deferiprone / Generic mfg. - LARVOL DELTA

Infratentorial Superficial Siderosis following Cervical Laminectomy and Durotomy: A Case Report (PAINWeek 2026) - "Iron chelators have shown promise such as Deferiprone, however also with mixed and inconsistent outcomes, underscoring the need for more research. An 87-year-old male with a past medical history of chronic low back pain status post multiple lumbar spine injections initially presented to the emergency room with bilateral lower extremity and right upper extremity weakness. He was found to have a cervical intramedullary lesion for which he underwent a C4-C5 and C5-C6 laminectomy with durotomy with resection of the lesion. Pathology showed inflammatory changes without evidence of neoplasm."

Case report • Clinical • Ataxia • Back Pain • CNS Disorders • Lumbar Back Pain • Movement Disorders • Musculoskeletal Pain • Subarachnoid Hemorrhage

INOVA-2023-159: Early Screening and Treatment of Heart Complication in Sickle Cell Disease (clinicaltrials.gov) - P2 | N=100 | Recruiting | Sponsor: Inova Health Care Services | Trial completion date: Jun 2027 ➔ Dec 2027 | Trial primary completion date: Jun 2026 ➔ Jun 2027

Trial completion date • Trial primary completion date • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Deferiprone Modulates Ferroptosis to Protect Macrophages and Liver during Neospora caninum Infection. (PubMed, Acta Parasitol) - "In summary, these results establish ferroptosis as a critical pathogenic mechanism in N. caninum infection and highlight DFP's dual role in mitigating oxidative injury while possibly impairing host metabolic responses. Our findings provide novel insights into iron-targeted therapeutic strategies for combating intracellular parasitic infections, paving the way for future investigations into optimizing host-directed antiparasitic interventions."

Journal • Alzheimer's Disease • Beta-Thalassemia • CNS Disorders • Genetic Disorders • Hematological Disorders • Infectious Disease • FTH1 • GPX4

Green Tea Extract Attenuates Oxidative Stress and Prolongs Red Blood Cell Survival in Iron-Overloaded β-Thalassemic Mice. (PubMed, Antioxidants (Basel)) - "Mice subsequently received oral GTE (300 mg/kg/day) or deferiprone (DFP, 50 mg/kg/day) for 12 weeks...The protective effects of GTE were comparable to DFP treatment. These findings suggest that GTE may serve as a promising natural adjunctive strategy for reducing oxidative injuries in β-thalassemia and iron-overload disorders."

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

Brazilin reduces toxic iron species and oxidative damage in iron-loaded β-thalassemic mice. (PubMed, Biomed Pharmacother) - "Iron overload was induced with a 0.2% TMH-ferrocene diet, and mice were treated with deferiprone (DFP 50 mg/kg body weight) or brazilin (50 and 100 mg/kg body weight)...It also modulated HAMP and TfR expression, and histologically reduced hepatic iron deposition, although the reduction in cardiac iron levels was modest. These findings demonstrate that brazilin attenuates iron overload and associated oxidative stress in β-thalassemic mice, consistent with its iron-chelating and antioxidant properties, and support its potential as an adjunctive approach for managing iron overload in β-thalassemia."

Journal • Preclinical • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • HAMP

Machine learning-assisted colorimetric sensor array for identification and quantitative analysis of iron chelators. (PubMed, Biosens Bioelectron) - "The three clinical chelators, namely deferoxamine (DFO), deferasirox (DFX), and deferiprone (DFP), exhibit distinct iron-chelating capacities (DFO > DFX > DFP). By incorporating machine learning algorithms, this sensor array enables high-precision discrimination and quantitative prediction of multi-chelator levels in human serum samples with good recoveries ranging from 90.9 % to 108.1 %, which can effectively avoid the cross-interference in conventional single-signal readout methods. This mimetic enzyme-based sensor array is simple to operate, cost-effective, and capable of multi-component recognition, offering a new methodological framework for iron chelators and intelligent colorimetric analysis of complex drug systems."

Journal • Genetic Disorders • Hematological Disorders

The role of CNNM4 in the progression of cholangiocarcinoma: implications for ferroptosis and therapeutic potential (EASL 2025) - "This study demonstrates that increased CNNM4 expression drives CCA progression and malignancy and that its inhibition may be an effective therapeutic strategy to limit proliferation and metastasis in CCA patients."

