P1522: LONG-TERM EFFICACY AND SAFETY OF THE ORAL PYRUVATE KINASE ACTIVATOR MITAPIVAT IN ADULTS WITH NON–TRANSFUSION-DEPENDENT ALPHA- OR BETA-THALASSEMIA by K. H. M. Kuo, D. M. Layton, A. Lal, H. Al-Samkari, J. Bhatia, P. A. Kosinski, B. Tong, M. Lynch, K. Uhlig, E. P. Vichinsky

Get full text

P1562: CHARACTERIZING IRON OVERLOAD BY AGE IN PATIENTS DIAGNOSED WITH PYRUVATE KINASE DEFICIENCY – A DESCRIPTIVE ANALYSIS FROM THE PEAK REGISTRY by P. Bianchi, R. F. Grace, J.-L. Vives Corrons, B. Glader, A. Glenthøj, H. Kanno, K. H. Kuo, C. Lander, D. M. Layton, D. Pospíŝilová, V. Viprakasit, J. Williams, Y. Yan, B. McGee, E. J. van Beers

Get full text

P1548: LONG-TERM TREATMENT WITH ORAL MITAPIVAT IS ASSOCIATED WITH NORMALIZATION OF HEMOGLOBIN LEVELS IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY by W. Barcellini, R. F. Grace, H. Al-Samkari, A. Glenthøj, J. A. Rothman, M. Morado Arias, D. M. Layton, O. Andres, M. DiBacco, P. Hawkins, M. P. Judge, F. Tai, J. Morales-Arias, V. Beynon, E. J. van Beers

Get full text

P1544: BONE MINERAL DENSITY REMAINS STABLE IN PYRUVATE KINASE DEFICIENCY PATIENTS RECEIVING LONG-TERM TREATMENT WITH MITAPIVAT by H. Al-Samkari, R. F. Grace, A. Glenthøj, O. Andres, W. Barcellini, F. Galactéros, K. H. M. Kuo, D. M. Layton, M. Morado Arias, V. Viprakasit, Y. Dong, F. Tai, L. Grekas, K. Khoja, S. Gheuens, B. McGee, J. B. Porter, E. J. van Beers

Get full text

P1545: DURABILITY OF HEMOGLOBIN RESPONSE AND REDUCTION IN TRANSFUSION BURDEN IS MAINTAINED OVER TIME IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY TREATED WITH MITAPIVAT IN A LONG-TE... by R. F. Grace, A. Glenthøj, W. Barcellini, M. Verhovsek, J. A. Rothman, M. Morado Arias, D. M. Layton, O. Andres, F. Galactéros, E. J. van Beers, K. Onodera, V. Viprakasit, S. Chonat, J. B. Porter, M. P. Judge, P. A. Kosinski, P. Hawkins, S. Gheuens, R. Xu, B. McGee, V. Beynon, H. Al-Samkari

Get full text