Exostoses, Multiple Hereditary Clinical Trial
Official title:
The Health-Related Quality of Life in Patients With Hereditary Multiple Exostoses
The purpose of this study is to assess the health-related quality of life of subjects who have Hereditary Multiple Exostoses and to develop a disease specific quality of life survey. The investigators hypothesize that there are a wide range of quality of life experiences for patients with this syndrome.
At the time of recruitment, subjects will be asked to indicate whether they would like to participate in a one-time survey, or are willing to participate in the initial survey plus two additional questionnaire administrations, thereafter, which will be used to develop the disease-specific quality of life (QOL) score. All participants will complete either the SF-36 survey (for adult HME subjects) or the CHQ PF (for parents of children with HME), as applicable, in addition to a blank form with categorical subheadings (such as recreation, social function, etc.), which they will use to address any issues not covered in the standardized questionnaire. Standardized questionnaires will be scored and analyzed. The items listed on the second form will be compiled and reapplied to subjects that indicated interest in extended participation in survey completion. Subjects will be asked to determine which items apply to them and to rank these applicable items in order of importance. The results from this will be used to develop a 30 question, disease-specific questionnaire. The final step will be to readminister this survey, in conjunction with the SF-36 or CHQ PF, and follow up, in two weeks, with a second administration of the aforementioned surveys, in order to test the variability and validity of the disease-specific, health-related, quality of life score. ;
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