Endocrine Function During Deferasirox Therapy

National Survey on Endocrine Function and Bone Metabolism in Transfusion--dependent Patients Treated With Deferasirox

A national survey on the prevalence and natural history of endocrine complications in thalassemia transfusion--dependent patients treated with deferasirox was designed, in order to assess a larger population during a longer follow up and improve the quality of previous investigations.

Study Overview

• Status: Completed
• Conditions: Iron Overload; Endocrine; Complications; Transfusion-dependent Thalassemia

• Study Type: Observational
• Enrollment (Actual): 426

Contacts and Locations

Study Locations

• Italy
  • Naples, Italy, 80138
    • Università degli studi della Campania Luigi Vanvitelli

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

All consecutive patients visited at the participating sites since September 2009 were recruited into the cohort provided the inclusion criteria were met: affected by TDT and on deferasirox monotherapy during the study period.

Description

Inclusion Criteria:

• Adult and pediatric patients with transfusion---dependent thalassemia;
• Chelation with deferasirox as assigned chelation therapy;
• Available medical history including relevant clinical data (age at start of transfusion regimen, age at start of chelation therapy, prior chelation therapy, concomitant diseases and concomitant treatments, including hormonal replacement treatments if appropriate) and laboratory data (e.g TSH, FT3 and FT4, fasting serum glucose, OGTT serum glucose, bone mineral density z---score, T---score, g/cm2, PTH, FSH, LH, testosterone and estradiol, serum ferritin, liver function tests,renal function tests, MRI T2* value) at baseline and at the end of study

Exclusion Criteria:

• Non transfusion- dependent patients;
• Other chelation therapy than deferasirox or combination with other chelators during the observation;
• Absence of complete medical history as above specified

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline number of endocrine disorders at study completion	Absolute change in number of patients diagnosed with any endocrine disorder at the baseline and at the study completion	baseline through study completion, a minimum of 5 years

Collaborators and Investigators

• Sponsor: University of Campania "Luigi Vanvitelli"

Study record dates

Study Major Dates

• Study Start (Actual): November 2, 2016
• Primary Completion (Actual): January 30, 2019
• Study Completion (Actual): January 30, 2019

Study Registration Dates

• First Submitted: August 6, 2020
• First Submitted That Met QC Criteria: August 13, 2020
• First Posted (Actual): August 17, 2020

Study Record Updates

• Last Update Posted (Actual): August 17, 2020
• Last Update Submitted That Met QC Criteria: August 13, 2020
• Last Verified: August 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Iron Metabolism Disorders; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Iron Overload; Thalassemia
• Other Study ID Numbers: CICL670AIT16T

Drug and device information, study documents

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