Vincent Barlogis1, Nizar Mahlaoui2, Pascal Auquier3, Isabelle Pellier4, Fanny Fouyssac5, Camille Vercasson3, Maya Allouche6, Carolina Brito De Azevedo7, Felipe Suarez8, Despina Moshous2, Bénédicte Neven2, Marlène Pasquet9, Eric Jeziorski10, Nathalie Aladjidi11, Nicolas Schleinitz12, Caroline Thomas13, Virginie Gandemer14, Françoise Mazingue15, Patrick Lutz16, Olivier Hermine17, Capucine Picard2, Stéphane Blanche2, Gérard Michel18, Alain Fischer19. 1. Department of Pediatric Hematology-Oncology, Assistance Publique-Hôpitaux de Marseille, Marseille, France; French National Reference Center for Primary Immune Deficiency (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Department of Public Health-EA 3279 Research Unit, University Hospital Marseille, Aix-Marseille University, Marseille, France. Electronic address: vincent.barlogis@ap-hm.fr. 2. French National Reference Center for Primary Immune Deficiency (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Pediatric Immuno-Haematology and Rheumatology Unit, Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; INSERM UMR1163, Imagine Institute, Necker Medical School, Sorbonne Paris Cité, University Paris Descartes, Paris, France. 3. Department of Public Health-EA 3279 Research Unit, University Hospital Marseille, Aix-Marseille University, Marseille, France. 4. French National Reference Center for Primary Immune Deficiency (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Department of Pediatric Oncohematology, University Hospital of Angers, Angers, France. 5. French National Reference Center for Primary Immune Deficiency (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Department of Pediatric Hematology-Oncology, University Hospital of Nancy, Nancy, France. 6. Department of Pediatric Hematology-Oncology, Assistance Publique-Hôpitaux de Marseille, Marseille, France. 7. French National Reference Center for Primary Immune Deficiency (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France. 8. French National Reference Center for Primary Immune Deficiency (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; INSERM UMR1163, Imagine Institute, Necker Medical School, Sorbonne Paris Cité, University Paris Descartes, Paris, France; Adult Hematology Unit, Necker University Hospital, APHP, University Paris Descartes, Paris, France; CNRS ERL8254, Imagine Institute, Necker Medical School, Sorbonne Paris Cité, University Paris Descartes, Paris, France. 9. French National Reference Center for Primary Immune Deficiency (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Département d'Hématologie, Centre Hospitalier Universitaire Toulouse Purpan and INSERM, CRCT, IUCT-Oncopole, Toulouse, France. 10. French National Reference Center for Primary Immune Deficiency (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Service de Pédiatrie Générale et Infectiologie et Immunologie Clinique, Hôpital Arnaud de Villeneuve, Montpellier University Hospital, Montpellier, France. 11. French National Reference Center for Primary Immune Deficiency (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Department of Pediatric Hematology and CIC 0005, INSERM CICP, University Hospital of Bordeaux, Bordeaux, France. 12. French National Reference Center for Primary Immune Deficiency (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Department of Internal Medicine, CHU Timone, Assistance Publique-Hôpitaux de Marseille, Marseille, France. 13. French National Reference Center for Primary Immune Deficiency (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Pediatric Hematology-Oncology Unit, Hôpital Mère Enfant, University Hospital of Nantes, Nantes, France. 14. French National Reference Center for Primary Immune Deficiency (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Department of Pediatric Hematology/Oncology, University Hospital of Rennes, Rennes, France. 15. French National Reference Center for Primary Immune Deficiency (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Department of Pediatrics, Hôpital Jeanne de Flandre, University Hospital of Lille, Lille, France. 16. French National Reference Center for Primary Immune Deficiency (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Pediatric Hematology-Oncology, EA3430, Strasbourg, France. 17. French National Reference Center for Primary Immune Deficiency (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; INSERM UMR1163, Imagine Institute, Necker Medical School, Sorbonne Paris Cité, University Paris Descartes, Paris, France; Adult Hematology Unit, Necker University Hospital, APHP, University Paris Descartes, Paris, France. 18. Department of Pediatric Hematology-Oncology, Assistance Publique-Hôpitaux de Marseille, Marseille, France; French National Reference Center for Primary Immune Deficiency (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Department of Public Health-EA 3279 Research Unit, University Hospital Marseille, Aix-Marseille University, Marseille, France. 19. French National Reference Center for Primary Immune Deficiency (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Pediatric Immuno-Haematology and Rheumatology Unit, Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; INSERM UMR1163, Imagine Institute, Necker Medical School, Sorbonne Paris Cité, University Paris Descartes, Paris, France; Collège de France, Paris, France.
