PMID- 35365177 OWN - NLM STAT- MEDLINE DCOM- 20220405 LR - 20220518 IS - 1750-1172 (Electronic) IS - 1750-1172 (Linking) VI - 17 IP - 1 DP - 2022 Apr 1 TI - Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS). PG - 145 LID - 10.1186/s13023-022-02289-7 [doi] LID - 145 AB - BACKGROUND: Limited real-world data from routine clinical care are available on the safety and effectiveness of treatment with taliglucerase alfa in patients with Gaucher disease (GD). METHODS: Taliglucerase Alfa Surveillance (TALIAS), a multinational prospective Drug Registry of patients with GD, was established to evaluate the long-term safety (primary objective) and effectiveness (secondary objective) of taliglucerase alfa. We present an interim analysis of the data from the Drug Registry collected over the 5-year period from September 2013 to January 2019. RESULTS: A total of 106 patients with GD (15.1% children aged < 18 years; 53.8% females) treated with taliglucerase alfa have been enrolled in the Drug Registry, as of January 7, 2019. The median duration of follow-up was 795 days with quartiles (Q1, Q3) of 567 and 994 days. Fifty-three patients (50.0%) were from Israel, 28 (26.4%) were from the United States, and 25 (23.6%) were from Albania. At the time of enrollment, most patients (87.7%) had received prior enzyme replacement therapy (ERT). Thirty-nine of the 106 patients had treatment-emergent adverse events (AEs). Twelve of the 106 patients experienced serious AEs; two patients experienced four treatment-related serious AEs. Four patients died, although none of the deaths was considered to be related to taliglucerase alfa treatment by the treating physicians. Nine patients discontinued from the study, including the four who died. At baseline, patients with prior ERT had a higher mean hemoglobin concentration and platelet counts than treatment-naive patients, likely reflecting the therapeutic effects of prior treatments. During follow-up, the hemoglobin concentration and platelet counts increased in the treatment-naive patients and remained relatively constant or increased slightly in patients with prior ERT. Spleen and liver volumes decreased in treatment-naive patients. CONCLUSIONS: The interim data showed no new or emergent safety signals. The overall interim data are consistent with the clinical program experience and known safety and effectiveness profile of taliglucerase alfa. CI - (c) 2022. The Author(s). FAU - Titievsky, Lina AU - Titievsky L AD - Pfizer, Inc., New York, NY, USA. FAU - Schuster, Tilman AU - Schuster T AD - Pfizer, Inc., New York, NY, USA. FAU - Wang, Ronnie AU - Wang R AD - Pfizer, Inc., New York, NY, USA. FAU - Younus, Muhammad AU - Younus M AUID- ORCID: 0000-0002-1103-296X AD - Pfizer Inc, 500 Arcola Road, Collegeville, PA, 19426, USA. Muhammad.Younus2@pfizer.com. FAU - Palladino, Andrew AU - Palladino A AD - Pfizer, Inc., New York, NY, USA. FAU - Quazi, Kabir AU - Quazi K AD - Pfizer, Inc., New York, NY, USA. FAU - Wajnrajch, Michael P AU - Wajnrajch MP AD - Pfizer, Inc., New York, NY, USA. AD - New York University Grossman School of Medicine, New York, NY, USA. FAU - Hernandez, Betina AU - Hernandez B AD - Pfizer, Inc., New York, NY, USA. FAU - Becker, Pamela S AU - Becker PS AD - University of California, Irvine, Irvine, CA, USA. AD - University of Washington School of Medicine, Seattle, WA, USA. FAU - Weinreb, Neal J AU - Weinreb NJ AD - University of Miami Miller School of Medicine, Miami, FL, USA. FAU - Chambers, Christina AU - Chambers C AD - University of California, San Diego, La Jolla, CA, USA. FAU - Mansfield, Roy AU - Mansfield R AD - Pfizer, Inc., New York, NY, USA. FAU - Taylor, Louise AU - Taylor L AD - Pfizer, Inc., New York, NY, USA. FAU - Tseng, Li-Jung AU - Tseng LJ AD - Pfizer, Inc., New York, NY, USA. FAU - Kaplan, Paige AU - Kaplan P AD - The Children's Hospital of Philadelphia, Philadelphia, PA, USA. LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20220401 PL - England TA - Orphanet J Rare Dis JT - Orphanet journal of rare diseases JID - 101266602 RN - EC 3.2.1.45 (Glucosylceramidase) RN - EC 3.2.1.45 (taliglucerase alfa) SB - IM MH - Adolescent MH - Child MH - Enzyme Replacement Therapy/adverse effects MH - Female MH - *Gaucher Disease/drug therapy MH - Glucosylceramidase/adverse effects MH - Humans MH - Male MH - Registries PMC - PMC8973565 OTO - NOTNLM OT - Effectiveness OT - Gaucher disease OT - Non-interventional study OT - Safety OT - Taliglucerase alfa COIS- LT is a former employee of Pfizer. TS, RW, MY, AP, KQ, MPW, BH, RM, LT, and L-JT are employees of and own stock in Pfizer. NW, CC, and PK served as consultants on the Scientific Steering Committee for this study and received grant/research support from Pfizer. PSB's institution received grant/research support from Pfizer for her participation on the Scientific Steering Committee for this study. EDAT- 2022/04/03 06:00 MHDA- 2022/04/06 06:00 PMCR- 2022/04/01 CRDT- 2022/04/02 05:22 PHST- 2021/12/20 00:00 [received] PHST- 2022/03/14 00:00 [accepted] PHST- 2022/04/02 05:22 [entrez] PHST- 2022/04/03 06:00 [pubmed] PHST- 2022/04/06 06:00 [medline] PHST- 2022/04/01 00:00 [pmc-release] AID - 10.1186/s13023-022-02289-7 [pii] AID - 2289 [pii] AID - 10.1186/s13023-022-02289-7 [doi] PST - epublish SO - Orphanet J Rare Dis. 2022 Apr 1;17(1):145. doi: 10.1186/s13023-022-02289-7.