Side Effects of deferiprone: A Synthesis of Findings from 24 Studies

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Original Abstract of the Article

Major Research Findings

Numerous studies have been conducted on the safety and effectiveness of deferiprone, many of which demonstrate its effectiveness in treating iron overload in patients with thalassemia. 13 , 10 , 2 , 22 , 8 These studies indicate that deferiprone is an effective oral iron chelating agent for the treatment of iron overload. Notably, it has shown potential to be superior to the conventional iron chelating agent, deferoxamine, in preventing iron accumulation in the heart. 13 However, it has also been suggested that using high doses could increase the risk of serious side effects. 12

Reasons for Side Effects

The side effects of deferiprone stem from its iron chelating action. Deferiprone binds to iron in the body, promoting its excretion in urine. This process may potentially affect cells during the binding process. Additionally, the small molecular structure of deferiprone is also considered a contributing factor to its side effects. The small molecular size allows it to easily penetrate cell membranes and readily enter cells. 15

Common Side Effects

Agranulocytosis

One of the most serious side effects of deferiprone is agranulocytosis, a condition where the number of granulocytes, a type of white blood cell, decreases significantly. Granulocytes play a crucial role in protecting the body from pathogens like bacteria. Agranulocytosis increases susceptibility to infections. 13 , 2 , 8 Agranulocytosis can occur a few weeks to months after starting deferiprone. If you experience symptoms like fever, sore throat, or cough while taking deferiprone, consult a doctor immediately.

Neutropenia

Neutropenia is a condition where the number of neutrophils, a type of white blood cell, decreases. Neutrophils are essential for protecting the body from bacterial infections. Neutropenia makes you more prone to infections. 20 , 8

Gastrointestinal Symptoms

Gastrointestinal symptoms such as nausea, vomiting, abdominal pain, and diarrhea have been reported. 13 , 10 , 2 , 8 These symptoms may appear soon after starting deferiprone. If you experience any gastrointestinal discomfort, consult a doctor.

Arthropathy

Arthropathy, joint pain, can occur with deferiprone treatment. It commonly affects the joints of hands and feet, often accompanied by pain and swelling. 13 , 10 , 2 , 8 If you experience joint pain, consult a doctor.

Liver Function Abnormalities

Liver function abnormalities can occur with deferiprone treatment. This indicates impaired liver function. The liver plays a vital role in detoxifying the body and storing nutrients. Liver function abnormalities can lead to symptoms like jaundice or ascites. 13 , 10 , 2 If you experience concerns about liver function abnormalities, consult a doctor.

Zinc Deficiency

Zinc deficiency can occur with deferiprone treatment. Zinc is crucial for growth and immune function in the body. Zinc deficiency can lead to symptoms like taste disturbances, hair loss, and skin rashes. 13 , 2 If you experience concerns about zinc deficiency, consult a doctor.

Other

In addition to the side effects mentioned above, others such as rash, headache, and fatigue have also been reported. 13 , 10 , 2 , 8 If you experience any concerning symptoms, consult a doctor.

Side Effects Management

Agranulocytosis Management

Agranulocytosis may improve by discontinuing deferiprone treatment. Antibiotics may also be administered to prevent infections. 2 If you experience fever, sore throat, or cough while taking deferiprone, consult a doctor immediately.

Neutropenia Management

Preventing infections is crucial for managing neutropenia. Practice good hygiene, like handwashing and gargling, and avoid crowded places. Antibiotics may also be prescribed as needed. 20

Gastrointestinal Symptoms Management

For gastrointestinal discomfort, dietary changes and medication may be recommended. Dietary modifications include consuming easily digestible foods and avoiding irritants. Anti-nausea or anti-diarrhea medications may be prescribed. 10 , 2

Arthropathy Management

To manage joint pain, painkillers or nonsteroidal anti-inflammatory drugs (NSAIDs) may be prescribed. Physical therapy such as heat therapy or massage may also be beneficial. 10 , 2

Liver Function Abnormalities Management

For liver function abnormalities, deferiprone treatment may be discontinued. Medications to support liver function may also be prescribed. 10

