Oral chelators in transfusion-dependent thalassemia major patients may prevent or reverse iron overload complications

Farmaki, Kallistheni; Tzoumari, Ioanna; Pappa, Christina

doi:10.1016/j.bcmd.2011.03.007

Cited by 82 publications

(81 citation statements)

References 49 publications

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“…In addition, patients could be safely prevented from future cardiac and hepatic problems at this SF level. To achieve this goal, it might be sensible to increase the dose of deferiprone up to 100 mg/kg/day [53,54] or combine deferiprone with 3-4 days a week of desferoxamine (40-60 mg kg 21 ) [55] in patients who are nonresponse patients after 6-month evaluation of deferiprone monotherapy or in patients who subsequently lose their primary response to deferiprone when their iron load status have declined from the start but remain higher than needed normalized levels [56]. However, this approach must be treated with caution under a rigorous assessment since experience with deferoxamine has suggested that lowering iron chelator doses should be used when iron levels are low to minimize drug toxicity [6].…”

Section: Discussionmentioning

confidence: 99%

Deferiprone (GPO‐L‐ONE^®) monotherapy reduces iron overload in transfusion‐dependent thalassemias: 1‐year results from a multicenter prospective, single arm, open label, dose escalating phase III pediatric study (GPO‐L‐ONE; A001) from Thailand

et al. 2013

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Accessibility to iron chelators including deferoxamine and deferasirox remains obscured in many developing countries. To provide an alternative, the government pharmaceutical organization of Thailand (GPO) manufactured deferiprone which has similar bioequivalent to the standard product. Seventy-three pediatric patients with severe b thalassemias, age range 3.2-19 years, were recruited to a 1-year multicenter prospective, single arm, open label, dose escalating Phase III study of deferiprone to determine its clinical efficacy and safety. Sixty-four patients (87.6%) completed the study with good compliance (>94%). Average deferiprone dose was 79.164.3 mg/kg/day. Overall, mean serum ferritin (SF) levels at 1 year were not significantly changed from baseline. However, 45% of patients (response group) had SF reduced >15% from baseline at 1 year with a median reduction of 1,065 ng ml 21 . Baseline SF was the major factor that predicts clinical efficacy; patients with baseline SF>3,500 ng ml 21 had the most significant fall of SF at 1 year. A subgroup analysis by MRI-T2* confirmed that the response group had higher baseline liver iron and deferiprone could significantly reduce liver iron overload and normalize levels of ALT at 1 year. Although, gastrointestinal irritation (20.5%) was the most common drug-related adverse events (AEs) followed by transaminitis (16.4%) and neutropenia (6.8%), all patients were well tolerated. There was no mortality and agranulocytosis found in this trial. Monotherapy of deferiprone with appropriate dose adjustment and monitoring for adverse events appeared to be an effective chelation therapy in some patients with good compliance and acceptable safety profiles. Am. J. Hematol. 88:251-260,

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Section: Discussionmentioning

confidence: 99%

Deferiprone (GPO‐L‐ONE^®) monotherapy reduces iron overload in transfusion‐dependent thalassemias: 1‐year results from a multicenter prospective, single arm, open label, dose escalating phase III pediatric study (GPO‐L‐ONE; A001) from Thailand

et al. 2013

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“…In the largest, 16 patients were treated with DFP 75-100 mg/kg per day in 3 divided doses together with DFX 20-25 mg/kg each day. 85 There was a fall in total body iron measured by serum ferritin, liver iron and cardiac iron measured by T2* MRI. Improvements occurred in LVEF, gonadal function, and glucose metabolism.…”

Section: Combined Therapy: Dfp and Dfxmentioning

confidence: 98%

How I treat transfusional iron overload

Hoffbrand

Taher

Cappellini

2012

Blood

180

150

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Patients with ␤-thalassemia major (TM) and other refractory anemias requiring regular blood transfusions accumulate iron that damages the liver, endocrine system, and most importantly the heart. The prognosis in TM has improved remarkably over the past 10 years. This improvement has resulted from the development of magnetic resonance imaging (MRI) techniques, especially T2*, to accurately measure cardiac and liver iron, and from the availability of 3 iron-chelating drugs. In this article we describe the use of MRI to determine which adult and pediatric patients need to begin iron chelation therapy and to monitor their progress. We summarize the properties of each of the 3 drugs, deferoxamine (DFO), deferiprone (DFP), and deferasirox (DFX), including their efficacy, patient acceptability, and side effects. We describe when to initiate or intensify therapy, switch to another drug, or use combined therapy. We also discuss the management of refractory anemias other than TM that may require multiple blood transfusions, including sickle cell anemia and myelodysplasia.

