TOPLINE:

Patients with Takayasu arteritis treated with mycophenolate mofetil (MMF) combined with methotrexate achieved greater overall response rates at week 52 than those treated with cyclophosphamide followed by azathioprine, with a favorable safety profile.

METHODOLOGY:

• Researchers conducted an open-label randomized trial across multiple centers in China to evaluate the efficacy of MMF combined with methotrexate in treating Takayasu arteritis.
• They included 111 adult patients with active Takayasu arteritis (median age at recruitment, 30 years; 85.6% women) between February 2017 and September 2021.
• Among the patients, 74 were randomly assigned to receive MMF (750 or 1000 mg twice daily) plus oral methotrexate (15 mg weekly), and 37 were assigned to receive 15 mg/kg intravenous cyclophosphamide followed by 100 mg oral azathioprine. All patients received high-dose glucocorticoids with a predefined tapering regime.
• The primary endpoint was the overall response rate, defined as the proportion of patients achieving complete or partial response according to resolution of signs and symptoms of active disease, normalization of erythrocyte sedimentation rate and C-reactive protein levels, absence of progression on imaging, and glucocorticoid dose < 15 mg/d, assessed at week 52.
• Secondary endpoints included response rates at weeks 28 and 52 and relapse rates; occurrence of adverse events was also assessed.

TAKEAWAY:

• By week 52, 55.4% of patients receiving MMF plus methotrexate achieved an overall response compared with 32.4% of those receiving cyclophosphamide followed by azathioprine (odds ratio [OR], 2.59; P = .022).
• Patients receiving MMF plus methotrexate had higher odds of achieving an overall response at week 28 than those receiving cyclophosphamide followed by azathioprine (OR, 2.89; P = .011).
• By week 52, relapse occurred in four patients who initially responded to MMF plus methotrexate and in two patients treated with cyclophosphamide followed by azathioprine; all patients were managed with treatment adjustments.
• The reports of adverse events were similar in both groups, with only one severe adverse event in a patient treated with cyclophosphamide followed by azathioprine leading to discontinuation of treatment.

IN PRACTICE:

“The [study] results provide much-needed trial-level evidence for immunosuppressive treatments in TAK [Takayasu arteritis], including the use of drugs that are currently much less expensive than biologic agents, an important factor in many countries with high prevalences of TAK,” the authors of the study wrote.

SOURCE:

This study was led by Xiaochuan Sun and Jing Li, Peking Union Medical College Hospital in Beijing, China. It was published online on August 22, 2025, in Annals of the Rheumatic Diseases.

LIMITATIONS:

The sample size was calculated as per efficacy estimates from studies on lupus nephritis owing to limited data on Takayasu arteritis. The sample size fell short of its target due to the COVID pandemic. A 52-week follow-up period may have been relatively short for a chronic disease prone to relapse.

DISCLOSURES:

This study received funding from CAMS Innovation Fund for Medical Sciences and National High Level Hospital Clinical Research Funding. Hunan Warrant Pharmaceutical supplied MMF for this study. One author reported receiving funds for consulting, research support, and royalties from and having stock options in various pharmaceutical, healthcare, and biotechnology companies, including AbbVie, Metagenomi, and Novartis.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.