This study tests the clinical outcomes of a preparative regimen of fludarabine (FLU), anti-thymocyte globulin (ATG)/or Campath, and melphalan; followed by hematopoietic stem cell transplant, and a post transplant regimen of Cyclosporin A (CsA) in patients with immunologic or histiocytic disorders. The researchers hypothesize that this regimen will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease (GVHD). Patients will be randomized biologically into one of 3 arms based upon donor availability: (a) human leukocyte antigen (HLA) genotypic matched sibling donor, (b) HLA phenotypic matched unrelated peripheral blood stem cell (PBSC) donor, (c) two HLA 0-2 antigen mismatched unrelated cord blood donors (double cord).
Prior to transplantation, subjects will receive Melphalan, Fludarabine and Anti-Thymocyte Globulin (ATG) or Campath. These three drugs are being given to subjects to help the new stem cells take and grow. On the day of transplantation, subjects will receive stem cells transfused via intravenous (IV) catheter. After stem cell transplantation, subjects will be given Cyclosporin A (CsA) and mycophenolate mofetil (MMF) to reduce the risk of graft-versus-host disease, the complication that occurs when the donor's stem cells react against the patient.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
19
IV on Day 0
30mg/m\^2 IV Day -7 through Day -3
140 mg/m\^2 IV Day -1
30 mg/kg IV Day -5 through Day -1
0.2 mg/kg IV X 5 days (used as an alternative to Anti-thymocyte globulin (ATG) if unable to tolerate ATG) Day -10 through Day -6
2.5 mg/kg IV every 12 hours (adults) or every 8 hours (children \<40 kg) maintaining a level of \>200mg/L Day -3 until Day +180 when, if no GVHD, the dose will be tapered 10% per week beginning on day 181
15 mg/kg IV or orally bid and discontinued on Day +45 unless GVHD is present
500 mg/kg IV weekly beginning on Day +7 until Day +100
Masonic Cancer Center University of Minnesota
Minneapolis, Minnesota, United States
Number of Subjects With Mixed Chimerism
\>10% Donor Cells at Day 100
Time frame: Day 100
Percentage of Donor Chimerism at 100 Days
The percent of recipient bone marrow and blood cells that are of donor origin.
Time frame: Day 100
Percentage of Donor Chimerism at 180 Days
The percent of recipient bone marrow and blood cells that are of donor origin.
Time frame: Day 180
Percentage of Donor Chimerism at 365 Days
The percent of recipient bone marrow and blood cells that are of donor origin.
Time frame: Day 365
Incidence of Grade 2-4 Acute Graft Versus Host Disease (aGVHD)
Acute graft versus host disease (aGVHD) is a reaction occurring within the first 100 days after transplant where the T- cells of the donor graft attacks the recipient's (host's) skin, GI tract, liver and other organs. The severity of aGVHD is graded on a scale of 1 - 4 with the highest number representing the most severe disease.
Time frame: Day 100
Incidence of Grade 3-4 Acute Graft Versus Host Disease (aGVHD)
Acute graft versus host disease (aGVHD) is a reaction occurring within the first 100 days after transplant where the T- cells of the donor graft attacks the recipient's (host's) skin, GI tract, liver and other organs. The severity of aGVHD is graded on a scale of 1 - 4 with the highest number representing the most severe disease.
Time frame: Day 100
Incidence of Chronic Graft Versus Host Disease (cGVHD)
Chronic graft versus host disease (cGVHD) is a reaction which typically develops 3 to 6 months after transplant where the T- cells of the donor graft attacks the recipient's (host's) skin, GI tract, liver and other organs.
Time frame: 6 months and 1 year
Number of Subjects Alive at 100 Days
Time frame: Day 100
Number of Subjects Alive at One Year
Time frame: Day 365
Compare Quality of Life (QOL)
Time frame: Pretransplant, 1 year, 2 years and 5 years
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