Biliary Cancer • Cholangiocarcinoma • Hepatology • Oncology • Solid Tumor • NICD

Neutropenia in adult patients with thalassemia receiving deferiprone: a 10-year experience from a large thalassemia cohort. (PubMed, Blood Res) - "In this large real-world cohort, DFP-induced neutropenia was uncommon but clinically significant, occurring predominantly within the first 6 months of therapy. Baseline cytopenia may help identify patients at a higher risk. With appropriate monitoring, early recognition, and individualized management, DFP remains a viable and effective iron chelation option for adult patients with thalassemia."

Journal • Agranulocytosis • Beta-Thalassemia • Genetic Disorders • Granulocytopenia • Hematological Disorders • Infectious Disease • Neutropenia • Septic Shock

Spatiotemporal iron-hijacking hydrogel reprograms host-pathogen iron homeostasis for fungal keratitis therapy. (PubMed, Cell Rep Med) - "To disrupt this, we develop a spatiotemporally controlled gallium-deferiprone (Ga-DFP) composite hydrogel (PGaDH) that reprograms host-pathogen iron homeostasis through an iron-hijacking strategy...In mouse and rabbit FK models, PGaDH shows prolonged ocular retention, potent antifungal efficacy, and reliable safety. These findings establish a dual iron-hijacking platform with translational potential for fungal and related diseases."

Journal • Hematological Disorders • Infectious Disease • Inflammation • Keratitis • Ocular Inflammation • Ophthalmology

Metal ion dyshomeostasis in neurological disorders: from pathological mechanisms to targeted neuroprotective and regenerative strategies. (PubMed, Front Pharmacol) - "Furthermore, we evaluate the translational challenges of clinical-stage metal chelators (e.g., deferiprone, PBT2) and highlight novel nano-delivery strategies designed to cross the blood-brain barrier to reverse subcellular metal toxicity. This provides a clear therapeutic roadmap for developing next-generation neuroprotective drugs."

Journal • Review • CNS Disorders • Vascular Neurology

Mitochondria-targeted chelators with potential to disrupt iron metabolism in cancer cells: The role of the linker structure in successful membrane translocation. (PubMed, Eur J Med Chem) - "A series of compounds with a 3-hydroxypyrid-4-one chelating unit (analogues of deferiprone, DFP, one of the clinically approved iron chelators) connected to a mitochondria-targeting triphenylphosphonium (TPP) moiety through linkers of different lengths and compositions was synthesised...Preliminary cell studies demonstrate that these chelators can cross biological membranes, and their cytotoxicity increases with increasing hydrophobic character of the linker used. A comparative study of molecules whose iron(III)-binding ability was blocked by modification of the chelating unit showed that general TPP-based membrane disruption and iron(III)-chelating ability cumulate to increase cytotoxicity."

Journal • Oncology

Amelioration of Cisplatin Nephrotoxicity by Fe-Pt Coordination Exchange-Triggered Ferroptosis Suppression. (PubMed, Adv Healthc Mater) - "The block copolymer, methoxyl polyethylene glycol-poly(glutamic acid) (mPEG-PGlu) was coupled with an iron chelator, deferiprone (Dfp) to generate the tailored amphiphilic conjugate, mPEG-P(Glu-Dfp) that could coordinate with activated cisplatin and self-assemble into micelles (Pt@MDfp)...Moreover, due to the controlled cargo activation, Pt@MDfp significantly enhanced the in vivo antitumor efficacy compared to Pt@MGlu and free cisplatin. Collectively, the coordination exchange approach is promising in controlled delivery of cisplatin for efficacy enhancement."

Journal • Breast Cancer • Fibrosis • Immunology • Oncology • Renal Disease • Solid Tumor

Precision Medicine in Transfusion-Dependent and Non-Transfusion-Dependent β-Thalassemia: Toward Personalized Diagnosis and Therapy. (PubMed, Hemoglobin) - "Literature indexed in PubMed and Scopus from 2000 to 2025 was reviewed using terms related to thalassemia, precision medicine, magnetic resonance imaging (MRI), chelation tailoring, next-generation sequencing (NGS), fetal hemoglobin (HbF) modifiers, luspatercept, mitapivat, hepcidin, gene therapy, gene editing, and artificial intelligence (AI)...Personalized chelation should include deferiprone, either alone or in combination, when cardiac iron is increased...AI currently has its strongest role in screening and diagnosis, whereas risk-stratification models remain exploratory. Equitable implementation requires standardized TDT/NTDT pathways, regional MRI and genomics access, longitudinal registries, and multidisciplinary interpretation."

