Results 81 to 90 of about 39,204 (167)

HOPE Kids 2: Phase 3, Randomized Trial of Voxelotor in Children With SCD and Conditional Cerebral Blood Flow Velocities

American Journal of Hematology, EarlyView.
ABSTRACT Children with SCD have elevated stroke risk, correlated with cerebral blood flow velocity (CBFV). HOPE Kids 2 was a phase 3, multicenter, double‐blind, placebo‐controlled trial evaluating the effect of voxelotor on CBFV. Participants aged 2 to < 15 years with SCD (HbSS/HbSβ0) and conditional CBFV (170 to < 200 cm/s) were randomized 1:1 to ...
Halima Bello‐Manga, Shehu Umar Abdullahi, Hoda Hassab, Biobele Brown, Videlis Nduba, Jessie Githanga, Adeseye Michael Akinsete, Amal El‐Beshlawy, Diana Hanna, Catherine Segbefia, Iheanyi Okpala, Azza Tantawy, Bernhards Ogutu, Venée N. Tubman, Vivian Paintsil, Yasser Wali, Mohammed Essa, Paul Telfer, Sandy Dixon, Anne Hickman, Jennifer F. Doss, Mark W. Davis, Jeremy Rupon, Fenella J. Kirkham, R. Clark Brown, on behalf of the HOPE Kids 2 Investigators, Amal El‐Beshlawy, Mohamed Badr, Diana Hanna*, Hoda Hassab, Azza Tantawy, Vivian Paintsil, Catherine Segbefia, Raffaella Colombatti, Daniela Cuzzubbo, Silverio Perrotta, Jessie Githanga, Videlis Nduba, Bernhards Ogutu, Shehu Umar Abdullahi, Adeseye Michael Akinsete, Halima Bello‐Manga, Biobele Brown, Iheanyi Okpala, Yasser Wali, Mohammed Essa, Banu Kaya, Paul Telfer, Ofelia Alvarez, Natasha Archer, Shayla Bergmann, R Clark Brown, Andrew Campbell, Carlton Dampier*, Richard Hurwitz, Erin Jackson, Janet Kwiatkowski, Melissa M. McNaull*, Venée N. Tubman   +58 more
wiley   +1 more source

Targeting B‐Cell‐Activating Factor and Its Receptor in Immune Thrombocytopenia and Warm Autoimmune Hemolytic Anemia

American Journal of Hematology, EarlyView.
ABSTRACT Current treatments for immune thrombocytopenia (ITP) and warm autoimmune hemolytic anemia (wAIHA), rare autoimmune diseases in which autoreactive B cells play a major role, can lead to high response rates; however, for many patients these responses are not durable or maintained after treatment discontinuation.
Matthieu Mahévas, Nichola Cooper, Vickie McDonald, Karina Yazdanbakhsh, Wilma Barcellini   +4 more
wiley   +1 more source

PB2546: DIAGNOSTIC POWER OF ERYTHROCYTE AND RETICULOCYTE AUTOMATIC PARAMETERS IN THE SCREENING FOR CONGENITAL HEMOLYTIC ANEMIAS

HemaSphere, 2023
Paola Bianchi, Azita Sharif, Cristina Vercellati, Elisa Fermo, Maria Laura Chiaretto, Anna Paola Marcello, Anna Zaninoni, Nadi Mirra, Simona Torricelli, Virna De Zordi, Stefania Bottalico, Wilma Barcellini, Ferruccio Ceriotti, Anna Chiara Migliorini   +13 more
doaj   +1 more source

The Presence and Percentage of Circulating Nucleated Red Blood Cells Reveal Distinct Characteristics in Adults With Sickle Cell Disease

American Journal of Hematology, EarlyView.
ABSTRACT Although circulating nucleated red blood cells (cNRBCs) have previously been detected in patients with sickle cell disease (SCD), their prevalence and clinical significance during steady‐state disease have not been established. We retrospectively analyzed 270 adults with an SS or Sβ0‐thalassemia genotype.
Ugo Boccadifuoco, Ivan Lerner, Auria Godard, Luc Darnige, Laure Joseph, Wassim El Nemer, Geoffrey Cheminet, Jean‐Benoit Arlet   +7 more
wiley   +1 more source

Splenic Tropism and Spleen‐Modulated Systemic Inflammation in Acute Plasmodium vivax Malaria

