GDF 15 in Sickle Cell Disease and Hereditary Spherocytosis (GDF 15)

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

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

Study Overview

• Status: Unknown
• Conditions: Patients With Thalassemia Intermedia,; Congenital Dyserythropoietic Anemia Type I

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.

• Study Type: Observational
• Enrollment (Anticipated): 80

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years and older (ADULT, OLDER_ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

The study will contain 40 patients with Sickle cell disease and 40 patients with hereditary spherocytosis.

After ICF (Informed Consent Form) has been signed by the patients the following laboratory tests will be taken once during the study:

1. GDF 15( 3ml of serum) (at the laboratory of hematology at Wolfson Medical Center/Israel)
2. Hepcidine (3ml of serum) (at the laboratory of Prof. T. Ganz, USA). The blood samples should be taken at least one week apart from blood transfusion.

In case of infection or acute inflammation , blood samples should be taken only one week after resolution of these conditions.

Description

Inclusion Criteria:

• non

Exclusion Criteria:

• non

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
Sickle cell disease
hereditary spherocytosis.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
GDF 15	year

Secondary Outcome Measures

Outcome Measure	Time Frame
Hepcidine	year

Collaborators and Investigators

• Sponsor: Wolfson Medical Center

Study record dates

Study Major Dates

• Study Start: September 1, 2010
• Primary Completion (ANTICIPATED): September 1, 2011
• Study Completion (ANTICIPATED): September 1, 2011

Study Registration Dates

• First Submitted: September 12, 2010
• First Submitted That Met QC Criteria: September 12, 2010
• First Posted (ESTIMATE): September 14, 2010

Study Record Updates

• Last Update Posted (ESTIMATE): September 14, 2010
• Last Update Submitted That Met QC Criteria: September 12, 2010
• Last Verified: September 1, 2010

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell; Thalassemia; beta-Thalassemia; Anemia, Dyserythropoietic, Congenital; Spherocytosis, Hereditary
• Other Study ID Numbers: GDF-15CTIL