Treating Uncommon Dermatoses in Children: Off-Label Strategies, Biologics

For many rare and uncommon pediatric skin conditions, randomized controlled trial data simply do not exist. Clinicians must instead build treatment rationales from disease pathophysiology, case series, registry data, and expert consensus, while remaining transparent with families about the limits of available evidence.1

At Maui Derm NP+PA Summer 2026 in Colorado Springs, Dawn Eichenfield, MD, PhD, of Rady Children's Health, presented the session, “Outside the Box: Treating the Uncommon Dermatosis.” In the Q&A below, Eichenfield outlines how she approaches off-label treatment decisions and informed consent when the evidence base is thin and how advances in targeted biologics and JAK inhibitors have reshaped thinking about inflammatory skin diseases even in the absence of formal pediatric indications.

She also addresses where the field has made progress, including expanded hormonal therapy options for adolescent acne, and where gaps remain, particularly in hormonally driven disease in younger patients and rare inflammatory and genetic skin disorders that still lack prospective data, validated outcome measures, and approved therapies. This is part 2 of a 2-part Q&A; part 1 covered differential diagnosis and recognizing atypical presentations.2

HCPLive: When there are no approved therapies and no controlled trial data, how do you build a treatment rationale? What evidence sources do you lean on?

Eichenfield: This is a common challenge in pediatric dermatology. I build a treatment rationale using multiple layers of evidence: disease pathophysiology, adult literature, case series, registry data, expert consensus statements, and mechanistic understanding of available therapies. The rarity of many pediatric conditions means that randomized controlled trials are often unavailable. We frequently rely on thoughtful extrapolation while being transparent about the limitations of the evidence.

HCPLive: How do you approach off-label use with your patients, particularly the informed consent conversation when the evidence base is thin?

Eichenfield: Transparency is critical. I explain what is known, what is unknown, why I think the treatment may be beneficial, and what alternatives exist. Families appreciate understanding the rationale behind a recommendation, especially when formal approvals are lacking. I discuss expected benefits, potential risks, monitoring requirements, and the uncertainty surrounding outcomes. Shared decision-making becomes particularly important in these situations.

HCPLive: Has the emergence of targeted biologics and JAK inhibitors changed how you think about some of these conditions, even without formal indications?

Eichenfield: Absolutely. Advances in immunology have fundamentally changed how we conceptualize many inflammatory skin diseases. Even when a biologic or JAK inhibitor does not have a formal indication, understanding the relevant cytokine pathways can help guide treatment decisions and identify therapeutic targets. These medications have also demonstrated that diseases previously thought to be heterogeneous may actually share common inflammatory mechanisms.

HCPLive: Are there one or two conditions in this category where you feel the field has made meaningful progress in the last few years, even without formal approvals?

Eichenfield: In acne, the growing evidence supporting hormonal therapies, including combined oral contraceptives and spironolactone in appropriately selected adolescent patients, has expanded our ability to individualize treatment and reduce reliance on prolonged antibiotic use.

HCPLive: Conversely, are there conditions where you feel clinicians are most under-equipped—where you see the biggest gap between what patients need and what we currently have to offer?

Eichenfield: In acne, one important gap is the recognition and management of hormonally driven disease in younger adolescents. We still have limited pediatric-specific data regarding optimal hormonal treatment strategies, long-term outcomes, and predictors of response.

More broadly, rare inflammatory and genetic skin disorders continue to suffer from a lack of prospective studies, validated outcome measures, and approved therapies.

HCPLive: How much does multidisciplinary collaboration factor into managing these patients—rheumatology, hematology, and pathology—and how do you structure those relationships in practice?

Eichenfield: Multidisciplinary collaboration is often essential. For example, for disease states such as Crohn’s disease presenting as vulvar edema or Langerhans cell histiocytosis mimicking diaper dermatosis, we need multidisciplinary care to deliver the best clinical care. For hormonally mediated acne, endocrinology and adolescent medicine can be invaluable, particularly when evaluating hyperandrogenism or menstrual abnormalities.

HCPLive: Are there resources—networks, registries, or literature sources—you would point colleagues to when they encounter a case that stumps them?

Eichenfield: Pediatric dermatologists are fortunate to have a highly collaborative community. I encourage colleagues to leverage professional networks such as the Society for Pediatric Dermatology and Pediatric Dermatology Research Alliance, which provide opportunities for case discussion and collaboration.

Disease-specific registries, multidisciplinary specialty clinics, and consultation with experienced dermatopathologists are also invaluable. Finally, carefully reviewing recent case series and expert consensus statements can often provide practical guidance when high-level evidence is unavailable.

References

1. Eichenfield D. Outside the Box: Treating the Uncommon Dermatosis. Session presented at Maui Derm Summer 2026 in Colorado Springs on June 25.

2. Eichenfield D. Diagnosing Uncommon Dermatoses in Children: LCH, Psoriasis, and More. HCPLive. Published June 25, 2026. Accessed June 25, 2026. https://www.hcplive.com/view/diagnosing-uncommon-dermatoses-children-lch-psoriasis