Targeted treatment of severe vascular malformations harboring PIK3CA and TEK mutations with alpelisib is highly effective with limited toxicity

Autor: Martin Sterba, Petra Pokorna, Renata Faberova, Blanka Pinkova, Jarmila Skotakova, Anna Seehofnerova, Jan Blatny, Lucia Janigova, Olga Koskova, Hana Palova, Michal Mahdal, Lukas Pazourek, Petr Jabandziev, Ondrej Slaby, Peter Mudry, Jaroslav Sterba

Publikováno v: Scientific Reports, Vol 13, Iss 1, Pp 1-9 (2023)

Abstract This was a prospective cohort study of eighteen patients with large and debilitating vascular malformations with one or more major systemic complications. In all patients, we discovered activating alterations in either TEK or PIK3CA. Based o

Externí odkaz: https://doaj.org/article/a577de5101bc4a5b9cfe353e1382fac2

Capnometry during neonatal transport—Mini review

Autor: Hana Fucikova, Jan Blatny, Jan Stingl, Jan Miletin

Publikováno v: Acta Paediatrica. 112:919-923

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::6e449071f8f1eedb8bc7a1deae37835c
https://doi.org/10.1111/apa.16729

The prospective Hemophilia Inhibitor PUP Study reveals distinct antibody signatures prior to FVIII inhibitor development

Autor: Amy D. Shapiro, B. Gangadharan, J. Bowen, Christoph Male, Helmut Schweiger, C. J. Hofbauer, Verena Berg, Elena Santagostino, Michael Recht, Margaret V. Ragni, Janice M. Staber, Deborah L Brown, Jenny Klintman, Shannon L. Meeks, Birgit M. Reipert, Karin Fijnvandraat, Hassan M. Yaish, Jan Blatny, Catherine E. McGuinn, Eric S. Mullins, Vlad C. Radulescu

Publikováno v: Blood Adv
Blood advances, 4(22), 5785-5796. American Society of Hematology

Preventing factor VIII (FVIII) inhibitors following replacement therapies with FVIII products in patients with hemophilia A remains an unmet medical need. Better understanding of the early events of evolving FVIII inhibitors is essential for risk ide

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::681fd965767be44a9de0a71548b5d13f
https://doi.org/10.1182/bloodadvances.2020002731

Future needs for continuing innovation in hemophilia: improving outcomes for individuals of all severities, including women and those in resource-constrained regions

Autor: Jan Blatný, Jan Astermark, Cristina Catarino, Gerry Dolan, Karin Fijnvandraat, Cédric Hermans, Katharina Holstein, Víctor Jiménez-Yuste, Robert Klamroth, Michelle Lavin, Peter J. Lenting, Sébastien Lobet, Maria Elisa Mancuso, Jayashree Motwani, James S. O’Donnell, Christoph Königs

Publikováno v: Therapeutic Advances in Hematology, Vol 15 (2024)

Over recent decades, management of people with hemophilia (PwH) has been greatly improved by scientific advances that have resulted in a rich and varied therapeutic landscape. Nevertheless, treatment limitations continue to drive innovation, and emer

Externí odkaz: https://doaj.org/article/49b84b74635547b39a4cb8316ffbb30c

Using pharmacokinetics for tailoring prophylaxis in people with hemophilia switching between clotting factor products: A scoping review

Autor: Jacky K. Yu, Alfonso Iorio, Andrea N. Edginton, Sanjay Ahuja, Ma Teresa Álvarez Román, Ma E. Arrieta, Mikko Arola, Giovanni Barillari, Vinod Balasa, Mark Belletrutti, Ruben Berrueco Moreno, Philippe Beurrier, Cristoph Bidlingmaier, Victor Blanchette, Jan Blatny, Santiago Bonanad, Kelsey Brose, Deborah Brown, Paulette C. Byant, Mariana Canaro, Manuela Carvalho, Cristina Catarino, Meera Chitlur, Erin Cockrell, Pratima Chowdary, Marjon Cnossen, Peter Collins, Michial Coppens, Stacy Croteau, Dorina Cultrera, Raimundo de Cristofaro, Emmauelle de Raucourt, Dominique Desprez, Amy Dunn, Magda El‐Ekiabi, Barbara Faganel Kotnik, Kathleen Fischer, Brigit Frotscher, Susana Garbiero, Raquel Garrido Ruiz, Joan Gill, Carmen Gomez del Castillo, Saskia Gottstein, Giuseppe Lassandro, Paola Giordano, Daniel Hart, Inga Hegemann, Cedric Hermans, Baolai Hua, Nina Hwang, Shannon Jackson, Paula James, Olga Katsarou, Kaan Kavakli, Christine Kempton, Karim Kentouche, Osman Khan, Rainer Kobelt, Rebecca Kruse‐Jarres, Edward Laane, Eric Larson, Riitta Lassila, Adrienne Lee, Man‐Chiu Poon, Jennifer Lissick, Satu Langstrom, Johnny Mahlangu, Michael Makris, Emmanuela Marchesini, Jose Mateo, Pacual Marco Vera, Marta Martorell, Tadashi Matsushita, Simon McCrae, Eva Mignot‐Castellano, Caitlin Montcrieff, Philip Maes, Veerle Mondelars, Marlies Bekart, Elena Mora, Juan Cristóbal Morales, Guillaume Mourey, Marie Ann Bertrand, Mariasanta Napolitano, Sergio Siragusa, Claude Negrier, Daniela Neme, Ritta Niinimaki, Johannes Oldenburg, Thilo Albert, Deborah Ornstein, Margarete Ozelo, John Carl Panetta, Ellis J. Neufeld, Stephanie P'Ng, Kathelijne Peerlinck, Berardino Pollio, Claire Pouplard, Yves Gruel, Alessandra Prezotti, Vicky Price, Fitri Primacakti, Mathieu Puyade, Paolo Radossi, Leslie Raffini, Margaret Ragni, Savita Rangarajan, Mark T. Reding, Robin Reid, Jose Restrepo, Jose Ramirez, Michael Recht, Manuel Rodriguez Lopez, Arlette Ruiz‐Sàez, Mahasen Saleh, Amy Shapiro, Anjali Sharathkumar, Anna Selmeczi, Mindy Simpson, Tami Singleton, Maria Sol Cruz, Veronica Soto, MacGregor Steele, Werner Streif, Hao Wei Sun, Bruce Ritchie, Jing Sun, Xiaqin Feng, Takashi Suzuki, Asuza Nagao, Cliff Takemoto, Heather Tapp, Jerry Teitel, Alan Tinmouth, Courtney Thornburg, Alberto Tosseto, Oliver Turnstall, Catherine Vezina, Beth Warren, Allison Wheeler, Juan D. Wilches Gutierrez, John K.M. Wu, Tung Wynn, Renchi Yang, Guy Young, Ezio Zanon, Irena Zupan

Publikováno v: Research and practice in thrombosis and haemostasis, Vol. 3, no.3, p. 528-541 (2019)
Research and Practice in Thrombosis and Haemostasis
Research and Practice in Thrombosis and Haemostasis, Vol 3, Iss 3, Pp 528-541 (2019)

The objective of this scoping review is to summarize the current use of pharmacokinetics for tailoring prophylaxis in hemophilia patients switching between clotting factor products. Patients with hemophilia may require switching of clotting factor co

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8855ec59f7541838e90c9de25930d966
https://hdl.handle.net/2078.1/224098