This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.
Based on diagnosis and clinical history, a determination of the most appropriate regimen will be made based on the following prep plans: Arm A: Fully Myeloablative Preparative Regimen, Arm B: Reduced Toxicity Ablative Preparative Regimen, Arm C: Reduced Intensity Conditioning, Arm D: No Preparative Regimen
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
57
0.3 mg/kg intravenously (IV) on days -12 through -10
cyclophosphamide 50 mg/kg IV on days -9 through -6
busulfan 0.8 or 1.1 mg/kg IV on days -5 through -2
Unrelated donor bone marrow will be collected in the usual manner using established parameters determined by the National Marrow Donor Program. A minimum of 3 x 10\^8 nucleated cells/kg recipient weight will be collected with a goal of ≥ 5 x 10\^8 nucleated cells/kg recipient weight. Umbilical cord blood selection will be per the current University of Minnesota Cord Blood Unit Selection algorithm. One or two units may be used to obtain the minimum cell dose. One of the UCB units selected for transplantation must contain ≥ 3.5 x 10\^7 nucleated cells/kg recipient weight based on cell numbers at time of cryopreservation, and the total combined cell dose of both units must be \> 5.0 x 10\^7 nucleated cells/kg.
40 mg/m\^2 IV on days -5 through -2 (for children \< 6 months and/or \< 10 kg weight dose at 1.33 mg/kg)
140 mg/m\^2 IV on day -3
0.2 mg/kg intravenously (IV) on days -14 through -10
busulfan 0.8 or 1.1 mg/kg IV on days -9 through -6
fludarabine 30 mg/m\^2 IV on days -8 through -4
administered as per the standard institutional protocol.
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States
Neutrophil Engraftment
Neutrophil engraftment is defined as the first day of three consecutive days where the neutrophil count (absolute neutrophil count) is 500 cells/mm3 (0.5 x 109/L) or greater.
Time frame: Day 42
Incidence of Graft Failure
Graft failure is defined as not accepting donated cells. The donated cells do not make the new white blood cells, red blood cells and platelets.
Time frame: Day 100
Incidence of Chimerism
a state in bone marrow transplantation in which bone marrow and host cells exist compatibly without signs of graft-versus-host rejection disease.
Time frame: Day 100, 6 Months, 1 Year
Incidence of Acute Graft-Versus-Host Disease
Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host.
Time frame: Day 100
Incidence of Chronic Graft-Versus-Host Disease
Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host.
Time frame: 6 Months and 1 Year
Incidence of Transplant-Related Mortality
In the field of transplantation, toxicity is high and all deaths without previous relapse or progression are usually considered as related to transplantation.
Time frame: 6 Months
Disease-Free Survival
the length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works.
Time frame: 6 Months
Overall Survival
Overall survival will be defined as time from enrollment to date of death or censored at the date of last documented contact for patients still alive.
Time frame: 6 Months
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