Donor Stem Cell Transplant Followed by Cyclophosphamide in Treating Patients With Hematological Diseases

PRIMARY OBJECTIVES:

I. To determine if haploidentical stem cell transplant using post-transplant cyclophosphamide
results in 60% or better disease free survival (DFS) at 12 months at our institution.

SECONDARY OBJECTIVES:

I. To determine the rate of acute and chronic graft-versus-host disease (GvHD), non-relapse
mortality, and relapse.

OUTLINE:

PREPARATIVE REGIMEN: Patients receive fludarabine phosphate intravenously (IV) once daily
(QD) on days -6 to -2. Patients receiving myeloablative conditioning receive busulfan IV
every 6 hours for 16 doses on days -7 to -4 and patients receiving reduced intensity
conditioning receive busulfan IV every 6 hours for 8 doses on days -5 to -4. Patients also
receive cyclophosphamide IV QD on days -3 and -2

TRANSPLANT: Patients undergo stem cell transplant on day 0.

GVHD PROPHYLAXIS: Patients receive cyclophosphamide QD on days 3 and 4, tacrolimus on days
5-180, and mycophenolate mofetil on days 5-35.

After completion of study treatment, patients are followed up periodically for 2 years.

Inclusion Criteria:

- Diagnosis of a hematological malignancy requiring an allogeneic stem cell transplant
consistent with the standard of care

- Hematologic remission of disease

- Lack of 8 out of 8 human leukocyte antigen (HLA) loci-matched related donor or
HLA-matched unrelated donor

- Available familial haploidentical (4 to 7 out of 8 HLA loci-matched) donor

Exclusion Criteria:

- Significant organ dysfunction > grade 3 by Common Terminology Criteria for Adverse
Events (CTCAE), and a left ventricular ejection fraction < 40% (evaluated by
echocardiogram) or < 30% (evaluated by magnetic resonance imaging [MRI]) within the
first 30 days after stem cell transplant (SCT)

- Human immunodeficiency virus (HIV) positive (recipients who are positive for hepatitis
B [hepatitis B virus (HBV)], hepatitis C [hepatitis C virus (HCV)] or human T-cell
lymphotropic virus [HTLV-]I/II are not excluded from participation)

- Positive pregnancy test for women of childbearing age

- Estimated probability of surviving less than 3 months

- Major anticipated illness or organ failure incompatible with survival form transplant

- Severe psychiatric illness or mental deficiency sufficiently severe as to make
compliance with the transplant treatment unlikely and informed consent impossible