Unrelated Umbilical Cord Blood (UBC)Transplantation
| Status: | Recruiting |
|---|---|
| Conditions: | Cancer, Blood Cancer, Lymphoma, Anemia, Hematology |
| Therapuetic Areas: | Hematology, Oncology |
| Healthy: | No |
| Age Range: | 16 - 70 |
| Updated: | 5/30/2018 |
| Start Date: | February 2009 |
| End Date: | December 2019 |
| Contact: | Pam Bunner, MT, CCRC |
| Email: | bunnerp@wvumedicine.com |
| Phone: | 304-598-4511 |
Unrelated Umbilical Cord Blood (UCB) Transplantation
Hematopoietic progenitor cell (HPC- primitive cells in the blood, bone marrow and umbilical
cord that can restore the bone marrow) transplant can be a curative therapy for the treatment
of hematologic malignancies (a disease of the bone marrow and lymph nodes). The source of
cells used for the transplant comes from related (sibling) and in cases where there is no
sibling match, from unrelated donors through the National Marrow Donor Program. The
availability of a suitable donor can be a significant obstacle for patients who need a
transplant but do not have a matched donor. Cord blood that has been harvested from an
umbilical cord shortly after birth has a rich supply of cells needed for transplant. These
stored cord bloods are now being used to transplant adults without a matched donor
Advantages to using cord blood includes a readily available source of cells with no risk to
the donor during the collection process, immediate source of cells in urgent situations (no
lengthy donor work-up)and a reduction in infectious disease transmission to the recipient.
One of the main disadvantages is the cord blood has a small number of cells needed for
transplant. In an adult, usually two cords are needed and large recipients do not qualify
because they need too many cells.
This study will use two different preparative regimens (chemotherapy and radiation) followed
by one or two umbilical cord units (UBC). The preparative regimen used will be chosen by the
physician and is based on patient's age, disease and medical condition at the time of
transplant.
Multiple objectives for this study include disease-free and overall survival, treatment
related mortality, rate of cells taking hold, and the incidence and severity of the
transplant complication called graft versus host disease (GVHD).
cord that can restore the bone marrow) transplant can be a curative therapy for the treatment
of hematologic malignancies (a disease of the bone marrow and lymph nodes). The source of
cells used for the transplant comes from related (sibling) and in cases where there is no
sibling match, from unrelated donors through the National Marrow Donor Program. The
availability of a suitable donor can be a significant obstacle for patients who need a
transplant but do not have a matched donor. Cord blood that has been harvested from an
umbilical cord shortly after birth has a rich supply of cells needed for transplant. These
stored cord bloods are now being used to transplant adults without a matched donor
Advantages to using cord blood includes a readily available source of cells with no risk to
the donor during the collection process, immediate source of cells in urgent situations (no
lengthy donor work-up)and a reduction in infectious disease transmission to the recipient.
One of the main disadvantages is the cord blood has a small number of cells needed for
transplant. In an adult, usually two cords are needed and large recipients do not qualify
because they need too many cells.
This study will use two different preparative regimens (chemotherapy and radiation) followed
by one or two umbilical cord units (UBC). The preparative regimen used will be chosen by the
physician and is based on patient's age, disease and medical condition at the time of
transplant.
Multiple objectives for this study include disease-free and overall survival, treatment
related mortality, rate of cells taking hold, and the incidence and severity of the
transplant complication called graft versus host disease (GVHD).
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.
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.
Inclusion Criteria:
- Age: 16-70 years
- Available 4/6, 5/6, or 6/6 HLA antigen match (using A, B, and DRB1) cord blood unit.
- ECOG performance status of 0-2 (Karnofsky greater than or equal to 70%)
- Serum bilirubin less than 2 x upper limit of normal
- Serum creatinine less than 2 mg/dl
- DLCO or FEV1 greater than or equal to 50% predicted
- Left ventricular ejection fraction greater than or equal to 35%
- no uncontrolled infection
- If female, not pregnant
- Informed consent given
- No major organ dysfunction precluding transplantation.
- One of the following malignancies or bone marrow failure syndromes:
- Chronic myelogenous leukemia (CML)
- Acute myelogenous leukemia (AML)
- Myelodysplastic syndrome
- Multiple myeloma
- Hodgkin lymphoma
- Non-Hodgkin lymphoma
- Chronic lymphocytic leukemia (CLL)
- Acute lymphocytic leukemia (ALL)
- Severe Aplastic Anemia
Exclusion Criteria:
- Patient pregnant
- Age less than 16, greater than 70
- ECOG performance status of greater than 2 (Karnofsky less than 70%)
- Psychiatric disorder or mental deficiency of the patient sufficiently severe as to
make compliance with the BMT treatment unlikely, or making informed consent impossible
- Serum bilirubin greater than or equal to 2 x upper limit of normal, transaminases
greater than 3 x upper limit of normal
- Serum creatinine greater than or equal to 2 mg/dl
- DLCO less than 50% predicted
- Left ventricular ejection fraction less than 35%
- Major anticipated illness or organ failure incompatible with survival from BMT
We found this trial at
1
site
Morgantown, West Virginia 26506
Principal Investigator: Michael Craig, MD
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