Describing Pediatric Oncology Patients Enrolled in Phase 1 Clinical Trials

Background

Phase 1 trials may expose pediatric oncology patients to potential adverse effects beyond drug-related toxicity, including delays in advance care planning and suboptimal quality of end-of-life (EoL) care. Pediatric palliative care (PPC) can provide symptom management support and assist with EoL planning and care for patients and families enrolling in Phase 1 trials; however, little is known about children with cancer who enroll in Phase 1 studies.

Methods

A retrospective medical record review of pediatric oncology patients enrolled on a Phase 1 clinical trial over a 9-year period was completed at an academic cancer hospital. Data collected included sociodemographic, clinical, and EoL-related variables. Descriptive analyses were performed.

Results

A total 539 patient charts met the inclusion criteria for review; 53% (n = 285) were male, 76% (n = 411) were Caucasian, and 82% (n = 442) were deceased. Central nervous system (CNS) tumors were the most common diagnosis (44%, n = 237), followed by leukemias (33%, n = 179), solid tumors (20%, n = 108), and lymphomas (3%, n = 15). All patients enrolled in at least one Phase 1 study, with 15% (n = 83) enrolling in two or more. The median length of time on study was 62 days, with time on study decreasing for subsequent Phase 1 trial participation. Two-thirds of patients (68%, n = 368) had PPC consultation; however, only 21% (n = 76) had PPC consulted prior to Phase 1 trial enrollment.

Conclusions

Although most patients enrolled in Phase 1 studies have PPC involvement, referrals are typically made far beyond trial enrollment. Earlier and systematic PPC integration in this high-risk patient population may offer the potential benefit of maximal EoL support, care, and planning.