Li Yi,Dai Shuyang,Zhan Yong,et al.Correlation analysis of clinical features and outcomes of infantile fibrosarcoma[J].Journal of Clinical Pediatric Surgery,2024,23(07):641-647.[doi:10.3760/cma.j.cn101785-202204074-007]
婴儿型纤维肉瘤的临床特征及预后影响因素分析
- Title:
- Correlation analysis of clinical features and outcomes of infantile fibrosarcoma
- Keywords:
- Infantial Fibrosarcoma; Prognosis; Surgical Procedures; Operative; Child
- 摘要:
- 目的 分析婴儿型纤维肉瘤(infantile fibrosarcoma,IFS)的临床特征及治疗效果,并探讨其预后相关因素。方法 本研究为回顾性研究,以2010年1月至2022年1月复旦大学附属儿科医院收治的23例术后病理检查证实为IFS的患儿为研究对象,采用Kaplan-Meier法计算患儿总体生存率和无事件生存率,患儿预后相关因素的单因素分析采用Log-Rank 检验,多因素分析采用Cox回归。结果 23例患儿中,男15例,女8例;发病年龄最小为1天,最大为46个月;发病部位:头颈部4例,躯干13例,四肢6例,首诊时伴肺转移1例;17例肿瘤长径＞5 cm;11例ETS变异转录因子6(ETS variant transcription factor 6,ETV6)-神经营养受体酪氨酸激酶3(neurotrophic receptor tyrosine kinase 3,NTRK3)基因融合检测结果为阳性;16例首诊接受切除手术,7例接受活检手术;术后按照美国横纹肌肉瘤研究组(Intergroup Rhabdomyosarcoma Study,IRS)分期:Ⅰ期9例,Ⅱ期4例,Ⅲ期9例,Ⅳ期1例;5例接受新辅助化疗,其中3例疗效不明显;5例口服拉罗替尼,均疗效良好。截至随访时间,23例中6例因随访时间不满3年删失,IFS 3年总体生存率为(15/17,88.24%),3年无事件生存率为(11/17,64.71%);9例因随访时间不满5年删失,IFS 5年总体生存率为(12/14,85.71%),5年无事件生存率为(9/14,64.29%)。单因素分析结果表明首次手术方式选择切除手术与IFS良好预后相关(χ2=4.938,P=0.026);多因素Cox回归分析显示首次手术方式及术后IRS分期非IFS预后独立影响因素(P＞0.05)。结论 IFS总体预后较好;首诊时接受切除手术对IFS生存有利。新辅助化疗在IFS中的有效性有待探究;拉罗替尼是携带NTRK基因融合、难治性IFS患儿的有效治疗手段。
- Abstract:
- Objective To explore the clinical characteristics and therapeutic outcomes of infantile fibrosarcoma (IFS) and explore the related prognostic factors at a single center. Methods Between January 2010 and January 2022,the relevant clinical data were collected from 23 IFS children.Kaplan-Meier analysis was performed for calculating the values of overall survival (OS) and event-free survival (EFS).Univariate analysis was performed with Log-rank test and multivariate analysis by Cox regression. Results With a male-to-female ratio of 1.86:1,the age was from birth to 46 months.Tumor locations included head & neck (n=4),trunk (n=13) and extremities (n=6).Lung metastasis (n=1) was detected at an initial diagnosis.Seventeen children had tumors with a diameter >5 cm and 11 cases tested positive for ETS variant transcription factor 6 (ETV6)-neurotrophic receptor tyrosine kinase 3 (NTRK3) gene fusion.Primary resection (n=16) and biopsy (n=7) were performed.According to the staging scheme of Intergroup Rhabdomyosarcoma Study (IRS),the clinical stages were Ⅰ(n=9),Ⅱ(n=4),Ⅲ(n=9) and Ⅳ(n=1).Among 5 cases on neoadjuvant chemotherapy,efficacy was indefinite (n=3).Five cases taking oral larotrectinib showed a promising efficacy.At the end of follow-up, 6 of 23 cases had been followed for less than 3 years,and 9 less for 5 years.The 3-year OS was (15/17,88.24%) and the 5-year OS was (12/14,85.71%); the 3-year EFS (11/17, 64.71%) and the 5-year EFS rate was (9/14, 64.29%). Univariate analysis revealed that primary resection at an initial diagnosis was associated with a favorable prognosis for IFS (χ2=4.938,P=0.026).Multivariate Cox regression analysis revealed that neither the initial surgical method nor the postoperative IRS stage were independent prognostic factors for IFS (P>0.05). Conclusions The overall prognosis of IFS is favorable.Primary resection at an initial diagnosis is beneficial for the survival of IFS children.The effectiveness of neoadjuvant chemotherapy for IFS requires further investigations.Larotrectinib represents a potential effective therapy for refractory IFS children with NTRK gene fusion.
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备注/Memo
收稿日期:2022-4-24。
基金项目:唐仲英基金会项目(ZSBK0070)
通讯作者:董瑞,Email:rdong@fudan.edu.cn