Agrawal Shipra, Ransom Richard F, Saraswathi Saras, Garcia-Gonzalo Esperanza, Webb Amy, Fernandez-Martinez Juan L, Popovic Milan, Guess Adam J, Kloczkowski Andrzej, Benndorf Rainer, Sadee Wolfgang, Smoyer William E
Center for Clinical and Translational Research, Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.
Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH 43210, USA.
J Clin Med. 2021 Feb 2;10(3):523. doi: 10.3390/jcm10030523.
Glucocorticoid (GC) resistance complicates the treatment of ~10-20% of children with nephrotic syndrome (NS), yet the molecular basis for resistance remains unclear. We used RNAseq analysis and in silico algorithm-based approaches on peripheral blood leukocytes from 12 children both at initial NS presentation and after ~7 weeks of GC therapy to identify a 12-gene panel able to differentiate steroid resistant NS (SRNS) from steroid-sensitive NS (SSNS). Among this panel, subsequent validation and analyses of one biologically relevant candidate, sulfatase 2 (SULF2), in up to a total of 66 children, revealed that both SULF2 leukocyte expression and plasma arylsulfatase activity Post/Pre therapy ratios were greater in SSNS vs. SRNS. However, neither plasma SULF2 endosulfatase activity (measured by VEGF binding activity) nor plasma VEGF levels, distinguished SSNS from SRNS, despite VEGF's reported role as a downstream mediator of SULF2's effects in glomeruli. Experimental studies of NS-related injury in both rat glomeruli and cultured podocytes also revealed decreased SULF2 expression, which were partially reversible by GC treatment of podocytes. These findings together suggest that SULF2 levels and activity are associated with GC resistance in NS, and that SULF2 may play a protective role in NS via the modulation of downstream mediators distinct from VEGF.
糖皮质激素(GC)抵抗使约10-20%的肾病综合征(NS)患儿的治疗变得复杂,但其抵抗的分子基础仍不清楚。我们对12名初发NS患儿及GC治疗约7周后的外周血白细胞进行了RNA测序分析和基于计算机算法的方法,以确定一个能够区分激素抵抗性NS(SRNS)和激素敏感性NS(SSNS)的12基因panel。在这个panel中,随后对多达66名儿童中的一个生物学相关候选基因硫酸酯酶2(SULF2)进行验证和分析,结果显示,与SRNS相比,SSNS中SULF2白细胞表达和血浆芳基硫酸酯酶活性治疗后/治疗前比值更高。然而,尽管有报道称VEGF是SULF2在肾小球中作用的下游介质,但血浆SULF2内切硫酸酯酶活性(通过VEGF结合活性测量)和血浆VEGF水平均无法区分SSNS和SRNS。大鼠肾小球和培养的足细胞中NS相关损伤的实验研究也显示SULF2表达降低,而足细胞的GC治疗可部分逆转这种降低。这些发现共同表明,SULF2水平和活性与NS中的GC抵抗相关,并且SULF2可能通过调节不同于VEGF的下游介质在NS中发挥保护作用。