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Article ; Online: Commentary concerning the publication by Zheng X et al.: Metabolic activation of deferiprone mediated by CYP2A6 (Xenobiotica, published online 18 May 2021).

Tricta, Fernando

Xenobiotica; the fate of foreign compounds in biological systems

2021 Volume 52, Issue 3, Page(s) 332–333

MeSH term(s)	Activation, Metabolic ; Cytochrome P-450 CYP2A6 ; Deferiprone/pharmacology
Chemical Substances	Deferiprone (2BTY8KH53L) ; Cytochrome P-450 CYP2A6 (EC 1.14.14.1)
Language	English
Publishing date	2021-10-06
Publishing country	England
Document type	Journal Article
ZDB-ID	120287-x
ISSN	1366-5928 ; 0049-8254
ISSN (online)	1366-5928
ISSN	0049-8254
DOI	10.1080/00498254.2021.1986650
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Article ; Online: Real-world evidence: Long-term safety of deferiprone in a large cohort of patients with sickle cell disease enrolled in a registry for up to 10 years.

Kwiatkowski, Janet L / Thompson, Alexis A / Tricta, Fernando / Temin, Noemi Toiber / Rozova, Anna / Fradette, Caroline / Badawy, Sherif M

American journal of hematology

2024

Abstract: Patients with sickle cell disease (SCD) and other anemias who receive blood transfusions are at risk of organ damage due to transfusional iron overload. Deferiprone is an iron chelator with a well-established safety and efficacy profile that is indicated ...

Abstract	Patients with sickle cell disease (SCD) and other anemias who receive blood transfusions are at risk of organ damage due to transfusional iron overload. Deferiprone is an iron chelator with a well-established safety and efficacy profile that is indicated for the treatment of transfusional iron overload. Here, we report safety data from the large-scale, retrospective Ferriprox® Total Care Registry, which involved all patients with SCD taking deferiprone following the 2011 approval of deferiprone in the United States through August 2020. A total of 634 patients who had initiated deferiprone treatment were included. The mean (SD) duration of deferiprone exposure in the registry was 1.6 (1.6) years (range 0 to 9.7 years). In the overall patient population (N = 634), 64.7% (n = 410) of patients reported a total of 1885 adverse events (AEs). In subgroup analyses, 54.6% (n = 71) of pediatric patients and 67.3% (n = 339) of adult patients reported AEs. The most common AEs reported in patients receiving deferiprone were sickle cell crisis (22.7%), nausea (12.1%), vomiting (8.7%), abdominal discomfort (5.4%), and fatigue (5.4%). Neutropenia was reported in four (0.6%) patients and severe neutropenia/agranulocytosis (defined as absolute neutrophil count <0.5 × 10
Language	English
Publishing date	2024-03-01
Publishing country	United States
Document type	Journal Article
ZDB-ID	196767-8
ISSN	1096-8652 ; 0361-8609
ISSN (online)	1096-8652
ISSN	0361-8609
DOI	10.1002/ajh.27276
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Article ; Online: Rates of severe neutropenia and infection risk in patients treated with deferiprone: 28 years of data.

Badawy, Sherif M / Palmblad, Jan / Tricta, Fernando / Toiber Temin, Noemi / Fradette, Caroline / Lin, Leo / Rozova, Anna / Sheth, Sujit

Blood advances

2024

Abstract: Patients treated with deferiprone for transfusional iron overload may experience idiosyncratic drug-induced neutropenia (IDIN) that may put them at risk of infection. The purpose of this analysis was to examine the rates of severe IDIN and risk of ... ...

