hrp0089fc11.1 | Bone, Growth Plate & Mineral Metabolism 2 | ESPE2018

Successful Immune Tolerance Induction in the First Case of Neutralizing Antibody Mediated Loss of Efficacy of Asfotase Alfa Treatment in Hypophosphatasia

Martos-Moreno Gabriel A , Gonzalez-Vicent Marta , Sebastian Elena , Argente Jesus

Introduction: Generation of neutralizing antibodies (Nab) is a complication in enzyme replacement therapies and can lead to loss of treatment efficacy. Asfotase alfa (AA) was recently approved as the first replacement therapy in severe hypophosphatasia (congenital deficiency of alkaline phosphatase [TNSALP]). We report a case of neutralizing antibody mediated loss of efficacy of AA treatment in hypophosphatasia and the successful result of immune tolerance ind...