Transplant strategies for idiopathic myelofibrosis

Abstract

Idiopathic myelofibrosis (IMF) is a clonal stem cell disorder and is one of the four major myeloproliferative disorders, which include essential thrombocythemia (ET), polycythemia vera (PV), and chronic myelogenous leukemia (CML). Patients may be asymptomatic at the early stages, but later progress to marrow fibrosis, splenomegaly with pancytopenia leading to anemia, and other constitutional symptoms. Most of the care available is supportive and only palliates the constitutional symptoms. Prognosis for these patients is dependent on karyotype, hemoglobin count, and age. Stem cell transplantation is the only curative therapy, which results in eradication of the stem cell clone, with the cessation of extramedullary hematopoiesis and resolution of marrow fibrosis and its sequelae. Stem cell replacement therapy using either autologous or allogeneic stem cells has been attempted in small populations of patients with variable benefit. A nonmyeloablative approach has shown promise in a very small number of patients, but additional investigation is required for the ideal management of these patients.