Identifying early-phase clinical trial (EP-CT) participants at risk for poor outcomes.
Authors: Lundquist Debra , Jimenez Rachel , Healy Megan , Johnson Andrew , Durbin Sienna , Capasso Virginia , McIntyre Casandra , Cashavelly Barbara J. , Juric Dejan , Nipp Ryan David 
Published: September 2021

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Background: EP-CTs investigate novel treatment options, with recent advances in personalized therapy leading to increased response rates, decreased toxicity, and improved survival. Identifying EP-CT participants at risk for poor outcomes could help identify those who may benefit most from targeted supportive care efforts. Methods: We retrospectively reviewed the electronic health records of consecutive patients enrolled in EP-CTs from 2017-2019 to obtain baseline characteristics (demographics and clinical factors), clinical outcomes (survival, time on trial, completion of dose-limiting toxicity [DLT] period, emergency room [ER] visits, hospitalizations, and hospice use), and receipt of supportive care services before/during trial (palliative care, social work, physical therapy [PT], and nutrition). We calculated the validated Royal Marsden Hospital (RMH) prognosis score using data at the time of EP-CT enrollment based on patients’ lactate dehydrogenase, serum albumin, and number of sites of metastasis. RMH scores range from 0-3, with scores of 2+ indicating a poor prognosis. We examined differences in patient characteristics, clinical outcomes, and receipt of supportive care services based on the RMH prognosis score. Results: Among 350 patients (median age = 63.2 years [range 23.0-84.3]; 57.1% female, 98.0% metastatic cancer), the most common cancer types were lung (23.4%), gastrointestinal (20.3%), and breast (12.0%). Nearly one-third (31.7%) had an RMH score indicating a poor prognosis. Patients with a poor prognosis RMH score had a worse performance status (ECOG ≥1: 80.2% vs 58.1%, p <.001) and more prior treatment (3+ prior lines: 48.6% vs 34.7%, p =.001) than those with a better prognosis score. Those with a poor prognosis RMH score had worse survival (median: 147 vs 402 days, p <.001) and shorter time on trial (median: 49 vs 84 days, HR = 1.53, p <.001), as well as a lower likelihood of completing the DLT period (72.1% vs 80.8%, p =.015). Patients with a poor prognosis score had a higher risk for ER visits (HR 1.66; p =.037) and hospitalizations (HR 1.69; p =.016) while on trial, with earlier hospice enrollment (HR 2.22; p =.006) following the trial. Patients with a poor prognosis score were significantly more likely to receive palliative care before/during trial (46.8% vs 27.6% p =.001), but not social work (41.4% vs 41.4% p = 1.00), PT (44.1% vs 34.7%; p =.098), or nutrition (40.5% vs 37.2%; p =.557). Conclusions: EP-CT participants represent a unique population of patients with advanced cancer, and we identified a group at risk for particularly poor outcomes, including worse survival, shorter time on trial, and greater use of healthcare services. Although patients with a poor prognosis score had higher rates of palliative care use, under half received supportive care services, underscoring the need for efforts to prospectively target these patients with interventions that address their supportive care needs.