Study
Multi-Center Retrospective Study of Iron Overload in Children Receiving HSCT for Non-Malignant Blood Disorders
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Funding
This research was made possible through the generous support of Children's Hospital of Philadelphia.
Creative Commons license
Except where otherwised noted, this item's license is described as a CC-BY 4.0 Attribution license.
Publisher
PEDSnet
Project Description
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.
Study Aims
- To determine the prevalence and risk factors for pre- and post-HSCT iron overload.
- To analyze the effects of elevated liver iron concentration (LIC) and ferritin on transplant outcomes, such as survival rates, graft failure, and immune recovery.
- 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.