title: Development of the adult PedsQL™ neurofibromatosis type 1 module: initial feasibility, reliability and validity.
abstract: BACKGROUND
  Neurofibromatosis type 1 (NF1) is a common autosomal dominant genetic disorder with significant impact on health-related quality of life (HRQOL). Research in understanding the pathogenetic mechanisms of neurofibroma development has led to the use of new clinical trials for the treatment of NF1. One of the most important outcomes of a trial is improvement in quality of life, however, no condition specific HRQOL instrument for NF1 exists. The objective of this study was to develop an NF1 HRQOL instrument as a module of PedsQL™ and to test for its initial feasibility, internal consistency reliability and validity in adults with NF1.
  
  
  METHODS
  The NF1 specific HRQOL instrument was developed using a standard method of PedsQL™ module development - literature review, focus group/semi-structured interviews, cognitive interviews and experts' review of initial draft, pilot testing and field testing. Field testing involved 134 adults with NF1. Feasibility was measured by the percentage of missing responses, internal consistency reliability was measured with Cronbach's alpha and validity was measured by the known-groups method.
  
  
  RESULTS
  Feasibility, measured by the percentage of missing responses was 4.8% for all subscales on the adult version of the NF1-specific instrument. Internal consistency reliability for the Total Score (alpha =0.97) and subscale reliabilities ranging from 0.72 to 0.96 were acceptable for group comparisons. The PedsQL™ NF1 module distinguished between NF1 adults with excellent to very good, good, and fair to poor health status.
  
  
  CONCLUSIONS
  The results demonstrate the initial feasibility, reliability and validity of the PedsQL™ NF1 module in adult patients. The PedsQL™ NF1 Module can be used to understand the multidimensional nature of NF1 on the HRQOL patients with this disorder.
authors: Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA. knutakki@iupui.edu
authors: Kavitha
authors: K
authors: Nutakki
authors: Cynthia M
authors: CM
authors: Hingtgen
authors: Patrick
authors: P
authors: Monahan
authors: James W
authors: JW
authors: Varni
authors: Nancy L
authors: NL
authors: Swigonski
mesh_terms: D000328:Adult; D000368:Aged; D005260:Female; D017144:Focus Groups; D006801:Humans; D007407:Interviews as Topic; D008297:Male; D008875:Middle Aged; D009456:Neurofibromatosis 1 / Q000523:psychology*; D011788:Quality of Life / Q000523:psychology*; D015203:Reproducibility of Results; D011795:Surveys and Questionnaires / Q000592:standards; D055815:Young Adult