Objective: Chronic blood transfusion therapy reduces clinical events in children with sickle cell anemia but increases risk for an iron-related liver injury. Liver biopsy is the gold standard technique for quantifying liver iron content (LIC) and evaluating liver pathology. Ferritin, liver enzymes, and R2* magnetic resonance imaging of the liver are obtained as surrogate markers. In this study we compared surrogate markers with the gold standard, liver biopsy, in assessing liver histology.
Methods: We conducted a retrospective review of 259 liver biopsies in 109 children with sickle cell anemia on chronic transfusion therapy and chelation therapy during a 9-year period at a single center. Liver pathology was compared with LIC, ferritin, and alanine aminotransferase.
Results: Ferritin correlates with LIC (r = 0.74, P < 0.001), although there is a broad range of ferritin values for a given LIC. Furthermore, patients with a high LIC (≥7 mg Fe/g dry weight) demonstrated significantly higher ferritin as compared to the patients with lower LIC <7 (P < 0.001). Periportal/portal inflammation also showed a significant relation. There was no significance when comparing ferritin and lobular inflammation or ferritin and alanine aminotransferase. When evaluating LIC in relation to fibrosis, the present study revealed that there was only a significant correlation with severe fibrosis (F = 36, P < 0.001).
Conclusions: The results suggest that although correlations exist among ferritin and LIC and severe fibrosis and LIC, caution should be taken when they are used in isolation. Liver biopsy provides important pathologic information that cannot be obtained through surrogate markers.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1097/MPG.0000000000000235 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Knowledge, Attitude, and Practice of Patients Suffering From Sickle Cell Disease in an Endemic Zone.
J Pediatr Hematol Oncol
January 2025
Medical Oncology, Haemato-Oncology, BMT, Sarvodaya Hospital, Faridabad, Haryana, India.
Background And Objective: Sickle cell disease (SCD) is a genetically inherited disorder that is associated with morbidity and mortality.
Methods: This cross-sectional study was conducted on patients diagnosed with SCD to evaluate the knowledge, attitude, and practice of patients/guardians using a pretested questionnaire.
Results And Discussion: Of the 111 participants, 56 (50.
Major ABO Incompatibility in Non-Myeloablative Hematopoietic Stem Cell Transplant for Sickle Cell Disease-Not an Insurmountable Obstacle.
Pediatr Blood Cancer
January 2025
Blood and Marrow Transplant/Cellular Therapy Program, Division of Hematology/Oncology, The Hospital for Sick Children, Toronto, Ontario, Canada.
With advances in conditioning strategies and graft-versus-host disease (GvHD) prevention, hematopoietic stem cell transplantation (HSCT) is a safe, curative treatment option for pediatric patients with sickle cell disease (SCD). However, donor options have been limited in non-myeloablative matched sibling donor (MSD) setting by excluding recipients with major ABO blood group incompatible donors due to concern of the risk of significant complications such as pure red cell aplasia (PRCA). We present three cases of successful HSCT with major ABO incompatibility with their donors, and discuss strategies to safely expand the donor pool to include these donors.
View Article and Find Full Text PDFRespiratory manifestations of sickle cell disease in children: a comprehensive review for the pediatrician.
Expert Rev Respir Med
January 2025
Department of Surgery, Dentistry, Pediatrics and Gynaecology, Pediatric Division, University of Verona, Verona, Italy.
Introduction: Sickle cell disease (SCD) is an inherited hemoglobinopathy characterized by the production of sickle hemoglobin, leading to red blood cells sickling and hemolysis in hypoxic conditions. The resulting acute and chronic endothelial inflammation leads to chronic organ damage. Respiratory manifestations in SCD usually start from childhood and represent the leading causes of morbidity and mortality.
View Article and Find Full Text PDFThe modern use of hydroxyurea for children with sickle cell anemia.
Haematologica
January 2025
Division of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati OH; University of Cincinnati College of Medicine, Cincinnati OH; Global Health Center, Cincinnati Children's Hospital Medical Center, Cincinnati OH.
Over the past 40 years, the introduction and refinement of hydroxyurea therapy has led to remarkable progress for the care of individuals with sickle cell anemia (SCA). From initial small proof-of-principle studies to multi-center Phase 3 controlled clinical trials and then numerous open-label studies, the consistent benefits of once-daily oral hydroxyurea have been demonstrated across the lifespan. Elevated fetal hemoglobin (HbF) serves as the most important treatment response, as HbF delays sickle hemoglobin polymerization and reduces erythrocyte sickling.
View Article and Find Full Text PDFSuccessful Treatment of Bone Marrow Necrosis and Fat Embolism Syndrome Without Long-Term Sequelae in a Young Adult with Sickle Cell Disease: A Case Report and Recommendations for Diagnosis and Management.
Pediatr Blood Cancer
January 2025
Division of Pediatric Hematology/Oncology, Hasbro Children's Hospital, Providence, Rhode Island, USA.
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!