Modern management of myelofibrosis

Abstract

SummaryThe conventional treatment of myelofibrosis involves a wait‐and‐see approach for asymptomatic patients, oral chemotherapy for the hyperproliferative forms of the disease, androgens or erythropoietin for the anaemia, and splenectomy in selected patients. Low‐dose thalidomide plus prednisone is a well‐tolerated therapy for the anaemia and the thrombocytopenia of myelofibrosis, whereas imatinib has shown little efficacy. Allogeneic stem cell transplantation (allo‐SCT) is the only curative therapy for myelofibrosis. Its standard modality has an associated mortality of about 30% and can be applied to younger patients with high‐risk disease or resistant to conventional treatment. Reduced‐intensity conditioning allo‐SCT involves a low mortality and is a promising therapy for patients aged 45–70 years old with the above characteristics. Autologous SCT is a palliative therapy for patients resistant to conventional treatment who lack a suitable donor. The next candidates for the treatment of myelofibrosis are the thalidomide derivatives, the proteasome inhibitors, and vascular endothelial growth factor neutralizing antibodies.

Efficacy and tolerability of hydroxyurea in the treatment of the hyperproliferative manifestations of myelofibrosis: results in 40 patients

“…Except for allo-HSCT [7,8], the therapy of MF remains essentially palliative and is usually adjusted to the characteristics of the disease in every individual [6]. Thus, a wait-and-see approach is often adopted in asymptomatic patients, delaying treatment start until a change in the clinical situation is observed.…”

“…From the clinical point of view, MF is a heterogeneous disorder, with its spectrum ranging from patients who are asymptomatic at diagnosis and may not require treatment for years to others with symptoms mainly derived from anemia and splenomegaly and constitutional symptoms [6]. Except for allogeneic hemopoietic stem cell transplantation (allo-HSCT) [7,8], therapy of MF remains mostly palliative and is usually adjusted to the disease characteristics in each patient [6].…”

“…Except for allogeneic hemopoietic stem cell transplantation (allo-HSCT) [7,8], therapy of MF remains mostly palliative and is usually adjusted to the disease characteristics in each patient [6]. In the "hyperproliferative" forms of the disease, characterized by constitutional symptoms, marked splenomegaly, leukocytosis and/or thrombocytosis, and, more rarely, aquagenic pruritus or bone pain, cytolytic treatment is usually administered [6]. Although hydroxyurea (HU) is the drug most frequently used in such cases, information on its efficacy is limited to a few reports including a scarce number of patients [9,10].…”

Respective clustering of unfavorable and favorable cytogenetic clones in myelofibrosis with myeloid metaplasia with homozygosity for JAK2V617F and response to erythropoietin therapy

“…In general, current drug therapy for MMM remains suboptimal, and the response to specific agents is unpredictable. 15 Of the currently available drugs, those with demonstrated efficacy for alleviating anemia include Epo, 16 corticosteroids, 17 androgens, 18 danazol, 19 and thalidomide alone 20 or in combination with prednisone. 21 Response rates with these drugs generally have been meager, and accurate prediction of response has not been possible in most instances.…”

The Clinical Course and Prognosis of Patients With Essential Thrombocythemia Treated With Hydroxyurea and Low-Dose Aspirin in an 11-Year Follow-Up

“…Reactive thrombocytosis may be responsible for more than 85% of cases of thrombocytosis seen in routine clinical practice. The main clinical features of ET are splenomegaly, myelofibrosis with varying degrees in the bone marrow, developing into acute leukemia, and increased risk of thrombotic and bleeding events [4][5][6][7]. The risk of venous and arterial thrombosis in patients with ET is thought to be high.…”