Published online May 6, 2021. doi: 10.12998/wjcc.v9.i13.3056
Peer-review started: November 3, 2020
First decision: February 12, 2021
Revised: February 22, 2021
Accepted: March 11, 2021
Article in press: March 11, 2021
Published online: May 6, 2021
Processing time: 169 Days and 21.6 Hours
Crumbs homolog 2 (CRB2) is a recently discovered gene that is closely related to the maintenance of normal polarity in podocytes; mutations can directly lead to steroid-resistant nephrotic syndrome (SRNS). However, the characteristics of nephrotic syndrome (NS) caused by CRB2 mutations have not been described.
We report a novel compound heterozygous mutation of the CRB2 gene in two siblings with SRNS. The two siblings had edema, proteinuria, hypoproteinemia and hyperlipidemia. Both their father and mother had normal phenotypes (no history of NS). Whole exon sequencing (WES) of the family showed a novel compound heterozygous mutation, c.2290 (exon 8) C > T and c.3613 (exon 12) G > A. Glucocorticoid therapy (methylprednisolone pulse therapy or oral prednisone) and immunosuppressive agents (tacrolimus) had no effect. During a 3-year follow-up after genetic diagnosis by WES, proteinuria persisted, but the patient was healthy.
CRB2 mutations related to SRNS often occur in exons 7, 10, and 12. Clinical manifestations of SRNS caused by CRB2 mutations are often less severe than in other forms of SRNS.
Core Tip: Crumbs homolog 2 (CRB2) mutation is related to early-onset steroid-resistant nephrotic syndrome (SRNS). We here report two siblings with a novel compound heterozygous CRB2 mutation. We also reviewed the literature and found that CRB2 mutations related to SRNS often occur in exons 7, 10 and 12. Clinical manifestations of SRNS caused by CRB2 mutations, such as edema, are often less severe than in other forms of SRNS.