Centre Hospitalier Régional Universitaire de Lille, Hôpital Jeanne de Flandre, Lille, France.
Paediatric Rheumatology Department, Centre Hospitalier Régional Universitaire de Lille, France.
Clin Exp Rheumatol. 2023 Oct;41(10):2039-2043. doi: 10.55563/clinexprheumatol/bfyb6o. Epub 2023 Mar 9.
Cryopyrin-associated periodic syndromes (CAPS) belongs to the group of hereditary recurrent fever disorders characterised by interleukin1β-mediated systemic inflammation. Specific treatment by IL-1 targeting drugs has significantly modified the disease evolution. We aimed to evaluate the socio-professional impact of CAPS in the long term and the influence of genetic variants in the phenotype.
We made a multicentre, observational and descriptive study and collected retrospective data from childhood to adulthood, and until the last year of follow-up. We assessed the quality of life (QoL) of the patients by phone interviews. We also used the SF36 questionnaire including 8 domains: physical function, physical role, body pain, general health, vitality, social function, emotional role and mental health. A high score means a better QoL.
Fifty-four patients were evaluated (14 familial cold autoinflammatory syndrome, 27 Muckle-Wells syndrome, 7 chronic infantileneurological cutaneous and articular syndrome. The study showed improvement in symptoms in adulthood and good QoL in all domains apart from school (87%) and work (61%) absenteeism. The MWS group is intermediate in terms of symptoms but seems to describe a better QoL compared to the other groups. The genetic variant alone does not determine the expression of the disease.
Our study shows that CAPS patients have an improvement of symptoms in adulthood and a satisfactory QoL for most of them. Anti-IL1 treatment is the main factor linked to this improvement and therefore early initiation should be encouraged.
冷吡啉相关周期性综合征(CAPS)属于遗传性复发性发热疾病的一种,其特征为白细胞介素 1β介导的全身炎症。针对白细胞介素 1 的靶向药物治疗显著改变了疾病的演变过程。我们旨在评估 CAPS 的长期社会职业影响以及遗传变异对表型的影响。
我们进行了一项多中心、观察性和描述性研究,从儿童期到成年期收集回顾性数据,并随访至最后一年。我们通过电话访谈评估患者的生活质量(QoL)。我们还使用了 SF36 问卷,包括 8 个领域:身体功能、身体角色、身体疼痛、总体健康、活力、社会功能、情感角色和心理健康。高分表示 QoL 更好。
评估了 54 例患者(14 例家族性冷自身炎症综合征、27 例 Muckle-Wells 综合征、7 例慢性婴儿神经皮肤关节综合征)。研究显示,成年后症状改善,除了学校(87%)和工作(61%)缺勤外,所有领域的 QoL 均良好。MWS 组在症状方面处于中间水平,但与其他组相比,似乎描述了更好的 QoL。遗传变异本身并不能决定疾病的表达。
我们的研究表明,CAPS 患者在成年后症状改善,大多数患者的生活质量令人满意。抗白细胞介素 1 治疗是改善的主要因素,因此应鼓励早期开始治疗。