Extension Study of Iron Chelation Therapy With Deferasiro... | NCT00303329

Label	Type	Description	Intervention Names
Deferasirox	Experimental	Deferasirox daily oral dose between 5-40 mg/kg/day	Drug: Deferasirox

Name	Type	Description	Arm Group Labels	Other Names
Deferasirox	Drug	Deferasirox available as 125 mg, 250 mg or 500 mg tablets	Deferasirox

Measure	Description	Time Frame
The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths	Safety was assessed using reports of adverse events of all participants in this study. Serious adverse events are those events that resulted in death, were life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect.	Core study Baseline to the end of the study (up to 60 months)

Measure	Description	Time Frame
The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study	Liver iron concentration was monitored at the start of the core study, the end of the core study, and then at the end of the extension study. High-risk participants, like participants with rare anemia, were excluded from any further potential liver biopsy, except if required and justified by the Investigator for the general care of the participant.

Name	Affiliation	Role
Novartis Pharmaceuticals	Novartis Pharmaceuticals	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Children's Hospital and Research center at Oakland		Oakland	California	94609-1809	United States

ID	Title	Description
FG000	β-thalassemia Patients	Deferasirox (5-40 mg/kg/day)
FG001	Rare Anemias Patients	Deferasirox (5-40 mg/kg/day)

ID	Title	Description
BG000	β-thalassemia Patients	Deferasirox (5-40 mg/kg/day)
BG001	Rare Anemias Patients	Deferasirox (5-40 mg/kg/day)
BG002	Total	Total of all reporting groups

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths

Safety was assessed using reports of adverse events of all participants in this study. Serious adverse events are those events that resulted in death, were life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect.

The safety analysis set comprised all participants who received at least one dose of deferasirox during either the core or extension studies.

Posted

Number

Participants

Core study Baseline to the end of the study (up to 60 months)

ID	Title	Description
OG000	β-thalassemia Patients	Deferasirox (5-40 mg/kg/day)
OG001	Rare Anemias Patients	Deferasirox (5-40 mg/kg/day)

Units	Counts
Participants	OG00085 OG00199

Title

Denominators

Categories

Adverse Events

Title	Measurements
	OG00085 OG00199

Serious Adverse Events

Title	Measurements
	OG000

Secondary

The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study

Liver iron concentration was monitored at the start of the core study, the end of the core study, and then at the end of the extension study. High-risk participants, like participants with rare anemia, were excluded from any further potential liver biopsy, except if required and justified by the Investigator for the general care of the participant.

The full analysis Set (FAS) comprised all participants who received at least one dose of deferasirox during either the core or extension studies.

Posted

Mean

Standard Deviation

mg Fe/g dw

Core study Baseline to end of extension study (up to 60 months)

ID	Title	Description
OG000	β-thalassemia Patients	Deferasirox (5-40 mg/kg/day)
OG001	Rare Anemias Patients	Deferasirox (5-40 mg/kg/day)

Units	Counts
Participants	OG000

Secondary

The Absolute Change in Liver Iron Content (LIC) as Assessed by Superconducting Quantum Interference Device (SQUID) From Baseline to End of Study

Liver iron concentration was monitored at the end of the core study and then at the end of the extension study. High-risk participants, like participants with rare anemia, were excluded from any further potential liver biopsy, except if required and justified by the Investigator for the general care of the participant. Pediatric participants or participants with a medical contraindication to liver biopsy were allowed the use of SQUID in the extension study.

The FAS comprised all participants who received at least one dose of deferasirox during either the core or extension studies.

Posted

Mean

Standard Deviation

mg Fe/g dw

Core study Baseline to end of extension study (up to 60 months)

ID	Title	Description
OG000	β-thalassemia Patients	Deferasirox (5-40 mg/kg/day)
OG001	Rare Anemias Patients	Deferasirox (5-40 mg/kg/day)

Units	Counts
Participants

Secondary

The Absolute Change in Serum Ferritin (μg/L) Levels From Baseline to the End of the Study

Serum ferritin was monitored monthly and the dose of deferasirox was increased or decreased in steps of 5 to 10 mg/kg/day up to a maximum of 40 mg/kg/day if appropriate, every 3 months. If serum ferritin fell to 500 ng/mL or lower on two consecutive study visits, an interruption of treatment until serum ferritin was more than 500 ng/mL was considered.