Zinc Deficiency Management

For zinc deficiency, zinc-containing supplements are recommended. Foods rich in zinc include oysters, beef, and liver. 13 , 2

Comparison Across Studies

Commonalities Across Studies

The iron chelating effect of deferiprone has been confirmed across many studies. Numerous studies also employ common indicators to assess the effectiveness and safety of deferiprone. For instance, serum ferritin levels and heart T2* values are widely used metrics for evaluating deferiprone's effectiveness. Additionally, hematological side effects such as agranulocytosis and neutropenia are crucial indicators in the safety assessment of deferiprone.

Differences Across Studies

The dosage and duration of deferiprone administration vary between studies. The administration methods also differ, with oral administration, intravenous infusion, and other methods employed in different studies. These variations make it challenging to directly compare research findings. Additionally, comparative studies between deferiprone and other iron chelators often utilize diverse patient populations and assessment criteria, making comparison of results difficult.

Considerations for Real-World Application

Deferiprone is an effective medication for treating iron overload, but it is essential to be aware of the potential for serious side effects. When taking deferiprone, always follow your doctor's instructions and undergo regular blood tests. If you experience symptoms such as fever, sore throat, or cough, consult your doctor immediately.

Limitations of Current Research

Research on the safety and effectiveness of deferiprone remains limited. In particular, the long-term effects of deferiprone are not fully understood due to insufficient data. Additionally, the mechanisms underlying the side effects caused by deferiprone are not entirely elucidated. Further research is necessary to optimize the dosage and duration of deferiprone treatment.

Future Research Directions

Large-scale clinical trials are needed to thoroughly assess the safety and effectiveness of deferiprone. Basic research is required to uncover the mechanisms behind the side effects caused by deferiprone. Research on optimizing the dosage and duration of deferiprone treatment is also essential.

Conclusion

Deferiprone is an effective oral iron chelating agent for the treatment of iron overload. However, it is crucial to be aware of the potential for serious side effects. Always follow your doctor's instructions and undergo regular blood tests while taking deferiprone.

Keywords
Benefit Keywords
Iron overload treatment
2
Chelation therapy
3
Iron chelation
4
Reduced ferritin levels
5
Iron chelation therapy
6
BMT patient care
hematological cancer treatment
7
Iron chelation
Iron overload treatment
8
Brain iron reduction
Neurodegenerative disease treatment
9
Reduced serum ferritin
10
Cardiac function improvement
Thalassemia prognosis improvement
11
Chelate iron
Prevent iron overload
13
Improved survival
Iron chelation therapy
14
Thalassemia Treatment
16
Antibacterial effect
Reduced antibiotic use
Vibriosis treatment
17
Improved cardiac function
Reduced endocrine dysfunction
18
Effective iron chelation
19
Neurodegeneration treatment
21
Improved compliance
Iron chelation
22
Iron chelation for malaria
24
Risk Keywords
Agranulocytosis
Neutropenia
1
Agranulocytosis
Arthralgia
Fluctuating transaminase levels
Gastrointestinal symptoms
Zinc deficiency
2
Arthralgia
Nausea
Skin reactions
3
Iron poisoning
4
Arthropathy
Liver enzyme elevation
Nausea
Neutropenia
5
Side effects
6
noncompliance to therapy
relapse of diseases
side effects
7
Agranulocytosis
Neutropenia
8
Hematologic side effects
Neurologic side effects
9
Arthralgia
Gastrointestinal discomfort
10
Agranulocytosis
Arthropathy
Gastrointestinal symptoms
Neutropenia
Thrombocytopenia
11
Agranulocytosis
Alopecia
Anaphylactic reactions
Gastric intolerance
Gastrointestinal hemorrhage
Hepatic failure
Renal failure
Renal tubulopathy
Skin rash
12
Agranulocytosis
Arthropathy
Gastrointestinal symptoms
Liver enzyme changes
Neutropenia
Zinc deficiency
13
Iron overload complications
14
Neurological toxicity
Visual side effects
15
Thalassemia Complications
16
Side effects
18
Safety concerns
19
Increased risk of agranulocytosis
Increased risk of neutropenia
20
Discontinuation rates
Side effects
22
Life-threatening side effects
23
Literature analysis of 24 papers
Positive Content
19
Neutral Content
0
Negative Content
5
Article Type
2
1
2
8
23
3.

