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
- 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
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.
Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Event-free survival by disease assessment
at 100 days and at 6, 9, 12, 18, and 24 months
Mary J. Laughlin, MD
Ireland Cancer Center at University Hospitals Case Medical Center, Case Comprehensive Cancer Center
United States: Federal Government
|Ireland Cancer Center at University Hospitals Case Medical Center, Case Comprehensive Cancer Center||Cleveland, Ohio 44106-5065|