Results 101 to 110 of about 10,305 (260)

Eltrombopag Added to Standard Immunosuppressive Treatment as Front‐Line Therapy for Severe Aplastic Anemia: Long‐Term Outcomes of the Phase‐3 Randomized Superiority EBMT‐SAAWP RACE Study

American Journal of Hematology, EarlyView.
ABSTRACT The RACE study (NCT02009747) compared horse antithymocyte globulin (hATG) plus cyclosporine A (CsA) ± eltrombopag as initial immunosuppressive treatment (IST) for severe aplastic anemia. Here we report the final 2‐year analysis of this prospective randomized phase III study.
Antonio M. Risitano, Simona Iacobelli, Austin Kulasekararaj, Marleen van Os, Sofie R. Terwel, Joe Tuffnell, Brian Piepenbroek, Morag Griffin, Constantijn J. M. Halkes, Christian Recher, Fiorenza Barraco, Edouard Forcade, Juan Carlos Vallejo, Beatrice Drexler, Jean‐Baptiste Mear, Roochi Trikha, Shreyans Gandhi, Anna Maria Raiola, L. G. M. Daenen, Marco R. de Groot, Etienne Daguindau, Erfan Nur, Wilma Barcellini, Nigel H. Russell, Louis Terriou, Anna Paola Iori, Walter Barberi, Anna Sureda, Isabel Sánchez‐Ortega, Blanca Xicoy, Isidro Jarque, James Cavenagh, Flore Sicre de Fontbrune, Camilla Frieri, Talha Munir, Jennifer M. L. Tjon, Suzanne Tavitian, Aline Praire, Laurence Clement, Florence Rabian, Luana Marano, Anita Hill, Elena Palmisani, Petra Muus, Serena Marotta, Fabiana Cacace, Marica Laurino, Jakob R. Passweg, Gérard Socié, Ghulam J. Mufti, Carlo Dufour, Régis Peffault de Latour, Severe Aplastic Anaemia Working Party of the EBMT   +52 more
wiley   +1 more source

Cutaneous Manifestations of Inborn Errors of Immunity: Clinical Clues to Immune Disorders. [PDF]

Medicina (Kaunas)
Napiorkowska-Baran K, Pastuszczak M, Płocka-Karpińska M, Tykwińska M, Treichel P, Margossian GA, Margossian CL, Rogalska A, Czajkowski R.   +8 more
europepmc   +1 more source

Cardiovascular Toxicity Associated With Bispecific Antibodies in Hematological Malignancies: A Comprehensive Pharmacovigilance Analysis

American Journal of Hematology, EarlyView.
ABSTRACT Cardiovascular adverse events (CVAEs) associated with bispecific T‐cell engaging antibodies (BsAbs) have not been systematically investigated across approved agents. In this disproportionality analysis of FAERS (December 2014–September 2025), reports listing BsAbs as the primary suspected drug (n = 7647) were compared with all other drugs in ...
Malak Munir, Ahmed Sayed, Dae Hyun Lee, Sanam Ghazi, Mustafa M. Houmsse, Saad Badat, Abdul Moeed, Avirup Guha, Daniel Addison, Narendranath Epperla   +9 more
wiley   +1 more source

Vitiligo like depigmentation in a patient with NHEJ1 deficiency related radiosensitive SCID: A case report. [PDF]

JAAD Case Rep
Alhaider A, Almutairi M, Alomari A, Altuwaijri A, Almarshoud G, Alakrash L.   +5 more
europepmc   +1 more source

Systemic Bevacizumab for Severe Bleeding From Acquired Gastrointestinal Vascular Malformations

American Journal of Hematology, EarlyView.
Targeted antiangiogenic therapy with systemic bevacizumab for bleeding from acquired GI VMs was found to be safe and effective for chronic and severe bleeding from acquired vascular malformations due to idiopathic angiodysplasia, chronic liver disease, and deficiencies of von Willebrand factor, in a patient population comprised mostly of heavily ...
Nardeen E. Ayad, Jacob R. Anderson, Bimalangshu Dey, Rebecca Karp Leaf, Andrew B. Song, Hanny Al‐Samkari   +5 more
wiley   +1 more source

Atopic dermatitis reconsidered: Clinical mimics and diagnostic pearls. [PDF]

J Am Acad Dermatol
Lawrence CN, Karagounis TK, Cohen DE.
europepmc   +1 more source

The Homozygous p.(Arg215Ter) Variant in XRCC2 Is Associated With Atypical Fanconi Anemia Without Major Hematological Abnormalities in Childhood

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Fanconi Anemia (FA) is the most frequent inherited bone marrow failure syndrome. A role for the XRCC2 gene in FA was suspected in 2012 and confirmed in 2016, but only two affected individuals have been described thus far, and no long‐term follow‐up is available.
Sabina Cenciarelli, Giulia Bruna Marchetti, Maria Iascone, Maria Grazia Patricelli, Sara Giangiobbe, Gabriella Cinzia Pozzobon, Miriam Nella Savini, Fabio Giglio, Alessandro Aiuti, Paola Carrera, Francesca Ferrua, Angela Peron   +11 more
wiley   +1 more source

Opinion: Why paediatric rheumatologists need to understand inborn errors of immunity. [PDF]

Front Pediatr
Abinun M, Owens S.
europepmc   +1 more source

Defining Features of Gabriele‐de Vries Syndrome in Adults: A Case Report and Literature Review

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Gabriele‐de Vries syndrome (GADEVS) is a neurodevelopmental disorder caused by heterozygous pathogenic variants in the YY1 gene. Like most rare genetic syndromes, the adult manifestations of GADEVS remain poorly defined. Here, we describe the oldest patient reported to date with GADEVS—a 63‐year‐old woman with a c.1177_1179del YY1 variant ...
Ethan W. Hollingsworth, Changrui Xiao
wiley   +1 more source

Commentary: Anti-endothelial cell antibodies in pathogenesis of vasculitis. [PDF]

Front Immunol
Xie J, Shi L, Wang L, Dai J.
europepmc   +1 more source