Results 31 to 40 of about 5,736 (192)

A pilot trial of deferiprone in pantothenate kinase-associated neurodegeneration patients

Neurology International, 2018
Pantothenate kinase-associated neurodegeneration (PKAN) is the most common form of neurodegeneration with brain iron accumulation, it is an autosomal recessive disease due to mutation in PANK 2 on chromosome 20, which causes the accumulation of iron in ...
Mohammad Rohani, Saeed Razmeh, Gholam Ali Shahidi, Maryam Orooji   +3 more
doaj   +1 more source

Comparison of oral iron chelators in the management of transfusion-dependent β-thalassemia major based on serum ferritin and liver enzymes [version 2; peer review: 2 approved]

F1000Research, 2023
Background Excess iron deriving from a chronic transfusion and dietary intake increases the risk for cardiac complications in β-thalassemia major patients.
Khairunnisa Khairunnisa, Heru Noviat Herdata, Eka Destianti Edward, Sulaiman Yusuf   +3 more
doaj   +1 more source

Correction of Ineffective Erythropoiesis and Normalization of Iron Homeostasis After Exagamglogene Autotemcel in Transfusion‐Dependent β‐Thalassemia

American Journal of Hematology, EarlyView.
ABSTRACT Exagamglogene autotemcel (exa‐cel) is a one‐time, ex vivo, CRISPR‐Cas9 gene edited cell therapy approved for patients with transfusion dependent β‐thalassemia (TDT) aged 12–35 years. In a Phase 3 study (CLIMB THAL‐111), exa‐cel treatment resulted in reactivation of fetal hemoglobin and increases in total hemoglobin, leading to transfusion ...
Sujit Sheth, Selim Corbacioglu, Josu de la Fuente, Mattia Algeri, Joachim Rupprecht, Kevin H. M. Kuo, Ami J. Shah, Peter Lang, Hayley Merkeley, Ben Carpenter, Markus Y. Mapara, Robert I. Liem, Stephan Grupp, Yogi Chopra, Amanda M. Li, Janet L. Kwiatkowski, Melanie Kirby‐Allen, Maria Domenica Cappellini, Antonis Kattamis, Sakellarios Zairis, Tina Liu, William Hobbs, Haydar Frangoul, Franco Locatelli, Roland Meisel, on behalf of the CLIMB THAL‐111 and CLIMB‐131 Study Groups   +25 more
wiley   +1 more source

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

American Journal of Hematology, EarlyView.
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

Trial of Deferiprone in Parkinson\u27s Disease

, 2022
\ua9 2022 Massachusetts Medical Society. Background Iron content is increased in the substantia nigra of persons with Parkinson\u27s disease and may contribute to the pathophysiology of the disorder.
Ory-Magne F, Dexter DT, Labreuche J, Spino M, Maetzler W, Mangin J-F, Poewe W, Ouk T, Otto M, Chupin M, Thobois S, Lopes R, Pruvo J-P, Deplanque D, Ruzicka E, Fradette C, Cabantchik I, Habert M-O, Berg D, Bordet R, Kuchcinski G, Duhamel A, Moreau C, Gago M, Tranchant C, Nyholm D, Coelho MVS, Lehericy S, Behnke S, Garcon G, Devedjian J-C, Walter U, Tricta F, Compta Y, Post B, Corvol J-C, Rolland A-S, Worth P, Pavese N, Viard R, Marques A-R, Bush AI, Devos D, Carpentier J, Leclercq C, Kulisevsky J, Duce JA, Simonin O, Pigny P, Scherfler C, Potey C, Meissner WG, Ayton S, Rascol O, Dusek P, Ferreira J, De Bie RMA, Vilas D, Defebvre L, Dodel R, Eusebio A, Delannoy PG, Januario C, Bloem BR, Seppi K   +64 more
core  

Population Genomics Insights into Pharmacogenomic Differentiation Between East Asians and Europeans

Clinical Pharmacology &Therapeutics, EarlyView.
Genetic variation contributes substantially to interindividual and interpopulation differences in drug response, yet most pharmacogenomic studies remain biased toward European populations. Here, we systematically assessed pharmacogenomic variation across East Asians (EAS) and Europeans (EUR) using public genomic datasets and investigated the potential ...
Sihan Chen, Hongpu Chen, Shuhua Xu
wiley   +1 more source

Antiplatelet activity of deferiprone through cyclooxygenase-1 inhibition

, 2019
Thalassemia patients are susceptible to both iron overload and thromboembolism. Deferiprone is an iron chelator that shows an antiplatelet activity and thus may alleviate platelet hyperactivation in thalassemia.
Benjaporn Akkawat (7111757), Ngan Thi Tran (7111754), Ponlapat Rojnuckarin (4693228), Noppawan Phumala Morales (7111760), Rataya Luechapudiporn (7111763)   +4 more
core   +1 more source

Beta‐Thalassemia in Spain: Results From the National Thalassemia Registry and Molecular Analysis of Patients With Transfusion‐Dependent Thalassemia

Journal of Clinical Laboratory Analysis, EarlyView.
This nationwide registry‐based study describes the clinical and molecular characteristics of 78 patients with transfusion‐dependent beta‐thalassemia in Spain. Comprehensive genetic analysis revealed marked molecular heterogeneity, with 24 HBB mutations identified and a predominance of β0 genotypes, alongside generally effective transfusion and iron ...
Ana Villegas, Paloma Ropero, Fernando Ataulfo González, Marta Morado, María Menor, Silvia de la Iglesia, Montserrat López‐Rubio, Eduardo José Salido, Mariola Abío, María Leonor Senent, Valle Recasens, José Manuel Vagace, Cristina Fonte, Jesús María Hernández‐Rivas, José María Raya, Pablo Ríos, Valeria Luciana Peri, Keneth A. Herrera, María Concepción Tenorio, Laura Lo Riso, Juan Tena, María Angustias Molina, Miriam Vara, Isabel Rodríguez, Pilar Ricard, Miguel Gómez, Oana Popa Dusacovschi, Julia María Vidán, Jorge Groiss, Óscar Ferre, Irene Orbe, Rosa María López, Nazaret Ugalde, Manuela Inés Hidalgo, Cristina Hinojosa, Ana Isabel Rodríguez, Esther Herrera, María Josefa Murúzabal, María del Carmen Hernández, Josefa Esperanza Marco, Celina Benavente, Grupo Español de Eritropatología   +41 more
wiley   +1 more source

Safety and Efficacy of Iron Chelators in Patients With Chronic Kidney Disease: A Systematic Review and Meta‐Analysis

Hemodialysis International, EarlyView.
ABSTRACT Background Iron overload in chronic kidney disease contributes to oxidative injury. The role of iron chelators, standard in transfusion‐related overload, remains unclear in chronic kidney disease. Aim To evaluate the efficacy and safety of iron chelators in adults with chronic kidney disease.
Humam Emad Rajha, Richard Shamoon, Ameer Almeshagbeh, Samah Masry, Fadel Alqatati, Baha H. Abuajameia, Abdulqadir J. Nashwan   +6 more
wiley   +1 more source

Prevention of Iron Overload and Long Term Maintenance of Normal Iron Stores in Thalassaemia Major Patients using Deferiprone or Deferiprone Deferoxamine Combination

, 2017
Decrease in mortality and morbidity is observed in thalassaemia major patients with reduced iron load in comparison to heavy iron loaded patients. Effective and complete treatment of transfusional iron overload can be achieved by chelation protocols that
Christina Kontoghiorghe, Annita Kolnagou, George Kontoghiorghes   +2 more
core   +1 more source