Evidence map›Paper›PMID 39558913›Full record

ArticleResearch and practice in thrombosis and haemostasis2024

Prediction of the chance of successful immune tolerance induction in persons with severe hemophilia A and inhibitors: a clinical prediction model.

Ilja Oomen, Amal Abdi, Ricardo M Camelo, Fábia M R A Callado, Luany E M Carvalho, Ilenia L Calcaterra, Manuel Carcao, Giancarlo Castaman, Jeroen C J Eikenboom, Kathelijn Fischer and 18 more

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Article in Research and practice in thrombosis and haemostasis, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from it

What it found

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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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

28 authors.

Ilja OomenDepartment of Pediatric Hematology, Amsterdam University Medical Centers location University of Amsterdam, Amsterdam, the Netherlands.
Amal AbdiDepartment of Pediatric Hematology, Amsterdam University Medical Centers location University of Amsterdam, Amsterdam, the Netherlands.
Ricardo M CameloDepartment of Internal Medicine, Faculty of Medicine, Universidade Federal de Minas Gerais, Belo Horizonte, Brazil.
Fábia M R A CalladoFundação de Hematologia e Hemoterapia de Pernambuco, Recife, Brazil.
Luany E M CarvalhoCentro de Hematologia e Hemoterapia do Ceará, Fortaleza, Brazil.
Ilenia L CalcaterraDepartment of Clinical Medicine and Surgery, Federico II University, Naples, Italy.
Manuel CarcaoDepartment of Pediatrics, Division of Hematology and Oncology, Hospital for Sick Children, Toronto, Ontario, Canada.
Giancarlo CastamanDepartment of Oncology, Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy.
Jeroen C J EikenboomDepartment of Internal Medicine, Division of Thrombosis and Hemostasis, Leiden University Medical Center, Leiden, the Netherlands.
Kathelijn FischerDepartment of Hematology, Center for Benign Hematology, Thrombosis and Hemostasis, Van Creveldkliniek, University Medical Center Utrecht, Utrecht, the Netherlands.
Vivian K B FrancoCentro de Hematologia e Hemoterapia de Santa Catarina, Florianópolis, Brazil.
Martijn W HeymansDepartment of Epidemiology and Biostatistics, Amsterdam University Medical Centers, Vrije Universiteit University, Amsterdam, the Netherlands.
Frank W G LeebeekDepartment of Hematology, Erasmus University Medical Center, Rotterdam, the Netherlands.
David LillicrapDepartment of Pathology and Molecular Medicine, Queen's University, Kingston, Ontario, Canada.
Cláudia S LorenzatoCoagulopathy Clinic, Hemocentro do Paraná, Curitiba, Brazil.
Maria Elisa MancusoDepartment of Hematology, Center for Thrombosis and Hemorrhagic Diseases, Instituto di Ricovero e Cura a Carattere Scientifico Humanitas Research Hospital, Rozzano, Milan, Italy.
Davide MatinoDepartment of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Ontario, Canada.
Matteo N D Di MinnoDepartment of Clinical Medicine and Surgery, Federico II University, Naples, Italy.
Alex B MohsenyDepartment of Pediatrics, Leiden University Medical Center, Leiden, the Netherlands.
Johannes OldenburgInstitute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn, Germany.
Suely Meireles RezendeDepartment of Internal Medicine, Faculty of Medicine, Universidade Federal de Minas Gerais, Belo Horizonte, Brazil.
Georges-Etienne RivardMolecular Diagnostic Laboratory, Centre Hospitalier Universitaire Sainte-Justine, Montréal, Québec, Canada.
Natalia RydzDepartment of Hematology and Hematologic Malignancies, Foothills Medical Center, Calgary, Alberta, Canada.
Saskia E M ScholsDepartment of Hematology, Radboud University Medical Center, Nijmegen, the Netherlands.
Jan VoorbergDepartment of Molecular Hematology, Sanquin Research, Amsterdam, the Netherlands.
Karin FijnvandraatDepartment of Pediatric Hematology, Amsterdam University Medical Centers location University of Amsterdam, Amsterdam, the Netherlands.
Samantha C GouwDepartment of Pediatric Hematology, Amsterdam University Medical Centers location University of Amsterdam, Amsterdam, the Netherlands.
International Genetic and clinical determinants for the outcome of immune tolerance induction study group

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Inhibitor eradication to restore factor (F)VIII efficacy is the treatment goal for persons with severe hemophilia A (HA) and inhibitors. Immune tolerance induction (ITI) is demanding and successful in about 70% of people. Until now, it has remained difficult to quantify the probability of ITI success or failure, complicating the decision to initiate or not initiate ITI. Estimating the individual chance of ITI success allows clinicians, patients, and their families to support shared decision-making. Objectives: We aimed to identify clinical predictors of ITI success and to develop a clinical prediction model to estimate the chance of successful ITI in persons with severe HA. Methods: This multicenter study included persons with severe HA who received ITI. Clinical data were collected. Successful ITI was defined by a negative inhibitor titer and an adequate response to FVIII concentrates. A multivariable logistic regression model was developed. Model performance and internal validation were performed. Results: Of 206 participants with a median age of 19.8 months (IQR, 12.1-38.8) at ITI start, 148 (71.8%) achieved ITI success. Our clinical prediction model included 4 predictors of ITI success: cumulative number of FVIII exposure days at inhibitor development, peak inhibitor titer, ethnicity, and Conclusion: In our study, including 206 people with severe HA and inhibitors, we developed a clinical prediction model to estimate the chance of successful ITI. After future external validation, this clinical prediction model may be useful for informing clinicians and families.

Indexed as

factor VIIIhemophilia Aimmune toleranceprobabilitytreatment outcome

Identifiers

PMID39558913
PMCPMC11570954

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