Evidence map›Paper›PMID 36224704›Full record

ReviewHaemophilia : the official journal of the World Federation of Hemophilia2023

Prophylaxis use of clotting factor replacement products in people with non-severe haemophilia: A review of the literature.

Alfonso Iorio, Christoph Königs, Mark T Reding, Dawn Rotellini, Mark W Skinner, Maria Elisa Mancuso, Erik Berntorp

Open access · hybridAbstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Haemophilia : the official journal of the World Federation of Hemophilia, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
2.2field-weighted citation impact, top 10% 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

8 citing papers in PubMed, 14 citations in OpenAlex.

  1. Article
  2. Article
  3. Review
  4. Article
  5. The imperative to prevent joint bleeding in everyone living with hemophilia.Research and practice in thrombosis and haemostasis · 2024
    Article
  6. Review
  7. Article
  8. Review
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

7 authors at 7 institutions in 5 countries.

Alfonso IorioDepartment of Health Research Methods, Evidence and Impact, McMaster University, Hamilton, Ontario, Canada.ORCID https://orcid.org/0000-0002-3331-8766
Christoph KönigsClinical and Molecular Hemostasis, University Hospital Frankfurt, Goethe University, Department of Paediatrics, Frankfurt am Main, Germany.
Mark T RedingCenter for Bleeding and Clotting Disorders, University of Minnesota, Minneapolis, Minnesota, USA.ORCID https://orcid.org/0000-0003-3319-2806
Dawn RotelliniNational Hemophilia Foundation, New York, New York, USA.
Mark W SkinnerDepartment of Health Research Methods, Evidence and Impact, McMaster University, Hamilton, Ontario, Canada.ORCID https://orcid.org/0000-0002-0934-0680
Maria Elisa MancusoCenter for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital, Rozzano, Milan, Italy.
Erik BerntorpLund University, Skane University Hospital, Malmo, Sweden.ORCID https://orcid.org/0000-0002-1337-7195
American Association For The Advancement of Science · USCanadian Hemophilia Society · CAGoethe University Frankfurt · DEIRCCS Humanitas Research Hospital · ITLund University · SENational Hemophilia Foundation · USUniversity of Minnesota · US

Funding

Bayer, USA
6 · The paper itself

Abstract

introductionPeople with non-severe haemophilia appear to be under-treated in many countries, and this may lead to joint damage and worsen quality of life.

aimTo review literature for clotting factor replacement prophylaxis in people with non-severe haemophilia A and B (HA/HB) in relation to long-term outcomes to support clinical decision-making.

methodsA targeted literature search was performed to identify studies published between 2000 and 2021 that included prophylaxis in people with non-severe HA/HB and long-term outcomes, including annualized bleeding rates, joint health and quality of life.

resultsAlthough eligible articles included 2737 and 2272 people with mild or moderate HA, respectively, only 22% (n = 609) and 29% (n = 668) reported treatment regimens. A total of 549 people with moderate HA were treated with factor replacement prophylaxis and were from high-income countries. On the contrary, nearly all people with mild HA received desmopressin (n = 599). Details of treatment regimens for women with haemophilia and people with HB were sparse. Three studies provided long-term outcomes for people with moderate haemophilia who received prophylaxis with factor concentrate, supporting early prophylaxis in people with a frequent bleeding phenotype regardless of their endogenous clotting factor level to preserve joint health.

conclusionThere remain large knowledge gaps when considering how to provide optimal treatment for people with non-severe haemophilia. Nonetheless, there is a strong rationale that prophylaxis should be considered early in life according to similar strategies as for severe haemophilia for those with a frequent severe bleeding phenotype.

Indexed as

Hemophilia AHemophilia BBlood Coagulation FactorsFactor VIIIFemaleHemorrhageHumansQuality of LifeBlood Coagulation FactorsFactor VIIIarthropathybleedingepisodic/on-demand treatmentnon-severe (mild or moderate) haemophilia A or Bprophylactic FVIII or FIXwomen with haemophilia (symptomatic carriers)

Identifiers

PMID36224704
PMCPMC10091955
OpenAlexW4304775574

What OpenQuestion holds

Textmetadata
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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.