Know Cancer

forgot password

Phase 2
55 Years
Not Enrolling
Anemia, Aplastic, Fanconi Anemia, Hematologic Diseases, Leukemia, Neoplasms, Severe Combined Immunodeficiency, Hematopoietic Stem Cell Transplantation, Myelodysplastic Syndromes

Thank you

Trial Information


Bone marrow transplantation is an effective therapy for a variety of genetic and hematologic
disorders. Donated bone marrow, which provides a source of stem and progenitor cells for
bone marrow reconstitution, is obtained either from related donors, usually HLA-matched
siblings, or from HLA-matched unrelated donors. Because of the limited availability of
HLA-matched related donors, HLA-matched unrelated donors are frequently the only alternative
source of bone marrow for transplantation. To provide matched volunteer bone marrow donors
for unrelated recipients, the National Marrow Donor Program (NMDP) was established in 1986
to develop a national Registry of HLA-typed individuals who agree to donate bone marrow if
needed. More than 3 million potential marrow donors are now enrolled. A limitation of the
registry approach is the time needed to identify a donor and complete the necessary clinical
evaluation and laboratory testing for histocompatibility, infectious diseases, and general
good health. Moreover, a certain proportion of potential donors change their minds or
otherwise become unavailable between the time they enter the Registry and the time they are
called as a preliminary match for a patient. Another disadvantage is the marrow harvesting
procedure which requires the normal donor to be hospitalized and given general anesthesia in
an operating room. Finally, the NMDP Registry consists of potential donors that are
primarily of European Caucasian ancestry. Although great strides have been made in
increasing the numbers of African-Americans, Hispanics, Asian-Americans, and Native
Americans, it is still more difficult to find matches for these ethnic minorities than for
Caucasian patients.

Human umbilical cord blood is an alternative source of hematopoietic stem and progenitor
cells capable of reconstituting the bone marrow of recipients with a variety of diseases.
Cord blood stem and progenitor cells from related donors have been successfully transplanted
world-wide in children with genetic or hematologic diseases. These results suggested that
cord blood from unrelated, HLA-matched donors also can be used for patients who need a
transplant but don't have a related donor. The existing problems in unrelated-donor bone
marrow transplantation with donor recruitment, bone marrow harvesting, and matching for
antigens peculiar to a particular ethnic group would be reduced if typed, tested, and frozen
umbilical cord blood could be made easily and rapidly available. Such a system, which could
complement or partially replace the present process, has been shown to be feasible. In an
NHLBI-sponsored program, investigators have collected and cryopreserved a bank of about
9,000 human UCBUs. In addition, successful searches and matches (including many 4/6 antigen
matches) have resulted in more than 900 unrelated-donor, UCBU transplants to date.
Preliminary data from both these unrelated-donor and other related-donor cord blood
transplants suggest that there is less GvHD than if the source of the graft were adult bone
marrow. The severity of GvHD also seems less even in highly mismatched recipients. One
important question is whether or not 3/6 HLA matched transplants in children will have
acceptable 180-day disease free survival. It also remains to be determined if the
graft-vs-leukemia effect is also less or if unrelated-donor cord blood grafts are as durable
as those from matched, related-donors. Most transplants conducted thus far have been in
children. Hence another important question is whether or not there are sufficient numbers
of stem cells in cord blood to support transplantation to an unrelated adult.

The initiative was proposed by the Division of Blood Diseases and Resources staff and
approved by the February 1995 National Heart, Lung, and Blood Advisory Council. The
Requests for Proposals were released in June 1995. Contracts were awarded September 30,


The study is multicenter, with six Cord Blood Transplant Centers (CBTCs), two collecting and
storage centers (Cord Blood Banks -- CBBs) and one Coordinating Center. Each CBB used the
same protocol for recruiting donors, collecting, processing, testing, storage, retrieval
from storage, reprocessing from the frozen state, and shipping. Each participating CBTC
used the same patient selection criteria, preparative regimen for patients in the same
class, initial graft-versus-host disease (GvHD) prophylaxis, indications for the use of
cytokines, definitions for events and complications, and methods for evaluating immune
reconstitution. The main study evaluated the impact of HLA 3/6 and 4/6 matching on outcome.
The primary endpoint was 180-day disease free survival after UCBU transplantation.
Secondary endpoints included engraftment, the frequency and severity of acute and chronic
GvHD with stratification by degree of HLA match, overall survival, and immunologic
reconstitution. Data were collected on banked UCBUs to define unit quality by nucleated
cell count and flow cytometry of surface markers to identify effects on patient outcome.
Recruitment ended December 31, 2003 with the accrual of 326 subjects. .

Inclusion Criteria

Mothers of Infant-donors must complete a medical history form, have an uncomplicated
delivery, and deliver at UCLA or Duke.

Patients are those who need a transplant but don't have a matched marrow donor or can't
wait to find one.

Type of Study:


Study Design:

Primary Purpose: Treatment

Principal Investigator

Shelly Carter

Investigator Affiliation:

The EMMES Corporation


United States: Federal Government

Study ID:




Start Date:

September 1996

Completion Date:

October 2007

Related Keywords:

  • Anemia, Aplastic
  • Fanconi Anemia
  • Hematologic Diseases
  • Leukemia
  • Neoplasms
  • Severe Combined Immunodeficiency
  • Hematopoietic Stem Cell Transplantation
  • Myelodysplastic Syndromes
  • Anemia
  • Anemia, Aplastic
  • Neoplasms
  • Fanconi Anemia
  • Fanconi Syndrome
  • Hematologic Diseases
  • Immunologic Deficiency Syndromes
  • Leukemia
  • Myelodysplastic Syndromes
  • Preleukemia
  • Severe Combined Immunodeficiency