Evidence map›Paper›PMID 24338563›Full record

ArticleJournal of clinical immunology2014

Modeling primary immunodeficiency disease epidemiology and its treatment to estimate latent therapeutic demand for immunoglobulin.

Jeffrey S Stonebraker, Albert Farrugia, Benjamin Gathmann, ESID Registry Working Party, Jordan S Orange

Abstract read
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In one paragraph

Article in Journal of clinical immunology, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
2.2field-weighted citation impact, top 13% 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

10 citing papers in PubMed, 28 citations in OpenAlex.

  1. Review
  2. Observational
  3. Review
  4. Common Variable Immunodeficiency and Liver Involvement.Clinical reviews in allergy & immunology · 2018
    Review
  5. Article
  6. Review
  7. The Ethics of Paid Plasma Donation: A Plea for Patient Centeredness.HEC forum : an interdisciplinary journal on hospitals' ethical and legal issues · 2015
    Article
  8. Errors and Omissions: Donor Compensation Policies and Richard Titmuss.HEC forum : an interdisciplinary journal on hospitals' ethical and legal issues · 2015
    Article
  9. Article
  10. 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

5 authors at 4 institutions in 3 countries.

Jeffrey S StonebrakerDepartment of Business Management, Poole College of Management, North Carolina State University, Raleigh, NC, 27695, USA, jeff_stonebraker@ncsu.edu.
Albert Farrugia
Benjamin Gathmann
ESID Registry Working Party
Jordan S Orange
North Carolina State University · USBaylor College of Medicine · USThe University of Western Australia · AUUniversity of Freiburg · DE

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purposeEstimating the underlying demand for immunoglobulin (Ig) is important to ensure that adequate provision is made for patients with primary immune deficiency (PID) in the context of the competing demands for Ig and to ensure optimal therapeutic regimens. The concept of latent therapeutic demand (LTD) was used to estimate evidence-based requirements and compared to the actual Ig consumption in different countries. The estimates were performed for common variable immunodeficiency (CVID) and X-linked Agammaglobulinaemia (XLA), the two most commonly studied PIDs using Ig.

methodsThe LTD model for CVID and XLA was derived using decision analysis methodology. Data for the epidemiology and treatment variables were obtained from peer-reviewed publications, clinical registries and publicly-available patient surveys. Incomplete data records from registries were excluded from analysis. The variables impacting LTD were ranked in order of sensitivity through a tornado diagram. The uncertainty surrounding the variables was modeled using probabilistic distributions and evaluated using Monte Carlo simulation.

resultsTreatment dosage and prevalence were determined to be the most sensitive variables driving demand. The average potential usage of Ig for the treatment of CVID and XLA was estimated at 72 g per 1,000 population, which is higher than the estimated Ig usage in CVID and XLA of 27-41 g per 1,000 population in the US.

conclusionThe potential demand for treating CVID and XLA exceeds the currently observed usage of Ig in these disorders. Variable usage in different countries is due to varying prevalence and dosage practices. Under-reporting in patient registries represents a major obstacle to calculating the true prevalence of CVID and XLA. Modeling demand relies heavily upon accurate prevalence and practice estimates which reemphasize the importance of accurate registries and improved registry methods. As better data becomes available, revision of model variables provides opportunities to anticipate and prepare for evolving patient needs.

Indexed as

Models, StatisticalAdolescentAdultAgammaglobulinemiaAgedAged, 80 and overChildChild, PreschoolCommon Variable ImmunodeficiencyFemaleGenetic Diseases, X-LinkedHumansImmunoglobulinsImmunologic Deficiency SyndromesMaleMiddle AgedImmunoglobulins

Identifiers

PMID24338563
OpenAlexW2002051798

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.