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A Two Step Approach To Allogeneic Hematopoietic Stem Cell Transplantation for Hematologic Malignancies From HLA Partially-Matched Related Donors

Phase 1/Phase 2
18 Years
Not Enrolling
Hematologic Malignancies

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Trial Information

A Two Step Approach To Allogeneic Hematopoietic Stem Cell Transplantation for Hematologic Malignancies From HLA Partially-Matched Related Donors

Haploidentical hematopoietic stem cell transplant is a life-saving therapy for patients who
are without well matched donors. This type of therapy has been associated with poor
outcomes in the past due to complications such as infection. The Jefferson 2 Step approach
was designed to allow the infusion of an exact dose of tolerized lymphocytes in
haploidentical transplant in order to allow for immune reconstitution post transplant to
avoid infectious complications while still having acceptable rates of GVHD. In this
approach, patients with high-risk hematological malignancies undergo 8 fractions of TBI (12
Gy) followed by an exact dose of donor lymphocytes. The phase I portion of the study
determined the optimal dose of lymphocytes. Two days after receiving the donor lymphocytes,
the patients receive 2 daily doses of cyclophosphamide. One day after receiving
cyclophosphamide, the patients receive stem cell from their donor. Tacrolimus and
mycophenylate mofetil are used as GVHD prophylaxis.

Inclusion Criteria:

1. Any patient with a hematologic or oncologic diagnosis in which allogeneic HSCT is
thought to be beneficial, and in whom front-line therapy has already been applied.

2. Patients must have a related donor who is either a one, two or three out of six
antigen mismatch at the HLA-A;B;DR loci.

3. Patients without a well-matched unrelated donor or those who have a disease status
that precludes a wait for an identified unrelated donor.

4. Patients must adequate organ function:

- LVEF of >45%

- FVC or FEV1 >45% of predicted

- Adequate liver function as defined by a serum bilirubin <1.8, AST or ALT < 2.5X
upper limit of normal

- Serum creatinine < 2.0 mg/dl or creatinine clearance of > 40 ml/min

5. Performance status > 60% (Karnofsky)

6. Patients must be willing to use contraception if they have childbearing potential

7. Able to give informed consent

Exclusion Criteria:

1. An eligible HLA-identical sibling donor.

2. Performance status < 60% (Karnosfsky)

3. HIV positive

4. Active involvement of the central nervous system with malignancy

5. Psychiatric disorder that would preclude patients from signing an informed consent

6. Pregnancy

7. Patients with life expectancy of < 6 months for reasons other than their underlying
hematologic/oncologic disorder.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Overall Survival of Participants

Outcome Description:

To determine overall survival at 6 months post-transplant.

Outcome Time Frame:

6 months

Safety Issue:


Principal Investigator

Neal Flomenberg, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Thomas Jefferson University


United States: Institutional Review Board

Study ID:




Start Date:

January 2006

Completion Date:

June 2010

Related Keywords:

  • Hematologic Malignancies
  • Hematologic Malignancies
  • Two Step Approach
  • Haploidentical Transplant
  • Neoplasms
  • Hematologic Neoplasms



Thomas Jefferson UniversityPhiladelphia, Pennsylvania  19107-6541