This study focuses on iron overload in pediatric patients undergoing hematopoietic stem cell transplantation (HSCT) for non-malignant blood disorders (NMBD). In recent years, HSCT for NMBD has increased, but little data exists on how iron overload, often caused by blood transfusions, impacts post-transplant outcomes. The study aims to assess the prevalence of iron overload in these patients, its impact on clinical outcomes, and the effectiveness of different post-transplant iron reduction therapies.
The primary goals of the study are:
1. To determine the prevalence and risk factors for pre- and post-HSCT iron overload.
2. To analyze the effects of elevated liver iron concentration (LIC) and ferritin on transplant outcomes, such as survival rates, graft failure, and immune recovery.
3. To compare the effectiveness of phlebotomy versus iron-chelating medications for reducing post-transplant iron overload.
By addressing these gaps, this study aims to generate data that can improve clinical decision-making for children undergoing HSCT, particularly those from minoritized racial and ethnic groups, and establish an evidence-based standard of care for managing iron overload.
Defining the epidemiology and impacts of iron overload in children undergoing bone marrow transplants for hematological disorders.
Funding Source:
Participating PEDSnet Sites:
- Cincinnati Children’s Hospital Medical Center
- Children’s Hospital of Philadelphia
- Children’s National Hospital
- Ann & Robert H. Lurie Children’s Hospital of Chicago
- Nationwide Children’s Hospital
- Seattle Children’s Hospital
- Nemours Children’s Health
- Texas Children’s Hospital
Study Investigator:
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