Myelodysplastic Syndrome and Myelofibrosis
Myelodysplastic Syndrome and Myelofibrosis are chronic malignant hematologic diseases that stem from abnormal blood cell formation. The Myelodysplastic Syndrome (MDS) is a term for a group of frequent malignant stem cell diseases, mainly encountered in older individuals. People with MDS have low numbers of red blood cells (anemia), and the cells may have a mutation in their DNA. Overall, MDS is relatively uncommon, with an incidence of between four to five people per a population of 100. However, in patients over the age of 60, this increases to around 20 to 50 incidences per one hundred.
The World Health Organization divides myelodysplastic syndromes into subtypes based on the kind of blood cells — red cells, white cells and platelets — involved. In about 1 in 3 patients, MDS can progress to a quickly growing cancer of bone marrow cells called acute myeloid leukemia.
Myelofibrosis (MF) is rare disorder in which abnormal blood cells and fibers build up in the bone marrow. Myelofibrosis is part of a group of diseases called myeloproliferative disorders/neoplasms (MPN), and are characterized for abnormal cells which sometimes harbor mutations in the JAK pathway. The symptoms associated with primary myelofibrosis differ and are associated to a build of abnormal blood cells and fibers in the bone marrow. Affected people may stay symptom-free for many years, and then develop the florid disease. Myelofibrosis can affect anyone; however, it is most frequently diagnosed in people older than 50.
Myelodysplastic Syndrome and Myelofibrosis Symptoms
Symptoms of myelofibrosis can include fatigue, shortness of breath, belly discomfort, pain beneath the ribs, feeling full, muscle and bone pain, itching, and night sweats. Most patients with myelofibrosis have an enlarged spleen, and in some cases, an enlarged liver.
In primary myelofibrosis there are often low ranges of circulating red blood cells, a situation known as anemia. White blood cells and platelets are additionally misshapen and immature. Symptoms of anemia include fatigue, pallor, and shortness of breath.
Like Myelofibrosis, the symptoms for Myelodysplastic syndrome include fatigue, shortness of breath, skin pallor due to anemia and skin bruising due to low platelets.
Myelodysplastic Syndrome and Myelofibrosis Treatments
Myelodysplastic syndrome and Myelofibrosis symptoms and low blood counts can be treated with blood transfusions, where red blood cells or platelets are replaced. Active treatment with drugs called hypomethylating agents are used in MDS. Studies have shown that lenalidomide is extremely efficient in treating peripheral blood and bone marrow abnormalities in certain people with MDS (a condition called MDS with 5q minus). Another treatment for Myelodysplastic syndrome and Myelofibrosis is a bone marrow transplant.
The use of JAK inhibitors, leading to regulatory approval of ruxolitinib, represented a major therapeutic advance in myelofibrosis (MF). Some individuals with major myelofibrosis have been treated with allogeneic stem cell transplantation. In allogeneic stem cell transplantation, stem cells are donated from another particular person, often from a closely matched family member. There are current studies analyzing the best treatments after MF progresses on Ruxolitinib. Massive Bio uses artificial intelligence and patient support to match to novel treatments and clinical trials, including for Myelodysplastic Syndrome and Myelofibrosis Treatments.