Evidence map›Paper›PMID 30061443›Full record

Observational studyBMJ open2018

Costs and effects of on-demand treatment of hereditary angioedema in Italy: a prospective cohort study of 167 patients.

Carlo Federici, Francesca Perego, Ludovica Borsoi, Valentina Crosta, Andrea Zanichelli, Antonio Gidaro, Rosanna Tarricone, Marco Cicardi

Open access · goldAbstract readObservational Study
In one paragraph

Observational study in BMJ open, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
0.6field-weighted citation impact, top 31% 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

5 citing papers in PubMed, 12 citations in OpenAlex.

  1. Article
  2. Article
  3. Review
  4. Review
  5. 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

8 authors at 2 institutions in 1 country.

Carlo Federici *Centre for Research on Health and Social Care Management (CeRGAS), SDA Bocconi University, Milan, Italy.ORCID 0000-0002-0309-4669
Francesca Perego *Department of Biomedical and Clinical Sciences, "Luigi Sacco" University Hospital, University of Milan, Milan, Italy.
Ludovica BorsoiCentre for Research on Health and Social Care Management (CeRGAS), SDA Bocconi University, Milan, Italy.
Valentina CrostaDepartment of Biomedical and Clinical Sciences, "Luigi Sacco" University Hospital, University of Milan, Milan, Italy.
Andrea ZanichelliDepartment of Biomedical and Clinical Sciences, "Luigi Sacco" University Hospital, University of Milan, Milan, Italy.
Antonio GidaroDepartment of Biomedical and Clinical Sciences, "Luigi Sacco" University Hospital, University of Milan, Milan, Italy.
Rosanna TarriconeGovernment, Health and Non Profit Division, SDA Bocconi University, Milan, Italy.
Marco CicardiDepartment of Biomedical and Clinical Sciences, "Luigi Sacco" University Hospital, University of Milan, Milan, Italy.
University of Milan · ITBocconi University · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectivesTo explore treatment behaviours in a cohort of Italian patients with hereditary angioedema due to complement C1-inhibitor deficiency (C1-INH-HAE), and to estimate how effects and costs of treating attacks in routine practice differed across available on-demand treatments.

designCost analyses and survival analyses using attack-level data collected prospectively for 1 year.

settingNational reference centre for C1-INH-HAE.

participants167 patients with proved diagnosis of C1-INH-HAE, who reported data on angioedema attacks, including severity, localisation and duration, treatment received, and use of other healthcare services.

interventionsAttacks were treated with either icatibant, plasma-derived C1-INH (pdC1-INH) or just supportive care.

main outcome measuresTreatment efficacy in reducing attack duration and the direct costs of acute attacks.

resultsOverall, 133 of 167 patients (79.6%) reported 1508 attacks during the study period, with mean incidence of 11 attacks per patient per year. Only 78.9% of attacks were treated in contrast to current guidelines. Both icatibant and pdC1-INH significantly reduced attack duration compared with no treatment (median times from onset 7, 10 and 47 hours, respectively), but remission rates with icatibant were 31% faster compared with pdC1-INH (HR 1.31, 95% CI 1.14 to 1.51). However, observed treatment behaviours suggest patterns of suboptimal dosing for pdC1-INH. The average cost per attack was €1183 (SD €789) resulting in €1.58 million healthcare costs during the observation period (€11 912 per patient per year). Icatibant was 54% more expensive than pdC1-INH, whereas age, sex and prophylactic treatment were not associated to higher or lower costs.

conclusionsBoth icatibant and pdC1-INH significantly reduced attack duration compared with no treatment, however, icatibant was more effective but also more expensive. Treatment behaviours and suboptimal dosing of pdC1-INH may account for the differences, but further research is needed to define their role.

Indexed as

AdultAngioedemas, HereditaryBradykininComplement C1 Inhibitor ProteinCost-Benefit AnalysisFemaleHumansImmunologic FactorsItalyMaleMiddle AgedPlasmaProspective StudiesTreatment OutcomeBradykininComplement C1 Inhibitor ProteinicatibantImmunologic Factorscost-analysishealth economicshereditary angioedemaicatibantPDC1-INH

Identifiers

PMID30061443
PMCPMC6067408
OpenAlexW2886851188

What OpenQuestion holds

Textmetadata
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.