未经证实:肺泡软组织肉瘤(ASPS)是一种罕见的肉瘤,已被证明对抗血管生成药物和免疫检查点抑制剂非常有效,但大多数报道的关于ASPS的研究集中在成年人群。在这项研究中,我们旨在描述儿童ASPS的临床特征和治疗结果.
UNASSIGNED:我们回顾性回顾了自2015年1月以来我们机构的ASPS患者记录。本研究中包括的所有患者均为病理证实的ASPS,在最初诊断时年龄在12岁以下。人口特征,肿瘤大小,原发性肿瘤部位,转移,使用的治疗方法,评估了治疗反应和生存率.
UNASSIGNED:自2015年1月以来,我们确定了总共56例最初诊断为ASPS的患者。观察到头颈部高发生率(32.1%)(四肢为41.1%,躯干为21.4%)。26例(46.4%)患者在诊断时或随访期间发生了转移。舌部肿瘤,咽和喉转移的可能性最小(7.7%,P<0.05)。建议对15例仅有肺转移的IV期患者进行观察。7例(46.7%)患者在最后一次随访前保持稳定。1年PFS率为83.3%,中位无进展生存期(PFS)为29.4个月。15例进行性疾病患者接受单一或联合治疗。11例患者接受PD-1单药治疗。2例患者取得部分反响,5例疾病稳固。总有效率为18.2%。本组中位PFS为22.0个月,1年PFS率为70.0%。4例患者接受PD-1抑制剂加酪氨酸激酶抑制剂的联合治疗。他们都保持稳定。随访期间无疾病相关死亡发生。
UNASSIGNED:ASPS在儿童头颈部的发生率较高。起源于舌咽区的ASPS倾向于具有较低的转移率。ASPS在儿童中显示出更懒惰的生长模式,这使得观察成为唯一肺转移儿童的首选。小儿ASPS似乎对靶向治疗和免疫疗法的有效性不如成年人。儿童进行性ASPS的治疗仍然具有挑战性。
UNASSIGNED: Alveolar soft part sarcoma (ASPS) is a rare sarcoma that has been shown to be highly effective to antiangiogenic agents and immune checkpoint inhibitors, but most reported studies about ASPS were concentrated on adult population. In this
study, we aimed to describe the clinical features and therapeutic outcomes of ASPS in children.
UNASSIGNED: We retrospectively reviewed the records of patients with ASPS in our institution since Jan 2015. All patients included in this
study were pathologically confirmed ASPS and aged under 12 years at the time of initial diagnosis. Demographic characteristics, tumor sizes, primary tumor sites, metastasis, treatments used, therapeutic responses and survivals were evaluated.
UNASSIGNED: We identified a total of 56 patients to be initially diagnosed as ASPS since Jan 2015. A predisposition of high occurrence in head and neck (32.1%) was observed (versus 41.1% in limbs and 21.4% in trunk). 26 (46.4%) patients developed metastasis at the time of diagnosis or during follow-up. Tumors in tongue, pharynx and larynx had the least likelihood to metastasize (7.7%, P<0.05). Observation was recommended for 15 stage IV patients with only pulmonary metastasis. 7 (46.7%) patients remained stable until last follow up. The 1-year PFS rate was 83.3% and median progression-free survival time (PFS) was 29.4 months. 15 patients with progressive disease received mono or combined therapy. 11 patients received PD-1 monotherapy. 2 patients achieved partial response and 5 stable disease. The overall response rate was 18.2%. The median PFS of this group was 22.0 months, and the 1-year PFS rate was 70.0%. 4 patients received a combination therapy of PD-1 inhibitors plus tyrosine kinase inhibitors. All of them remained stable. No disease-related death occurred during follow-up.
UNASSIGNED: ASPS exhibits a higher occurrence in head and neck in children. ASPS originating from glossopharyngeal region tends to have a lower metastasis rate. ASPS displays a more indolent growth pattern in children, which makes observation a preferable choice for children with sole pulmonary metastasis. Pediatric ASPS appears to be less effective to targeted therapy and immunotherapy than adults. The treatment of progressive ASPS in children remains challenging.