Pyruvate kinase deficiency in a newborn with extramedullary hematopoiesis in the skin

por Voit, Richard A., Grace, Rachael F.
Publicado 2020

The role of romiplostim for pediatric patients with immune thrombocytopenia

por Al-Samkari, Hanny, Grace, Rachael F., Kuter, David J.
Publicado 2020

PB2619: THE IMPACT OF ITP-DIRECTED TREATMENT ON FATIGUE AMONG CHILDREN AND ADOLESCENTS WITH CHRONIC ITP: DATA FROM THE ITP NATURAL HISTORY STUDY REGISTRY

por Diraimo, Jennifer, Kruse, Caroline, Kruse, Alexandra, Won, Kevin Doyoon, Grace, Rachael F.
Publicado 2023

PB2620: NIGHTTIME SYMPTOMS ARE ASSOCIATED WITH FATIGUE AMONG CHILDREN AND ADOLESCENTS WITH CHRONIC ITP: DATA FROM THE ITP NATURAL HISTORY STUDY REGISTRY

por Diraimo, Jennifer, Kruse, Caroline, Kruse, Alexandra, Won, Kevin Doyoon, Grace, Rachael F.
Publicado 2023

Response to rituximab in children and adults with immune thrombocytopenia (ITP)

por Harris, Emily M., Hillier, Kirsty, Al‐Samkari, Hanny, Berbert, Laura, Grace, Rachael F.
Publicado 2021

P1477: MITAPIVAT EFFICACY IN ADULTS WITH PYRUVATE KINASE DEFICIENCY AND BASELINE HEMOGLOBIN LEVELS >10 G/DL

por Grace, Rachael F., Kwiatkowski, Janet, van Beers, Eduard, Tai, Feng, Mcgee, Bryan, Al-Samkari, Hanny
Publicado 2023

Erythrocyte pyruvate kinase deficiency: 2015 status report

por Grace, Rachael F., Zanella, Alberto, Neufeld, Ellis J., Morton, D. Holmes, Eber, Stefan, Yaish, Hassan, Glader, Bertil
Publicado 2015

Development of the pyruvate kinase deficiency diary and pyruvate kinase deficiency impact assessment: Disease‐specific assessments

por Salek, Sam, Boscoe, Audra N., Piantedosi, Sarah, Egan, Shayna, Evans, Christopher J, Wells, Ted, Cohen, Jennifer, Klaassen, Robert J., Grace, Rachael, Storm, Michael
Publicado 2020

Psychometric validation of the Pyruvate Kinase Deficiency Diary and Pyruvate Kinase Deficiency Impact Assessment in adults in the phase 3 ACTIVATE trial

por Andrae, David A., Grace, Rachael F., Jewett, Adrian, Foster, Brandon, Klaassen, Robert J., Salek, Sam, Li, Junlong, Tai, Feng, Boscoe, Audra N., Zagadailov, Erin
Publicado 2023

Exome sequencing results in successful diagnosis and treatment of a severe congenital anemia

por Lacy, Jessica N., Ulirsch, Jacob C., Grace, Rachael F., Towne, Meghan C., Hale, John, Mohandas, Narla, Lux, Samuel E., Agrawal, Pankaj B., Sankaran, Vijay G.
Publicado 2016

Phase 3 randomised trial of eltrombopag versus standard first-line pharmacological management for newly diagnosed immune thrombocytopaenia (ITP) in children: study protocol

por Shimano, Kristin A, Grace, Rachael F, Despotovic, Jenny M, Neufeld, Ellis J, Klaassen, Robert J, Bennett, Carolyn M, Ma, Clement, London, Wendy B, Neunert, Cindy
Publicado 2021

Immune dysregulation and multisystem inflammatory syndrome in children (MIS-C) in individuals with haploinsufficiency of SOCS1

por Lee, Pui Y., Platt, Craig D., Weeks, Sabrina, Grace, Rachael F., Maher, George, Gauthier, Kasey, Devana, Sridevi, Vitali, Sally, Randolph, Adrienne G., McDonald, Douglas R., Geha, Raif S., Chou, Janet
Publicado 2020

