Klinikum der Landeshauptstadt Stuttgart gKAöR, Olgahospital, Stuttgart Cancer Center, Zentrum für Kinder-, Jugend- und Frauenmedizin, Pädiatrie 5 (Pädiatrische Onkologie, Hämatologie, Immunologie), Stuttgart, Germany.
University of Medicine Tübingen, Tübingen, Germany.
Pediatr Blood Cancer. 2024 Jan;71(1):e30742. doi: 10.1002/pbc.30742. Epub 2023 Oct 25.
Parameningeal location of rhabdomyosarcoma (PM RMS) is known to be an unfavorable prognostic factor. Scarce data are available on radiotherapy (RT) concepts with regard to outcome.
Treatment and outcome of 395 children with PM RMS registered within two Cooperative Weichteilsarkom Studiengruppe (CWS) trials and one registry (1995-2021) were evaluated.
Patients were IRS group II (n = 15) and III (n = 380) and received systemic treatment according to the enrolled protocols: I2VA (n = 172), VAIA/CEVAIE (n = 223). Delayed resection was performed in 88/395 (22%) patients, and RT was additionally given in 79/88 (90%) resected patients. RT was the predominant local treatment in 355/395 (90%) patients: hyperfractionated accelerated photon (HART; n = 77), conventionally fractionated photon (n = 91) or proton beam (n = 126), brachytherapy (n = 4), heavy ions (n = 1), not available (n = 56). In the subgroup of RT as only local treatment (n = 278), no intracranial tumor extension and complete remission at end of treatment were significant positive prognostic factors. No significant difference on tumor outcome was seen between different radiotherapy concepts. Long-term toxicity with mostly endocrinological and visual deficiencies was reported in 161/279 (58%) surviving patients with a lower trend after proton beam RT (48%) when compared to HART or conventionally fractionated photon RT (71% and 72%, respectively). Ten-year event-free and overall survival in the overall group were 62% (±5, 95% confidence interval [CI]) and 67% (±5, 95% CI); in the RT-only group 67% (±6, 95% CI) and 71% (±6, 95% CI), respectively.
CWS data confirm the recent RT concept in PM RMS. Long-term sequelae as endocrinological and visual deficiencies need to be addressed.
横纹肌肉瘤（RMS）的脑脊膜旁位置是预后不良的已知因素。关于放疗（RT）结果的相关数据很少。
对在两个软组织肉瘤研究组（CWS）试验和一个登记处（1995-2021 年）中登记的 395 名脑脊膜旁 RMS 患儿的治疗和结局进行评估。
患者为国际横纹肌肉瘤研究组（IRS）Ⅱ组（n=15）和Ⅲ组（n=380），并根据入组方案接受全身治疗：I2VA（n=172）、VAIA/CEVAIE（n=223）。88/395（22%）例患者行延迟性切除术，79/88（90%）例切除患者接受 RT。355/395（90%）例患者的主要局部治疗为 RT：超分割加速光子（HART；n=77）、常规分割光子（n=91）或质子束（n=126）、近距离放疗（n=4）、重离子（n=1）、不可用（n=56）。在仅接受 RT 作为局部治疗的亚组（n=278）中，颅内肿瘤未扩展和治疗结束时完全缓解是显著的阳性预后因素。不同放疗概念之间肿瘤结局无显著差异。在 279 例存活患者中，报告了 161 例（58%）长期毒性反应，主要为内分泌和视觉缺陷，质子束 RT 时毒性反应呈下降趋势（48%），与 HART 或常规分割光子 RT 相比（分别为 71%和 72%）。总体人群的 10 年无事件生存率和总生存率分别为 62%（±5，95%置信区间[CI]）和 67%（±5，95% CI）；仅 RT 组分别为 67%（±6，95% CI）和 71%（±6，95% CI）。
CWS 数据证实了脑脊膜旁 RMS 的最新 RT 概念。需要解决内分泌和视觉缺陷等长期后遗症。
