Results 51 to 60 of about 339,316 (310)

Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study

Haematologica, 2019
Quality of response to immunosuppressive therapy and long-term outcomes for pediatric severe aplastic anemia remain incompletely characterized. Contemporary evidence to inform treatment of relapsed or refractory severe aplastic anemia for pediatric ...
Z. Rogers, Taizo A Nakano, T. Olson, A. Bertuch, Winfred C. Wang, A. Gillio, T. Coates, A. Chawla, Paul Castillo, P. Kurre, C. Gamper, Carolyn M Bennett, S. Joshi, A. Geddis, Jessica Boklan, G. Nalepa, J. Rothman, James N. Huang, G. Kupfer, M. Cada, B. Glader, K. Walkovich, A. Thompson, R. Hanna, A. Vlachos, M. Malsch, E. Weller, David A. Williams, A. Shimamura   +28 more
semanticscholar   +1 more source

Syngeneic peripheral blood stem cell transplantation with immunosuppression for hepatitis-associated severe aplastic anemia

Turkish Journal of Hematology, 2010
Hepatitis-associated aplastic anemia occurs in up to 10% of all aplastic anemia cases. Syngeneic bone marrow transplantation is rare in patients with severe aplastic anemia and usually requires pre-transplant conditioning to provide engraftment.
Aleksandar Savic, Bela Balint, Ivana Urosevic, Nebojsa Rajic, Milena Todorovic, Ivanka Percic, Stevan Popovic   +6 more
doaj   +3 more sources

CD34+ gene expression profiling of individual children with very severe aplastic anemia indicates a pathogenic role of integrin receptors and the proapoptotic death ligand TRAIL

Haematologica, 2012
Background Very severe aplastic anemia is characterized by a hypoplastic bone marrow due to destruction of CD34+ stem cells by autoreactive T cells. Investigation of the pathomechanism by patient-specific gene expression analysis of the attacked stem ...
Ute Fischer, Christian Ruckert, Bernd Hubner, Olaf Eckermann, Vera Binder, Tamam Bakchoul, Friedhelm R. Schuster, Sylvia Merk, Hans-Ulrich Klein, Monika Führer, Martin Dugas, Arndt Borkhardt   +11 more
doaj   +1 more source

Severe retinal hemorrhages at various levels with a serous retinal detachment in a pediatric patient with aplastic anemia–A case report

Frontiers in Medicine, 2023
BackgroundAplastic anemia can cause ophthalmic abnormalities in patients. Vision loss in a child with aplastic anemia due to massive retinal hemorrhages at various levels is rare.Case presentationA pediatric patient with aplastic anemia presented with ...
Xiaoshuang Jiang, Mengxi Shen, Licong Liang, Philip J. Rosenfeld, Fang Lu   +4 more
doaj   +1 more source

Appraisal of patient-reported outcome measures in analogous diseases and recommendations for use in phase II and III clinical trials of pyruvate kinase deficiency [PDF]

, 2018
Purpose: Pyruvate kinase deficiency (PKD) is a rare disease and understanding of its epidemiology and associated burden remains limited. With no current curative therapy, clinical manifestations can be life threatening, clinically managed by maintaining ...
Ionova, Tatyana, Johns, Jeffrey, Oliva, Esther Natalie, Salek, Mir-Saeed Shayegan   +3 more
core   +2 more sources

Aplastic anaemia: a review [PDF]

, 2003
Aplastic anaemia is featured by bone marrow hypocellularity and peripheral pancytopenia and is a potentially fatal disease. In recent years, insight in it pathogenesis has increased.
Lugtenburg, P.J. (Pieternella), Sleijfer, S. (Stefan)   +1 more
core   +6 more sources

A CASE OF APLASTIC ANEMIA. [PDF]

JAMA: The Journal of the American Medical Association, 1907
The recent and carefully prepared report of a case of aplastic anemia by Lavenson1embraces a thorough review of the literature and renders a detailed consideration of this disease unnecessary, but the cases are still so rare that it is deemed of importance to report every case. History. —Miss F. S., aged 18, was seen through the kindness of Dr. H. B.
openaire   +3 more sources

Diagnosis and treatment of pediatric acquired aplastic anemia (AAA): An initial survey of the North American Pediatric Aplastic Anemia Consortium (NAPAAC) [PDF]

, 2013
Peer Reviewedhttp://deepblue.lib.umich.edu/bitstream/2027.42/106711/1/pbc24875 ...
Bennett, Carolyn, Bertuch, Alison, Bessler, Monica, Coates, Thomas, Corey, Seth, Dror, Yigal, Huang, James, Lipton, Jeffrey, Olson, Timothy S., Reiss, Ulrike M., Rogers, Zora R., Shimamura, Akiko, Sieff, Colin, Vlachos, Adrianna, Walkovich, Kelly, Wang, Winfred, Williams, David A.   +16 more
core   +1 more source

T-cell expression of Bruton’s tyrosine kinase promotes autoreactive T-cell activation and exacerbates aplastic anemia

Cellular & Molecular Immunology, 2019
The role of Bruton’s tyrosine kinase (BTK) in BCR signaling is well defined, and BTK is involved in B-cell development, differentiation, and malignancies.
Simo Xia, Xiang Liu, Xuetao Cao, Sheng Xu   +3 more
semanticscholar   +1 more source

Effectiveness of Iptacopan Versus C5 Inhibitors in Complement Inhibitor‐Naive Patients With Paroxysmal Nocturnal Haemoglobinuria

eJHaem
Background Paroxysmal nocturnal haemoglobinuria (PNH) is characterised by haemolytic anaemia, bone marrow failure and thrombosis. The single‐arm phase 3 APPOINT‐PNH trial (NCT04820530) investigating iptacopan monotherapy in complement inhibitor‐naive ...
Matthew Holt, Richard J. Kelly, Jilles M. Fermont, Soudeh Ansari, Marion Dahlke, Isabelle Brindel, Lynda Maafa, Flore Sicre deFontbrune, Régis Peffault deLatour   +8 more
doaj   +1 more source