Association between patient-reported outcomes and objective disease indices in people with NF2.

ABSTRACT:

Objective

The association between patient-reported outcomes and currently used clinical trial endpoints, including total vestibular schwannoma (VS) volume and word recognition score (WRS) in neurofibromatosis type 2 (NF2), is not known.

Methods

A prospective observational study enrolling adult patients with NF2 was conducted at a single specialty center. Measures included: NF2 impact on quality of life (NFTI-QOL), short form (SF)-36; total VS volume, WRS; provider- and patient-reported disease severity (ProvSev, PatSev) measured with an institutionally derived multi-item (e.g., symptom burden, age-of-onset, and fatality-risk) and single-item Likert (mild, moderate, severe) scale.

Results

Fifty-one patients were enrolled between June 2014 and August 2017. Mean age was

SUBMITTER: Shukla A 

PROVIDER: S-EPMC6745746 | biostudies-literature | 2019 Aug

REPOSITORIES: biostudies-literature