Haematological effects of oral administration of bitopertin, a glycine transport inhibitor, in patients with non-transfusion-dependent β-thalassaemia

被引:15
|
作者
Taher, Ali T. [1 ]
Viprakasit, Vip [2 ,3 ]
Cappellini, Maria Domenica [4 ]
Kraus, Dominik [5 ]
Cech, Patrick [5 ]
Volz, Dietmar [5 ]
Winter, Erica [6 ]
Nave, Stephane [5 ]
Dukart, Juergen [5 ,7 ,8 ]
Khwaja, Omar [5 ]
Koerner, Annette [5 ]
Hermosilla, Ricardo [5 ]
Brugnara, Carlo [9 ,10 ]
机构
[1] Amer Univ Beirut, Med Ctr, Dept Internal Med, Div Hematol & Oncol, Beirut, Lebanon
[2] Mahidol Univ, Dept Pediat, Bangkok, Thailand
[3] Mahidol Univ, Div Hematol & Oncol, Siriraj Thalassemia Ctr, Bangkok, Thailand
[4] Univ Milan, Dept Med, CaGranda Fdn IRCCS, Milan, Italy
[5] Roche Innovat Ctr Basel, Roche Pharma Res & Early Dev, Basel, Switzerland
[6] Roche Innovat Ctr, Roche Pharma Res & Early Dev, New York, NY USA
[7] Res Ctr Julich, Inst Neurosci & Med Brain & Behav INM 7, Julich, Germany
[8] Heinrich Heine Univ Dusseldorf, Med Fac, Inst Syst Neurosci, Dusseldorf, Germany
[9] Boston Childrens Hosp, Dept Lab Med, Boston, MA USA
[10] Harvard Med Sch, Dept Pathol, Boston, MA 02115 USA
关键词
bitopertin; haem; iron; reactive oxygen species; beta-thalassemia; ERYTHROPOIESIS;
D O I
10.1111/bjh.17479
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
引用
收藏
页码:474 / 477
页数:4
相关论文
共 24 条
  • [21] Soluble form of transferrin receptor-1 level is associated with the age at first diagnosis and the risk of therapeutic intervention and iron overloading in patients with non-transfusion-dependent thalassemia
    Paolo Ricchi
    Antonella Meloni
    Silvia Costantini
    Anna Spasiano
    Tiziana Di Matola
    Alessia Pepe
    Patrizia Cinque
    Aldo Filosa
    Annals of Hematology, 2017, 96 : 1541 - 1546
  • [22] Clinical utility of serum ferritin thresholds for guiding iron chelation therapy when magnetic resonance imaging is unavailable in patients with non-transfusion-dependent thalassaemias - response to Ang et al
    Taher, Ali T.
    BRITISH JOURNAL OF HAEMATOLOGY, 2017, 176 (06) : 989 - 990
  • [23] Efficacy and safety of LentiGlobin gene therapy in patients with transfusion-dependent β-thalassaemia and non-β0/β0 genotypes: Updated results from the completed phase 1/2 Northstar and ongoing phase 3 Northstar-2 studies
    Porter, J. B.
    Walters, M. C.
    Kwiatkowski, J. L.
    Hongeng, S.
    Rasko, J. E. J.
    Sauer, M. G.
    Thrasher, A. J.
    Thuret, I.
    Schiller, G. J.
    Elliot, H.
    Deary, B.
    Chen, Y.
    Tao, G.
    Asmal, M.
    Locatelli, F.
    Thompson, A. A.
    BRITISH JOURNAL OF HAEMATOLOGY, 2019, 185 : 23 - 23
  • [24] Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial
    Kwiatkowski, Janet L.
    Walters, Mark C.
    Hongeng, Suradej
    Yannaki, Evangelia
    Kulozik, Andreas E.
    Kunz, Joachim B.
    Sauer, Martin G.
    Thrasher, Adrian J.
    Thuret, Isabelle
    Lal, Ashutosh
    Tao, Ge
    Ali, Shamshad
    Thakar, Himal L.
    Elliot, Heidi
    Lodaya, Ankit
    Lee, Ji
    Colvin, Richard A.
    Locatelli, Franco
    Thompson, Alexis A.
    LANCET, 2024, 404 (10468): : 2175 - 2186