Abstract	Patients treated with deferiprone for transfusional iron overload may experience idiosyncratic drug-induced neutropenia (IDIN) that may put them at risk of infection. The purpose of this analysis was to examine the rates of severe IDIN and risk of serious infections at different ANC levels in patients treated with deferiprone. Events of severe IDIN (ANC <0.5×109/L) and associated serious infections from clinical trials and postmarketing setting were analyzed by 3 discrete ANC levels: Group 1, 0.2-0.5×109/L; Group 2, 0.1-0.199×109/L; Group 3, <0.1×109/L. In clinical trials, 22 events of severe IDIN were observed (Group 1, n=9; Group 2, n=3; Group 3, n=10); total deferiprone exposure was 1990.26 patient-years; and rates of severe IDIN per 100 patient-years were 0.45 in Group 1, 0.15 in Group 2, and 0.50 in Group 3. All serious infections were in Group 3 (3/10, 30.0%). In the postmarketing setting, 176 events of severe IDIN were reported (Group 1, n=65; Group 2, n=20; Group 3, n=91); total deferiprone exposure was 111,570.24 patient-years; and rates of severe IDIN per 100 patient-years were 0.06 in Group 1, 0.02 in Group 2, and 0.08 in Group 3. Rates of serious infection were 7.7% (n=5/65) in Group 1, 10% (n=2/20) in Group 2, and 13.2% (n=12/91) in Group 3. Our findings suggest that in patients receiving deferiprone, ANC below 0.2×109/L carries a high risk of serious infections, consistent with the recent neutropenia guidelines that agranulocytosis with ANC <0.2×109/L is associated with a high risk of serious infections.
Language	English
Publishing date	2024-04-19
Publishing country	United States
Document type	Journal Article
ZDB-ID	2915908-8
ISSN	2473-9537 ; 2473-9529
ISSN (online)	2473-9537
ISSN	2473-9529
DOI	10.1182/bloodadvances.2023012316
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Article ; Online: The pharmacokinetic and safety profile of single-dose deferiprone in subjects with sickle cell disease.

Soulières, Denis / Mercier-Ross, Jules / Fradette, Caroline / Rozova, Anna / Tsang, Yu Chung / Tricta, Fernando

Annals of hematology

2022 Volume 101, Issue 3, Page(s) 533–539

Abstract: Patients with sickle cell disease (SCD) who undergo repeated blood transfusions often develop iron overload. Deferiprone (Ferriprox®) is an oral iron chelator indicated for the treatment of transfusional iron overload due to thalassemia syndromes and has ...

Abstract	Patients with sickle cell disease (SCD) who undergo repeated blood transfusions often develop iron overload. Deferiprone (Ferriprox®) is an oral iron chelator indicated for the treatment of transfusional iron overload due to thalassemia syndromes and has been recently approved as a treatment for iron overload in adult and pediatric patients with SCD and other anemias. The present study aims to characterize the pharmacokinetic (PK) profile of deferiprone (DFP) in adult subjects with SCD. In this phase I, open-label study, subjects with SCD were administered a single 1500 mg dose of DFP. Blood and urine samples were collected for PK assessments of DFP and its main metabolite, deferiprone 3-O-glucuronide (DFP-G). Eight subjects were enrolled and completed the study. Following drug administration, serum levels of DFP and DFP-G rose to maximum concentrations at 1.0 and 2.8 h post-dose, respectively. The half-lives of DFP and DFP-G were 1.5 and 1.6 h, respectively. The majority of administered drug was metabolized and excreted as DFP-G, with less than 4% excreted unchanged in urine up to 10 h post-dose. Subjects received a safety assessment 7 (± 3) days post-dose. Two subjects reported mild adverse events unrelated to the study drug, and no other safety concerns were reported. The PK profile of DFP in SCD subjects is consistent with previous reports in healthy adult volunteers, suggesting no special dosing adjustments are indicated for this population. These findings provide valuable insight for treating iron overload in patients with SCD, who have limited chelation therapy treatment options (trial registration number: NCT01835496, date of registration: April 19, 2013).
MeSH term(s)	Adult ; Anemia, Sickle Cell/complications ; Anemia, Sickle Cell/therapy ; Blood Transfusion ; Chelation Therapy/adverse effects ; Deferiprone/adverse effects ; Deferiprone/pharmacokinetics ; Deferiprone/therapeutic use ; Female ; Humans ; Iron Chelating Agents/adverse effects ; Iron Chelating Agents/pharmacokinetics ; Iron Chelating Agents/therapeutic use ; Iron Overload/drug therapy ; Iron Overload/etiology ; Male ; Young Adult
Chemical Substances	Iron Chelating Agents ; Deferiprone (2BTY8KH53L)
Language	English
Publishing date	2022-01-04
Publishing country	Germany
Document type	Clinical Trial, Phase I ; Journal Article
ZDB-ID	1064950-5
ISSN	1432-0584 ; 0939-5555 ; 0945-8077
ISSN (online)	1432-0584
ISSN	0939-5555 ; 0945-8077
DOI	10.1007/s00277-021-04728-0
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Article ; Online: Efficacy and safety of early-start deferiprone in infants and young children with transfusion-dependent beta thalassemia: Evidence for iron shuttling to transferrin in a randomized, double-blind, placebo-controlled, clinical trial (START).

