Burden of Poor Health Conditions and Quality of Life in 656 Children with Primary Immunodeficiency - 01/12/17

Doi : 10.1016/j.jpeds.2017.10.029

Vincent Barlogis, MD, PhD ^1,^2,^3,^* , Nizar Mahlaoui, MD ^2,^4,⁵, Pascal Auquier, MD, PhD ³, Fanny Fouyssac, MD ^2,⁶, Isabelle Pellier, MD, PhD ^2,⁷, Camille Vercasson, MS ³, Maya Allouche, PhD ¹, Carolina Brito De Azevedo, PhD ², Despina Moshous, MD, PhD ^2,^4,⁵, Bénédicte Neven, MD, PhD ^2,^4,⁵, Marlène Pasquet, MD, PhD ^2,⁸, Eric Jeziorski, MD, PhD ^2,⁹, Nathalie Aladjidi, MD ^2,¹⁰, Caroline Thomas, MD ^2,¹¹, Virginie Gandemer, MD, PhD ^2,¹², Françoise Mazingue, MD ^2,¹³, Capucine Picard, MD, PhD ^2,^4,⁵, Stéphane Blanche, MD ^2,^4,⁵, Gérard Michel, MD ^1,^2,³, Alain Fischer, MD, PhD ^2,^4,^5,¹⁴
¹ Department of Pediatric Hematology-Oncology, Assistance Publique-Hôpitaux de Marseille, Marseille, France
² French National Reference Center for Primary Immune Deficiency (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
³ Department of Public Health—EA 3279 Research Unit, Aix-Marseille University, Provence, France
⁴ Pediatric Immuno-Haematology and Rheumatology Unit, Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
⁵ INSERM UMR1163, Imagine Institute, Sorbonne Paris Cité, University Paris Descartes, Paris, France
⁶ Department of Pediatric Hematology-Oncology, University Hospital of Nancy, Nancy, France
⁷ Department of Pediatric Oncohematology, University Hospital of Angers, Angers, France
⁸ Department of Pediatric Hematology, University Hospital of Toulouse, IUCT-Oncopole, INSERM, Toulouse, France
⁹ Department of Pediatrics, Hôpital Arnaud de Villeneuve, Montpellier University Hospital, Montpellier, France
¹⁰ Department of Pediatric Hematology, CIC 0005, INSERM CICP, University Hospital of Bordeaux, Bordeaux, France
¹¹ Pediatric Hematology-Oncology Unit, University Hospital of Nantes, Nantes, France
¹² Department of Pediatric Hematology/Oncology, University Hospital of Rennes, Rennes, France
¹³ Department of Pediatrics, Hôpital Jeanne de Flandre, University Hospital of Lille, Lille, France
¹⁴ Division of Médecine Expérimentale, Collège de France, Paris, France

^*Reprint requests: Vincent Barlogis, MD, PhD, Department of Pediatric Hematology Oncology, Hôpital d'enfants La Timone, Université Aix-Marseille, 13385 Marseille Cedex5, Marseille, France.Department of Pediatric Hematology OncologyHôpital d'enfants La TimoneUniversité Aix-Marseille13385 Marseille Cedex5MarseilleFrance

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Abstract

Objective

To gain insight into how primary immunodeficiencies (PIDs) affect children's health status and quality of life.

Study design

The French Reference Center for PIDs conducted a prospective multicenter cohort that enrolled participants who met all criteria: patients included in the French Reference Center for PIDs registry, children younger than18 years, and living in France. Participants were asked to complete both a health questionnaire and a health-related quality of life (HR-QoL) questionnaire. A severity score was assigned to each health condition: grade 1 (mild) to grade 4 (life-threatening). HR-QoL in children was compared with age- and sex-matched French norms.

Results

Among 1047 eligible children, 656 were included in the study, and 117 had undergone hematopoietic stem cell transplantation; 40% experienced at least one grade 4 condition, and 83% experienced at least one grade 3 or 4 condition. Compared with the French norms, children with PID scored significantly lower for most HR-QoL domains. Low HR-QoL scores were associated strongly with burden of poor conditions.

Conclusions

Our results quantify the magnitude of conditions in children with PID and demonstrate that the deleterious health effects borne by patients already are evident in childhood. These results emphasize the need to closely monitor this vulnerable population and establish multidisciplinary healthcare teams from childhood.

Trial registration

ClinicalTrials.gov: NCT02868333 and EudraCT 2012-A0033-35.

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Keywords : primary immunodeficiency, health status, transition to adulthood

Abbreviations : CEREDIH, CTCAE, ENT, F-CILC, HR-QoL, HSCT, PAD, PID, VSP-A, VSP-Ae, VSP-Ap

Plan

Methods

Statistical Analyses

Results

Health Condition Outcomes

Quality of Life

Discussion

Supported by the French National Program for Clinical Research (Programme Hospitalier de Recherche Clinique, Ministry of Health). The authors declare no conflicts of interest.