Pilot Study of Tocilizumab Monotherapy for Active Chronic Periaortitis

This is a prospective study to investigate the treatment response of Tocilizumab on patients with active chronic periaortitis (CP).

Methods: patients with a definite or possible diagnosis of CP at acute active stage were enrolled for this study and accepted Tocilizumab monotherapy for 3 months.

Endpoints: The primary endpoint is to investigate the treatment response of Tocilizumab; the secondary endpoints include the improvement of inflammatory markers, the frequency of adverse events.

Study Overview

• Status: Completed
• Conditions: Chronic Periaortitis; Tocilizumab Monotherapy
• Intervention / Treatment: Drug: Tocilizumab monotherapy

Detailed Description

Patients enrolled received intravenous infusions of TCZ (8 mg/kg) at inclusion and then every 4 weeks for at least 3 months. Demographic and clinical features, laboratory findings and imaging examinations were recorded at baseline and during 3-month follow-up. Imaging improvement was converted into the ratio of perivascular soft tissues shrinkage by evaluating 2 dimensions of greatest change on computed tomography (CT) at baseline and after 3 months. Partial remission was defined as obtaining alleviation of symptoms and normalization of inflammatory markers including erythrocyte sedimentation rate (ESR) and high-sensitivity C-reactive protein (hsCRP), with shrinkage of soft tissue mass in imaging <70%. Further, complete remission was defined as normalization of inflammatory markers accompanied by shrinkage of soft tissue mass ≥70%.

• Study Type: Observational
• Enrollment (Actual): 12

Contacts and Locations

• Study Contact: Name: Yunyun Fei; Phone Number: +8613681125226; Email: feiyunyun@pumch.cn

Study Locations

• China
  • Beijing, China
    • Yunyun Fei

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Chronic periaortitis (CP) is characterized by the presence of mass that develops around the thoracic aorta, abdominal aorta or iliac arteries, with predominance in males of middle and elderly age.

Description

Inclusion Criteria:

1. adults ≥ 18 years of age at time of informed consent;
2. meet clinical diagnostic criteria including (1) imaging findings show perivascular soft tissue density mass surrounding thoracic aorta, abdominal aorta or iliac arteries; (2) histopathological findings show fibrous tissue with chronic inflammatory infiltrate comprised of lymphocytes, plasma cells and macrophages (Neutrophils and granulomas are rare). Patients who satisfied (1) but without histopathological examination were perceived as possible CP;
3. at active stage: clinical symptoms or organ involvement; a higher level of ESR and CRP than normal;

Exclusion Criteria:

1. Secondary forms of CP related to drugs, infections, malignancies, Erdheim-Chester disease or other autoimmune diseases were excluded.
2. Combined with other autoimmune diseases.
3. Known immunodeficiency disorder.
4. Pregnancy, lactation, or planning to become pregnant within 6 months of the test.
5. Positive test for, or prior treatment for, hepatitis B or HIV infection. A positive test for hepatitis B is detection of hepatitis B surface antigen (HBsAg) or HBV-DNA.
6. Evidence of active tuberculosis (TB), including Chest X-ray and PPD.
7. Severe abnormal liver function or cardiac insufficiency.
8. Any reason the investigator think that should not attend this trial.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Tocilizumab monotherapy; Patients with a definite or possible diagnosis of CP at acute active stage were enrolled for this study. Clinical diagnostic criteria consist of: (1) imaging findings show perivascular soft tissue density mass surrounding thoracic aorta, abdominal aorta or iliac arteries; (2) histopathological findings show fibrous tissue with chronic inflammatory infiltrate comprised of lymphocytes, plasma cells and macrophages (Neutrophils and granulomas are rare). Patients who satisfied (1) but without histopathological examination were perceived as possible CP. Secondary forms of CP related to drugs, infections, malignancies, Erdheim-Chester disease or other autoimmune diseases were excluded. Patients enrolled received intravenous infusions of TCZ (8 mg/kg) at inclusion and then every 4 weeks for at least 3 months.

Drug: Tocilizumab monotherapy; intravenous infusions of TCZ (8 mg/kg) at inclusion and then every 4 weeks for at least 3 months

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
treatment response	the rate of partial remission and complete remission after 3-month TCZ monotherapy	three months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
the frequency of treatment related adverse events	adverse events caused by TCZ during 3-month follow-up.	three months
the improvement of inflammatory markers	including erythrocyte sedimentation rate (ESR) and high-sensitivity C-reactive protein (hsCRP).	three months

Collaborators and Investigators

• Sponsor: Peking Union Medical College Hospital

Study record dates

Study Major Dates

• Study Start (Actual): July 15, 2020
• Primary Completion (Actual): December 30, 2022
• Study Completion (Actual): December 30, 2022

Study Registration Dates

• First Submitted: November 14, 2021
• First Submitted That Met QC Criteria: November 14, 2021
• First Posted (Actual): November 24, 2021

Study Record Updates

• Last Update Posted (Actual): March 2, 2023
• Last Update Submitted That Met QC Criteria: March 1, 2023
• Last Verified: March 1, 2023

More Information

Terms related to this study

• Keywords: Tocilizumab; treatment response; complete remission; partial remission; chronic periaortitis
• Additional Relevant MeSH Terms: Pathologic Processes; Fibrosis; Retroperitoneal Fibrosis
• Other Study ID Numbers: Tocilizumab for Periaortitis

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No