Elalfy, Mohsen S / Hamdy, Mona / Adly, Amira / Ebeid, Fatma S E / Temin, Noemi Toiber / Rozova, Anna / Lee, David / Fradette, Caroline / Tricta, Fernando

American journal of hematology

2023 Volume 98, Issue 9, Page(s) 1415–1424

Abstract: Children with transfusion-dependent thalassemia (TDT) require regular blood transfusions that, without iron-chelation therapy, lead to iron-overload toxicities. Current practice delays chelation therapy (late-start) until reaching iron overload (serum ... ...

Abstract	Children with transfusion-dependent thalassemia (TDT) require regular blood transfusions that, without iron-chelation therapy, lead to iron-overload toxicities. Current practice delays chelation therapy (late-start) until reaching iron overload (serum ferritin ≥1000 μg/L) to minimize risks of iron-depletion. Deferiprone's distinct pharmacological properties, including iron-shuttling to transferrin, may reduce risks of iron depletion during mild-to-moderate iron loads and iron overload/toxicity in children with TDT. The early-start deferiprone (START) study evaluated the efficacy/safety of early-start deferiprone in infants/young children with TDT. Sixty-four infants/children recently diagnosed with beta-thalassemia and serum ferritin (SF) between 200 and 600 μg/L were randomly assigned 1:1 to receive deferiprone or placebo for 12 months or until reaching SF-threshold (≥1000 μg/L at two consecutive visits). Deferiprone was initiated at 25 mg/kg/day and increased to 50 mg/kg/day; some recipients' dosages increased to 75 mg/kg/day based on iron levels. The primary endpoint was the proportion of patients ≥SF-threshold by month 12. Monthly transferrin saturation (TSAT) assessment evaluated iron-shuttling. At baseline, there was no significant difference in mean age (deferiprone: 3.03 years, placebo: 2.63 years), SF (deferiprone: 513.8 μg/L, placebo: 451.7 μg/L), or TSAT (deferiprone: 47.98%, placebo: 43.43%) between groups. At month 12, there was no significant difference in growth or adverse event (AE) rates between groups. No deferiprone-treated patients were iron-depleted. At month 12, 66% of patients receiving deferiprone remained below SF threshold versus 39% of placebo (p = .045). Deferiprone-treated patients showed higher TSAT levels and reached ≥60% TSAT threshold faster. Early-start deferiprone was well-tolerated, not associated with iron depletion, and efficacious in reducing iron overload in infants/children with TDT. TSAT results provide the first clinical evidence of deferiprone shuttling iron to transferrin.
MeSH term(s)	Humans ; Child ; Infant ; Child, Preschool ; Iron ; beta-Thalassemia/drug therapy ; Iron Chelating Agents/adverse effects ; Transferrin ; Ferritins ; Pyridones/adverse effects ; Iron Overload/drug therapy ; Iron Overload/etiology
Chemical Substances	Iron (E1UOL152H7) ; Iron Chelating Agents ; Transferrin ; Ferritins (9007-73-2) ; Pyridones
Language	English
Publishing date	2023-07-04
Publishing country	United States
Document type	Randomized Controlled Trial ; Journal Article ; Research Support, Non-U.S. Gov't
ZDB-ID	196767-8
ISSN	1096-8652 ; 0361-8609
ISSN (online)	1096-8652
ISSN	0361-8609
DOI	10.1002/ajh.27010
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Article ; Online: Deferiprone versus deferoxamine for transfusional iron overload in sickle cell disease and other anemias: Pediatric subgroup analysis of the randomized, open-label FIRST study.

