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Diseases

Genetic and Rare Diseases Information Center (GARD)

Myelofibrosis


Other Names for this Disease
  • Idiopathic myelofibrosis
  • Myeloid metaplasia
  • Agnogenic myeloid metaplasia
  • Primary myelofibrosis
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.

Your Question

I need to ascertain the incidence of myelofibrosis in the United States. Is myelofibrosis considered to be a rare disorder?

Our Answer

We have identified the following information that we hope you find helpful. If you still have questions, please contact us.

What is myelofibrosis?

Myelofibrosis is a disorder of the bone marrow, in which the marrow is replaced by fibrous (scar) tissue.[1] When the bone marrow is scarred, it cannot make enough blood cells. This leads to anemia, weakness, fatigue, and often, swelling of the liver and spleen.[1][2] The condition occurs when blood stem cells develop somatic mutations in the JAK2, MPL, CALR, and TET2 genes. Other genes may also be involved. The condition is generally not inherited.[3] Although myelofibrosis can occur at any age, it typically develops after the age of 50.[1][2] In most cases, myelofibrosis gets progressively worse. Treatment is aimed at relieving signs and symptoms and may include medications, blood transfusions, chemotherapy, radiation therapy and surgery.[2]   
Last updated: 6/22/2016

How rare is myelofibrosis?

Few epidemiologic studies are available to estimate the occurrence of myelofibrosis.[4] Through the review of several medical textbooks and journal articles, it appears as if the annual incidence (occurrence) in European, Australian, and North American populations ranges from 0.3 to 1.5 cases per 100,000 persons.[4][5][6][7][8]
Last updated: 6/22/2016

Is myelofibrosis a rare disorder?

According to the definition of a rare disorder per the Orphan Drug act, myelofibrosis is considered a rare disorder given the incidence discussed above. 
Last updated: 6/22/2016

References
Other Names for this Disease
  • Idiopathic myelofibrosis
  • Myeloid metaplasia
  • Agnogenic myeloid metaplasia
  • Primary myelofibrosis
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.