Stem cell transplants found to improve survival, quality of life for severe cases of scleroderma
January 8, 2018 | by Denise Heady
Most people are familiar with stem cell transplants as lifesaving procedures that are often used for those diagnosed with a blood cancer or blood disorder.
But now, a new study lead by Duke Health, has found that stem cell transplants are a safe and effective curative treatment option for patients with scleroderma, a serious autoimmune disorder that causes hardening of the skin and problems with internal organs like the lungs, heart and gastrointestinal system.
The National Institutes of Health-funded clinical trial findings, published this week in the in the New England Journal of Medicine, found that patients with a severe form of scleroderma who underwent chemotherapy, whole-body radiation and an autologous hematopoietic stem cell transplant (HSCT) had significant improved survival and quality of life than those treated with the immune-suppressing drug cyclophposphmide.
An HSCT is a procedure that involves the infusion of stem cells to treat patient malignancies or to repair diseased or defective bone marrow. An autologous HSCT involves taking healthy cells from the patient’s own blood or bone marrow and administering them to help the patient recover after high-dose chemotherapy.
Researchers found this type of transplantation offered significantly greater long-term benefits, but also carried well-known short-term risks, such as infections and low blood cell counts, when compared with cyclophosphamide.
“These results show that individuals with poor-prognosis scleroderma can improve and live longer and that these advances appear durable,” said lead author Keith Sullivan, M.D., James B. Wyngaarden Professor of Medicine and Cellular Therapy at Duke in a news release.
City of Hope, one of 26 clinical trial sites, is one of the country's largest and most successful bone marrow and blood stem cell transplant centers, and is one of only a few cancer centers offering this treatment for people diagnosed with the disease.
Our goal as physicians is to help give patients back their lives,” said co-author Stephen J. Forman, M.D., Francis & Kathleen McNamara Distinguished Chair in Hematology and Hematopoietic Cell Transplantation at City of Hope. "Stem cell transplants have been proven to help do that for patients with a variety of diseases, a list that now includes scleroderma.”
The clinical trial, called Scleroderma: Cyclopsphamide or Transplantation, was conducted over a 10-year period and compared the safety and potential benefits of the two treatment regimens among 75 participants with diffuse systemic sclerosis who had lung or kidney involvement.
Bone Marrow and Stem Cell Transplant Expertise at City of Hope
As a pioneer in advancing care for all hematologic cancers and related blood disorders, City of Hope uses leading-edge research to advance the safety and efficacy of HSCTs.
As one of the largest transplant centers in the United States, City of Hope has performed over 14,000 bone marrow/stem cell transplant procedures since 1976. For the past 13 consecutive years, City of Hope has been ranked as an “overperforming” transplant center with better than expected survival outcomes.
City of Hope’s Center for Stem Cell Transplantation team is working to reduce treatment toxicity and improve patient outcomes. Researchers have access to a large transplant patient population, rich historical data and a clinical research department with a reputation for outstanding transplant outcomes. The center’s research focuses on expansion of donor options for patients, treatment complications such as graft-vs-host disease, cytomegalovirus vaccine development and novel transplant regimens designed to reduce the risk of relapse.
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