The FAS comprised all participants who received at least one dose of deferasirox during either the core or extension studies.

Posted

Mean

Standard Deviation

μg/L

Core study Baseline to end of extension study (up to 60 months)

ID	Title	Description
OG000	β-thalassemia Patients	Deferasirox (5-40 mg/kg/day)
OG001	Rare Anemias Patients	Deferasirox (5-40 mg/kg/day)

Units	Counts
Participants	OG000

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Anaemia	Blood and lymphatic system disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0015 affected99 at risk
Disseminated intravascular coagulation	Blood and lymphatic system disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Extramedullary haemopoiesis	Blood and lymphatic system disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Febrile neutropenia	Blood and lymphatic system disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0012 affected99 at risk
Haemolysis	Blood and lymphatic system disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Lymphadenopathy	Blood and lymphatic system disorders	MedDRA	Systematic Assessment	EG0002 affected85 at risk EG0010 affected99 at risk
Neutropenia	Blood and lymphatic system disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0012 affected99 at risk
Pancytopenia	Blood and lymphatic system disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Thrombocytopenia	Blood and lymphatic system disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Angina pectoris	Cardiac disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Arrhythmia	Cardiac disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0011 affected99 at risk
Atrial fibrillation	Cardiac disorders	MedDRA	Systematic Assessment	EG0003 affected85 at risk EG0011 affected99 at risk
Cardiac arrest	Cardiac disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Cardiac disorder	Cardiac disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Cardiac failure	Cardiac disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Cardiac failure congestive	Cardiac disorders	MedDRA	Systematic Assessment	EG0002 affected85 at risk EG0010 affected99 at risk
Cardio-respiratory arrest	Cardiac disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Cardiogenic shock	Cardiac disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Cardiomyopathy	Cardiac disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Left ventricular failure	Cardiac disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Mitral valve incompetence	Cardiac disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Palpitations	Cardiac disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Tachyarrhythmia	Cardiac disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Ventricular fibrillation	Cardiac disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Ventricular tachyarrhythmia	Cardiac disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Sudden hearing loss	Ear and labyrinth disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Tympanic membrane perforation	Ear and labyrinth disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Primary hypogonadism	Endocrine disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Cataract	Eye disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0012 affected99 at risk
Abdominal pain	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0003 affected85 at risk EG0014 affected99 at risk
Constipation	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0012 affected99 at risk
Gastrointestinal haemorrhage	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0012 affected99 at risk
Ileus	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Melaena	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0011 affected99 at risk
Nausea	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0002 affected85 at risk EG0010 affected99 at risk
Varices oesophageal	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Vomiting	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0003 affected85 at risk EG0011 affected99 at risk
Catheter related complication	General disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Chest pain	General disorders	MedDRA	Systematic Assessment	EG0002 affected85 at risk EG0011 affected99 at risk
Chills	General disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0012 affected99 at risk
Drug withdrawal syndrome	General disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
General physical health deterioration	General disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Hyperpyrexia	General disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Malaise	General disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Oedema	General disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Pyrexia	General disorders	MedDRA	Systematic Assessment	EG0003 affected85 at risk EG00112 affected99 at risk
Bile duct stone	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Cholelithiasis	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Cholelithiasis obstructive	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Gallbladder disorder	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Hepatic failure	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Hepatic haemorrhage	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Hepatitis	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0012 affected99 at risk
Abdominal infection	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Anal abscess	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Arthritis infective	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Bacteraemia	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Bacterial sepsis	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0012 affected99 at risk
Bronchiolitis	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Bronchitis	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Bronchopulmonary aspergillosis	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Catheter related infection	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Erysipelas	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Gastroenteritis	Infections and infestations	MedDRA	Systematic Assessment	EG0002 affected85 at risk EG0011 affected99 at risk
Gastroenteritis yersinia	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Haematoma infection	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Hepatitis A	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Infection	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Influenza	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0011 affected99 at risk
Klebsiella infection	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Klebsiella sepsis	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Lobar pneumonia	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Lower respiratory tract infection	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0012 affected99 at risk
Lung infection	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0012 affected99 at risk
Meningitis viral	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Neutropenic sepsis	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Orchitis	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Peridiverticular abscess	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Pharyngitis	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Pharyngitis streptococcal	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Pneumonia	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0015 affected99 at risk
Pseudomonal sepsis	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Pyelonephritis	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Sepsis	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0015 affected99 at risk
