Division of Allergy and Immunology, Department of Internal Medicine, University of South Florida College of Medicine, Tampa, FL, USA.
Department of Pharmacology and Molecular Therapeutics, Uniformed Services University of the Health Sciences, 4301 Jones Bridge Road, C-2013, Bethesda, MD, 20814, USA.
J Clin Immunol. 2024 Jan 17;44(2):48. doi: 10.1007/s10875-023-01636-y.
The caspase activation and recruitment domain 11 (CARD11) gene encodes a scaffold protein required for lymphocyte antigen receptor signaling. Dominant-negative, loss-of-function (LOF) pathogenic variants in CARD11 result in CARD11-associated atopy with dominant interference of NF-κB signaling (CADINS) disease. Patients with CADINS suffer with severe atopic manifestations including atopic dermatitis, food allergy, and chronic spontaneous urticaria in addition to recurrent infections and autoimmunity. We assessed the response of dupilumab in five patients and omalizumab in one patient with CADINS for the treatment of severe atopic symptoms. CARD11 mutations were validated for pathogenicity using a T cell transfection assay to assess the impact on activation-induced signaling to NF-κB. Three children and three adults with dominant-negative CARD11 LOF mutations were included. All developed atopic disease in infancy or early childhood. In five patients, atopic dermatitis was severe and recalcitrant to standard topical and systemic medications; one adult suffered from chronic spontaneous urticaria. Subcutaneous dupilumab was initiated to treat atopic dermatitis and omalizumab to treat chronic spontaneous urticaria. All six patients had rapid and sustained improvement in atopic symptoms with no complications during the follow-up period. Previous medications used to treat atopy were able to be decreased or discontinued. In conclusion, treatment with dupilumab and omalizumab for severe, refractory atopic disease in patients with CADINS appears to be effective and well tolerated in patients with CADINS with severe atopy.
Caspase 激活和募集结构域 11(CARD11)基因编码一种支架蛋白,是淋巴细胞抗原受体信号所必需的。CARD11 的显性负、失功能(LOF)致病性变异导致 CARD11 相关变应性,NF-κB 信号呈显性干扰(CADINS)疾病。CADINS 患者表现为严重的变应性表现,包括特应性皮炎、食物过敏和慢性自发性荨麻疹,此外还伴有反复感染和自身免疫。我们评估了 dupilumab 在五名 CADINS 患者和 omalizumab 在一名 CADINS 患者中治疗严重特应性症状的反应。使用 T 细胞转染测定来评估对 NF-κB 激活诱导信号的影响,验证 CARD11 突变的致病性。纳入了三名儿童和三名成年人,他们均存在 CARD11 LOF 突变的显性失活。所有患者均在婴儿期或幼儿期发生特应性疾病。在五名患者中,特应性皮炎严重且对标准局部和全身药物治疗有抗药性;一名成人患有慢性自发性荨麻疹。开始皮下注射 dupilumab 治疗特应性皮炎,皮下注射 omalizumab 治疗慢性自发性荨麻疹。在随访期间,所有六名患者的特应性症状迅速且持续改善,没有并发症。以前用于治疗特应性的药物可以减少或停用。总之,CADINS 患者严重、难治性特应性疾病用 dupilumab 和 omalizumab 治疗似乎有效且耐受良好。