deferiprone / Generic mfg. - LARVOL DELTA

Deferiprone therapy improves the oxidative status of LDL in patients with β-thalassaemia/HbE. (PubMed, Drugs Context) - "LDL from the washout period showed the highest oxidative susceptibility when evaluated by NBD-Pen. Iron chelation therapy with L1 improves the oxidative status of LDL in patients with β-thalassaemia/HbE."

Journal • Beta-Thalassemia • Cardiovascular • Dyslipidemia • Hematological Disorders

A Cross-Sectional Study on Pain and Quality of Life of Adult Patients with Transfusion-Dependent Thalassemia in a Tertiary Hospital In Malaysia. (PubMed, Hemoglobin) - "No variable was independently associated with higher QoL, regardless at the age of diagnosis, baseline ferritin, severe MRI T2* liver results, taking oral deferiprone or deferasirox. With the identification of affecting variables, appropriate treatment plan may be formulated to reduce pain and improve the QoL of thalassemia patients."

HEOR • Journal • Observational data • Beta-Thalassemia • Genetic Disorders • Musculoskeletal Diseases • Orthopedics • Pain

FAIR-ALS II: Conservative Iron Chelation as a Disease-modifying Strategy in Amyotrophic Lateral Sclerosis (clinicaltrials.gov) - P2/3 | N=372 | Completed | Sponsor: University Hospital, Lille | Active, not recruiting ➔ Completed

Trial completion • Alzheimer's Disease • Amyotrophic Lateral Sclerosis • CNS Disorders

Iron Stress Reprograms Enterocyte Metabolism. (PubMed, Metabolites) - " Cells were treated with deferiprone (DFP) or ferric ammonium citrate (FAC) to induce ID or IE, respectively... ID impaired enterocyte proliferation and profoundly disrupted cellular metabolism, whereas IE enhanced cholesterol synthesis and depleted alpha-tocopherol levels. Restoration of cellular metabolism following iron repletion was observed, highlighting the resilience of enterocytes."

Journal • Hematological Disorders • Inflammation • Inflammatory Bowel Disease • CXCL8 • CYBRD1 • TLR4

Plasma protein biomarkers of traumatic brain injury in the lateral fluid percussion injury model: a multicenter study (AES 2025) - "LFPI rats also received treatments that are either used clinically in the acute post-TBI period, e.g. levetiracetam (LEV), or modify iron load from hemorrhages, i.e. the iron chelator deferiprone (DEF). Plasma GFAP, S100B, UCHL1 levels show biphasic responses after LFPI with an acute decrease at 2hr and subsequent increases, more consistent for GFAP (1-7d) and only transient (2d) for S100B and UCHL1. Overall, the temporal trajectories of these plasma biomarker levels after LFPI differ from the changes reported after human TBI. Establishing the temporal profile of changes helps to understand and address the differences between experimental modeling and clinical reality."

Biomarker • Clinical • Cerebral Hemorrhage • CNS Disorders • Epilepsy • Hematological Disorders • Vascular Neurology • GFAP • S100B • UCHL1

Transcriptomic Signature and PROTAC Strategy Revealed Histone Lysine Demethylase as a Target of Anticancer Activity of Deferiprone. (PubMed, bioRxiv) - "Moreover, DFP-derived PROTACs elicited enhanced cancer cell selective antiproliferative activities and intracellular on-target effects, downregulating several KDMs implicated in the etiology of BCa cells, including a strong degradation of KDMs 2A, 3A and 5B, and a moderate degradation of KDMs 4A-C, 5C, 6B. Collectively, our data supports KDM inhibition as a key mechanism of anticancer activity of DFP and identifies PROTAC is a viable strategy to obtain novel DFP analogs with improved potency and therapeutic index."

Journal • Breast Cancer • Genetic Disorders • Hematological Disorders • Oncology • Solid Tumor • Targeted Protein Degradation • HIF1A

Phase I trial to evaluate safety of Deferiprone-Chitogel to prevent epidural fibrosis in lumbar spine surgery. (PubMed, Eur Spine J) - No abstract available

Journal • P1 data • Fibrosis • Immunology • Orthopedics

Administration of green tea polyphenols mitigates iron-overload-induced bone loss in a β-thalassemia mouse model. (PubMed, NPJ Sci Food) - "This study assessed the effects of GTE on bone health in β-thalassemia knockout mice subjected to 4 weeks of iron dextran injections, followed by 2 months of daily oral treatment with deionized water, deferiprone (50 mg/kg), GTE (50 mg EGCG/kg), the combination of deferiprone and GTE, EGCG (50 mg/kg), or vitamin D3 (0.5 μg/kg ). GTE treatment reduced systemic iron burden, malondialdehyde, alkaline phosphatase, and parathyroid hormone, while improving femoral microarchitecture, bone mineral density, plasma calcium, and bone morphogenetic protein expression. These findings suggest GTE protects against iron-induced bone loss through combined chelation and antioxidation, supporting its potential as a therapeutic strategy."

