Emerging Therapies and Advances in Sickle Cell Disease with a Focus on Renal Manifestations.
Emerging Therapies and Advances in Sickle Cell Disease with a Focus on Renal Manifestations.
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DOI：
10.34067/kid.0000000000000162
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发表时间：
2023-07-01
期刊：
影响因子：
--
通讯作者：
Little J
中科院分区：
文献类型：
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作者：
Obadina M;Wilson S;Derebail VK;Little J
镰状细胞病（SCD）的潜在机制导致多方面的肾病，通常表现为蛋白尿。在严重的SCD基因型中（例如，血红蛋白SS [HbSS]）、白蛋白尿和CKD是该人群死亡率的主要预测因素。因此，肾功能的监测和管理是SCD综合护理的内在组成部分。SCD中肾病的管理可以通过SCD定向疗法和/或CKD定向疗法来完成。在过去的5年中，新的疾病修饰和姑息疗法已被批准用于SCD，以靶向疾病的各个方面，如贫血、炎症和血管病变。沿着常规的羟基脲和慢性输血，l-谷氨酰胺、crizanlizumab和voxelotor均显示可减轻SCD的一些不良反应，并且正在研究其对肾病的影响。CKD导向治疗，如肾素-血管紧张素-醛固酮系统阻滞剂，长期以来一直用于SCD肾病;然而，需要更完整的长期获益研究。考虑到肾脏疾病对生存率的影响，进一步评估这些SCD导向或CKD导向治疗药物的机制和疗效至关重要。
The underlying mechanisms of disease in sickle cell disease (SCD) contribute to a multifaceted nephropathy, commonly manifested as albuminuria. In severe SCD genotypes (e.g., Hemoglobin SS [HbSS]), albuminuria and CKD are major predictors of mortality in this population. Therefore, the monitoring and management of renal function is an intrinsic part of comprehensive care in SCD. Management of nephropathy in SCD can be accomplished with SCD-directed therapies and/or CKD-directed therapies. In the past 5 years, novel disease-modifying and palliative therapies have been approved in SCD to target aspects of the disease, such as anemia, inflammation, and vasculopathy. Along with conventional hydroxyurea and chronic transfusion, l-glutamine, crizanlizumab, and voxelotor have all been shown to mitigate some adverse effect of SCD, and their effect on nephropathy is being investigated. CKD-directed therapies such as renin–angiotensin–aldosterone system blockers have long been used in SCD nephropathy; however, more complete long-term studies on benefits are needed. Given the effect of renal disease on survival, further assessment of the mechanisms and efficacy of these SCD-directed or CKD-directed therapeutic agents is essential.