Is there a
cure for sickle cell disease?A small number of children with
severe sickle cell disease have been cured through a blood stem cell transplant.
The stem cells (immature cells that develop into blood cells) come from bone
marrow, or less frequently from umbilical cord blood, usually donated by
siblings who are a good genetic match. Most children with sickle cell disease,
however, do not have siblings who are good genetic matches. For this reason,
researchers have recently begun performing stem cell transplants using umbilical
cord blood from unrelated donors with apparent success.7
About 150 children
worldwide with sickle cell disease have had blood stem cell transplants, and
about 85 percent of them appear to be cured of the disease.8 However, this
approach carries a high risk: about 5 percent of children who underwent bone
marrow transplants died.8 The transplant did not cure the disease in another 10
percent.8 Gene therapy may someday offer a cure with less risk.
Currently, researchers
are studying a number of new drug treatments, in addition to hydroxyurea, for
reducing complications of the disease. Like hydroxyurea, decitabine and butyrate
(a food additive) appear to cause the body to make a form of hemoglobin (fetal
hemoglobin) that the baby makes before birth.7,9 Increased levels of fetal
hemoglobin appear to help prevent red blood cells from sickling. Recent studies
also suggest that inhaling the gas nitric oxide or taking an oral dietary
supplement called L-arginine (which appears to boost the body's production of
nitric oxide) may also help prevent sickling, as may treatment with an
antifungal drug called clotimazole or some blood-thinning drugs.7,9 There has
already been a great deal of progress in medical care that reduces serious
complications and improves survival in individuals with sickle cell disease.
These and other new treatments may further improve the quality of life in
affected individuals.
cure for sickle cell disease?A small number of children with
severe sickle cell disease have been cured through a blood stem cell transplant.
The stem cells (immature cells that develop into blood cells) come from bone
marrow, or less frequently from umbilical cord blood, usually donated by
siblings who are a good genetic match. Most children with sickle cell disease,
however, do not have siblings who are good genetic matches. For this reason,
researchers have recently begun performing stem cell transplants using umbilical
cord blood from unrelated donors with apparent success.7
About 150 children
worldwide with sickle cell disease have had blood stem cell transplants, and
about 85 percent of them appear to be cured of the disease.8 However, this
approach carries a high risk: about 5 percent of children who underwent bone
marrow transplants died.8 The transplant did not cure the disease in another 10
percent.8 Gene therapy may someday offer a cure with less risk.
Currently, researchers
are studying a number of new drug treatments, in addition to hydroxyurea, for
reducing complications of the disease. Like hydroxyurea, decitabine and butyrate
(a food additive) appear to cause the body to make a form of hemoglobin (fetal
hemoglobin) that the baby makes before birth.7,9 Increased levels of fetal
hemoglobin appear to help prevent red blood cells from sickling. Recent studies
also suggest that inhaling the gas nitric oxide or taking an oral dietary
supplement called L-arginine (which appears to boost the body's production of
nitric oxide) may also help prevent sickling, as may treatment with an
antifungal drug called clotimazole or some blood-thinning drugs.7,9 There has
already been a great deal of progress in medical care that reduces serious
complications and improves survival in individuals with sickle cell disease.
These and other new treatments may further improve the quality of life in
affected individuals.
Warm autoimmune hemolytic anemia is an extremely rare form of anemia affecting only one in eighty thousand individuals. This type of anemia is caused when the red blood cells are destroyed by warm antibodies which are activated by warm temperatures such as the body temperature. Warm autoimmune hemolytic anemia can occur in individuals of all ages but typically occurs in people after the age of forty and is seen more commonly in women than in men.
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