Septic shock	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0013 affected99 at risk
Staphylococcal sepsis	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Subcutaneous abscess	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Urosepsis	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Viral infection	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0011 affected99 at risk
Clavicle fracture	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Contusion	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Device failure	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Facial bones fracture	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Fall	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Fibula fracture	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Forearm fracture	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Head injury	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Hepatic haematoma	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Radius fracture	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Rib fracture	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Road traffic accident	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Skin laceration	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Spinal fracture	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0011 affected99 at risk
Splenic rupture	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Subdural haematoma	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Tibia fracture	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Transfusion reaction	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Blood phosphorus increased	Investigations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Electrocardiogram abnormal	Investigations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Neutrophil count decreased	Investigations	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Transaminases increased	Investigations	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0012 affected99 at risk
Anorexia	Metabolism and nutrition disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0012 affected99 at risk
Dehydration	Metabolism and nutrition disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Hyperglycaemia	Metabolism and nutrition disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Hypocalcaemia	Metabolism and nutrition disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Hypoglycaemia	Metabolism and nutrition disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Hypoglycaemic seizure	Metabolism and nutrition disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Hypokalaemia	Metabolism and nutrition disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Hypophosphataemia	Metabolism and nutrition disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Type 1 diabetes mellitus	Metabolism and nutrition disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Back pain	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0011 affected99 at risk
Bone cyst	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Knee deformity	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0002 affected85 at risk EG0010 affected99 at risk
Musculoskeletal chest pain	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Musculoskeletal pain	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Myalgia	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Osteoarthritis	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Acute leukaemia	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0012 affected99 at risk
Myelodysplastic syndrome	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Cerebral haemorrhage	Nervous system disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0012 affected99 at risk
Coma	Nervous system disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Dementia	Nervous system disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Dizziness	Nervous system disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Encephalitis	Nervous system disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Headache	Nervous system disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Hypokinesia	Nervous system disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Loss of consciousness	Nervous system disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Migraine	Nervous system disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Psychomotor hyperactivity	Nervous system disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Sciatica	Nervous system disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Pregnancy	Pregnancy, puerperium and perinatal conditions	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Adjustment disorder	Psychiatric disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Anxiety	Psychiatric disorders	MedDRA	Systematic Assessment	EG0002 affected85 at risk EG0010 affected99 at risk
Delirium	Psychiatric disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Depression	Psychiatric disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0013 affected99 at risk
Illusion	Psychiatric disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Mental disorder	Psychiatric disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Suicidal ideation	Psychiatric disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Suicide attempt	Psychiatric disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Anuria	Renal and urinary disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Calculus bladder	Renal and urinary disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Calculus ureteric	Renal and urinary disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Nephrolithiasis	Renal and urinary disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Renal colic	Renal and urinary disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Renal mass	Renal and urinary disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Urethral haemorrhage	Renal and urinary disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Testicular disorder	Reproductive system and breast disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Testicular torsion	Reproductive system and breast disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Acute respiratory failure	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Dyspnoea	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0012 affected99 at risk
Epistaxis	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Hypoxia	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Nasal congestion	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Pharyngeal haemorrhage	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Pleurisy	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Pneumonitis	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Pulmonary haemorrhage	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Respiratory failure	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Tonsillar hypertrophy	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Rash	Skin and subcutaneous tissue disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0010 affected99 at risk
Stasis dermatitis	Skin and subcutaneous tissue disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Toe amputation	Surgical and medical procedures	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Hypotension	Vascular disorders	MedDRA	Systematic Assessment	EG0001 affected85 at risk EG0011 affected99 at risk
Hypovolaemic shock	Vascular disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk
Poor venous access	Vascular disorders	MedDRA	Systematic Assessment	EG0000 affected85 at risk EG0011 affected99 at risk

