Changes in Pituitary Iron and Volume With Deferasirox

Children's Hospital Los Angeles

Status: Completed

Conditions: Iron Overload

Study type: Observational

Funder types: Other; Industry

Identifiers

NCT01376622

CCI-08-00143 (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 130 patients

Sex: All

Ages: 2 to 25 years old

Volunteers: Accepts Healthy Volunteers

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.

Trial design

130 participants in 2 patient groups

Chronically Transfusion Patients

Description:

Patients with transfusion dependent anemia (excluding sickle cell disease), ages 2-25, on Deferasirox chelation therapy, to be monitored over 2 years.

Controls

Description:

Normal controls, ages 2-25, with no known brain abnormality or endocrine dysfunction.