Outcome of 449 adult patients with rhabdomyosarcoma: an observational ambispective nationwide study

摘要

Five‐year overall survival (OS) of localized RMS exceeds 70% in children (<18) but is very poor in adult patients. We analyzed the outcome and prognostic factors (PF) of a national series of adult patients with RMS in a large study. The study population consisted of two different cohorts: a retrospective cohort (157 adult patients treated in 13 reference centers between 05/1981 and 02/2010) and the prospective cohort (292 patients with RMS diagnosed and treated between 01/2010 and 12/2014 in France) included in the NetSarc database. A descriptive analysis of patients’ characteristics and prognostic factors was conducted on both series which were compared. In the retrospective series, histological subtypes were embryonal (E‐RMS) for 21% of patients, alveolar (A‐RMS) for 35% of patients, and “adult‐type” P‐RMS (pleomorphic, spindle cell RMS, not otherwise specified) (P) for 44% patients. This distribution significantly differed in the prospective cohort: A‐RMS: 18%; E‐RMS: 17%; and P‐RMS 65%. With a median follow‐up of 8.5 years, 5‐year OS for localized RMS and advanced RMS (with nodes and/or metastases) was 43% and 5%, respectively, (P < 0.0001), and median style="fixed-case">OS was 51, 33, and 16 months for E‐ style="fixed-case">RMS, A‐ style="fixed-case">RMS, and P‐ style="fixed-case">RMS, respectively, in the retrospective cohort. The median style="fixed-case">OS was less than 40 months for the prospective nationwide cohort for the entire population. In a multivariate analysis of the retrospective study, independent prognostic factors for style="fixed-case">OS were A‐ style="fixed-case">RMS, R0 resection, and adjuvant radiotherapy ( style="fixed-case">RT). For localized style="fixed-case">RMS, age and use of pediatric chemotherapy ( style="fixed-case">CT) regimen are independent prognostic factors. Adult patients with style="fixed-case">RMS have a poorer overall survival than pediatric patients, and survival varies considerably across histological subtypes.