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.
Read more: UI Health validates cure for sickle cell in adults
The Latest on: Sickle cell disease
[google_news title=”” keyword=”Sickle cell disease” num_posts=”10″ blurb_length=”0″ show_thumb=”left”]
via Google News
The Latest on: Sickle cell disease
- May 18 brunch will offer sickle cell disease support in Albany areaon May 8, 2024 at 2:02 pm
The MTS Sickle Cell Foundation has partnered with the Georgia Public Library Service and the Dougherty County Library System to help raise awareness about sickle cell disease in the community. The ...
- New Orleans child receives gift of life, cured from sickle cell diseaseon May 8, 2024 at 4:26 am
According to NMDP, ethnically diverse patients with blood cancers and disorders historically have had less than a 50 percent chance of finding a transplant match.
- Black 12-Year-Old Patient Begins New Sickle Cell Therapy Treatmenton May 7, 2024 at 8:39 am
A 12-year-old Black patient is the first to undergo a new gene therapy treatment for sickle cell disease, according to reports.
- What is sickle cell disease, the illness that led to a 16-year-old's death?on May 6, 2024 at 6:46 pm
After a 16-year-old Dauphin County boy died suddenly, the coroner determined his cause of death was complications from sickle cell disease. It has some people wondering: What is this illness? Sickle ...
- A 12-Year-Old Is First to Start New Sickle Cell Treatmenton May 6, 2024 at 1:25 pm
He's the first patient outside of a clinical trial to start a new genetic treatment that was shown in clinical trials to cure 88% of people of the hallmark symptoms of sickle cell disease.
- Bluebird, Vertex prep for first commercial use of sickle cell gene therapieson May 6, 2024 at 9:33 am
A 12-year-old boy in the Washington, D.C., area is set become the first patient treated with Lyfgenia since its U.S. approval last December.
- First Patient Begins Newly Approved Sickle Cell Gene Therapyon May 6, 2024 at 4:01 am
A 12-year-old boy in the Washington, D.C., area faces months of procedures to remedy his disease. “I want to be cured,” he said.
- ‘Life-changing’ drug for sickle cell disease to be offered by NHS in Englandon May 2, 2024 at 5:29 pm
Voxelotor, which can help reduce need for blood transfusions, is administered as a daily tablet ...
- 'Life-changing' sickle cell disease treatment made available on NHSon May 2, 2024 at 4:43 pm
A "life-changing" new treatment for people living with sickle cell disease is to be made available on the NHS. Around 4,000 people living with the potentially fatal blood disorder could see their ...
- ‘Life-changing’ sickle cell drug approved for NHS roll-out after patient-led campaignon May 2, 2024 at 4:11 pm
Despite being the fastest-growing genetic condition in the UK, sickle cell disorder has historically been underserved by treatment options.
via Bing News