Re: “The Effect of Atorvastatin on Vascular Function and Structure in Young Adult Survivors of Childhood Cancer: A Randomized,Placebo-Controlled Pilot Clinical Trial” by Marlatt et al. (J Adolesc Young Adult Oncol. 2017 [Epub ahead of print];DOI: 10.1089/jayao.2017.0075)

In a recently published article in the Journal of Adolescent and Young Adult Oncology (JAYAO), Marlatt et al. describe a randomized, placebo-controlled pilot clinical trial wherein the effect of atorvastatin on vascular function and structure in young adult survivors of childhood cancer (YASCC) was examined. ¹ Cancer therapies are thought to damage the lining of the artery walls, including those of the heart, and place a survivor at a high risk of developing cardiovascular disease later in life. As a clinician that provides care to YASCC, I regularly describe the risk of a potential late effect directly to a childhood cancer survivor and then I must share that no evidence-based intervention exists to help lower this risk or prevent the late effect altogether. The lack of evidence-based interventions leaves me and other providers caring for YASCC frustrated and continually surprised at the small number of pharmaceutical interventions reported in and supported by the survivorship literature. As a physician caring for YASCC, I believe we need more evidence-based interventions, including those that are pharmacologic, to better inform our survivor-focused practice for YASCC.

Despite the scarcity of pharmacologic-based interventions to help YASCC avoid late effects, there is a disturbing gap in the literature surrounding clinical trials examining the role of pharmaceuticals in the prevention or amelioration of late effects that arise from the use of most childhood cancer treatments. Most regrettably, the number of pilot studies pertaining to this topic is just as scarce. Although usually not powered to truly assess the effect of a drug intervention, pilot studies with small samples sizes are critical to assess feasibility and provide important preliminary statistical information to calculate power in future large-scale clinical trials (e.g., effect size or standard deviation).

Although publication bias ² (or the differential publishing rate between positive and negative results) likely plays a role in why so few pilot studies have been published, there may also be a competitive advantage for investigators to not share their early findings in an effort to include unique preliminary studies to support their work in large upcoming grant proposals. Regardless, there needs to be a general understanding and acceptance of pilot studies with small sample sizes, yet rigorous methodologies, so that the entire field of pharmacologic intervention studies in YASCC can move forward. JAYAO not only understood this when publishing Marlatt et al., ¹ but the journal has also provided this much needed forum to discuss gaps in the survivorship literature. We hope other survivorship-minded outlets will do the same.