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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:...

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Autores principales: Tayyaba Rehan, Syeda, Hussain, Hassan ul, Malik, Farheen, Usama, Rana Muhammad, Tahir, Muhammad Junaid, Asghar, Muhammad Sohaib
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2022
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.
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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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