Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria.

Autor: John Devin Peipert, Austin G Kulasekararaj, Anna Gaya, Saskia M C Langemeijer, Susan Yount, F Ataulfo Gonzalez-Fernandez, Emilio Ojeda Gutierrez, Christa Martens, Amy Sparling, Kimberly A Webster, David Cella, Ioannis Tomazos, Masayo Ogawa, Caroline I Piatek, Richard Wells, Flore Sicre de Fontbrune, Alexander Röth, Lindsay Mitchell, Anita Hill, Karen Kaiser

Publikováno v: PLoS ONE, Vol 15, Iss 9, p e0237497 (2020)

BackgroundEculizumab has transformed management of paroxysmal nocturnal hemoglobinuria (PNH) since its approval. However, its biweekly dosing regimen remains a high treatment burden. Ravulizumab administered every 8 weeks demonstrated noninferiority

Externí odkaz: https://doaj.org/article/f886a2dbac2e4004b37122b11f34a4e1

Autosomal Recessive Congenital Dyserythropoietic Anemia Type III Is Caused By Mutations in the Centralspindlin RACGAP1 Component

Autor: Lídia Romero-Cortadellas, Concepción Pérez de Soto, Rosario M. Morales-Camacho, Gonzalo Hernández, Ana Villegas, Santiago Pérez-Montero, Cristian Tornador, Mayka Sanchez, Xènia Ferrer-Cortès, Mireia Olivella, Veronica Venturi, Marta Morado, F. Ataulfo Gonzalez-Fernandez

Publikováno v: Blood. 138:847-847

An autosomal dominant form of congenital dyserythropoietic anemia type III (CDA III) is caused by a missense mutation in the KIF23 gene whose protein product, mitotic kinesin-like protein (MKLP1), is part of the centralspindlin complex involved in cy

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::764d4072965d994b7ebd3f5153750958
https://doi.org/10.1182/blood-2021-150097

Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor-experienced adult patients with PNH: the 302 study

Autor: Régis Peffault de Latour, Saskia Langemeijer, Anna Gaya, Lori Shafner, Scott T. Rottinghaus, Austin G. Kulasekararaj, Jeff Szer, Caroline I. Piatek, Stephan Ortiz, Emilio Ojeda Gutierrez, Anita J. Hill, Richard A. Wells, F. Ataulfo Gonzalez-Fernandez, Eric Bachman, Antonio M. Risitano, Andrew I. Damokosh, Jong Wook Lee, Shinji Nakao, Alexander Röth

Publikováno v: Blood, 133, 6, pp. 540-549
Repositorio Institucional de la Consejería de Sanidad de la Comunidad de Madrid
Consejería de Sanidad de la Comunidad de Madrid
Blood, 133, 540-549

Contains fulltext : 202845.pdf (Publisher’s version ) (Open Access) Ravulizumab, a new complement component C5 inhibitor administered every 8 weeks, was noninferior to eculizumab administered every 2 weeks in complement-inhibitor-naive patients wit

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f87c292ea6251462031e05173408aaff
https://pubmed.ncbi.nlm.nih.gov/30733201

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