DEFERIPRONE

Details

Stereochemistry	ACHIRAL
Molecular Formula	C7H9NO2
Molecular Weight	139.1519
Optical Activity	NONE
Defined Stereocenters	0 / 0
E/Z Centers	0
Charge	0

SHOW SMILES / InChI

Structure Search

SMILES

CN1C=CC(=O)C(O)=C1C

InChI

InChIKey=TZXKOCQBRNJULO-UHFFFAOYSA-N

InChI=1S/C7H9NO2/c1-5-7(10)6(9)3-4-8(5)2/h3-4,10H,1-2H3

HIDE SMILES / InChI

Molecular Formula	C7H9NO2
Molecular Weight	139.1519
Charge	0
Count

Stereochemistry	ACHIRAL
Additional Stereochemistry	No
Defined Stereocenters	0 / 0
E/Z Centers	0
Optical Activity	NONE

Description
Sources: http://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf
Curator's Comment: description was created based on several sources, including https://pubchem.ncbi.nlm.nih.gov/compound/deferiprone#section=Top; http://www.ncbi.nlm.nih.gov/pubmed/12825969

Deferiprone (trade name Ferriprox) is an iron chelator indicated for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate. Deferiprone is an orally bioavailable bidentate ligand with iron chelating activity. Deferiprone binds to iron in a 3:1 (ligand:iron) molar ratio. By binding to iron, deferiprone is able to remove excess iron from the body. All the adverse effects of deferiprone are considered reversible, controllable and manageable. These include agranulocytosis with frequency of about 0.6%, neutropenia 6%, musculoskeletal and joint pains 15%, gastrointestinal complains 6% and zinc deficiency 1%.

CNS Activity

Originator

Approval Year

Targets

Primary Target	Pharmacology	Condition	Potency
Iron 19 Target ID: CHEMBL2363058 Sources: http://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf	Chelating Agent 143

Conditions

Condition	Modality	Targets	Highest Phase	Product
Thalassemia syndrome 1 Sources: http://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf	Secondary		Approved 8360	FERRIPROX Approved Use Indicated for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate. Approval is based on a reduction in serum ferritin levels. There are no controlled trials demonstrating a direct treatment benefit, such as improvement in disease-related symptoms, functioning, or increased survival Launch Date 1.31855037E12
Iron overload 4 Sources: http://www.webmd.com/drugs/2/drug-158383/deferiprone-oral/details/list-conditions	Primary		Approved 8360	FERRIPROX Approved Use Indicated for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate. Approval is based on a reduction in serum ferritin levels. There are no controlled trials demonstrating a direct treatment benefit, such as improvement in disease-related symptoms, functioning, or increased survival. Safety and effectiveness have not been established for the treatment of transfusional iron overload in patients with other chronic anemias. Launch Date 1.31855037E12

C_max

Value	Dose	Analyte	Population
64.8 μM EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/27975237/	25 mg/kg single, oral dose: 25 mg/kg route of administration: Oral experiment type: SINGLE co-administered:	DEFERIPRONE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
118.8 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE serum	Homo sapiens
150.8 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE serum	Homo sapiens
60.8 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE serum	Homo sapiens
35.2 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE plasma	Homo sapiens population: healthy age: sex: food status:
51.4 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	50 mg/kg single, oral dose: 50 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE plasma	Homo sapiens population: healthy age: sex: food status:
54.4 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	50 mg/kg single, oral dose: 50 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE plasma	Homo sapiens population: healthy age: sex: food status:
34.1 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE plasma	Homo sapiens population: healthy age: sex: food status:
30.6 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE serum	Homo sapiens
33.4 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE serum	Homo sapiens
43.3 ug/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE serum	Homo sapiens

