N-acetylcysteine treatment for skin-picking in children and young adults with pws: a randomized placebo-controlled cross-over trial

Demi Trueba-Timmermans; Anne Rochtus; Lionne Grootjen; Gerthe Kerkhof; Edmond Rings; Anita Hokken-Koelega

doi:10.1530/endoabs.110.P624

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Endocrine Abstracts (2025) 110 P624 | DOI: 10.1530/endoabs.110.P624

ECEESPE2025 Poster Presentations Growth Axis and Syndromes (91 abstracts)

N-acetylcysteine treatment for skin-picking in children and young adults with pws: a randomized placebo-controlled cross-over trial

Demi Trueba-Timmermans ^{1 2 3} , Anne Rochtus ⁴ , Lionne Grootjen ^{1 2 5} , Gerthe Kerkhof ¹ , 5 , Edmond Rings ⁶ & Anita Hokken-Koelega ^{1 2 7}

Author affiliations

¹Dutch Reference Center for Prader-Willi Syndrome, Rotterdam, Netherlands; ²Dutch Growth Research Foundation, Rotterdam, Netherlands; ³Erasmus MC Sophia Children’s Hospital, Department of Pediatrics, Subdivision of Endocrinology, Rotterdam, Netherlands; ⁴University of Leuven, Department of Pediatrics, Leuven, Belgium; ⁵Erasmus MC Sophia Children’s Hospital, Department of Pediatrics, Subdivision of Endocrinology, Rotterdam, Netherlands; ⁶Erasmus MC Sophia Children’s Hospital, Department of Pediatrics, Rotterdam, Netherlands; ⁷Erasmus MC Sophia Children’s Hospital, Rotterdam, Netherlands

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Introduction: Skin-picking is the most common form of self-injurious behavior in Prader Willi Syndrome (PWS) and can lead to serious, life-threatening infections and severe scarring. An open-label pilot study reported that N-acetylcysteine (NAC) reduced skin-picking behavior in patients with PWS. Thus, NAC might be a promising treatment option for patients with PWS and skin-picking behavior. A placebo-controlled study was needed to show evidence.

Methods: A randomized, double-blind, placebo-controlled, cross-over trial in 23 patients with PWS aged 6-25 years in The Netherlands and Belgium. Cross-over intervention with NAC (dose range 600-2400 mg/day) and placebo, both during 3 months, with a wash-out period of 3 months.

Results: Overall, NAC lead to less skin-picking lesions compared to placebo, albeit not significantly (P = 0. 07). In boys, NAC showed a trend towards less skin-picking lesions compared to placebo (P = 0. 06) and the Clinical Global Impression Scale improved (2 (2-3) vs. 3 (2-3), P < 0. 01)), while no difference was observed in girls. In addition, median (IQR) Skin-Picking Symptom Assessment-score was lower after NAC compared to placebo (18 (10. 75-26) vs. 24 (13. 5-30. 75), P = 0. 05)). NAC was well-tolerated and there were no serious adverse events.

Conclusion: NAC appears to have beneficial effects on skin-picking in a subgroup of patients with PWS, particularly in boys, without safety concerns. While NAC may be considered in children and young adults with PWS, its effects should be assessed on an individual basis. Treatment should be discontinued if no significant benefits are observed.