Hamdy, Mona / El-Beshlawy, Amal / Veríssimo, Mônica P A / Kanter, Julie / Inusa, Baba / Williams, Suzan / Lee, David / Temin, Noemi Toiber / Fradette, Caroline / Tricta, Fernando / Ebeid, Fatma S E / Kwiatkowski, Janet L / Elalfy, Mohsen S

Pediatric blood & cancer

2023 Volume 71, Issue 1, Page(s) e30711

Abstract: Background: Children with sickle cell disease (SCD) who are chronically transfused often, require iron chelation therapy. There are limited data that allow for comparison of the efficacy and safety of the iron chelator deferiprone versus deferoxamine in ...

Abstract	Background: Children with sickle cell disease (SCD) who are chronically transfused often, require iron chelation therapy. There are limited data that allow for comparison of the efficacy and safety of the iron chelator deferiprone versus deferoxamine in children with SCD. Methods: This post hoc analysis of the phase 3b/4, randomized, open-label FIRST (Ferriprox in Patients with IRon Overload in Sickle Cell Disease Trial) study (NCT02041299) included patients 17 years and younger with SCD or other anemias receiving deferiprone or deferoxamine. Results: Overall, 142 patients were evaluated; mean ages were 10.5 and 11.7 years in the deferiprone and deferoxamine groups, respectively. At 12 months: mean change from baseline in liver iron concentration was -3.3 mg/g dry weight (dw) with deferiprone and -3.4 mg/g dw with deferoxamine (p = .8216); relative mean change (coefficient of variation %) in log cardiac T2* magnetic resonance imaging was 1.02 (21.8%) with deferiprone and 0.95 (19.5%) with deferoxamine (p = .0717); and the mean (standard error) change in serum ferritin levels was -133.0 (200.3) μg/L with deferiprone and -467.1 (244.1) μg/L with deferoxamine (p = .2924). The most common deferiprone-related adverse events (AEs) were upper abdominal pain (20.2%), vomiting (13.8%), pyrexia (9.6%), decreased neutrophil count (9.6%), increased alanine aminotransferase (ALT; 9.6%), and increased aspartate aminotransferase (AST; 9.6%). All cases of increased ALT, increased AST, and neutropenia resolved, most without intervention. Conclusions: This post hoc analysis of pediatric patients from FIRST corroborated previous findings in adults that deferiprone is comparable to deferoxamine in reducing iron overload. No new safety concerns were observed. Deferiprone is an oral chelation option that could improve adherence and outcomes in children.
MeSH term(s)	Adult ; Child ; Humans ; Anemia, Sickle Cell/complications ; Anemia, Sickle Cell/drug therapy ; beta-Thalassemia/therapy ; Deferiprone/therapeutic use ; Deferoxamine/adverse effects ; Iron ; Iron Chelating Agents/adverse effects ; Iron Overload/drug therapy ; Iron Overload/etiology ; Neutropenia/chemically induced ; Pyridones/adverse effects
Chemical Substances	Deferiprone (2BTY8KH53L) ; Deferoxamine (J06Y7MXW4D) ; Iron (E1UOL152H7) ; Iron Chelating Agents ; Pyridones
Language	English
Publishing date	2023-10-09
Publishing country	United States
Document type	Clinical Trial, Phase III ; Clinical Trial, Phase IV ; Journal Article
ZDB-ID	2131448-2
ISSN	1545-5017 ; 1545-5009
ISSN (online)	1545-5017
ISSN	1545-5009
DOI	10.1002/pbc.30711
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Article ; Online: Rational Design of Novel Therapies for Pantothenate Kinase-Associated Neurodegeneration.

Thakur, Nivedita / Klopstock, Thomas / Jackowski, Suzanne / Kuscer, Enej / Tricta, Fernando / Videnovic, Aleksandar / Jinnah, Hyder A

Movement disorders : official journal of the Movement Disorder Society

2021 Volume 36, Issue 9, Page(s) 2005–2016

Abstract: Background: This review highlights the recent scientific advances that have enabled rational design of novel clinical trials for pantothenate kinase-associated neurodegeneration (PKAN), a rare autosomal recessive neurogenetic disorder associated with ... ...

