A Phase II Study of Umbilical Cord Blood Transplantation Following Myeloablative or Reduced-Intensity Conditioning
Allogeneic stem cell transplantation (SCT) following myeloablative and non-myeloablative
conditioning therapy has proven curative treatment for a number of inherited and acquired
hematologic disorders. The success of allogeneic transplantation is largely determined by
compatibility between donor and recipient, which predicts the risk of fatal
graft-versus-host disease (GVHD). Unfortunately, less than one third of patients needing an
allogeneic transplant have an available compatible donor in their family. Registries have
been established to match patients with compatible volunteer (unrelated) donors, but many
patients, and in particular minority patients, still lack stem cell donors.
Umbilical cord blood (UCB) is a rich source of hematopoietic stem cells, which is readily
available from the placenta following childbirth. Blood banks have been established in the
United States and abroad to collect, process and store UCB for use in allogeneic
transplantation. To date, more than 2000 UCB transplants have been performed in adults and
children around the world.
Rationale for use of Umbilical Cord Blood in Transplantation
UCB has a number of proven and theoretical advantages as an alternative source of
hematopoietic stem cells for transplantation:
1. Placental or umbilical cord blood is an abundantly available source of stem cells,
which is currently discarded and can be harvested at no risk to the mother or infant.
2. Important infectious agents, particularly CMV, are much less common in the newborn than
adults, and are less likely to contaminate UCB collections.
3. UCB collections, typed, cryopreserved and banked, are available on demand, eliminating
delays and uncertainties that now complicate marrow collection from unrelated donors.
At present, UCB can be delivered for infusion within days of the initiation of a
search. This compares with a median of 3 months from search to delivery of stem cells
through the registries of volunteer adult donors.
4. The intensity of graft-versus-host reactivity of fetal lymphocytes appears to be less
than that of adult cells and consequently fetal lymphocytes are more tolerant of HLA
incompatibility. Published studies have shown that transplantation of UCB matched at
4-5/6 antigens results in a comparable incidence of GVHD to transplantation of
unrelated stem cells fully matched at 6/6 antigens.
5. Frozen UCB can be easily shipped, stored at the treating institution, and thawed for
use when needed, compared to freshly donated stem cells which have a limited shelf-life
of one day or less, necessitating coordination between harvesting surgeons,
transportation, and transplantation teams.
This research study has been designed for people who have been diagnosed with a blood tumor,
which has not responded to treatment or has recurred, a bone marrow failure state such as
aplastic anemia, or one of certain inherited metabolic disorders; and whose doctor feels the
best treatment is an allogeneic stem cell transplant (alloSCT) but a related or unrelated
adult donor is not available. Instead, a single unit of umbilical cord blood (UCB) will be
used as the source of the subject's immune system. This study is designed to determine
whether a single unit of UCB can be substituted for adult bone marrow cells in the standard
stem cell transplant regimens used at this hospital for subjects who do not have stem cell
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Determine the rate of neutrophil engraftment in adult patients receiving umbilical cord blood for hematopoietic rescue following myeloablative or non-myeloablative conditioning.
+45 and 90 days
Andreas Klein, MD
Tufts Medical Center
United States: Institutional Review Board
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