Article
Medicine, General & Internal
Raffaella Origa, Monia Cinus, Maria Paola Pilia, Barbara Gianesin, Antonietta Zappu, Valeria Orecchia, Maria Grazia Clemente, Carla Pitturru, Anna Rita Denotti, Francesco Corongiu, Simona Piras, Susanna Barella
Summary: The study evaluated the safety and efficacy of long-term combination therapy of deferasirox plus desferrioxamine and deferasirox plus deferiprone in a large group of transfusion-dependent thalassemia patients. Both combinations were effective in reducing serum ferritin and liver iron concentration, but patient adherence to the two drugs was low.
JOURNAL OF CLINICAL MEDICINE
(2022)
Review
Multidisciplinary Sciences
Yesim Aydinok
Summary: Combination chelation therapy is a viable option for transfusion-dependent thalassemia patients who have not achieved iron balance with monotherapy or need intensified treatment for rapid iron reduction. It allows for a more personalized approach, improving tolerability, adherence, and quality of life. Real-life data highlights the importance of compliance in achieving meaningful reduction in iron overload compared to monotherapy.
ANNALS OF THE NEW YORK ACADEMY OF SCIENCES
(2023)
Review
Medicine, General & Internal
Antonis Kattamis, Janet L. Kwiatkowski, Yesim Aydinok
Summary: Thalassaemia is a genetic disorder that affects globin chain synthesis and has a worldwide distribution. The unbalanced production of globin chains leads to ineffective erythropoiesis, increased haemolysis, and disrupted iron homeostasis. The clinical phenotype varies from mild to severe, with treatment options including transfusion, iron chelation, allogeneic haematopoietic stem cell transplantation, and gene therapy.
Article
Hematology
John Chapin, Alan R. Cohen, Ellis J. Neufeld, Elliott Vichinsky, Patricia J. Giardina, Jeanne Boudreaux, Binh C. Le, Kristy Kenney, Sean Trimble, Alexis A. Thompson
Summary: Thalassaemia is a genetic disorder causing anaemia and dependence on transfusions, with recent clinical advancements leading to reduced mortality and potential future gene therapies. Research revealed older patients with more iron-related comorbidities and complications, while younger patients had excess liver and heart iron and were on combination chelation therapy.
BRITISH JOURNAL OF HAEMATOLOGY
(2022)
Article
Chemistry, Multidisciplinary
Pritam Kumar Pain, Dipanwita Palit, Meenakshi Shegane, Rajnish Pratap Singh, Debasish Manna
Summary: Copper can be both harmful and essential, and its concentration needs to be precisely controlled within cells. We have developed a system using a photocaged chelator and light to regulate cellular copper concentration. This method allows us to control copper levels in cells in a spatiotemporal manner.
CHEMICAL COMMUNICATIONS
(2023)
Review
Chemistry, Medicinal
George J. Kontoghiorghes
Summary: The iron chelating orphan drug deferiprone (L1), which has been used daily by patients at high doses for over 30 years with no serious toxicity, has unique properties that have played a major role in transforming thalassaemia from a fatal to a chronic disease. These properties include oral effectiveness, highly effective iron removal from organs, rapid absorption and clearance from the body, and the ability to cross the blood-brain barrier. Additionally, L1 exhibits specific protein interactions, antioxidant effects, and the ability to inhibit iron-containing proteins associated with various pathological conditions.
Article
Hematology
Mohsen S. Elalfy, Mona Hamdy, Amira Adly, Fatma S. E. Ebeid, Noemi Toiber Temin, Anna Rozova, David Lee, Caroline Fradette, Fernando Tricta
Summary: Traditionally, iron-chelation therapy for children with transfusion-dependent thalassemia was delayed until iron overload occurred. This study investigated the use of early-start deferiprone in infants and young children to reduce iron load, and found it to be effective, well-tolerated, and not associated with iron depletion in this population.
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Article
Chemistry, Multidisciplinary
Mateusz Pawlaczyk, Grzegorz Schroeder
Summary: Deferoxamine was successfully immobilized on three different supports, leading to materials with high adsorption capacity for Fe(III) ions. The adsorption processes were intense, endothermic, and spontaneous, showing the high adsorptive potential of the DFO-functionalized materials.
