[MOL] Subject: Drug for Iron Overload Passes Major Safety Hurdle.... [00795] Medicine On Line


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[MOL] Subject: Drug for Iron Overload Passes Major Safety Hurdle....



Good Morning All and our angel Lil,

Was thinking about Lils Grandbaby and saw this message about Iron
Overload and wondered if Lil posted this and knew about this. She is so
up on things and probably this is something that she knew, but maybe
didnt. Hope this info is beneficial.

Subject: Drug for Iron Overload Passes Major Safety Hurdle....

Drug for Iron Overload Passes Major Safety Hurdle, May Benefit Patients
With
Thalassemia and Other Blood Disorders, According to Researchers At The
Children's Hospital of Philadelphia

PHILADELPHIA, Feb. 22 /PRNewswire/ -- An oral medication that may be
helpful
for patients with thalassemia and related blood disorders has fewer
adverse
effects on white blood cells than previously predicted.  The drug,
deferiprone, may help patients with thalassemia who do not benefit from
the
conventional treatment for iron overload, a life-threatening
complication of
their disease.  A research team headed by a physician from The
Children's
Hospital of Philadelphia announced the results of their multicenter
study
today in the British Journal of Haematology.

Thalassemia, an inherited disease of the blood in which hemoglobin fails
to
form properly, may be challenging to treat.  Depending on its severity,
the
disease may require regular blood transfusions starting in early
childhood
and may cause an enlarged spleen, bone weakness and growth problems. 
The
blood transfusions lead to the most serious complication,
life-threatening
multiple organ damage caused by accumulation of excessive iron. 
Therefore,
patients with iron overload receive frequent treatments with
desferrioxamine,
which removes iron from the tissues.  However, this drug is difficult to
administer, because it must be pumped into the skin or bloodstream for
an
8-to-12-hour period every night.  Many patients do not comply with the
arduous regimen, and others are unable to use it because of adverse
reactions.

Therefore, for the past 20 years, researchers have been seeking an
effective
oral medicine for iron overload.  The leading candidate has been
deferiprone,
but previous studies showed the medicine had a serious side effect
called
agranulocytosis, a sharp reduction in white blood cells.  The current
study
measured the frequency of that side effect in 187 pediatric and adult
patients at four sites, one at The Children's Hospital of Philadelphia
and
the other three at thalassemia centers in Italy.  The patients were
unable or
unwilling to use desferrioxamine.

Patients in the study received weekly blood tests to monitor
deferiprone's
effects.  Agranulocytosis occurred in only one patient, while nine
patients
had a milder reduction in white blood cells (neutropenia).  "The study
showed
that toxicity to white blood cells is a rare side effect of deferiprone,
and
is not frequent enough to disqualify the drug as a potential treatment
for
iron overload," said Alan R. Cohen, M.D., chief of Hematology at The
Children's Hospital of Philadelphia and lead author of the study. 
"Although
much research remains to be done, deferiprone might also be used to
treat
patients with other hemoglobin disorders, such as sickle cell disease,
in
which regular blood transfusions are often used."

Dr. Cohen added that since the study began, other possible side effects
of
deferiprone have been suggested, such as liver toxicity, and that
further
studies need to address such concerns.  Like desferrioxamine,
deferiprone is
an iron chelator -- a chemical that binds to excess iron and removes it
from
the body.  "We are not proposing that deferiprone should replace
desferrioxamine as a first-line therapy for iron overload," he said. 
"But it
may become an option for patients who do not respond to desferrioxamine
or
who have severe adverse reactions to that conventional therapy."

The Children's Hospital of Philadelphia, the nation's first children's
hospital, is a leader in patient care, education and research.  This
373-bed
multispecialty hospital provides comprehensive pediatric services,
including
home care, to children from before birth through age 19.  The hospital
is
second in the United States among all children's hospitals in total
research
funding from the National Institutes of Health.

CONTACT:  Maria Stearns of The Children's Hospital of Philadelphia,
215-590-4091, or Stearnsm@email.chop.edu

 SOURCE  The Children's Hospital of Philadelphia

------------------------------
God Bless All,
marty auslander
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