M YELOFIBROSIS is a disease in which the normal blood-forming elements of the bone marrow are replaced by fibrous tissue with compensatory extramedullary hemopoiesis arising in other organs of the reticulo-endothelial system. Clinically, it is characterized by pains in the long bones, back or abdomen, progressive weakness, pallor and subsequent loss ofweight. The spleen, liver and sometimes the...

Myelofibrosis (MF) is a BCR-ABL1-negative myeloproliferative neoplasm characterized by clonal myeloproliferation, dysregulated kinase signaling, and release of abnormal cytokines. In recent years, important progress has been made in the knowledge of the molecular biology and the prognostic assessment of MF. Conventional treatment has limited impact on the patients' survival; it includes a wait-...

A 55-year-old woman with chronic refractory idiopathic thrombocytopenic purpura (ITP) presented with gum bleeding for 1 day. Over the course of several years, she had failed, or became resistant to, multiple treatments including glucocorticoids, intravenous immunoglobulin, splenectomy, rituximab, and, recently, a 9-month course of weekly romiplostim with moderate compliance. A complete blood co...

With the use of the International Working Group for Myelofibrosis Treatment and Research consensus criteria, we re-assessed the efficacy of thalidomide and lenalidomide in 125 patients with myelofibrosis treated in 3 consecutive phase 2 trials: 44 received single-agent thalidomide, 41 single-agent lenalidomide, and 40 a combination of lenalidomide plus prednisone. The thalidomide group included...

The review by Kremyanskaya and colleagues[1] regarding the utilization of ruxolitinib (Jakafi) in the therapy of myelofibrosis (MF) raises many important issues regarding how we assess drug efficacy in MF, the heterogeneous nature and impact of the disease, the published data regarding utilization of ruxolitinib, and practical advice for potential prescribers. At its core, the key question pose...

A case of primary myelofibrosis was identified with a previously unreported complex karyotype with two abnormal clones in addition to a proportion of normal cells: 46,XY, 2, 1 1, + der(2)t(2; 1) (q24/31;q13), + mar and 45,XY, 2, 1 1, + der(2)t(2;1 1)(q24/31;q13), + mar, 17,del(7q). Study of circulating committed progenitors from this patient consistently showed (1) an absence of erythroid proge...

Transforming growth factor-β1 (TGF-β1) is the most important cytokine involved in the promotion of myelofibrosis. Mechanisms leading to its local activation in the bone marrow environment remain unclear. As a recent study has highlighted the role of thrombospondin-1 (TSP-1) in platelet-derived TGF-β1 activation, we investigated the role of TSP-1 in the TPO(high) murine model of myelofibrosis. T...

References APPLEBY, A., BATSON, G.A., LASSMAN, L.P. & SIMPSON, C.A. (1964) Spinal cord compression by extramedullary haematopoiesis in myelosclerosis. J. Neurol. Neurosurg. Psychiat. 27, 313. ASK-UPMARK, E. (1945) Tumour-simulating intra-thoracic heterotopia of bone marrow, Acta radiol. (Stockh.), 26, 425. BRANNAN, D. (1927) Extramedullary hematopoiesis in anaemias. Bull. Johns Hopk. Hosp. 41 1...

Studies by the International Working Group showed that the prognosis of myelofibrosis patients is predicted by the Dynamic International Prognostic Scoring System (DIPSS) risk categorization, which includes patient age, constitutional symptoms, hemoglobin, leukocyte count, and circulating blasts. We evaluated the prognostic usefulness of the DIPSS in 170 patients with myelofibrosis, 12 to 78 ye...