Results 11 to 20 of about 256,813 (405)

The clinical spectrum of SMA‐PME and in vitro normalization of its cellular ceramide profile

Annals of Clinical and Translational Neurology, Volume 9, Issue 12, Page 1941-1952, December 2022., 2022
Abstract Objective The objectives of this study were to define the clinical and biochemical spectrum of spinal muscular atrophy with progressive myoclonic epilepsy (SMA‐PME) and to determine if aberrant cellular ceramide accumulation could be normalized by enzyme replacement.
Michelle M. Lee, Graeme S. V. McDowell, Darryl C. De Vivo, Daniel Friedman, Samuel F. Berkovic, Maria Spanou, Argirios Dinopoulos, Katheryn Grand, Pedro A. Sanchez‐Lara, Michelle Allen‐Sharpley, Jodi Warman‐Chardon, Alexander Solyom, Thierry Levade, Edward H. Schuchman, Steffany A. L. Bennett, David A. Dyment, Toni S. Pearson   +16 more
wiley   +1 more source

Donation and transplantation of allogeneic hematopoietic stem cells [PDF]

Canadian Medical Association Journal, 2011
Only 30% of patients with indications for a transplant have a matched related donor; the remaining 70% seek an unrelated donor on the world’s registry. Nonwhite Canadians have a particularly low likelihood of finding a match.1,2 Appendix 1 lists common indications for transplant (available at ...
Ilana Kopolovic, Robert Turner
openaire   +3 more sources

The consensus from The Chinese Society of Hematology on indications, conditioning regimens and donor selection for allogeneic hematopoietic stem cell transplantation: 2021 update

Journal of Hematology & Oncology, 2021
The consensus recommendations in 2018 from The Chinese Society of Hematology (CSH) on indications, conditioning regimens and donor selection for allogeneic hematopoietic stem cell transplantation (allo-HSCT) facilitated the standardization of clinical ...
Xiao-hui Zhang, Jing Chen, Mingzhe Han, He Huang, E. Jiang, M. Jiang, Y. Lai, Daihong Liu, Qi-fa Liu, Ting Liu, H. Ren, Yong-Ping Song, Zi-min Sun, Xiaowen Tang, Jian-min Wang, De-Pei Wu, Lanping Xu, Xi Zhang, Dao-bin Zhou, Xiao-jun Huang   +19 more
semanticscholar   +1 more source

Recognizing early MRI signs (or their absence) is crucial in diagnosing metachromatic leukodystrophy

Annals of Clinical and Translational Neurology, Volume 9, Issue 12, Page 1999-2009, December 2022., 2022
Abstract Objectives Metachromatic leukodystrophy (MLD) has characteristic white matter (WM) changes on brain MRI, which often trigger biochemical and genetic confirmation of the diagnosis. In early or pre‐symptomatic disease stages, these typical MRI changes might be absent, hampering early diagnosis.
Daphne H. Schoenmakers, Shanice Beerepoot, Ingeborg Krägeloh‐Mann, Saskia Elgün, Benjamin Bender, Marjo S. van der Knaap, Nicole I. Wolf, Samuel Groeschel   +7 more
wiley   +1 more source

Optimizing Allogeneic Grafts in Hematopoietic Stem Cell Transplantation [PDF]

Stem Cells Translational Medicine, 2021
AbstractAllogeneic hematopoietic stem cell transplantation (allo-HSCT) is widely used in the treatment of hematological diseases. It is well known that allogeneic grafts play a key role in predicting transplantation prognosis. Hematopoietic stem cells (HSCs) are a functional part of grafts and are capable of reconstructing hematopoiesis and immunity ...
Zheng-Li Xu, Xiao-Jun Huang
openaire   +4 more sources

Successful allogeneic hematopoietic stem cell transplantation in patients with VEXAS syndrome: a 2-center experience

Blood Advances, 2021
Key Points Allogeneic hematopoietic stem cell transplantation is a curative option in patients with VEXAS syndrome and severe manifestations.
Ava Diarra, Nicolas Duployez, E. Fournier, C. Preudhomme, V. Coiteux, L. Magro, B. Quesnel, M. Heiblig, P. Sujobert, F. Barraco, M. Balsat, Quentin Scanvion, E. Hachulla, D. Launay, I. Yakoub-Agha, L. Terriou   +15 more
semanticscholar   +1 more source

Allogeneic hematopoietic stem cell transplantation in Tunisia [PDF]

Bone Marrow Transplantation, 2008
In 1998, the Tunisian team of the 'Centre National de Greffe de Moelle Osseuse' initiated allogeneic hematopoietic SCT (AHSCT) in Tunisia. As of June 2007, information was collected about 299 patients with a first AHSCT and 12 additional retransplants. The median age was 19 years (range 2-49 years). The main indications were aplastic anemia (n=106, 36%)
Abderrahman Abdelkefi, T. Ben Othman, Saloua Ladeb, Lamia Torjemane, A. Ben Abdeladhim, H. Slama, L. Ben Hamed, Amel Lakhal   +7 more
openaire   +2 more sources

Outpatient allogeneic hematopoietic stem-cell transplantation: a review [PDF]

Therapeutic Advances in Hematology, 2022
Hematopoietic stem-cell transplantation (HSCT) is usually performed in well-equipped units inside a hospital. The cost of this in-hospital transplant is usually very high; therefore, this procedure is more difficult to perform in low- and middle-income countries.
David Gómez-Almaguer, Andrés Gómez-De León, Perla R. Colunga-Pedraza, Olga G. Cantú-Rodríguez, César Homero Gutierrez-Aguirre, Guillermo Ruíz-Arguelles   +5 more
openaire   +3 more sources

Combining CD34+ stem cell selection with prophylactic pathogen and leukemia directed T‐cell immunotherapy to simultaneously reduce graft versus host disease, infection, and leukemia recurrence after allogeneic stem cell transplant

American Journal of Hematology, Volume 98, Issue 1, Page 159-165, January 2023., 2023
Donor stem cell product undergoes CD34 stem cell selection. CD34 positive stem cells are used for transplantation. Pathogen‐ and leukemia‐specific T‐cells are manufactured from CD34 negative fraction and infused prophylactically 21 days after transplant.
David J. Gottlieb, Gaurav Sutrave, Wei Jiang, Selmir Avdic, Janine A. Street, Renee Simms, Leighton E. Clancy, Vicki Antonenas, Brian S. Gloss, Caroline Bateman, David C. Bishop, Kenneth P. Micklethwaite, Emily Blyth   +12 more
wiley   +1 more source

Maintenance therapy in acute myeloid leukemia after allogeneic hematopoietic stem cell transplantation

Journal of Hematology & Oncology, 2021
Relapse remains the main cause of treatment failure in acute myeloid leukemia (AML) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT).
L. Xuan, Qi-fa Liu
semanticscholar   +1 more source