Patient and caregiver experiences with selumetinib for the treatment of pediatric patients with neurofibromatosis type 1 and plexiform neurofibromas

Background Plexiform neurofibromas (PN) affect 20–50% of patients with neurofibromatosis type 1 (NF1) and can lead to pain, disfigurement, motor dysfunction, compression of vital structures, and risk of malignant degeneration. Selumetinib was the first pharmacotherapy approved for children aged ≥2 years with symptomatic, inoperable PN in the USA and other countries. This qualitative study was conducted to better understand the drivers for initiating selumetinib and the impact of treatment on quality of life (QoL) from the perspective of pediatric patients with NF1-PN and their caregivers. Methods The study included pediatric patients in the USA (aged 9–18 years) who had been prescribed selumetinib (≥6 months), and their caregivers. Interviews were conducted, and thematic analyses were performed to identify key concepts. A saturation approach established the point at which no new key concepts were being identified with successive interviews. Results Prior to initiating selumetinib, children (N = 10) and their caregivers (N = 19) reported that PN-related issues, such as pain, impacted the lives of those with NF1-PN. Caregivers played a key role in treatment decisions, and initiation of selumetinib helped meet treatment goals including PN size reduction, pain improvement, and improved QoL. Patients experienced increased energy and improvements in pain and all QoL domains post-selumetinib initiation. Conclusions Overall, patients and caregivers reported improvements in pain and QoL after selumetinib initiation. The qualitative, real-world nature of this study provides insights into patient and caregiver perspectives, and the impact of selumetinib on the patient journey.