Safety monitoring of cardiac and hepatic systems in β‐thalassemia patients with chelating treatment in Taiwan*

Peng, Ching‐Tien; Chow, Kuan‐Chih; Chen, Jeon‐Hor; Chiang, Yen-Ting; Lin, Tze–Yi; Tsai, Chang‐Hai

doi:10.1034/j.1600-0609.2003.00071.x

Cited by 54 publications

(47 citation statements)

References 38 publications

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“…Results of randomized clinical trials comparing DFO with DFP demonstrated no difference after 12 months of treatment in serum ferritin levels or liver iron concentration [23,24]. Similar results have been obtained in other studies [25,26,27]. …”

Section: Dfp Efficacysupporting

confidence: 76%

“…In two studies, DFP at 75 mg/kg/day was as effective as DFO at 50 mg/kg/day for 5–6 days per week at reducing cardiac iron by MR signal intensity ratio [24,32]. Peng et al [27] compared cardiac iron, estimated by MR signal intensity and LVEF over 3 years in 13 patients taking DFO (50 mg/kg/day at least 5 days per week) with 11 patients taking DFP (75 mg/kg/day). Cardiac iron was markedly improved in 5 patients on DFP but only in 2 patients on DFO treatment.…”

Section: Dfp Efficacymentioning

confidence: 99%

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Deferiprone Chelation Therapy for Thalassemia Major

Galanello

Campus²

2009

Acta Haematol

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Iron overload is one of the major causes of morbidity in patients with thalassemia major. Deferiprone (DFP), an orally active iron chelator, emerged from an extensive search for new drugs to treat iron overload. Comparative studies have shown that at comparable doses the efficacy of DFP in removing body iron is similar to that of desferoxamine (DFO). In retrospective and prospective studies, DFP monotherapy was significantly more effective than DFO in the treatment of myocardial siderosis in thalassemia major. DFP can be used in combination with DFO in the management of severe iron overload. This chelation regimen is tolerable and attractive for patients unable to comply with standard DFO infusions or with inadequate response to DFP monotherapy. DFP has a well-known long-term safety profile. Agranulocytosis is the most serious side effect associated with its use, occurring in about 1% of the patients. More common but less serious side effects are gastrointestinal symptoms, arthralgia, zinc deficiency, and fluctuating transaminase levels.

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Section: Dfp Efficacysupporting

confidence: 76%

Section: Dfp Efficacymentioning

confidence: 99%

Deferiprone Chelation Therapy for Thalassemia Major

Galanello

Campus²

2009

Acta Haematol

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“…The incidence of fluctuation of hepatic transaminase levels after deferiprone exposure found in our Thai patients seemed to be higher than Caucasian population [21,42,54] but somewhat similar to Chinese patients [41,59]. Around 50% of the incidences were mild and transient; patients could continue on the study drug without interruption.…”

Section: Discussionsupporting

confidence: 54%

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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“…The researchers found no progression of hepatic fibrosis or increased liver iron stores in most patients with deferiprone treatment [46]. Another Taiwanese study group conducted a prospective 3 year study comparing cardiac and hepatic efficacy and safety of chelating treatment with deferiprone and deferoxamine [47]. Among 24 patients studied, 11 were switched from deferoxamine to deferiprone due to unwillingness or intolerability while the remaining 13 patients stayed on deferoxamine.…”

Section: Clinical Experiencementioning

confidence: 97%

“…There was a marginal benefit in reducing cardiac iron accumulation and increasing cardiac function in the deferiprone group. The study group hence recommended the use of deferiprone in patients who failed on deferoxamine treatment [47].…”

Section: Clinical Experiencementioning

confidence: 99%

Iron chelation therapy in the management of thalassemia: the Asian perspectives

et al. 2009

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Worldwide, thalassemia is the most commonly inherited hemolytic anemia, and it is most prevalent in Asia and the Middle East. Iron overload represents a significant problem in patients with transfusion-dependent beta-thalassemia. Chelation therapy with deferoxamine has traditionally been the standard therapeutic option but its usage is tempered by suboptimal patient compliance due to the discomfort and demands associated with the administration regimen. Therefore, a great deal of attention has been focused on the development of oral chelating agents. Deferiprone, even though available for nearly two decades in Asia with recent encouraging data on cardiac iron removal and long-term efficacy, has serious adverse effects including agranulocytosis and neutropenia which has impeded it from routine clinical practice. A novel oral chelator; deferasirox is effective throughout a 24 h dosing period and both preclinical and clinical data indicate that it successfully removes both hepatic and cardiac iron. In Asia, optimal management of severe thalassemia patients and the availability and access to oral iron chelators still presents a major challenge in many countries. In this regard, the development and implementation of consensus guidelines for management of Asian patients with transfusion-dependent thalassemia will be a major step towards improving and maintaining the continuity of patient care.

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Safety monitoring of cardiac and hepatic systems in β‐thalassemia patients with chelating treatment in Taiwan*

Cited by 54 publications

References 38 publications

Deferiprone Chelation Therapy for Thalassemia Major

Deferiprone Chelation Therapy for Thalassemia Major

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

Iron chelation therapy in the management of thalassemia: the Asian perspectives

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Safety monitoring of cardiac and hepatic systems in β‐thalassemia patients with chelating treatment in Taiwan*

Cited by 54 publications

References 38 publications

Deferiprone Chelation Therapy for Thalassemia Major

Deferiprone Chelation Therapy for Thalassemia Major

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

Iron chelation therapy in the management of thalassemia: the Asian perspectives

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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