deferasirox / Generic mfg. - LARVOL DELTA

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 | Not yet recruiting ➔ Recruiting

Enrollment open • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

INOVA-2023-159: Early Screening and Treatment of Heart Complication in Sickle Cell Disease (clinicaltrials.gov) - P2 | N=100 | Not yet recruiting | Sponsor: Inova Health Care Services | Initiation date: Jun 2025 ➔ Sep 2025

Trial initiation date • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

THE RELATIONSHIP BETWEEN IRON OVERLOAD AND GLUCOSE DYSREGULATION IN CHILDREN WITH BETA THALASSEMIA MAJOR (EHA 2025) - "Glucose metabolism abnormalities were found at a low rate in our TM patients, dependent on their adherence to chelation therapy. The study emphasizes the significance of chelation therapy compliance and close glucose metabolism monitoring in preventing glucose metabolism disorders and diabetes development in children with TM."

Clinical • Anemia • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • Hepatology • Liver Failure • Metabolic Disorders

Early Screening and Treatment of Heart Complication in Sickle Cell Disease (clinicaltrials.gov) - P2 | N=100 | Not yet recruiting | Sponsor: Inova Health Care Services

New P2 trial • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Low Dose Deferasirox Yields Promising Transfusion Independence in MDS (Cancer Network) - P2 | N=39 | LODEFI (NCT03387475) | "Results from the single-arm, multi-center, open-label phase 2 LODEFI study (NCT03387475) shared at the 2024 Hematology Annual Meeting & Exposition (ASH), showed that low-dose deferasirox (Jadenu) elicited promising transfusion independence (TI) and a favorable tolerance profile in patients with myelodysplastic syndrome (MDS) who are low-risk anemic. At 12 months, a TI of 47.4% (95% CI, 31.0%-64.2%) was observed in patients treated with deferasirox. At a stricter definition of transfusion dependence, a burden of 4 or more red blood cell units every 8 weeks, 68.4% (95% CI, 51.3%-82.5%) of patients had an observed TI at 12 months...According to the primary end point definition, MDS-RS had a lower rate of transfusion dependence than all other types of MDS, 28.6% vs 58.3%, respectively."

P2 data • Myelodysplastic Syndrome

Comparison of the effects of deferasirox film-coated tablets (Jadenu®) and deferasirox dispersible tablets (Exjade®) in patients with beta thalassemia major: a preliminary report of the effects on the satisfaction, convenience, cardiac/liver MRI T2*, serum ferritin level, and biochemical profiles. (PubMed, Front Pharmacol) - "This, in turn, may help mitigate cardiovascular and hepatic complications from iron overload in the long term. https://irct.behdasht.gov.ir/search/result?query=IRCT20210830052346N1."

Journal • Beta-Thalassemia • Cardiovascular • Gastrointestinal Disorder • Genetic Disorders • Hematological Disorders

Study to Evaluate the Efficacy and Safety of Deferasirox Film-coated Tablet Versus Phlebotomy in Patients With Hereditary Hemochromatosis (HH) (clinicaltrials.gov) - P2 | N=45 | Terminated | Sponsor: Novartis Pharmaceuticals | Completed ➔ Terminated; Due to enrolment challenge

Trial termination • Genetic Disorders • Hematological Disorders • Hepatology

Deferasirox film-coated tablet-associated ulcerative colitis: An emerging pattern in thalassemia patients? (PubMed, Br J Haematol) - No abstract available

Journal • Gastroenterology • Gastrointestinal Disorder • Genetic Disorders • Immunology • Inflammatory Bowel Disease • Ulcerative Colitis

Switching from Deferasirox Dispersible Tablets to Deferasirox Film-Coated Tablets: Is There an Effect on Ferritin Levels in Children and Adolescents with Transfusion-Dependent Anemia? (PubMed, Turk Arch Pediatr) - "Overall, higher SF value was observed with FCT compared to DT in short term. There were different results between patient groups. Our results suggest that FCT is more effective than DT in patients with high basal ferritin and who are actually incompatible with treatment and should be preferred first in these patients."

Journal • Anemia • Hematological Disorders

Deferoxamine Graded Challenge in a Patient with Diamond-Blackfan Anemia (AAAAI 2024) - "There are currently three FDA-approved iron chelating agents: deferoxamine mesylate (Desferal), deferasirox (Jadenu), and deferiprone (Ferriprox). Here, we present the case of a patient with DBA with a self-reported allergy history to deferoxamine. After consideration of risk factors, she underwent a graded challenge in the hospital setting without adverse reaction and is now receiving daily deferoxamine to manage her transfusion-related iron overload."

