“From Drowning to Treading Water”: Adolescents and Young Adults Living with Incurable and Indolent Metastatic Soft Tissue Sarcoma for More than Two Years
Authors: D’Alessandro Paul R. , Homanick Caitlin E. , Cooper Brittany D. , Ferguson Katelyn , Rutan Hillary , Pressey Joseph G. 
Published: January 2025

Introduction: Adolescent/young adult (AYA) patients with metastatic soft tissue sarcoma (STS) typically face a dismal prognosis. However, a subset of patients with incurable disease lives beyond two years. Due to the rarity of diagnoses and inherent heterogeneity within this population, a paucity of data exists regarding the experiences of AYAs with an indolent course (and how to best capture these experiences). With increasing biological insight and clinical experience, including the use of targeted or immune therapies, it is anticipated that more such patients will experience prolonged survival. Our pilot study aimed to describe the clinical characteristics and illness experiences of AYAs with incurable yet indolent metastatic STS who were living two years after their diagnoses. Our exploratory aim was to generate a conceptual framework that could subsequently be tested in a multi-center study with a larger cohort of patients. Materials and Methods: Patients with metastatic incurable STS, aged 15–39 years at diagnosis, and at least two years from diagnosis, were eligible. Patients were recruited over a two-year period at a quaternary children’s hospital with a comprehensive AYA oncology program. Participants completed a demographic form and PROMIS short form questionnaires for seven domains and answered an open-ended question. Responses to open-ended questions were coded independently by two authors and utilized to generate themes. Clinical variables were collected from medical records. Results: Five patients completed questionnaires. Mean age was 29.4 years (18.5–39.8 years) at diagnosis and 34 years (23.2–45.7 years) at study. Three patients were female; two were male; four were White; and one was Black/African American. Diagnoses included ASPSCR1::TFE3 alveolar soft part sarcoma; WWTR1::CAMTA1 epithelioid hemangioendothelioma; INI-1 deficient epithelioid sarcoma; EWSR1::NR4A3 extra-skeletal myxoid chondrosarcoma; and low-grade ARHGAP23::FER spindle cell malignancy, a novel fusion-driven sarcoma. Mean time since diagnosis was 4.5 years (2.6–6 years), and mean treatment duration was 4.2 years (1.5–6 years). On average, patients received 4.8 lines (range 2–8 lines) of antineoplastic therapy. All patients received at least one targeted therapy or immune checkpoint inhibitor. Patients reported increased fatigue and anxiety and decreased physical function compared to the standardized US reference population. Themes emerging from qualitative responses included managing physical symptoms, navigating feelings of guilt and inadequacy, self-reflection generating gratitude, and changing illness experiences over time. Conclusions: AYA patients living with incurable metastatic soft tissue sarcoma for more than two years were treated with multiple lines of antineoplastic therapy longitudinally. PROMIS data identified fatigue, anxiety, and decreased physical function within this population. Exploratory thematic analysis of qualitative responses generated concepts that could be further tested in an expanded cohort of patients.