Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease

Autor: Jane E. Potter, Gemma Petts, Arunabha Ghosh, Fiona J. White, Jane L. Kinsella, Stephen Hughes, Jane Roberts, Adam Hodgkinson, Kathryn Brammeier, Heather Church, Christine Merrigan, Joanne Hughes, Pamela Evans, Helen Campbell, Denise Bonney, William G. Newman, Brian W. Bigger, Alexander Broomfield, Simon A. Jones, Robert F. Wynn

Publikováno v: Orphanet Journal of Rare Diseases, Vol 16, Iss 1, Pp 1-14 (2021)

Abstract Background Wolman disease is a rare, lysosomal storage disorder in which biallelic variants in the LIPA gene result in reduced or complete lack of lysosomal acid lipase. The accumulation of the substrates; cholesterol esters and triglyceride

Externí odkaz: https://doaj.org/article/d3be9f8f7dcb489b904a47d7c2643970

ALL-REZ BFM 2002 Is Associated with Improved Outcome as Compared to ALL-R3 Strategy in Children with Standard Risk Isolated CNS Relapse of Acute Lymphoblastic Leukemia, while Maintaining Comparable Efficacy in Patients with Bone Marrow Relapse. Results of the Multi-National, Multi-Center Trial IntReALL SR 2010

Autor: Arend von Stackelberg, Jean-Pierre Bourquin, Martin Zimmermann, Tamas Revesz, Andishe Attarbaschi, Alina Ferster, Lucie Sramkova, Thomas Leth Frandsen, Päivi Maria Lähteenmäki, Ronit Elhasid, Hidemi Toyoda, Peter M. Hoogerbrugge, Inga M. Johannsdottir, Ximo Duarte, Denise Bonney, Vaskar Saha, Ingo G. Steffen, Andrej Lissat, Christiane Chen-Santel, Cornelia Eckert, Andre Baruchel, Arnaud Petit, Hélène Cavé, Carmelo Rizzari, Giovanni Cazzaniga, Luciana Vinti, Pierre Rohrlich, Franco Locatelli

Publikováno v: Blood. 140:3247-3249

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::a8e400d044c6a4b6e73bad19aaf30d52
https://doi.org/10.1182/blood-2022-166234

Reduced Intensity Reinduction Followed By Blinatumomab Achieves Excellent MRD Clearance with Reduced Toxicity and Facilitates Timely Transplant in Children and Young People with Relapsed B-Precursor Acute Lymphoblastic Leukemia

Autor: Angus Hodder, Avijeet K Mishra, Katherine Clesham, Susan Baird, Kaljit Bhuller, Ismail Bisho, Denise Bonney, Anna Castleton, Michelle Cummins, Emmy Dickens, Lindsay George, Sara Ghorashian, Brenda Gibson, Chris Hasley, Nicholas Heaney, Rachael E Hough, Danielle Ingham, Galina Jigoulina, Katherine Lindsay, Donna Lancaster, Majid Madni, Andrea Malone, Bethany Mitchell, Anthony V. Moorman, John Moppett, Vanessa McLelland, Jayashree Motwani, Emma Nicholson, Caroline Osborne, Katharine Patrick, Lamia Samrin, Sanjay Tewari, Indu Rakesh Thakur, Sujith Samarasinghe, Ajay Vora, David O'Connor

Publikováno v: Blood. 140:3268-3270

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::64a97d220552d88872d3c949db3523e0
https://doi.org/10.1182/blood-2022-162565

Dual Antigen Targeting with Co-Transduced CD19/22 CAR T Cells May Prevent Antigen-Negative Relapse after CAR T Cell Therapy for Relapsed/Refractory ALL

Autor: Sara Ghorashian, Giovanna Lucchini, Rachel Richardson, Kyvi Nguyen, Craig Terris, Macarena Oporto-Espuelas, Jenny Yeung, Danielle Pinner, Jan Chu, Lindsey Williams, Ka-yuk Ko, Chloe Walding, Kelly Watts, Sarah Inglott, Stuart Adams, Emma Gravett, Kimberly Gilmour, Alka Lal, Sangeetha Kunaseelan, Bilyana Popova, Andre Lopes, Yenting Ngai, Evangelia K Kokalaki, Milena Balasch Carulla, Khushnuma Mullanfiroze, Arina Lazareva, Vesna Pavasovic, Anupama Rao, Jack Bartram, Ajay Vora, Robert Chiesa, Juliana Silva, Kanchan Rao, Katherine Clesham, Denise Bonney, Robert F Wynn, Martin Pule, Rachael E Hough, Persis J Amrolia

Publikováno v: Blood. 140:10352-10354

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::ea12492684ee229651c495469b5c0218
https://doi.org/10.1182/blood-2022-164879

Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease

Autor: Denise Bonney, Alexander Broomfield, Fiona White, Jane Roberts, Jane Louise Kinsella, Arunabha Ghosh, Robert Wynn, Joanne Hughes, Jane E. Potter, Helen Campbell, Christine Merrigan, Pamela Evans, Adam Hodgkinson, Gemma Petts, Simon Jones, Heather J. Church, Stephen M. Hughes, Brian W. Bigger, William G. Newman, Kathryn Brammeier

Publikováno v: Orphanet Journal of Rare Diseases, Vol 16, Iss 1, Pp 1-14 (2021)
Potter, J, Newman, W, Jones, S, Wynn, R, Ghosh, A, Broomfield, A & Bigger, B 2021, ' Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease ', Orphanet Journal of Rare Diseases, vol. 16, no. 1, 235 . https://doi.org/10.1186/s13023-021-01849-7
Orphanet Journal of Rare Diseases

BackgroundWolman disease is a rare, lysosomal storage disorder in which biallelic variants in theLIPAgene result in reduced or complete lack of lysosomal acid lipase. The accumulation of the substrates; cholesterol esters and triglycerides, significa

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::646bbf105331fe78fffe045b41d6488f
https://doaj.org/article/d3be9f8f7dcb489b904a47d7c2643970