Open Access Study protocol

Development of a disease-specific quality of life questionnaire for adult patients with hereditary angioedema due to C1 inhibitor deficiency (HAE-QoL): Spanish multi-centre research project

Nieves Prior1*, Eduardo Remor2, Carmen Gómez-Traseira1, Concepción López-Serrano1, Rosario Cabañas1, Javier Contreras1, Ángel Campos3, Victoria Cardona4, Stefan Cimbollek5, Teresa González-Quevedo5, Mar Guilarte4, Dolores Hernández Fernández de Rojas3, Carmen Marcos6, María Rubio7, Miguel Ángel Tejedor-Alonso8 and Teresa Caballero19

Author Affiliations

1 Allergy Department, IdiPaz (Hospital La Paz Health Research Institute), Madrid, Spain

2 Universidad Autónoma de Madrid, Madrid, Spain

3 Hospital Universitario La Fe, Valencia, Spain

4 Hospital Universitario Vall d’Hebron, Barcelona, Spain

5 Hospital Universitario Virgen del Rocío, Sevilla, Spain

6 Hospital Xeral Cíes, Vigo, Spain

7 Hospital General Universitario Gregorio Marañón, Madrid, Spain

8 Allergy Unit, Hospital Universitario Fundación Alcorcón, Madrid, Spain

9 Biomedical Research Network on Rare Diseases U754 (CIBERER), Madrid, Spain

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Health and Quality of Life Outcomes 2012, 10:82  doi:10.1186/1477-7525-10-82

Published: 20 July 2012

Abstract

Background

There is a need for a disease-specific instrument for assessing health-related quality of life in adults with hereditary angioedema due to C1 inhibitor deficiency, a rare, disabling and life-threatening disease. In this paper we report the protocol for the development and validation of a specific questionnaire, with details on the results of the process of item generation, domain selection, and the expert and patient rating phase.

Methods/Design

Semi-structured interviews were completed by 45 patients with hereditary angioedema and 8 experts from 8 regions in Spain. A qualitative content analysis of the responses was carried out. Issues raised by respondents were grouped into categories. Content analysis identified 240 different responses, which were grouped into 10 conceptual domains. Sixty- four items were generated. A total of 8 experts and 16 patients assessed the items for clarity, relevance to the disease, and correct dimension assignment. The preliminary version of the specific health-related quality of life questionnaire for hereditary angioedema (HAE-QoL v 1.1) contained 44 items grouped into 9 domains.

Discussion

To the best of our knowledge, this is the first multi-centre research project that aims to develop a specific health-related quality of life questionnaire for adult patients with hereditary angioedema due to C1 inhibitor deficiency. A preliminary version of the specific HAE-QoL questionnaire was obtained. The qualitative analysis of interviews together with the expert and patient rating phase helped to ensure content validity. A pilot study will be performed to assess the psychometric properties of the questionnaire and to decide on the final version.

Keywords:
Hereditary angioedema; C1 inhibitor deficiency; Quality of Life; Questionnaire; Qualitative methodology