Joint model robustness compared with the time-varying covariate Cox model to evaluate the association between a longitudinal marker and a time-to-event endpoint

Autor: Maeregu W. Arisido, Laura Antolini, Davide P. Bernasconi, Maria G. Valsecchi, Paola Rebora

Publikováno v: BMC Medical Research Methodology, Vol 19, Iss 1, Pp 1-13 (2019)

Abstract Background The recent progress in medical research generates an increasing interest in the use of longitudinal biomarkers for characterizing the occurrence of an outcome. The present work is motivated by a study, where the objective was to e

Externí odkaz: https://doaj.org/article/6143077b51de43b0bfd52ee425ebe9bb

Unraveling the cellular origin and clinical prognostic markers of infant B-cell acute lymphoblastic leukemia using genome-wide analysis

Autor: Antonio Agraz-Doblas, Clara Bueno, Rachael Bashford-Rogers, Anindita Roy, Pauline Schneider, Michela Bardini, Paola Ballerini, Gianni Cazzaniga, Thaidy Moreno, Carlos Revilla, Marta Gut, Maria G. Valsecchi, Irene Roberts, Rob Pieters, Paola De Lorenzo, Ignacio Varela, Pablo Menendez, Ronald W. Stam

Publikováno v: Haematologica, Vol 104, Iss 6 (2019)

B-cell acute lymphoblastic leukemia is the commonest childhood cancer. In infants, B-cell acute lymphoblastic leukemia remains fatal, especially in patients with t(4;11), present in ~80% of cases. The pathogenesis of t(4;11)/KMT2A-AFF1+ (MLL-AF4+) in

Externí odkaz: https://doaj.org/article/e28a2faf5de741be80bab67752885060

Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome

Autor: Bernhard, Gentner, Francesca, Tucci, Stefania, Galimberti, Francesca, Fumagalli, Maurizio, De Pellegrin, Paolo, Silvani, Chiara, Camesasca, Silvia, Pontesilli, Silvia, Darin, Francesca, Ciotti, Marina, Sarzana, Giulia, Consiglieri, Chiara, Filisetti, Giulia, Forni, Laura, Passerini, Daniela, Tomasoni, Daniela, Cesana, Andrea, Calabria, Giulio, Spinozzi, Maria-Pia, Cicalese, Valeria, Calbi, Maddalena, Migliavacca, Federica, Barzaghi, Francesca, Ferrua, Vera, Gallo, Simona, Miglietta, Erika, Zonari, Patali S, Cheruku, Claudia, Forni, Marcella, Facchini, Ambra, Corti, Michela, Gabaldo, Stefano, Zancan, Serena, Gasperini, Attilio, Rovelli, Jaap-Jan, Boelens, Simon A, Jones, Robert, Wynn, Cristina, Baldoli, Eugenio, Montini, Silvia, Gregori, Fabio, Ciceri, Maria G, Valsecchi, Giancarlo, la Marca, Rossella, Parini, Luigi, Naldini, Alessandro, Aiuti, Maria-Ester, Bernardo, Ilaria, Visagalli

Publikováno v: New England Journal of Medicine. 385:1929-1940

Background Allogeneic hematopoietic stem-cell transplantation is the standard of care for Hurler syndrome (mucopolysaccharidosis type I, Hurler variant [MPSIH]). However, this treatment is only partially curative and is associated with complications.

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::dc127ebe27e8c45f4441f81d50e24d74
https://doi.org/10.1056/nejmoa2106596

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Central nervous system involvement in childhood acute lymphoblastic leukemia is linked to upregulation of cholesterol biosynthetic pathways

Autor: A. Cousins, O. Olivares, E. Markert, A. Manoharan, X. Bubnova, S. Bresolin, M. Degn, Z. Li, D. Silvestri, G. McGregor, S. Tumanov, D. Sumpton, J. J. Kamphorst, A. M. Michie, P. Herzyk, M. G. Valsecchi, A. E. Yeoh, K. Schmiegelow, G. te Kronnie, E. Gottlieb, C. Halsey

Publikováno v: Cousins, A, Olivares, O, Markert, E, Manoharan, A, Bubnova, X, Bresolin, S, Degn, M, Li, Z, Silvestri, D, McGregor, G, Tumanov, S, Sumpton, D, Kamphorst, J J, Michie, A M, Herzyk, P, Valsecchi, M G, Yeoh, A E, Schmiegelow, K, te Kronnie, G, Gottlieb, E & Halsey, C 2022, ' Central nervous system involvement in childhood acute lymphoblastic leukemia is linked to upregulation of cholesterol biosynthetic pathways ', Leukemia, vol. 36, no. 12, pp. 2903-2907 . https://doi.org/10.1038/s41375-022-01722-x

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::66908e5220f89b723d10588796e1cfe2
https://hdl.handle.net/11577/3483125