Deferiprone, efficacy and safety

Abstract

Deferiprone is an effective iron chelator, but arthropathy and neutropenia are very frequent side effects and need strict monitoring during therapy. Most of the neutropenia are neither very severe nor recur with re-challenge with the drug. Similarly, arthropathy does not need withdrawal of drug in majority of patients.

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

“…AEs identified in our present trial were manageable and comparable with previous studies and with other generic product (Kelfer V R , Cipla, Mumbai, India) used previously in Thai patients [28] (Supporting Information Table VIII). Moreover, the incidence of three common adverse events including gastrointestinal irritation, raised liver enzyme and neutropenia (22.7, 18 and 7.5/100 patient-year, respectively) found in our study were similar with previous reports in other ethnicities [21,39,40,57] with a relatively higher incidence of neutropenia in our population but none developed agranulocytosis. The majority of Thai patients with neutropenia had intact spleen and the average ANC counts were relatively higher in patients with splenectomy throughout the study period as observed previously (Fig.…”

“…Interestingly, the total incidence of arthropathy found in our present study (3%/100 patient-year) seemed to be relatively low among Asian patients so far compared to other ethnicities, in particular, Indian population where this adverse event was found in up to 40% [40]. Although, different genetic background, levels of iron loaded, dose of deferiprone might play an important role on such difference, however different manufacturing products used in different studies might be partially contributing to such dissimilarity as the incidence of arthropathy was also higher in another Thai study in patients who received imported deferiprone with an incidence of 8.3%, similar to those of young Indian pediatric patients reported previously (9.1%) [57] (Supporting Information Table VIII).…”

“…These groups of patients were inadequately treated or chelation naive with very high levels of baseline serum ferritin and high iron intake from blood transfusion [11,[36][37][38][39][40][41]. Furthermore our findings that SF values were more likely to decrease in those patients who began with the highest SF values and lower iron intake from lower transfusion rate (<0.25 mg/kg/day in our study) are consistent with previous individual studies [42][43][44] and review of trends from several studies [38].…”

Iron overload in beta thalassemia major patients

“…Moreover, similar studies carried out in other South Asian countries have reported high mean ferritin levels when compared to values of this study. Mean serum ferritin levels reported to be 4236.5 ng/ml, 6723 ng/ml and 3272.5 ng/ml in studies conducted in Pakistan [18], India [33] and Saudi Arabia [34] respectively. However, Cunningham et al reported that serum ferritin level was 1696 ng/ml and Eghbali et al reported that mean serum ferritin was 1927 ng/ml in studies conducted in North America [35] and Iran [36] respectively.…”

The synthesis, molecular structure and spectra properties of sulphur and selenium deferiprone analogues

“…Some compounds found in plants have similar capabilities of chelating metal ions, among them are maltol, mimosine and 1,2-tropolone with structures similar to that of deferiprone. 2 Due to its iron(III) ion chelation properties, deferiprone has been extensively studied and used in treating thalassemia in children 3 and adults. 4 Unlike desferrioxamine, which was used previously, deferiprone can be administered orally.…”