Langerhans Cell Histiocytosis, Rosai-Dorfman Disease
Conditions
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.
Interventions
DRUGMycophenolate mofetil combined with oral prednisone
Mycophenolate mofetil oral 500mg twice a day combined with standard of care of oral prednisone 5mg-15mg/day from baseline to week 108
Sponsors
Tongji Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Intervention model description
Mycophenolate mofetil combined with stand of care of oral prednisone 5mg-15mg/day
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| Cumulative Oral Corticosteroid Dose | From 12th, 24th, 36th ,52th and 108 Weeks | Calculate the Cumulative Oral Corticosteroid Dose |
Countries
China
Contacts
Primary ContactYIKAI YU, M.D
Backup ContactXIAO LUO, M.D
Outcome results
None listed