Treatment Study to assess of safety and efficiency of conditioning with Plerixafor and G-CSF as additional agents for prevention of graft failure after transplantation in patients with chronic granulomatous disease
Severe primary or secondary graft dysfunction is one of major problem in patients with Chronic granulomatous disease (CGD). In this study the hypothesis is that the use of plerixafor and G-CSF as additional agents in conditioning regimen would offers advantages. The effect is based on mobilizing bone marrow stem cells into the peripheral blood and blocking CXCR4 chemokine receptors to prevent stem cell homing. Thus, some have hypothesized that plerixafor and G-CSF make free stromal space of the bone marrow available for donor stem cell engraftment. Moreover, stem cell release probably leads to liberation of host stem cells from the anti-apoptotic effects of the BM stroma for the more powerful effect of chemotherapy. Thus, the purpose of this study is to evaluate the safety and efficiency of myeloablative conditioning with Plerixafor and G-CSF as additional agents for prevention of graft failure after stem cell transplantation in patients with chronic granulomatous disease.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
17
Plerixafor for Conditioning before HSCT.
GCSF for Conditioning before HSCT.
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology
Moscow, Russia
RECRUITINGEvent free survival
The EFS probability compared with historical control. We mean event as primary (non-engraftment) and secondary (rejection) graft dysfunction.
Time frame: 1 year
1. Overall survival
The OS probability compared with historical control
Time frame: 1 year
Proportion of patients with full/mixed donor chimerism
Evaluation of the percentage of patients with the full/mixed donor chimerism (whole blood and CD3+ lineage). In addition, patients will be divided in accordance with % of donors cells: \>95%; 50%-95%; 10%-49%; \<10%. All data will be compared with historical control
Time frame: 30 days
3. Transplant related mortality
The TRM probability compared with historical control.
Time frame: 1 year
4. Acute Graft Versus Host Diseases
Cumulative Incidence of aGVHD
Time frame: 100 days
5. Incidence of Plerixafor related toxicity
severity, features, incidence
Time frame: 100 days
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