Pedini Veronica, Savore Isabella, Danieli Maria Giovanna
Clinical Medicine, Department of Clinical and Molecular Sciences, Marche Polytechnic University, Riuniti Hospital.
Isr Med Assoc J. 2017 Jul;19(7):420-423.
Common variable immunodeficiency (CVID) is the most common symptomatic primary immune deficiency of adulthood. Besides recurrent infections, autoimmune disorders-mainly cytopenias-affect 30% of patients with CVID.
To describe the efficacy and safety of facilitated subcutaneous immunoglobulin (fSCIg), which is a combination of 10% [human] SCIg with recombinant human hyaluronidase for the treatment of CVID-linked cytopenias.
We describe four women (mean age 54 years) with CVID associated with idiopathic thrombocytopenic purpura (ITP) (n=3) and autoimmune hemolytic anemia (AIHA) (n=1). Diagnosis of CVID was made according to the European Society of Immune Deficiencies / Pan-American Group for Immune Deficiency criteria. All were treated with fSCIg (bi-monthly, 20 g).
After a median follow-up of 22 months, all patients achieved a stable remission from the cytopenias, characterized by increased platelet values in ITP (mean values 93000/mmc), and resolution of anemia. A reduction of the daily prednisone dose was documented in the patient with AIHA. No systemic adverse drug reactions were observed.
Our preliminary data documented the efficacy and safety of fSCIg in the treatment of CVID associated with autoimmune cytopenias, with a good tolerability. We also noted the role of fSCIg as a steroid sparing agent. It is thus possible to suppose an immunomodulatory role for fSCIg, but linked to different mechanisms than IVIg, due to the peculiar pharmacokinetic and administration route of fSCIg.
普通可变免疫缺陷(CVID)是成年期最常见的有症状的原发性免疫缺陷。除反复感染外,自身免疫性疾病(主要是血细胞减少症)影响30%的CVID患者。
描述即用型皮下免疫球蛋白(fSCIg)的疗效和安全性,fSCIg是10%[人]SCIg与重组人透明质酸酶的组合,用于治疗与CVID相关的血细胞减少症。
我们描述了4名女性(平均年龄54岁),她们患有与特发性血小板减少性紫癜(ITP)相关的CVID(n = 3)和自身免疫性溶血性贫血(AIHA)(n = 1)。根据欧洲免疫缺陷学会/泛美免疫缺陷小组标准诊断为CVID。所有患者均接受fSCIg治疗(每两个月一次,20 g)。
中位随访22个月后,所有患者的血细胞减少症均实现稳定缓解,其特征为ITP患者血小板值升高(平均值93000/mmc)以及贫血症状消失。AIHA患者的每日泼尼松剂量有所减少。未观察到全身性药物不良反应。
我们的初步数据证明了fSCIg治疗与自身免疫性血细胞减少症相关的CVID的疗效和安全性,且耐受性良好。我们还注意到fSCIg作为一种类固醇节约剂的作用。因此,可以推测fSCIg具有免疫调节作用,但由于其独特的药代动力学和给药途径,其作用机制与静脉注射免疫球蛋白(IVIg)不同。