Comparison between desferrioxamine and combined therapy with desferrioxamine and deferiprone in iron overloaded thalassaemia patients.

Author: MouradFadi H, HoffbrandA Victor, Sheikh-TahaMarwan, KoussaSusane, KhoriatyAdlette I, TaherAli

Chelation therapy
Arthralgia
Nausea
Skin reactions

Combined therapy with deferiprone and desferrioxamine was as effective as desferrioxamine alone in chelating iron in patients with thalassaemia.

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
Comparative StudyResearch that compares multiple groups/interventions to determine differences/similarities in outcomes/characteristics.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.

Language : English

4.

Iron; Benefits or threatens (with emphasis on mechanism and treatment of its poisoning).

Author: Rafati RahimzadehMehrdad, Rafati RahimzadehMehravar, KazemiSohrab, MoghadamniaAhmad Reza, Ghaemi AmiriMaryam, MoghadamniaAli Akbar

Iron chelation
Iron poisoning

Iron poisoning causes clinical manifestations in the gastrointestinal tract, liver, heart, kidneys, and hematopoietic system. Treatment includes whole bowel irrigation, esophagogastroduodenoscopy, and chelation therapy with agents like deferoxamine mesylate, deferasirox, deferiprone, and deferitrin.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

5.

The effect of combined therapy with deferoxamine and deferiprone on serum ferritin level of beta-thalassemic patients.

Author: MirbehbahaniNarges, JahaziAzam, Rahim AbadHanif Hashem Nejad

Reduced ferritin levels
Arthropathy
Liver enzyme elevation
Nausea
Neutropenia

Combined therapy with deferoxamine and deferiprone significantly decreased serum ferritin level in major beta-thalassemic patients.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

6.

The Efficacy of Iron Chelators for Removing Iron from Specific Brain Regions and the Pituitary-Ironing out the Brain.

Author: CrichtonRobert R, WardRoberta J, HiderRobert C

Iron chelation therapy
Side effects

Iron chelation therapy is an essential therapy for beta thalassemia patients. This study discussed the potential role of inflammation in chelation efficacy.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

8.

Deferiprone for the treatment of transfusional iron overload in thalassemia.

Author: BelmontAmi, KwiatkowskiJanet L

Iron chelation
Iron overload treatment
Agranulocytosis
Neutropenia

This article reviews the efficacy, adverse effects, and use of deferiprone, an oral iron chelator used to treat transfusional iron overload.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

9.

Selective iron chelation in Friedreich ataxia: biologic and clinical implications.

Author: BoddaertNathalie, Le Quan SangKim Hanh, RötigAgnès, Leroy-WilligAnne, GalletSerge, BrunelleFrancis, SidiDaniel, ThalabardJean-Christophe, MunnichArnold, CabantchikZ Ioav

Brain iron reduction
Neurodegenerative disease treatment
Hematologic side effects
Neurologic side effects

Deferiprone, a chelator, was able to reduce iron accumulation in the brain of Friedreich ataxia patients.

Clinical Trial, Phase IThe first stage of clinical trials in humans. Evaluates the safety, tolerability, and pharmacokinetics of a new drug/treatment.
Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

10.

Safety and effectiveness of 100 mg/kg/day deferiprone in patients with thalassemia major: a two-year study.

Author: TaherAli, Sheikh-TahaMarwan, ShararaAla, InatiAdlette, KoussaSuzane, EllisGareth, DhillonA P, HoffbrandA V

Reduced serum ferritin
Arthralgia
Gastrointestinal discomfort

Deferiprone at a dose of 100 mg/kg/day is safe and effective for long-term therapy in patients with iron overload. The most common side effects were transient aspartate aminotransferase increase, gastrointestinal discomfort, and arthralgia.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

14.

