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Background: Phase 1 clinical trials introduce new therapies to humans with the goal of establishing their safety. A prior Children's Oncology Group (COG) study analyzed the proportional enrollment of patients by race, ethnicity, sex, and age for all trial phases. The current study evaluated the representation of patients by race, ethnicity, sex, and age in phase 1 clinical trials.Methods: This study evaluated 1348 children with 128 diagnoses enrolled in COG and Pediatric Brain Tumor Consortium phase 1 clinical trials in the United States from February 28, 2000 to December 29, 2008. Observed and expected proportions were calculated according to an established methodology with a representative population from Surveillance, Epidemiology, and End Results data, which included 27,766 children with the same International Classification of Diseases for Oncology (third edition) diagnostic codes.Results: Underrepresentation in phase 1 trials was seen for lymphohematopoietic (LH) tumors (9.3% observed vs 37% expected) versus solid tumors (90.6% observed vs 63% expected). Although representation was fairly proportional, Hispanics (12.6% observed vs 27% expected), particularly Hispanic females (6% observed vs 18% expected), were significantly underrepresented. The 0- to 4-year age group was underrepresented (11.7% observed vs 36.5% expected). By tumor type, the most significantly underrepresented groups were 0- to 4-year-old children and Hispanics for both solid cancers (11% observed vs 34.4% expected for 0- to 4-year-old children and 12% observed vs 24% expected for Hispanics) and LH cancers (16% observed vs 40% expected for 0- to 4-year-old children and 19.4% observed vs 33% expected for Hispanics).Conclusions: Although sex and racial/ethnic groups are mostly proportionally represented in phase 1 trials, some specific subgroups such as Hispanic children are underrepresented and may benefit from focused accrual. Cancer 2016;122:3207-14. © 2016 American Cancer Society. [ABSTRACT FROM AUTHOR]

Background: The last 3 decades have witnessed limited therapeutic advances in small cell lung cancer (SCLC) management. This study evaluated real-world trends in the use of systemic therapies and the impact on patient outcomes in the United States.Methods: The Surveillance, Epidemiology, and End Results-Medicare database was used to find patients diagnosed with SCLC between 1985 and 2005. The 1985-1990 period served as the baseline for a temporal analysis conducted at 5-year intervals (1985-1990, 1991-1995, 1996-2000, and 2001-2005). Cox proportional models were used to estimate the effect of chemotherapy on survival. Results were validated with a propensity-matched analysis.Results: There were 47,351 eligible patients: 52% were male; the median age was 71 years; and 87% were white, 7% were black, and 1.4% were Asian. The proportion of patients treated with chemotherapy was low but increased over time (38%, 55%, 50%, and 53%; P < .001). Race, diagnosis period, age, stage, and location of residence significantly predicted chemotherapy use. Females (51%), Asians (53%), and rural residents (60%) were more likely to receive chemotherapy. The median overall survival with and without chemotherapy was 9.6 and 3.6 months, respectively. Linear trend analyses showed a modest reduction in the impact of chemotherapy on survival for patients treated with chemotherapy versus untreated patients (hazard ratios [HRs], 0.59, 0.61, 0.64, and 0.62; P < .001) but an overall trend of improved survival for treated (HRs, 1.0, 1.03, 1.00, and 0.96; P = .005) and untreated patients (HRs, 1.0, 0.99, 0.94, and 0.92; P < .001). There was no survival difference between patients treated with carboplatin and patients treated with cisplatin (HR, 0.99; confidence interval [CI], 0.81-1.19; P = .875). Additional therapy beyond platinum-based chemotherapy was associated with a survival benefit (HR, 0.78; CI, 0.75-0.81; P < .001).Conclusions: Chemotherapy use was associated with a survival benefit in Medicare patients with SCLC treated in a real-world setting. [ABSTRACT FROM AUTHOR]