Article
Hematology
Denis Soulieres, Jules Mercier-Ross, Caroline Fradette, Anna Rozova, Yu Chung Tsang, Fernando Tricta
Summary: This study characterized the pharmacokinetic profile of deferiprone in adult patients with sickle cell disease (SCD). The results showed that deferiprone had similar metabolism and excretion characteristics in SCD patients compared to healthy volunteers, suggesting no special dosing adjustments are needed for this population.
ANNALS OF HEMATOLOGY
(2022)
Article
Clinical Neurology
Yurie Nose, Ikuko Uwano, Ukihide Tateishi, Makoto Sasaki, Takanori Yokota, Nobuo Sanjo
Summary: Deferiprone administration significantly improved radiological and clinical outcomes in patients with postoperative superficial siderosis. Earlier and longer courses of deferiprone could result in better patient prognosis.
JOURNAL OF NEUROLOGY
(2022)
Article
Chemistry, Medicinal
Martina Bortolami, Fabiana Pandolfi, Antonella Messore, Daniele Rocco, Marta Feroci, Roberto Di Santo, Daniela De Vita, Roberta Costi, Paola Cascarino, Giovanna Simonetti, Luigi Scipione
Summary: In specific conditions, certain derivatives of deferiprone show potential in inhibiting the growth and biofilm formation of Candida albicans, with low toxicity and interesting activity against planktonic cells. Additionally, these compounds exhibit chelating abilities with Fe(III) and Cu(II), suggesting a possible mechanism for their antifungal activity.
BIOORGANIC & MEDICINAL CHEMISTRY LETTERS
(2021)
Article
Biochemistry & Molecular Biology
Athina G. Mantelou, Alexandra Barbouti, Anna Goussia, Argyro Zacharioudaki, Alexandra Papoudou-Bai, Chara Vlachou, Stelios Kokkoris, Apostolos Papalois, Dimitrios Galaris, Georgios K. Glantzounis
Summary: The study showed that a combination of iron chelators with varying lipophilicity and iron-binding properties can enhance protection against hepatic I/R injury. Through investigations in cell lines and a rabbit model, the protective effects of these iron chelator combinations were observed.
FREE RADICAL BIOLOGY AND MEDICINE
(2022)
Review
Biochemistry & Molecular Biology
Alessandra Di Paola, Chiara Tortora, Maura Argenziano, Maria Maddalena Marrapodi, Francesca Rossi
Summary: Iron is a crucial element for mammalian cells and its alteration could lead to the onset of disorders. Iron chelation has been proposed as a novel therapeutic strategy to counteract inflammation in autoimmune and inflammatory diseases. Several iron chelators, such as deferiprone, deferoxamine, deferasirox, and Dp44mT, have anti-inflammatory effects. Eltrombopag has been reported to reduce inflammation by chelating iron and modulating iron efflux.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Oncology
Mona Hamdy, Amal El-Beshlawy, Monica P. A. Verissimo, Julie Kanter, Baba Inusa, Suzan Williams, David Lee, Noemi Toiber Temin, Caroline Fradette, Fernando Tricta, Fatma S. E. Ebeid, Janet L. Kwiatkowski, Mohsen S. Elalfy
Summary: This post hoc analysis of pediatric patients from the FIRST study confirms that deferiprone is comparable to deferoxamine in reducing iron overload, and it could improve adherence and outcomes in children.
PEDIATRIC BLOOD & CANCER
(2023)
Article
Pediatrics
Rajeswari Rethinaswamy DivakarJose, C. G. Delhikumar, G. Ram Kumar
Summary: The study aimed to evaluate the efficacy and safety of dual oral iron chelation therapy in reducing iron overload in transfusion-dependent thalassemic children. The results showed a significant decrease in serum ferritin levels with the combination therapy, while liver and myocardial iron concentrations did not change significantly. Adverse effects such as red color urine and transient elevation in creatinine and liver enzymes were observed during the study period.
INDIAN JOURNAL OF PEDIATRICS
(2021)