Abstract	Background: This review highlights the recent scientific advances that have enabled rational design of novel clinical trials for pantothenate kinase-associated neurodegeneration (PKAN), a rare autosomal recessive neurogenetic disorder associated with progressive neurodegenerative changes and functional impairment. PKAN is caused by genetic variants in the PANK2 gene that result in dysfunction in pantothenate kinase 2 (PANK2) enzyme activity, with consequent disruption of coenzyme A (CoA) synthesis, and subsequent accumulation of brain iron. The clinical phenotype is varied and may include dystonia, rigidity, bradykinesia, postural instability, spasticity, loss of ambulation and ability to communicate, feeding difficulties, psychiatric issues, and cognitive and visual impairment. There are several symptom-targeted treatments, but these do not provide sustained benefit as the disorder progresses. Objectives: A detailed understanding of the molecular and biochemical pathogenesis of PKAN has opened the door for the design of novel rationally designed therapeutics that target the underlying mechanisms. Methods: Two large double-blind phase 3 clinical trials have been completed for deferiprone (an iron chelation treatment) and fosmetpantotenate (precursor replacement therapy). A pilot open-label trial of pantethine as a potential precursor replacement strategy has also been completed, and a trial of 4-phosphopantetheine has begun enrollment. Several other compounds have been evaluated in pre-clinical studies, and additional clinical trials may be anticipated. Conclusions: Experience with these trials has encouraged a critical evaluation of optimal trial designs, as well as the development of PKAN-specific measures to monitor outcomes. PKAN provides a valuable example for understanding targeted drug development and clinical trial design for rare disorders. © 2021 International Parkinson and Movement Disorder Society.
MeSH term(s)	Brain/metabolism ; Humans ; Iron ; Pantothenate Kinase-Associated Neurodegeneration/drug therapy ; Pantothenate Kinase-Associated Neurodegeneration/genetics ; Phenotype ; Phosphotransferases (Alcohol Group Acceptor)/genetics ; Randomized Controlled Trials as Topic
Chemical Substances	Iron (E1UOL152H7) ; Phosphotransferases (Alcohol Group Acceptor) (EC 2.7.1.-)
Language	English
Publishing date	2021-05-18
Publishing country	United States
Document type	Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't ; Review
ZDB-ID	607633-6
ISSN	1531-8257 ; 0885-3185
ISSN (online)	1531-8257
ISSN	0885-3185
DOI	10.1002/mds.28642
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Article ; Online: Extramedullary haematopoiesis in patients with thalassemia: a cross-sectional description of its prevalence, clinical features and survival.

Chapchap, Eduardo Cerello / Silva, Murilo Marques Almeida / Baroni, Ronaldo Hueb / Araujo, Aderson da Silva / de Assis, Reijane Alves / Loggetto, Sandra Regina / Junior, Antonio Fabron / Verissimo, Monica Pinheiro de Almeida / Baldanzi, Giorgio Roberto / Fertrin, Kleber Yotsumoto / Tricta, Fernando / Piga, Antonio Giulio / Hamerschlak, Nelson

Hematology, transfusion and cell therapy

2023

Abstract: Introduction: Despite knowledge advances on extramedullary haematopoiesis (EMH) in thalassemic patients, the real picture remains an open issue.: Objectives: To assess EMH prevalence in patients with thalassemia major (TM) and intermedia (TI), to ... ...