AUC

Value	Dose	Analyte	Population
15001 μM × min EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/27975237/	25 mg/kg single, oral dose: 25 mg/kg route of administration: Oral experiment type: SINGLE co-administered:	DEFERIPRONE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
205.5 ug*h/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE plasma	Homo sapiens population: healthy age: sex: food status:
331.2 ug*h/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	50 mg/kg single, oral dose: 50 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE plasma	Homo sapiens population: healthy age: sex: food status:
152.1 ug*h/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	50 mg/kg single, oral dose: 50 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE plasma	Homo sapiens population: healthy age: sex: food status:
95.4 ug*h/mL Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE plasma	Homo sapiens population: healthy age: sex: food status:

T_1/2

Value	Dose	Analyte	Population
168 min EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/27975237/	25 mg/kg single, oral dose: 25 mg/kg route of administration: Oral experiment type: SINGLE co-administered:	DEFERIPRONE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FASTED
2.58 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE serum	Homo sapiens
2.58 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE serum	Homo sapiens
3.34999999999999 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE serum	Homo sapiens
2.5 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE plasma	Homo sapiens population: healthy age: sex: food status:
2.6 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	50 mg/kg single, oral dose: 50 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE 3-O-BETA-D-GLUCURONIDE plasma	Homo sapiens population: healthy age: sex: food status:
1.8 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	50 mg/kg single, oral dose: 50 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE plasma	Homo sapiens population: healthy age: sex: food status:
1.8 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01860703	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE plasma	Homo sapiens population: healthy age: sex: food status:
1.77 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE serum	Homo sapiens
2.03 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE serum	Homo sapiens
2.2 h Clinical Trial https://clinicaltrials.gov/ct2/show/NCT01770652	33 mg/kg single, oral dose: 33 mg/kg route of administration: oral experiment type: single co-administered:	DEFERIPRONE serum	Homo sapiens

