Myelodysplastic/myeloproliferative diseases (MDS/MPD) are diseases of the bone marrow. They share characteristics of myeloproliferative disorders and myelodysplastic syndromes.

Myeloproliferative disorders are diseases in which too many of certain types of blood cells are made in the bone marrow. The bone marrow, the tissue inside the large bones in the body, makes red blood cells (which carry oxygen to all the tissues in the body), white blood cells (which fight infection), and platelets (which make the blood clot). Myelodysplastic syndromes, also called preleukemia or “smoldering” leukemia, are diseases in which the bone marrow does not function normally and not enough normal blood cells are made.

Myelofibrosis

Myelofibrosis is a disease of the bone marrow where collagen creates scar tissue inside the marrow cavity. It is caused by a disturbance of the immune system. You may also know this disorder as primary or idiopathic myelofibrosis or agnogenic myeloid metaplasia. These names describe a situation where myelofibrosis is the primary disorder. It is also possible to have myelofibrosis as a consequence of another disease such as polycythemia vera (PV) or essential thrombocythemia (ET).

Commonly Prescribed (On-Label) Drugs: None

Off-Label Prescription Drugs Breakthrough Options: Interferon Alfa-2b, Thalidomide

Polycythemia Vera

Polycythemia vera (PV) is a myeloproliferative disorder (MPD). In this type, there is uncontrolled production of mature red blood cells leading to an increase in red blood cells. If not controlled, this can lead to complications involving clotting or bleeding episodes. PV is fairly rare, occurring in approximately 1 in 100,000 and is considered an orphan disease (a disease that pharmaceutical companies do not dedicate much research time to).

Commonly Prescribed (On-Label) Drugs: Mechlorethamine

Off-Label Prescription Drugs Breakthrough Options: Anagrelide, Hydroxyurea, Interferon Alfa-2b

Thrombocythemia

Platelets (thrombocytes) are normally produced in the bone marrow by cells called megakaryocytes. In thrombocythemia, megakaryocytes increase in number and produce too many platelets. Thrombocythemia usually occurs in people older than 50 and more frequently in women. The cause of thrombocythemia is unknown.

Commonly Prescribed (On-Label) Drugs: Anagrelide

Off-Label Prescription Drugs Breakthrough Options: Hydroxyurea, Interferon Alfa-2b, Peginterferon Alfa-2b

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