Progress in controlling symptoms and maximizing health-related quality of life lags behind advances in disease-directed therapies. This work addresses the paucity of knowledge regarding symptom management in children with advanced cancer.
Part I. Low enrollment and high attrition in an already small population hamper meaningful controlled studies, in particular, the gold-standard randomized controlled trial (RCT). Novel RCT designs, however, may enhance participation. Understanding patient willingness to participate in various RCT designs may facilitate conduct of rigorous, feasible studies.
Part II. Fatigue causes significant suffering but is under-treated in children with advanced cancer. Methylphenidate (MPH) improves fatigue in adults with cancer and HIV. MPH is used extensively for children with ADHD but data regarding its efficacy and side effect profile in children with advanced cancer are lacking.
I. To understand which pediatric cancer patients enter symptom-related studies and which designs promote participation.
II. To evaluate efficacy and side effects of MPH for cancer-related fatigue in children.
I. We will survey parents of children and adolescent patients being treated for cancer at Dana-Farber/Children痴 Hospital. Survey domains include patient and parent characteristics, perceived benefits and burdens, and preferences for RCT designs. Oncologists will be surveyed regarding their perspectives on RCT designs and their likelihood of offering participation. The primary endpoint for both surveys will be likelihood of participating/offering participation in each RCT design.
II. We will conduct an RCT involving 30 children with the design determined by Part I findings. The primary endpoint will be change in median fatigue scores on the Symptom Distress Scale during treatment and placebo periods.
This study will contribute to a foundation for sound, compassionate symptom-related research in palliative care and will employ knowledge gained to evaluate an intervention to alleviate suffering in this population.