Overcoming barriers to clinical trial enrollment in patients with bone and soft tissue sarcoma: a paradigm for an increasingly heterogeneous cancer population
Abstract
Background: Clinical trials remain a cornerstone in the development of new cancer therapies. Patients diagnosed with bone and soft tissue sarcoma typically have comparatively low rates of trial participation, which may contribute to lack of progress. This cross-sectional survey study was designed to characterize patient attitudes, knowledge, perceived ability (ie, self-efficacy), receptivity, and willingness to participate in trials. As well, we explored perceptions related to tumor molecular profiling (TMP).
Methods: Patients with sarcoma who were evaluated at Northwestern Medicine (NM) between 2012 and 2017 were identified through the Enterprise Data Warehouse (NMEDW). A link to an online self-administered survey was emailed to patients. Data were analyzed using Spearman correlations and the Mann-Whitney test.
Results: Surveys were e-mailed to 750 patients, of which 20 emails bounced back and 309 were opened; 283 patients completed at least a portion of the survey and this population was used in this analysis (37.7% of total and 91.6% of opened). Of the responding patients, median age was 56 years, 59.4% were female, and 26.8% reported metastatic disease. Greater knowledge of trials correlated with increased positive attitudes toward trial participation (r=0.5; P<0.001) and positive attitudes correlated with greater trial perceived ability (r=0.4; P<0.02). Patients with metastatic disease had more positive attitudes compared with patients with non-metastatic disease (P=0.03). Trial enrollment was associated with greater knowledge (P=0.002) and positive attitudes (P=0.001). Among patients who reported knowledge of TMP (n=46), 65% credit TMP with a >50% chance of isolating a targetable result. Of patients who had TMP performed (n=18), important considerations included wanting to live as long as possible and helping future cancer patients, but not wanting to be part of research.
Conclusions: Increasing awareness, knowledge, and attitudes towards trials among patients with sarcoma may lead to increased trial enrollment and greater progress in cancer treatment. Patient-focused interventions and research-site optimization are critical to maximizing accrual rates and biomarker-driven therapeutics. Lessons from a sarcoma patient population can be used to optimize trial enrollment in an era of increasingly heterogeneous cancer populations.
Statistical analysis
Responses were separated from the personal data for complete anonymization. Descriptive statistical analysis was performed for demographics and disease variables and were summarized using frequencies and percentages. Median and range were used for age. Correlations between continuous variables were analyzed using Spearman correlations. Scores were compared between subgroups using the Mann-Whitney test. Descriptive statistics for knowledge, attitude, and ability scores include means and 95% confidence intervals. Correlations were interpreted as small (r=0.10), medium (r=0.30), or large (r=0.50).15 Statistical significance was indicated when P<0.05.
Results
Patients
Seven hundred fifty patients were eligible to participate in the survey and received the initial and two follow-up e-mails. Twenty e-mailed surveys bounced back. Three hundred nine patients opened the initial e-mail and 283 patients (37.7% of total and 91.6% of opened) completed at least a portion of the survey, with 182 patients completing the entire survey (FIGURE 1). Data for analysis were used from patients who completed at least a portion of the survey.
Baseline characteristics of patients who responded can be seen in TABLE 2. Patients had a median age of 56, the majority were female (59.4%), white (88.2%), and most had college or university graduate degrees or higher educational level (69.0%). Patients had various different histological subtypes, with the most common being liposarcoma (16.5%) and leiomyosarcoma (16.0%). Slightly more than a quarter (26.8%) of patients had metastatic disease, and 84.2% had never been enrolled in a clinical trial. Previous treatments included surgery (91.1%), radiation (53.2%), and chemotherapy (51.6%). Prior to completing the survey, 85.4% reported being receptive to a cancer clinical trial, while 60.7% of patients reported willingness to participate in a clinical trial.
