Evidence map›Paper›PMID 35365177›Full record

ArticleOrphanet journal of rare diseases2022

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 and 5 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Orphanet journal of rare diseases, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
1.3field-weighted citation impact, top 21% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

7 citing papers in PubMed, 11 citations in OpenAlex.

  1. Plant-Based Systems for Producing Therapeutic Proteins: Current Status and Future Prospects.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2026
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

15 authors at 5 institutions in 1 country.

Lina TitievskyPfizer, Inc., New York, NY, USA.
Tilman SchusterPfizer, Inc., New York, NY, USA.
Ronnie WangPfizer, Inc., New York, NY, USA.
Muhammad YounusPfizer Inc, 500 Arcola Road, Collegeville, PA, 19426, USA. Muhammad.Younus2@pfizer.com.ORCID 0000-0002-1103-296X
Andrew PalladinoPfizer, Inc., New York, NY, USA.
Kabir QuaziPfizer, Inc., New York, NY, USA.
Michael P WajnrajchPfizer, Inc., New York, NY, USA.
Betina HernandezPfizer, Inc., New York, NY, USA.
Pamela S BeckerUniversity of California, Irvine, Irvine, CA, USA.
Neal J WeinrebUniversity of Miami Miller School of Medicine, Miami, FL, USA.
Christina ChambersUniversity of California, San Diego, La Jolla, CA, USA.
Roy MansfieldPfizer, Inc., New York, NY, USA.
Louise TaylorPfizer, Inc., New York, NY, USA.
Li-Jung TsengPfizer, Inc., New York, NY, USA.
Paige KaplanThe Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Pfizer (United States) · USChildren's Hospital of Philadelphia · USUniversity of California San Diego · USUniversity of Miami · USUniversity of Washington · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundLimited 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).

methodsTaliglucerase 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.

resultsA 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.

conclusionsThe 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.

Indexed as

Gaucher DiseaseAdolescentChildEnzyme Replacement TherapyFemaleGlucosylceramidaseHumansMaleRegistriesGlucosylceramidasetaliglucerase alfaEffectivenessGaucher diseaseNon-interventional studySafetyTaliglucerase alfa

Identifiers

PMID35365177
PMCPMC8973565
OpenAlexW4220855934

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.