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Impact of voxelotor on hemoglobin electrophoretic and chromatographic profiles

Par : Contributeur(s) : Type de matériel : TexteTexteLangue : français Détails de publication : 2023. Ressources en ligne : Abrégé : Voxelotor (GBT440, OXBRYTA®) has appeared recently as one of the possible treatments for sickle cell disease. By binding the alpha globin of hemoglobin, this molecule 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, and consequently the French HAS (National Health Authority) college issued an early access authorization and, since 2021, this treatment has been available to patients under a temporary authorization for use. Consequently, the laboratories that carry out the biological monitoring of sickle cell patients will be faced with new profiles that are characteristic of the presence of hemoglobin combined with GBT440. This work presents a collection of images obtained using different techniques: HPLC, capillary electrophoresis, isoelectric focusing, alkaline gel, and acid agar gel electrophoresis in transfused or non-transfused sickle cell disease patients. The ability to observe the presence of GBT440 using these analyses could be useful for defining patients’ therapeutic follow-up.
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Voxelotor (GBT440, OXBRYTA®) has appeared recently as one of the possible treatments for sickle cell disease. By binding the alpha globin of hemoglobin, this molecule 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, and consequently the French HAS (National Health Authority) college issued an early access authorization and, since 2021, this treatment has been available to patients under a temporary authorization for use. Consequently, the laboratories that carry out the biological monitoring of sickle cell patients will be faced with new profiles that are characteristic of the presence of hemoglobin combined with GBT440. This work presents a collection of images obtained using different techniques: HPLC, capillary electrophoresis, isoelectric focusing, alkaline gel, and acid agar gel electrophoresis in transfused or non-transfused sickle cell disease patients. The ability to observe the presence of GBT440 using these analyses could be useful for defining patients’ therapeutic follow-up.

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