New transplant method aims to tame dangerous side effects for rare blood disease patients

NCT ID NCT04965597

Summary

This study tested a new combination of drugs given before a bone marrow or blood stem cell transplant. The goal was to see if this new 'conditioning' regimen could help patients with rare bone marrow failure diseases have fewer and less severe complications after their transplant. The trial involved 40 patients under age 50 with specific genetic blood disorders. Researchers measured success by looking at survival without major side effects like graft-versus-host disease one year after the procedure.

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Contacts and locations

Locations

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30329, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Cincinnati Children's Hospital

    Cincinnati, Ohio, 45229, United States

  • Cohen Children's Hospital of NY

    Queens, New York, 11040, United States

  • Duke University Medical Center

    Durham, North Carolina, 27705, United States

  • Fred Hutch/University of Washington Cancer Consortium

    Seattle, Washington, 98109, United States

  • Johns Hopkins University

    Baltimore, Maryland, 21231, United States

  • MD Anderson Cancer Center

    Houston, Texas, 77030, United States

  • Medical College of Wisconsin/Children's Hospital of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Oregon Health & Science University

    Portland, Oregon, 97239, United States

  • Primary Children's/University of Utah

    Salt Lake City, Utah, 84113, United States

  • Rady Children's Hospital/UCSD

    San Diego, California, 92123, United States

  • Roswell Park Comprehensive Cancer Center

    Buffalo, New York, 14203, United States

  • St. Louis Children's Hospital

    St Louis, Missouri, 63110, United States

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • University of California San Francisco

    San Francisco, California, 94143, United States

  • University of Michigan Medical Center

    Ann Arbor, Michigan, 48109, United States

  • University of Minnesota

    Minneapolis, Minnesota, 55455, United States

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

Conditions

Explore the condition pages connected to this study.