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pediatric

Denys-Drash Syndrome

ICD-10 Code
Q87.89

Rare triad of diffuse mesangial sclerosis (early-onset nephrotic syndrome), pseudohermaphroditism (XY genital ambiguity), and a very high risk of Wilms tumor. Caused by WT1 gene mutations.

Clinical Presentation & Protocol

Patient Usually Complains Of

Patient presents with early-onset nephrotic syndrome characterized by refractory proteinuria and rapid progression to end-stage renal disease (ESRD). History significant for ambiguous genitalia (XY karyotype) noted at birth. Family history negative or positive for WT1-related nephropathy. No current evidence of abdominal mass or hematuria.

Clinical Examination Findings

General: Patient appears chronically ill. Abdomen: Soft, non-tender, no palpable masses suggestive of Wilms tumor. Genitourinary: Ambiguous genitalia noted; testes may be undescended. Renal: Hypertension noted secondary to renal failure. Edema: Generalized anasarca present due to nephrotic syndrome.

Treatment Protocol

Management plan: 1. Nephrology: Aggressive management of nephrotic syndrome; preparation for renal replacement therapy (dialysis/transplant). 2. Oncology: Quarterly renal ultrasound and serum AFP/HCG monitoring for early detection of Wilms tumor. 3. Endocrinology: Evaluation for gonadal dysgenesis and hormone replacement therapy. 4. Surgical: Prophylactic bilateral nephrectomy may be considered due to extremely high risk of Wilms tumor.

Detailed clinical guide coming soon.