Clinical • Allergy • Anemia • Aplastic Anemia • Genetic Disorders • Hematological Disorders • Immunology • Oncology

Audiologic and vestibular assessment in children and adolescents with transfusion dependent beta thalassemia major: The era of deferasirox film coated tablet. (PubMed, Int J Pediatr Otorhinolaryngol) - "We conclude that the incidence of SNHL and vestibular dysfunction was high among transfusion dependent β-thalassemia patients. Therefore, we recommend performing pre-treatment baseline audio-vestibular assessment and yearly audio-vestibular monitoring to early detect high risk patients and initiate timely management to prevent permanent damage."

Journal • Beta-Thalassemia • Genetic Disorders • Otorhinolaryngology

Iron Chelation in Patients with Myelodysplastic Syndromes and Myeloproliferative Neoplasms-Real-World Data from the German Noninterventional Study EXCALIBUR. (PubMed, J Clin Med) - "Iron chelation was effective in lowering serum ferritin levels, with the observed adverse drug reactions being in line with the known safety profile. Hematologic response occurred in a few patients, comparable to other studies that examined hematologic improvement in patients with MDS."

Journal • Observational data • Real-world • Real-world evidence • Aplastic Anemia • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Oncology

Patient preference for deferasirox film-coated versus dispersible tablet formulation: a sequential-design phase 2 study in patients with thalassemia. (PubMed, Ann Hematol) - "The most common treatment-emergent AEs were proteinuria, pyrexia, urine protein/creatinine ratio increase, diarrhea, upper respiratory tract infections, transaminase increase, and pharyngitis. Overall, this study reinforced the observations from the previous study by showing a distinct patient preference for FCT over DT formulation and further supported the potential benefits of life-long compliance with ICT."

Journal • P2 data • Gastrointestinal Disorder • Genetic Disorders • Infectious Disease • Renal Disease • Respiratory Diseases

Study to Evaluate the Efficacy and Safety of Deferasirox Film-coated Tablet Versus Phlebotomy in Patients With Hereditary Hemochromatosis (HH) (clinicaltrials.gov) - P2 | N=45 | Completed | Sponsor: Novartis Pharmaceuticals | Active, not recruiting ➔ Completed

Trial completion • Genetic Disorders • Hematological Disorders • Hepatology • Solid Tumor

Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia. (PubMed, Cochrane Database Syst Rev) - "The medication comparisons included in this review had higher than average adherence rates not accounted for by differences in medication administration or side effects, though often follow-up was not good (high dropout over longer trials), with adherence based on a per protocol analysis. Participants may have been selected based on higher adherence to trial medications at baseline. Also, within the clinical trial context, there is increased attention and involvement of clinicians, thus high adherence rates may be an artefact of trial participation. Real-world, pragmatic trials in community and clinic settings are needed that examine both confirmed or unconfirmed adherence strategies that may increase adherence to iron chelation therapy. Due to lack of evidence this review cannot comment on intervention strategies for different age groups."

Adherence • Journal • Review • Cystic Fibrosis • Fibrosis • Genetic Disorders • Hematological Disorders • Immunology • Pulmonary Disease • Respiratory Diseases • Sickle Cell Disease

Renal Function in Patients with Thalassemia Major Receiving Exjade Dispersible Tablets and a New Film-coated Tablet Formulation of Deferasirox (Nanojade). (PubMed, Adv Biomed Res) - "The proportion of S, BUN, eGFR, 24-h U, U/U ratio, and U/U ratio was not significantly different in TM patients treated with Nanojade compared to patients' received Exjade. Nanojade had similar effects to Exjade, and therefore, the use of Nanojade is safe in TM patients and does not seem to be associated with increased renal failure, proteinuria, and hypercalciuria."

Journal • Beta-Thalassemia • Genetic Disorders • Nephrology • Renal Disease

Effect of Aging on Deferasirox Therapy in Transfusion-dependent Patients. A prospective-retrospective, cohort-study. (PubMed, Curr Drug Metab) - "The study evidenced that to ensure the efficacy of deferasirox in terms of control over LIC and, at the same time, a lesser influence on renal function, the dose of the drug to be administered to an elderly patient could be reduced."

Journal • Retrospective data • Hematological Disorders • UGT1A1

Crushed Deferasirox Film-Coated Tablets in Pediatric Patients With Transfusional Hemosiderosis: Results From a Single-Arm, Interventional Phase 4 Study (MIMAS). (PubMed, Am J Hematol) - No abstract available

Journal • P4 data • Pediatrics

MEDICATION ADHERENCE AND HABIT STRENGTH AMONG CHILDREN WITH CANCER OR CHRONIC HEMATOLOGIC CONDITIONS (ASPHO 2022) - "Most prescribed medications included Bactrim (18%), Hydroxyurea (16%), Penicillin (15%), 6-Mercaptopurine (13%), and Jadenu (11%).Median medication adherence levels were high for the PMAS, ARMS-7 and VAS (44 [IQR 4]; 9 [IQR 4]; 95 [IQR 20], respectively). Most participants reported high adherence to their oral medications. The PMAS correlated well with validated medication adherence measures. PMAS is a comprehensive, patient-centered assessment of medication adherence rates and barriers."

