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

Sponsors and Collaborators:	Ospedale Microcitemico ASL - 8 - Cagliari,
Information provided by:	Ospedale Microcitemico
ClinicalTrials.gov Identifier:	NCT00800761

Purpose

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	Intervention	Phase
Iron Overload Cardiomyopathy	Drug: Deferoxamine and Deferiprone Drug: Deferoxamine	Phase IV

Genetics Home Reference related topics: beta thalassemia

MedlinePlus related topics: Cardiomyopathy Heart Diseases Thalassemia

Drug Information available for: Deferoxamine 1,2-Dimethyl-3-hydroxypyrid-4-one Deferoxamine mesylate

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Parallel Assignment, Efficacy Study
Official Title:	Increased Survival and Reversion of Iron-Induced Cardiac Disease in Patients With Thalassemia Major Receiving Intensive Combined Chelation Therapy

Further study details as provided by Ospedale Microcitemico:

Primary Outcome Measures:

Our primary objective: to assess the prevalence of cardiovascular deaths and hospitalisations for cardiovascular disease in the 2 treatment groups [ Time Frame: 42 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

monitor the left ventricular ejection fraction (LVEF) and serum ferritin levels for evidence of improvement. [ Time Frame: 42 months ] [ Designated as safety issue: Yes ]

Study Start Date:	December 2001
Study Completion Date:	June 2006
Primary Completion Date:	June 2006 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Deferoxamine alone: Active Comparator 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
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 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

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Cardiomyopathy secondary to iron overload

Exclusion Criteria:

Heart failure

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00800761

Locations

Italy, Sardinia
Adult Talassemic Center, Ospedale Microcitemico
Cagliari, Sardinia, Italy, 09121

Sponsors and Collaborators

Ospedale Microcitemico

ASL - 8 - Cagliari,

Investigators

Study Director:

Maria E Lai, MD

Department of Internal Medicine, University of Cagliari-Italy

More Information

No publications provided

Responsible Party:	University ( Maria Eliana Lai, Prof, MD, Adult Thalassemic Center, Director )
Study ID Numbers:	DFO-DFP in TM, DFOplusDFPLAI
Study First Received:	December 1, 2008
Last Updated:	December 1, 2008
ClinicalTrials.gov Identifier:	NCT00800761 History of Changes
Health Authority:	Italy: Ethics Committee

Study placed in the following topic categories:

Heart Diseases
Metabolic Diseases
Hematologic Diseases
Deferiprone
Beta-thalassemia
Anemia
Anemia, Hemolytic
Iron Metabolism Disorders
Cardiomyopathies
Thalassemia
Anemia, Hemolytic, Congenital

Thalassemia Minor
Genetic Diseases, Inborn
Beta-Thalassemia
Hemoglobinopathies
Chelating Agents
Iron Overload
Hemoglobinopathy
Iron
Metabolic Disorder
Deferoxamine

Additional relevant MeSH terms:

Metabolic Diseases
Heart Diseases
Molecular Mechanisms of Pharmacological Action
Hematologic Diseases
Deferiprone
Iron Chelating Agents
Anemia
Anemia, Hemolytic
Iron Metabolism Disorders
Thalassemia
Cardiomyopathies

Pharmacologic Actions
Siderophores
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Beta-Thalassemia
Hemoglobinopathies
Cardiovascular Diseases
Chelating Agents
Iron Overload
Deferoxamine

ClinicalTrials.gov processed this record on May 07, 2009