Evidence map›Paper›PMID 39842018›Full record

ArticleThe New England journal of medicine2025

Platelet-Targeted Gene Therapy for Hemophilia A with Inhibitor History.

Mary Eapen, Lynn M Malec, Myriam A Armant, Bryon D Johnson, Qizhen Shi, Huiqing Xu, Lily M Du, James H Jerkins, Lisa J Duffy, Frederic D Bushman and 7 more

Registry-linked trialAbstract readLetter
In one paragraph

Article in The New England journal of medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03818763 (Phase I Study Evaluating Safety and Feasibility of Hematopoietic Stem Cell Gene Transfer That Targets Factor VIII Delivery From Platelets for Patients With Hemophilia A), which is not on this map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing 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.

NCT03818763 phase1active not recruitingnot on this map

Phase I Study Evaluating Safety and Feasibility of Hematopoietic Stem Cell Gene Transfer That Targets Factor VIII Delivery From Platelets for Patients With Hemophilia A

TypeinterventionalSponsorMedical College of WisconsinRan2020 to 2033Enrolled5ConditionsHemophilia AArmsAuto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII
3 · Its place in the literature

Who cites it

10 citing papers in PubMed.

  1. Article
  2. Review
  3. Article
  4. Review
  5. Review
  6. Advancements in CRISPR-basedFrontiers in genome editing · 2026
    Review
  7. Review
  8. Gene therapy for hemophilia - From basic science to first approvals of "one-and-done" therapies.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  9. Article
  10. Research and practice in thrombosis and haemostasis · 2025
    Article
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

17 authors.

Mary EapenMedical College of Wisconsin, Milwaukee, WI meapen@mcw.edu.ORCID 0000-0001-6193-4243
Lynn M MalecMedical College of Wisconsin, Milwaukee, WI meapen@mcw.edu.
Myriam A ArmantBoston Children's Hospital, Boston, MA.
Bryon D JohnsonMedical College of Wisconsin, Milwaukee, WI.
Qizhen ShiVersiti Blood Research Institute, Milwaukee, WI.
Huiqing XuMedical College of Wisconsin, Milwaukee, WI.
Lily M DuMedical College of Wisconsin, Milwaukee, WI.
James H JerkinsMedical College of Wisconsin, Milwaukee, WI.
Lisa J DuffyIndiana University, Indianapolis, IN.
Frederic D BushmanUniversity of Pennsylvania, Philadelphia, PA.ORCID 0000-0003-4740-4056
Carole LeeUniversity of Pennsylvania, Philadelphia, PA.
Angelina PetrichenkoUniversity of Pennsylvania, Philadelphia, PA.
Peiman HemattiMedical College of Wisconsin, Milwaukee, WI.
Ruta BrazauskasMedical College of Wisconsin, Milwaukee, WI.
Shawn M JobeMichigan State University, East Lansing, MI.
Parameswaran N HariMedical College of Wisconsin, Milwaukee, WI.
David A WilcoxMedical College of Wisconsin, Milwaukee, WI.

Funding

A Phase I Clinical Trial Testing Feasibility of Hematopoietic Stem Cell Gene Therapy Using Platelet Factor VIII to Safely Improve Hemostasis for Severe Hemophilia A with Inhibitory AntibodiesR01HL142791 · NHLBI · MEDICAL COLLEGE OF WISCONSIN · PI EAPEN, MARY, WILCOX, DAVID ALLEN · 2019 to 2023
$8.2M
Disseminating Curative Biological Therapies for Rare Pediatric DiseasesU01TR001814 · NCATS · BOSTON CHILDREN'S HOSPITAL · PI CANCELAS, JOSE A., KOHN, DONALD B · 2016 to 2020
$6.7M
PURPOSE OF THE NGVB CONTRACT IS TO CONTINUE SUPPORTING GENE THERAPY RESEARCH.75N92019D00018 · OD · INDIANA UNIVERSITY INDIANAPOLIS · 2019 to 2023
$1.7M
NCATS NIH HHS U01 TR001814NCATS NIH HHS UO1TR0018414NHLBI NIH HHS 75N92019D00018NHLBI NIH HHS 75N92019D00018-0-759201900074-1NHLBI NIH HHS R01 HL142791NHLBI NIH HHS RO1HL142791
6 · The paper itself

Abstract

PubMed holds no abstract for this paper.

Identifiers

PMID39842018
PMCPMC11756741

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

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.