Vultaggio Alessandra, Azzari Chiara, Milito Cinzia, Finocchi Andrea, Toppino Claudia, Spadaro Giuseppe, Trizzino Antonino, Baldassarre Martire, Paganelli Roberto, Moschese Viviana, Soresina Annarosa, Matucci Andrea
Immunoallergology Unit, Department of Biomedicine, Policlinico di Careggi, Viale Morgagni, 85, 50134, Florence, Italy.
Clin Drug Investig. 2015 Mar;35(3):179-85. doi: 10.1007/s40261-015-0270-1.
Subcutaneous immunoglobulin (SCIG) therapy is becoming increasingly popular as self-administration is possible because intravenous access is unnecessary, and there is a lower frequency of systemic adverse events. The aim of this study was to evaluate the shifting from intravenous immunoglobulins (IVIGs) replacement therapy to SCIG in patients with primary immunodeficiency (PID) in a routine real-life situation.
In a multicenter prospective observational study, we enrolled 50 patients suffering from PID who were monitored for 24 months; 44 patients switched from IVIG and six from different SCIG preparations. The study preparation (human IgG 16 %, Vivaglobin(®), CSL Behring GmbH, Germany) was subcutaneously infused weekly (maximum volume 15 mL/site; maximum infusion rate 22 mL/h). The study endpoints were: annual rate of severe bacterial infections (SBIs), local adverse reactions, quality of life, days off school/work, and days of hospitalization.
Thirty-three of 39 (84.6 %) patients who completed the study experienced an infection or signs thereof. Only five SBIs were observed, corresponding to an annual rate of 0.056 episodes per patient in 44 subjects [intention-to-treat (ITT) population]. A significant decrease in both days of hospitalization (1.93 ± 4.08 vs. 0.64 ± 2.94) and days off school/work (15.27 ± 23.17 vs. 2.26 ± 4.45) was recorded at 24 months. Local reactions were observed in 14/50 (28 %) patients, mainly consisting of skin manifestations at the injection site. Only three (6.8 %) patients discontinued due to infusion site reactions. In patients shifting from IVIG to SCIG, the total mean score of Life Quality Index (LQI) improved from 76.9 ± 16.8 to 90.7 ± 11.6 (P < 0.01) at 6 months; there was an improvement also in the overall patients' evaluation.
A total of 93.2 % patients tolerated the new route of administration and reported a significant improvement in their LQI. Our results from a routine clinical practice in a real-life population are consistent with those of phase III clinical studies.
皮下注射免疫球蛋白（SCIG）疗法越来越受欢迎，因为无需静脉通路即可自行给药，且全身不良事件的发生率较低。本研究的目的是评估在常规实际生活情况下，原发性免疫缺陷（PID）患者从静脉注射免疫球蛋白（IVIG）替代疗法转换为SCIG疗法的情况。
在一项多中心前瞻性观察研究中，我们纳入了50例患有PID的患者，并对其进行了24个月的监测；44例患者从IVIG转换而来，6例患者从不同的SCIG制剂转换而来。研究制剂（16%人IgG，Vivaglobin(®)，德国CSL Behring GmbH公司）每周皮下注射一次（最大剂量15 mL/部位；最大输注速率22 mL/h）。研究终点包括：严重细菌感染（SBI）的年发生率、局部不良反应、生活质量、缺课/缺勤天数和住院天数。
39例完成研究的患者中有33例（84.6%）发生了感染或出现感染迹象。仅观察到5例SBI，在44例受试者中，每位患者的年发生率为0.056次[意向性分析（ITT）人群]。在24个月时，住院天数（1.93±4.08对0.64±2.94）和缺课/缺勤天数（15.27±23.17对2.26±4.45）均显著减少。14/50（28%）的患者出现了局部反应，主要表现为注射部位的皮肤表现。仅有3例（6.8%）患者因输注部位反应而停药。在从IVIG转换为SCIG的患者中，生活质量指数（LQI）的总平均分在6个月时从76.9±16.8提高到了90.7±11.6（P<0.01）；患者的总体评价也有所改善。
共有93.2%的患者耐受了新的给药途径，并报告其LQI有显著改善。我们在实际生活人群中进行的常规临床实践结果与III期临床研究结果一致。
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