Journal • Preclinical • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • Osteoporosis • Rheumatology

Iron Chelation Efficiency in Human Hepatocytes Is Enhanced By Exogenous Hepcidin (ASH 2023) - "Here we have examined whether application of exogenous hepcidin to a human hepatocyte cell line HepG2 can increase the chelation efficiency of deferiprone and whether this effect is also seen with other iron chelators such as deferasirox and deferoxamine. Two mechanisms for enhanced chelation are possible in this model; the first is by increased iron loading of hepatocytes incubated with hepcidin and secondly through the blocking by hepcidin of labile intracellular iron egress through ferroportin channels, thereby increasing the magnitude of chelatable intracellular iron pools. Future work in primary human hepatocytes will examine whether manipulation of intracellular hepcidin levels by SLN124 has similar chelation-enhancing effects."

Beta-Thalassemia • Genetic Disorders • Hematological Disorders • TMPRSS6

Exploring Novel Therapeutic Avenues: Drug Repurposing for Neurodegenerative Movement Disorders. (PubMed, Curr Drug Res Rev) - "Several studies on the potential of pre-existing drugs such as isradipine, tetracycline, ambroxol, metformin, deferiprone, simvastatin, etc., which have been repurposed for neurodegenerative movement disorders, including Parkinson's disease, Huntington's disease, Alzheimer's disease, Multiple Sclerosis, etc. have been discussed. Further, the current scenario and future prospective of drug repurposing have also been touched upon."

Journal • Review • Alzheimer's Disease • Amyotrophic Lateral Sclerosis • CNS Disorders • Huntington's Disease • Movement Disorders • Multiple Sclerosis • Parkinson's Disease

Lipid nanoparticle-mediated delivery of CP mRNA to reverse aceruloplasminemia phenotype in a mouse model (Neuroscience 2025) - "The treatments for ACP primarily relies on oral iron chelators, such as deferasirox and deferiprone...We establish an ACP mouse model to evaluate the therapeutic effects of using a lipid nanoparticle (LNP)-delivered CP mRNA in vivo. We expect to see an exogenous ceruloplasmin expression to at least partially reverse the pathological and phenotypic manifestations of ACP."

Preclinical • CNS Disorders • CP • FTL

Intranasal deferiprone ameliorates behavioral abnormalities in mice with brain iron loading (Neuroscience 2025) - "IN DFP may provide a non-invasive, brain-targeted alternative to oral administration for CNS conditions such as neuropsychiatric disorders. Further studies are needed to elucidate the underlying mechanisms and assess the translational potential of IN DFP in treating neuropsychiatric conditions."

Preclinical • CNS Disorders • Mental Retardation • Psychiatry • FAP • GFAP

The novel antioxidant iron chaperone ATH434 suppresses heme-induced oxidative stress in glutamatergic HT22 neurons (Neuroscience 2025) - "In a menadione-induced oxidative stress model in HT22 neurons with mitochondrial superoxide production, ATH434 rescued deficits in mitochondrial membrane potential while deferiprone (DFP), a strong ferric iron chelator did not. Hemin-induced saturation of the labile iron pool was evident at 6 h. The dose-dependent susceptibility of neurons to hemin-induced lipid peroxidation support this as a cellular model for damage induced by intracranial hemorrhage or microbleeds. ATH434's novel iron chaperone and antioxidant activities show potential for neuroprotective therapeutic activity after neurovascular injury."

Oxidative stress • Cerebral Hemorrhage • CNS Disorders • HMOX1

Identification of Lung Adenocarcinoma Subtypes Based on Nicotinamide Metabolism-Related Genes to Assess Prognosis and Immunotherapy. (PubMed, J Environ Pathol Toxicol Oncol) - "We successfully developed a predictive model for the survival and immunotherapy response of LUAD patients."

Journal • Lung Adenocarcinoma • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor

RBC Exchange Transfusion As an Adjunct Therapy to Control Iron Overload in Patients with Transfusion-Dependent Thalassemia (ASH 2023) - "Deferoxamine, deferiprone, and deferasirox are the iron chelators currently licensed for clinical use to manage iron overload in TDT patients with side effects ranging from nausea to nephrotoxicity and hearing loss. Blood utilization increased after transitioning to RBCX. Next steps in evaluating our practice for the patient group will be to evaluate iron deposition status with MRI T2* imaging."

