Adrenocorticotropic Hormone for Steroid-Resistant and Oral Steroid-Intolerant Children With Minimal Change Nephrotic Syndrome

Background: The most frequent cause of nephrotic syndrome (NS) in childhood, minimal change disease, is generally steroid-responsive. Children who are intolerant of oral steroids or steroid-resistant still present a significant therapeutic challenge. Additionally, adverse effects of long-term corticosteroid administration warrant the search for an alternative safe and effective treatment. Here we report the use of adrenocorticotropic hormone (ACTH; H.P. Acthar® Gel), an agent originally introduced in the 1950s, for the treatment of these patients.

Methods: We report 3 children with NS who were not able to attain remission with standard steroid therapy due to oral steroid intolerance or steroid resistance.

Results: Two of the patients attained remission with a long-acting formulation of ACTH.

Conclusions: ACTH is an option for the treatment of pediatric patients with NS who are oral steroid-intolerant. This approach may also be useful for the treatment of other steroid-responsive conditions in patients with limiting gastrointestinal toxicity to oral preparations. The use of ACTH for the treatment of steroid-resistant NS requires further investigation.