From the Research
Ustekinumab (Stelara) is not recommended for treating pediatric Graft-Versus-Host Disease (GVHD) due to limited evidence supporting its use in this population. The primary approvals for ustekinumab are for psoriasis, psoriatic arthritis, Crohn's disease, and ulcerative colitis in adults and some pediatric populations 1. For pediatric GVHD, standard first-line treatments include corticosteroids, calcineurin inhibitors (tacrolimus, cyclosporine), and other immunosuppressants. In refractory cases where standard therapies fail, physicians sometimes consider off-label biologics, but ustekinumab is not commonly used for this purpose.
Mechanism and Potential Use
Ustekinumab works by blocking interleukin-12 and interleukin-23, which are inflammatory cytokines involved in immune responses. While this mechanism could theoretically help in GVHD by reducing T-cell-mediated inflammation, clinical evidence specifically for pediatric GVHD is lacking.
Alternative Treatments
Other treatments have shown promise in steroid-refractory acute and chronic GVHD, such as ruxolitinib, which has a high overall response rate of 77% and 89%, respectively, in acute and chronic GVHD 2. Basiliximab has also been effective as a second-line treatment in children with steroid-refractory acute GVHD, particularly for skin involvement 3. Etanercept has been used successfully as a second-line treatment for steroid-refractory acute GVHD in pediatric patients, with an overall response rate of 78% 4.
Considerations for Ustekinumab Use
Any consideration of ustekinumab for pediatric GVHD would be experimental and should occur only in specialized transplant centers under careful monitoring for efficacy and adverse effects, with appropriate informed consent discussions with patients and families. The lack of strong evidence supporting its use in pediatric GVHD, combined with the availability of other treatments with more established efficacy, makes ustekinumab a less preferred option for this condition.
Key Points
- Ustekinumab is not FDA-approved for pediatric GVHD.
- Limited evidence supports its use in pediatric GVHD.
- Standard treatments for pediatric GVHD include corticosteroids and calcineurin inhibitors.
- Alternative biologics like ruxolitinib, basiliximab, and etanercept have shown promise in refractory GVHD.