{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T16:36:19Z","timestamp":1790786179138,"version":"4.1.0"},"reference-count":19,"publisher":"American Society of Hematology","issue":"5","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2003,9,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>The identification of a safe, orally active iron chelator is critically important for the prevention of morbidity and early death in patients receiving regular red cell transfusions. Based on our findings in a 1-year multicenter, prospective study of the safety and efficacy of deferiprone in patients with thalassemia major, we have extended the treatment period to 4 years. The mean dose of the chelator was 73 mg\/kg per day during 531 patient-years. The rates of agranulocytosis (absolute neutrophil count [ANC] &amp;lt; 500 \u00d7 109\/L) and milder forms of neutropenia (ANC, 500-1500 \u00d7 109\/L) were 0.2 and 2.8 per 100 patient-years, respectively. Neutropenia occurred significantly more commonly in patients with intact spleens. Gastrointestinal and joint symptoms decreased significantly after the first year of therapy, and led to discontinuation of deferiprone in only one patient in years 2 to 4. The mean alanine aminotransferase (ALT) value of 71 U\/L after 4 years of therapy was significantly higher than the baseline value of 61 U\/L. Trend analysis showed no increase in the ALT levels or the percentage of patients with ALT levels greater than twice the upper limit of the reference range. Ferritin levels did not change significantly from the values at the time of change from deferoxamine to deferiprone in either the intention-to-treat analysis or in the 84 patients who completed 4 years of therapy. Because of concerns regarding the effectiveness of the studied dose of deferiprone, 47 patients discontinued therapy, whereas 15 patients interrupted therapy because of concerns regarding low iron levels. The results of this study help to define the safety and effectiveness of long-term therapy with deferiprone.<\/jats:p>","DOI":"10.1182\/blood-2002-10-3280","type":"journal-article","created":{"date-parts":[[2003,5,26]],"date-time":"2003-05-26T20:16:07Z","timestamp":1053980167000},"page":"1583-1587","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":236,"title":["Safety and effectiveness of long-term therapy with the oral iron chelator deferiprone"],"prefix":"10.1182","volume":"102","author":[{"given":"Alan R.","family":"Cohen","sequence":"first","affiliation":[{"name":"From the Children's Hospital of Philadelphia, Philadelphia, PA; Ospedale Regionale Microcitemie ASL8, Dipartimento Scienze Biomediche e Biotecnologie, Universita\u0300 di Cagliari, Cagliari, Italy; Dipartimento Scienze Pediatriche e dell'Adolescenza, Universita\u0300 di Torino, Torino, Italy; Divisione di Pediatria ed Adolescentologia, Arcispedale S. Anna, Ferrara, Italy; and Apotex Research, Toronto, ON, Canada."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Renzo","family":"Galanello","sequence":"additional","affiliation":[{"name":"From the Children's Hospital of Philadelphia, Philadelphia, PA; Ospedale Regionale Microcitemie ASL8, Dipartimento Scienze Biomediche e Biotecnologie, Universita\u0300 di Cagliari, Cagliari, Italy; Dipartimento Scienze Pediatriche e dell'Adolescenza, Universita\u0300 di Torino, Torino, Italy; Divisione di Pediatria ed Adolescentologia, Arcispedale S. Anna, Ferrara, Italy; and Apotex Research, Toronto, ON, Canada."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonio","family":"Piga","sequence":"additional","affiliation":[{"name":"From the Children's Hospital of Philadelphia, Philadelphia, PA; Ospedale Regionale Microcitemie ASL8, Dipartimento Scienze Biomediche e Biotecnologie, Universita\u0300 di Cagliari, Cagliari, Italy; Dipartimento Scienze Pediatriche e dell'Adolescenza, Universita\u0300 di Torino, Torino, Italy; Divisione di Pediatria ed Adolescentologia, Arcispedale S. Anna, Ferrara, Italy; and Apotex Research, Toronto, ON, Canada."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vincenzo","family":"De Sanctis","sequence":"additional","affiliation":[{"name":"From the Children's Hospital of Philadelphia, Philadelphia, PA; Ospedale Regionale Microcitemie ASL8, Dipartimento Scienze Biomediche e Biotecnologie, Universita\u0300 di Cagliari, Cagliari, Italy; Dipartimento Scienze Pediatriche e dell'Adolescenza, Universita\u0300 di Torino, Torino, Italy; Divisione di Pediatria ed Adolescentologia, Arcispedale S. Anna, Ferrara, Italy; and Apotex Research, Toronto, ON, Canada."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fernando","family":"Tricta","sequence":"additional","affiliation":[{"name":"From the Children's Hospital of Philadelphia, Philadelphia, PA; Ospedale Regionale Microcitemie ASL8, Dipartimento Scienze Biomediche e Biotecnologie, Universita\u0300 di Cagliari, Cagliari, Italy; Dipartimento Scienze Pediatriche e dell'Adolescenza, Universita\u0300 di Torino, Torino, Italy; Divisione di Pediatria ed Adolescentologia, Arcispedale S. Anna, Ferrara, Italy; and Apotex Research, Toronto, ON, Canada."