Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS)

Lina Titievsky; Tilman Schuster; Ronnie Wang; Muhammad Younus; Andrew Palladino; Kabir Quazi; Michael P Wajnrajch; Betina Hernandez; Pamela S Becker; Neal J Weinreb; Christina Chambers; Roy Mansfield; Louise Taylor; Li-Jung Tseng; Paige Kaplan

doi:10.1186/s13023-022-02289-7

Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS)

Orphanet J Rare Dis. 2022 Apr 1;17(1):145. doi: 10.1186/s13023-022-02289-7.

Authors

Lina Titievsky¹, Tilman Schuster¹, Ronnie Wang¹, Muhammad Younus², Andrew Palladino¹, Kabir Quazi¹, Michael P Wajnrajch^{1

3}, Betina Hernandez¹, Pamela S Becker^{4

5}, Neal J Weinreb⁶, Christina Chambers⁷, Roy Mansfield¹, Louise Taylor¹, Li-Jung Tseng¹, Paige Kaplan⁸

Affiliations

¹ Pfizer, Inc., New York, NY, USA.
² Pfizer Inc, 500 Arcola Road, Collegeville, PA, 19426, USA. Muhammad.Younus2@pfizer.com.
³ New York University Grossman School of Medicine, New York, NY, USA.
⁴ University of California, Irvine, Irvine, CA, USA.
⁵ University of Washington School of Medicine, Seattle, WA, USA.
⁶ University of Miami Miller School of Medicine, Miami, FL, USA.
⁷ University of California, San Diego, La Jolla, CA, USA.
⁸ The Children's Hospital of Philadelphia, Philadelphia, PA, USA.

Abstract

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-naïve patients, likely reflecting the therapeutic effects of prior treatments. During follow-up, the hemoglobin concentration and platelet counts increased in the treatment-naïve patients and remained relatively constant or increased slightly in patients with prior ERT. Spleen and liver volumes decreased in treatment-naïve 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.

Keywords: Effectiveness; Gaucher disease; Non-interventional study; Safety; Taliglucerase alfa.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Adolescent
Child
Enzyme Replacement Therapy / adverse effects
Female
Gaucher Disease* / drug therapy
Glucosylceramidase / adverse effects
Humans
Male
Registries

Substances

Glucosylceramidase
taliglucerase alfa