Department of Hematology, Oncology and Cardiovascular Medicine, Kyushu University Hospital, Fukuoka, Japan.
Department of Medical Oncology Organization, Hamanomachi Hospital, Fukuoka, Japan.
BMC Cancer. 2023 Oct 31;23(1):1046. doi: 10.1186/s12885-023-11528-4.
Rhabdomyosarcoma is the most common soft tissue sarcoma in children, but rare in adults. Para-meningeal rhabdomyosarcoma in head and neck (PM-HNRMS) is less applicable for surgery due to the anatomic reason. PM-HNRMS has a poor prognosis in children. However, its clinical outcomes remain unclear in adults due to the rarity. Further, there is almost no detailed data about salvage therapy.
We retrospectively examined the adult patients with PM-HNRMS treated at institutions belonging to the Kyushu Medical Oncology Group from 2009 to 2022. We evaluated the overall survival (OS) and progression-free survival (PFS) of the patients who received a first-line therapy. We also reviewed the clinical outcomes of patients who progressed against a first-line therapy and received salvage therapy.
Total 11 patients of PM-HNRMS received a first-line therapy. The characteristics were as follows: median age: 38 years (range 25 - 63 years), histology (alveolar/spindle): 10/1, and risk group (intermediate/high): 7/4. As a first-line therapy, VAC and ARST0431-based regimen was performed in 10 and 1 patients, respectively. During a first-line therapy, definitive radiation for all lesions were performed in seven patients. The median PFS was 14.2 months (95%CI: 6.0 - 25.8 months): 17.1 months (95%CI: 6.0 - not reached (NR)) for patients with stage I-III and 8.5 months (95%CI: 5.2 - 25.8 months) for patients with stage IV. The 1-year and 3-year PFS rates were 54.5% and 11.3% for all patients. Median OS in all patients was 40.8 months (95%CI: 12.1 months-NR): 40.8 months (95%CI: 12.1 - NR) for patients with stage I-III and NR for patients with stage IV. The 5-year OS rate was 48.5% for all patients. Among seven patients who received salvage therapy, three are still alive, two of whom remain disease-free for over 4 years after completion of the last therapy. Those two patients received multi-modal therapy including local therapy for all detected lesions.
The cure rate of adult PM-HNRMS is low in spite of a first-line therapy in this study. Salvage therapy might prolong the survival in patients who received the multi-modal therapy including local therapy for all detected lesions.
横纹肌肉瘤是儿童中最常见的软组织肉瘤，但在成人中较为罕见。发生于头颈部的脑脊膜旁横纹肌肉瘤（PM-HNRMS）由于解剖原因，手术治疗的适用性较差。儿童 PM-HNRMS 的预后较差。然而，由于其罕见性，其在成人中的临床结局仍不清楚。此外，关于挽救性治疗几乎没有详细的数据。
我们回顾性分析了 2009 年至 2022 年期间隶属于九州肿瘤内科治疗组的机构中治疗的成人 PM-HNRMS 患者。我们评估了接受一线治疗的患者的总生存期（OS）和无进展生存期（PFS）。我们还回顾了对一线治疗进展并接受挽救性治疗的患者的临床结局。
共有 11 例 PM-HNRMS 患者接受了一线治疗。其特征如下：中位年龄 38 岁（范围 25-63 岁），组织学（腺泡/梭形）10/1，风险组（中危/高危）7/4。一线治疗中，10 例患者接受 VAC 方案，1 例患者接受 ARST0431 方案。在一线治疗期间，7 例患者对所有病灶进行了根治性放疗。中位 PFS 为 14.2 个月（95%CI：6.0-25.8 个月）：I-III 期患者为 17.1 个月（95%CI：6.0-NR），IV 期患者为 8.5 个月（95%CI：5.2-25.8 个月）。所有患者的 1 年和 3 年 PFS 率分别为 54.5%和 11.3%。所有患者的中位 OS 为 40.8 个月（95%CI：12.1-NR）：I-III 期患者为 40.8 个月（95%CI：12.1-NR），IV 期患者为 NR。所有患者的 5 年 OS 率为 48.5%。在接受挽救性治疗的 7 例患者中，有 3 例仍存活，其中 2 例在最后一次治疗后超过 4 年无疾病。这两名患者接受了包括所有检测到的病变局部治疗在内的多模式治疗。
尽管本研究中对成人 PM-HNRMS 进行了一线治疗，但治愈率仍较低。挽救性治疗可能会延长接受包括所有检测到的病变局部治疗在内的多模式治疗的患者的生存时间。
