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Case report: Safety and efficacy of voxelotor in a patient with sickle cell disease and stage IV chronic kidney disease
Sickle cell disease (SCD) is a heterogeneous group of inherited disorders characterized by the production of sickle hemoglobin which is less soluble than an adult or fetal hemoglobin. Voxelotor is a hemoglobin S polymerization inhibitor that has been approved for sickle cell disease treatment in the...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Frontiers Media S.A.
2022
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9520303/ https://www.ncbi.nlm.nih.gov/pubmed/36186817 http://dx.doi.org/10.3389/fmed.2022.931924 |
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author | Alshurafa, Awni Yassin, Mohamed A. |
author_facet | Alshurafa, Awni Yassin, Mohamed A. |
author_sort | Alshurafa, Awni |
collection | PubMed |
description | Sickle cell disease (SCD) is a heterogeneous group of inherited disorders characterized by the production of sickle hemoglobin which is less soluble than an adult or fetal hemoglobin. Voxelotor is a hemoglobin S polymerization inhibitor that has been approved for sickle cell disease treatment in the adult and adolescent populations. It acts as a hemoglobin modulator by increasing its affinity to oxygen which prevents red blood cells from sickling. Chronic kidney disease is a common but under-reported complication of SCD and it is a leading cause of morbidity and mortality. The data about the safety and efficacy of voxelotor use in chronic kidney disease is limited. Herein we report a 49-year-old man, with sickle cell disease and stage IV chronic kidney disease, who was managed successfully with voxelotor and resulted in decreasing transfusion requirement and vaso-occlusive painful crisis without affecting kidney function. |
format | Online Article Text |
id | pubmed-9520303 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | Frontiers Media S.A. |
record_format | MEDLINE/PubMed |
spelling | pubmed-95203032022-09-30 Case report: Safety and efficacy of voxelotor in a patient with sickle cell disease and stage IV chronic kidney disease Alshurafa, Awni Yassin, Mohamed A. Front Med (Lausanne) Medicine Sickle cell disease (SCD) is a heterogeneous group of inherited disorders characterized by the production of sickle hemoglobin which is less soluble than an adult or fetal hemoglobin. Voxelotor is a hemoglobin S polymerization inhibitor that has been approved for sickle cell disease treatment in the adult and adolescent populations. It acts as a hemoglobin modulator by increasing its affinity to oxygen which prevents red blood cells from sickling. Chronic kidney disease is a common but under-reported complication of SCD and it is a leading cause of morbidity and mortality. The data about the safety and efficacy of voxelotor use in chronic kidney disease is limited. Herein we report a 49-year-old man, with sickle cell disease and stage IV chronic kidney disease, who was managed successfully with voxelotor and resulted in decreasing transfusion requirement and vaso-occlusive painful crisis without affecting kidney function. Frontiers Media S.A. 2022-09-15 /pmc/articles/PMC9520303/ /pubmed/36186817 http://dx.doi.org/10.3389/fmed.2022.931924 Text en Copyright © 2022 Alshurafa and Yassin. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms. |
spellingShingle | Medicine Alshurafa, Awni Yassin, Mohamed A. Case report: Safety and efficacy of voxelotor in a patient with sickle cell disease and stage IV chronic kidney disease |
title | Case report: Safety and efficacy of voxelotor in a patient with sickle cell disease and stage IV chronic kidney disease |
title_full | Case report: Safety and efficacy of voxelotor in a patient with sickle cell disease and stage IV chronic kidney disease |
title_fullStr | Case report: Safety and efficacy of voxelotor in a patient with sickle cell disease and stage IV chronic kidney disease |
title_full_unstemmed | Case report: Safety and efficacy of voxelotor in a patient with sickle cell disease and stage IV chronic kidney disease |
title_short | Case report: Safety and efficacy of voxelotor in a patient with sickle cell disease and stage IV chronic kidney disease |
title_sort | case report: safety and efficacy of voxelotor in a patient with sickle cell disease and stage iv chronic kidney disease |
topic | Medicine |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9520303/ https://www.ncbi.nlm.nih.gov/pubmed/36186817 http://dx.doi.org/10.3389/fmed.2022.931924 |
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