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

页码：94 / 100

页数：7

共 26 条

[21] HYPOURICEMIA DUE TO AN INCREMENT IN RENAL TUBULAR URATE SECRETION [J].

SHICHIRI, M ;

MATSUDA, O ;

SHIIGAI, T ;

TAKEUCHI, J ;

KANAYAMA, M .

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 [J].

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 .

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 [J].

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 .

BRITISH JOURNAL OF HAEMATOLOGY, 2011, 154 (03) :387-397

[24] Deferiprone as an oral iron chelator in sickle cell disease [J].

Voskaridou, E ;

Douskou, M ;

Terpos, E ;

Stamoulakatou, A ;

Meletis, J ;

Ourailidis, A ;

Papassotiriou, I ;

Loukopoulos, D .

ANNALS OF HEMATOLOGY, 2005, 84 (07) :434-440

[25] Deferasirox effectively decreases iron burden in patients with double heterozygous HbS/β-thalassemia [J].

Voskaridou, Ersi ;

Plata, Eleni ;

Douskou, Marousa ;

Sioni, Anastasia ;

Mpoutou, Efrosini ;

Christoulas, Dimitrios ;

Dimopoulou, Maria ;

Terpos, Evangelos .

ANNALS OF HEMATOLOGY, 2011, 90 (01) :11-15

[26] Iron Metabolism and Iron Chelation in Sickle Cell Disease [J].

Walter, Patrick B. ;

Harmatz, Paul ;

Vichinsky, Elliott .

ACTA HAEMATOLOGICA, 2009, 122 (2-3) :174-183

← 1 2 3 →