Journal • Review • Beta-Thalassemia • Gene Therapies • Genetic Disorders • Hematological Disorders • GDF15 • HBA2

Iron overload cardiomyopathy. (PubMed, Heart Fail Rev) - "Phlebotomy remains the cornerstone of treatment in primary haemochromatosis, while iron chelators, including deferoxamine, deferiprone and deferasirox, is the standard for transfusion-dependent patients. Novel disease-modifying or curative treatments for thalassaemia, such as luspatercept, mitapivat and gene therapy offer the prospect of addressing the root cause of iron loading. This review provides an updated comprehensive, evidence-based overview of the pathophysiology, diagnosis, and management of IOC."

Journal • Review • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Gene Therapies • Heart Failure • Hematological Disorders • Metabolic Disorders

Sappanwood extract modulates hepatic structure-function in hepatomegaly and hepcidin related iron regulatory pathways in a phenylhydrazine induced hemolytic anemia rat model relevant to thalassemia. (PubMed, Animal Model Exp Med) - "These findings confirm the potential of sappanwood as a potential therapeutic candidate for the management of iron overload in thalassemia."

Journal • Preclinical • Anemia • Genetic Disorders • Hematological Disorders • Hepatology • Metabolic Disorders • BMP6

Iron metabolism and ferroptosis in cardiovascular diseases: progress in the mechanism and pharmacological intervention. (PubMed, Redox Biol) - "Notably, various compounds like iron chelators (e.g., deferoxamine, deferiprone), chloroiodohydroxyquin and its derivatives, antioxidants (e.g., vitamin E, lipoic acid, selenium), Ferrostatin-1 (Fer-1), etc., have demonstrated efficacy in animal studies against CVDs, offering cardiovascular protection. In summary, this review outlines the role of ferroptosis in CVDs, the regulatory effects of relevant drugs (including natural products) on ferroptosis within cardiovascular contexts, aiming to provide valuable insights for CVDs management and the development of novel therapeutics."

Journal • Review • Atherosclerosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • Myocardial Ischemia • Reperfusion Injury

Diagnosis and Management of Transfusion-Dependent Thalassemia: Evidence-Based Guidelines From the Pediatric Hematology Oncology Chapter of the Indian Academy of Pediatrics. (PubMed, Indian Pediatr) - "Regular transfusion therapy with leukodepleted packed red cells to maintain pre-transfusion hemoglobin of 9.5-10.5 g/dL is the cornerstone of management in transfusion-dependent thalassemia (TDT), with individualized adjustment in special situations such as cardiac dysfunction. Iron chelation therapy should be initiated after 10-20 transfusions or when serum ferritin (SF) exceeds 1000 ng/mL; oral deferasirox is the preferred first-line agent. Combination therapy using deferasirox, and/or deferiprone and/or desferrioxamine is recommended when monotherapy with either deferasirox or deferiprone fails to reduce SF to < 2500 ng/mL or in the case of poor tolerance to one agent or when there is evidence of significant organ iron overload on T2*MRI (magnetic resonance imaging); de-escalation of chelation is recommended when SF approaches < 500 ng/mL."

Journal • Review • Beta-Thalassemia • Bone Marrow Transplantation • Genetic Disorders • Hematological Disorders • Oncology • Pediatrics • Transplantation

The therapeutic opportunities and pitfalls of using iron ion chelators to treat neurodegenerative diseases. (PubMed, FEBS Lett) - "In using iron chelators, it is important to remember the basics of their redox chemistry, which could explain their lack of therapeutic effects. This issue is explored here, and the potential of iron ion chelation in the treatment of neurodegeneration is critically evaluated."

Journal • Review • Alzheimer's Disease • CNS Disorders • Dementia • Movement Disorders • Parkinson's Disease

Ferritin and Iron Burden in SAH sIRB (clinicaltrials.gov) - P1/2 | N=66 | Active, not recruiting | Sponsor: Duke University | Recruiting ➔ Active, not recruiting

Enrollment closed • Alzheimer's Disease • Cardiovascular • CNS Disorders • Dementia • Hematological Disorders • Subarachnoid Hemorrhage

Classical superficial siderosis: a rare neuropsychiatric disorder. (PubMed, BMJ Case Rep) - "She was started on amantadine up to 200 mg/day, deferiprone up to 1000 mg/day, vitamin E supplementation and donepezil. There was a modest improvement in ataxia and frequency of falls."

