Central China Rosai-Dorfman Disease Registry

Phase 4

• Conditions: Rosai-Dorfman Disease; Langerhans Cell Histiocytosis
• Interventions: Drug: Mycophenolate mofetil combined with oral prednisone

• Registration Number: NCT05284942
• Lead Sponsor: Tongji Hospital

Brief Summary

This is a research study among patients with Rosai-Dorfman disease.

Detailed Description

Rosai-Dorfman disease is a rare disorder characterized by overproduction (proliferation) and accumulation of a specific type of white blood cell (histiocyte) in the lymph nodes of the body (lymphadenopathy), most often those of the neck (cervical lymphadenopathy). In some cases, abnormal accumulation of histiocytes may occur in other areas of the body besides the lymph nodes (extranodal). These areas include the skin, central nervous system, kidney, and digestive tract. The symptoms and physical findings associated with Rosai-Dorfman disease vary depending upon the specific areas of the body that are affected. The disorder predominantly affects mainly adolescents or young adults. The exact cause of Rosa Dorfman disease is unknown. The purpose of this research study is to learn the prevalence , therapies and long term prognosis of Rosa Dorfman disease.

Study Timeline

• Study Start: 2011-10-01
• Study First Submitted: 2022-01-18
• Status Verified: 2022-03
• Study First Submitted QC: 2022-03-16
• Last Update Submitted: 2022-03-16
• Study First Posted: 2022-03-17
• Last Update Posted: 2022-03-17
• Primary Completion: 2022-06-01
• Study Completion: 2022-06-01

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

Diagnosis of a histiocytic disorder as determined by a corroborating constellation of histopathology, clinical, and/or radiologic findings. All participants will undergo Standard of Care MRA, CTA or imaging examination.

Exclusion Criteria

Known intracranial involvement of histiocytosis (including dura, leptomeninges and brain parenchyma) Prior stroke or intracranial hemorrhage Other (non-histiocytic) intracranial neoplasm or neurological disorder deemed by the PI or Co-PI to confound neuroimaging studies (e.g., demyelinating disease) Existing diagnosis of a psychiatric disorder or untreated mood disturbance Existing diagnosis of a neurodegenerative disease, such as Alzheimer's disease Chronic or daily excessive alcohol consumption as determined by the PI. History of chronic use of corticosteroids, defined as continuous treatment for six months or longer at any time in the past History of severe claustrophobia or other contraindications to patient SOC brain MRI Prior intravenous cytarabine or cladribine Other current or prior treatments (e.g., high-dose chemotherapy for a different cancer) deemed by the PI or Co-PI to confound imaging studies or cognitive performance

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Mycophenolate mofetil	Mycophenolate mofetil combined with oral prednisone	Mycophenolate mofetil oral 500mg twice a day from baseline to week 108

Primary Outcome Measures

Name	Time	Method
Autoimmune bullous disease quality of life (ABQoL)	From baseline to 108 Weeks	Total ABQoL scores range from 0 to 30 . The ABQoL score is calculated by summing the score of each question resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired.

Secondary Outcome Measures

Name	Time	Method
Cumulative Oral Corticosteroid Dose	From 12th, 24th, 36th ,52th and 108 Weeks	Calculate the Cumulative Oral Corticosteroid Dose

Trial Locations

Locations (1)

Tongji Hospital; 🇨🇳 Wuhan, Hubei, China