Intensive Combined Chelation Therapy for Iron-Induced Cardiac Disease in Patients With Thalassemia Major

Phase 4

Completed

• Conditions: Iron Overload; Cardiomyopathy
• Interventions: Drug: Deferoxamine and Deferiprone; Drug: Deferoxamine

• Registration Number: NCT00800761
• Lead Sponsor: Ospedale Microcitemico

Brief Summary

Myocardial iron overload is the leading cause of death in patients with beta-thalassemia major (TM). Therapy with deferoxamine (DFO) combined with deferiprone (DFP) reduces myocardial iron and improves cardiac function. However, the prognosis for TM patients with established cardiac disease switched from DFO monotherapy to combined DFP/DFO chelation is unknown. Twenty-eight TM patients with cardiac disease were enrolled in a prospective study lasting 42±6 months. Fifteen (9 high-ferritin and 6 low-ferritin) were placed on DFP/DFO (DFP, 75 mg/kg t.i.d.; DFO, 40-50 mg/kg over 8-12 h at night 5-7 d/wk), while 13 (5 high- and 8 low-ferritin) received DFO alone. No cardiac events were observed among high-ferritin patients on combination therapy, whereas 4 cardiac events (p=0.0049), including three deaths, occurred in high-ferritin patients on DFO monotherapy. These findings demonstrate that in TM patients with well-established cardiac disease combined iron-chelation therapy with DFP/DFO is superior to DFO monotherapy.

Detailed Description

Not available

Study Timeline

• Status Verified: 2001-12
• Study Start: 2001-12
• Primary Completion: 2006-06
• Study Completion: 2006-06
• Study First Submitted: 2008-12-01
• Study First Submitted QC: 2008-12-01
• Last Update Submitted: 2008-12-01
• Study First Posted: 2008-12-02
• Last Update Posted: 2008-12-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Cardiomyopathy secondary to iron overload

Exclusion Criteria

Heart failure

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Deferoxamine alone	Deferoxamine and Deferiprone	comparison of deferoxamine subcutaneous 40mg/kg/die alone versus combined therapy deferoxamine-deferiprone
Deferoxamine plus Deferiprone	Deferoxamine and Deferiprone	comparison of two arms: the first one treated with deferoxamine subcutaneous vials,40 mg/kg,12 hours/die plus deferiprone tablets 75 mg/kg three times/die versus the second one treated with deferoxamine subcutaneous vials,40 mg/kg,12 hours/die
Deferoxamine alone	Deferoxamine	comparison of deferoxamine subcutaneous 40mg/kg/die alone versus combined therapy deferoxamine-deferiprone

Primary Outcome Measures

Name	Time	Method
Our primary objective: to assess the prevalence of cardiovascular deaths and hospitalisations for cardiovascular disease in the 2 treatment groups	42 months

Secondary Outcome Measures

Name	Time	Method
monitor the left ventricular ejection fraction (LVEF) and serum ferritin levels for evidence of improvement.	42 months

Trial Locations

Locations (1)

Adult Talassemic Center, Ospedale Microcitemico; 🇮🇹 Cagliari, Sardinia, Italy