Risk Factors for Survival in Pediatric Maxillofacial Rhabdomyosarcoma: A Single-Center Retrospective Cohort Study.
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BACKGROUND: Maxillofacial rhabdomyosarcoma (RMS) is a rare and poorly defined subset of pediatric head and neck RMS. Its anatomical complexity and overlap between parameningeal and nonparameningeal sites pose significant challenges in risk stratification and treatment. PURPOSE: The purpose of the study was to estimate and identify risk factors for the 5-year overall survival (OS) and event-free survival (EFS) among pediatric patients with rhabdomyosarcoma. STUDY DESIGN, SETTING, AND SAMPLE: This retrospective cohort study was conducted at Beijing Children's Hospital between July 2016 and April 2023. The sample included subjects under 18 years old with a primary diagnosis of maxillofacial RMS. Exclusions included unconfirmed histopathology, primary tumors outside the defined region, incomplete records, or follow-up of <3 months unless due to death. PREDICTOR VARIABLE: A set of heterogeneous risk factors were evaluated and grouped into demographic variables, tumor characteristics, and treatment factors. MAIN OUTCOME VARIABLE: The main outcome variables were therapeutic outcomes defined as OS and EFS, calculated from the date of diagnosis to the last follow-up visit, death, or first event. COVARIATES: Not applicable. ANALYSES: Survival analyses were performed using the Kaplan-Meier method, and subgroup differences were compared via the log-rank test. Cox proportional hazards regression models were utilized to estimate and identify risk factors associated with outcomes. The level of statistical significance was set at P < .05. RESULTS: The sample comprised 46 subjects. The median (interquartile range) age was 70.5 (42.3 to 111.5) months. With a median (interquartile range) for follow-up of 51.4 (37.9 to 81.6) months, the 5-year OS and EFS were 70.2 and 66.0%, respectively. Local relapse was the most common pattern of treatment failure (50.0% of events). Multivariable Cox regression indicated that a lack of response to neoadjuvant chemotherapy (SD/PD) was a highly significant independent risk factor for both poor OS and EFS, while positive FOXO1 fusion status remained an independent risk factor for inferior EFS. CONCLUSIONS AND RELEVANCE: Pediatric maxillofacial RMS represents a distinct subgroup with a prognosis intermediate to that of other head and neck subsites. Outcomes are heavily influenced by early chemotherapy response, as well as tumor biology.