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The purpose of this original research study by UPMC Children’s Hospital of Pittsburgh is to determine whether pediatric and young adult patients with high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) can benefit from a novel myeloablative conditioning (MAC) regimen or reduced-intensity conditioning (RIC) prior to hematopoietic stem cell transplant (HSCT) as treatment for their illness. Conditioning before transplant destroys blood-forming cells in the patient’s bone marrow to prepare the marrow for new donor cells. The test regimens are designed to reduce overall toxicity so patients with these high-risk conditions, who might otherwise have been ineligible to proceed to HSCT, can now receive potentially life-saving treatment.
Subject to certain exclusion criteria, this study is enrolling participants of both genders through age 26, who will be receiving HSCT for treatment of AML or MDS.
Males and Females: Through age 26
Based on medical evaluations, participants will be assigned to either the novel MAC group or the RIC group about 3 weeks before the HSCT. Participants will begin taking the conditioning drugs 12 or 14 days prior to transplant. For conditioning, patients will be admitted to a special isolation room to minimize contact with germs, and conditioning drugs will be administered via intravenous infusion or orally.
Visits: Requires hospitalization for conditioning and transplant, and there will be 5 visits following transplant
Duration: Patients will be followed for 1 year, post transplant
Study Description at National Institutes of Health
Blood and Marrow Transplantation and Cellular Therapies Research
Randy Windreich, MD
For more information about the study or enrollment, please contact:
Shawna McIntyre, Clinical Research Supervisor
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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