Advances In The Treatment Of Primary Myelofibrosis

Primary Myelofibrosis (PMF) is a rare and serious hematologicmalignancy classified as a Philadelphia chromosome-negativemyeloproliferative neoplasm (MPN). The disease is more common in malesand in older individuals. Of the MPNs, PMF presents with the most severemorbidity and greatest mortality. The disease is characterized bybeinmargsproliferasjon of megakaryocytes and granulocyttforstadiene,disturbed cytokine and reactive fibrosis involving bone marrow failure andextramedullary hematopoiesis.The clinical hallmarks of PMF areprogressive splenomegaly, anemia， and constitutional symptoms (e.g,fatigue, night sweats, fever). At present,treatment of myelofibrosis remainsa challenge.Conventional drugs for myelofibrosis are driven by clinicalneeds, primarily anemia and splenomegaly. With these therapies, stem celltransplantation remains the only potentially curative approach which arereserved only for a minority of patients. This year, RIC-allo-HSCTapplication broadens the indications for transplantation. It takes advantagefor monitoring of minimal residual disease and early detection ofrecurrence on molecular lever after transplantation.It also provides the benefit for DLI treatment timely. Meanwhile, the research of thepathogenesis of PMF has promoted the development and research ofmolecular targeted therapy drugs.The recent research found the disease hasthe relationship with mutation of JAK2,over50%PMF patient has beendetected the mutation of JAK2. In the WHO, The mutation of JAK2hasbeen considered as the main testing standards of PMF.Some new drugs oftargeted JAK2mutation and other broader mechanism of tumor has beenused for the treatment of PMF and has recieved the previews efficacy.Itprovides a available and effective treatment for patients who could not dothe Stem Cell transplant.Ruxilitinib, the most representative drug has beenapproved by PDA to be used as the medicine of PMF.