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“…This may be kept by the patients every day and can be reviewed by the treating physician at relevant time periods. 19 This can help physiciansto make note of the serum ferritin results and of the remaining total body iron load indices. Limitations: As the data available in this clinical study is very less due to less number of participated subjects been involved in the study, a large number of subjects can provide extensive results regarding the efficacy and safety of Deferasirox.…”

Section: Effect On Renal Functionmentioning

confidence: 99%

Study on Safety and Efficacy of Deferasirox in the treatment of Thalassemia in a South Indian Tertiary Care Hospital

Y¹,

Kamrthi²,

Kumar³

2015

IJOPP

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Background: There were very less number of research studies been done in Indian settings or around the world about the efficacy and safety of Deferasirox in thalassemia patients. The objective of this study was to assess the efficacy and safety of Deferasirox in thalassemia patients. Methodology: It is a prospective, observational study conducted for a period of eight months that assessed the laboratory data of thalassemia patients aged ≤ 15 years who were on Deferasirox therapy. The study was conducted in a tertiary care hospital situated in Nellore Andhra Pradesh, India. The study involved the day-to-day review of medication orders and laboratory data in the pediatrics wards. Evaluation included the collection of laboratory data such as serum ferritin levels for the efficacy assessment and alanine transaminase (ALT), serum creatinine, White Blood Cells (WBC) counts and platelet counts for safety assessment. Results: Total numbers of patients enrolled in the study were twenty-four out of 63 thalassemia patients treated in the hospital. Efficacy outcomes resulted that 18 (75%) out of 24 patients responded positively to the Deferasirox, whereas remaining 6 (25%) patients were observed with no changes in their serum ferritin levels. The safety outcomes stated that 11 (45.83%) out of 24 patients were observed with increased ALT levels whereas, the other safety parameters such as serum creatinine, WBCs and platelet counts were observed to be with mild deviations causing no clinical harm to the patients. Conclusion: This study has shown a consistent efficacy in 75% of patients and a manageable safety profile. Thus, it demonstrates that the oral chelation therapy of Deferasirox was efficient and well tolerated, especially in moderately iron loaded transfusion-dependent thalassemia patients.

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Oral chelators in transfusion-dependent thalassemia major patients may prevent or reverse iron overload complications

Cited by 82 publications

References 49 publications

Deferiprone (GPO‐L‐ONE^®) monotherapy reduces iron overload in transfusion‐dependent thalassemias: 1‐year results from a multicenter prospective, single arm, open label, dose escalating phase III pediatric study (GPO‐L‐ONE; A001) from Thailand

Deferiprone (GPO‐L‐ONE^®) monotherapy reduces iron overload in transfusion‐dependent thalassemias: 1‐year results from a multicenter prospective, single arm, open label, dose escalating phase III pediatric study (GPO‐L‐ONE; A001) from Thailand

How I treat transfusional iron overload

Study on Safety and Efficacy of Deferasirox in the treatment of Thalassemia in a South Indian Tertiary Care Hospital

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Oral chelators in transfusion-dependent thalassemia major patients may prevent or reverse iron overload complications

Cited by 82 publications

References 49 publications

Deferiprone (GPO‐L‐ONE®) monotherapy reduces iron overload in transfusion‐dependent thalassemias: 1‐year results from a multicenter prospective, single arm, open label, dose escalating phase III pediatric study (GPO‐L‐ONE; A001) from Thailand

Deferiprone (GPO‐L‐ONE®) monotherapy reduces iron overload in transfusion‐dependent thalassemias: 1‐year results from a multicenter prospective, single arm, open label, dose escalating phase III pediatric study (GPO‐L‐ONE; A001) from Thailand

How I treat transfusional iron overload

Study on Safety and Efficacy of Deferasirox in the treatment of Thalassemia in a South Indian Tertiary Care Hospital

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Product

Resources

About

Deferiprone (GPO‐L‐ONE^®) monotherapy reduces iron overload in transfusion‐dependent thalassemias: 1‐year results from a multicenter prospective, single arm, open label, dose escalating phase III pediatric study (GPO‐L‐ONE; A001) from Thailand

Deferiprone (GPO‐L‐ONE^®) monotherapy reduces iron overload in transfusion‐dependent thalassemias: 1‐year results from a multicenter prospective, single arm, open label, dose escalating phase III pediatric study (GPO‐L‐ONE; A001) from Thailand