Evidence map›Paper›PMID 28632502›Full record

ReviewCurrent opinion in hematology2017

Targeting activated protein C to treat hemophilia.

Stéphanie G I Polderdijk, Trevor P Baglin, James A Huntington

Open access · hybridAbstract readReview
In one paragraph

Review in Current opinion in hematology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers.

0numbers the graph read from it
0cells of the map it votes in
29citing papers in PubMed
2.7field-weighted citation impact, top 9% 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

29 citing papers in PubMed, 52 citations in OpenAlex.

  1. Trial
  2. Article
  3. Review
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  5. Review
  6. Article
  7. Article
  8. Article
  9. Article
  10. Molecular therapy. Nucleic acids · 2023
    Article
  11. Article
  12. Review
  13. Thrombin generation and implications for hemophilia therapies: A narrative review.Research and practice in thrombosis and haemostasis · 2023
    Review
  14. Management of haemophilia A with inhibitors: A regional cross-talk.Haemophilia : the official journal of the World Federation of Hemophilia · 2022
    Review
  15. Article
  16. Article
  17. Article
  18. Article
  19. Natural anticoagulants: A missing link in mild to moderate bleeding tendencies.Haemophilia : the official journal of the World Federation of Hemophilia · 2021
    Review
  20. 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

3 authors at 2 institutions in 1 country.

Stéphanie G I PolderdijkaDepartment of Haematology, University of Cambridge, Cambridge Institute for Medical Research bDepartment of Haematology, Addenbrooke's Hospital, Cambridge University Hospitals NHS Trust, Cambridge, UK.
Trevor P Baglin
James A Huntington
University of Cambridge · GBCambridge University Hospitals NHS Foundation Trust · GB

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purpose of reviewHemophilia is a debilitating disease, marked by frequent, painful bleeding events, joint deterioration and early death. All current treatments consist of i.v. infusions of replacement factor or other procoagulant factors, and are incompletely effective, due in part to the short half-lives of the proteins. An alternative approach is to rebalance hemostasis by inhibiting natural anticoagulant mechanisms. In this article, we explain why activated protein C (APC) is an appropriate and safe target for the treatment of hemophilia. RECENT

findingsA serpin (serine protease inhibitor) was engineered to specifically inhibit APC and was found to rescue hemostasis in a hemophilia mouse model, even after a severe tail clip injury. However, APC is also anti-inflammatory and has cytoprotective activities, raising safety concerns over the use of an APC inhibitor to treat hemophilia. We summarize the molecular basis of the anticoagulant and signaling activities of APC to assess the potential impact of targeting APC. SUMMARY: We conclude that the signaling and anticoagulant functions of APC are in spatially and kinetically distinct compartments, and that it is possible to specifically inhibit the anticoagulant activity of APC. Targeting APC with a serpin is remarkably effective and may be safe for long-term prophylactic use in the treatment of hemophilia.

Indexed as

AnimalsDisease Models, AnimalDrug Delivery SystemsHemophilia AHumansMiceProtein CSerpinsProtein CSerpins

Identifiers

PMID28632502
PMCPMC5548501
OpenAlexW2656022649

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.