Abstract Background Central nervous system (CNS) cancers remain the leading cause of cancer death in children. Precision medicine, which aims to identify targeted therapeutic options, offers affected families hope given insufficient effective treatment options. This study explored the experiences of families of children with CNS cancers participating in the Australian ZERO Childhood Cancer Program’s precisionmedicine trial for poor prognosis cancers (PRISM). Methods In this mixed-methods prospective longitudinal study (PRISM-Impact) we examined parents’ and patients’ expectations, recall, and experiences of receiving treatment recommendations. Ninety-six families participated. Parents and patients (aged 12-17 years) completed questionnaires at trial enrolment (n = 116 parents and n = 6 patients) and following return of any treatment recommendations (n = 54 and n = 2, respectively). Twenty-one parents also completed an interview exploring their trial experiences. Results At enrolment all patients and most parents reported expecting at least some benefit from trial participation (100% and 88% respectively). Most parents (77%) thought it was at least somewhat likely that PRISM would identify a treatment recommendation for their child. According to trial records, 79% of families received a treatment recommendation. Forty-seven percent of parents recalled this information, and a further 28% reported being unsure. Parents shared that treatment recommendations offered hope and potential treatment options; their absence brought disappointment, but also reassurance that all options were explored. Parents felt they understood treatment recommendations well (median score: 88/100) and reported high involvement (88/100) and satisfaction (88/100) with treatment decisions. Conclusions Families of children with CNS cancers approach precision medicine trials with expectations of benefit. Our findings indicate that families value precision medicine trials, irrespective of the receipt of treatment recommendations. Improved communication around treatment recommendations may strengthen parent recall and thus facilitate shared decision making in this setting.
Hetherington et al. (Tue,) studied this question.
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