Evidence map›Paper›PMID 33012060›Full record

ArticleHaemophilia : the official journal of the World Federation of Hemophilia2020

Real-world data demonstrate improved bleed control and extended dosing intervals for patients with haemophilia B after switching to recombinant factor IX Fc fusion protein (rFIXFc) for up to 5 years.

Amy Shapiro, Ateefa Chaudhury, Michael Wang, Miguel Escobar, Elisa Tsao, Christopher Barnowski, Jing Feng, Nisha Jain, Doris V Quon

Open access · hybridAbstract readMulticenter Study
In one paragraph

Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.

0numbers the graph read from it
0cells of the map it votes in
12citing papers in PubMed
1.4field-weighted citation impact, top 16% 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

12 citing papers in PubMed, 19 citations in OpenAlex.

  1. Extended Half-Life Factor Concentrates in Haemophilia Treatment.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2026
    Review
  2. Article
  3. Article
  4. Article
  5. Article
  6. Article
  7. Article
  8. Review
  9. Observational
  10. [Advances in long-acting recombinant factor Ⅸ for the treatment of hemophilia B].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2022
    Article
  11. Article
  12. 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

9 authors at 4 institutions in 1 country.

Amy ShapiroIndiana Hemophilia & Thrombosis Center, Inc., Indianapolis, IN, USA.ORCID https://orcid.org/0000-0003-2821-7159
Ateefa ChaudhuryCenter for Inherited Blood Disorders, Orange, CA, USA.
Michael WangHemophilia and Thrombosis Center, University of Colorado, Aurora, CO, USA.ORCID https://orcid.org/0000-0001-9289-4862
Miguel EscobarMcGovern Medical School at The University of Texas Health Science Center at Houston, Houston, TX, USA.ORCID https://orcid.org/0000-0002-2944-0240
Elisa TsaoSanofi, Waltham, MA, USA.
Christopher BarnowskiSanofi, Waltham, MA, USA.
Jing FengSanofi, Waltham, MA, USA.
Nisha JainSanofi, Cambridge, MA, USA.
Doris V QuonOrthopedic Hemophilia Treatment Center, Los Angeles, CA, USA.
Sanofi (United States) · USCenter for Inherited Blood Disorders · USIndiana Hemophilia and Thrombosis Center · USThe University of Texas Health Science Center at Houston · US

Funding

Sanofi
6 · The paper itself

Abstract

introductionIn clinical trials, recombinant factor IX fusion protein (rFIXFc) has demonstrated safety, efficacy and prolonged activity with extended dosing intervals for treatment of haemophilia B.

aimTo assess the real-world clinical utility of rFIXFc in a variable patient population and routine clinical practice.

methodsA multicentre, retrospective chart review was conducted of patients with haemophilia B who had received rFIXFc prophylaxis or on-demand treatment for ≥6 months across six sites in the United States.

resultsSixty-four eligible patients were identified who had a median (range) duration on rFIXFc of 2.7 (0.5-5.0) years. Of 32 patients on rFIXFc prophylaxis who switched from prophylaxis with another factor treatment (ie pre-rFIXFc) and had a known pre-rFIXFc dosing interval, the initial dosing interval was lengthened for 26 (81%) patients and maintained for the remaining 6 (19%) patients. Most (n = 48 [91%]) patients who received rFIXFc prophylaxis from the beginning to the end of the chart review period (n = 53) maintained or lengthened the dosing interval from first through last dose of rFIXFc. For patients receiving rFIXFc prophylaxis, there was an approximate 50% reduction in weekly factor consumption compared with pre-rFIXFc prophylaxis. Overall annualized bleed rates, annualized spontaneous bleed rates and annualized joint bleed rates decreased after switching to rFIXFc prophylaxis (n = 24 with bleed data). Compliance to recommended treatment improved or remained stable in most patients with available data (30/31).

conclusionRecombinant factor IX fusion protein prophylaxis improved bleed control, reduced overall consumption, reduced frequency of infusion and improved compliance for patients with haemophilia B in a real-world setting.

Indexed as

AdolescentAdultAgedChildChild, PreschoolFactor IXFemaleHemophilia BHemorrhageHumansImmunoglobulin Fc FragmentsMaleMiddle AgedRecombinant Fusion ProteinsRetrospective StudiesTime FactorsFactor IXfactor IX Fc fusion proteinImmunoglobulin Fc FragmentsRecombinant Fusion Proteinsextended half-life factorfactor IX switchinghaemophilia Bprolonged factor IX activityrFIXFc

Identifiers

PMID33012060
PMCPMC7821220
OpenAlexW3089829826

What OpenQuestion holds

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LicenceCC BY-NC
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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.