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In this study, the investigators will determine the utility of Tamoxifen, a non-cytotoxic agent, to improve quality of life, biochemical parameters, and bone marrow involvement in systemic mastocytosis patients having 1) up to 40% bone marrow infiltration by mast cells and/or 2) mediator-release symptoms which are not controlled by tolerated doses of standard "non-cytotoxic" medications regardless of the percentage bone marrow involvement by mastocytosis. The dose of Tamoxifen will be 40 mg/day and the duration of treatment will be for one year. Patients currently taking interferon alfa, imatinib mesylate, or cladribine will be excluded until these medications have been stopped.
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| Measure | Description | Time Frame |
|---|---|---|
| Stability or reduction of the percent bone marrow involvement by mastocytosis, including stable mast cell morphology and phenotype. | up to 40% bone marrow infiltration by mast cells and/or 2) mediator-release symptoms which are not controlled by tolerated doses of standard "non-cytotoxic" medications regardless of the percentage bone marrow involvement by mastocytosis. The dose of Tamoxifen will be 40mg/day and the duration of treatment will be for one year. | 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| Stability or improvement in biochemical markers of systemic mastocytosis | Stability or improvement in biochemical markers of systemic mastocytosis (serum tryptase, calcitonin, urinary N-methyl histamine and prostaglandin F2 excretion/24 hours, liver function studies, lactic dehydrogenase, complete blood count with leukocyte differential) | 1 year |
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Inclusion Criteria:
Exclusion Criteria:
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Patients with systemic mastocytosis having up to 20% bone marrow involvement or clinical symptoms not controlled on current medications.
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| Name | Affiliation | Role |
|---|---|---|
| Joseph H Butterfield, MD | Mayo Clinic | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mayo Clinic | Rochester | Minnesota | 55901 | United States |
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| ID | Term |
|---|---|
| D034721 | Mastocytosis, Systemic |
| ID | Term |
|---|---|
| D008415 | Mastocytosis |
| D009372 | Neoplasms, Connective Tissue |
| D018204 | Neoplasms, Connective and Soft Tissue |
| D009370 | Neoplasms by Histologic Type |
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| D009369 | Neoplasms |
| D000090362 | Mast Cell Activation Disorders |
| D007154 | Immune System Diseases |