Rare diseases: Myelodysplastic/ Myeloproliferative diseases |
Understanding Myelodysplastic/myeloproliferative diseases
Myelodysplastic/myeloproliferative diseases are a category of rare diseases in which the patient's bone marrow does not properly fulfil its role as regards the production of blood cells. In a healthy individual, the bone marrow makes stem cells which then mature into red or white blood cells or platelets. The red blood cells carry oxygen in the blood throughout the body, while the white blood cells fight infection and the platelets make blood clot. The rare diseases known as Myelodysplastic/myeloproliferative diseases are clonal myeloid disorders which have both dysplastic and proliferative features1. In a Myelodysplastic disease, the stem cells do not become mature blood cells or platelets, instead remaining in the immature or blast phase. These blasts die prematurely resulting in a decrease in the number of healthy blood cells and platelets in the patient's blood. In myeloproliferative diseases, an unusually large number of stem cells mature into blood cells or platelets. This means that the total number of blood cells gradually increases.
Three major myeloid disorders constitute the category of rare diseases known as Myelodysplastic/myeloproliferative diseases:2
Chronic myelomonocytic leukaemia (CMML)
Atypical chronic myeloid leukaemia (aCML)
Juvenile myelomonocytic leukaemia (JMML)
These documents present research conducted to evaluate the use of imatinib to treat certain types of Myelodysplastic/Myeloproliferative Diseases.
Investigations of Targeted Therapy With Imatinib for Atypical Chronic Myeloproliferative Disorders*
Treatment Options for Atypical Chronic Myeloproliferative Disorders*
Dermatofibrosarcoma protuberans (DFSP) is an extremely rare disease which occurs slightly more frequently in men than in women. It is a variety of malignant tumour that occurs in the dermis, the skin's second layer. For more information concerning this rare disease and treatment options, including molecular targeted therapy, click the link below:
Dermatofibrosarcoma protuberans (DFSP)
References :
1. National Cancer Institute. Myelodysplastic/Myeloproliferative Diseases (PDQ®): Treatment. Health Professional Version. Available at: http://www.cancer.gov/cancertopics/pdq/treatment/mds-mpd/healthprofessional/allpages. Accessed April 24, 2006.
2. Vardiman JW, Harris NL, Brunning RD. The World Health Organization (WHO) classification of the myeloid neoplasms. Blood. 2002;100:2292-2302.
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