{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,5]],"date-time":"2026-08-05T04:31:09Z","timestamp":1785904269490,"version":"3.56.0"},"reference-count":59,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2013,3,5]],"date-time":"2013-03-05T00:00:00Z","timestamp":1362441600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["American J Hematol"],"published-print":{"date-parts":[[2013,4]]},"abstract":"<jats:p>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\u2010three pediatric patients with severe \u03b2 thalassemias, age range 3.2\u201319 years, were recruited to a 1\u2010year multicenter prospective, single arm, open label, dose escalating Phase III study of deferiprone to determine its clinical efficacy and safety. Sixty\u2010four patients (87.6%) completed the study with good compliance (&gt;94%). Average deferiprone dose was 79.1\u00b14.3 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 &gt;15% from baseline at 1 year with a median reduction of 1,065 ng ml<jats:sup>\u22121<\/jats:sup>. Baseline SF was the major factor that predicts clinical efficacy; patients with baseline SF&gt;3,500 ng ml<jats:sup>\u22121<\/jats:sup> had the most significant fall of SF at 1 year. A subgroup analysis by MRI\u2010T2* 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\u2010related 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\u2013260, 2013. \u00a9 2013 Wiley Periodicals, Inc.<\/jats:p>","DOI":"10.1002\/ajh.23386","type":"journal-article","created":{"date-parts":[[2013,1,7]],"date-time":"2013-01-07T04:57:10Z","timestamp":1357534630000},"page":"251-260","source":"Crossref","is-referenced-by-count":42,"title":["Deferiprone (GPO\u2010L\u2010ONE<sup>\u00ae<\/sup>) monotherapy reduces iron overload in transfusion\u2010dependent thalassemias: 1\u2010year results from a multicenter prospective, single arm, open label, dose escalating phase III pediatric study (GPO\u2010L\u2010ONE; A001) from Thailand"],"prefix":"10.1002","volume":"88","author":[{"given":"Vip","family":"Viprakasit","sequence":"first","affiliation":[{"name":"Hematology\/Oncology Division, Department of Pediatrics and Thalassemia Center Faculty of Medicine Siriraj Hospital, Mahidol University Bangkok 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