婴儿型纤维肉瘤的临床特征及预后影响因素分析OA北大核心CSTPCD

Objective To explore the clinical characteristics and therapeutic outcomes of infantile fibro-sarcoma(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 chil-dren 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 chemo-therapy,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 di-agnosis was associated with a favorable prognosis for IFS(x2=4.938,P=0.026).Multivariate Cox regression analysis revealed that neither the initial surgical method nor the postoperative IRS stage were independent prog-nostic 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 chemothera-py for IFS requires further investigations.Larotrectinib represents a potential effective therapy for refractory IFS children with NTRK gene fusion.