Evidence map›Paper›PMID 40140214›Full record

Observational studyJournal of clinical immunology2025

Multi-Year Registry Study of Elapegademase Treatment in Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Requiring Enzyme Replacement Therapy.

Morna J Dorsey, Manish J Butte, Jay A Lieberman, Heather Lehman, Tracy Fausnight, Michael D Keller, Caroline Fradette, Michael S Hershfield, Tamara C Pozos, Anna Rozova and 10 more

Abstract readMulticenter StudyObservational Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study in Journal of clinical immunology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

20 authors.

Morna J DorseyUniversity of California San Francisco Medical School, San Francisco, CA, USA. Morna.Dorsey@ucsf.edu.ORCID https://orcid.org/0000-0002-9432-3542
Manish J ButteUniversity of California Los Angeles, Los Angeles, CA, USA.ORCID https://orcid.org/0000-0002-4490-5595
Jay A LiebermanUniversity of Tennessee Health Science Center, Memphis, TN, USA.ORCID https://orcid.org/0000-0002-1374-8929
Heather LehmanUniversity of Buffalo Jacobs School of Medicine and Biomedical Sciences, Buffalo, NY, USA.ORCID https://orcid.org/0000-0002-7553-9751
Tracy FausnightPenn State Health Hershey Medical Center, Hershey, PA, USA.ORCID https://orcid.org/0000-0002-0251-2486
Michael D KellerChildren's National Hospital, Washington, DC, USA.ORCID https://orcid.org/0000-0001-8323-3085
Caroline FradetteChiesi Global Rare Diseases, Toronto, ON, Canada.ORCID https://orcid.org/0009-0009-8004-0304
Michael S HershfieldDuke University School of Medicine, Durham, NC, USA.ORCID https://orcid.org/0000-0003-3589-8554
Tamara C PozosChildren's Minnesota, Minneapolis, MN, USA.ORCID https://orcid.org/0000-0003-0763-8763
Anna RozovaChiesi Global Rare Diseases, Toronto, ON, Canada.ORCID https://orcid.org/0009-0007-9128-3710
Luke A WallLouisiana State University Health Sciences Center and Children's Hospital New Orleans, New Orleans, LA, USA.ORCID https://orcid.org/0000-0001-9125-8530
Jeffrey J BednarskiWashington University School of Medicine, St. Louis, MO, USA.ORCID https://orcid.org/0000-0001-6902-0876
Teresa K TarrantDuke University School of Medicine, Durham, NC, USA.ORCID https://orcid.org/0000-0003-4067-5363
Hey J ChongUniversity of Pittsburgh School of Medicine, Pittsburgh, PA, USA.ORCID https://orcid.org/0000-0002-6938-9267
Bob GengSchool of Medicine, University of California San Diego, San Diego, CA, USA.ORCID https://orcid.org/0000-0002-4618-4445
Noemi Toiber TeminChiesi Global Rare Diseases, Toronto, ON, Canada.ORCID https://orcid.org/0009-0000-4347-9988
Susan S LaubachSchool of Medicine, University of California San Diego, San Diego, CA, USA.ORCID https://orcid.org/0000-0002-7470-9616
Leo LinChiesi Global Rare Diseases, Toronto, ON, Canada.ORCID https://orcid.org/0009-0000-2957-4567
Talal MousallemDuke University School of Medicine, Durham, NC, USA.ORCID https://orcid.org/0000-0002-3692-8930
Jolan E WalterUniversity of South Florida, Tampa, FL, USA.ORCID https://orcid.org/0000-0002-6192-0703

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purposeThe safety and tolerability of elapegademase (elapegademase-lvlr; Revcovi

methodsPatients were managed by routine clinical care and treating physicians' judgement from September 2019 to January 2023. Primary endpoints included trough plasma ADA activity and total trough erythrocyte deoxyadenosine nucleotides (dAXP). Secondary outcomes included lymphocyte counts, hospitalizations, infections, and safety outcomes.

resultsThirty-two patients were grouped as ERT-naïve (n = 7; infants and children with no prior ERT [EN]); pegademase-transitioning (n = 21; from pegademase to elapegademase [PT]); and patients who had participated in the Phase 3 clinical trial (n = 4; STP-2279-002; [STP]). The EN group maintained optimal plasma ADA activity, increased lymphocyte counts, had manageable infections, and had no mortality for up to 30 months while on elapegademase. The STP group and 66.7% of the PT group continued to maintain satisfactory levels of both ADA and dAXP with stable rates of infections and hospitalizations and stable lymphocyte counts for up to 48.6 months. Variability on all measures was seen, but overall, patients did not deteriorate while on elapegademase.

conclusionEffectiveness of elapegademase was maintained up to 4 years of use and with no new safety concerns.

Indexed as

Adenosine DeaminaseAgammaglobulinemiaEnzyme Replacement TherapySevere Combined ImmunodeficiencyAdolescentChildChild, PreschoolFemaleHumansInfantMaleRegistriesTreatment OutcomeAdenosine Deaminasepegademase bovineADA-SCIDElapegademaseEnzyme ReplacementPrimary ImmunodeficiencyReal-worldSafety

Identifiers

PMID40140214
PMCPMC11947034

What OpenQuestion holds

Textmetadata
LicenceCC BY
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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.