Abstract	Introduction: Despite knowledge advances on extramedullary haematopoiesis (EMH) in thalassemic patients, the real picture remains an open issue. Objectives: To assess EMH prevalence in patients with thalassemia major (TM) and intermedia (TI), to describe magnetic resonance imaging (MRI) findings and to explore clinical risk factors. Methods: In this cross-sectional study, images and clinical records of 184 consecutive patients with thalassemia who underwent T2* MRI between 2004 and 2011 were reviewed. Association of EMH with survival was investigated for patients with available follow-up charts. Results: EMH was detected in 16/168 (9.5%) patients with TM (aged 19-49 years) and in 3/16 (18.8%) with TI (aged 36-41 years). Most (88%) had paravertebral thoracic and/or abdominal masses. Age was significantly associated with EMH risk (hazard ratio, [HR] 1.10/year; confidence interval [CI]: 1.03-1.18; p-value < 0.001), while lower pancreatic iron content by T2MRI (HR: 0.94/ms; CI: 0.89-0.99; p-value = 0.049) was a protective factor. Estimated survival rate was superior for EMH-positive (n = 19) when compared to EMH-negative patients (n = 75) (p-value = 0.013). Conclusions:* The prevalence of EMH was 10.3% (19/184), presented mainly as tumoral masses of 3 to 10 cm. Age was a risk factor for EMH development, while lower pancreatic iron might be a protective factor in this cohort.
Language	English
Publishing date	2023-08-26
Publishing country	Brazil
Document type	Journal Article
ISSN	2531-1387
ISSN (online)	2531-1387
DOI	10.1016/j.htct.2023.07.005
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Article ; Online: The safety and acceptability of twice-daily deferiprone for transfusional iron overload: A multicentre, open-label, phase 2 study.

Badawy, Sherif M / Kattamis, Antonis / Ezzat, Hatoon / Deschamps, Benoît / Sicard, Eric / Fradette, Caroline / Zhao, Feng / Tricta, Fernando / Chung Tsang, Yu / Sheth, Sujit / Piga, Antonio

British journal of haematology

2021 Volume 197, Issue 1, Page(s) e12–e15

MeSH term(s)	Deferiprone/adverse effects ; Humans ; Iron Chelating Agents/adverse effects ; Iron Overload/drug therapy
Chemical Substances	Iron Chelating Agents ; Deferiprone (2BTY8KH53L)
Language	English
Publishing date	2021-12-21
Publishing country	England
Document type	Clinical Trial, Phase II ; Letter ; Multicenter Study ; Research Support, Non-U.S. Gov't
ZDB-ID	80077-6
ISSN	1365-2141 ; 0007-1048
ISSN (online)	1365-2141
ISSN	0007-1048
DOI	10.1111/bjh.17999
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Article ; Online: Cost-utility of chelators in transfusion-dependent β-thalassemia major patients: a review of the pharmacoeconomic literature.

Lee, Todd A / von Riedemann, Sarah / Tricta, Fernando

Expert review of pharmacoeconomics & outcomes research

2014 Volume 14, Issue 5, Page(s) 651–660

Abstract: In the inherited hematologic disorder β-thalassemia major, patients receive regular, lifelong blood transfusions, which carry excess iron that the body is unable to eliminate. Chelation therapy (deferoxamine, deferiprone, deferasirox or deferoxamine- ... ...

Abstract	In the inherited hematologic disorder β-thalassemia major, patients receive regular, lifelong blood transfusions, which carry excess iron that the body is unable to eliminate. Chelation therapy (deferoxamine, deferiprone, deferasirox or deferoxamine-deferiprone combination) is required to reduce iron accumulation in target organs and the associated morbidity and mortality. Each chelation regimen has a distinct safety/efficacy profile and particular costs associated with its use. This review aims to provide an overview of published cost-utility analyses of currently used chelation regimens, and to comment on the potential relevance of their findings in the USA market, where deferiprone has recently been introduced.
MeSH term(s)	Blood Transfusion/economics ; Cost-Benefit Analysis ; Drug Costs ; Drug Therapy, Combination ; Humans ; Iron Chelating Agents/economics ; Iron Chelating Agents/therapeutic use ; Models, Economic ; Transfusion Reaction ; Treatment Outcome ; beta-Thalassemia/blood ; beta-Thalassemia/diagnosis ; beta-Thalassemia/economics ; beta-Thalassemia/therapy
Chemical Substances	Iron Chelating Agents
Language	English
Publishing date	2014-10
Publishing country	England
Document type	Journal Article ; Review
ZDB-ID	2208481-2
ISSN	1744-8379 ; 1473-7167
ISSN (online)	1744-8379
ISSN	1473-7167
DOI	10.1586/14737167.2014.927314
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