OFF Label ON Children

Jim Treat, MD, Professor of Clinical Pediatrics and Dermatology, Perelman School of Medicine, Associate Chair for IT for the Department of Pediatrics, Fellowship Director, Pediatric Dermatology, Education Director, Pediatric Dermatology, Children’s Hospital of Philadelphia

December 2022

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Dr. Treat presented off-label uses of systemic medications for rare and common pediatric diseases.

Historically, most systemic pediatric medications, such as cyclosporine, methotrexate, and azathioprine, broadly affect the immune system. Newer, targeted therapies are more precise and target specific aspects of the immune system because they are based on the underlying pathophysiology and research findings. If the targeted therapy works for a particular disease, it may help elucidate the pathophysiology of that disease. However, the off-label use of targeted therapies is based on published studies of small numbers of patients. If the data were more robust (i.e., collected during randomized, controlled clinical trials), these therapies would be FDA-approved for the intended indication (i.e., on label). Importantly, off-label use of targeted therapies may not work for every patient.

Several systemic therapies are approved (i.e., indicated) for atopic dermatitis in children. These therapies also can be used off-label for rare and uncommon pediatric diseases affected by the same inflammatory pathways.

Dr. Treat presented examples of off-label use for dupilumab (IL-4/IL-13 inhibitor), omalizumab (IgE inhibitor), etanercept (TNF-alpha inhibitor), ustekinumab (IL-12/IL-23 inhibitor), methotrexate, and chimeric antigen T cell (CAR-T) therapy. Studies have reported off-label uses of dupilumab for diseases affecting other inflammatory pathways, including pruritus, prurigo nodularia, allergic contact dermatitis, eosinophillic annular erythema, bullous pemphigoid, papuloerythroderma of Ofuji.

An understanding of pathophysiology is important because medications that specifically target the immune system are now available.