Myelofibrosis with Myeloid Metaplasia

Treatment

Deciding how to treat myelofibrosis is a difficult decision for the patient and physician to make together. The severity and prognosis of myelofibrosis are quite variable. In some patients the disease remains stable for many years without treatment. Others will be very sick at the time of diagnosis.

Stable patients early in their disease may simply be observed closely, without treatment, watching for signs of progression of the disease.

Treatments

Only bone marrow transplantation has been shown to cure myelofibrosis. But other treatments can improve symptoms of the disease. Decreases in red and white blood cells and platelets can be treated with androgens, erythropoietin and thalidomide. An enlarged spleen can be helped in some cases with surgery, oral chemotherapy with hydroxyurea or low-dose radiation. These treatments have side effects and must be carefully managed so that the benefits outweigh the risks.

Mayo physicians have significant clinical expertise in using currently available medications to treat myelofibrosis. Many clinical trials of medications have been performed at Mayo, including Suramin, Pirfenidone, Interferon-Alpha, Etanercept, Thalidomide, Thalidomide plus Prednisone, Imatinib Mesylate (Gleevec), Cladribine and Zarnestra. Additionally, Mayo researchers (see Research on Myelofibrosis) are actively evaluating possible new therapies for myelofibrosis in the laboratory. Many clinical trials are currently under way for myelofibrosis and related diseases.

Bone Marrow Transplant

Aggressive chemotherapy followed by a transplant of normal blood or marrow stem cells from a matched family member or unrelated donor, known as myeloablative transplant, has been shown to cure some patients with this disease. But this treatment has a very high risk of life-threatening side effects. Most myelofibrosis patients, because of age, stability of the disease or other health problems, will not qualify for this treatment.

Clinical Trials

Mayo Clinic is studying another potentially curative transplant treatment called non-myeloablative transplant ("mini-transplant"). Patients can participate in this treatment through clinical trials. This approach involves administering low doses of chemotherapy, followed by an infusion of healthy peripheral blood stem cells from a matched (related or unrelated) donor. While this approach still has side effects, physicians hope that it will be safer but still as effective as myeloablative transplant. (See information on the bone marrow transplant pages.)