Changes in Pituitary Iron and Volume With Deferasirox

Changes in Pituitary Iron and Volume With Deferasirox in Transfusional Iron Overload

Despite continuing advances in iron chelation therapy, iron toxicity of endocrine glands, particularly the pituitary gland, remains common in patients with transfusion dependent anemias. We would like to establish accurate population norms of pituitary R2 and volume and understand the progression of pituitary iron in transfused patients on Deferasirox.

Study Overview

• Status: Completed
• Conditions: Iron Overload

• Study Type: Observational
• Enrollment (Actual): 130

Contacts and Locations

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90027
      • Children's Hospital Los Angeles

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 25 years (Child, Adult)
• Accepts Healthy Volunteers: Yes

• Sampling Method: Non-Probability Sample

Study Population

Transfusion Dependent Anemia

Description

Inclusion Criteria:

• Currently on chronic transfusion therapy.
• Duration of chronic transfusion >1 year.
• Age 2 to 25 years
• On deferasirox monotherapy for the duration of the study.
• Informed consent from legal guardian and/or patient.
• On deferasirox for a minimum of 3 months at start of study.

Exclusion Criteria:

• Sickle cell disease or sickle-beta zero genotype.
• Combination of deferasirox and another iron chelator.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
Chronically Transfusion Patients; Patients with transfusion dependent anemia (excluding sickle cell disease), ages 2-25, on Deferasirox chelation therapy, to be monitored over 2 years.
Controls; Normal controls, ages 2-25, with no known brain abnormality or endocrine dysfunction.

Collaborators and Investigators

• Sponsor: Children's Hospital Los Angeles
• Collaborators: Novartis Pharmaceuticals
• Investigators: Principal Investigator: John C Wood, MD, PhD, Children's Hospital Los Angeles

Publications and helpful links

General Publications

• Wood JC, Noetzl L, Hyderi A, Joukar M, Coates T, Mittelman S. Predicting pituitary iron and endocrine dysfunction. Ann N Y Acad Sci. 2010 Aug;1202:123-8. doi: 10.1111/j.1749-6632.2010.05545.x.

Study record dates

Study Major Dates

• Study Start: November 1, 2008
• Primary Completion (Actual): July 1, 2011
• Study Completion (Actual): July 1, 2011

Study Registration Dates

• First Submitted: June 17, 2011
• First Submitted That Met QC Criteria: June 17, 2011
• First Posted (Estimated): June 20, 2011

Study Record Updates

• Last Update Posted (Actual): October 31, 2023
• Last Update Submitted That Met QC Criteria: October 27, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Iron Metabolism Disorders; Iron Overload
• Other Study ID Numbers: CCI-08-00143 (Committee on Clinical Investigations)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No