Real-World Experiences with Taliglucerase Alfa Home Infusions for Patients with Gaucher Disease: A Global Cohort Study

Revel-Vilk, S; Mansfield, R; Feder-Krengel, N; Machtiger-Azoulay, N; Kuter, D; Szer, J; Rosenbaum, H; Ferreira, DC; Ruhrman-Shahar, N; Wajnrajch, M; Zimran, A

Author(s): Revel-Vilk, S; Mansfield, R; Feder-Krengel, N; Machtiger-Azoulay, N; Kuter, D; Szer, J; Rosenbaum, H; Ferreira, DC; Ruhrman-Shahar, N; Wajnrajch, M; Zimran, A;
Details: Publication Year 2023-09-12,Volume 12,Issue #18,Page 5913
Journal Title: Journal of Clinical Medicine
Publication Type: Research article
Abstract: 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.
Publisher: MDPI
Keywords: Gaucher disease; adverse event; enzyme replacement therapy; home infusion; infusion-related adverse event; safety
Department(s): Clinical Haematology
PubMed ID: 37762854
Publisher's Version: https://doi.org/10.3390/jcm12185913
Open Access at Publisher's Site: https://doi.org/10.3390/jcm12185913
Terms of Use/Rights Notice: Refer to copyright notice on published article.

Creation Date: 2023-11-21 05:54:28

Last Modified: 2023-11-21 05:55:40