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In current practice, options for venous and lymphatic malformations remain limited. Recently an oral medication, sirolimus, has been found to benefit patients when taken once or twice a day for several months. Unfortunately there are many side effects associated with this medication, some of which can be severe including, neutropenia, oral ulcerations, and lab abnormalities. The purpose of this study is to determine if once weekly dosed sirolimus will be effective for the treatment of venous and lymphatic malformations. Additionally, the study will evaluate patient satisfaction and identify adverse effects. Participants will be on the medication for 6 months with an option to continue after this time period.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment Group | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Sirolimus | Drug | Participants will get Sirolimus (1.5-2 2mg/m2) weekly for 6 months. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in size of lesion | Will be measuring the size of the lesions (mm) at each patient visit | Baseline and 6 months |
| Change in size of lesion through photograph | Will be evaluating clinical photographs of lesions at each patient visit | Baseline and 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Number of side effects experienced | Patient will complete side effect questionnaires at each visit | Month One |
| Number of side effects experienced | Patient will complete side effect questionnaires at each visit |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Stephnie Munie, BS | Contact | 843-566-2453 | munie@musc.edu |
| Name | Affiliation | Role |
|---|---|---|
| Chelsea Shope, MSCR | Medical University of South Carolina | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Medical University of South Carolina | Recruiting | Charleston | South Carolina | 29403 | United States |
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| ID | Term |
|---|---|
| D044148 | Lymphatic Abnormalities |
| ID | Term |
|---|---|
| D008206 | Lymphatic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| D020123 | Sirolimus |
| ID | Term |
|---|---|
| D018942 | Macrolides |
| D007783 | Lactones |
| D009930 | Organic Chemicals |
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| Month Two |
| Number of side effects experienced | Patient will complete side effect questionnaires at each visit | Month Three |
| Number of side effects experienced | Patient will complete side effect questionnaires at each visit | Month Four |
| Number of side effects experienced | Patient will complete side effect questionnaires at each visit | Month Five |
| Number of side effects experienced | Patient will complete side effect questionnaires at each visit | Month Six |
| Change in quality of life as assessed by questionnaire | Patient will complete quality of life questionnaire at each visit | Baseline and 6 months |
| Number of participants with laboratory abnormalities | Standard of care laboratory results (CBC, CMP, triglycerides) will be monitored | From baseline visit to 2 month visit |