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自我注射透明质酸酶辅助皮下免疫球蛋白治疗复杂原发性抗体缺陷症

Self-administered hyaluronidase-facilitated subcutaneous immunoglobulin therapy in complicated primary antibody deficiencies.

作者信息

Danieli Maria Giovanna, Pulvirenti Federica, Rocchi Valeria, Morariu Ramona, Quinti Isabella

机构信息

Clinica Medica, Dipartimento di Scienze Cliniche e Molecolari, Università Politecnica delle Marche & Ospedali Riuniti, Ancona, Italy.

Department of Molecular Medicine, Sapienza Medical University, Rome, Italy.

出版信息

Immunotherapy. 2016 Sep;8(9):995-1002. doi: 10.2217/imt-2016-0035.

Abstract

Hyaluronidase-facilitated subcutaneous immunoglobulin (fSCIg) is a new immunoglobulin product for replacement therapy in patients with primary antibody deficiencies (PAD). The pre-administration of recombinant human hyaluronidase associated with 10% immunoglobulin allowed the infusion of larger (up to 600 ml) amounts of immunoglobulin at a single infusion site, enabling patients to receive the necessary treatment in a single monthly dose. Here, we report the effectiveness and the tolerability of fSCIg in patients with severe PAD-related comorbidities: refractory autoimmune thrombocytopenia; systemic granulomatous disease; severe enteropathy, and Type I diabetes. We conclude that fSCIg could be a feasible option to improve the adherence to replacement therapy also by patients with severe PAD.

摘要

透明质酸酶辅助皮下注射免疫球蛋白(fSCIg)是一种用于原发性抗体缺陷(PAD)患者替代治疗的新型免疫球蛋白产品。与10%免疫球蛋白联合使用的重组人透明质酸酶预处理,可在单一输注部位输注更大剂量(高达600毫升)的免疫球蛋白,使患者能够每月单次接受必要的治疗。在此,我们报告了fSCIg在患有严重PAD相关合并症(难治性自身免疫性血小板减少症、系统性肉芽肿病、严重肠病和I型糖尿病)患者中的有效性和耐受性。我们得出结论,fSCIg可能是一种可行的选择,可提高重度PAD患者对替代治疗的依从性。

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