Doses

Dose	Population	Adverse events
30 mg/kg 2 times / day multiple, oral Recommended Dose: 30 mg/kg, 2 times / day Route: oral Route: multiple Dose: 30 mg/kg, 2 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/31620867 Page: p.3	unhealthy, 29–71 n = 10 Health Status: unhealthy Condition: Infratentorial superficial siderosis Age Group: 29–71 Sex: M+F Population Size: 10 Sources: https://pubmed.ncbi.nlm.nih.gov/31620867 Page: p.3	Disc. AE: Neutropenic sepsis, Fatigue... AEs leading to discontinuation/dose reduction: Neutropenic sepsis (30%) Fatigue (10%) Sources: https://pubmed.ncbi.nlm.nih.gov/31620867 Page: p.3
100 mg/kg multiple, oral (max\|total daily dose) Recommended Dose: 100 mg/kg Route: oral Route: multiple Dose: 100 mg/kg Sources: https://pubmed.ncbi.nlm.nih.gov/32470438 Page: e475	unhealthy n = 193 Health Status: unhealthy Condition: Transfusion-dependent haemoglobinopathies Sex: M+F Population Size: 193 Sources: https://pubmed.ncbi.nlm.nih.gov/32470438 Page: e475	Disc. AE: Arthralgia, Joint effusion... AEs leading to discontinuation/dose reduction: Arthralgia (non-serious) Joint effusion (non-serious) Nausea (non-serious) Abdominal discomfort (non-serious) Fatigue (non-serious) Joint swelling (non-serious) Epistaxis (non-serious) Upper respiratory tract infections (non-serious) Upper abdominal pain (non-serious) Vomiting (non-serious) Palpitation (non-serious) Sources: https://pubmed.ncbi.nlm.nih.gov/32470438 Page: e475
33 mg/kg 3 times / day multiple, oral (max) Recommended Dose: 33 mg/kg, 3 times / day Route: oral Route: multiple Dose: 33 mg/kg, 3 times / day Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf Page: p.3	unhealthy n = 642 Health Status: unhealthy Condition: Transfusional iron overload due to thalassemia syndromes Population Size: 642 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf Page: p.3	Disc. AE: Aspartate aminotransferase increased, Alanine aminotransferase increased... AEs leading to discontinuation/dose reduction: Aspartate aminotransferase increased (0.78%) Alanine aminotransferase increased (0.16%) Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf Page: p.3
33 mg/kg 3 times / day multiple, oral (max) Recommended Dose: 33 mg/kg, 3 times / day Route: oral Route: multiple Dose: 33 mg/kg, 3 times / day Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf Page: p.4	unhealthy n = 642 Health Status: unhealthy Condition: Transfusional iron overload due to thalassemia syndromes Population Size: 642 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf Page: p.4	Disc. AE: Nausea, Vomiting... AEs leading to discontinuation/dose reduction: Nausea (1.6%) Vomiting (1.6%) Abdominal pain (1.6%) Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf Page: p.4
33 mg/kg 3 times / day multiple, oral (max) Recommended Dose: 33 mg/kg, 3 times / day Route: oral Route: multiple Dose: 33 mg/kg, 3 times / day Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf Page: p.1	unhealthy Health Status: unhealthy Condition: Transfusional iron overload due to thalassemia syndromes Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf Page: p.1	Disc. AE: Agranulocytosis, Fetal damage... AEs leading to discontinuation/dose reduction: Agranulocytosis (grade 3-5) Fetal damage Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/021825lbl.pdf Page: p.1
15 mg/kg 2 times / day steady, oral Dose: 15 mg/kg, 2 times / day Route: oral Route: steady Dose: 15 mg/kg, 2 times / day Sources: https://clinicaltrials.gov/ct2/show/NCT01741532	unhealthy n = 58 Health Status: unhealthy Condition: Pantothenate Kinase-associated Neurodegeneration Population Size: 58 Sources: https://clinicaltrials.gov/ct2/show/NCT01741532	Other AEs: Neutropenia, Intestinal dilatation... Other AEs: Neutropenia (serious, 5 patients) Intestinal dilatation (serious, 1 patient) Intestinal obstruction (serious, 1 patient) Volvulus (serious, 1 patient) Obstruction (serious, 1 patient) Pyrexia (serious, 1 patient) Bacterial disease carrier (serious, 1 patient) Bronchitis (serious, 2 patients) Device related infection (serious, 1 patient) Pneumonia (serious, 2 patients) Wound infection (serious, 1 patient) Chemical eye injury (serious, 1 patient) Postoperative ileus (serious, 1 patient) Toxicity to various agents (serious, 1 patient) Unintentional medical device removal (serious, 1 patient) Oromandibular dystonia (serious, 1 patient) Device malfunction (serious, 1 patient) Urinary bladder rupture (serious, 1 patient) Choking (serious, 1 patient) Cough (serious, 1 patient) Respiratory disorder (serious, 1 patient) Colectomy (serious, 1 patient) Intestinal anastomosis (serious, 1 patient) Intrathecal pump insertion (serious, 1 patient) Laparotomy (serious, 1 patient) Medical device battery replacement (serious, 2 patients) Tracheostomy (serious, 1 patient) Tracheostomy tube removal (serious, 1 patient) Wound treatment (serious, 1 patient) Thrombosis (serious, 1 patient) Anaemia (below serious, 12 patients) Influenza (below serious, 3 patients) Pain (below serious, 4 patients) Bronchitis (below serious, 7 patients) Nasopharyngitis (below serious, 11 patient) Rhinitis (below serious, 4 patients) Upper respiratory tract infection (below serious, 8 patients) Viral infection (below serious, 4 patients) Laceration (below serious, 6 patients) Body temperature increased (below serious, 3 patients) Neutrophil count decreased (below serious, 10 patients) Serum ferritin decreased (below serious, 19 patients) Iron deficiency (below serious, 9 patients) Arthralgia (below serious, 8 patients) Muscle spasms (below serious, 3 patients) Pain in extremity (below serious, 10 patients) Dystonia (below serious, 25 patients) Migraine (below serious, 3 patients) Urinary incontinence (below serious, 3 patients) Cough (below serious, 10 patients) Oropharyngeal pain (below serious, 9 patients) Rhinorrhoea (below serious, 4 patients) Hyperhidrosis (below serious, 3 patients) Rash (below serious, 4 patients) Sources: https://clinicaltrials.gov/ct2/show/NCT01741532

