DFODFPTM: Intensive Combined Chelation Therapy for Iron-Induced Cardiac Disease in Patients With Thalassemia Major
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 4 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Active Comparator: Deferoxamine alone comparison of deferoxamine subcutaneous 40mg/kg/die alone versus combined therapy deferoxamine-deferiprone |
Drug: 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
Drug: Deferoxamine
deferoxamine vials,40 mg/kg,12 hours/die
|
| Active Comparator: Deferoxamine plus 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 |
Drug: 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
|
Outcome Measures
Primary Outcome Measures
- 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
- monitor the left ventricular ejection fraction (LVEF) and serum ferritin levels for evidence of improvement. [42 months]
Eligibility Criteria
Criteria
Inclusion Criteria:
Cardiomyopathy secondary to iron overload
Exclusion Criteria:
Heart failure
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Adult Talassemic Center, Ospedale Microcitemico | Cagliari | Sardinia | Italy | 09121 |
Sponsors and Collaborators
- Ospedale Microcitemico
- Azienda Sanitaria Locale di Cagliari
Investigators
- Study Director: Maria E Lai, MD, Department of Internal Medicine, University of Cagliari-Italy
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- DFO-DFP in TM
- DFOplusDFPLAI