Safety and effectiveness of long-term therapy with the oral iron chelator deferiprone

被引:235
|
作者
Cohen, AR
Galanello, R
Piga, A
De Sanctis, V
Tricta, F
机构
[1] Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
[2] Univ Cagliari, Dipartimento Sci Biomed & Biotecnol, Osped Reg Microcitemie ASL8, Cagliari, Italy
[3] Univ Turin, Dipartimento Sci Pediat & Adolescenza, Turin, Italy
[4] Arcispedale St Anna, Div Pediat & Adolescentol, Ferrara, Italy
[5] Apotex Res, Toronto, ON, Canada
关键词
D O I
10.1182/blood-2002-10-3280
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
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 x 10(9)/L) and milder forms of neutropenia (ANC, 500-1500 x 10(9)/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.
引用
收藏
页码:1583 / 1587
页数:5
相关论文
共 50 条
  • [41] EFFECTIVENESS OF AN ORAL LONG-TERM THERAPY OF PAROXYSMAL VENTRICULAR TACHYARRHYTHMIAS WITH MEXILETIN
    RATHGEN, K
    ROSTOCK, KJ
    PFEIFFER, D
    SCHIRDEWAN, A
    ZEITSCHRIFT FUR KLINISCHE MEDIZIN-ZKM, 1986, 41 (15): : 1167 - 1169
  • [42] Long-term safety and tolerability of the once-daily, oral iron chelator deferasirox (Exjade®, ICL670) in patients with transfusional iron overload.
    Cappellini, M. D.
    Giardina, P.
    Porter, J.
    Coates, T.
    Della Porta, M. G.
    Siegel, J.
    Glimm, E.
    Ford, J.
    Cappellini, Maria Dominica
    BLOOD, 2006, 108 (11) : 501A - 501A
  • [43] Assessment of the effect of the oral iron chelator deferiprone on asymptomatic Plasmodium falciparum parasitemia in humans
    Thuma, PE
    Olivieri, NF
    Mabeza, GF
    Biemba, G
    Parry, D
    Zulu, S
    Fassos, FF
    McClelland, RA
    Koren, G
    Brittenham, GM
    Gordeuk, VR
    AMERICAN JOURNAL OF TROPICAL MEDICINE AND HYGIENE, 1998, 58 (03): : 358 - 364
  • [44] Effect of deferiprone, an oral iron chelator, in diabetic and non-diabetic glomerular disease
    Rajapurkar, Mohan M.
    Hegde, Umapati
    Bhattacharya, Amalkumar
    Alam, Muhammad G.
    Shah, Sudhir V.
    TOXICOLOGY MECHANISMS AND METHODS, 2013, 23 (01) : 5 - 10
  • [45] THE EFFECT OF THE ORAL IRON CHELATOR DEFERIPRONE ON THE LIVER DAMAGE INDUCED BY TAMOXIFEN IN FEMALE RATS
    Cerna, Pavla
    Kotyzova, Dana
    Eybl, Vladislav
    HEMOGLOBIN, 2011, 35 (03) : 255 - 261
  • [46] Use of the oral chelator deferiprone in the treatment of iron overload in patients with Hb H disease
    Chan, JCW
    Chim, CS
    Ooi, CGC
    Cheung, B
    Liang, R
    Chan, TK
    Chan, V
    BRITISH JOURNAL OF HAEMATOLOGY, 2006, 133 (02) : 198 - 205
  • [47] Long-term improvement under deferiprone in a case of neurodegeneration with brain iron accumulation
    Kwiatkowski, A.
    Ryckewaert, G.
    Tchofo, P. Jissendi
    Moreau, C.
    Vuillaume, I.
    Chinnery, P. F.
    Destee, A.
    Defebvre, L.
    Devos, D.
    PARKINSONISM & RELATED DISORDERS, 2012, 18 (01) : 110 - 112
  • [48] Long-Term Neuroradiological and Clinical Evaluation of NBIA Patients Treated with a Deferiprone Based Iron-Chelation Therapy
    Romano, Nicola
    Baiardi, Giammarco
    Pinto, Valeria Maria
    Quintino, Sabrina
    Gianesin, Barbara
    Sasso, Riccardo
    Diociasi, Andrea
    Mattioli, Francesca
    Marchese, Roberta
    Abbruzzese, Giovanni
    Castaldi, Antonio
    Forni, Gian Luca
    JOURNAL OF CLINICAL MEDICINE, 2022, 11 (15)
  • [49] Long-term safety and effectiveness of lamictal in adults
    Risner, ME
    Groenke, DA
    Messenheimer, JA
    EPILEPSIA, 1999, 40 : 140 - 140
  • [50] Long-term safety of antihypertensive therapy
    Grossman, Ehud
    Messerh, Franz H.
    PROGRESS IN CARDIOVASCULAR DISEASES, 2006, 49 (01) : 16 - 25