AEs

Overview

CYP3A4	CYP2C9	CYP2D6	hERG
inhibitor 1579	inhibitor 1752	inhibitor 1837	inhibitor 1599

OverviewOther

Other Inhibitor	Other Substrate	Other Inducer
CYP1A1 110 CYP1A2 1509 CYP2E1 876	UGT1A6 307 UGT1A7 236 UGT1A8 283 UGT1A9 380 UGT1A10 290 UGT1A1 418 UGT1A3 422 UGT1A4 347 UGT1A5 55 UGT2B4 249 UGT2B7 389 UGT2B10 127 UGT2B15 203 UGT2B17 213 UGT2B28 63

Drug as perpetrator

Target	Modality	Activity
CYP1A1 218	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=68 Page: 68.0	no
CYP1A2 1673	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=68 Page: 68.0	no
CYP2C9 1803	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=68 Page: 68.0	no
CYP2D6 1875	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=68 Page: 68.0	no
CYP2E1 964	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=68 Page: 68.0	no
CYP3A4 1909	inhibitor 3240 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=68 Page: 68.0	no

Drug as victim

Target	Modality	Activity
UGT1A6 307	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=18, https://pubmed.ncbi.nlm.nih.gov/18971318/ Page: 18, 26, 27-28, 31, 33, 41	major
UGT1A10 290	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=31, https://pubmed.ncbi.nlm.nih.gov/18971318/ Page: 31.0	minor
UGT1A8 283	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=31, https://pubmed.ncbi.nlm.nih.gov/18971318/ Page: 31.0	minor
UGT1A9 380	substrate 3326 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=31, https://pubmed.ncbi.nlm.nih.gov/18971318/ Page: 31.0	minor
UGT1A1 418	substrate 3326 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	no
UGT1A3 422	substrate 3326 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	no
UGT1A4 347	substrate 3326 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	no
UGT1A5 55	substrate 3326 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	no
UGT2B10 127	substrate 3326 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	no
UGT2B17 213	substrate 3326 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	no
UGT2B28 63	substrate 3326 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	no
UGT2B4 249	substrate 3326 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	no
UGT1A7 236	substrate 3326 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	yes
UGT2B15 203	substrate 3326 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	yes
UGT2B7 389	substrate 3326 Sources: https://pubmed.ncbi.nlm.nih.gov/18971318/	yes

Tox targets

Target	Modality	Activity	Metabolite	Clinical evidence
hERG 1632	inhibitor 1613 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/021825Orig1s000ClinPharmR.pdf#page=73 Page: 73.0

Sourcing

Vendor/Aggregator	ID	URL
ChEBI	CHEBI:68554	https://www.ebi.ac.uk/chebi/searchId.do?chebiId=CHEBI:68554
ChemicalBook	CB0308124	https://www.chemicalbook.com/ChemicalProductProperty_EN_CB0308124
MolPort	MolPort-001-792-505	https://www.molport.com/shop/molecule-link/MolPort-001-792-505
Selleck Chemicals	deferiprone	http://www.selleckchem.com/products/deferiprone.html
ZINC	ZINC000000006226	http://zinc15.docking.org/substances/ZINC000000006226
eMolecules	502935	https://www.emolecules.com/cgi-bin/more?vid=502935

