Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands.
SIREDO Oncology Center (Care, Innovation and Research for Children, Adolescents and Young Adults with Cancer), Institut Curie, PSL University, Paris, France.
JCO Precis Oncol. 2023 Sep;7:e2300323. doi: 10.1200/PO.23.00323.
Inflammatory myofibroblastic tumors (IMTs) are often driven by anaplastic lymphoma kinase fusions and less frequently by alternative fusions such as . We describe the clinical characteristics, treatment approach, and outcome for a series of young patients with IMTs and alterations.
This was a retrospective, international, multicenter study analyzing young patients (younger than 21 years) with -altered IMTs treated in 10 European referral centers between 2014 and 2022. Patients were included in the European Soft tissue sarcoma Study Group NRSTS-2005 protocol or registered in the Soft Tissue Sarcoma Registry. Primary surgery was recommended if a microscopic radical resection was feasible without mutilation. No standard systemic treatment protocol was available, but several medical options were recommended.
A total of 19 patients (median age 8.3 years) were included. Most patients had a biopsy at diagnosis (Intergroup Rhabdomyosarcoma Study [IRS] I; n = 2, IRS II; n = 1, IRS III biopsy; n = 11, IRS III resection; n = 3, IRS IV; n = 2). Twelve patients received neoadjuvant systemic therapy in first line (four received multiple treatments): high-dose steroids (n = 2), vinorelbine/vinblastine with methotrexate (n = 6), or inhibitors (n = 8). After a median follow-up of 2.8 years (range, 0.2-13.4), seven patients developed an event. The 3-year event-free survival was 41% (95% CI, 11 to 71), and the 3-year overall survival was 100%.
Outcome for -altered IMTs appears excellent. A complete resection at diagnosis was often not feasible, and most patients needed neoadjuvant therapy. Patients who developed a tumor event could be cured with reinitiation of systemic therapy and/or surgery. This approach illustrates a switch in treatment philosophy moving from immediate, often mutilating, surgery to systemic (targeted) therapy as a bridge to more conservative surgery later in the treatment course.
炎症性肌纤维母细胞瘤（IMTs）常由间变性淋巴瘤激酶融合驱动，较少由替代性融合驱动，如 。我们描述了一系列具有 改变的 IMT 年轻患者的临床特征、治疗方法和结局。
这是一项回顾性的国际多中心研究，分析了 2014 年至 2022 年间在 10 个欧洲转诊中心接受治疗的 10 名年轻患者（年龄小于 21 岁）的 -改变 IMTs。患者被纳入欧洲软组织肉瘤研究组 NRSTS-2005 方案，或在软组织肉瘤登记处注册。如果可行的话，建议进行微小根治性切除，以避免严重致残。没有标准的系统治疗方案，但推荐了几种医疗方案。
共纳入 19 名患者（中位年龄 8.3 岁）。大多数患者在诊断时进行了活检（国际横纹肌肉瘤研究 [IRS] I；n = 2，IRS II；n = 1，IRS III 活检；n = 11，IRS III 切除；n = 3，IRS IV；n = 2）。12 名患者在一线接受了新辅助系统治疗（4 名接受了多次治疗）：高剂量类固醇（n = 2）、长春瑞滨/长春碱联合甲氨蝶呤（n = 6）或 抑制剂（n = 8）。中位随访 2.8 年后（范围：0.2-13.4），7 名患者出现了疾病进展。3 年无事件生存率为 41%（95%CI：11-71），3 年总生存率为 100%。
-改变的 IMT 患者的预后似乎很好。诊断时通常无法进行完全切除，大多数患者需要新辅助治疗。出现肿瘤进展的患者可以通过重新开始系统治疗和/或手术来治愈。这种方法说明了治疗理念的转变，从立即进行往往致残的手术，转变为将系统（靶向）治疗作为后期更保守手术的桥梁。
