Lymphocyte Count, ATG Dose and Incidence and Severity of GVHD in Pediatric Recipients of HSCT

NCT04869254 | Completed

• 1 other identifier: RC 08/2019
• Study Type: observational
• Target: 102
• Locations: 1 country
• Sites: 1

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.

Conditions

Hematopoietic Stem Cell Transplantation

Keywords

Gvhd prophylaxis; pediatric patients; hematopoietic cell transplantation

Outcome Measures

Primary Outcomes (1)

• Development of GVHD according to lymphocyte/ATG ratio

  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

  24 months after transplant

Secondary Outcomes (6)

• Transplant related mortality according to lymphocyte/ATG ratio

  24 months after transplant

• Incidence of Graft Failure according to lymphocyte/ATG ratio

  24 months after transplant

• Number of episodes of sepsis during the post-transplant period according to lymphocyte/ATG ratio

  24 months after transplant

• Number of episodes of fungal infections during the post transplant period according to lymphocyte/ATG ratio

  24 months after transplant

• Number of episodes of viral reactivations during the post transplant period according to lymphocyte/ATG ratio

  24 months after transplant

Study Arms (1)

Hematopoietic stem cells transplantation

patients undergone allogeneic or autologous bone marrow transplant who received immunomodulatory therapy with Thymoglobulin

Other: Lower lymphocyte/ATG ratio; Other: Higher lymphocyte/ATG ratio

Interventions

Lower lymphocyte/ATG ratio OTHER

Also known as: Lymphocyte/ATG ratio <0.01

Hematopoietic stem cells transplantation

Higher lymphocyte/ATG ratio OTHER

Also known as: Lymphocyte/ATG ratio >0.01

Hematopoietic stem cells transplantation

Eligibility Criteria

• Age: 1 Day - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Pediatric recipients of hematopoietic stem cells transplantation

You may qualify if:

• Age of the patients between 0 and 17
• Diagnosis of hematological or oncological disease undergoing allogeneic bone marrow transplantation
• Patients undergoing myeloablative conditioning
• Patients that received ATG as GVHD prophylaxis
• Patients whose consent has already been acquired for the processing of data for research purposes
• Minimum follow-up of 12 months

You may not qualify if:

• Bacterial and / or fungal infection present at the time of bone marrow transplantation
• Use of Thymoglobulin in the 3 months prior to transplantation
• Allergy and / or intolerance to the active substances or excipients contained in Thymoglobulin

Sponsors & Collaborators

• IRCCS Burlo Garofolo: lead

Study Sites (1)

Institute for Maternal and Child Health - IRCCS "Burlo Garofolo"

Trieste, 34137, Italy

Location

Study Officials

• Natalia Maximova, MD

  IRCCS Burlo Garofolo

  STUDY CHAIR

• Alessandra Maestro, PharmD PhD

  IRCCS Burlo Garofolo

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 28, 2021
• First Posted: May 3, 2021
• Study Start: January 1, 2000
• Primary Completion: December 31, 2018
• Study Completion: December 31, 2020
• Last Updated: May 3, 2021

Record last verified: 2021-04

Locations

IT (1)