The DOI matches its Crossref record; 16 of the 16 references it cites were found in a registry or an index.
Safety and effectiveness of long-term therapy with the oral iron chelator deferiprone
Abstract
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] < 500 × 109/L) and milder forms of neutropenia (ANC, 500-1500 × 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.
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Citations
Cites
Iron-Chelating Therapy and the Treatment of Thalassemia
Olivieri, N. F., & Brittenham, G. M. (1997) · Blood · doi:10.1182/blood.v89.3.739 · Cited by 1,089
Survival in Medically Treated Patients with Homozygous β-Thalassemia
Olivieri, N. F., Nathan, D. G., MacMillan, J. H., Wayne, A. S., Liu, P. P., McGee, A., Martin, M., Koren, G., & Cohen, A. R. (1994) · New England Journal of Medicine · doi:10.1056/nejm199409013310903 · Cited by 852
Self-citationBrittenham, G. M., Griffith, P. M., Nienhuis, A. W., McLaren, C. E., Young, N. S., Tucker, E. E., Allen, C. J., Farrell, D. E., & Harris, J. W. (1994) · New England Journal of Medicine · doi:10.1056/nejm199409013310902 · Cited by 829
Survival in β-thalassaemia major in the UK: data from the UK Thalassaemia Register
Modell, B., Khan, M., & Darlison, M. (2000) · The Lancet · doi:10.1016/s0140-6736(00)02357-6 · Cited by 583
Long-Term Safety and Effectiveness of Iron-Chelation Therapy with Deferiprone for Thalassemia Major
Olivieri, N. F., Brittenham, G. M., McLaren, C. E., Templeton, D. M., Cameron, R. G., McClelland, R. A., Burt, A. D., & Fleming, K. A. (1998) · New England Journal of Medicine · doi:10.1056/nejm199808133390701 · Cited by 420
Practical management of iron overload
Porter, J. B. (2001) · British Journal of Haematology · doi:10.1046/j.1365-2141.2001.03195.x · Cited by 378
Results of Long-Term Iron-Chelating Therapy
Gabutti, V., & Piga, A. (2009) · Acta Haematologica · doi:10.1159/000203853 · Cited by 378
Self-citationIron-Chelation Therapy with Oral Deferiprone in Patients with Thalassemia Major
Olivieri, N. F., Brittenham, G. M., Matsui, D., Berkovitch, M., Blendis, L. M., Cameron, R. G., McClelland, R. A., Liu, P. P., Templeton, D. M., & Koren, G. (1995) · New England Journal of Medicine · doi:10.1056/nejm199504063321404 · Cited by 324
Combined therapy with deferiprone and desferrioxamine
Wonke, Wright, & Hoffbrand (1998) · British Journal of Haematology · doi:10.1046/j.1365-2141.1998.01002.x · Cited by 218
The safety and effectiveness of deferiprone in a large‐scale, 3‐year study in Italian patients
Ceci, A., Baiardi, P., Felisi, M., Cappellini, M. D., Carnelli, V., De Sanctis, V., Galanello, R., Maggio, A., Masera, G., Piga, A., et al. (2002) · British Journal of Haematology · doi:10.1046/j.1365-2141.2002.03554.x · Cited by 215
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Cited by
Cao, A., & Galanello, R. (2010) · Genetics in Medicine · doi:10.1097/gim.0b013e3181cd68ed · Cited by 722
Self-citationRachmilewitz, E. A., & Giardina, P. J. (2011) · Blood · doi:10.1182/blood-2010-08-300335 · Cited by 417
Selective iron chelation in Friedreich ataxia: biologic and clinical implications
Boddaert, N., Le Quan Sang, K. H., Rötig, A., Leroy-Willig, A., Gallet, S., Brunelle, F., Sidi, D., Thalabard, J.-C., Munnich, A., & Cabantchik, Z. I. (2007) · Blood · doi:10.1182/blood-2006-12-065433 · Cited by 409
Greenberg, P. L., Attar, E., Bennett, J. M., Bloomfield, C. D., De Castro, C. M., Deeg, H. J., Foran, J. M., Gaensler, K., Garcia-Manero, G., Gore, S. D., et al. (2011) · Journal of the National Comprehensive Cancer Network · doi:10.6004/jnccn.2011.0005 · Cited by 290
Iron homeostasis and toxicity in retinal degeneration
He, X., Hahn, P., Iacovelli, J., Wong, R., King, C., Bhisitkul, R., Massaro-Giordano, M., & Dunaief, J. L. (2007) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2007.07.004 · Cited by 260
Metadata
- Authors
- Cohen, A. R., Galanello, R., Piga, A., De Sanctis, V., & Tricta, F.
- Venue
- Blood
- Publisher
- American Society of Hematology
- Published
- 2003-09-01
- Volume
- 102
- Issue
- 5
- Pages
- 1583-1587
- ISSN
- 0006-4971 (ISSN-L)
- 1528-0020
- OpenAlex
- W2137723066
- Cited by
- 323
- References
- 16
- FWCI
- 8.67
- Type
- journal article
- Language
- en
- Open access
- closed
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