Results 131 to 140 of about 3,873 (265)

NOVEL THERAPEUTIC APPROACHES IN THALASSEMIAS, SICKLE CELL DISEASE AND OTHER RED CELL DISORDERS

A deeper understanding of the pathophysiology of hereditary red cell disorders and the development of novel classes of pharmacologic agents in this last decade have provided novel therapeutic approaches to thalassemias, sickle cell disease (SCD) and ...
Lucia De Franceschi, Filippo Mazzi, Valeria Maria Pinto   +2 more
core  

A Multicenter, Open‐Label, Phase 2 Trial Comparing Crizanlizumab Combined With Standard Therapy to Standard Therapy Alone on Renal Function in Patients With Sickle Cell Nephropathy (STEADFAST)

American Journal of Hematology, Volume 101, Issue 10, Page 2666-2671, October 2026.
Kenneth I. Ataga, Pablo Bartolucci, Santosh L. Saraf, Vimal K. Derebail, Adlette Inati, Jeffrey D. Lebensburger, Laurie DeBonnett, Rodolfo D. Cancado, Claire C. Sharpe   +8 more
wiley   +1 more source

Inpatient Exposure, Confidence, and Knowledge in Pediatric Hematology/Oncology: Evaluating General Pediatric Residents During 2025 ACGME Curriculum Change

Pediatric Blood &Cancer, Volume 73, Issue 9, September 2026.
ABSTRACT Background General pediatricians often evaluate hematologic and oncologic presentations before subspecialty consultation, yet the 2025 Accreditation Council for Graduate Medical Education (ACGME) pediatric requirements reduce inpatient pediatric hematology/oncology (PHO) time, raising questions about resident readiness.
Colburn Yu, Rohini Jain
wiley   +1 more source

Synergistic HMGN1 and VP64 Fusions Potentiate High‐Precision and PAM‐Flexible Base Editing

Advanced Science, Volume 13, Issue 50, 7 September 2026.
A novel CDA1Δ‐SpRY architecture fused with HMGN1 and VP64 yields a nearly PAM‐less base editing platform. By focusing cytosine conversion predominantly at position −18, this synergistic complex ensures highly precise targeting. Demonstrating enhanced efficiency across diverse models, including yeast and rice, the platform offers a robust solution for ...
Xi Luo, Yuyao Qu, Zhengyan Ye, Zheng Li, Yuanyan Zhang, Longjiong Luo, Shaokang Li, Wei Zhao, Ming Wang, Ralph Bock, Jianmin Wan, Junjie Tan   +11 more
wiley   +1 more source

Therapeutic Silencing of Tmprss6 Reduces Iron‐Induced Inflammation and Prolongs Survival in MDS Mice

American Journal of Hematology, Volume 101, Issue 9, Page 2105-2122, September 2026.
ABSTRACT Myelodysplastic syndromes (MDS) are a heterogeneous group of clonal hematopoietic disorders characterized by ineffective hematopoiesis, cytopenias, and an increased risk of progression to acute myeloid leukemia (AML). Despite advances in supportive and targeted therapies, disease‐modifying interventions remain limited.
Shahla Vilcassim, Nuttanan Pholngam, Rattanawan Thubthed, Tiwaporn Nualkaew, Saovaros Svasti, Pornthip Chaichompoo, Kai Kysenius, Peter J. Crouch, Sibylle Dames, Mona Eisermann, Alberto Martinez, Ute Schaeper, Jim Vadolas, George Grigoriadis   +13 more
wiley   +1 more source

Talassemias alfa Alpha thalassemias

Revista Brasileira de Hematologia e Hemoterapia, 2006
Rodolfo D. Cançado
doaj   +1 more source

Myelodysplastic Syndromes: 2026 Update on Diagnosis, Risk‐Stratification and Management

American Journal of Hematology, Volume 101, Issue 9, Page 2393-2411, September 2026.
ABSTRACT Disease Overview The myelodysplastic syndromes (MDS) are a heterogeneous group of myeloid disorders characterized by peripheral blood cytopenias and increased risk of transformation to acute myelogenous leukemia (AML). MDS occurs more frequently in older males and in individuals with prior exposure to cytotoxic therapy.
Guillermo Garcia‐Manero
wiley   +1 more source

Undetectable Hydroxyurea Levels in the Majority of Sickle Cell Disease Patients, Especially in Young Children

American Journal of Hematology, Volume 101, Issue 9, Page 2297-2310, September 2026.
ABSTRACT Hydroxyurea (HU) is the most widely prescribed disease‐modifying treatment in sickle cell disease (SCD), though treatment responses vary due to metabolism and adherence. We examined HU blood levels and treatment response in patients with homozygous sickle cell disease (HbSS).
Sigrid van der Veen, Bart J. Biemond, Marjon H. Cnossen, Pablo Bartolucci, Maria P. Boaro, Betzabel Cajiao Garcia, Raffaella Colombatti, M. D'Agnolo, Karin Fijnvandraat, Anna Collado Gimbert, Amira Idrizovic, Petros Kountouris, Mar Mañú‐Pereira, Elisabetta Mezzalira, Mariane de Montalembert, Erfan Nur, A. Pham Hung D' Alexandry D'Orengiani, Tiziana Sanavia, Saskia E. M. Schols, Marissa J. M. Traets, Minke A. E. Rab, Sara I. Reidel, Anita W. Rijneveld, Yosr Zaouali, Nanda M. Verhoeven‐Duif, Judith J. M. Jans, Eduard J. van Beers   +26 more
wiley   +1 more source

The shifting global thalassemia burden and lessons from Chineseintegrated control strategy. [PDF]

iScience
Jin C, Hu Z, Li Y, Lin Q, Yu J, Zhang Q, Luo M, Yang C, Zhou X, Wen F, Jin W, Liu S.   +11 more
europepmc   +1 more source

Severe Multiorgan Failure Triggered by Infection in an Adult With Decades of Untreated Sickle Cell Disease: A Case Report

Clinical Case Reports, Volume 14, Issue 9, September 2026.
ABSTRACT A 67‐year‐old man with decades of untreated sickle cell disease developed septic shock with multiorgan failure, splenic infarction, and marrow fibrosis; his genotype could not be confirmed. He recovered with intensive supportive care, with hydroxyurea initiated for long‐term disease modification, illustrating catastrophic infection‐triggered ...
Sara Shahidi, Gisha Mohan
wiley   +1 more source