Preventing liver and pancreas complications in children after stem cell transplants
Identification of Risk Factors and Measures to Prevent Liver and Pancreas Complications in Pediatric Patients Undergoing a Hematopoietic Stem Cell Transplant (HSCT)
This study looks at children who had stem cell transplants to see how we can prevent liver and pancreas problems after their treatment.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 39 (estimated) |
| Ages | N/A to 17 Years |
| Sex | All |
| Sponsor | University of Pisa Academic / other |
| Locations | 1 site (Trieste) |
| Trial ID | NCT04423237 on ClinicalTrials.gov |
What this trial studies
This observational study focuses on pediatric patients who have undergone hematopoietic stem cell transplantation (HSCT) to assess the risk factors and preventive measures for liver and pancreas complications. It will collect retrospective data from patients treated between 2010 and 2018, including their underlying diseases, pre- and post-transplant liver and pancreatic function, and iron content levels. The study aims to analyze the tolerability and effectiveness of deferasirox treatment in managing iron overload and its associated risks. Data will be processed using statistical analyses to identify significant trends and outcomes related to these complications.
Who should consider this trial
Good fit: Ideal candidates include pediatric patients who have undergone one or more allogeneic HSCTs and have moderate-to-severe iron overload requiring treatment.
Not a fit: Patients who do not have liver biopsies or complete laboratory analysis results post-transplant may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved management strategies for preventing liver and pancreas complications in pediatric HSCT patients.
How similar studies have performed: Other studies have shown success in managing iron overload in HSCT patients, but this specific approach is novel in its comprehensive retrospective analysis.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * one or more allogeneic HSCT * any type of disease (blood-oncological or genetic), from any type of donor (sibling, MUD, haploidentical) and with any source of stem cells (spinal cord, peripheral blood stem cells, cord blood) * diagnosis of moderate-to-severe siderosis (by nuclear magnetic resonance imaging, MRI) and who needed a chelation treatment with deferasirox * one or more liver biopsies in the post-transplant period to perform histological examinations * Complete results from lab analyses * 2-year follow-up after HSCT * therapeutic drug monitoring (TDM) protocol for deferasirox plasma concentration as per clinical routine * Sign of the informed consent by the parents or legal representatives Exclusion Criteria: * Lack of liver biopsies after transplantation, results from laboratory analyses or TDM or MRI * Lack of informed consent
Where this trial is running
Trieste
- IRCCS Burlo Garofolo — Trieste, Italy (Recruiting)
Study contacts
- Study coordinator: Natalia Maximova, MD
- Email: natalia.maximova@burlo.trieste.it
- Phone: +39.040.3785.565
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.