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

Abstract

Marlène Pasquet,1 Isabelle Pellier,2 Nathalie Aladjidi,3 Anne Auvrignon,4 Patrick Cherin,5 Pierre Clerson,6 Gregoire Jacques Noël Cozon,7 Roland Jaussaud,8 Boris Bienvenu,9 Cyrille Hoarau10 1Pediatric Hematology and Oncology Department, University Hospital Centre of Toulouse, Toulouse, 2University Hospital of Angers, Angers, 3Paediatric Hematology Unit, CEREVANCE, CIC 1401, Inserm CICP, Hospital Pellegrin, 4Trousseau Hospital, 5Internal Medicine Department, Paris, 6Soladis Clinical Studies, Roubaix, 7Clinical Immunology, Edouard Herriot Hospital, Lyon, 8Internal Medicine and Clinical Immunology Department, University Hospital Centre of Nancy, Nancy, 9Internal Medicine Department, University Hospital Centre of Caen, Caen, 10Renal Transplantation and Clinical immunology Department, University Hospital Centre of Tours, Tours, France 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. Keywords: primary immunodeficiency, pediatric, immunotherapy, immunoglobulins, satisfaction, preference

Keywords

• Primary immunodeficiency Pediatric Immunotherapy Immunoglobulins Satisfaction Preference