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Open Access Research

Development of the Adult PedsQL™ Neurofibromatosis Type 1 Module: Initial Feasibility, Reliability and Validity

Kavitha Nutakki1*, Cynthia M Hingtgen2, Patrick Monahan3, James W Varni45 and Nancy L Swigonski16*

Author Affiliations

1 Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA

2 Department of Clinical Neurosciences, Spectrum Health Medical Group and College of Human Medicine, Michigan State University, Grand Rapids, MI, USA

3 Department of Biostatistics, Indiana University School of Medicine, Indianapolis, IN, USA

4 Department of Pediatrics, College of Medicine, Texas A&M University, College Station, TX, USA

5 Department of Landscape Architecture and Urban Planning, College of Architecture, Texas A&M University, College Station, TX, USA

6 Department of Public Health, Indiana University School of Medicine, Indianapolis, IN, USA

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Health and Quality of Life Outcomes 2013, 11:21  doi:10.1186/1477-7525-11-21

Published: 21 February 2013

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.

Keywords:
PedsQL™; Neurofibromatosis type 1 (NF1); Health-related quality of life; Patient reported outcomes