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

June 2, 2025 updated by: Antonello Di Paolo, M.D., Ph.D., University of Pisa

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed 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.

Study Type

Observational

Enrollment (Actual)

125

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Italy
      • Trieste, Italy, 34137
        • IRCCS Burlo Garofolo

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patients who underwent allo-HSCT procedure between January 2011and December 2022 at IRCC Burlo Garofolo

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
TAC pharmacokinetics
Time Frame: 0-30 days
Evaluation of factors affecting TAC blood concentration/dose (C/D) ratio
0-30 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
GVHD
Time Frame: 0-180 days
Evaluation of GVHD in patients according to TAC C/D
0-180 days
Graft failure
Time Frame: 0-180 days
Evaluation of graft failure in patients according to TAC C/D
0-180 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Pisa

Investigators

  • Principal Investigator: Natalia Maximova, MD, IRCCS Burlo Garofolo - Trieste - ITALY

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 29, 2023

Primary Completion (Actual)

September 29, 2024

Study Completion (Actual)

December 31, 2024

Study Registration Dates

First Submitted

October 6, 2023

First Submitted That Met QC Criteria

October 6, 2023

First Posted (Actual)

October 12, 2023

Study Record Updates

Last Update Posted (Estimated)

June 5, 2025

Last Update Submitted That Met QC Criteria

June 2, 2025

Last Verified

June 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GEN/INT 0001973

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

IPD Plan Description

IPD sharing plan is under evaluation by local IRB

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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