Treatment of mastocytosis depends on the symptoms and the classification of disease.
Indolent mastocytosis may require no treatment, though most patients experience symptoms that require regular doses of H1 and H2 antihistamines. If symptoms are not adequately relieved, additional medications, including mast cell stabilizing drugs such as cromolyn sodium and/or ketotifen fumarate, a leukotriene inhibitor (such as zafirlukast, montelukast sodium, or zileuton), a proton pump inhibitor (if needed to treat stomach hyperacidity) and where tolerated, a nonsteroidal anti-inflammatory drug. Bisphosphanates should be used if osteoporosis or significant osteopenia is present. Most specialist agree that symptomatic treatment by blocking the formation or action of mast cell mediators is appropriate when the disease is indolent, and that cytoreductive treatment should be reserved for other categories of disease.
Mastocytosis with an associated clonal, non-mast cell hematologic disorder should be treated as two separate disease entities: the mastocytosis should be treated appropriately for the disease, and the non-mast cell hematologic disorder should be treated appropriately for the type of disorder.
Aggressive mastocytosis, mast cell sarcoma, and mast cell leukemia should be treated to block the effects of mediator release as well with cytoreductive therapies. Several targeted therapeutic agents are in clinical trials and offer potentially more effective and specific treatments in the near future. Bone marrow transplant has not yet been effective for more than a short time, and rapid return and progression of the mastocytosis may occur.