To describe the outcomes of rhIGF-1 treatment in patients with Donohue syndrome (DS) and Rabson-Mendenhall syndrome (RMS) and to highlight the significance of comparing these outcomes to previously published cases.
Key Findings:
Metabolic benefits of rhIGF-1 included improved glycemic control, fasting tolerance in early life, and some growth enhancement.
Two patients exhibited poor response or intolerance to rhIGF-1 and died in infancy.
The two longest-lived patients progressed to diabetes mellitus despite rhIGF-1 therapy.
New features associated with severe insulin receptoropathies included cataract, liver hemangioma, and impaired hepatic protein synthesis.
Interpretation:
Current treatment options for DS and RMS remain unsatisfactory, with rhIGF-1 providing limited benefits and not preventing diabetes mellitus decompensation; thus, there is an urgent need for alternative treatment strategies.
Limitations:
Small sample size and retrospective nature of the study.
Lack of controlled studies and potential biases in existing literature, including selection and reporting biases.
Conclusion:
Further research is urgently needed to identify alternative treatment strategies for extreme forms of insulin resistance, as high-dose rhIGF-1 has only limited beneficial effects.
Harold Burstein, MD, PhD, and Jennifer A. Ligibel, MD discuss new research from the Breast Cancer Weight Loss (BWEL) trial, as well as recommendations for lifestyle, diet and GLP1 agonists.
