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UI Health Validates Cure For Sickle Cell In Adults by chrisrock(m): 12:20pm On Jun 14, 2016 |
Physicians at the University of Illinois Hospital & Health Sciences
System have cured 12 adult patients of sickle cell disease using a
unique procedure for stem cell transplantation from healthy, tissue-
matched siblings.
The transplants were the first to be performed outside of the National
Institutes of Health campus in Maryland, where the procedure was
developed. Physicians there have treated 30 patients, with an 87
percent success rate. The results of the phase I/II clinical trial at UI
Health, in which 92 percent of treated patients were cured, are
published online in the journal Biology of Blood & Marrow
Transplantation.
The new technique eliminates the need for chemotherapy to prepare
the patient to receive the transplanted cells and offers the prospect of
cure for tens of thousands of adults suffering from sickle cell disease.
About 90 percent of the approximately 450 patients who have received
stem cell transplants for sickle cell disease have been children.
Chemotherapy has been considered too risky for adult patients, who
are often more weakened than children by the disease.
“Adults with sickle cell disease are now living on average until about
age 50 with blood transfusions and drugs to help with pain crises, but
their quality of life can be very low,” says Dr. Damiano Rondelli, chief of
hematology/oncology and director of the blood and marrow transplant
program at UI Health, and corresponding author on the paper.
“Now, with this chemotherapy-free transplant, we are curing adults
with sickle cell disease, and we see that their quality of life improves
vastly within just one month of the transplant,” said Rondelli, who is
also the Michael Reese Professor of Hematology in the UIC College of
Medicine. “They are able to go back to school, go back to work, and
can experience life without pain.”
Sickle cell disease is inherited. It primarily affects people of African
descent, including about one in every 500 African Americans born in
the U.S. The defect causes the oxygen-carrying red blood cells to be
crescent shaped, like a sickle. The misshapen cells deliver less oxygen
to the body’s tissues, causing severe pain and eventually
stroke or organ damage.
Doctors have known for some time that bone marrow transplantation
from a healthy donor can cure sickle cell disease. But few adults
were transplanted because high-dose chemotherapy was needed to kill
off the patients’ own blood-forming cells — and their entire immune
system, to prevent rejection of the transplanted cells, leaving
patients open to infection.
In the new procedure, patients receive immunosuppressive
drugs just before the transplant, along with a very low dose of total
body irradiation — a treatment much less harsh and with fewer
potentially serious side effects than chemotherapy.
Next, donor cells from a healthy and tissue-matched sibling
are transfused into the patient. Stem cells from the donor produce
healthy new blood cells in the patient, eventually in sufficient quantity
to eliminate symptoms. In many cases, sickle cells can no longer be
detected. Patients must continue to take immunosuppressant drugs
for at least a year.
In the reported trial, the researchers transplanted
13 patients, 17 to 40 years of age, with a stem cell
preparation from the blood of a tissue-matched
sibling. Healthy sibling donor-candidates and
patients were tested for human leukocyte antigen, a
set of markers found on cells in the body. Ten of
these HLA markers must match between the donor
and the recipient for the transplant to have the best
chance of evading rejection.
In a further advance of the NIH procedure,
physicians at UI Health successfully transplanted
two patients with cells from siblings who matched
for HLA but had a different blood type.
In all 13 patients, the transplanted cells successfully
took up residence in the marrow and produced
healthy red blood cells. One patient who failed to
follow the post-transplant therapy regimen reverted
to the original sickle cell condition.
None of the patients experienced graft-versus-host
disease, a condition where immune cells originating
from the donor attack the recipient’s body.
One year after transplantation, the 12 successfully transplanted
patients had normal hemoglobin concentrations in their blood and
better cardiopulmonary function. They reported less pain and improved
health and vitality.
Four of the patients were able to stop post-transplantation
immunotherapy without transplant rejection or other complications.
“Adults with sickle cell disease can be cured without chemotherapy –
the main barrier that has stood in the way for them for so long,”
Rondelli said. “Our data provide more support that this therapy is safe
and effective and prevents patients from living shortened lives,
condemned to pain and progressive complications.”
This research was supported in part by a Michael Reese Research &
Education Foundation Grant to Rondelli. |
Re: UI Health Validates Cure For Sickle Cell In Adults by easymax: 2:53pm On Jun 14, 2016 |
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