PMID- 37762854 OWN - NLM STAT- PubMed-not-MEDLINE LR - 20230930 IS - 2077-0383 (Print) IS - 2077-0383 (Electronic) IS - 2077-0383 (Linking) VI - 12 IP - 18 DP - 2023 Sep 12 TI - Real-World Experiences with Taliglucerase Alfa Home Infusions for Patients with Gaucher Disease: A Global Cohort Study. LID - 10.3390/jcm12185913 [doi] LID - 5913 AB - 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. FAU - Revel-Vilk, Shoshana AU - Revel-Vilk S AUID- ORCID: 0000-0001-9151-0337 AD - Gaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel. AD - Faculty of Medicine, The Hebrew University of Jerusalem, Ein Kerem, Jerusalem 9112102, Israel. FAU - Mansfield, Royston AU - Mansfield R AD - Pfizer R&D UK Ltd., Sandwich, Kent CT13 9NJ, UK. FAU - Feder-Krengel, Neta AU - Feder-Krengel N AD - Pfizer, Inc., New York, NY 10001, USA. FAU - Machtiger-Azoulay, Noya AU - Machtiger-Azoulay N AD - Pfizer, Inc., New York, NY 10001, USA. FAU - Kuter, David AU - Kuter D AD - Hematology Division, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA. FAU - Szer, Jeff AU - Szer J AUID- ORCID: 0000-0001-6783-2301 AD - Clinical Haematology at Peter MacCallum Cancer Centre, The Royal Melbourne Hospital, Melbourne 3050, Australia. FAU - Rosenbaum, Hanna AU - Rosenbaum H AD - Hematology Day Care and Gaucher Clinic, The Center of Consulted Medicine, Clalit Services, Nazareth 1603701, Israel. FAU - Ferreira, David Cavalcanti AU - Ferreira DC AD - Internal Medicine Department, Federal University of Santa Catarina, Florianopolis 88040-900, Brazil. FAU - Ruhrman-Shahar, Noa AU - Ruhrman-Shahar N AUID- ORCID: 0009-0009-8725-2596 AD - Raphael Recanati Genetic Institute, Rabin Medical Center, Beilinson Hospital, Petach Tikva 4941492, Israel. FAU - Wajnrajch, Michael AU - Wajnrajch M AUID- ORCID: 0000-0002-8211-9237 AD - Pfizer, Inc., New York, NY 10001, USA. AD - Department of Pediatrics, Grossman School of Medicine, New York University, New York, NY 10016, USA. FAU - Zimran, Ari AU - Zimran A AUID- ORCID: 0000-0003-0077-8608 AD - Gaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel. AD - Faculty of Medicine, The Hebrew University of Jerusalem, Ein Kerem, Jerusalem 9112102, Israel. LA - eng PT - Journal Article DEP - 20230912 PL - Switzerland TA - J Clin Med JT - Journal of clinical medicine JID - 101606588 PMC - PMC10531841 OTO - NOTNLM OT - Gaucher disease OT - adverse event OT - enzyme replacement therapy OT - home infusion OT - infusion-related adverse event OT - safety COIS- The SZMC Gaucher Unit receives support from Sanofi/Genzyme for participation in the ICGG Registry, Takeda for the GOS Registry, and Pfizer for TALIAS. The Unit also receives research grants from Takeda, Pfizer, Sanofi/Genzyme, and Centogene/Germany. S.R.-V. reported receiving grant/research support, honoraria, and advisory fees from Takeda, Pfizer, and Sanofi/Genzyme. R.M., N.F-K., N.M-A. and M.W. are Pfizer employees. D.K. reported receiving research support from Alnylam, Biocryst, Novartis, Rigel, Sanofi (Principia), Takeda (Bioverativ), UCB, being a consultant for AIRx, Alexion (Syntimmune), Alnylam, Alpine, Amgen, Argenx, BioCryst, Bristol Myers Squibb (BMS), Caremark, Cellularity, Cellphire, Chugai, CRICO, Daiichi Sankyo, Dianthus, Electra Therapeutics, Fuji, Hemopure, Hengrui, Immunovant, Incyte, Inmagenebio, Kezar, Kyowa-Kirin, Merck Sharp Dohme, Momenta, Novartis, Nuvig, Pfizer, Platelet Biogenesis, Platelet Disorder Support Association, Protagonist, Rigel, Sanofi (Bioveratif), Sanofi (Principia), Sanofi (Genzyme), Sobi (Dova), Takeda, UCB, Up-To-Date, and Zafren, and owning stock at Rubius. J.S. reported receiving honoraria and advisory fees from Takeda, Sanofi/Genzyme, Alexion, Eli-Lilly, Sobi, Regeneron, BioCryst, and Novartis. H.R. reported receiving research grants from Genzyme and Pfizer, and travel grants and honoraria from Genzyme-Sanofi and Pfizer. D.C.F. declared no conflicts of interest. N.R-S. reported receiving honoraria and advisory fees from Sanofi, Takeda, and Pfizer. A.Z. reported receiving honoraria from Takeda, Pfizer, and Bio-Events, being a consultant for Takeda, and having ownership at Agyany Pharma Ltd. EDAT- 2023/09/28 06:42 MHDA- 2023/09/28 06:43 PMCR- 2023/09/12 CRDT- 2023/09/28 01:22 PHST- 2023/08/02 00:00 [received] PHST- 2023/09/04 00:00 [revised] PHST- 2023/09/06 00:00 [accepted] PHST- 2023/09/28 06:43 [medline] PHST- 2023/09/28 06:42 [pubmed] PHST- 2023/09/28 01:22 [entrez] PHST- 2023/09/12 00:00 [pmc-release] AID - jcm12185913 [pii] AID - jcm-12-05913 [pii] AID - 10.3390/jcm12185913 [doi] PST - epublish SO - J Clin Med. 2023 Sep 12;12(18):5913. doi: 10.3390/jcm12185913.