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Voxelotor versus other therapeutic options for sickle cell disease: Are we still lagging behind in treating the disease?
BACKGROUND: Sickle cell disease (SCD) is one of the most prevalent hemoglobinopathies that affects around 275,000 neonates annually. Until 2017, hydroxyurea was the only available drug for SCD treatment. Later on, L‐glutamine and crizanlizumab have shown promising results in SCD therapy. OBJECTIVES:...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9213830/ https://www.ncbi.nlm.nih.gov/pubmed/35774831 http://dx.doi.org/10.1002/hsr2.713 |
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author | Tayyaba Rehan, Syeda Hussain, Hassan ul Malik, Farheen Usama, Rana Muhammad Tahir, Muhammad Junaid Asghar, Muhammad Sohaib |
author_facet | Tayyaba Rehan, Syeda Hussain, Hassan ul Malik, Farheen Usama, Rana Muhammad Tahir, Muhammad Junaid Asghar, Muhammad Sohaib |
author_sort | Tayyaba Rehan, Syeda |
collection | PubMed |
description | BACKGROUND: Sickle cell disease (SCD) is one of the most prevalent hemoglobinopathies that affects around 275,000 neonates annually. Until 2017, hydroxyurea was the only available drug for SCD treatment. Later on, L‐glutamine and crizanlizumab have shown promising results in SCD therapy. OBJECTIVES: There were limited pharmacological options for the disease when in November, 2019, voxelotor was approved for the treatment of SCD patients after showing promising results in the clinical HOPE trial. Despite its favorable results, some life‐threatening side effects were also observed. Uncertainty regarding the use of available pharmaceutical therapies for SCD is the major hurdle for the survival of patients. DISCUSSION & CONCLUSION: An immediate attention needs to be drawn towards the drawbacks of limited pharmacological options for SCD. Article calls out to conduct more extensive trials in this advanced era of medicine where ambiguity regarding the use of SCD drugs still prevails. |
format | Online Article Text |
id | pubmed-9213830 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | John Wiley and Sons Inc. |
record_format | MEDLINE/PubMed |
spelling | pubmed-92138302022-06-29 Voxelotor versus other therapeutic options for sickle cell disease: Are we still lagging behind in treating the disease? Tayyaba Rehan, Syeda Hussain, Hassan ul Malik, Farheen Usama, Rana Muhammad Tahir, Muhammad Junaid Asghar, Muhammad Sohaib Health Sci Rep Perspective BACKGROUND: Sickle cell disease (SCD) is one of the most prevalent hemoglobinopathies that affects around 275,000 neonates annually. Until 2017, hydroxyurea was the only available drug for SCD treatment. Later on, L‐glutamine and crizanlizumab have shown promising results in SCD therapy. OBJECTIVES: There were limited pharmacological options for the disease when in November, 2019, voxelotor was approved for the treatment of SCD patients after showing promising results in the clinical HOPE trial. Despite its favorable results, some life‐threatening side effects were also observed. Uncertainty regarding the use of available pharmaceutical therapies for SCD is the major hurdle for the survival of patients. DISCUSSION & CONCLUSION: An immediate attention needs to be drawn towards the drawbacks of limited pharmacological options for SCD. Article calls out to conduct more extensive trials in this advanced era of medicine where ambiguity regarding the use of SCD drugs still prevails. John Wiley and Sons Inc. 2022-06-21 /pmc/articles/PMC9213830/ /pubmed/35774831 http://dx.doi.org/10.1002/hsr2.713 Text en © 2022 The Authors. Health Science Reports published by Wiley Periodicals LLC. https://creativecommons.org/licenses/by/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited. |
spellingShingle | Perspective Tayyaba Rehan, Syeda Hussain, Hassan ul Malik, Farheen Usama, Rana Muhammad Tahir, Muhammad Junaid Asghar, Muhammad Sohaib Voxelotor versus other therapeutic options for sickle cell disease: Are we still lagging behind in treating the disease? |
title | Voxelotor versus other therapeutic options for sickle cell disease: Are we still lagging behind in treating the disease? |
title_full | Voxelotor versus other therapeutic options for sickle cell disease: Are we still lagging behind in treating the disease? |
title_fullStr | Voxelotor versus other therapeutic options for sickle cell disease: Are we still lagging behind in treating the disease? |
title_full_unstemmed | Voxelotor versus other therapeutic options for sickle cell disease: Are we still lagging behind in treating the disease? |
title_short | Voxelotor versus other therapeutic options for sickle cell disease: Are we still lagging behind in treating the disease? |
title_sort | voxelotor versus other therapeutic options for sickle cell disease: are we still lagging behind in treating the disease? |
topic | Perspective |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9213830/ https://www.ncbi.nlm.nih.gov/pubmed/35774831 http://dx.doi.org/10.1002/hsr2.713 |
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