Prediction of the chance of successful immune tolerance induction in persons with severe hemophilia A and inhibitors: a clinical prediction model.
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Oomen Ilja, Abdi Amal, Camelo Ricardo M, Callado Fábia M R A, Carvalho Luany E M, Calcaterra Ilenia L, Carcao Manuel, Castaman Giancarlo, Eikenboom Jeroen C J, Fischer Kathelijn, Franco Vivian K B, Heymans Martijn W, Leebeek Frank W G, Lillicrap David, Lorenzato Cláudia S, Mancuso Maria Elisa, Matino Davide, Di Minno Matteo N D, Mohseny Alex B, Oldenburg Johannes, Rezende Suely Meireles, Rivard Georges-Etienne, Rydz Natalia, Schols Saskia E M, Voorberg Jan, Fijnvandraat Karin, Gouw Samantha C, International Genetic and clinical determinants for the outcome of immune tolerance induction study group
Аннотация
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 F8 mutation type. The C statistic was 0.801 (95% CI, 0.70-0.87).
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.