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.
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.
J Allergy Clin Immunol. 2017 Apr;139(4):1275-1281.e7. doi: 10.1016/j.jaci.2016.08.027. Epub 2016 Sep 30.
Most children with primary immunodeficiencies (PIDs) now reach adulthood. However, few studies have evaluated their health status and health-related quality of life (HRQoL).
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.
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.
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.
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.
大多数原发性免疫缺陷病（PID）患儿现已成年。然而，鲜有研究评估他们的健康状况和健康相关生活质量（HRQoL）。
为了调查长期发病率，法国 PID 参考中心启动了一项前瞻性多中心队列研究：法国儿童期免疫缺陷长期队列研究。收集的数据用于评估成年患者的身体健康状况及其对生活质量的影响。
患者需完成健康状况问卷。为每个健康状况分配严重程度评分（1 级[轻度]至 4 级[危及生命]）。使用 36 项简短健康状况调查（SF-36）HRQoL 问卷将患者的 HRQoL 与年龄和性别匹配的法国正常值进行比较。
在 329 名参与者中，评估时的平均年龄为 27.6 岁，诊断后平均随访 21 年；43%的患者至少有 1 种 4 级健康状况，86%的患者至少有 1 种 3 级（严重）或 4 级健康状况。25 名（7.6%）患者曾接受过癌症治疗。与法国正常值相比，PID 成人在所有 HRQoL 领域的得分明显较低。HRQoL 与健康状况负担密切相关。与 4 级或 3-4 级健康状况的关联在所有身体和精神领域均高度显著。
在儿童期诊断出 PID 的成年人经历了严重的健康状况负担，这影响了他们的 HRQoL。我们的研究结果强调需要密切监测这一弱势群体。
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