Stem Cell Transplantation to Treat Systemic Mastocytosis

Inclusion Criteria

• •Ages greater than or equal to 2 to 80.
• •Wt greater than 40 kg.
• •Patients with systemic mastocytosis proven by a bone marrow biopsy and one of the following:
• •Category I disease with poor prognosis secondary to extensive bone marrow involvement of mast cells (i.e. greater than 50 percent of the bone marrow cavity replaced by mast cells) and evidence of a bone marrow failure state defined as presence of one of the following for greater than or equal to 2 months:
• •Neutrophil count less than 500/ mm3.
• •Platelet count less than 20,000/mm3.
• •Dependency on transfusion of RBC or platelets.
• •Category II disease with an associated hematologic disorder:
• •Myelodysplastic syndromes.
• •Myeloproliferative states except essential thrombocytosis or polycythemia vera.
• •Increased blasts in peripheral blood or bone marrow greater than 10 percent.
• •Bone marrow failure states as described above.
• •Category III disease with enlarged peripheral lymph nodes with histopathological evidence of the replacement of the entire lymph node architecture with mast cells and/or accompanying peripheral eosinophilia. We also consider aggressive systemic mastocytosis with local tissue invasion as category III disease.
• •Category IV disease with mast cell leukemia, characterized by the presence of malignant mast cells in the peripheral blood, which constitute greater than 10 percent of the nucleated cells.
• •No major organ dysfunction precluding transplantation.
• •DLCO greater than or equal to 65 percent predicted.
• •Left ventricular ejection fraction of greater than or equal to 40%.
• •ECOG performance status of 0 or 1.
• •Life expectancy of more than 3 months.
• •HLA-identical sibling available as donor.
• •Patients or their parent(s)/responsible guardian(s) must be able to comprehend the investigational nature of the study and be willing to sign an informed consent.
• •HLA identical family donor, (only HLA identical).
• •Weight greater than or equal to 18 kg.
• •Age less than or equal to 80 years old.
• •Informed consent given.