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

P1542: COMORBIDITIES AND COMPLICATIONS ACROSS GENOTYPES IN ADULT PATIENTS WITH PYRUVATE KINASE DEFICIENCY: ANALYSIS FROM THE PEAK REGISTRY

por Glenthøj, A., Grace, R. F., van Beers, E. J., Vives Corrons, J.-L., Glader, B., Kuo, K. H. M., Lander, C., Pospíŝilová, D., Williams, J., Yan, Y., McGee, B., Bianchi, P.
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

Ektacytometry Analysis of Post-splenectomy Red Blood Cell Properties Identifies Cell Membrane Stability Test as a Novel Biomarker of Membrane Health in Hereditary Spherocytosis

por Berrevoets, M. C., Bos, J., Huisjes, R., Merkx, T. H., van Oirschot, B. A., van Solinge, W. W., Verweij, J. W., Lindeboom, M. Y. A., van Beers, E. J., Bartels, M., van Wijk, R., Rab, M. A. E.
Publicado 2021

P1562: CHARACTERIZING IRON OVERLOAD BY AGE IN PATIENTS DIAGNOSED WITH PYRUVATE KINASE DEFICIENCY – A DESCRIPTIVE ANALYSIS FROM THE PEAK REGISTRY

por Bianchi, P., Grace, R. F., Vives Corrons, J.-L., Glader, B., Glenthøj, A., Kanno, H., Kuo, K. H., Lander, C., Layton, D. M., Pospíŝilová, D., Viprakasit, V., Williams, J., Yan, Y., McGee, B., van Beers, E. J.
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

P1065: CONGENITAL ERYTHROCYTOSIS DUE TO HETEROZYGOUS VARIANTS IN THE BISPHOSPHOGLYCERATE MUTASE GENE

por van Dijk, M. J., van Oirschot, B. A., Stam-Slob, M. C., van der Zwaag, B., van Beers, E. J., Jans, J. J., van der Linden, P., Torregrosa Diaz, J. M., Gardie, B., Girodon, F., Schots, R., Thielen, N., van Wijk, R.
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

P1501: FOLLOW-UP RESULTS OF A PHASE 2 STUDY ASSESSING THE SAFETY AND EFFICACY OF MITAPIVAT TREATMENT, AN ORAL PYRUVATE KINASE ACTIVATOR, FOR UP TO 60 WEEKS IN SUBJECTS WITH SICKLE...

por van Dijk, M. J., Rab, M. A., van Oirschot, B. A., Bos, J., Derichs, C., Rijneveld, A. W., Cnossen, M. H., Nur, E., Biemond, B. J., Bartels, M., Jans, J. J., van Solinge, W. W., Schutgens, R. E., van Wijk, R., van Beers, E. J.
Publicado 2022

5589320 FOLLOW-UP RESULTS OF A PHASE 2 STUDY ASSESSING THE SAFETY AND EFFICACY OF MITAPIVAT TREATMENT, AN ORAL PYRUVATE KINASE ACTIVATOR, FOR UP TO 60 WEEKS IN SUBJECTS WITH SICKLE...

por van Dijk, M.J., Rab, M.A.E., van Oirschot, B.A., Bos, J.F., Derichs, C., Rijneveld, A.W., Cnossen, M.H., Nur, E., Biemond, B.J., Bartels, M., Jans, J.J.M., van Solinge, W.W., Schutgens, R.E.G., van Wijk, R., van Beers, E.J.
Publicado 2023

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