Long Term Clinical Outcome and Quality of Life of Children, Adolescents and Young Adults Treated with Pencil Beam Scanning Proton Therapy for Rhabdomyosarcoma

Abstract

To assess the long-term clinical outcomes of children, adolescents and young adults (CAYAs) with rhabdomyosarcoma (RMS) treated with pencil beam scanning proton therapy (pbsPT). One hundred twenty-one RMS (embryonal, n = 102; 84.3%) patients treated between January 2000 and December 2020 were included in this analysis. The median age was 4.7 years (range, 0.1-35.3). All patients received systemic chemotherapy according to prospective protocols. The median total dose delivered was 54 Gy (RBE) (range, 41.4-74.0). After a median follow-up time of 85.5 month (range,3.4-240.0), we observed 23 local/regional and 12 distant failures. The estimated 5-year local control (LC) and overall survival (OS) was 81.5% and 80%, respectively. For the subgroups of parameningeal (PM), orbital, urogenital and H&N non-PM the 5-year LC was 74.7%, 92%, 100% and 66.7%. The corresponding figures for OS were 68.8%, 100%, 100%, and 66.7, respectively. The 5-year non-ocular Grade 3 toxicity free survival was 78.9%. At the start of pbsPT children and their caregivers reported QoL significantly worse than the norm group. QoL improved however over the follow up period to normal values in nearly all domains. Median of PEDQOL PROXY reported QoL in Normgroup (children: 4- 18 yrs) and RMS patients (n = 27-42 answers per domain): CONCLUSION: Excellent clinical outcome was observed for CAYAs with RMS treated with pbsPT. Two thirds of treatment failures were local. High-grade late non-ocular toxicity was manageable. QoL improved towards normal scores in nearly all domains after therapy.