PubMed

Title	Date	PubMed
Antimalarial effect of iron chelators.	2002	12572998
Long term deferiprone chelation therapy.	2002	12572992
Anti-leukemia activity of 7-hydroxy-2-substituted-methyl-5H-oxazolo[3,2-a]pyrimidin-5-one derivatives.	2002 Dec	12683674
Experience with the oral iron chelator deferiprone in transfusion-dependent children.	2002 Dec	12661341
Deferiprone (L1) as an adjuvant therapy for Plasmodium falciparum malaria.	2002 Jan	12424933
Iron toxicity and chelation therapy.	2002 Oct	12416732
Academic freedom in clinical research.	2002 Oct 24	12397199
Immune status of thalassemic patients receiving deferiprone or combined deferiprone and desferrioxamine chelation treatment.	2003	14663175
Iron chelation in chemotherapy.	2003	12964245
Benefits and risks of deferiprone in iron overload in Thalassaemia and other conditions: comparison of epidemiological and therapeutic aspects with deferoxamine.	2003	12825969
Clinical research: a tale of two studies.	2003	12813922
Iron chelating agents for treating malaria.	2003	12804409
[Effect of desferrioxamine and deferiprone on osteocalcin secretion in osteoblast-type cells].	2003	12778850
Antihistone and other autoantibodies in beta-thalassemia major patients receiving iron chelators.	2003	12486321
Multidentate pyridinones inhibit the metabolism of nontransferrin-bound iron by hepatocytes and hepatoma cells.	2003 Apr	12694182
Comparison between desferrioxamine and combined therapy with desferrioxamine and deferiprone in iron overloaded thalassaemia patients.	2003 Apr	12670352
Do we need more iron-chelating drugs?	2003 Aug 9	12927446
Combined therapy with desferrioxamine and deferiprone in thalassemic patients: effect on urinary iron excretion.	2003 Dec	14687998
Synthesis and antiviral evaluation of 3-hydroxy-2-methylpyridin-4-one dideoxynucleoside derivatives.	2003 Dec 15	14643328
Polyamine-iron chelator conjugate.	2003 Dec 4	14640556
The Olivieri case.	2003 Feb 27	12608401
The Olivieri case.	2003 Feb 27	12606746
The Olivieri Report--a compelling study of the growing tensions in clinical research.	2003 Jan	12645235
Patients' health or company profits? The commercialisation of academic research.	2003 Jan	12645227
Deferiprone versus desferrioxamine in thalassaemia, and T2* validation and utility.	2003 Jan 11	12531621
Deferiprone versus desferrioxamine in thalassaemia, and T2* validation and utility.	2003 Jan 11	12531619
Deferiprone versus desferrioxamine in thalassaemia, and T2* validation and utility.	2003 Jan 11	12531618
Deferiprone versus desferrioxamine in thalassaemia, and T2* validation and utility.	2003 Jan 11	12531617
Clinical trial of deferiprone iron chelation therapy in beta-thalassaemia/haemoglobin E patients in Thailand.	2003 Jul	12846901
Iron withdrawal strategies fail to prevent the growth of SiHa-induced tumors in mice.	2003 Jul	12821347
Role of deferiprone in chelation therapy for transfusional iron overload.	2003 Jul 1	12637334
Pulmonary dysfunction in transfusion-dependent patients with thalassemia major.	2003 Jul 15	12738605
Iron-dependent activation of NF-kappaB in Kupffer cells: a priming mechanism for alcoholic liver disease.	2003 Jun	12957294
Monitoring long-term efficacy of iron chelation therapy by deferiprone and desferrioxamine in patients with beta-thalassaemia major: application of SQUID biomagnetic liver susceptometry.	2003 Jun	12786807
Safety monitoring of cardiac and hepatic systems in beta-thalassemia patients with chelating treatment in Taiwan.	2003 Jun	12756022
Emerging understanding of the advantage of small molecules such as hydroxypyridinones in the treatment of iron overload.	2003 Jun	12678676
Iron chelator research: past, present, and future.	2003 Jun	12678671
Deferiprone and hepatic fibrosis.	2003 Jun 15	12788794
Examination of novel zinc-binding groups for use in matrix metalloproteinase inhibitors.	2003 Jun 2	12767177
Potential myocardial iron content evaluation by magnetic resonance imaging in thalassemia major patients treated with Deferoxamine or Deferiprone during a randomized multicenter prospective clinical study.	2003 May	12779268
Comparative effects of deferiprone and deferoxamine on survival and cardiac disease in patients with thalassemia major: a retrospective analysis.	2003 May	12745268
Treatment of cardiac iron overload in thalassemia major.	2003 May	12745264
Glutathione depletion increases nitric oxide-induced oxidative stress in primary rat hepatocyte cultures: involvement of low-molecular-weight iron.	2003 May 15	12726916
Signaling role of intracellular iron in NF-kappaB activation.	2003 May 16	12637578
Pharmacotherapy of iron overload in thalassaemic patients.	2003 Oct	14521486
Labile plasma iron in iron overload: redox activity and susceptibility to chelation.	2003 Oct 1	12805056
Chromosomal aberration frequencies in patients with thalassaemia major undergoing therapy with deferiprone and deferoxamine in a comparative crossover study.	2003 Sep	12960415
Safety and effectiveness of long-term therapy with the oral iron chelator deferiprone.	2003 Sep 1	12763939
Bone mass and metabolism in thalassemic children and adolescents treated with different iron-chelating drugs.	2004	14691688
Superoxide, H2O2, and iron are required for TNF-alpha-induced MCP-1 gene expression in endothelial cells: role of Rac1 and NADPH oxidase.	2004 Mar	14576080

