Burden of adult neurofibromatosis 1: development and validation of a burden assessment tool.
Marie-Laure ArmandCharles TaïebAline BourgeoisMireille BourlierMohammed BennaniChristine BodemerPierre Wolkensteinnull nullPublished in: Orphanet journal of rare diseases (2019)
BoN is a specific tool for assessing the burden that NF1 generates on many practical aspects of the patient daily activities, beyond the notion of quality of life". Given the increasing relevance that regulatory authorities attribute to patient-reported outcomes, the BoN questionnaire provides such supplementary information while accounting for the burden of NF1 patients in the broadest sense.
Keyphrases
- patient reported outcomes
- end stage renal disease
- signaling pathway
- risk factors
- ejection fraction
- oxidative stress
- chronic kidney disease
- newly diagnosed
- nuclear factor
- physical activity
- healthcare
- pi k akt
- peritoneal dialysis
- transcription factor
- case report
- patient reported
- prognostic factors
- young adults
- inflammatory response