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

NCT00800761 | Completed Phase 4

• 2 other identifiers: DFO-DFP in TMDFOplusDFPLAI
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Iron Overload; Cardiomyopathy

Outcome Measures

Primary Outcomes (1)

• Our primary objective: to assess the prevalence of cardiovascular deaths and hospitalisations for cardiovascular disease in the 2 treatment groups

  42 months

Secondary Outcomes (1)

• monitor the left ventricular ejection fraction (LVEF) and serum ferritin levels for evidence of improvement.

  42 months

Study Arms (2)

Deferoxamine alone

ACTIVE COMPARATOR

comparison of deferoxamine subcutaneous 40mg/kg/die alone versus combined therapy deferoxamine-deferiprone

Drug: Deferoxamine and Deferiprone; Drug: Deferoxamine

Deferoxamine plus Deferiprone

ACTIVE COMPARATOR

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

Drug: Deferoxamine and Deferiprone

Interventions

Deferoxamine and Deferiprone DRUG

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 plus Deferiprone

Deferoxamine DRUG

deferoxamine vials,40 mg/kg,12 hours/die

Deferoxamine alone

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Cardiomyopathy secondary to iron overload

You may not qualify if:

• Heart failure

Sponsors & Collaborators

• Ospedale Microcitemico: lead
• Azienda Sanitaria Locale di Cagliari: collaborator

Study Sites (1)

Adult Talassemic Center, Ospedale Microcitemico

Cagliari, Sardinia, 09121, Italy

Location

Related Publications (1)

• Padhani ZA, Gangwani MK, Sadaf A, Hasan B, Colan S, Alvi N, Das JK. Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with transfusion-dependent beta thalassaemia. Cochrane Database Syst Rev. 2023 Nov 17;11(11):CD011626. doi: 10.1002/14651858.CD011626.pub3.

  PMID: 37975597 DERIVED

MeSH Terms

Conditions

Iron Overload; Cardiomyopathies

Interventions

Deferoxamine; Deferiprone

Condition Hierarchy (Ancestors)

Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Heart Diseases; Cardiovascular Diseases

Intervention Hierarchy (Ancestors)

Hydroxamic Acids; Hydroxylamines; Amines; Organic Chemicals; Hydroxy Acids; Carboxylic Acids; Pyridones; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Maria E Lai, MD

  Department of Internal Medicine, University of Cagliari-Italy

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: December 1, 2008
• First Posted: December 2, 2008
• Study Start: December 1, 2001
• Primary Completion: June 1, 2006
• Study Completion: June 1, 2006
• Last Updated: December 2, 2008

Record last verified: 2001-12

Locations

IT (1)