Development and validation of an epidermolysis bullosa family/parental burden score.
BACKGROUND
The notion of the individual burden associated with a disease has been introduced to determine 'disability' in the broadest sense: psychological, social, economic and physical. Subtypes of epidermolysis bullosa (EB) are rare, life-threatening, untreatable chronic genodermatoses.


OBJECTIVE
To develop and validate a specific questionnaire assessing the burden on families of children with EB: Epidermolysis Bullosa Burden of Disease (EB-BoD).


METHODS
Items were generated by a verbatim report from parents of children with EB. Subsequently, a study was implemented for psychometric analysis. An epidermolysis bullosa burden-of-disease questionnaire was refined via item reduction according to inter-question correlations, consensus among experts and exploratory factor analysis. Internal consistency was determined by calculating Cronbach's α. Concurrent validity was determined by calculating the correlation between EB-BoD and the Short-Form 12 items (SF-12) questionnaire.


RESULTS
From a primary list of 30 items, EB-BoD was reduced to a 20-item questionnaire, covering four disease aspects based on the exploratory factor analysis. Construct validity was demonstrated and the EB-BoD questionnaire showed good internal consistency (Cronbach's α = 0·9). The resulting EB-BoD score was significantly correlated to the mental dimension of SF-12 (r = -0·61), but it was not correlated to it's physical dimension (r = 0·04). EB-BoD scores were significantly discriminating between EB subtypes.


CONCLUSIONS
The EB-BoD questionnaire appears to be a useful assessment tool regarding medical and socioeconomic issues in patients with EB and their families. EB-BoD scores correlate well with the family/parental burden experienced by the families of patients with EB.
Department of Dermatology, Reference Center for Genodermatoses and Rare Skin Diseases (MAGEC), Institut Imagine, Hôpital Universitaire Necker-Enfants Malades, 149 Rue de Sèvres, 75015, Paris, France.
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Dufresne
Department of Dermatology, Reference Center for Genodermatoses and Rare Skin Diseases (MAGEC), Institut Imagine, Hôpital Universitaire Necker-Enfants Malades, 149 Rue de Sèvres, 75015, Paris, France.|NSERM U1163, Université Paris Descartes - Sorbonne Paris Cité, Institut Imagine, 149 Rue de Sèvres, 75015, Paris, France.
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Hadj-Rabia
Public Health and Quality of Life, Pierre Fabre, Paris, France.
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Taieb
Department of Dermatology, Reference Center for Genodermatoses and Rare Skin Diseases (MAGEC), Institut Imagine, Hôpital Universitaire Necker-Enfants Malades, 149 Rue de Sèvres, 75015, Paris, France.|NSERM U1163, Université Paris Descartes - Sorbonne Paris Cité, Institut Imagine, 149 Rue de Sèvres, 75015, Paris, France.
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Bodemer
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D017281:Cost of Illness; D004820:Epidermolysis Bullosa / Q000523:psychology*; D005192:Family Health; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D010290:Parents / Q000523:psychology; D011446:Prospective Studies; D011788:Quality of Life; D011795:Surveys and Questionnaires / Q000592:standards*