Clinical • Acute Kidney Injury • Cardiovascular • Genetic Disorders • Hematological Disorders • Otorhinolaryngology • Pulmonary Arterial Hypertension • Pulmonary Disease • Renal Disease • Respiratory Diseases • Sickle Cell Disease

Overcoming the Cost Barrier for Thalassemia Innovative Treatments (ASH 2023) - "The direct cost of oral iron chelators, Deferiprone and Deferasirox, was updated, because chelator patents expired (Tab... The cost of TDT patient in WPFHS decreased over the last years, because of the oral iron chelator patents expiring. This cost changes according to age, because of the different incidence of complications, increasing from € 1. 035."

Beta-Thalassemia • Genetic Disorders • Hematological Disorders • Pediatrics

Utilization Patterns and Outcomes from Iron Chelation in Elderly Patients with Low-Risk Myelodysplastic Syndrome (ASH 2023) - "The initial agent of choice was deferasirox in 203 (51.7%), deferoxamine in 188 (47.8%), and deferiprone in 2 (0.5%)...Chelated patients were younger (median age 79 vs 81, p<0.01), more likely to have histology with ringed sideroblasts [RS] (17.8% vs 7%, p<0.01), had less comorbid burden (median CCI 1 vs 2, p<0.01), and were more frequently treated with ESA (42.8% vs 33%, p<0.01), HMA (59% vs 49.8%), and lenalidomide (27% vs 11.1%, p<0.01), than those not treated with ICT... Our results suggest that ICT is associated with fewer transfusions, improved OS, lower risk of AML, and less adverse cardiac outcomes in LR-MDS. Our older, unselected cohort better reflects real-world LR-MDS patients and builds upon OS benefit reported in more restricting settings. Given the link between clonal hematopoiesis, cardiovascular disease and LR-MDS, defining ICT's effect on molecular and clonal dynamics could clarify the mechanism behind improved cardiac outcomes."

Clinical • Acute Myelogenous Leukemia • Atrial Fibrillation • Cardiovascular • Congestive Heart Failure • Coronary Artery Disease • Heart Failure • Hematological Disorders • Hematological Malignancies • Hepatology • Immunology • Leukemia • Myelodysplastic Syndrome • Oncology

Analysis of the Risk of Infection Stratified By Absolute Neutrophil Counts during Deferiprone-Associated Severe Neutropenia or Agranulocytosis (ASH 2023) - "Conclusion Patients receiving deferiprone who become neutropenic are generally at a higher risk of infections or mortality with lower neutrophil levels (ie, ANC <0.2 or <0.1 x 109/L compared with ANC ≥0.2 to <0.5 x 109/L). Our findings are consistent with the recently published guidelines from the European Hematology Association and the European Network for Innovative Diagnosis and Treatment of Chronic Neutropenias that used agranulocytosis to describe events with ANC of ≤0.2 x 109/L in association with a high risk of severe and life-threatening infections."

Agranulocytosis • Anemia • Candidiasis • Genetic Disorders • Granulocytopenia • Hematological Disorders • Herpes Simplex • Infectious Disease • Neutropenia • Pneumonia • Respiratory Diseases • Septic Shock • Sickle Cell Disease

Red Is Not Always Bad: Vin Rosé Urine in a Kidney Transplant Patient with Sickle Cell Crisis (KIDNEY WEEK 2025) - "His treatment included Tacrolimus ER, Cellcept, Prednisone, Hydroxyurea, Deferiprone, Apixaban, Pregabalin, Nifedipine and Methadone. However, as urinalysis experts nephrologists should be aware of it. Figure 1: Vin rosé urine"

Clinical • Anemia • Chronic Kidney Disease • CNS Disorders • Epilepsy • Genetic Disorders • Glomerulonephritis • Infectious Disease • Lupus Nephritis • Nephrology • Renal Calculi • Renal Disease • Sickle Cell Disease • Transplantation

Long Term Follow up of a Child with a Rare Hemoglobinopathy: Hemoglobin Little Venice (ASH 2024) - "At 14, she was treated for asymptomatic babesiosis with atovaquone and azithromycin.At present, she continues to require regular transfusions every 4 weeks due to severe hemolytic anemia and has significant thrombocytosis along with leukocytosis and nucleated red blood cells. She has significant transfusion-associated hemosiderosis and receives double chelation with deferasirox and deferiprone with marked history of noncompliance...At present, little is known regarding treatment choices for this rare hemoglobinopathy therefore, we will continue chronic blood transfusions and encourage compliance with iron chelation. A similar variant affecting the same codon, Hb Hammersmith [β42 Phe→Ser, HBB : c.128T>C] has comparatively more data in the literature which may offer some general guidance."

