Pathogenesis and management of idiopathic myelofibrosis

Baillieres Clin Haematol. 1998 Dec;11(4):751-67. doi: 10.1016/s0950-3536(98)80037-1.

Abstract

Idiopathic myelofibrosis is the least common and carries the worst prognosis of the chronic myeloproliferative disorders. The primary disease process is a clonal haematopoietic stem cell disorder which results in a chronic myeloproliferation and an atypical megakaryocyte hyperplasia. In contrast, the characteristic stromal proliferation is a reactive phenomenon, resulting from the inappropriate release of megakaryocyte/platelet-derived growth factors, including PDGF, TGF-beta bFGF and calmodulin. The median survival is approximately 4 years, although individual survival varies greatly. A variety of prognostic schema have been developed which enable the identification of high-risk patients, for whom bone marrow transplantation should be considered. Management for the majority of patients, however, is directed towards the alleviation of symptoms and improvement in quality of life. This review summarizes the recent advances in our understanding of the disease's pathogenesis and discusses the limited therapeutic options available to clinicians.

Publication types

  • Review

MeSH terms

  • Anemia / etiology
  • Animals
  • Bone Marrow / pathology
  • Bone Marrow Transplantation
  • Cladribine / therapeutic use
  • Combined Modality Therapy
  • Disease Models, Animal
  • Erythropoietin / therapeutic use
  • Growth Substances / metabolism
  • Hematopoietic Stem Cells / pathology
  • Humans
  • Hydroxyurea / therapeutic use
  • Interferon-alpha / therapeutic use
  • Karyotyping
  • Megakaryocytes / metabolism
  • Megakaryocytes / pathology
  • Mice
  • Polyethylene Glycols / toxicity
  • Primary Myelofibrosis / drug therapy
  • Primary Myelofibrosis / etiology
  • Primary Myelofibrosis / pathology*
  • Primary Myelofibrosis / radiotherapy
  • Primary Myelofibrosis / therapy
  • Prognosis
  • Recombinant Proteins / toxicity
  • Splenectomy
  • Stromal Cells / pathology
  • Thrombopoietin / toxicity
  • Vitamin D / therapeutic use

Substances

  • Growth Substances
  • Interferon-alpha
  • Recombinant Proteins
  • polyethylene glycol-recombinant human megakaryocyte growth and development factor
  • Erythropoietin
  • Vitamin D
  • Polyethylene Glycols
  • Cladribine
  • Thrombopoietin
  • Hydroxyurea