pediatric patient / parent

PNH (paroxysmal nocturnal hemoglobinuria hemoglobinuria: (hee-muh-gloe-buh-NYOOR-ee-uh) The presence of hemoglobin in the urine. ) is a very rare blood disease that causes red blood cells to break apart. PNH occurs because of a genetic change to some of your blood-forming stem cells stem cells: Cells in the body that develop into other cells. There are two main sources of stem cells. Embryonic stem cells come from human embryos and are used in medical research. Adult stem cells in the body repair and maintain the organ or tissue in which they are found. Blood-forming (hemapoietic) stem… . These defective stem cells become PNH blood cells that are missing an important coating (protein) that protects them from your immune system. If you have PNH, there will be times when your immune system destroys many red blood cells very quickly, causing low red blood cell red blood cell: The most numerous type of blood cell in healthy people. Red blood cells contain hemoglobin, a protein that picks up oxygen in the lungs and brings it to cells in all parts of the body. Also called erythrocyte, RBC. counts, as well as blood clots, muscle spasms and dark urine. This destruction of blood cells is called hemolysis hemolysis: (hi-MOL-uh-suss) The destruction of red blood cells. . 

The exact number of people who have MDS is unknown, but about 10,300 new cases were reported in the U.S. in 2003. This number is the result of data collected by the Surveillance, Epidemiology Epidemiology: The study of patterns and causes of disease in groups of people. Epidemiology researchers study how many people have a disease, how many new cases are diagnosed each year, where patients are located, and environmental or other factors that influence disease. , and End Results (SEER) Program of the Centers for Disease Control (CDC) and the National Cancer Institute (NCI). This program only started collecting data in 2001. The number of people may be higher than reported because some doctors are still don’t know that they need to report MDS cases to a national registry. Some doctors believe there are as many as 12,000 to 15,000 new MDS cases a year.

People diagnosed with MDS are:

• More often men than women
• Most often age 60 or older; 72 is the average age of diagnosis
• Most often white (Caucasian)

Native Americans, African-Americans, Inuits, Asians, and Pacific Islanders are less likely to get MDS.

MDS (myelodysplastic syndromes) is a group of disorders in which your blood-forming stem cells stem cells: Cells in the body that develop into other cells. There are two main sources of stem cells. Embryonic stem cells come from human embryos and are used in medical research. Adult stem cells in the body repair and maintain the organ or tissue in which they are found. Blood-forming (hemapoietic) stem… are defective and fail to make healthy, mature blood cells. Doctors consider MDS to be a slow growing form of blood and bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. cancer. With MDS, patients have low blood counts for at least one blood cell type and blood cells look abnormal under a microscope. Doctors must do a bone marrow biopsy bone marrow biopsy: A medical procedure to remove a small piece of solid bone marrow using a needle that goes into the marrow of the hip bone. The solid bone marrow is examined for cell abnormalities, the number of different cells and checked for scarring of the bone marrow. to be certain of the diagnosis. Symptoms and the course of MDS vary for each patient depending on which blood cells are affected.

Between 600 and 900 people are diagnosed with aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… each year in the U.S. It can strike people of any age, race or gender, but it’s more common among children, teenagers and older adults. It is also more likely to occur in people of Asian heritage.

Aplastic anemia Aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… happens when your bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. stops making enough blood-forming stem cells stem cells: Cells in the body that develop into other cells. There are two main sources of stem cells. Embryonic stem cells come from human embryos and are used in medical research. Adult stem cells in the body repair and maintain the organ or tissue in which they are found. Blood-forming (hemapoietic) stem… . Most experts believe aplastic anemia occurs because your immune system attacks and kills your stem cells in your bone marrow. This causes you to have low blood counts for all three types of blood cells.

Low blood counts result in symptoms such as fatigue, tiredness, bleeding, bruising and a higher risk of infection. The disease can be classified as moderate (MAA), severe (SAA) or very severe (VAA) depending on how low your blood counts are.