Central China Rosai-Dorfman Disease Registry

Study Overview
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Brief Summary
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This is a research study among patients with Rosai-Dorfman disease.

Detailed Description
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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.

Official Title
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A Trial to Evaluate the Long Term Prognosis in Rosai-Dorfman Disease

Conditions
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Rosai-Dorfman Disease, Langerhans Cell Histiocytosis

Intervention / Treatment
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* Drug: Mycophenolate mofetil combined with oral prednisone


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Mycophenolate mofetil combined with stand of care of oral prednisone 5mg-15mg/day


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Mycophenolate mofetil<br>Mycophenolate mofetil oral 500mg twice a day from baseline to week 108 | Drug: Mycophenolate mofetil combined with oral prednisone<br>* Mycophenolate mofetil oral 500mg twice a day combined with standard of care of oral prednisone 5mg-15mg/day from baseline to week 108<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Autoimmune bullous disease quality of life (ABQoL) | 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. | From baseline to 108 Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Cumulative Oral Corticosteroid Dose | Calculate the Cumulative Oral Corticosteroid Dose | From 12th, 24th, 36th ,52th and 108 Weeks |