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  • Presentation

Biologics in Congenital Ichthyosis: Are They Effective?

Description

The discussion centered around the effectiveness of biologics in treating congenital ichthyosis, prompted by observed cytokine alterations and the potential to repurpose existing biologics. A retrospective study involving 98 patients from various countries, primarily with types such as Netherton syndrome and congenital ichthyosis from erythroderma, was conducted to assess the impact of biologics. Results indicated that efficacy varied significantly—with 46% of patients responding positively to treatment, while 54% were non-responders, including some experiencing worsening symptoms. Among responders, improvements in pruritus, erythema, and scaling were noted, and some patients reported secondary benefits like enhanced hair growth and quality of life. However, a substantial proportion withdrew from treatment due to ineffectiveness or side effects, with a notable portion experiencing only transient improvements. Analysis suggested that IL 12/23 and IL 4 inhibitors showed better efficacy compared to IL 17. The findings highlight limitations such as the heterogeneous patient population, treatment regimens, and a lack of robust statistical comparisons due to study design. Despite some positive outcomes, high success rates in published literature likely reflect a publication bias, emphasizing that biologics should be cautiously considered primarily for severe erythrodermic cases. The study calls for more stringent clinical trials to substantiate these findings and better understand the utility of biologics in congenital ichthyosis.

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Conclusions

  • Biologics may be effective in treating severe congenital ichthyosis, particularly in erythrodermic forms like Netherton syndrome.
  • The response rate to biologics was 46%, indicating variability in efficacy.
  • 54% of patients were non-responders, including some with worsened conditions while on treatment.
  • Most responders exhibited only mild to moderate improvements, with marked improvement seen in only 1% of cases.
  • Positive benefits included reductions in pruritus and scaling, with some patients experiencing improvements in quality of life.
  • A trend suggested higher response rates for IL-12/23 and IL-4/13 inhibitors compared to IL-17 inhibitors.
  • Subgroup analysis was limited by the heterogeneity of the patient population and potential indication biases.
  • There were few side effects reported, but physicians should remain cautious about treatment efficacy and potential worsening of the condition.
  • Biologics are considered off-label for this use and are costly, necessitating careful consideration for each patient.
  • Future clinical trials are needed to better define the role of biologics in treating congenital ichthyosis.
  • Hernández-Martín A et al. N Engl J Med. 2019
  • Süßmuth K et al. J Eur Acad Dermatol Venereol. 2021 ;35 :e152-e155
  • Br J Dermato/ 2025; 192:327-334
  • Advance access publication date: 29 October 2024
  • Medical Dermatology