[Clinical efficacy and safety of lonafarnib in children with Hutchinson-Gilford progeria syndrome].

Authors

Y Huang, J J Wang, C J Wang, X X Tang, Q M Yu, H D Fu, L D Hu, J H Mao

Published in

Zhonghua er ke za zhi = Chinese journal of pediatrics. Volume 64. Issue 9. Pages 1009-1014. Aug 17, 2026. Epub Aug 17, 2026.

Abstract

Objective: To evaluate the efficacy and safety of lonafarnib, in children with Hutchinson-Gilford progeria syndrome(HGPS). Methods: This was a prospective cohort study. A total of 12 children with HGPS who were treated with lonafarnib at the Children's Hospital of Zhejiang University School of Medicine between April 2022 and November 2025 were enrolled. Clinical data collected within 1 week before initial medication were taken as the baseline. The first follow-up was performed 4 to 12 months after treatment initiation, with complete laboratory tests and imaging examinations collected. The follow-up period ended in March 2026. Paired-samples t test and Wilcoxon signed-rank test were used to compare indicators including height, weight, blood lipid levels, pulse wave velocity, and areal bone mineral density before and after treatment. Results: Among the 12 children, 6 were boys and 6 girls. The age at diagnosis was 0.8 (0.3, 4.8) years, including 10 cases of classic HGPS and 2 cases of non-classic HGPS. The follow-up duration was (7±2) months. Two patients were lost to follow-up. In the 10 children who completed follow-up, height increased significantly from baseline (93 (83, 102) vs. 97 (85, 103) cm, Z=2.81, P=0.002), while no significant change was observed in height-for-age Z-score (Z=0.46, P=0.682). Low-density lipoprotein decreased significantly (2.8 (2.3, 3.5)vs. 2.5 (2.1, 2.8) mmol/L, Z=2.13, P=0.031). No statistically significant change was found in weight, liver and renal function, homeostasis model assessment of insulin resistance, pulse wave velocity, carotid intima-media thickness and areal bone mineral density before and after treatment (all P>0.05). All children experienced gastrointestinal adverse reactions of varying degrees during treatment, which were predominantly mild to moderate and relieved after dose adjustment. One child discontinued treatment due to drug allergy. Conclusions: Lonafarnib demonstrated an overall favorable safety and tolerability profile. Early gastrointestinal adverse events were mostly reversible and manageable, with no significant drug-related hepatic or renal impairment observed. Short-term follow-up showed that only low-density lipoprotein improved significantly, with no clear evidence of growth benefit, and no progressive aggravation of vascular or skeletal lesions was detected.

PMID:
42605084
Bibliographic data and abstract were imported from PubMed on 17 Aug 2026.

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