Clinical • Anemia • Gastroenterology • Hematological Disorders • Hepatology • Infectious Disease • Novel Coronavirus Disease • Thrombocytopenia • Thrombocytosis

Efficacy and Safety of Combination Therapy with Oral Iron Chelators Deferiprone and Deferasirox in Patients with β-Thalassemia Major: A Systematic Literature Review (ASH 2024) - "Currently, 3 iron chelators are approved for clinical use : deferoxamine (DFO), deferiprone (DFP), and deferasirox (DFX), all of them with comparable efficacy. Adherence, defined as percentage of dose taken in relation to the prescribed dosage, was higher with DFP+DFX compared with other combinations, ranging from 60% to 95% across the 4 studies.ConclusionThis systematic literature review suggests that combination of oral iron chelators DFP and DFX can effectively reduce overall iron overload from liver and/or heart without new safety concerns. Limitations include small sample size, variability in dosages, and nonrandomized patient population."

Clinical • Combination therapy • Review • Agranulocytosis • Beta-Thalassemia • Gastroenterology • Genetic Disorders • Granulocytopenia • Hematological Disorders • Hepatology • Musculoskeletal Pain • Neutropenia • Rheumatology • Thrombocytopenia

Abrupt Increases in Ferritin Levels May Indicate a Malignant Process and Not Changes in Iron Overload in Thalassemic Patients (ASH 2024) - "Chelation therapy included deferasirox (DFX) (8pts), deferoxamine (DFO) or deferiprone (DFP) (3 pts each), DFP and DFO (5 pts), DFO and DFX or DFP and DFX (1 pt each).The results showed that ferritin levels, while they were no different between 1 year and 5 years prior to cancer diagnosis (p=0.13), rise sharply during 0-6 months prior to cancer diagnosis, with a mean increase of 933±1728(129% of baseline). Thus, our data support that ferritin levels is a sensitive marker for malignancy even in thalassemic patients with iron overload. Unexplained changes in serum ferritin levels in this group of patients should prompt for timely evaluation for an underlying malignancy."

Clinical • Biliary Cancer • Cholangiocarcinoma • Colorectal Cancer • Endocrine Cancer • Genetic Disorders • Genito-urinary Cancer • Hematological Malignancies • Hepatocellular Cancer • Lung Cancer • Lymphoma • Non Small Cell Lung Cancer • Non-Hodgkin’s Lymphoma • Oncology • Pancreatic Cancer • Renal Cell Carcinoma • Solid Tumor • Thyroid Gland Carcinoma

Risk Assessment of Complications Prevalence in Italian Chelated Transfusion Dependent Thalassemia Patients (ASH 2024) - "The three main chelators used in current clinical practice are deferoxamine (DFO), deferiprone (DFP) and deferasirox (DFX). Using MCRM, DFP confirmed advantage over DFO with similar result in comparison with DFX. Overall, this study suggested, as oral chelation with DFP had advantage over DFO and it confirmed to have more efficacy for cardiac complication over DFX."

Clinical • Genetic Disorders

Comparative Efficacy and Safety of Iron Chelators in Sickle Cell Disease: A Meta-Analysis (ASH 2024) - "The standard of care for transfusional iron overload involves iron chelation using agents such as deferoxamine (DFO), deferiprone (DFP), and deferasirox (DFX). These findings suggest that while DFO and DFX achieve similar efficacy in reducing serum ferritin and LIC, DFX may offer a better safety profile for SCD patients, with no significant differences in patient compliance. Further studies and multicenter collaborations are warranted to enhance the evidence base for optimizing chelation strategies in this unique patient population and to confirm these findings."

Retrospective data • Genetic Disorders • Hematological Disorders • Musculoskeletal Pain • Sickle Cell Disease

Ferritin and Iron Burden in SAH sIRB (clinicaltrials.gov) - P1/2 | N=66 | Recruiting | Sponsor: Duke University | Trial completion date: May 2026 ➔ Oct 2026 | Trial primary completion date: Nov 2025 ➔ Apr 2026

Trial completion date • Trial primary completion date • Alzheimer's Disease • Cardiovascular • CNS Disorders • Dementia • Hematological Disorders • Subarachnoid Hemorrhage