American Journal of Hematology, EarlyView.
ABSTRACT In chronic Plasmodium vivax (Pv) infection, the spleen accounts for over 98% of total‐body parasite biomass. Whether splenic tropism also occurs in acute infection and how the spleen influences pathogenesis have not been systematically explored. In Papua, Indonesia, we compared clinical and hematology data in 24 spleen‐intact and 25 previously
Steven Kho, Hasrini Rini, Noy N. Kambuaya, Syawal Satria, Freis Candrawati, Putu A. I. Shanti, King Alexander, Benediktus Andries, Aisah R. Amelia, Anjana Rai, Kim A. Piera, Agatha M. Puspitasari, Ristya Amalia, Pak Prayoga, Leo Leonardo, Maulina Hafidzah, Theo Situmorang, Dewi S. Margayani, Desandra A. Rahmayenti, Pengxing Cao, Enny Kenangalem, Leily Trianty, Damian Oyong, Julie A. Simpson, Rintis Noviyanti, Pierre A. Buffet, Jeanne R. Poespoprodjo, Nicholas M. Anstey   +27 more
wiley   +1 more source

A rare variant of dyskeratosis congenita: RTEL1 defect

Hematology, Transfusion and Cell Therapy, 2020
C. Coskun, S. Unal, N. Akarsu
doaj   +1 more source

Cerebral Small Vessel Disease in Immune‐Mediated Thrombotic Thrombocytopenic Purpura Patients During the Acute Phase and Disease Remission

American Journal of Hematology, EarlyView.
ABSTRACT Immune‐mediated thrombotic thrombocytopenic purpura (iTTP) is a life‐threatening thrombotic microangiopathy characterized by acute neurological manifestations and long‐term sequelae related to microvascular brain injury. Cerebral small vessel disease (cSVD), detectable on MRI through lacunes, microbleeds, white matter hyperintensities, and ...
Addolorata Truma, Francesco Maria Lo Russo, Giorgio Conte, Ilaria Mancini, Andrea Artoni, Juri Alessandro Giannotta, Barbara Ferrari, Pasquale Agosti, Maria Abbattista, Matteo Gagliardi, Eleonora Piccin, Marco Stroppi, Fabio Maria Triulzi, Flora Peyvandi   +13 more
wiley   +1 more source

Severe ADEM‐Like Neuroinflammatory Disease and Cerebrovascular Fragility With Recurrent Pseudoaneurysms and Moyamoya in a Familial Germline CBL Mutation: Expanding the Clinical Phenotype

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Heterozygous germline variants in CBL disrupt its function as a negative regulator of the Ras/MAPK pathway, classically predisposing to Juvenile myelomonocytic leukemia (JMML) and moyamoya. We describe two affected siblings carrying a paternally inherited CBL variant (c.1210 T> C, p.
Michal Bar‐Hakim, Anat Abramovich, Sara Via‐Dorembus, Gali Heimer, Bruria Ben‐Zeev, Etai Adam, Assaf A. Barg, Jonathan Roth, Basheer Sheick‐Yousif, Rony Beeri Berkowiz, Michal Feldon, Haim Bassan, Moran Hausman‐Kedem   +12 more
wiley   +1 more source

Viral Hepatitis B/C Co-Infection and Its Association With Haematological and Virological Parameters in HIV Patients in Northern Ghana

Anemia
Human immunodeficiency virus (HIV) and viral hepatitis B (HBV) and C (HCV) share common routes of transmission and increase the morbidity and mortality of infected patients.
Uzzah Mohammed Forgor, George Doopaar Billak, Nsoh Godwin Anabire, Gideon Kofi Helegbe   +3 more
doaj   +1 more source

Concurrent Germline RB1 & Mosaic TP53 in a Child With Multiple Childhood Cancers

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT We report a patient with a pathogenic germline variant (PGV) in RB1 and somatic mosaicism for a pathogenic TP53 variant who developed three distinct types of childhood cancer: retinoblastoma, osteosarcoma, and myelodysplastic syndrome (MDS) before the age of 6 years.
Ole Haubjerg Nielsen, Ulrik Kristoffer Stoltze, Pernille Axél Gregersen, Marianne Hoffmann, Morten Lyng Høgild, Mette Klarskov Andersen, Lise Barlebo Ahlborn, Lisa Lyngsie Hjalgrim, Karin A. W. Wadt   +8 more
wiley   +1 more source