Trial Information

Unrelated Umbilical Cord Blood (UCB) Transplantation

Allogeneic hematopoietic cell transplantation (allo- HCT) is a curative therapy for the
treatment of hematological and non-hematological malignancies and certain non-malignant
conditions. Bone marrow or peripheral blood from a Human Leukocyte Antigen (HLA) matched
sibling donor is the most commonly used source of allogeneic stem cells. However, HLA
matched siblings are available for less than one third of the patients who require allo-
SCT. In the absence of an HLA matched sibling, volunteer unrelated donors or partially
mismatched related donors (PMRD), stored cord blood may be used as a source of allogeneic
stem cells. Stored cord blood has been used as a source of allogeneic stem cells in infants
and children, but had early skepticism in adults because of concerns about the engraftment
potential of the relatively limited number of stem cells. The number of stem cells in a unit
of cord blood is generally one log less than the number of stem cells on an average
collection of bone marrow from an adult for transplantation.

After the success of the first allogeneic umbilical cord blood transplantation in 1988,
programs for banking screened unrelated donor CBSC have been initiated both in the United
States and Europe. Dr Pablo Rubenstein started the first such bank at the New York Blood
Center (NYBC) in 1993. Since its inception, the NYBC has provided unrelated donor cord blood
stem cells for over 1000 transplants. Analysis of outcomes for the initial 562 transplant
recipients from the NYBC revealed a cumulative rate of engraftment of 81% by day 42 for
PMNs. and 85% by day 180 for platelets. Currently, approximately more than 100,000 cord
blood units are available in cord blood banks worldwide and more than 2000 patients have
received cord blood transplants from these banks. NetCord, an international cooperative
group of cord blood banks, has developed a detailed set of standards for cord blood banking
to facilitate international exchanges and to guarantee the quality of these products.

Cord Blood Unit Selection:

UCB units will be required to be a 4 to 6 of 6 HLA-A, -B antigen and -DRB1 allele match with
the patient. Typing at HLA-C and -DQ will be obtained but not required in the match
strategy. A minimum total nucleated cell (TNC) dose of >2.0 x 107/kg at the time of
freezing will be utilized when possible. When using double units, each unit should contain
a minimum pre-cryopreserved TNC dose of 1.5 x 107/kg.

UCB Transplant Procedure:

There will be a myeloablative and reduced-intensity preparative regimen that can be given
prior to infusion of cord product. The myeloablative approach will be selected in younger
patients (<50yo) with a HCT-CI score <3. The reduced-intensity regimen will be selected for
all older patients (>50) or younger patients with a HCT-CI score >3. The reduced-intensity
regimen will also be chosen for any patients being transplanted for indolent/follicular
lymphomas, CLL, myeloma, or Hodgkin lymphoma; irrelevant of age or HCT-CI score. On a case
by case basis, patients may receive a preparative regimen outside of their designated
category as noted above with the approval of the PI, if deemed in the patient's best
interest.