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Notes	Statistical Information
Lymphadenopathy	Blood and lymphatic system disorders	MedDRA	Systematic Assessment		EG0005 affected85 at risk EG0011 affected99 at risk
Palpitations	Cardiac disorders	MedDRA	Systematic Assessment		EG00012 affected85 at risk EG0018 affected99 at risk
Tachycardia	Cardiac disorders	MedDRA	Systematic Assessment		EG00012 affected85 at risk EG0016 affected99 at risk
Ear pain	Ear and labyrinth disorders	MedDRA	Systematic Assessment		EG00010 affected85 at risk EG0016 affected99 at risk
Tinnitus	Ear and labyrinth disorders	MedDRA	Systematic Assessment		EG0006 affected85 at risk EG0010 affected99 at risk
Vertigo	Ear and labyrinth disorders	MedDRA	Systematic Assessment		EG0006 affected85 at risk EG0016 affected99 at risk
Vision blurred	Eye disorders	MedDRA	Systematic Assessment		EG0005 affected85 at risk EG0011 affected99 at risk
Abdominal discomfort	Gastrointestinal disorders	MedDRA	Systematic Assessment		EG0007 affected85 at risk EG0013 affected99 at risk
Abdominal distension	Gastrointestinal disorders	MedDRA	Systematic Assessment		EG00010 affected85 at risk EG0015 affected99 at risk
Abdominal pain	Gastrointestinal disorders	MedDRA	Systematic Assessment		EG00032 affected85 at risk EG00131 affected99 at risk
Abdominal pain upper	Gastrointestinal disorders	MedDRA	Systematic Assessment		EG00030 affected85 at risk EG00113 affected99 at risk
Constipation	Gastrointestinal disorders	MedDRA	Systematic Assessment		EG00023 affected85 at risk EG00114 affected99 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA	Systematic Assessment		EG00040 affected85 at risk EG00150 affected99 at risk
Dyspepsia	Gastrointestinal disorders	MedDRA	Systematic Assessment		EG0009 affected85 at risk EG0017 affected99 at risk
Gastritis	Gastrointestinal disorders	MedDRA	Systematic Assessment		EG0001 affected85 at risk EG0017 affected99 at risk
Gingival bleeding	Gastrointestinal disorders	MedDRA	Systematic Assessment		EG0001 affected85 at risk EG0015 affected99 at risk
Nausea	Gastrointestinal disorders	MedDRA	Systematic Assessment		EG00036 affected85 at risk EG00134 affected99 at risk
Toothache	Gastrointestinal disorders	MedDRA	Systematic Assessment		EG00012 affected85 at risk EG0013 affected99 at risk
Vomiting	Gastrointestinal disorders	MedDRA	Systematic Assessment		EG00023 affected85 at risk EG00136 affected99 at risk
Asthenia	General disorders	MedDRA	Systematic Assessment		EG00014 affected85 at risk EG00119 affected99 at risk
Chest pain	General disorders	MedDRA	Systematic Assessment		EG00019 affected85 at risk EG0019 affected99 at risk
Fatigue	General disorders	MedDRA	Systematic Assessment		EG00017 affected85 at risk EG00118 affected99 at risk
Influenza like illness	General disorders	MedDRA	Systematic Assessment		EG0009 affected85 at risk EG0014 affected99 at risk
Malaise	General disorders	MedDRA	Systematic Assessment		EG0005 affected85 at risk EG0014 affected99 at risk
Oedema	General disorders	MedDRA	Systematic Assessment		EG0002 affected85 at risk EG0018 affected99 at risk
Oedema peripheral	General disorders	MedDRA	Systematic Assessment		EG0007 affected85 at risk EG00112 affected99 at risk
Pyrexia	General disorders	MedDRA	Systematic Assessment		EG00039 affected85 at risk EG00136 affected99 at risk
Hepatomegaly	Hepatobiliary disorders	MedDRA	Systematic Assessment		EG0004 affected85 at risk EG0015 affected99 at risk
Seasonal allergy	Immune system disorders	MedDRA	Systematic Assessment		EG0008 affected85 at risk EG0016 affected99 at risk
Bronchitis	Infections and infestations	MedDRA	Systematic Assessment		EG0009 affected85 at risk EG00120 affected99 at risk
