Transplantation of Umbilical Cord Blood From Unrelated Donors in Patients With Haematological Diseases Using a Reduced Intensity Conditioning Regimen
- To assess the safety and efficacy of unrelated-donor umbilical cord blood
transplantation (UCBT) using a nonmyeloablative preparative regimen in patients with
hematological disease, in a multi-institution UK setting.
- To confirm that unrelated-donor UCBT following nonmyeloablative conditioning is
associated with consistent and durable engraftment in these patients.
- To assess transplant-related mortality at day 100 associated with nonmyeloablative UCBT
in these patients.
- To assess the incidence of grades II-IV and III-IV acute graft-vs-host disease (GVHD)
in these patients.
- To assess the risk of relapse and progressive disease in these patients at 1 year post
transplant after nonmyeloablative UCBT.
- To assess overall and progression-free survival of these patients at 1 year after
- To assess immune reconstitution at 1, 2, 3, 6, 12, and 24 months after transplant as
measured by quantitative recovery of B, T, and NK cells (flow cytometry), qualitative
recovery of T cells (TREC and spectratyping), in vivo functional T-cell responses (EBV
and CMV tetramers), and quantitative immunoglobulins.
OUTLINE: This is a multicenter study.
- Reduced-intensity conditioning regimen: Patients receive cyclophosphamide IV over 2
hours on day -6 and fludarabine phosphate IV over 1 hour on days -6 to -2. Patients
undergo a single fraction of total-body irradiation on day -1.
- Umbilical cord blood (UCB) transplantation: Patients undergo umbilical cord blood
transplantation on day 0.
- Graft-vs-host disease prophylaxis: Patients receive cyclosporine IV or orally on days
-3 to 100 followed by taper and mycophenolate mofetil IV or orally on days -3 to 35
followed by taper.
Blood and bone marrow samples are collected periodically for analysis.
After completion of study treatment, patients are followed up every 3 months in year 1,
every 4 months in year 2, every 6 months until 5 years, and then annually thereafter.
Peer Reviewed and Funded or Endorsed by Cancer Research UK.
Allocation: Non-Randomized, Masking: Open Label, Primary Purpose: Treatment
Non-relapse mortality at day 100
Rachael Hough, MD
University College London Hospitals