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, to watch for signs of disease progression.

Treatments

Only bone marrow transplantation has been shown to cure myelofibrosis. But other treatments can improve symptoms. 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 current medications to treat myelofibrosis. Many medications have been studied in clinical trials 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 evaluating possible new therapies for myelofibrosis. Clinical trials are underway that address 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 (myeloablative transplant) has cured 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, do not qualify for this treatment.

Clinical Trials

Mayo Clinic is studying another potentially curative transplant treatment called non-myeloablative transplant ("mini-transplant"). Patients may be able to participate in clinical trials studying this treatment. Low doses of chemotherapy are administered, 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 than but as effective as myeloablative transplant. (See information on the bone marrow transplant pages.)