None listed
Conditions
Brief summary
The main purpose of our study is to identify an effective and tolerable treatment for Erdheim-Chester disease. To date, various regimens have been used with variable results and often burdened by severe toxic effects. On the basis of rapamycin characteristics and of the information available about this disease, we think Sirolimus (one of the commercially available forms of rapamycin) associated with steroids, such as prednisone, could be an alternative therapeutic approach for this disease. Prednisone is given orally at the initial dose of 0.75 mg/kg/day for 1 month, tapered to 2.5-5 mg/day over 6 months. Rapamycin is given orally at a daily dose of 2-4 mg, with a target trough level of 8-12 ng/mL. The minimum duration of treatment to assess response to therapy is 6 months; if disease stabilisation or remission is achieved, the treatment is continued usually until the end of the second year; then, the treatment can be continued or stopped at the discretion of the treating clinician.
Interventions
Prednisone is given orally at the initial dose of 0.75 mg/kg/day for 1 month, tapered to 2.5-5 mg/day over 6 months.
Rapamycin is given orally at a daily dose of 2-4 mg, with a target trough plasmatic level of 8-12 ng/mL.
The minimum duration of treatment to assess response to therapy is 6 months. If disease stabilisation or remission is achieved, we plan to continue treatment for an overall duration of 2 years. After the end of year 2, the decision as to whether treatment has to be continued o
Prednisone is given orally at the initial dose of 0.75 mg/kg/day for 1 month, tapered to 2.5-5 mg/day over 6 months. Rapamycin is given orally at a daily dose of 2-4 mg, with a target trough plasmatic level of 8-12 ng/mL. The minimum duration of treatment to assess response to therapy is 6 months. If disease stabilisation or remission is achieved, we plan to continue treatment for an overall duration of 2 years. After the end of year 2, the decision as to whether treatment has to be continued or stopped is left at the discretion of the treating clinician and the patient. The response to treatment and the monitoring for side effects is assessed by means of clinical examination and appropriate laboratory and imaging studies (CT, MRI, PET/CT, bone scintigraphy). To monitor the adherence to sirolimus therapy and to modulate sirolimus dose, we evaluate plasma concentration of sirolimus (trough levels) which must be between 8 and 12 ng/mL.
Sponsors
University of Parma, Department of Clinical and Experiment Medicine
Study design
Allocation
Non-randomised trial
Intervention model
Single group
Primary purpose
Treatment
Masking
Open (masking not used)
Eligibility
Sex/Gender
All
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
- Diagnosis of Erdheim-Chester with systemic involvement - Active disease (newly diagnosed progressive disease in patients undergoing other treatments)
Exclusion criteria
- Concurrent active malignancies or serious infections - Hypersensitivity to sirolimus and/or prednisone and/or contraindications to their use (uncontrolled diabetes mellitus, severe osteoporosis with previous fractures) - Proteinuria > 1g/24h - Recent major surgery and/or post-surgical complications - Pregnancy - Patients partecipating in other trials
Outcome results
None listed