A Bone Marrow Disease With a Brighter Prognosis
New drugs, transplantation helping people with aplastic anemia live
longer, fuller lives
By Dennis Thompson
Posted 3/23/08
SUNDAY, March 23 (HealthDay News) -- Blood is life. And the rare
disease known as aplastic anemia robs the body of life by robbing the
body of blood.

The aplastic anemia patient's blood thins as the bone marrow slows its
production of blood cells. The results can range from chronic fatigue
to heart disease or from endless infections to cuts that won't clot,
depending on the type of blood cells that are lacking.

But there's hope: Considered fatal as recently as two decades ago,
aplastic anemia is becoming a far more manageable disease. Advances in
drug therapies and improvements in the field of transplantation have
slashed the death toll, allowing patients to live longer, fuller
lives.

"We are getting better at treating aplastic anemia, either in getting
rid of it or treating its symptoms," said Dr. Jaroslaw P. Maciejewski,
with the Cleveland Clinic's Department of Hematologic Oncology and
Blood Disorders.

And those advances are helping doctors gain greater insights into
other, more prevalent, health conditions, such as heart disease and
leukemia.

An estimated 50,000 people develop aplastic anemia in the United
States each year, according to the U.S. National Institutes of Health.
(A related blood disorder, myelodysplastic syndrome, or MDS, occurs
when the bone marrow begins to produce poorly functioning or immature
blood cells. About 20,000 to 30,000 new cases of MDS occur each year.)

It's important to note that many symptoms of aplastic anemia, such as
fatigue and infection, can also be caused by other diseases, said Dr.
Ronald Paquette, a blood disease researcher with the University of
California, Los Angeles' Jonsson Comprehensive Cancer Center.

"If everyone who was fatigued thought they had aplastic anemia, we'd
be swamped," Paquette said.

Bone marrow -- the spongy material inside bones -- produces stem cells
that normally develop into the three main types of blood cells -- red
blood cells, white blood cells, and platelets.

"Essentially, the bone marrow is a factory of blood," Maciejewski
said.

In patients with aplastic anemia, the stem cells have been damaged,
slowing or stopping the production of all blood cells.

The cause of the damage to stem cells remains unknown in more than
half of people with aplastic anemia. Some research has suggested that
stem cell damage occurs when the immune system attacks the body's own
cells by mistake, according to the National Institutes of Health.

Aplastic anemia has also been linked to exposure to toxins such as
pesticides, arsenic and benzene. Some infectious diseases also can
cause the disorder, including hepatitis, Epstein-Barr virus,
cytomegalovirus, parvovirus B19, and HIV, as well as autoimmune
diseases like lupus and rheumatoid arthritis. Finally, some genetic
disorders have been linked to it.

Symptoms vary depending on the type of blood cells in shortage:

Too few red blood cells can mean not enough oxygen is carried to the
body, according to the NIH. People who have a low red blood cell count
often feel tired. Because the heart has to work harder to pump blood
to get enough oxygen to the body's organs and tissues, heart disease
can develop over time.
Too few white blood cells weaken the body's defense against infection.
The patient may become ill more often, and the illness can be severe.
Too few platelets hamper the blood's ability to clot. Patients with a
low platelet count may bruise or bleed easily, and their bleeding may
be hard to stop.
Once aplastic anemia is detected, swift treatment is essential,
Paquette said. "Because it's a rare disease, it's important to be
treated at a specialized center," he said. "The most important thing
is to be seen by someone with a lot of experience treating the disease
early on."

For patients younger than 30, stem cell transplantation is often the
preferred treatment. For those with a matched sibling donor, stem cell
transplantation replaces the defective bone marrow with healthy cells,
and as many as 80 percent of patients enjoy a complete recovery,
according to the Aplastic Anemia & MDS International Foundation Inc.

Advances in stem cell research and anti-rejection drugs have meant
that transplantations from unrelated donors also are becoming more
successful, Paquette said.

One promising avenue of treatment involves transplantation using stem
cells harvested from the umbilical cord of new mothers. "The cells can
be cryopreserved [frozen] and saved, then given to unrelated donors,"
Paquette said. "It's quite encouraging."

For these patients, again, speed is of the essence. "The data show the
earlier you do a transplant, the better the outcome," Paquette said.

Patients whose transplants fail, or for whom transplantation is not an
option, often receive successful immunosuppressive therapy with agents
like anti-thymocyte globulin and cyclosporine. Response rates
typically range from 70 percent to 80 percent, according to the
Aplastic Anemia & MDS International Foundation Inc.

Blood transfusions from matched donors also are used to keep blood
counts high and help relieve symptoms, although they are not an
effective long-term treatment.

"Whether we cure the disease or not, patients are getting better
across the board," Maciejewski said. "We now can maintain life, keep
these patients alive longer."

More information

To learn more, visit the Aplastic Anemia & MDS International
Foundation Inc.

Copyright © 2008 ScoutNews, LLC. All rights reserved.

"Complete recovery after iron chelation in aplastic anemia"

Park SJ, Han CW
Complete hematopoietic recovery after continuous iron chelation
therapy in a patient with severe aplastic anemia with secondary
hemochromatosis. [Journal Article]
J Korean Med Sci 2008 Apr; 23(2):320-3.

A 16-yr-old male patient with hemochromatosis due to multiple packed
red blood cell transfusions was referred to our emergency center for
the treatment of severe aplastic anemia and dyspnea. He was diagnosed
with aplastic anemia at 11-yr of age. He had received continuous
transfusions because an HLA-matched marrow donor was unavailable.
Following a continuous, approximately 5-yr transfusion, he was noted
to develop hemochromatosis. He had a dilated cardiomyopathy and
required diuretics and digitalis, multiple endocrine and liver
dysfunction, generalized bleeding, and skin pigmentation. A total
volume of red blood cell transfusion before deferoxamine therapy was
about 96,000 mL. He received a regular iron chelation therapy
(continuous intravenous infusion of deferoxamine, 50 mg/kg/day for 5
days q 3-4 weeks) for approximately seven years after the onset of
multiple organ failures. His cytopenia and organ dysfunctions began
to
be gradually recovered since about 2002, following a 4-yr
deferoxamine
treatment. He showed completely normal ranges of peripheral blood
cell
counts, heart size, and liver function two years ago. He has not
received any transfusions for the last four years. This finding
suggests that a continuous deferoxamine infusion may play a role in
the immune regulation in addition to iron chelation effect.
Journal of Korean medical science [J Korean Med Sci]
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