Evidence map›Paper›PMID 21935333›Full record

ArticleClinicoEconomics and outcomes research : CEOR2011

Cost-utility analysis of prophylaxis versus treatment on demand in severe hemophilia A.

Giorgio L Colombo, Sergio Di Matteo, Maria Elisa Mancuso, Elena Santagostino

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in ClinicoEconomics and outcomes research : CEOR, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
17citing papers in PubMed, 3 pooled it
1.5field-weighted citation impact, top 18% 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

17 citing papers in PubMed, 3 syntheses or guidelines pooled it, 41 citations in OpenAlex.

  1. Pooled it
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  9. Reflections on the Importance of Cost of Illness Analysis in Rare Diseases: A Proposal.International journal of environmental research and public health · 2021
    Article
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  12. Review
  13. Treatment adherence in hemophilia.Patient preference and adherence · 2017
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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

4 authors at 1 institution in 1 country.

Giorgio L ColomboUniversity of Pavia, School of Pharmacy, Pavia, Italy;
Sergio Di Matteo
Maria Elisa Mancuso
Elena Santagostino
University of Pavia · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundIndividuals with severe hemophilia A have reduced blood levels of clotting factor VIII (FVIII) leading to recurrent bleeding into joints and muscles. Primary prophylaxis with clotting factor concentrates started early in childhood prevents joint bleeds, thus avoiding joint damage and improving people's quality of life. There remain significant differences in the implementation of primary prophylaxis worldwide mainly due to the cost of prophylaxis compared with treatment on demand.

objectiveTo evaluate the cost-effectiveness of primary prophylaxis with FVIII concentrates versus secondary prophylaxis, versus treatment on demand, and versus a "hybrid" (primary prophylaxis followed by on-demand treatment in adults) in individuals with severe hemophilia A.

methodsA Markov model was developed and run using different sources of clinical, cost, and utility data. The model was populated with a hypothetical cohort of 100 individuals with severe hemophilia A. The perspective of the Italian National Health System was used.

resultsThe baseline results showed that primary and secondary prophylaxis is cost-effective compared both with treatment on demand and with a hybrid strategy. The incremental costs per quality-adjusted life-year gained for individuals with hemophilia A receiving primary and secondary prophylaxis were €40,229 to €40,236 versus an on-demand strategy. However, the sensitivity analyses performed showed that the results were sensitive to the unit cost of clotting FVIII, bleeding frequency, and the discount rate.

conclusionAlthough primary prophylaxis is a costly treatment, our results show that it is cost-effective compared with treatment on demand.

Indexed as

cost-utilityfactor VIIIhemophiliaprophylaxisquality of lifetreatment on demand

Identifiers

PMID21935333
PMCPMC3169977
OpenAlexW2056900097

What OpenQuestion holds

Textmetadata
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.