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A Two Step Approach to Haploidentical Hematopoietic Stem Cell Transplantation for Patients in Remission From HLA Partially-Matched Related Donors-Effect of Maternal Donors on Outcomes

Phase 2
18 Years
Open (Enrolling)
Malignant Neoplasm

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Trial Information

A Two Step Approach to Haploidentical Hematopoietic Stem Cell Transplantation for Patients in Remission From HLA Partially-Matched Related Donors-Effect of Maternal Donors on Outcomes


I. Examine the 1 year disease free survival (DFS) rate of patients with maternal donors or
sibling donors who share the maternal haplotype (maternal group) and compare them to
patients receiving cells from donors who have points from other characteristics such as
killer immunoglobulin-like receptor (KIR) ligand mismatching, minor histocompatibility
antigen (MHag) differences, or number of human leukocyte antigen (HLA) mismatches
(non-maternal group).


I. Assess the incidences of relapse and graft-versus-host disease (GVHD) in maternal
recipients whose only eligible donors are offspring.

II. Assess the incidence of grades III-IV GVHD in female recipients with male donors.

III. Compare the rates of DFS in recipient-donor combinations in which there is at least 1
KIR ligand mismatch versus those without a KIR ligand mismatch.


Patients undergo total body irradiation (TBI) twice daily (BID) on days -9 to -6, undergo
donor lymphocyte infusion (DLI) on day -6, and receive cyclophosphamide intravenously (IV)
over 2 hours on days -3 and -2.

TRANSPLANT: Patients undergo haploidentical allogeneic hematopoietic stem cell transplant on
day 0.

GVHD PROPHYLAXIS: Patients receive tacrolimus IV beginning on day -1 with taper beginning on
day 42, and mycophenolate mofetil IV BID from day -1 to day 28.

After completion of study treatment, patients are followed up at 90, 180, and 270 days, and
1 year.

Inclusion Criteria:

1. Any patient with a hematologic or oncologic diagnosis without morphological evidence
of disease in which allogeneic HSCT is thought to be beneficial.

2. Patients must have a related donor who is a two or more allele mismatch at the HLA-A;
B; C; DR loci.

3. Patients must have adequate organ function:

1. LVEF (Left ventricular ejection fraction) of >50%

2. Diffusion Capacity for Carbon Monoxide (DLCO) >50% of predicted corrected for

3. Adequate liver function as defined by a serum bilirubin <1.8, Aspartate
Aminotransferase (AST) or alanine aminotransferase (ALT) < 2.5X upper limit of

4. Creatinine clearance of > 60 ml/min

4. Performance status > 80% (TJU Karnofsky)

5. Hematopoietic Comorbidity Index (HCT-CI) Score < 5 Points

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

7. Able to give informed consent, or if decisionally impaired, have a legal next of kin
or guardian that can give informed consent

Exclusion Criteria:

1. Performance status < 80 % (TJU Karnofsky)

2. HCT-CI Score > 5 Points

3. Combination of Performance status of < 80% (TJU Karnofsky) and an HCT-CI of 4 points
or more.

4. HIV positive

5. Active involvement of the central nervous system with malignancy

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

7. Pregnancy

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

9. Patients who have received alemtuzumab within 8 weeks of the transplant admission, or
who have recently received horse or rabbit anti-thymocyte globulin and have an ATG
level of > 2 ugm/ml

10. Patients who cannot receive cyclophosphamide

11. Patients with evidence of another malignancy, exclusive of a skin cancer that
requires only local treatment, should not be enrolled on this protocol

Type of Study:


Study Design:

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

Outcome Measure:

Disease-free survival (DFS)

Outcome Description:

Defined as the time to death, relapse or disease progression. The difference in DFS between maternal and non-maternal groups will be tested using log-rank test. The survival curves for each group will be estimated using the Kaplan-Meier estimator.

Outcome Time Frame:

1 year

Safety Issue:


Principal Investigator

Dolores Grosso, DNP, CRNP

Investigator Role:

Principal Investigator

Investigator Affiliation:

Thomas Jefferson University


United States: Institutional Review Board

Study ID:




Start Date:

May 2013

Completion Date:

Related Keywords:

  • Malignant Neoplasm
  • Neoplasms
  • Hematologic Neoplasms



Thomas Jefferson UniversityPhiladelphia, Pennsylvania  19107-6541