The variable manifestations of disease in pyruvate kinase deficiency and their management

por Al-Samkari, Hanny, van Beers, Eduard J., Kuo, Kevin H.M., Barcellini, Wilma, Bianchi, Paola, Glenthøj, Andreas, del Mar Mañú Pereira, María, van Wijk, Richard, Glader, Bertil, Grace, Rachael F.
Publicado 2020

P1497: MITAPIVAT IMPROVES IRON OVERLOAD IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY WHO ARE REGULARLY TRANSFUSED

por van Beers, Eduard, Al-Samkari, Hanny, Grace, Rachael F., Barcellini, Wilma, Glenthøj, Andreas, Beynon, Vanessa, Wind-Rotolo, Megan, Xu, Rengyi, Dibacco, Melissa, Patel, Parija, Porter, John B., Kuo, Kevin H.M.
Publicado 2023

P1479: COMORBIDITIES AND COMPLICATIONS IN ADULTS WITH PYRUVATE KINASE DEFICIENCY ACCORDING TO HEMOGLOBIN STRATA – A DESCRIPTIVE ANALYSIS FROM THE PEAK REGISTRY

por Pospíŝilová, Dagmar, Grace, Rachael F., Glenthøj, Andreas, Lander, Carl, van Beers, Eduard, Glader, Bertil, Layton, Mark, Kuo, Kevin H.M., Vives-Corrons, Juan-Luis, Yan, Yan, Mcgee, Bryan, Higa, Sara, Bianchi, Paola
Publicado 2023

Tapering thrombopoietin receptor agonists in primary immune thrombocytopenia: Expert consensus based on the RAND/UCLA modified Delphi panel method

por Cuker, Adam, Despotovic, Jenny M., Grace, Rachael F., Kruse, Caroline, Lambert, Michele P., Liebman, Howard A., Lyons, Roger M., McCrae, Keith R., Pullarkat, Vinod, Wasser, Jeffrey S., Beenhouwer, David, Gibbs, Sarah N., Yermilov, Irina, Broder, Michael S.
Publicado 2020

P1473: CLINICALLY RELEVANT HEMOGLOBIN RESPONSE IN ADULTS WITH PYRUVATE KINASE DEFICIENCY TREATED WITH MITAPIVAT – A SUB-ANALYSIS OF THE ACTIVATE TRIAL

por Al-Samkari, Hanny, Grace, Rachael F., Glenthøj, Andreas, Barcellini, Wilma, Verhovsek, Madeleine, Rothman, Jennifer A., Morado, Marta, Layton, Mark, Andres, Oliver, Galactéros, Frédéric, Onodera, Koichi, Chonat, Satheesh, Xu, Rengyi, Mcgee, Bryan, Dibacco, Melissa, Morales, Jaime, van Beers, Eduard
Publicado 2023

Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency

por Al-Samkari, Hanny, van Beers, Eduard J., Morton, D. Holmes, Eber, Stefan W., Chonat, Satheesh, Kuo, Kevin H. M., Kollmar, Nina, Wang, Heng, Breakey, Vicky R., Sheth, Sujit, Sharma, Mukta, Forbes, Peter W., Klaassen, Robert J., Grace, Rachael F.
Publicado 2022

The Pyruvate Kinase Deficiency Global Longitudinal (Peak) Registry: rationale and study design

por Grace, Rachael F, van Beers, Eduard J, Vives Corrons, Joan-Lluis, Glader, Bertil, Glenthøj, Andreas, Kanno, Hitoshi, Kuo, Kevin H M, Lander, Carl, Layton, D Mark, Pospíŝilová, Dagmar, Viprakasit, Vip, Li, Junlong, Yan, Yan, Boscoe, Audra N, Bowden, Chris, Bianchi, Paola
Publicado 2023

Comorbidities and complications in adults with pyruvate kinase deficiency

por Boscoe, Audra N., Yan, Yan, Hedgeman, Elizabeth, van Beers, Eduard J., Al‐Samkari, Hanny, Barcellini, Wilma, Eber, Stefan W., Glader, Bertil, Yaish, Hassan M., Chonat, Satheesh, Sharma, Mukta, Kuo, Kevin H. M., Neufeld, Ellis J., Wang, Heng, Verhovsek, Madeleine, Sheth, Sujit, Grace, Rachael F.
Publicado 2021