Results 41 to 50 of about 334,579 (212)

Myelodysplastic syndrome evolving from aplastic anemia treated with immunosuppressive therapy: efficacy of hematopoietic stem cell transplantation

Haematologica, 2014
A proportion of patients with aplastic anemia who are treated with immunosuppressive therapy develop clonal hematologic disorders, including post-aplastic anemia myelodysplastic syndrome.
Sung-Yong Kim, Jennifer Le Rademacher, Joseph H. Antin, Paolo Anderlini, Mouhab Ayas, Minoo Battiwalla, Jeanette Carreras, Joanne Kurtzberg, Ryotaro Nakamura, Mary Eapen, H. Joachim Deeg   +10 more
doaj   +1 more source

Treatment optimization and genomic outcomes in refractory severe aplastic anemia treated with eltrombopag.

Blood, 2019
Eltrombopag (EPAG) received approval from the US Food and Drug Administration for the treatment of refractory severe aplastic anemia (rSAA) based on treatment of 43 patients with doses escalating from 50 to 150 mg daily for 12 weeks.
T. Winkler, Xing Fan, J. Cooper, R. Desmond, David J. Young, D. Townsley, P. Scheinberg, Sophia Grasmeder, A. Larochelle, M. Desierto, Janet Valdez, J. Lotter, Colin O. Wu, R. Shalhoub, K. Calvo, N. Young, C. Dunbar   +16 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

THPO gene variants in patients with acquired aplastic anemia

Hematology, Transfusion and Cell Therapy, 2018
Background: Human aplastic anemia is a hematologic disease characterized by low peripheral blood cell counts associated with reduced numbers of hematopoietic stem and progenitor cells and a hypocellular bone marrow.
Pedro Henrique Padilha, Gustavo Borges, Barbara Amélia Santana, Larissa Alessandra Medeiros, Samir Kanaan Nabhan, Ricardo Pasquini, Flavia Sacilotto Donaires, Rodrigo Tocantins Calado   +7 more
doaj   +1 more source

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, Anjulika Chawla, Paul Castillo, P. Kurre, C. Gamper, C. 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

Eltrombopag in Aplastic Anemia [PDF]

Seminars in Hematology, 2015
The treatment of aplastic anemia is currently with immunosuppressive therapy (IST) with anti-thymocyte globulin (ATG) and cyclosporine, to which two thirds of patients respond. However, a significant proportion of these responders relapse and many have persistent cytopenias. The management of these patients is challenging.
Ronan, Desmond, Danielle M, Townsley, Cynthia, Dunbar, Neal S, Young   +3 more
openaire   +2 more sources

Using cell‐free RNA to identify B‐ and T‐cell clonality for diagnosis and monitoring of B‐ and T‐cell neoplasms

FEBS Open Bio, EarlyView.
Using peripheral blood for determining B‐cell or T‐cell clonality is more reliable when we use cell‐free RNA (cfRNA) because cells release blood significantly more RNA than DNA. Next‐generation sequencing (NGS) of cfRNA allows us to evaluate fragment cfRNA and evaluate clonality reliably without the need for prior determination of the specific dominant
Adam Albitar, Sally Agersborg, Ahmed Charifa, Pooja Phull, Noa Biran, David Vesole, Harsh Parmar, Andrew Pecora, Andrew Ip, Andre Goy, David Siegel, Maher Albitar   +11 more
wiley   +1 more source

Hepatitis-associated aplastic anemia from workout supplement: Rare but potentially fatal entity

SAGE Open Medical Case Reports, 2020
Hepatitis-associated aplastic anemia (HAAA) is a rare clinical syndrome characterized by bone marrow failure 1–3 months after development of hepatitis. Untreated, hepatitis-associated aplastic anemia has poor outcome and the mainstay of treatment remains
Sanjog Bastola, Ojbindra Kc, Sumesh Khanal, Alexandra Halalau   +3 more
doaj   +1 more source

The Lupus Damage Index Revision Program: Results From the Item Generation and Reduction Phases

Arthritis Care &Research, EarlyView.
Objective A data‐driven and expert/patient consensus‐based project to develop a revised Systemic Lupus International Collaborating Clinics (SLICC)/American College of Rheumatology (ACR) Damage Index (SDI) is under way supported by SLICC, ACR, and the Lupus Foundation of America. Our objective is to report the item generation and reduction phase results
Burak Kundakci, Megan R. W. Barber, Ann E. Clarke, Sindhu R. Johnson, Anselm Mak, Hermine I. Brunner, Murat Inanc, Rosalind Ramsey‐Goldman, Guillermo Ruiz‐Irastorza, Ellen M. Ginzler, John G. Hanly, Alexandra Legge, Cynthia Aranow, Laurent Arnaud, Nathalie Costedoat‐Chalumeau, Dafna D. Gladman, Søren Jacobsen, Michelle Petri, Clovis A. Silva, Évelyne Vinet, Alexandre E. Voskuyl, SDI Revision Literature Review Groups, SDI Revision Delphi Panel, SDI Revision Item Reduction Committee, SDI Revision Clinical Domain Groups, Ian N. Bruce   +25 more
wiley   +1 more source

Response of Paroxysmal Nocturnal Hemoglobinuria Clone with Aplastic Anemia to Rituximab

Case Reports in Hematology, 2012
Paroxysmal nocturnal hemoglobinuria is caused by expansion of a hematopoietic stem cell clone with an acquired somatic mutation in the PIG-A gene. This mutation aborts the synthesis and expression of the glycosylphosphatidylinositol anchor proteins CD55
Radha Raghupathy, Olga Derman
doaj   +1 more source