Clinical Trial: Pilot Study Of Unrelated UCB Transplant for Non-Malignant Hematologic Conditions
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Patients With Severe Aplastic Anemia, Inborn Errors in Metabolism, or Inherited Hematologic Stem Cell Disorders
Brief Summary:
RATIONALE: Umbilical cord blood transplantation may allow doctors to give higher doses of chemotherapy or radiation therapy and kill more cancer cells.
PURPOSE: This phase II trial is studying how well umbilical cord blood transplantation works in treating patients with severe aplastic anemia, malignant thymoma, or myelodysplasia.
Detailed Summary:
OBJECTIVES:
- Determine the rates of durable engraftment in patients with severe aplastic anemia, myelodysplastic syndrome, inborn errors of metabolism, or inherited hematopoietic disorders, refractory to medical management, who are undergoing high-dose chemoradiotherapy followed by unrelated cord blood (UCB) transplantation.
- Evaluate the rate and quality of immunologic reconstitution in this patient population.
OUTLINE: Patients are stratified according to weight (under 45 kg vs over 45 kg).
Patients receive high-dose chemotherapy and/or radiotherapy as a conditioning regimen beginning 6-9 days before the umbilical cord blood transplant (UCBT). The regimen varies according to the underlying cause of the anemia, but could include busulfan, cyclophosphamide or melphalan, anti-thymocyte globulin or methylprednisolone, and/or radiation therapy. One day after the conditioning regimen is completed, patients receive the UCBT.
Patients are followed weekly for 3 months, at 6 months, then every 6 months for 2.5 years, then annually thereafter.
PROJECTED ACCRUAL: A total of 4-90 patients will be accrued for this study within 5 years.
Sponsor: Case Comprehensive Cancer Center
Current Primary Outcome: Event-free survival by disease assessment [ Time Frame: at 100 days and at 6, 9, 12, 18, and 24 months ]
Original Primary Outcome:
Current Secondary Outcome: Umbilical cord blood donor engraftment by chimerism and complete blood count (CBC) at time of myeloid recovery. [ Time Frame: 100 days and at 6, 9, 12, 18, and 24 months ]
Original Secondary Outcome:
Information By: Case Comprehensive Cancer Center
Dates:
Date Received: November 1, 1999
Date Started: January 1998
Date Completion:
Last Updated: June 10, 2010
Last Verified: June 2010
