Table 4.

Treatment of Manifestations in Individuals with a WT1 Disorder

Manifestation/ConcernTreatmentConsiderations/Other
Glomer-
ulopathy 		Persistent
proteinuria		Consider renin-angiotensin-aldosterone system (RAAS) inhibition: ACE inhibitor, AT1 receptor blocker. 1
  • Avoid immunosuppressants, which are ineffective & potentially toxic. 1
  • Nephropathy does not recur post renal transplantation. 2
SRNS
CKD
CNS
Disorder of testicular
development 		See Nonsyndromic Disorders of Testicular Development.Management is often by a multidisciplinary team incl medical geneticist, endocrinologist, urologist, & psychologist.
Wilms tumor Standard oncology protocols; surgery w/nephron-sparing approach whenever applicableBilateral prophylactic nephrectomy after reaching ESRD (i.e., at time of kidney transplantation or placement of a peritoneal dialysis catheter) 3
CAKUT Urologic intervention may be applicable.Per treating nephrologist &/or urologist
Gonadoblastoma Gonadectomy per DSD teamNo consensus re timing of surgery
Diaphragmatic hernia As per treating surgeonRepair to be performed prior to start of peritoneal dialysis

CAKUT = congenital anomalies of the kidney and urinary tract; CKD = chronic kidney disease; CNS = congenital nephrotic syndrome; DSD = disorders of sex development; ESRD = end-stage renal disease; SRNS = steroid-resistant nephrotic syndrome

1.
2.

For a child to be eligible for kidney transplantation, most centers require that children weigh 10 kg and/or be at least one year post-completion of treatment for Wilms tumor.

3.

From: WT1 Disorder

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