Tacrolimus Blood Concentration and Transplant-related Outcomes in Pediatric HSCT Recipients

Graft Vs Host Disease Clinical Trial

Official title:

Factors Affecting the Tacrolimus Blood Concentration and Its Impact on Transplant-related Outcomes in Pediatric HSCT Recipients: a Single-center Retrospective Study

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06080490
• Other study ID #: GEN/INT 0001973
• Status: Recruiting
• Start date: September 29, 2023
• Est. completion date: September 29, 2025

Study information

• Verified date: October 2023
• Source: University of Pisa
• Contact: Natalia Maximova, MD
• Phone: 040.3785.111
• Email: natalia.maximova[@]burlo.trieste.it
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The goal of this retrospective observational study is to investigate any possible association among tacrolimus (TAC) blood concentrations, clinical efficacy and tolerability.

Therefore, the main questions it aims to answer are:

1. to clarify which variables, how and to what extent influence daily TAC blood concentrations in pediatric allo-hematopoietic stem cell transplantation (HSCT) recipients;

2. to investigate the incidence of graft-versus-host disease (GVHD) and graft failure according to TAC exposure.

Pediatric patients administered TAC to prevent GVHD after an allogeneic bone marrow transplantation.

Description:

Patients undergoing allo-HSCT will be recruited at IRCC Burlo Garofolo, collecting demographic and clinical characteristics from electronic medical records in a retrospective manner: age, sex, primary disease, donor type and stem cell source, body weight (BW), white blood cell (WBC) count, platelet count, red blood cell (RBC) counts, hemoglobin (HGB) and hematocrit (HCT) levels, red cell concentrate (RCC) transfusions. Other clinical laboratory data (i.e., liver and kidney function) will be included, as well as concomitant drugs administered to patients after allo-HSCT (i.e, mycophenolate mofetil, steroids, pantoprazole, voriconazole, methotrexate). Data will be collected, validated, and presented according to Good Clinical Practice (GCP) principles to support the statistical analyses and subsequent reporting. Once selected according to predefined inclusion requirements, data will be collected anonymously; an identification code will be assigned to each case. As per GCP, all collected information will be recorded, handled, and stored in a way that allows its accurate reporting, interpretation, and verification with user access restriction, and data will be retrievable by designated personnel only.

Because of the retrospective nature of the present research protocol, all eligible patients will be included in the study population only if their medical and laboratory records will be complete.

Statistical analyses (for descriptive and comparative aims) will be performed stratifying patients by body weight, age, gender, and source of stem cells. Univariate and multivariate tests will be used, together with Kaplan-Meyer tes, Cox proportional hazard model and ROC curve analysis.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: September 29, 2025
• Est. primary completion date: September 29, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Month to 18 Years

Eligibility

Inclusion Criteria:

• Age of the patients between 0 and 18 years (pediatric)

• Patients undergoing allogeneic bone marrow transplantation

• Patients' GVHD prophylaxis with tacrolimus

• Signed informed consent

Exclusion Criteria:

• Patients undergoing autologous bone marrow transplantation

• Patients not undergoing GVHD prophylaxis

• Patients undergoing cyclosporin prophylaxis

• Incomplete pharmacokinetic/pharmacodynamic data

• Lack of any informed consent

Related Conditions & MeSH terms

• Graft Vs Host Disease
• Stem Cell Transplant Complications
• Transplant Failure

Intervention

Drug:
• TAC C/D ratio
Pharmacokinetics of TAC according to possible influencing factors

Locations

Country	Name	City	State
Italy	IRCCS Burlo Garofolo	Trieste

Sponsors (1)

Lead Sponsor	Collaborator
University of Pisa

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	TAC pharmacokinetics	Evaluation of factors affecting TAC blood concentration/dose (C/D) ratio	0-30 days
Secondary	GVHD	Evaluation of GVHD in patients according to TAC C/D	0-180 days
Secondary	Graft failure	Evaluation of graft failure in patients according to TAC C/D	0-180 days