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特发性炎性肌病患者的皮下免疫球蛋白治疗:真实世界、单中心经验。

Subcutaneous immunoglobulin for patients with idiopathic inflammatory myopathies: a real-world, single-centre experience.

机构信息

Divsion of Rheumatology, Department of Medicine, University of Alberta, Edmonton, AB, Canada.

Division of Dermatology, Department of Medicine, University of Alberta, Edmonton, AB, Canada.

出版信息

Rheumatology (Oxford). 2024 Aug 1;63(8):2118-2122. doi: 10.1093/rheumatology/kead521.

Abstract

OBJECTIVES

Idiopathic inflammatory myopathies (IIMs) are heterogeneous diseases characterized by skeletal muscle inflammation associated with cutaneous, pulmonary and/or other visceral organ involvement. IVIG has been recommended as an adjunct therapy for IIM patients refractory to conventional therapy. However, IVIG has high resource needs and increased risk of adverse reactions. Subcutaneous immunoglobulin (SCIG) therapy has been used as an alternative to IVIG in primary immunodeficiencies and neuroinflammatory disorders. We assessed the satisfaction, patient preference and effectiveness in IIM patients who transitioned from IVIG to SCIG.

METHODS

We retrospectively reviewed consecutive 20 patients with IIM who were transitioned from IVIG to SCIG therapy for >12 months. Patient preference between IVIG and SCIG was surveyed using a questionnaire previously used in studies of neuroinflammatory conditions. In addition, disease flares, changes in immunosuppression, cumulative prednisone doses and global disease activity were evaluated using the Myositis Intention to Treat Index (MITAX) 12 months pre- and post-SCIG initiation.

RESULTS

Most patients (78.9%) preferred SCIG over IVIG and preferred home-based therapies to hospital-based therapies. There was no significant difference in global disease activity (MITAX 3.31 vs 3.02) or in cumulative steroid doses 12 months pre- or post-SCIG initiation. Three patients experienced disease flares, five escalated in immunosuppression, while four patients deescalated in immunosuppressive medications.

CONCLUSIONS

SCIG is preferred by most patients over IVIG without a substantial increased disease activity or need for additional CS. Future cost-effectiveness studies may provide an additional rationale for utilizing SCIG over IVIG for maintenance therapy for IIM.

摘要

目的

特发性炎性肌病(IIM)是一组异质性疾病,其特征为骨骼肌炎症伴有皮肤、肺部和/或其他内脏器官受累。对于常规治疗无效的 IIM 患者,已推荐使用 IVIG 作为辅助治疗。然而,IVIG 需要大量资源,且不良反应风险增加。皮下免疫球蛋白(SCIG)治疗已在原发性免疫缺陷和神经炎症性疾病中替代 IVIG。我们评估了从 IVIG 转为 SCIG 治疗的 IIM 患者的满意度、患者偏好和疗效。

方法

我们回顾性分析了连续 20 例接受 IVIG 转为 SCIG 治疗且持续时间>12 个月的 IIM 患者。使用先前在神经炎症性疾病研究中使用的问卷评估患者对 IVIG 和 SCIG 的偏好。此外,通过肌炎意向治疗指数(MITAX)评估疾病活动度、免疫抑制药物的变化、累积泼尼松剂量和治疗前和治疗后 12 个月的总体疾病活动度。

结果

大多数患者(78.9%)更喜欢 SCIG 而非 IVIG,且更喜欢家庭治疗而非医院治疗。治疗后 12 个月,总体疾病活动度(MITAX 3.31 与 3.02)或累积激素剂量无显著差异。3 例患者出现疾病活动度增加,5 例患者免疫抑制药物升级,4 例患者免疫抑制药物降级。

结论

与 IVIG 相比,SCIG 更受大多数患者的青睐,且不会显著增加疾病活动度或需要额外的 CS。未来的成本效益研究可能为将 SCIG 用于 IIM 的维持治疗替代 IVIG 提供额外的依据。

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