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

Ospedale Microcitemico

Status and phase: Completed; Phase 4

Conditions: Cardiomyopathy; Iron Overload

Treatments: Drug: Deferoxamine and Deferiprone; Drug: Deferoxamine

Study type: Interventional

Funder types: Other

Identifiers

NCT00800761

DFOplusDFPLAI

DFO-DFP in TM

Details and patient eligibility

About

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.

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

Cardiomyopathy secondary to iron overload

Exclusion criteria

Heart failure

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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0 participants in 2 patient groups

Deferoxamine alone

Active Comparator group

Description:

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

Treatment:

Drug: Deferoxamine and Deferiprone

Drug: Deferoxamine

Deferoxamine plus Deferiprone

Active Comparator group

Description:

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

Treatment:

Drug: Deferoxamine and Deferiprone