Hematopoietic Stem Cell Transplantation
Conditions
Keywords
Gvhd prophylaxis, pediatric patients, hematopoietic cell transplantation
Brief summary
Despite increasing success rate in hematopoietic stem cell transplantation (HSCT) control of graft versus host disease (GVHD) remains a significative burden in mortality and morbidity. A lot of strategies could lower the incidence and gravity of the disease and immunosuppressive treatment as GVHD prophylaxis still represent the main method. Although immunosuppressive treatment showed a good effect on GVHD mortality a lot of studies also highlight an increase of relapse and infection related mortality that jeopardize the effect on overall survival of HSCT recipient. Using anti thymocyte globulin (ATG) as GVHD prophylaxis shares the same double-edge effect as other immunosuppressive treatment although is still unclear how manage dose and timing of the infusion to minimize promoting effect on infections and maximize protective effect on GVHD. Biological effect of ATG lead to a dose- related delay in all class of T-cell reconstitution but our data are mostly from adult studies with high doses between 30 and 60 mg/kg due to the more important burden of GVHD in HSCT adult population. As for other treatment in HSCT conditioning we would like to study a personalized approach for ATG treatment: some studies focus on tuning of ATG dose for kilos but previous evidence showed that the same dose could made too little or too much immunosuppressive effect for different patients, even though same age and same stem cell source.
Interventions
OTHERLower lymphocyte/ATG ratio
OTHERHigher lymphocyte/ATG ratio
Sponsors
IRCCS Burlo Garofolo
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
1 Days to 17 Years
Healthy volunteers
No
Inclusion criteria
1. Age of the patients between 0 and 17 2. Diagnosis of hematological or oncological disease undergoing allogeneic bone marrow transplantation 3. Patients undergoing myeloablative conditioning 4. Patients that received ATG as GVHD prophylaxis 5. Patients whose consent has already been acquired for the processing of data for research purposes 6. Minimum follow-up of 12 months
Exclusion criteria
1. Bacterial and / or fungal infection present at the time of bone marrow transplantation 2. Use of Thymoglobulin in the 3 months prior to transplantation 3. Allergy and / or intolerance to the active substances or excipients contained in Thymoglobulin
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Development of GVHD according to lymphocyte/ATG ratio | 24 months after transplant | Differences in the frequency of GVHD according to the two exposure's groups (lymphocyte/ATG ratio \<0.01 vs \>0.01). Adjustment for potentially associated variables (e.g., dose of drug actually received by patients, age of patients, type of conditioning, underlying disease, type of donor) will be carried out |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Transplant related mortality according to lymphocyte/ATG ratio | 24 months after transplant | Comparison of transplant related mortality 24 months after HSCT in the two exposure's groups (lymphocyte/ATG ratio \<0.01 vs \>0.01) |
| Incidence of Graft Failure according to lymphocyte/ATG ratio | 24 months after transplant | Frequency of engraftment failure and hematological reconstitution time (defined as the first post-transplant day of at least three consecutive days with the presence of at least 500 neutrophils / mmc for myeloid engraftment and the first appearance of lymphoid cell subpopulations) in the two exposure's groups (lymphocyte/ATG ratio \<0.01 vs \>0.01) |
| Number of episodes of sepsis during the post-transplant period according to lymphocyte/ATG ratio | 24 months after transplant | Number of episodes of sepsis after HSCT and correlated mortality and morbidity in the two exposure's groups (lymphocyte/ATG ratio \<0.01 vs \>0.01) |
| Number of episodes of fungal infections during the post transplant period according to lymphocyte/ATG ratio | 24 months after transplant | Number of episodes of fungal infections after HSCT and correlated mortality and morbidity in the two exposure's groups (lymphocyte/ATG ratio \<0.01 vs \>0.01) |
| Number of episodes of viral reactivations during the post transplant period according to lymphocyte/ATG ratio | 24 months after transplant | Number of episodes of viral reactivations after HSCT and correlated mortality and morbidity in the two exposure's groups (lymphocyte/ATG ratio \<0.01 vs \>0.01) |
| Entity of GVHD according to lymphocyte/ATG ratio | 24 months after transplant | Severity and organ involvement of acute and chronic GVHD defined by the Glucksberg classification by degrees of severity in the two exposure's groups (lymphocyte/ATG ratio \<0.01 vs \>0.01) |
Countries
Italy
Outcome results
None listed