Stuttgart Cancer Center, Zentrum für Kinder-, Jugend- und Frauenmedizin, Pediatrics 5 (Oncology, Hematology, and Immunology), Klinikum Stuttgart-Olgahospital, Stuttgart, Germany.
Department of Pediatric Surgery, University Children's Hospital Marburg, Marburg, Germany.
Pediatr Blood Cancer. 2018 May;65(5):e26943. doi: 10.1002/pbc.26943. Epub 2018 Jan 5.
Treatment algorithms for patients with aggressive fibromatosis (AF) are challenging. There are limited data available about the use of systemic therapy (ST) in pediatric patients with AF.
Patient-, tumor-, and treatment-related factors of 90 children and adolescents with AF treated on multiple prospective trials of the Cooperative Weichteilsarkom Studiengruppe (1981-2015) were analyzed with focus on response and outcome of ST.
Median age was 9.48 years (0.02-18.05). Primary resection was performed in 54 patients and ST was administered in 29 of 54 patients because of disease progression or relapse. In 35 patients, ST was the initial treatment modality. A secondary resection was performed in 21 of 35 patients after ST. A total of 64 patients received ST, mainly methotrexate and vinblastine (40%) with a median duration of 380 days. The most frequent radiological response to ST was stable disease at 3 months (39%) and partial response at 6 months (53%). Radiotherapy was administered to 15 of 90 patients. One patient remained on observation only. The 5-year overall survival was 100% and the 5-year event-free survival (EFS) was 44%. Patients who had a primary resection showed a 5-year EFS of 35% versus 59% in patients who had received primary ST (P = 0.08). Functional deficiencies as long-term sequelae following resection occurred in 11 patients. At a median follow-up of 5.05 years (0.25-14.88), complete remission was achieved in 51 patients and partial remission in 28 patients.
ST seems appropriate if a primary complete resection is not feasible and at relapse/progression after resection.
对于侵袭性纤维瘤病（AF）患者，治疗方案的选择具有挑战性。目前，关于在儿科 AF 患者中应用系统治疗（ST）的数据有限。
对在多个协作软组织肉瘤研究组（1981-2015 年）前瞻性研究中接受治疗的 90 例儿童和青少年侵袭性纤维瘤病患者的患者、肿瘤和治疗相关因素进行了分析，重点关注 ST 的反应和结果。
中位年龄为 9.48 岁（0.02-18.05 岁）。54 例患者进行了初次手术切除，其中 29 例因疾病进展或复发而接受 ST。35 例患者首次接受 ST 治疗。21 例接受 ST 治疗后再次进行手术切除。共有 64 例患者接受 ST 治疗，主要采用甲氨蝶呤联合长春新碱（40%），中位治疗时间为 380 天。ST 治疗 3 个月时最常见的影像学反应为疾病稳定（39%），6 个月时为部分缓解（53%）。15 例患者接受放疗。1 例患者仅接受观察。5 年总生存率为 100%，5 年无事件生存率（EFS）为 44%。接受初次手术切除的患者 5 年 EFS 为 35%，而接受初次 ST 治疗的患者 5 年 EFS 为 59%（P=0.08）。11 例患者在接受切除手术后出现长期的功能缺陷。中位随访 5.05 年（0.25-14.88 年）后，51 例患者达到完全缓解，28 例患者达到部分缓解。
如果初次完全切除不可行，或在切除后复发/进展时，ST 似乎是合适的选择。