Ear infection	Infections and infestations	MedDRA	Systematic Assessment		EG0007 affected85 at risk EG0013 affected99 at risk
Gastroenteritis	Infections and infestations	MedDRA	Systematic Assessment		EG00014 affected85 at risk EG00117 affected99 at risk
Influenza	Infections and infestations	MedDRA	Systematic Assessment		EG00014 affected85 at risk EG00115 affected99 at risk
Nasopharyngitis	Infections and infestations	MedDRA	Systematic Assessment		EG00033 affected85 at risk EG00126 affected99 at risk
Oral herpes	Infections and infestations	MedDRA	Systematic Assessment		EG0003 affected85 at risk EG0018 affected99 at risk
Otitis media	Infections and infestations	MedDRA	Systematic Assessment		EG0003 affected85 at risk EG0016 affected99 at risk
Pharyngitis	Infections and infestations	MedDRA	Systematic Assessment		EG00026 affected85 at risk EG0014 affected99 at risk
Rhinitis	Infections and infestations	MedDRA	Systematic Assessment		EG00021 affected85 at risk EG00112 affected99 at risk
Sinusitis	Infections and infestations	MedDRA	Systematic Assessment		EG0003 affected85 at risk EG0016 affected99 at risk
Tonsillitis	Infections and infestations	MedDRA	Systematic Assessment		EG0009 affected85 at risk EG0018 affected99 at risk
Tooth abscess	Infections and infestations	MedDRA	Systematic Assessment		EG0007 affected85 at risk EG0011 affected99 at risk
Tooth infection	Infections and infestations	MedDRA	Systematic Assessment		EG0007 affected85 at risk EG0012 affected99 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA	Systematic Assessment		EG00023 affected85 at risk EG00116 affected99 at risk
Urinary tract infection	Infections and infestations	MedDRA	Systematic Assessment		EG00010 affected85 at risk EG00110 affected99 at risk
Viral infection	Infections and infestations	MedDRA	Systematic Assessment		EG0005 affected85 at risk EG0012 affected99 at risk
Viral upper respiratory tract infection	Infections and infestations	MedDRA	Systematic Assessment		EG00010 affected85 at risk EG0011 affected99 at risk
Fall	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment		EG0005 affected85 at risk EG0013 affected99 at risk
Procedural pain	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment		EG00014 affected85 at risk EG0013 affected99 at risk
Transfusion reaction	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment		EG00013 affected85 at risk EG00113 affected99 at risk
Blood creatinine increased	Investigations	MedDRA	Systematic Assessment	This is the number patients with clinically notable increases in serum creatinine, not just patients with increases.	EG00016 affected85 at risk EG00119 affected99 at risk
Cardiac murmur	Investigations	MedDRA	Systematic Assessment		EG0005 affected85 at risk EG0015 affected99 at risk
Transaminases increased	Investigations	MedDRA	Systematic Assessment		EG0003 affected85 at risk EG0015 affected99 at risk
Weight decreased	Investigations	MedDRA	Systematic Assessment		EG0002 affected85 at risk EG0015 affected99 at risk
Anorexia	Metabolism and nutrition disorders	MedDRA	Systematic Assessment		EG0007 affected85 at risk EG0017 affected99 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment		EG00018 affected85 at risk EG00112 affected99 at risk
Back pain	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment		EG00032 affected85 at risk EG00120 affected99 at risk
Bone pain	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment		EG0008 affected85 at risk EG00110 affected99 at risk
Muscle spasms	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment		EG00010 affected85 at risk EG0019 affected99 at risk
Musculoskeletal pain	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment		EG0009 affected85 at risk EG0012 affected99 at risk
