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Ann Biol Clin (Paris) ; 81(3): 320-326, 2023 07 21.
Artigo em Francês | MEDLINE | ID: mdl-37475172

RESUMO

Voxelotor (GBT440, OXBRYTA®) appeared recently as one of the possible treatments for sickle cell disease. This molecule, by binding the alpha globin of hemoglobin, causes hyperaffinity of the latter for oxygen and reduces its polymerization properties. Several therapeutic trials have been able to show its effectiveness on certain aspects of sickle cell disease; thus, the french HAS (High Authority of Health) college issued an early access authorization and, since 2021, this treatment can be offered to patients under a temporary authorization for use. Consequently, the laboratories that carry out the biological monitoring of sickle cell patients will be confronted with new profiles characteristic of the presence of hemoglobin combined with GBT440. This work presents a collection of images obtained by different techniques: HPLC, capillary electrophoresis, isoelectrofocusing, alkaline gel and acid agar gel electrophoresis in transfused or non-transfused sickle cell disease patients. The ability to observe the presence of GBT440 by these analyzes could be useful in order to characterize the therapeutic follow-up of patients.


Assuntos
Anemia Falciforme , Hemoglobina Falciforme , Humanos , Hemoglobina Falciforme/química , Hemoglobina Falciforme/metabolismo , Hemoglobina Falciforme/uso terapêutico , Hemoglobinas/metabolismo , Anemia Falciforme/diagnóstico , Anemia Falciforme/terapia , Benzaldeídos/efeitos adversos
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