgical assessment of regional nodes as significant ( P ≤ .05 each) in the EFS model, and era of enrollment, age, tumor size, and histology ( P ≤ .05 each) in the OS model.
Localized PT RMS has a favorable prognosis. Age ≥ 10 years at diagnosis and tumor size larger than 5 cm are unfavorable prognostic features. Surgical assessment of regional nodes is important in patients age ≥ 10 years and in those with N1 nodes as it affects EFS.
北美和欧洲对于局限性睾丸旁横纹肌肉瘤（PT RMS）的治疗建议有所不同。我们进行了一项汇总分析，以确定影响预后的人口统计学特征和治疗选择。
我们回顾性分析了五个合作组开展的12项研究中九个人口统计学变量和四种治疗选择对无事件生存期（EFS）和总生存期（OS）的影响。
纳入了1988年至2013年登记的842例局限性PT RMS患者。年龄≥10岁的患者比年轻患者更有可能出现肿瘤>5 cm、淋巴结肿大（N1）或病理累及淋巴结（各P≤0.05）。中位随访7.5年，Kaplan-Meier法估计的5年EFS和OS分别为87.7%和94.8%。在人口统计学变量中，合作组、入组时代、年龄类别、肿瘤大小、横纹肌肉瘤协作组和T分期影响EFS（各P≤0.05）。对区域淋巴结进行手术评估的患者占23.5%，通常是年龄≥10岁或有可疑或N1淋巴结的患者，在年龄≥1岁的患者中，单变量和多变量分析显示，这是唯一与EFS相关的治疗变量（各P≤0.05）。在EFS模型中，比例风险回归模型的变量选择程序选择入组时代、年龄、肿瘤大小和区域淋巴结手术评估为显著变量（各P≤0.05），在OS模型中选择入组时代、年龄、肿瘤大小和组织学（各P≤0.05）。
局限性PT RMS预后良好。诊断时年龄≥10岁和肿瘤大小大于5 cm是不良预后特征。对年龄≥10岁和有N1淋巴结的患者进行区域淋巴结手术评估很重要，因为它会影响EFS。
