Shire US Inc., a Takeda company, 650 E Kendall St, Cambridge, MA, 02142, USA.
Baxalta US Inc., a Takeda company, Cambridge, MA, USA.
BMC Immunol. 2020 May 4;21(1):24. doi: 10.1186/s12865-020-00346-z.
Often, patients with primary immunodeficiency diseases (PID), which are marked by the absence or loss of functional antibodies, require lifelong treatment with immunoglobulin (IG) replacement therapy administered either intravenously (intravenous immunoglobulin [IVIG]) or subcutaneously (subcutaneous immunoglobulin [SCIG]). In patients with PID, the 20% SCIG product, Ig20Gly, was shown to be efficacious and well tolerated in 2 phase 2/3 trials conducted in North America and Europe. This analysis evaluated patient satisfaction with Ig20Gly therapy and treatment preferences.
This prespecified post hoc analysis showed combined data from 2 Ig20Gly pivotal trials. Treatment satisfaction was assessed in the pre-Ig20Gly period and after ≥11 months of Ig20Gly treatment using the Life Quality Index (LQI; both studies) and the Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9; North American study only). Treatment preference was assessed using a survey at the end of the European study. Median within-patient differences in LQI and TSQM-9 scores between the pre-Ig20Gly period and the end of the Ig20Gly treatment period were assessed using the Wilcoxon signed-rank test.
A total of 113 patients (n = 68 [North American]; n = 45 [Europe]) with PID were included in the analysis. In the combined LQI analysis (n = 110), significant improvements were observed in the treatment interference (median ∆: + 2.8; P = 0.006) and therapy setting (median ∆: + 5.6; P < 0.0001) domains, and in the item-level scores for convenience (median ∆: + 1.0; P < 0.0001) and interference with work/school (median ∆: + 1.0; P = 0.0001) categories. In the subgroup analyses, significant improvements in the treatment interference and therapy setting domains and the convenience and interference with work/school items were observed for those who had previously received treatment outside the home, those who had previously received IVIG, and those in the North American study. Significant improvements were observed in the TSQM-9 treatment convenience domain (median ∆: + 11.1; P < 0.0001) and selected item-level scores in the North American study. In the European study, most (88.9%) patients preferred to continue Ig20Gly versus other IG treatments.
After ≥11 months of taking Ig20Gly, patients reported high levels of treatment satisfaction, convenience, and preference for Ig20Gly, with consistent results across studies and use of multiple patient-reported outcome measures.
原发性免疫缺陷病（PID）患者常因功能性抗体缺失或丧失而需要终身接受免疫球蛋白（IG）替代治疗，治疗方式包括静脉内（静脉注射免疫球蛋白 [IVIG]）或皮下（皮下免疫球蛋白 [SCIG]）注射。在 PID 患者中，两项在北美和欧洲开展的 2 期/3 期临床试验显示，20%的 SCIG 产品 Ig20Gly 具有疗效且可良好耐受。本分析评估了 Ig20Gly 治疗的患者满意度和治疗偏好。
本事后分析是对两项 Ig20Gly 关键性试验的联合数据进行分析。采用生活质量指数（LQI；两项研究）和药物治疗满意度问卷-9（TSQM-9；仅北美研究），在 Ig20Gly 治疗前和治疗后≥11 个月评估患者对 Ig20Gly 治疗的满意度。在欧洲研究结束时采用一项调查评估治疗偏好。采用 Wilcoxon 符号秩检验评估 LQI 和 TSQM-9 评分在 Ig20Gly 治疗前和治疗结束时的患者内中位数差异。
共纳入 113 例 PID 患者（北美 68 例[68 例]；欧洲 45 例[45 例]）进行分析。在联合 LQI 分析（n=110）中，治疗干扰（中位数差值：+2.8；P=0.006）和治疗设定（中位数差值：+5.6；P<0.0001）以及便利性（中位数差值：+1.0；P<0.0001）和工作/学校干扰（中位数差值：+1.0；P=0.0001）项目评分均观察到显著改善。亚组分析显示，对于那些曾在家外接受治疗、曾接受 IVIG 治疗以及在北美研究中的患者，治疗干扰和治疗设定领域以及便利性和工作/学校干扰项目的改善具有统计学意义。在北美研究中，TSQM-9 治疗便利性领域（中位数差值：+11.1；P<0.0001）和选定项目评分观察到显著改善。在欧洲研究中，大多数（88.9%）患者更愿意继续使用 Ig20Gly 而非其他 IG 治疗。
在接受 Ig20Gly 治疗≥11 个月后，患者报告了较高水平的治疗满意度、便利性和对 Ig20Gly 的偏好，各项研究和使用多种患者报告结局测量指标的结果一致。
