September 22, 2008
Clinical trial to determine effectiveness of unrelated
marrow and cord blood transplants.
A first-of-its-kind clinical trial may provide new treatment options to children with sickle cell disease (SCD), an inherited blood disorder that affects 70,000 people in the United States. The Sickle Cell Unrelated Transplant (SCURT) research study is a Phase II, multi-site clinical trial that will start accepting qualified patients later this month.
Currently, marrow or blood stem cell transplantation is the only potential cure for severe SCD. Most people with SCD who have undergone a transplant have received stem cells from family members, usually siblings. In this nationwide, multicenter clinical trial, researchers will evaluate the role of unrelated donor transplants in treating severe SCD and the effectiveness of a less-intensive regimen of chemotherapy to prepare patients for transplant.
SCD causes blood cells to clump together, which can block blood vessels. This blockage can damage the brain, bones, muscles, lungs, kidneys, liver, intestines and other organs and cause excruciating episodes of pain. Over time, people with SCD can experience permanent damage to vital organs and even sudden death. The study seeks to enroll 45 children ages 3-16 with symptoms of severe SCD, such as strokes, frequent pain crises or episodes of severe lung problems. Those who meet the eligibility requirements will receive a bone marrow or umbilical cord blood transplant from a suitably matched, unrelated donor to replace their own red blood cells.
Shalini Shenoy, M.D., medical director of the pediatric bone marrow transplant program at St. Louis Children's Hospital and Naynesh Kamani, M.D., of Children's National Medical Center in Washington, D.C. will serve as the lead transplant physicians for the SCURT trial. The multi-site trial will also include at least 20 other medical institutions across the United States.
"Doctors have performed related donor blood cell transplants to treat SCD, but few unrelated donor transplants have been preformed nationally" said Shenoy. "Although the overall success must be demonstrated, preliminary results have shown that most patients tolerate the treatment well, which compelled us to start this national trial."
Unrelated donor bone marrow and cord blood transplant is a proven treatment for leukemia, lymphoma and many other diseases. Overall, people in need of a bone marrow transplant have a 30 percent chance of finding a matched donor within their family. Those without a family donor can turn to the National Marrow Donor Program to search for an unrelated adult donor or cord blood unit.
With SCD, only 14 percent of potential transplant recipients find an appropriate matched donor within their family. Because SCD is inherited, there is a greater likelihood that other family members have SCD, which prevents them from being donors.
"Examining the success of unrelated donor transplants in treating SCD can potentially offer a matched unrelated donor and a curative treatment option," said Richard Labotka, M.D., a representative of the Sickle Cell Disease Clinical Research Network (SCD CRN) and director of the Pediatric Sickle Cell Program at the University of Illinois. "If the clinical trial results reflect the initial data we've seen, many more SCD families may be able to take advantage of an important treatment option that was not previously available to them."
Children in this trial will receive lower doses of chemotherapy than currently used for SCD transplants to reduce the treatment side effects. The study participants will be followed for two years after their transplant to determine whether they have been cured of SCD and assess the side effects from the transplant procedure.
The trial is facilitated by the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) and is funded by multiple organizations, including the National Heart, Lung, and Blood Institute; National Cancer Institute; National Marrow Donor Program; Sickle Cell Disease Clinical Research Network (SCD CRN) and the National Center on Minority Health and Health Disparities. Potential transplant candidates and their families can visit www.marrow.org for more information. Referring physicians can visit www.bmtctn.net.
About the BMT CTNThe BMT CTN was established in October 2001 to conduct large multi-institutional clinical trials. The trials address important issues in hematopoietic stem cell transplantation (HSCT), thereby furthering understanding of the best possible treatment approaches. The infrastructure of the BMT CTN is designed to permit continuity of operations and facilitate effective communication and cooperation among participating transplant centers, the National Heart, Lung, and Blood Institute, National Cancer Institute, National Marrow Donor Program and National Center on Minority Health and Health Disparities. The BMT CTN offers clinical trial participation to patients in all regions of the U.S.
The BMT CTN is committed to including widespread transplant community participation in these trials. However, due to the limited resources, not all requests can be accommodated for each protocol. If your center is interested in participating, please print and fax the completed Affiliate Center Application to BMT CTN DCC/EMMES at 301-251-1355 (or you may complete the short form if currently participating as an affiliate center in one of the BMT CTN trials). You will be contacted regarding your center's application for participation.
About the SCD CRN
The goal of the SCD CRN is to translate results from basic and Phase I/II studies into Phase III trials in patients with SCD and when appropriate thalassemia. The network tests new therapies to treat and prevent complications associated with SCD; create data sets that can be used to better characterize patients and their clinical course; apply genomic and proteomic techniques for improved diagnostic and therapeutic approaches; and expand the clinical application of multimodal therapies in SCD. Patients who receive care in institutions which are not members of the SCD CRN may be referred for evaluation of eligibility for this study.