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Objective: To assess quality of life and satisfaction regarding immunoglobulin-replacement therapy (IgRT) treatment according to the route (intravenous Ig [IVIg] or subcutaneous Ig [SCIg]) and place of administration (home-based IgRT or hospital-based IgRT). Subjects and methods: Children 5-15 years old treated for primary immunodeficiency disease (PIDD) with IgRT for ≥3 months were included in a prospective, noninterventional cohort study and followed over 12 months. Quality of life was assessed with the Child Health Questionnaire - parent form (CHQ-PF)-50 questionnaire. Satisfaction with IgRT was measured with a three-dimensional scale (Life Quality Index [LQI] with three components: factor I [FI], treatment interference; FII, therapy-related problems; FIII, therapy settings). Results: A total of 44 children (9.7±3.2 years old) receiving IgRT for a mean of 5.6±4.5 years (median 4.1 years) entered the study: 18 (40.9%) were receiving hospital-based IVIg, two (4.6%) were receiving home-based IVIg, and 24 (54.6%) were treated by home-based SCIg. LQI FIII was higher for home-based SCIg than for hospital-based IVIg (P=0.0003), but there was no difference for LQI FI or LQI FII. LQI FIII significantly improved in five patients who switched from IVIg to SCIg during the follow-up when compared to patients who pursued the same regimen (either IVIg or SCIg). No difference was found on CHQ-PF50 subscales, LQI FI, or LQI FII. Conclusion: Home-based SCIg gave higher satisfaction regarding therapy settings than hospital-based IVIg. No difference was found on other subscales of the LQI or CHQ-PF50 between hospital-based IVIG and home-based SCIG. [ABSTRACT FROM AUTHOR]

Abstract: Registries of patients with rare diseases are important and represent valuable sources of information on epidemiology, natural history of the disease, treatment response and medical practices. The French ‘observatoire’ on Gaucher''s disease has both an epidemiological and educational objective: It will describe a large population of adult patients with special attention to complications and quality of life assessment. All adult patients with a confirmed diagnosis of Gaucher''s disease will be enrolled at the time of their regular visit to the investigational centre. Collection will be as comprehensive as possible, covering the classical parameters of Gaucher''s disease with patients'' complaints, clinical abnormalities, imaging and biological assessments and concomitant medications. Special attention will be devoted to investigating the nature, frequency and severity of associated complications with special emphasis on neurological and bone involvement. Quality of life will be assessed using the SF-36 questionnaire. Recruitment will last for 1 year from May 2005. These large database collected will enhance disease knowledge and ultimately lead to improved long-term management of Gaucher''s disease patients. [Copyright &y& Elsevier]