Safety Profile - Deferasirox (Including SCAR)

Deferasirox safety: GI, renal, hepatic and hematological events plus severe cutaneous reactions (SJS/TEN/DRESS) requiring discontinuation.

Deferasirox has a well-characterised safety profile from pivotal trials, large open-label registries and decades of post-authorisation experience. Most adverse reactions are mild-to-moderate gastrointestinal events; serious risks - including renal failure, hepatic failure, GI hemorrhage and severe cutaneous reactions - require active monitoring and prompt response.

Common Adverse Reactions #

SystemReactions
GastrointestinalNausea, vomiting, diarrhoea, abdominal pain, dyspepsia
SkinRash (usually mild-to-moderate), pruritus
LaboratoryIncreased serum creatinine (often dose-related, frequently reversible); increased liver enzymes (ALT/AST)
GeneralHeadache, fatigue

Serious Adverse Reactions #

Important Safety Information. Cases with fatal outcome have been reported across several serious-reaction categories. Active monitoring and prompt discontinuation criteria are essential.
Renal
  • Acute renal failure, including fatal cases - particularly in elderly or volume-depleted patients
  • Tubulopathy (Fanconi-like) reported, especially in pediatric patients
  • Persistent creatinine elevations require dose reduction or discontinuation
Hepatic
  • Hepatic dysfunction, including hepatic failure
  • Liver enzymes should be monitored before initiation and at regular intervals during therapy
Gastrointestinal
  • GI hemorrhage and ulceration - particularly in elderly patients with advanced underlying disease, those on NSAIDs, corticosteroids, oral bisphosphonates or anticoagulants
  • Patients should report black/tarry stools or coffee-ground vomiting urgently
Haematological
  • Cytopenias including agranulocytosis, neutropenia and thrombocytopenia - predominantly in MDS patients with pre-existing risk
  • CBC monitoring during initiation in at-risk populations

Severe Cutaneous Adverse Reactions (SCAR) - Critical Box #

Severe Cutaneous Adverse Reactions: SJS, TEN and DRESS
Serious cutaneous adverse reactions have been reported, including:
  • Stevens-Johnson Syndrome (SJS) - epidermal detachment < 10% of body surface, mucosal involvement
  • Toxic Epidermal Necrolysis (TEN) - epidermal detachment > 30% of body surface, life-threatening
  • Drug Reaction with Eosinophilia and Systemic Symptoms (DRESS) - rash, fever, lymphadenopathy, eosinophilia, multi-organ involvement
If signs/symptoms of severe cutaneous reaction occur (progressive rash often with blisters or mucosal lesions, fever, lymphadenopathy), DEFRATAJ must be discontinued immediately and not re-introduced. Patients should be counselled to recognise warning signs and seek urgent care.

Hypersensitivity Reactions #

Anaphylactic reactions, including angiedema, have been reported. Symptoms typically appear within the first month of therapy. Discontinue immediately if hypersensitivity occurs.

Class-Specific Considerations #

  • Combination chelation (with deferoxamine or deferiprone) only under specialist supervision - increased risk of toxicity
  • