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Kidney Week

Abstract: TH-PO530

Adrenocorticotropic Hormone (ACTH) in the Treatment of Refractory Childhood Nephrotic Syndrome (NS)

Session Information

  • Trainee Case Reports - I
    October 25, 2018 | Location: Exhibit Hall, San Diego Convention Center
    Abstract Time: 10:00 AM - 12:00 PM

Category: Trainee Case Reports

  • 1600 Pediatric Nephrology


  • Chinnadurai, Amirtha, Yale University School of Medicine, New Haven, Connecticut, United States
  • Goodwin, Julie, Yale University School of Medicine, New Haven, Connecticut, United States

Treatment resistance or failure to respond to conventional therapies in NS is associated with poor prognosis. ACTH is recognized as therapy for podocytopathies, though these data are based on small observational studies in adults. There are no published data on the use of ACTH therapy in the pediatric population. We report a case of steroid-resistant NS in a 10 year-old boy who failed multiple secondary therapies but showed a partial response to ACTH injection (ACTHAR) therapy.

Case Description

A 10-year-old Hispanic boy who was diagnosed with NS at the age of 2. He was initially steroid sensitive but frequently relapsing. Cyclosporine A (CsA) was initiated as a steroid-sparing agent and he achieved a 5-year period of stability. However, he eventually began to relapse while on CsA. Tacrolimus and mycophenolate mofetil (MMF) were also ineffective. During this time he became steroid resistant. Triple therapy with steroids, tacrolimus and MMF was unable to induce even a partial remission. A renal biopsy showed early focal segmental glomerulosclerosis (FSGS). Whole exome sequencing showed only a heterozygous variant in PLCE1. He became dependent on twice weekly albumin infusions due to anasarca and persistent hypoalbuminemia (less than 1.2g/dl). Urine protein/creatinine (UPC) ratios were persistently in the nephrotic range. Over 18 months he persisted in uncontrolled relapse, complicated with 5 episodes of acute kidney injury. His creatinine plateaued at 0.7-0.8 mg/dl, whereas in remission it had been 0.3-0.4 mg/dL. We initiated ACTHAR as a last resort given concerns for progressive renal impairment. Initial therapeutic response was minimal with dose of 40 units biweekly. After increment to 80 units biweekly, he demonstrated significant improvement. His serum creatinine improved to his baseline of 0.30 mg/dl and UPCs decreased by more than 50%. Serum albumin improved spontaneously to 1.9-2.0g/dl, resulting in discontinuation of scheduled albumin infusions.


This case highlights that ACTHAR may be a viable therapeutic alternative for children who are resistant to other therapies for NS. Additionally, the role of his PLCE1 mutation is unclear but we suspect a compound heterozygous state for another unidentified pathogenic mutation and/or epigenetic modifications may be playing a role in his refractory NS.