Abstract
BACKGROUND: Most children with primary immunodeficiencies (PIDs) now reach adulthood. However, few studies have evaluated their health status and health-related quality of life (HRQoL). OBJECTIVE: To investigate long-term morbidity, the French Reference Center for PIDs initiated a prospective multicenter cohort: the French Childhood Immune Deficiency Long-term Cohort. The data collected were used to assess the physical health condition of patients who reached adulthood and the effect on their quality of life. METHODS: Patients were asked to complete health status questionnaires. A severity score (grade 1 [mild] to grade 4 [life-threatening]) was assigned to each health condition. The HRQoL of patients was compared with age- and sex-matched French normal values by using the 36-item Short-Form Survey (SF-36) HRQoL questionnaire. RESULTS: Among 329 participants, the mean age at evaluation was 27.6 years, with a 21-year mean follow-up after diagnosis; 43% reported at least 1 grade 4 health condition, and 86% reported at least 1 grade 3 (severe) or 4 health condition. Twenty-five (7.6%) patients had been treated for cancer. Compared with the French normal values, adults with PIDs scored significantly lower for all HRQoL domains. HRQoL was strongly associated with the burden of health conditions. The association with grade 4 or grade 3-4 health conditions was highly significant for all physical and mental domains. CONCLUSION: Adults with PIDs diagnosed during childhood experienced a heavy burden of health conditions, which affected their HRQoL. Our results emphasize the need to closely monitor this vulnerable population.
BACKGROUND: Most children with primary immunodeficiencies (PIDs) now reach adulthood. However, few studies have evaluated their health status and health-related quality of life (HRQoL). OBJECTIVE: To investigate long-term morbidity, the French Reference Center for PIDs initiated a prospective multicenter cohort: the French Childhood Immune Deficiency Long-term Cohort. The data collected were used to assess the physical health condition of patients who reached adulthood and the effect on their quality of life. METHODS:Patients were asked to complete health status questionnaires. A severity score (grade 1 [mild] to grade 4 [life-threatening]) was assigned to each health condition. The HRQoL of patients was compared with age- and sex-matched French normal values by using the 36-item Short-Form Survey (SF-36) HRQoL questionnaire. RESULTS: Among 329 participants, the mean age at evaluation was 27.6 years, with a 21-year mean follow-up after diagnosis; 43% reported at least 1 grade 4 health condition, and 86% reported at least 1 grade 3 (severe) or 4 health condition. Twenty-five (7.6%) patients had been treated for cancer. Compared with the French normal values, adults with PIDs scored significantly lower for all HRQoL domains. HRQoL was strongly associated with the burden of health conditions. The association with grade 4 or grade 3-4 health conditions was highly significant for all physical and mental domains. CONCLUSION: Adults with PIDs diagnosed during childhood experienced a heavy burden of health conditions, which affected their HRQoL. Our results emphasize the need to closely monitor this vulnerable population.
Authors: Thomas F Michniacki; Kelly J Walkovich; Lauren E Merz; Julie Sturza; Roshini S Abraham Journal: J Clin Immunol Date: 2019-10-26 Impact factor: 8.317
Authors: Irina Odnoletkova; Gerhard Kindle; Isabella Quinti; Bodo Grimbacher; Viviane Knerr; Benjamin Gathmann; Stephan Ehl; Nizar Mahlaoui; Philippe Van Wilder; Kris Bogaerts; Esther de Vries Journal: Orphanet J Rare Dis Date: 2018-11-12 Impact factor: 4.123