Pediatric Nephrology and Hypertension Unit, MassGeneral Hospital for Children at Massachusetts General Hospital, Boston, Massachusetts, USA; Department of Pediatrics, Harvard Medical School, Boston, Massachusetts, USA.
Kidney Int. 2022 Sep;102(3):471-473. doi: 10.1016/j.kint.2022.06.005.
Among youth with incident nephrotic syndrome, those with steroid-resistant nephrotic syndrome (SRNS) often have an ominous clinical course. Identifying them at or shortly after diagnosis would potentially prevent substantial morbidity and even mortality. For those with a specific monogenic form, targeted therapy might be possible, as is the case presently for CoQ10 insufficiency cases. Further, dissecting specific causes and pathways that lead to SRNS may lead to other targeted, potentially highly effective treatments.
在患有肾病综合征的年轻人中,那些患有类固醇抵抗性肾病综合征(SRNS)的人通常预后不良。如果能在诊断时或诊断后不久识别出他们,就有可能预防严重的发病率,甚至死亡率。对于那些具有特定单基因形式的患者,可能可以进行靶向治疗,目前 CoQ10 缺乏症的情况就是如此。此外,剖析导致 SRNS 的具体原因和途径可能会导致其他靶向治疗,这些治疗可能具有很高的疗效。