Evidence map›Paper›PMID 31294906›Full record

SynthesisHaemophilia : the official journal of the World Federation of Hemophilia2019

Burden of mild haemophilia A: Systematic literature review.

Flora Peyvandi, Fatemeh Tavakkoli, Diana Frame, Jennifer Quinn, Benjamin Kim, Adebayo Lawal, Mimi C Lee, Wing Y Wong

Abstract readSystematic Review
In one paragraph

Synthesis in Haemophilia : the official journal of the World Federation of Hemophilia, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
17citing papers in PubMed, 1 pooled it
–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

17 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Burden of mild haemophilia A: Systematic literature review.Haemophilia : the official journal of the World Federation of Hemophilia · 2019
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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

8 authors.

Flora PeyvandiAngelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.ORCID https://orcid.org/0000-0001-7423-9864
Fatemeh TavakkoliBioMarin Pharmaceutical Inc., Novato, California.
Diana FrameFrame Research, LLC, Brooklyn, New York.
Jennifer QuinnBioMarin Pharmaceutical Inc., Novato, California.
Benjamin KimBioMarin Pharmaceutical Inc., Novato, California.
Adebayo LawalBioMarin Pharmaceutical Inc., Novato, California.
Mimi C LeeBioMarin Pharmaceutical Inc., Novato, California.
Wing Y WongBioMarin Pharmaceutical Inc., Novato, California.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionAlthough the clinical manifestations of severe haemophilia A (HA) are well studied, the challenges, if any, of living with mild HA are not clearly delineated to date.

aimTo assess available evidence of clinical risks and societal/economic impacts of disease in adult patients with mild HA using a systematic literature review.

methodsPrespecified study selection criteria were applied in a comprehensive literature search. Included studies varied in design and reported outcomes of interest for adults (≥13 years of age) with mild HA.

resultsSeventeen studies with a total of 3213 patients met eligibility criteria (published or presented in English, 1966-2017). Most studies were observational, and the outcomes reported were too sparse and dissimilar to support a formal meta-analysis. Mean annual bleeding rates ranged from 0.44 to 4.5 episodes per patient per year. Quality of life (QoL; SF-36 General Health) was impacted compared to healthy controls. Health care costs and productivity were seldom assessed and no robust comparisons to healthy controls were available.

conclusionQuantifying outcomes for adult patients with mild HA remains challenging, with estimates of key QoL and cost data often based on small data sets and without comparison to population norms. Therefore, the clinical impact of mild haemophilia may be under-represented and unmet needs may remain unaddressed. As paradigm-changing therapies for HA emerge, stronger knowledge of mild HA can guide the development of care options that minimize burden and enhance the QoL for this segment of the haemophilia community, and for the haemophilia community in totality.

Indexed as

Hemophilia AAdolescentAdultHumansYoung Adultacute bleedingdisease burdenhaemophiliaquality of life

Identifiers

PMID31294906
PMCPMC6852304

What OpenQuestion holds

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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.