P1543: MITAPIVAT DECREASES THE NEED FOR TRANSFUSIONS SECONDARY TO POORLY TOLERATED ANEMIA AND ACUTE EVENTS COMPARED TO PLACEBO IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY WHO ARE N...

por Al-Samkari, H., Morales-Arias, J., Xu, R., Beynon, V., Grace, R. F.
Publicado 2022

S116: LONG-TERM EFFICACY AND SAFETY OF THE ORAL PYRUVATE KINASE ACTIVATOR MITAPIVAT IN ADULTS WITH NON—TRANSFUSION-DEPENDENT ALPHA- OR BETA-THALASSEMIA

por Kuo, K, Layton, D, Lal, A, Al-Samkari, H, Bhatia, J, Kosinski, P, Tong, B, Lynch, M, Uhlig, K, Vichinsky, E
Publicado 2022

P1522: LONG-TERM EFFICACY AND SAFETY OF THE ORAL PYRUVATE KINASE ACTIVATOR MITAPIVAT IN ADULTS WITH NON–TRANSFUSION-DEPENDENT ALPHA- OR BETA-THALASSEMIA

por Kuo, K. H. M., Layton, D. M., Lal, A., Al-Samkari, H., Bhatia, J., Kosinski, P. A., Tong, B., Lynch, M., Uhlig, K., Vichinsky, E. P.
Publicado 2022

P1735: IMPROVEMENTS IN PATIENT-REPORTED OUTCOMES IN MITAPIVAT-TREATED PATIENTS WITH PYRUVATE KINASE DEFICIENCY: A DESCRIPTIVE ANALYSIS FROM THE PHASE 3 ACTIVATE TRIAL

por Kuo, K. H., Grace, R. F., van Beers, E. J., Barcellini, W., Glenthøj, A., Holzhauer, S., Patel, P., Beynon, V., Li, J., Zagadailov, E., Al-Samkari, H.
Publicado 2022

P112: ENERGIZE AND ENERGIZE-T: TWO PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDIES OF MITAPIVAT IN ADULTS WITH NON--TRANSFUSION-DEPENDENT OR TRANSFUSION-DEPENDENT ALP...

por Kuo, K, Layton, D, Al-Samkari, H, Kattamis, A, Sheth, S, Taher, A, Viprakasit, V, Chamberlain, C, Czapla, L, Gheuens, S, Lynch, M, Tong, B, Uhlig, K, Cappellini, M
Publicado 2022

P1565: MITAPIVAT IMPROVES INEFFECTIVE ERYTHROPOIESIS AND REDUCES IRON OVERLOAD IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY

por van Beers, E. J., Al-Samkari, H., Grace, R. F., Barcellini, W., Glenthøj, A., Judge, M. P., Kosinski, P. A., Xu, R., Beynon, V., McGee, B., Porter, J. B., Kuo, K. H. M.
Publicado 2022

P1548: LONG-TERM TREATMENT WITH ORAL MITAPIVAT IS ASSOCIATED WITH NORMALIZATION OF HEMOGLOBIN LEVELS IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY

por Barcellini, W., Grace, R. F., Al-Samkari, H., Glenthøj, A., Rothman, J. A., Morado Arias, M., Layton, D. M., Andres, O., DiBacco, M., Hawkins, P., Judge, M. P., Tai, F., Morales-Arias, J., Beynon, V., van Beers, E. J.
Publicado 2022

P1544: BONE MINERAL DENSITY REMAINS STABLE IN PYRUVATE KINASE DEFICIENCY PATIENTS RECEIVING LONG-TERM TREATMENT WITH MITAPIVAT

por Al-Samkari, H., Grace, R. F., Glenthøj, A., Andres, O., Barcellini, W., Galactéros, F., Kuo, K. H. M., Layton, D. M., Morado Arias, M., Viprakasit, V., Dong, Y., Tai, F., Grekas, L., Khoja, K., Gheuens, S., McGee, B., Porter, J. B., van Beers, E. J.
Publicado 2022

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...

por Grace, R. F., Glenthøj, A., Barcellini, W., Verhovsek, M., Rothman, J. A., Morado Arias, M., Layton, D. M., Andres, O., Galactéros, F., van Beers, E. J., Onodera, K., Viprakasit, V., Chonat, S., Porter, J. B., Judge, M. P., Kosinski, P. A., Hawkins, P., Gheuens, S., Xu, R., McGee, B., Beynon, V., Al-Samkari, H.
Publicado 2022