Clinical trial NCT04869254: Lymphocyte Count, ATG Dose and Incidence and Severity of GVHD in Pediatric Recipients of HSCT
Official title: Correlation Between the Pre-transplantation Lymphocyte Count, Administered Dose of Thymoglobulin and Graft Versus Host Disease (GVHD) in Pediatric Recipients of Hematopoietic Stem Cells Transplantation: a Retrospective Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hematopoietic Stem Cell Transplantation
Keywords: Gvhd prophylaxis
pediatric patients
hematopoietic cell transplantation
Lead sponsor: IRCCS Burlo Garofolo
Sponsor class: OTHER
First posted: 2021-05-03
Enrollment: 102
Sex: ALL
Minimum age (years): 1
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Pediatric recipients of hematopoietic stem cells 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.


Eligibility criteria: 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

Primary outcomes:
measure: Development of GVHD according to lymphocyte/ATG ratio
timeFrame: 24 months after transplant
Secondary outcomes:
measure: Transplant related mortality according to lymphocyte/ATG ratio
timeFrame: 24 months after transplant
measure: Incidence of Graft Failure according to lymphocyte/ATG ratio
timeFrame: 24 months after transplant
measure: Number of episodes of sepsis during the post-transplant period according to lymphocyte/ATG ratio
timeFrame: 24 months after transplant
measure: Number of episodes of fungal infections during the post transplant period according to lymphocyte/ATG ratio
timeFrame: 24 months after transplant
measure: Number of episodes of viral reactivations during the post transplant period according to lymphocyte/ATG ratio
timeFrame: 24 months after transplant
measure: Entity of GVHD according to lymphocyte/ATG ratio
timeFrame: 24 months after transplant
Other outcomes:
Locations:
city: Trieste
country: Italy
facility: Institute for Maternal and Child Health - IRCCS "Burlo Garofolo"
Study officials:
affiliation: IRCCS Burlo Garofolo
name: Natalia Maximova, MD
role: STUDY_CHAIR
affiliation: IRCCS Burlo Garofolo
name: Alessandra Maestro, PharmD PhD
role: STUDY_DIRECTOR
References: