This expanded access protocol studies bone marrow transplantation using CD34-selectedstem cells from related or unrelated donors in treating participants with cancer or otherdisorders. Stem cells collected from the donor will be processed using a new devicecalled CliniMACS CD34 Reagent System which marks the blood cells collected from the donorwith a special protein called "antibody" that tags only the donor stem cells, sorting outother cells of the blood and immune system. This is done to remove, at least partially,some of the T cells. T cells are the cells in the blood that work as scavengers of theimmune system deciding what belongs and what does not. These cells can sometimes causerejection of the donor graft or a condition called graft-versus host disease (GVHD),where the donor cells can attack the body of the recipient. A bone marrow transplantationusing CD34-selected stem cells may reduce the risk of these unwanted side effects oftransplant as much as possible.
PRIMARY OBJECTIVES:
I. To provide a source of CD34-selected stem cells for patients with malignant and
nonmalignant disorders undergoing bone marrow transplantation from haploidentical (human
leukocyte antigen [HLA]-mismatched) related donors or HLA-compatible unrelated donors
SECONDARY OBJECTIVES:
I. Monitoring the safety of the CD34-selected stem cells for the recipient, as measured
by adverse events related to stem cell infusion, incidence of engraftment of neutrophils
and platelets, incidence of acute and chronic GVHD, and one year overall survival,
disease-free survival, and primary disease recurrence.
OUTLINE:
Donor stem cells undergo CD34 selection ex vivo using the CliniMACS CD34 Reagent System
using standard operating procedures (SOPs) from the manufacturer. Recipients undergo
standard of care preparative regimen, bone marrow transplantation with CD34-selected
peripheral blood stem cells via infusion over 1 to 2 hours on day 0, and then receive
standard of care GVHD prophylaxis.
After completion of study treatment, recipients are followed up at least once weekly
while inpatient until transplant day 100, every 1-3 months for the first year and yearly
thereafter.
Procedure: Bone Marrow Transplantation
Undergo BMT
Other Name: BMT,Bone Marrow Grafting,Bone marrow transplant,Marrow Transplantation
Device: CliniMACS CD34 Reagent System
CD34 selection ex vivo
Procedure: Peripheral Blood Stem Cell Transplantation
Receive CD34-selected peripheral blood stem cells from related or unrelated donors
Other Name: PBPC transplantation,PBSCT,Peripheral Blood Progenitor Cell Transplantation,Peripheral Stem Cell Support,Peripheral Stem Cell Transplant,Peripheral Stem Cell Transplantation
Inclusion Criteria:
- Patients with malignant or nonmalignant diseases that can benefit from alternative
stem cell transplantation according to institutional standard practice guidelines
- Patients lacking a healthy human leukocyte antigen (HLA)-identical related donor and
having one of the following alternative donor sources:
- HLA-haploidentical related (HLA antigen genotypic match >= 4/8 and <= 7/8) or
- Unrelated donor that is HLA matched at >= 7/8 HLA antigen loci
- The selected donor must also be:
- Able to receive granulocyte colony-stimulating factor (G-CSF, filgrastim) and
undergo apheresis either through placement of peripheral or temporary central
venous catheter, based on institutional guidelines for peripheral blood stem
cell collection
- Meet all institutional standard criteria for clearance for peripheral stem cell
collection
- Recipients must meet institutional treatment standards based on pre-transplant
evaluations including:
- Adequate major organ functions
- Free of major systemic infections
- Not pregnant, if female of childbearing age (post-pubertal)
- Recipient (if >= 18 years) or their parent/legal guardian (if < 18 years) must be
able to sign the informed consent form for the treatment plan; assent will be
obtained from recipients 7-17 years of age, in accordance to our institutional
guidelines
Exclusion Criteria:
- Patients not meeting clearance criteria for bone marrow transplantation (BMT) based
on standard institutional guidelines
- Presence of anti-HLA antibodies against donor antigens in a recipient or anti-HLA
antibodies against recipient antigens in a donor
- Known allergy to murine (mouse) protein or iron-dextran
OHSU Knight Cancer Institute
Portland, Oregon, United States
Investigator: Eneida R. Nemecek
Contact: 503-494-0829
nemeceke@ohsu.edu
Investigator: Eneida R. Nemecek
Eneida Nemecek, MD
503-494-0829
nemeceke@ohsu.edu
Eneida Nemecek, MD, Principal Investigator
OHSU Knight Cancer Institute