SAFETY AND EFFICACY OF 4 YEARS OF DEFERASIROX TREATMENT FOR SICKLE CELL DISEASE PATIENTS

被引：3
作者：
Vlachaki, Efthymia [1 ]
Mainou, Maria [2 ]
Bekiari, Eleni [2 ]
Vetsiou, Evaggelia [1 ]
Tsapas, Apostolos [2 ]
机构：
[1] Aristotle Univ Thessaloniki, Hippokratio Gen Hosp, Thalassemia Unit, Dept Med 2, Thessaloniki 54642, Greece
[2] Aristotle Univ Thessaloniki, Metab Dis Unit, Dept Med 2, Thessaloniki 54642, Greece
来源：
HEMOGLOBIN | 2013年 / 37卷 / 01期
关键词：
Deferasirox (DFRA); Chelation treatment; Sickle cell disease; ORAL IRON CHELATOR; THERAPY; DEFERIPRONE; OVERLOAD; THALASSEMIA; MORBIDITY; MORTALITY;
D O I：
10.3109/03630269.2012.746696
中图分类号：
Q5 [生物化学]; Q7 [分子生物学];
学科分类号：
071010 ; 081704 ;
摘要：
Deferasirox (DFRA) is a novel oral chelator agent for treatment of iron overload. Although well established in the treatment of beta-thalassemia major (beta-TM), it has not yet been fully investigated in patients with sickle cell disease. The aim of this report is to present the preliminary results of a pilot study assessing the effect of 4 years of DFRA treatment in six patients with sickle cell disease who are in need of recurrent transfusions. Our results show a significant reduction of ferritin levels and improvement of liver hemosiderosis, assessed by means of magnetic resonance imaging T2* (MRI T2*). None of the patients presented any serious adverse effects and the treatment was well tolerated. These results are in accordance with previous studies about the use of DFRA in sickle cell disease.
引用
收藏
页码：94 / 100
页数：7
相关论文
共 26 条
  • [21] HYPOURICEMIA DUE TO AN INCREMENT IN RENAL TUBULAR URATE SECRETION
    SHICHIRI, M
    MATSUDA, O
    SHIIGAI, T
    TAKEUCHI, J
    KANAYAMA, M
    [J]. ARCHIVES OF INTERNAL MEDICINE, 1982, 142 (10) : 1855 - 1857
  • [22] A randomised comparison of deferasirox versus deferoxamine for the treatment of transfusional iron overload in sickle cell disease
    Vichinsky, Elliott
    Onyekwere, Onyinye
    Porter, John
    Swerdlow, Paul
    Eckman, James
    Lane, Peter
    Files, Beatrice
    Hassell, Kathryn
    Kelly, Patrick
    Wilson, Felicia
    Bernaudin, Francoise
    Forni, Gian Luca
    Okpala, Iheanyi
    Ressayre-Djaffer, Catherine
    Alberti, Daniele
    Holland, Jaymes
    Marks, Peter
    Fung, Ellen
    Fischer, Roland
    Mueller, Brigitta U.
    Coates, Thomas
    [J]. BRITISH JOURNAL OF HAEMATOLOGY, 2007, 136 (03) : 501 - 508
  • [23] Long-term safety and efficacy of deferasirox (Exjade®) for up to 5 years in transfusional iron-overloaded patients with sickle cell disease
    Vichinsky, Elliott
    Bernaudin, Francoise
    Forni, Gian Luca
    Gardner, Renee
    Hassell, Kathryn
    Heeney, Matthew M.
    Inusa, Baba
    Kutlar, Abdullah
    Lane, Peter
    Mathias, Liesl
    Porter, John
    Tebbi, Cameron
    Wilson, Felicia
    Griffel, Louis
    Deng, Wei
    Giannone, Vanessa
    Coates, Thomas
    [J]. BRITISH JOURNAL OF HAEMATOLOGY, 2011, 154 (03) : 387 - 397
  • [24] Deferiprone as an oral iron chelator in sickle cell disease
    Voskaridou, E
    Douskou, M
    Terpos, E
    Stamoulakatou, A
    Meletis, J
    Ourailidis, A
    Papassotiriou, I
    Loukopoulos, D
    [J]. ANNALS OF HEMATOLOGY, 2005, 84 (07) : 434 - 440
  • [25] Deferasirox effectively decreases iron burden in patients with double heterozygous HbS/β-thalassemia
    Voskaridou, Ersi
    Plata, Eleni
    Douskou, Marousa
    Sioni, Anastasia
    Mpoutou, Efrosini
    Christoulas, Dimitrios
    Dimopoulou, Maria
    Terpos, Evangelos
    [J]. ANNALS OF HEMATOLOGY, 2011, 90 (01) : 11 - 15
  • [26] Iron Metabolism and Iron Chelation in Sickle Cell Disease
    Walter, Patrick B.
    Harmatz, Paul
    Vichinsky, Elliott
    [J]. ACTA HAEMATOLOGICA, 2009, 122 (2-3) : 174 - 183
123