Phase II Study: HSCT Using CD34 Selected Mismatched Related Donor and One Umbilical Cord Unit
This method of stem cell transplantation is designed to overcome some of the limitations of
other alternative donor transplant options. Use of unrelated umbilical cord unit (UCB)
donors appears to allow a greater degree of HLA mismatch with acceptable rates of GVHD.
However, when UCB transplant was studied in the adult population, investigators discovered
several limitations. One major limitation with UCB was delayed engraftment, resulting in
higher risk of infection in the early post transplant period. The limitations to cord blood
transplant involve delayed engraftment resulting in early complications such as infections.
The main limitation associated with haploidentical donors is the significant
immunosuppression required to prevent/treat aGVHD. Use of this combined modality of
transplantation appears to allow for rapid neutrophil engraftment from the haploidentical
donor and coupled with long term hematopoiesis from the UCB donor, thus requiring less long
This study tests a new method of bone marrow transplantation called combined
haploidentical-cord blood transplantation. In this procedure, some of the blood forming
cells (the stem cells) from a partially HLA matched (haploidentical) related donor are
collected from the blood, as well as cells from an umbilical cord are transplanted into the
patient (the recipient) after administration of a "conditioning regimen". A conditioning
regimen consists of chemotherapy and sometimes radiation to the entire body (total body
irradiation, or TBI),
One of two 'conditioning regimens' which will be determined by the physician.
1. FLUDARABINE, MELPHALAN, ATG
Fludarabine 30mg/m2(Days-7,-6,-5,-4,-3)-,Melphalan 70mg/m2(Day -3,-2), ATG
2. FLUDARABINE, BUSULFAN, 400 CGY TBI, ATG Fludarabine 50mg/m2(Day
-6,-5,-4,-3,-2),Busulfan 3.2mg/kg(Day -5,-4,-3,-2) 400cGY Total Body
Day 0 -Haploidentical donor and one umbilical cord blood unit infusion
Filgrastim will be administered daily from day +1 until blood counts have completely
recovered. Tacrolimus and another immunosuppressant, Cellcept, starting before transplant
also to reduce the risks of graft versus host disease and to promote the growth of the
graft. Tacrolimus will be given daily from two days before the transplant until at least
three months after transplantation. Cellcept, will be tapered after the cells engraft.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
The primary objective is to estimate the overall survival, separately in the two risk strata.
Jeanne Palmer, M.D.
Medical College of Wisconsin
United States: Institutional Review Board
|Froedtert Hospital/ Medical College of Wisconsin - Clinical Cancer Center||Milwaukee, Wisconsin 53226|