A cohort of French pediatric patients with primary immunodeficiencies: are patient preferences regarding replacement immunotherapy fulfilled in real-life conditions?

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 [F _I ], treatment interference; F _II , therapy-related problems; F _III , 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 F _III was higher for home-based SCIg than for hospital-based IVIg ( P =0.0003), but there was no difference for LQI F _I or LQI F _II . LQI F _III 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 F _I , or LQI F _II .
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.
Competing Interests: Disclosure The study was sponsored by Octapharma France. The sponsor validated the study design, participated in the interpretation of data, had no role in the writing of the study report, and supported the decision to submit the manuscript for publication. BB, GJC, CH, PC, and RJ are part of the scientific committee for the VISAGES study and earned fees from Octapharma. IP, NA, AA, PC, GJC, RJ, BB, and CH were investigators for the VISAGES study. BB, PC, GJC, CH, and RJ take part in several scientific boards and also in several studies led by Octapharma. PC works as an independent statistician and earned fees from Octapharma to conduct the statistical analysis and draft the manuscript. The authors report no other conflicts of interest in this work.