The purpose of this study is to determine whether stem cells collected from a donor's blood
stream will be as safe and effective as using bone marrow collected from a donor's pelvic
This is a pilot study to assess the safety and potential efficacy of haploidentical
peripheral blood stem cell transplantation using a nonmyeloablative preparative regimen and
post-transplant cyclophosphamide. The overall objective of this study is to collect the
efficacy and safety data to provide the basis to decide whether a larger study of clinical
efficacy is warranted in this setting.
1. Age< 70.
2. Molecular based HLA typing will be performed for the HLA-A, -B, -Cw, DRB1 and -DQB1
loci to the resolution adequate to establish haplo identity. A minimum match of 5/10
is required. An unrelated donor search is not required for a patient to be eligible
for this protocol if the clinical situation dictates an urgent transplant. Clinical
urgency is defined as 6-8 weeks from referral or low-likelihood of finding a matched,
3. Subjects must meet one of the disease classifications listed below:
Acute leukemias (includes T lymphoblastic lymphoma). Remission is defined as < 5%
blasts with no morphological characteristics of acute leukemia (e.g., Auer Rods) in a
bone marrow with > 20% cellularity, peripheral blood counts showing ANC >1000/ul,
including patients in CRp.
Acute Lymphoblastic Leukemia in high risk CR1 as defined by at least one of the
Adverse cytogenetics such as t(9;22), t(1;19), t(4;11), MLL rearrangements White blood
cell counts >30,000/mcL Patients over 30 years of age Time to complete remission >4
weeks Presence of extramedullary disease
Acute Myelogeneous Leukemia in high risk CR1 as defined by at least one of the
Greater than 1 cycle of induction therapy required to achieve remission Preceding
myelodysplastic syndrome (MDS) Presence of Flt3 abnormalities FAB M6 or M7 leukemia or
Adverse cytogenetics for overall survival such as:
those associated with MDS Complex karyotype (≥ 3 abnormalities) Any of the following:
inv(3) or t(3;3), t(6;9), t(6;11), + 8 [alone or with other abnormalities except for
t(8;21), t(9;11), inv(16) or t(16;16)], t(11;19)(q23;p13.1)
Acute Leukemias in 2nd or subsequent remission
Biphenotypic/Undifferentiated Leukemias in 1st or subsequent CR.
High-risk MDS status-post cytotoxic chemotherapy
Burkitt's lymphoma: second or subsequent CR.
Chemotherapy-sensitive (complete or partial response; see response criteria Appendix
C) large cell, Mantle Cell or Hodgkin's lymphomas that have failed at least 1 prior
regimen of multi-agent chemotherapy and are ineligible for an autologous transplant or
relapsed/progressed after autologous stem cell transplant.
Marginal zone B-cell lymphoma or follicular lymphoma that has progressed after at
least two prior therapies (excluding single agent Rituxan) and are ineligible for an
autologous transplant or relapsed/progressed after autologous stem cell transplant..
4. Patients with adequate physical function as measured by:
Cardiac: left ventricular ejection fraction at rest must be ≥ 35%.
Hepatic: bilirubin ≤ 2.5 mg/dL; and ALT, AST, and Alkaline Phosphatase < 5 x ULN.
Renal: serum creatinine within normal range for age, or if serum creatinine outside
normal range for age, then renal function(creatinine clearance or GFR) > 40
Pulmonary: FEV1, FVC, DLCO (diffusion capacity) ≥ 40% predicted (corrected for
hemoglobin); if unable to perform pulmonary function tests, then O2 saturation > 92%
on room air.
Performance status: Karnofsky/Lansky score ≥ 60%.
5. Patients who have received a prior allogeneic HSCT and who have either rejected their
grafts or who have become tolerant of their grafts with no active GVHD requiring
1. Donors must be HLA-haploidentical first-degree or second degree relatives of the
2. Age ≥ 18 years
3. Weight ≥ 40 kg
1. HLA-matched donor able to donate.
2. Pregnancy or breast-feeding.
3. Current uncontrolled bacterial, viral or fungal infection (currently taking medication
with evidence of progression of clinical symptoms or radiologic findings).
1) Positive anti-donor HLA antibody.