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"234","reference":[{"key":"2020021300113290800_REF1","doi-asserted-by":"crossref","unstructured":"Olivieri NF, Brittenham GM. Iron-chelating therapy and the treatment of thalassemia. Blood. 1997;89: 739-761.","DOI":"10.1182\/blood.V89.3.739"},{"key":"2020021300113290800_","unstructured":"Cohen AR, Porter JB. Transfusion and iron chelation therapy in thalassemia and sickle cell anemia. In: Steinberg MH, Forget BG, Higgs DR, Nagel RL, eds. Disorders of Hemoglobin. Cambridge, United Kingdom: Cambridge University Press; 2001: 979-1027."},{"key":"2020021300113290800_REF3","doi-asserted-by":"crossref","unstructured":"Porter JB. Practical management of iron overload. Brit J Haematol. 2001;115: 239-252.","DOI":"10.1046\/j.1365-2141.2001.03195.x"},{"key":"2020021300113290800_REF4","doi-asserted-by":"crossref","unstructured":"Olivieri NO, Nathan DG, MacMillan JH, et al. Survival of medically treated patients with homozygous \u03b2-thalassemia. N Engl J Med. 1994;331: 574-578.","DOI":"10.1056\/NEJM199409013310903"},{"key":"2020021300113290800_","doi-asserted-by":"crossref","unstructured":"Brittenham GM, Griffith PM, Nienhuis AW, et al. Efficacy of deferoxamine in preventing complications of iron overload in patients with thalassemia major. N Engl J Med. 1994;331: 567-573.","DOI":"10.1056\/NEJM199409013310902"},{"key":"2020021300113290800_","doi-asserted-by":"crossref","unstructured":"Gabutti V, Piga A. Results of long-term iron-chelating therapy. Acta Haematologica. 1996;95: 26-36.","DOI":"10.1159\/000203853"},{"key":"2020021300113290800_REF7","doi-asserted-by":"crossref","unstructured":"Modell B, Khan M, Darlison M. Survival in \u03b2-thalassaemia major in the UK: data from the UK Thalassaemia Register. Lancet. 2000;355: 2051-2052.","DOI":"10.1016\/S0140-6736(00)02357-6"},{"key":"2020021300113290800_REF8","doi-asserted-by":"crossref","unstructured":"Cohen A, Galanello R, Piga A, Vullo C, Tricta F. A multi-center safety trial of the oral iron chelator deferiprone. Ann N Y Acad Sci. 1998;850: 223-226.","DOI":"10.1111\/j.1749-6632.1998.tb10478.x"},{"key":"2020021300113290800_REF9","doi-asserted-by":"crossref","unstructured":"Cohen AR, Galanello R, Piga A, DiPalma A, Vullo C, Tricta F. Safety profile of the oral iron chelator deferiprone: a multicentre study. Brit J Haematol. 2000;108: 305-312.","DOI":"10.1046\/j.1365-2141.2000.01866.x"},{"key":"2020021300113290800_REF10","doi-asserted-by":"crossref","unstructured":"Olivieri NF, Brittenham GM, McLaren CE, et al. Long-term safety and effectiveness of iron-chelation therapy with deferiprone for thalassemia major. N Engl J Med. 1998;339: 417-423.","DOI":"10.1056\/NEJM199808133390701"},{"key":"2020021300113290800_REF11","doi-asserted-by":"crossref","unstructured":"Arboretti R, Tognoni G, Alberti D. Pharmacosur-veillance and quality of care of thalassaemic patients. Eur J Clin Pharmacol. 2001;56: 915-922.","DOI":"10.1007\/s002280000251"},{"key":"2020021300113290800_REF12","doi-asserted-by":"crossref","unstructured":"Al-Refaie FN, Hershko C, Hoffbrand AV, et al. Results of long-term deferiprone (L1) therapy: a report by the International Study Group on Oral Iron Chelators. 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Lancet. 1989;2: 457.","DOI":"10.1016\/S0140-6736(89)90641-7"},{"key":"2020021300113290800_REF16","doi-asserted-by":"crossref","unstructured":"Al-Refaie FN, Wonke B, Hoffbrand AV. Deferiprone-associated myelotoxicity. Eur J Haematol. 1994;53: 298-301.","DOI":"10.1111\/j.1600-0609.1994.tb01323.x"},{"key":"2020021300113290800_REF17","doi-asserted-by":"crossref","unstructured":"Ceci A, Baiardi P, Felisi M, et al. The safety and effectiveness of deferiprone in a large-scale, 3-year study in Italian patients. Brit J Haematol. 2002;118: 330-336.","DOI":"10.1046\/j.1365-2141.2002.03554.x"},{"key":"2020021300113290800_REF18","doi-asserted-by":"crossref","unstructured":"Agarwal MB, Gupte SS, Viswanathan C, et al. Long-term assessment of efficacy and safety of L1, an oral iron chelator, in transfusion dependent thalassaemia: Indian trial. 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Brit J Haematol. 1998;103: 361-364.","DOI":"10.1046\/j.1365-2141.1998.01002.x"}],"container-title":["Blood"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/ashpublications.org\/blood\/article-pdf\/102\/5\/1583\/1689829\/h81703001583.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"http:\/\/ashpublications.org\/blood\/article-pdf\/102\/5\/1583\/1689829\/h81703001583.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2020,3,20]],"date-time":"2020-03-20T00:46:32Z","timestamp":1584665192000},"score":1,"resource":{"primary":{"URL":"https:\/\/ashpublications.org\/blood\/article\/102\/5\/1583\/17504\/Safety-and-effectiveness-of-longterm-therapy-with"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2003,9,1]]},"references-count":19,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2003,9,1]]}},"URL":"https:\/\/doi.org\/10.1182\/blood-2002-10-3280","relation":{},"ISSN":["0006-4971","1528-0020"],"issn-type":[{"value":"0006-4971","type":"print"},{"value":"1528-0020","type":"electronic"}],"subject":[],"published":{"date-parts":[[2003,9,1]]}}}