deferiprone and Hemoglobinopathies studies

Deferiprone: A pyridone derivative and iron chelator that is used in the treatment of IRON OVERLOAD in patients with THALASSEMIA.
deferiprone : A member of the class of 4-pyridones that is pyridin-4(1H)-one substituted at positions 1 and 2 by methyl groups and at position 3 by a hydroxy group. A lipid-soluble iron-chelator used for treatment of thalassaemia.

Hemoglobinopathies: A group of inherited disorders characterized by structural alterations within the hemoglobin molecule.

Research Excerpts

Excerpt	Relevance	Reference
"Iron overload is a common complication experienced by transfusion-dependent children with hemoglobin disorders."	7.01	No difference in myocardial iron concentration and serum ferritin with deferasirox and deferiprone in pediatric patients with hemoglobinopathies: A systematic review and meta-analysis. ( Ahmed, J; Moeed, A; Naeem, U; Rais, H; Saleem, A; Shuja, SH; Waqar, E, 2023)
"In paediatric patients with transfusion-dependent haemoglobinopathies, deferiprone was effective and safe in inducing control of iron overload during 12 months of treatment."	5.34	Evaluation of the efficacy and safety of deferiprone compared with deferasirox in paediatric patients with transfusion-dependent haemoglobinopathies (DEEP-2): a multicentre, randomised, open-label, non-inferiority, phase 3 trial. ( Bejaoui, M; Bonifazi, D; Ceci, A; Christou, S; Cosmi, C; Cuccia, L; Del Vecchio, GC; Della Pasqua, O; El-Beshlawy, A; Felisi, M; Filosa, A; Hassab, H; Kattamis, A; Kreka, M; Maggio, A; Origa, R; Putti, MC; Reggiardo, G; Sherief, L; Spino, M; Telfer, P; Tempesta, B; Tricta, F; Tsang, YC; Vitrano, A; Zaka, A, 2020)
"Iron overload is a common complication experienced by transfusion-dependent children with hemoglobin disorders."	3.01	No difference in myocardial iron concentration and serum ferritin with deferasirox and deferiprone in pediatric patients with hemoglobinopathies: A systematic review and meta-analysis. ( Ahmed, J; Moeed, A; Naeem, U; Rais, H; Saleem, A; Shuja, SH; Waqar, E, 2023)

Research

Studies (3)

Authors

Clinical Trials (1)

Trial Overview

Trial Outcomes

Cardiac MRI T2*

Timeframe	Studies, this research(%)	All Research%
pre-1990	0 (0.00)	18.7374
1990's	0 (0.00)	18.2507
2000's	0 (0.00)	29.6817
2010's	0 (0.00)	24.3611
2020's	3 (100.00)	2.80

Authors	Studies
Borella, E	1
Oosterholt, S	1
Magni, P	1
Della Pasqua, O	2
Saleem, A	1
Waqar, E	1
Shuja, SH	1
Naeem, U	1
Moeed, A	1
Rais, H	1
Ahmed, J	1
Maggio, A	1
Kattamis, A	1
Felisi, M	1
Reggiardo, G	1
El-Beshlawy, A	1
Bejaoui, M	1
Sherief, L	1
Christou, S	1
Cosmi, C	1
Del Vecchio, GC	1
Filosa, A	1
Cuccia, L	1
Hassab, H	1
Kreka, M	1
Origa, R	1
Putti, MC	1
Spino, M	1
Telfer, P	1
Tempesta, B	1
Vitrano, A	1
Tsang, YC	1
Zaka, A	1
Tricta, F	1
Bonifazi, D	1
Ceci, A	1

Trial	Phase	Enrollment	Study Type	Start Date	Status
Multicentre, Randomised, Open Label, Non-inferiority Trial to Evaluate the Efficacy and Safety of Deferiprone Compared to Deferasirox in Patients Aged From 1 Month to Less Than 18 Years Affected by Transfusion Dependent Haemoglobinopathies[NCT01825512]	Phase 3	435 participants (Actual)	Interventional	2014-03-17	Completed
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Change in cardiac iron concentration (measured using cardiac MRI T2*), assessed as difference between value at 12 months minus value at baseline. MRI T2* is a non-invasive method based on gradient echo (GRE) sequences, where T2* represents the spin-spin relaxation times, measured in milliseconds. The faster the curve decreases (ie, the smaller T2*), the greater amount of iron is in the tissue. Treatment success was assessed as follows: if baseline cardiac T2* was less than 20 ms, an increase of 10% or more after 1 year of treatment was defined as treatment success; if baseline cardiac T2* was more than 20 ms, any increase or a decrease of less than 10% after 1 year of treatment was defined as treatment success. (NCT01825512)
Timeframe: at baseline and after 12 months

Ferritin Level

Intervention	milliseconds (ms) (Mean)
Deferiprone	0.488
Deferasirox	1.121

Change in serum ferritin level, assessed as difference between value at 12 months minus value at baseline. (NCT01825512)
Timeframe: at baseline and after 12 months

Liver MRI

Intervention	ng/mL (Mean)
Deferiprone	-397.583
Deferasirox	-398.184

Change in liver iron concentration (measured using liver MRI), assessed as difference between value at 12 months minus value at baseline. (NCT01825512)
Timeframe: at baseline and after 12 months

Percentage of Successfully Chelated Patients

Intervention	mg/g (Mean)
Deferiprone	-0.848
Deferasirox	-2.975

Percentage of successfully chelated patients is assessed by serum ferritin levels (in all patients) and cardiac MRI T2* (in patients above 10 years of age able to perform an MRI scan without sedation) (NCT01825512)
Timeframe: at baseline and after 12 months

deferiprone and Hemoglobinopathies