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Don’t fight Myelofibrosis alone.
Find your community on the free RareGuru App.Myelofibrosis is a disorder of the spongy tissue inside the bone (bone marrow) that contains the stem cells that will form blood cells. In myelofibrosis, the bone marrow is replaced by fibrous (scar) tissue. 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. The disorder occurs when blood stem cells develop somatic mutations in the JAK2, MPL, CALR, _and TET2 _genes. Other genes may also be involved. The disorder is generally not inherited because this type of mutation does not affect the reproductive cells (sperm and egg) only certain cells of the body (somatic). Although myelofibrosis can occur at any age, it typically develops after the age of 50 years. 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. Bone marrow or stem cell transplant may improve symptoms, and may cure the disease.
Source: GARD Last updated on 07-13-20
Myelofibrosis is a disorder of the spongy tissue inside the bone (bone marrow) that contains the stem cells that will form blood cells. In myelofibrosis, the bone marrow is replaced by fibrous (scar) tissue. 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. The disorder occurs when blood stem cells develop somatic mutations in the JAK2, MPL, CALR, _and TET2 _genes. Other genes may also be involved. The disorder is generally not inherited because this type of mutation does not affect the reproductive cells (sperm and egg) only certain cells of the body (somatic). Although myelofibrosis can occur at any age, it typically develops after the age of 50 years. 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. Bone marrow or stem cell transplant may improve symptoms, and may cure the disease.
Last updated on 05-01-20
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 on 05-01-20
Few epidemiologic studies are available to estimate the occurrence of myelofibrosis. 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.
Last updated on 05-01-20
The Myeloproliferative Disorders Research Consortium (MPD- RC) is an international, multi-institutional non- profit consortium funded by the National Cancer Institute (NCI) at the National Institutes of Health and set up to coordinate, facilitate, and perform basic and clinic research investigating the genetics of MPDs with the goal of developing therapy. To learn more, click on the link.
Last updated on 04-27-20
The Leukemia & Lymphoma Society has an article on idiopathic myelofibrosis. Click on Leukemia & Lymphoma Society to view the article.
Last updated on 04-27-20
Note, these links are external searches against the National Laboratory of Medicine's drug database. You may need to adjust the search if there are no results found.
| Drug Name | Generic Name |
|---|---|
| Inrebic | Fedratinib |
| Jakafi | ruxolitinib phosphate |
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