NCT01201135

GDF 15 in Sickle Cell Disease and Hereditary Spherocytosis

The Impact of Growth Differentiating Factor (GDF) 15 in Sickle Cell Disease and Hereditary Spherocytosis

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 80 (estimated)
• Sponsor: Wolfson Medical Center · Other Government
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

Patients with thalassemia intermedia, congenital dyserythropoietic anemia type I , and sideroblastic anemia were found to express very high levels of serum GDF15, and this contributed to the inappropriate suppression of hepcidin with subsequent secondary iron overload.The aim of our present study is to asses the levels of GDF15 and hepcidin in patients with Sickle cell disease and hereditary spherocytosis

Detailed description

The identification of the ferroportin/hepcidin axis has allowed the effect of erythroid activity on iron balance to be studied and has created the basis for better defining the erythroid regulators. In iron-loading anemias, ineffective erythropoiesis suppresses hepcidin production, which result in dysregulating iron homeostasis. Miller and co-workers showed that release of cytokines like growth differentiation factor 15 (GDF15) during the process of ineffective erythropoiesis inhibits hepcidin production, thus defining a molecular link between ineffective erythropoiesis, suppression of hepcidin production and parenchymal iron loading.

Conditions

• Patients With Thalassemia Intermedia,
• Congenital Dyserythropoietic Anemia Type I

Timeline

• Start date: 2010-09-01
• Primary completion: 2011-09-01
• Completion: 2011-09-01
• First posted: 2010-09-14
• Last updated: 2010-09-14