Iron Chelators in Treatment of Iron Overload.

Author: EntezariSarina, HaghiSeyedeh Mona, NorouzkhaniNarges, SahebnazarBarsa, VosoughianFatemeh, AkbarzadehDiba, IslampanahMuhammad, NaghshNavid, AbbasalizadehMohammad, DeraviNiloofar

Improved survival
Iron chelation therapy
Iron overload complications

Iron overload can cause serious complications, and iron chelation therapy has significantly improved patient survival. This review introduces three FDA-approved chelators (deferoxamine, deferiprone, and deferasirox), their advantages, disadvantages, and potential of combination therapy.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

15.

Neurological Complications and Cataract in a Child With Thalassemia Major Treated With Deferiprone.

Author: ParakhNupur, SharmaRajni, PrakashOm, MahtoDeoNath, DhingraBhavna, SharmaSuvasini, ChandraJagdish

Neurological toxicity
Visual side effects

Deferiprone is an oral iron chelator. This case report describes rare neurological manifestations of cerebellar ataxia, hypertonia, and bilateral cataract in an 11-year-old boy with thalassemia major on deferiprone therapy.

Case ReportsIn the medical field, a report that describes in detail a unique case, an unusual medical condition, or the effect of a treatment. Provides new insights and possibilities for treatment through individual patient cases.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

18.

A systematic review and meta-analysis of deferiprone monotherapy and in combination with deferoxamine for reduction of iron overload in chronically transfused patients with β-thalassemia.

Author: KuoKevin H M, MrkobradaMarko

Improved cardiac function
Reduced endocrine dysfunction
Side effects

β-Thalassemia major patients require life-long blood transfusions, leading to iron overload. This meta-analysis of 15 RCTs found that deferiprone was more effective than deferoxamine in improving cardiac ejection fraction and endocrine dysfunction, while the combination of both drugs showed better results for cardiac ejection fraction. More research is needed to confirm these findings and determine the best treatment approach for iron overload in β-Thalassemia major.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

19.

Overview of iron chelation therapy with desferrioxamine and deferiprone.

Author: CappelliniMaria D, MusallamKhaled M, TaherAli T

Effective iron chelation
Safety concerns

This article discusses the use of iron chelators, desferrioxamine and deferiprone, to treat chronic iron overload from frequent blood transfusions. While both drugs are effective, deferiprone offers the advantage of oral administration. However, regular monitoring is required due to potential safety concerns.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

21.

A pilot trial of deferiprone for neurodegeneration with brain iron accumulation.

Author: AbbruzzeseGiovanni, CossuGiovanni, BaloccoManuela, MarcheseRoberta, MurgiaDaniela, MelisMaurizio, GalanelloRenzo, BarellaSusanna, MattaGildo, RuffinengoUberto, BonuccelliUbaldo, ForniGian Luca

Neurodegeneration treatment

Deferiprone is a safe and potentially effective treatment for neurodegeneration with brain iron accumulation. In a pilot study, 4 patients with pantothenate kinase-associated neurodegeneration and 2 patients with parkinsonism and focal dystonia received deferiprone. Two patients with pantothenate kinase-associated neurodegeneration showed decreased iron accumulation in the globus pallidus, and 3 patients experienced mild-to-moderate motor improvement.

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

23.

A record number of fatalities in many categories of patients treated with deferasirox: loopholes in regulatory and marketing procedures undermine patient safety and misguide public funds?

Author: KontoghiorghesGeorge J

Life-threatening side effects

Deferasirox, despite warnings of life-threatening toxic side effects, has been associated with a high mortality rate. Concerns exist regarding its indiscriminate use.

EditorialAn article in which the editor or publisher expresses their opinion or perspective on a particular topic or issue. Its purpose is to convey a specific message or idea to the reader.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

24.

Iron chelating agents for treating malaria.

Author: SmithH J, MeremikwuM

Iron chelation for malaria

This study examines the effects of iron chelators in improving patient outcomes in malaria, exploring the potential of iron chelation as an adjunctive therapy to standard antimalarial regimens.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

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