| Autor: |
Revel-Vilk S; Gaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.; Faculty of Medicine, The Hebrew University of Jerusalem, Ein Kerem, Jerusalem 9112102, Israel., Mansfield R; Pfizer R&D UK Ltd., Sandwich, Kent CT13 9NJ, UK., Feder-Krengel N; Pfizer, Inc., New York, NY 10001, USA., Machtiger-Azoulay N; Pfizer, Inc., New York, NY 10001, USA., Kuter D; Hematology Division, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA., Szer J; Clinical Haematology at Peter MacCallum Cancer Centre, The Royal Melbourne Hospital, Melbourne 3050, Australia., Rosenbaum H; Hematology Day Care and Gaucher Clinic, The Center of Consulted Medicine, Clalit Services, Nazareth 1603701, Israel., Ferreira DC; Internal Medicine Department, Federal University of Santa Catarina, Florianópolis 88040-900, Brazil., Ruhrman-Shahar N; Raphael Recanati Genetic Institute, Rabin Medical Center, Beilinson Hospital, Petach Tikva 4941492, Israel., Wajnrajch M; Pfizer, Inc., New York, NY 10001, USA.; Department of Pediatrics, Grossman School of Medicine, New York University, New York, NY 10016, USA., Zimran A; Gaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.; Faculty of Medicine, The Hebrew University of Jerusalem, Ein Kerem, Jerusalem 9112102, Israel. |
| Abstrakt: |
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. |
