Evidence map›Paper›PMID 34220802›Full record

Trial reportFrontiers in immunology2021

Immune Tolerance-Adjusted Personalized Immunogenicity Prediction for Pompe Disease.

Anne S De Groot, Ankit K Desai, Sandra Lelias, S M Shahjahan Miah, Frances E Terry, Sundos Khan, Cindy Li, John S Yi, Matt Ardito, William D Martin and 1 more

Open access · goldAbstract readClinical Trial
In one paragraph

Trial report in Frontiers in immunology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
13citing papers in PubMed, 2 pooled it
1.9field-weighted citation impact, top 14% 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

13 citing papers in PubMed, 2 syntheses or guidelines pooled it, 19 citations in OpenAlex.

  1. Pooled it
  2. What's new and what's next for gene therapy in Pompe disease?Expert opinion on biological therapy · 2022
    Pooled it
  3. Review
  4. Article
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  7. Review
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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

11 authors at 3 institutions in 1 country.

Anne S De GrootEpiVax, Inc., Providence, RI, United States.
Ankit K DesaiDivision of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, United States.
Sandra LeliasEpiVax, Inc., Providence, RI, United States.
S M Shahjahan MiahEpiVax, Inc., Providence, RI, United States.
Frances E TerryEpiVax, Inc., Providence, RI, United States.
Sundos KhanEpiVax, Inc., Providence, RI, United States.
Cindy LiDivision of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, United States.
John S YiDepartment of Surgery, Duke University Medical Center, Durham, NC, United States.
Matt ArditoEpiVax, Inc., Providence, RI, United States.
William D MartinEpiVax, Inc., Providence, RI, United States.
Priya S KishnaniDivision of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, United States.
Duke Medical Center · USProvidence College · USEpiVax (United States) · US

Funding

GAA-ITEM: Personalizing the Prediction of Anti-therapeutic Antibody Response in Pompe Disease PatientsR43TR002441 · NCATS · EPIVAX, INC. · PI DEGROOT, ANNE SEARLS · 2018 to 2019
$325k
NCATS NIH HHS R43 TR002441
6 · The paper itself

Abstract

Infantile-onset Pompe disease (IOPD) is a glycogen storage disease caused by a deficiency of acid alpha-glucosidase (GAA). Treatment with recombinant human GAA (rhGAA, alglucosidase alfa) enzyme replacement therapy (ERT) significantly improves clinical outcomes; however, many IOPD children treated with rhGAA develop anti-drug antibodies (ADA) that render the therapy ineffective. Antibodies to rhGAA are driven by T cell responses to sequences in rhGAA that differ from the individuals' native

Indexed as

alpha-GlucosidasesComputational BiologyEnzyme Replacement TherapyEpitope MappingFemaleGlycogen Storage Disease Type IIHLA-DR AntigensHumansImmune ToleranceInfantMalePrecision MedicinePrognosisRegression AnalysisRiskT-Lymphocytes, Regulatoryalpha-GlucosidasesGAA protein, humanHLA-DR Antigensacid alpha-glucosidase (GAA)anti-drug antibodies (ADA)cross-reactive immunological material (CRIM)enzyme replacement therapy (ERT)immune tolerance induction (ITI)personalized immunogenicity assessment (PIMA)Pompe Disease (glycogen storage disease type II)Tregitope

Identifiers

PMID34220802
PMCPMC8242953
OpenAlexW3171080219

What Socratic 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 Socratic graph.