Tripterygium wilfordii Hook F. in the treatment of synovitis, acne, pustulosis, hyperostosis, and osteitis syndrome: a clinical trial

Objective: This study aimed to investigate the efficacy and safety of Tripterygium wilfordii Hook F. (TwHF) in the treatment of osteoarticular lesions in synovitis, acne, pustulosis, hyperostosis, and osteitis (SAPHO) syndrome. Methods: Eligible SAPHO patients were recruited to this single-center trial to receive 12-week TwHF treatment. Two dose groups (1.0-mg/kg/day group and 1.5-mg/kg/day group) were designed and patients were allocated (1:1) to these two groups. The primary endpoint was the change from baseline in Ankylosing Spondylitis Disease Activity Score on the basis of C-reactive protein level (ASDAS) at week 12. Results: All the 30 included patients completed the trial. At week 12, both dose groups showed significant change from baseline in ASDAS (1.0-mg/kg/day group: − 1.34 (1.10), p = 0.000; 1.5-mg/kg/day group: − 1.53 (1.19), p = 0.000). Similar improvement was also found in the Visual Analogue Scale in global osteoarticular pain, Bath Ankylosing Spondylitis Disease Activity Index, and other efficacy measures. The results showed a fast-acting characteristic of TwHF that the maximum efficacy was achieved within the first 2–4 weeks and maintained at a stable level for the rest of the study. No significant differences were observed between the two dose groups under the current sample size. TwHF was well tolerated that no severe adverse events or irregular menstruation were recorded, except for one patient who developed severe alanine aminotransferase elevation at the last follow-up and has stopped the TwHF treatment after the 12-week follow-up. Conclusions: TwHF should be considered for the treatment of osteoarticular lesions in SAPHO syndrome in clinical practice because of significant efficacy, reliable safety, and high socioeconomic value. Trial registration: ChiCTR1900025912• This is the first clinical trial to evaluate Tripterygium wilfordii Hook F. (TwHF) in the treatment of synovitis, acne, pustulosis, hyperostosis, and osteitis (SAPHO) syndrome.• Twelve-week TwHF treatment in both dose groups designed (1.0-mg/kg/day group and 1.5-mg/kg/day group) was well tolerated and could lead to significant disease remission of SAPHO syndrome.• No significant differences were observed between the two dose groups under the current sample size.• TwHF should be considered for the treatment of osteoarticular lesions in SAPHO syndrome in clinical practice because of significant efficacy, reliable safety, and high socioeconomic value.