Clinical • Genetic Disorders • Hematological Disorders • Hematological Malignancies • Oncology • Sickle Cell Disease

Use of Deferasirox Film-Coated Tablets in Pediatric Patients with Transfusion Dependent Thalassemia: A Single Center Experience. (PubMed, Biology (Basel)) - "Study results confirmed DFX FCT safety in older children in a manner similar to adults, but demonstrated increased frequency of adverse events in younger patients, mainly, involving liver function. With regards to efficacy, study results confirmed the preventive role of DFX FCT in iron loading of liver and heart, however, higher doses than generally recommended were required in order to ensure adequate chelation."

Journal • Genetic Disorders • Hematological Disorders • Ophthalmology • Pediatrics

Pilot Study to Assess the Safety, PK and Iron Chelating Activity of DST-0509 (Deferasirox) in Thalassemia Patients Refractory to Chelation (clinicaltrials.gov) - P2 | N=25 | Completed | Sponsor: DisperSol Technologies, LLC | Recruiting ➔ Completed | N=36 ➔ 25 | Trial primary completion date: Sep 2021 ➔ Jun 2021

Enrollment change • Trial completion • Trial primary completion date • Beta-Thalassemia • Genetic Disorders • MRI

Perforated Duodenal Ulcer Associated with Deferasirox in a Child with β-Thalassemia Major. (PubMed, Hemoglobin) - "However, perforated duodenal ulcer (PDU) has been previously reported in only one patient with β-thalassemia major (β-TM) on Exjade (DFXE). She is back on DFX, but with the film-coated tablet, Jadenu or DFXJ. Perforated duodenal ulcer should be suspected in patients with severe GI symptoms, abdominal distension and tenderness while on DFXE, especially at high doses (30+ mg/kg)."

Clinical • Journal • Beta-Thalassemia • Celiac Disease • Gastrointestinal Disorder • Genetic Disorders • Hematological Disorders • Immunology • Peptic Ulcer

Transfusional Iron Overload in Sickle Cell Patients:Outcomes of Chelation Therapy (ASH 2021) - "Most common side effects of deferasirox were gastrointestinal (abdominal pain, nausea, vomiting, diarrhea), which were seen less commonly with the newer oral formulation (Jadenu). Several conclusions can be drawn from our observation on relatively small number of patients: 1) Deferasirox is effective in decreasing Fe overload as shown by serum ferritin levels 2) Second generation of oral deferasirox is better tolerated, and therefore is associated with improved adherence, 3) Documentation of a decrease in LIC with chelation will be important for the reversal of Fe induced organ damage, and 4) Parallel studies of the levels of Fe regulatory peptides hepcidin and erythroferrone (ERFE) will clarify the effect of chelation therapy on biomarkers of Fe metabolism."

Clinical • Beta-Thalassemia • Cardiovascular • Gastrointestinal Disorder • Genetic Disorders • Hematological Disorders • Immunology • Pain • Sickle Cell Disease • MRI

A Prospective Observational Multicenter Study on Deferiprone & Deferasirox in Clinical Practice of Pediatric Patients with Transfusion-Dependent B-Thalassemia (ASH 2021) - "infants and young children with TDB-T naïve to chelation were put on mono-therapy at younger age than reported before. Both chelators were effective, well tolerated and with no severe safety concerns; however deviation from guidelines was observed as; unnecessary over-dosing of DFX and under-reporting of AEs in both groups in real life practice."

Clinical • Observational data • Agranulocytosis • Beta-Thalassemia • Genetic Disorders • Granulocytopenia • Hematological Disorders • Musculoskeletal Pain • Neutropenia • Pediatrics

Iron Overload in Patients With Heavily Transfused Sickle Cell Disease-Correlation of Serum Ferritin With Cardiac T2 MRI (CMRTools), Liver T2 MRI, and R2-MRI (Ferriscan®). (PubMed, Front Med (Lausanne)) - "Among the patients currently receiving chelation, 26 (96%) are on Deferasirox (DFX) [Jadenu® (24) or Exjade® (2)], with good compliance and tolerance. However, one patient is still receiving IV deferoxamine (DFO), in view of the significantly high systemic iron burden...It also confirms the cardiac sparing effect in patients with SCD, even with the significant transfusion-related iron burden. About 14 (24%) patients of the original cohort died over the past 15 years, indicative of a negative impact of iron overload on disease morbidity and mortality."

Clinical • Journal • Bone Marrow Transplantation • Cardiovascular • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • Transplantation • MRI