Overview
The goal of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-threatening disease for which HSCT is indicated. These patients are not eligible for other Children's Hospital of Philadelphia IRB approved protocols that utilize CliniMACs technology for T depletion.
Description
Only 25-30% of patients who may benefit from HSCT have a matched related donor. There is a higher rate of complications using cells from an unrelated or partially matched related donor. T cells within the donor cells may cause a complication called graft vs. host disease (GVHD). The goal of this study is to use the CliniMACs device to remove the T cells that cause GVHD, called T cell depletion.
Eligibility
Inclusion Criteria:
- Patients who lack a fully HLA matched sibling and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT) but do not meet criteria for current open institutional protocols using ClinMACs device for CD3+/CD19+ depletion.
- Patients with the following transplantable diseases:
Non-malignant diseases:
Metabolic storage diseases correctable by HSCT
Bone marrow failure syndromes
Immunodeficiencies/immune dysregulation syndromes
Malignant diseases:
Acute leukemias
Chronic leukemias
Lymphomas
Myelodyplastic syndrome
- Organ function criteria:
Lansky or Karnofsky performance ≥60
Serum creatinine ≤3xupper limit of normal for age
Hepatic: Transaminases ≤10xnormal
Cardiac shortening fraction ≥27%
Bilirubin <2.5x normal (unless elevation due to Gilberts disease)
- No active untreated infection
- Signed informed consent
- No fully HLA matched sibling donor available
- Females of childbearing potential must have negative pregnancy test
Exclusion Criteria:
- Uncontrolled bacterial, viral or fungal infections
- Fully HLA matched sibling donor
- Donor unable to donate peripheral stem cells
- Pregnant Females