Pediatrics 5 (Oncology, Hematology, and Immunology), Stuttgart Cancer Center, Klinikum Stuttgart, Olgahospital, Stuttgart, Germany.
Kiel Pediatric Tumor Registry, Department of Pediatric Pathology, University of Kiel, Kiel, Germany.
Pediatr Blood Cancer. 2018 Jun;65(6):e27012. doi: 10.1002/pbc.27012. Epub 2018 Feb 26.
Inflammatory myofibroblastic tumors (IMTs) are a rare subgroup of soft tissue tumors. The outcome of patients with IMT has been reported as favorable when the tumor is completely resected. If surgical resection is not possible, systemic therapy has to be considered. However, the best systemic treatment and response rates are currently unclear.
Thirty-eight patients under the age of 21, who were registered between 2000 and 2014 with a primary diagnosis of IMT, were analyzed.
IMT was typically localized intra-abdominally or in the pelvis. In 20 patients, the tumor was resected without further therapy; 17 patients were in complete remission at last evaluation and two patients were in partial remission. Eighteen patients received systemic therapy, 15 of whom had macroscopically incomplete resection. Systemic therapy most commonly consisted of regimens with dactinomycin, ifosfamide or cyclophosphamide, and vincristine, with or without doxorubicin, and it seemed to reduce tumor extension in individual cases. Five-year event-free survival was 74 ± 14% and 5-year overall survival was 91 ± 10% for all patients. The patients who died due to the disease were those with incomplete resection (n = 3).
Surgery without further systemic therapy was a feasible and acceptable therapeutic option for every second patient with IMT. Standard chemotherapy for pediatric soft tissue sarcoma produced favorable results in individual cases and was able to shrink the tumor enough to enable resection. Superior efficacy of new targeted therapies such as anaplastic lymphoma kinase-inhibitors compared to standard chemotherapy has to be proven in the future.
炎性肌纤维母细胞瘤（IMT）是一种罕见的软组织肿瘤亚群。当肿瘤完全切除时，患者的预后已被报道为良好。如果不能进行手术切除，则必须考虑全身治疗。然而，目前尚不清楚最佳的全身治疗和缓解率。
分析了 2000 年至 2014 年间登记的 38 名年龄在 21 岁以下、原发性诊断为 IMT 的患者。
IMT 通常位于腹腔内或骨盆内。20 名患者行肿瘤切除术而无进一步治疗；17 名患者在最后评估时完全缓解，2 名患者部分缓解。18 名患者接受了全身治疗，其中 15 名患者有肉眼不完全切除。全身治疗最常见的方案是使用放线菌素 D、异环磷酰胺或环磷酰胺和长春新碱，联合或不联合多柔比星，并且在个别情况下似乎能降低肿瘤的扩展。所有患者的 5 年无事件生存率为 74±14%，5 年总生存率为 91±10%。因疾病而死亡的患者是那些肿瘤不完全切除的患者（n=3）。
对于每二分之一的 IMT 患者，不进行进一步的全身治疗而行手术是一种可行且可接受的治疗选择。儿科软组织肉瘤的标准化疗在个别病例中产生了良好的效果，并能使肿瘤缩小到足以进行切除。新型靶向治疗（如间变性淋巴瘤激酶抑制剂）与标准化疗相比的优越性，将来需要进一步证实。
