Revel-Vilk Shoshana, Mansfield Royston, Feder-Krengel Neta, Machtiger-Azoulay Noya, Kuter David, Szer Jeff, Rosenbaum Hanna, Ferreira David Cavalcanti, Ruhrman-Shahar Noa, Wajnrajch Michael, Zimran Ari
Gaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.
Faculty of Medicine, The Hebrew University of Jerusalem, Ein Kerem, Jerusalem 9112102, Israel.
J Clin Med. 2023 Sep 12;12(18):5913. doi: 10.3390/jcm12185913.
Taliglucerase alfa is an enzyme replacement therapy approved for Gaucher disease. We assessed the duration/compliance/safety of such home infusions in commercial use in four countries where home infusion programs are available. The treatment duration/compliance study included 173 patients (Israel, 58; US, 61; Brazil, 48; Australia, 6) who received ≥1 taliglucerase alfa home infusion through 6/2021. The median age at home therapy initiation was 38 (range, 2-87) years; 58% were females. The median treatment duration (at home) was 2.7 (range, 0.04-9.0) years. The annual compliance rate was stable (≥95%) throughout the study period. A search of the Pfizer global safety database (through 6/2021), identified 19 adverse events (AEs) as related to "definite home use" and 14 to "possible home use" of taliglucerase alfa; 42.4% of these AEs were serious; none were fatal. Twelve serious AEs in five separate case reports were considered treatment related: one case of chest discomfort/pain and hypertension and one case of erythema associated with a toe blister, for which causality could not be excluded; pain in extremity; projectile vomiting and chills, alongside excessive eye blinking; and an infusion-related AE (pruritus). In conclusion, this real-life global study demonstrated that taliglucerase alfa home infusions are safe with high compliance rates.
塔利葡萄糖脑苷脂酶α是一种被批准用于治疗戈谢病的酶替代疗法。我们评估了在四个有家庭输液项目的国家中，这种用于商业用途的家庭输液的持续时间/依从性/安全性。治疗持续时间/依从性研究纳入了173例患者（以色列58例；美国61例；巴西48例；澳大利亚6例），这些患者在2021年6月前接受了≥1次塔利葡萄糖脑苷脂酶α家庭输液。在家开始治疗的中位年龄为38岁（范围2 - 87岁）；58%为女性。在家治疗的中位持续时间为2.7年（范围0.04 - 9.0年）。在整个研究期间，年依从率稳定（≥95%）。检索辉瑞全球安全数据库（截至2021年6月），确定了19例与塔利葡萄糖脑苷脂酶α“明确家庭使用”相关的不良事件（AE）和14例与“可能家庭使用”相关的不良事件；这些不良事件中有42.4%为严重不良事件；无一例死亡。五份独立病例报告中的12例严重不良事件被认为与治疗相关：1例胸部不适/疼痛和高血压，1例与脚趾水泡相关的红斑，因果关系无法排除；肢体疼痛；喷射性呕吐和寒战，伴有过度眨眼；以及1例输液相关不良事件（瘙痒）。总之，这项真实世界的全球研究表明，塔利葡萄糖脑苷脂酶α家庭输液安全且依从率高。
