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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Detalles Bibliográficos
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:
Perspective
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
Descripción
Sumario: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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