Bone Marrow Transplantation Using CD34-Selected Stem Cells From Related or Unrelated Donors in Treating Participants With Cancer or Other Disorders

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.

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