A National Modified Delphi Consensus on the Referral and Management of NF1 Plexiform Neurofibroma.

Abstract

Background and objectives: Neurofibromatosis type 1 (NF1) is a genetic disorder. Up to 50% of NF1 patients develop plexiform neurofibromas (PN). Despite revisions in diagnostic standards, there remains a lack of consensus on referral, treatment, monitoring and transition processes for NF1-PN. The study aimed to establish a Canada-wide consensus on the best practice for referral and management of patients with NF1-PN to help generate guidance where evidence on the long-term use of MEK inhibitors is lacking.

Methods: The study used a modified Delphi method. The steering committee (SC) identified 4 topics of focus and developed 44 consensus statements. Following ratification, 43 statements were developed into an online survey sent to 113 healthcare practitioners (HCPs) involved in NF1-PN management across Canada. Respondents used a 4-point Likert scale to indicate agreement with each statement. The threshold for consensus agreement was 75%.

Results: A total of 56 responses were received, predominantly from Ontario. Most respondents were neuro-oncologists (34%) and had over 11 years of experience (57%). Consensus was reached on 41 of 43 statements (95%), enabling the SC to develop recommendations for NF1-PN patient care and a treatment algorithm outlining key timings for treatment and management.

Conclusions: To our knowledge, this is the first national Delphi consensus on NF1-PN. Strong agreement was seen from HCPs on critical timings in NF1-PN treatment and management. The proposed recommendations and treatment algorithm provide a framework to enhance patient care and support ongoing research into optimizing care for NF1-PN patients, not just in Canada but globally.