Immune thrombocytopenia (ITP) is a disorder that can lead to easy or excessive bruising and bleeding. The bleeding results from unusually low levels of platelets — the cells that help blood clot.
Formerly known as idiopathic thrombocytopenic purpura, ITP can cause purple bruises, as well as tiny reddish-purple dots that look like a rash.
Children may develop ITP after a viral infection and usually recover fully without treatment. In adults, the disorder is often long term.
If you don't have signs of bleeding and your platelet count isn't too low, you may not need any treatment. If your symptoms are more severe, treatment may include medications to boost your platelet count or surgery to remove your spleen.
Primary immunodeficiency disorders — also called primary immune disorders or primary immunodeficiency — weaken the immune system, allowing infections and other health problems to occur more easily.
Many people with primary immunodeficiency are born missing some of the body's immune defenses or with the immune system not working properly, which leaves them more susceptible to germs that can cause infections.
So far, researchers have identified more than 300 forms of primary immunodeficiency disorders. Some forms are so mild they can go unnoticed until adulthood. Other types are severe enough that they're discovered soon after an affected baby is born.
Treatments can boost the immune system in many types of primary immunodeficiency disorders. Research is ongoing, leading to improved treatments and enhanced quality of life for people with the condition.
Diamond-Blackfan anemia (DBA) is a very rare blood disorder that affects people’s bone marrow and disrupts red blood cell production. It’s a genetic disorder that happens when certain genes mutate or change. Diamond-Blackfan anemia is a chronic condition. People who have this condition will need life-long medical care and support.
Diamond-Blackfan anemia (DBA) is a very rare blood disorder that affects people’s bone marrow, preventing bone marrow from producing enough red blood cells. People rely on red blood cells to carry oxygen throughout their bodies.
This type of anemia is a genetic disorder that happens when certain genes mutate, or change. Those genetic changes determine what kind of symptoms people have and whether those symptoms are mild or severe. That said, nearly everyone with Diamond-Blackfan anemia has anemia symptoms. They may have other conditions that affect their overall health and quality of life. As people with Diamond-Blackfan anemia grow older, they have an increased risk of developing certain kinds of cancer.
Diamond-Blackfan anemia is a chronic condition. People who have this condition will need lifelong medical care so their healthcare providers can treat the inherited condition, manage treatment side effects and monitor complications.
Familial hemophagocytic lymphohistiocytosis is a disorder in which the immune system produces too many activated immune cells (lymphocytes) called T cells, natural killer cells, B cells, and macrophages (histiocytes). Excessive amounts of immune system proteins called cytokines are also produced.
Familial hemophagocytic lymphohistiocytosis also destroys blood-producing cells in the bone marrow, a process called hemophagocytosis. As a result, affected individuals have low numbers of red blood cells (anemia) and a reduction in the number of platelets, which are involved in clotting. A reduction in platelets may cause easy bruising and abnormal bleeding.
Hemophilia is a rare disorder in which the blood doesn't clot in the typical way because it doesn't have enough blood-clotting proteins (clotting factors). If you have hemophilia, you might bleed for a longer time after an injury than you would if your blood clotted properly.
Small cuts usually aren't much of a problem. If you have a severe form of the condition, the main concern is bleeding inside your body, especially in your knees, ankles and elbows. Internal bleeding can damage your organs and tissues and be life-threatening.
Hemophilia is almost always a genetic disorder. Treatment includes regular replacement of the specific clotting factor that is reduced. Newer therapies that don't contain clotting factors also are being used.
Juvenile myelomonocytic leukemia (JMML) is a rare cancer of the blood that affects young children. JMML happens when types of white blood cells called monocytes and myelocytes do not mature normally. JMML can happen spontaneously (by chance) or can be associated with other genetic disorders in some children.
Langerhans cell histiocytosis is a rare disorder that can damage tissue or cause lesions to form in one or more places in the body. Langerhans cell histiocytosis (LCH) is a rare disease that begins in LCH cells. LCH cells are a type of dendritic cell that normally helps the body fight infection.
Mixed-phenotype acute leukemia (MPAL) is a rare type of blood cancer. Typically, a doctor can classify the cancer as acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL), depending on the cells involved. However, MPAL presents with features of both AML and ALL.
Leukemia describes a cancer of the blood or bone marrow. There are many types of leukemia, and doctors typically classify them as either acute (sudden) or chronic (slow), depending on how quickly the cancer develops. They can then further classify these cancers depending on whether they affect myeloid or lymphocytic blood cells.
MPAL refers to a rare subtype of leukemia that displays no clear sign of origin and presents with features of both AML and ALL.
Myelodysplastic Syndrome are a group of disorders caused by blood cells that are poorly formed or don't work properly. Myelodysplastic Syndrome result from something amiss in the spongy material inside your bones where blood cells are made (bone marrow).
Management of Myelodysplastic Syndrome is most often intended to slow the disease, ease symptoms and prevent complications. Common measures include blood transfusions and medications to boost blood cell production. In certain situations, a bone marrow transplant, also known as a stem cell transplant, may be recommended to replace your bone marrow with healthy bone marrow from a donor.
Pancytopenia is a laboratory finding rather than a disease. It means a person has fewer platelets, red blood cells, and white blood cells than usual.
Bone marrow issues can lead to pancytopenia. Bone marrow is a spongy tissue within the bones that contains stem cells, immature cells that can turn into blood cells. A problem with stem cells can mean fewer blood cells enter the bloodstream.
Nutritional deficiencies, autoimmune diseases, certain viruses or toxins, and cancer treatments can also cause pancytopenia.
Thrombocytopenia is a condition in which you have a low blood platelet count. Platelets (thrombocytes) are colorless blood cells that help blood clot. Platelets stop bleeding by clumping and forming plugs in blood vessel injuries.
Thrombocytopenia might occur as a result of a bone marrow disorder such as leukemia or an immune system problem. Or it can be a side effect of taking certain medications. It affects both children and adults.
Thrombocytopenia can be mild and cause few signs or symptoms. In rare cases, the number of platelets can be so low that dangerous internal bleeding occurs. Treatment options are available.
Von Willebrand disease is a lifelong bleeding disorder in which your blood doesn't clot properly. People with the disease have low levels of von Willebrand factor, a protein that helps blood clot, or the protein doesn't perform as it should.
Most people with the disease are born with it, having inherited it from one or both parents. However, warning signs, such as heavy bleeding after a dental procedure, might not show up for years.
Von Willebrand disease can't be cured. But with treatment and self-care, most people with this disease can lead active lives.
Dr. Vipin Khandelwal is a highly acclaimed clinician in the field of Paediatric Oncology, Paediatric Hematology and Bone Marrow Transplant (BMT). With more than 10 years of experience, he has helped in accomplishment of more than 1000 bone marrow transplants, predominantly in paediatric patients suffering from various hematological disorders.
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