背景:免疫球蛋白G替代疗法(IgRT),静脉内(IV)和皮下(SC)途径,是治疗原发性免疫缺陷(PID)的关键。近年来,促进皮下免疫球蛋白(fSCIG),rHuPH20和10%IgG的组合已成为一种结合IV和SC优点的递送方法。
方法:在观察性前瞻性队列中,我们调查了来自5个PID中心的PID患者使用fSCIG长达12个月的经历.我们使用患者/护理人员和医生报告的指标评估了这种治疗的有效性和安全性。此外,我们分析了患者治疗满意度(TSQM-9)和生活质量(QoL).
结果:我们招募了29名患者(22名儿科患者和7名成人患者;14名女性和15名男性患者;(中位数:15,最小-最大时间:2-40.9年),这些患者作为IgRT-naive(n=1),从常规快速推送10%SCIG(n=6)或IVIG(n=22)切换。在参与者中,19(65%)表现出抗体缺乏,8(27%)联合免疫缺陷,和2(7%)免疫失调。值得注意的是,在所有以前的IgRTs和fSCIG下都达到了靶向的谷值免疫球蛋白G水平.没有严重的全身药物不良反应记录,尽管存在普遍的局部(%86.45)和轻微的全身(%26.45)不良反应,但fSCIG仍观察到.由于轻微的全身症状,2例患者从fSCIG改为10%SCIG。患者满意度调查显示,与基线相比,2-4个月(p=0.102);5-8个月(p=0.006)和9-12个月(p<0.001)显着增加。在QoL调查中没有观察到显著的趋势。
结论:fSCIG在管理PID方面表现出可接受的耐受性和有效性,此外患者对IgRT的药物满意度也显著提高。尽管存在局部反应,但已确定的益处仍支持该疗法的继续。
BACKGROUND: Immunoglobulin G replacement therapy (IgRT), intravenous (IV) and subcutaneous (SC) routes, is pivotal in treatment of primary immunodeficiencies (PID). In recent years, facilitated subcutaneous immunoglobulin (fSCIG), a combination of rHuPH20 and 10% IgG has emerged as a delivery method to combine advantages of both IV and SC.
METHODS: In an observational prospective cohort, we investigated patient experience with fSCIG in PID patients from 5 PID centers for up to 12 months. We assessed the efficacy and safety of this treatment with patient/caregiver- and physician-reported indicators. Additionally, we analyzed patient treatment satisfaction (TSQM-9) and quality of life (QoL).
RESULTS: We enrolled 29 patients (22 pediatric and 7 adults; 14 females and 15 males; (median: 15, min-max: 2-40.9 years) who initiated fSCIG as IgRT-naive (n = 1), switched from conventional rapid-push 10% SCIG (n = 6) or IVIG (n = 22). Among the participants, 19 (65%) exhibited antibody deficiencies, 8 (27%) combined immunodeficiencies, and 2 (7%) immune dysregulations. Remarkably, targeted trough immunoglobulin G levels were achieved under all previous IgRTs as well as fSCIG. No severe systemic adverse drug reactions were documented, despite prevalent local (%86.45) and mild systemic (%26.45) adverse reactions were noted with fSCIG. Due to mild systemic symptoms, 2 patients switched from fSCIG to 10% SCIG. The patient satisfaction survey revealed a notable increase at 2-4th (p = 0.102); 5-8th (p = 0.006) and 9-12th (p < 0.001) months compared to the baseline. No significant trends were observed in QoL surveys.
CONCLUSIONS: fSCIG demonstrates admissable tolerability and efficacy in managing PIDs in addition to notable increase of patients\' drug satisfaction with IgRT. The identified benefits support the continuation of this therapy despite the local reactions.