Tayyaba Rehan Syeda, Hussain Hassan Ul, Malik Farheen, Usama Rana Muhammad, Tahir Muhammad Junaid, Asghar Muhammad Sohaib
Department of Medicine Dow University of Health Sciences Karachi Pakistan.
Lahore General Hospital Lahore Pakistan.
Health Sci Rep. 2022 Jun 21;5(4):e713. doi: 10.1002/hsr2.713. eCollection 2022 Jul.
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.
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.
镰状细胞病（SCD）是最常见的血红蛋白病之一，每年影响约27.5万名新生儿。直到2017年，羟基脲是唯一可用于治疗SCD的药物。后来，L-谷氨酰胺和crizanlizumab在SCD治疗中显示出了有前景的结果。
2019年11月，voxelotor在临床HOPE试验中显示出有前景的结果后被批准用于治疗SCD患者，当时该疾病的药物选择有限。尽管有良好的结果，但也观察到了一些危及生命的副作用。SCD现有药物治疗使用的不确定性是患者生存的主要障碍。
需要立即关注SCD药物选择有限的缺点。文章呼吁在这个医学先进时代进行更广泛的试验，因为SCD药物使用的不确定性仍然存在。
