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Determinants of successful immune tolerance induction in persons with hemophilia A – systematic review and meta-analysis

1Department of Pediatric Hematology, Emma Children’s Hospital, Amsterdam University Medical Centers, location AMC, University of Amsterdam, Amsterdam, the Netherlands; Department of Molecular Hematology, Sanquin Research and Landsteiner Laboratory, Amsterdam, The Netherlands, Amsterdam, Noord-Holland, Netherlands, 2Faculty of Medicine, Universidade Federal de Minas Gerais, Belo Horizonte, Brazil; Department of Clinical Epidemiology, Leiden University Medical Center, Leiden University, Leiden, The Netherlands, Belo Horizonte, Minas Gerais, Brazil, 3Department of Internal Medicine, Faculty of Medicine, Universidade Federal de Minas Gerais, Belo Horizonte, Brazil, Belo Horizonte, Minas Gerais, Brazil, 4Department of Molecular Hematology, Sanquin Research and Landsteiner Laboratory, Amsterdam, The Netherlands, Amsterdam, Noord-Holland, Netherlands, 5IRCCS Humanitas Research Hospital, Milan, Lombardia, Italy, 6University Clinic Bonn, Bonn, Nordrhein-Westfalen, Germany, 7Division of Hematology/Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada, 8McMaster University Medical Centre, Hamilton, Ontario, Canada, 9Department of Pathology and Molecular Medicine, Queen’s University, Kingston, Ontario, Canada, 10University Medical Center Utrecht, University Utrecht, Utrecht, The Netherlands, Utrecht, Utrecht, Netherlands, 11Department of Pediatric Hematology, Emma Children’s Hospital, Amsterdam University Medical Centers, location AMC, University of Amsterdam, Amsterdam, the Netherlands; Department of Clinical Epidemiology, Leiden University Medical Center, Leiden University, Leiden, the Netherlands, Amsterdam, Noord-Holland, Netherlands

Abstract Number: PB1146

Meeting: ISTH 2022 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical

Background: Immune tolerance induction (ITI) is the only therapy to eradicate anti-factor VIII (FVIII) antibodies (inhibitors) in persons with hemophilia A (PwHA). However, this burdensome treatment fails in 10-40% of patients. In order to predict the ITI outcome is it important to identify predictors of ITI success.

Aims: To summarize the current evidence on factors associated with ITI outcome in PwHA.

Methods: On February 15 2021, a comprehensive literature search was conducted, which was updated on December 29 2021. Randomized controlled trials (RCT), cohort -, or case-control studies reporting on predictors for ITI outcome in PwHA were eligible for inclusion. ITI success was defined according to the methods of the investigators of the original studies. Data were extracted on study characteristics, outcome definitions, and predictors of ITI outcome, using a data collection form. Methodological quality was assessed by using an adapted Joanna Briggs Institute checklist for cohort studies. Studies were rated as high quality if ≥11/13 criteria were met.

Results: The literature searches yielded 1,079 unique papers of which 27 studies were included. We included 1 (4%) RCT and 26 (96%) cohort studies, involving a total of 1,734 PwHA. Six studies were rated as high quality (22%). ITI success was predominantly defined as negative inhibitor titer ( < 0.6 BU/mL), FVIII recovery ≥66% of expected, and FVIII half-life ≥6 hours (16/27; (59.3%)). Historical peak titer < 100 BU/mL, pre-ITI titer < 10 BU/mL, and peak titer during ITI < 100 BU/mL were associated with ITI success with pooled odds ratios of 1.50 (95% CI, 1.19-1.89), 1.68 (95% CI, 1.33-2.13), and 2.43 (95% CI, 1.58-3.74), respectively.

Conclusion(s): This systematic review and meta-analysis summarized and analyzed all available evidence of single variables that influence ITI outcome. However, the effects of multiple variables on ITI outcome were not investigated in this review.

To cite this abstract in AMA style:

Oomen I, Camelo R, Rezende S, Voorberg J, Mancuso M, Oldenburg J, Carcao M, Matino D, Lillicrap D, Fischer K, Fijnvandraat K, Gouw S. Determinants of successful immune tolerance induction in persons with hemophilia A – systematic review and meta-analysis [abstract]. https://abstracts.isth.org/abstract/determinants-of-successful-immune-tolerance-induction-in-persons-with-hemophilia-a-systematic-review-and-meta-analysis/. Accessed August 16, 2022.

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