Journal • Ataxia • Cognitive Disorders • Mental Retardation • Movement Disorders • Otorhinolaryngology • Psychiatry

Fertility and endocrinopathies among adults with β-thalassemia major treated at Dubai thalassemia center. (PubMed, Front Endocrinol (Lausanne)) - "Despite the improved care and introduction of oral chelators, the prevalence of endocrinopathies namely hypogonadism, short stature, hypothyroidism, hypoparathyroidism and diabetes mellitus still pose a major concern for patients with BTM. More emphasis needs to be placed on improving compliance with chelation, and further studies are required to investigate new therapies options to reduce these complications."

Journal • Observational data • Retrospective data • Beta-Thalassemia • Diabetes • Endocrine Disorders • Genetic Disorders • Hematological Disorders • Hypoparathyroidism • Infertility • Metabolic Disorders • Sexual Disorders

A Bayesian reappraisal of disease-modifying drug candidates for Parkinson's Disease (EAN 2026) - " We performed a Bayesian reappraisal of major DMT trials—covering cinpanemab, deferiprone, CoQ10, ambroxol, NLY01, liraglutide, lixisenatide, and exenatide—focusing on motor and non-motor outcomes, with standardized effect sizes, Bayes Factors (BF₁₀), and posterior probabilities. Bayesian analysis aligns with the strongest frequentist signals while clarifying ambiguous or inconsistent results, underscoring that the magnitude of the effect and evidential strength vary substantially across compounds. GLP-1R agonists show promising efficacy as DMT candidates for PD treatment. Pharmacokinetic and pharmacodynamic differences among the compounds could underlie the different efficacy profiles concerning the disease stage and different symptoms (i.e, motor or non-motor)."

CNS Disorders • Movement Disorders • Parkinson's Disease

Rationally designed copper-deferiprone-phenanthroline complex as a dual anticancer and SARS-CoV-2 inhibitor. (PubMed, RSC Adv) - "Compared to cisplatin, doxorubicin, oxaliplatin, fluorouracil, and deferiprone, the copper complex demonstrated significantly enhanced anticancer potency. In addition, molecular docking revealed strong binding affinities of the complex toward key SARS-CoV-2 targets, including the main protease (Mpro), papain-like protease (PLpro), nucleocapsid N-terminal RNA-binding domain, and RNA-dependent RNA polymerase (RdRp), surpassing several currently available antiviral agents. Collectively, these findings identify the copper-deferiprone-phenanthroline complex as a promising candidate for dual biomedical applications, serving as both an anticancer agent and a potential SARS-CoV-2 inhibitor."

IO biomarker • Journal • Infectious Disease • Novel Coronavirus Disease • Oncology • Respiratory Diseases • BAX • BCL2 • CASP3

Therapeutic Impact of Iron Chelation in PKAN and Related NBIA Subtypes: a systematic review of 552 cases (EAN 2026) - "PubMed, BVS and Google Scholar were searched using the keywords “Neurodegeneration with Brain Iron”, “Accumulation”, “NBIA1”, “Chelation Therapy”, “Deferiprone”... Iron chelation therapy with Deferipone is associated with reduced brain iron and clinical stabilization in most NBIA1 patients, although neurological reversal is uncommon. Hematologic adverse effects limit treatment, emphasizing the need for safer long-term therapies and standardized outcome measures."

Clinical • Review • CNS Disorders • FTL

BONE DISEASE IN ADULTS WITH THALASSAEMIA: CHARACTERISTICS, INVESTIGATION AND MANAGEMENT INSIGHTS FROM A TERTIARY SERVICE IN THE UK (EHA 2026) - "77 were receiving iron chelation: 63.6% on a single chelator (most commonly deferasirox) and 36.4% on dual chelation therapy, predominantly deferasirox plus deferiprone...6 patients received Denosumab, 2 received Teriparatide, and 3 had Romosozumab...Summary/Conclusion Adults with thalassaemia develop low BMD at a relatively young age with an equal sex distribution. A structured, multidisciplinary approach enables systematic investigation, optimisation of reversible factors, and rational pharmacological decision-making in this high-risk cohort."

Clinical • Beta-Thalassemia • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Musculoskeletal Diseases • Orthopedics • Osteoporosis