Neck pain	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment		EG0008 affected85 at risk EG0012 affected99 at risk
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment		EG00017 affected85 at risk EG0019 affected99 at risk
Dizziness	Nervous system disorders	MedDRA	Systematic Assessment		EG0005 affected85 at risk EG0018 affected99 at risk
Headache	Nervous system disorders	MedDRA	Systematic Assessment		EG00045 affected85 at risk EG00134 affected99 at risk
Paraesthesia	Nervous system disorders	MedDRA	Systematic Assessment		EG0005 affected85 at risk EG0011 affected99 at risk
Anxiety	Psychiatric disorders	MedDRA	Systematic Assessment		EG0006 affected85 at risk EG0014 affected99 at risk
Depression	Psychiatric disorders	MedDRA	Systematic Assessment		EG00011 affected85 at risk EG0018 affected99 at risk
Insomnia	Psychiatric disorders	MedDRA	Systematic Assessment		EG0008 affected85 at risk EG0017 affected99 at risk
Dysuria	Renal and urinary disorders	MedDRA	Systematic Assessment		EG0007 affected85 at risk EG0013 affected99 at risk
Proteinuria	Renal and urinary disorders	MedDRA	Systematic Assessment		EG0005 affected85 at risk EG0013 affected99 at risk
Renal failure	Renal and urinary disorders	MedDRA	Systematic Assessment		EG0000 affected85 at risk EG0016 affected99 at risk
Dysmenorrhoea	Reproductive system and breast disorders	MedDRA	Systematic Assessment		EG0006 affected85 at risk EG0012 affected99 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment		EG00036 affected85 at risk EG00134 affected99 at risk
Dyspnoea	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment		EG00010 affected85 at risk EG00112 affected99 at risk
Epistaxis	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment		EG0002 affected85 at risk EG00110 affected99 at risk
Nasal congestion	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment		EG0009 affected85 at risk EG0013 affected99 at risk
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment		EG00030 affected85 at risk EG00115 affected99 at risk
Productive cough	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment		EG00011 affected85 at risk EG0011 affected99 at risk
Rhinorrhoea	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment		EG0006 affected85 at risk EG0013 affected99 at risk
Erythema	Skin and subcutaneous tissue disorders	MedDRA	Systematic Assessment		EG0005 affected85 at risk EG0013 affected99 at risk
Pruritus	Skin and subcutaneous tissue disorders	MedDRA	Systematic Assessment		EG0007 affected85 at risk EG0019 affected99 at risk
Rash	Skin and subcutaneous tissue disorders	MedDRA	Systematic Assessment		EG00014 affected85 at risk EG00113 affected99 at risk
Rash pruritic	Skin and subcutaneous tissue disorders	MedDRA	Systematic Assessment		EG0007 affected85 at risk EG0011 affected99 at risk
Haematoma	Vascular disorders	MedDRA	Systematic Assessment		EG0001 affected85 at risk EG0015 affected99 at risk
Hypertension	Vascular disorders	MedDRA	Systematic Assessment		EG0001 affected85 at risk EG0017 affected99 at risk

ID	Term
D000740	Anemia
D006486	Hemosiderosis
D019190	Iron Overload

ID	Term
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases
D019189	Iron Metabolism Disorders
D008659	Metabolic Diseases
D009750	Nutritional and Metabolic Diseases

ID	Term
D001565	Benzoates
D000146	Acids, Carbocyclic
D002264	Carboxylic Acids
D009930	Organic Chemicals
D001555	Benzene Derivatives
D006841	Hydrocarbons, Aromatic
D006844	Hydrocarbons, Cyclic
D006838	Hydrocarbons
D014230	Triazoles
D001393	Azoles
D006573	Heterocyclic Compounds, 1-Ring
D006571	Heterocyclic Compounds

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Beta-thalassemia	Deferasirox (5-40 mg/kg/day)			36	85	84	85
EG001	Rare Anemias	Deferasirox (5-40 mg/kg/day)			50	99	96	99