Nakazawa Michael S, Livingston J Andrew, Zarzour Maria A, Bishop Andrew J, Ratan Ravin, Ludwig Joseph A, Araujo Dejka M, Somaiah Neeta, Ravi Vinod, Nassif Elise F, Roland Christina L, Lazar Alexander J, Guadagnolo B Ashleigh, Harrison Douglas J, Benjamin Robert S, Patel Shreyaskumar R, Conley Anthony P
Department of Sarcoma Medical Oncology, University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Department of Radiation Oncology, University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Rare Tumors. 2024 Aug 4;16:20363613241271669. doi: 10.1177/20363613241271669. eCollection 2024.
Rhabdomyosarcomas are the most common soft tissue sarcoma in children, and pediatric alveolar rhabdomyosarcoma (ARMS) prognosis has improved based on cooperative studies. However, in adults, ARMS is significantly rarer, has poorer outcomes, and currently lacks optimal treatment strategies. This study aimed to evaluate the clinical outcome of an adult ARMS population with different front-line systemic chemotherapies and determine if any chemotherapy regimen is associated with improved survival. This is a retrospective study of histologically confirmed fusion-positive ARMS patients over 18 years of age, who were treated at MD Anderson Cancer Center (MDACC) from 2004 to 2021 and received systemic chemotherapy. Descriptive clinical statistics were performed, including staging, front-line chemotherapy, multimodal therapy usage, response rates, and survival analyses. 49 ARMS patients who received upfront chemotherapy were identified. Locoregional treatments included radiotherapy (RT) alone (29%, = 14), surgery alone (10%, = 5), or both (45%, = 22). Median overall survival (OS) for the entire cohort was 3.6 years, and the overall response rate to systemic therapy was 89%. No chemotherapy regimen showed OS benefit, specifically analyzing the pediatric-based vincristine, actinomycin-D, cyclophosphamide (VAC) or adult-based vincristine, doxorubicin, ifosfamide (VDI) regimens, even when controlled for other clinical risk factors. In this single-center contemporary series, adult ARMS patient outcomes remain poor. There was no statistically significant OS difference in patients who did or did not receive adult or pediatric based ARMS regimens, although a high overall response rate to chemotherapy was seen across the entire cohort. Based on these observations, further randomized prospective studies are necessary to delineate which frontline chemotherapy regimen is most beneficial in this rare adult cancer.
横纹肌肉瘤是儿童中最常见的软组织肉瘤，基于合作研究，小儿肺泡横纹肌肉瘤（ARMS）的预后有所改善。然而，在成人中，ARMS明显更为罕见，预后较差，目前缺乏最佳治疗策略。本研究旨在评估接受不同一线全身化疗的成人ARMS患者的临床结局，并确定是否有任何化疗方案与生存率提高相关。这是一项对组织学确诊的18岁以上融合阳性ARMS患者的回顾性研究，这些患者于2004年至2021年在MD安德森癌症中心（MDACC）接受治疗并接受了全身化疗。进行了描述性临床统计，包括分期、一线化疗、多模式治疗的使用、缓解率和生存分析。确定了49例接受 upfront化疗的ARMS患者。局部区域治疗包括单纯放疗（RT）（29%，n = 14）、单纯手术（10%，n = 5）或两者皆有（45%，n = 22）。整个队列的中位总生存期（OS）为3.6年，全身治疗的总缓解率为89%。没有化疗方案显示出OS获益，特别是分析基于小儿的长春新碱、放线菌素-D、环磷酰胺（VAC）或基于成人的长春新碱、阿霉素、异环磷酰胺（VDI）方案时，即使在控制了其他临床风险因素的情况下也是如此。在这个单中心当代系列研究中，成人ARMS患者的结局仍然很差。接受或未接受基于成人或小儿的ARMS方案的患者在OS上没有统计学上的显著差异，尽管整个队列对化疗的总缓解率较高。基于这些观察结果，有必要进行进一步的随机前瞻性研究，以确定哪种一线化疗方案对这种罕见的成人癌症最有益。