Patents

Sample Use Guides

In Vivo Use Guide

25 mg/kg to 33 mg/kg body weight, orally, three times per day, for a total daily dose of 75 mg/kg to 99 mg/kg body weight.

Route of Administration: Oral

In Vitro Use Guide

Proliferating CD4+ T cells from control and RRMS subjects, cultured with or without IL-2, decreased in response to 75 μM deferiprone, although the extent of decreased proliferation of CD4+ T cells from RRMS subjects was less than for control subjects. Proliferating CD8+ T cells from control subjects, cultured with or without IL-2, also decreased in response to 75 μM deferiprone, and this decrease was seen in proliferating CD8+ T cells from RRMS cultured with IL-2.

Substance Class	Chemical Created by `admin` on `Wed Jul 05 23:16:22 UTC 2023` Edited by `admin` on `Wed Jul 05 23:16:22 UTC 2023`
Record UNII	2BTY8KH53L
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

DEFERIPRONE

Official Name

English

4(1H)-PYRIDINONE, 3-HYDROXY-1,2-DIMETHYL-

Systematic Name

English

DEFERIPRONE [EP MONOGRAPH]

Common Name

English

NSC-758880

Code

English

L-1

Code

English

3-Hydroxy-1,2-dimethylpyridin-4(1H)-one

Systematic Name

English

APO-66

Code

English

DEFERIPRONE [MART.]

Common Name

English

Deferiprone [WHO-DD]

Common Name

English

L1

Code

English

DEFERIPRONE [ORANGE BOOK]

Common Name

English

3-HYDROXY-1,2-DIMETHYL-4(1H)-PYRIDONE

Systematic Name

English

deferiprone [INN]

Common Name

English

DEFERIPRONE [EMA EPAR]

Common Name

English

CP-20

Code

English

DN-180-01-AF

Code

English

DEFERIPRONE [VANDF]

Common Name

English

DN-18001AF

Code

English

CP20

Code

English

DEFERIPRONE [USAN]

Common Name

English

APO-066

Code

English

DEFERIPRONE [MI]

Common Name

English

DEFERIPRON

Common Name

English

PL1

Code

English

PL-1

Code

English

Classification Tree Code System Code

EU-Orphan Drug

EU/3/10/832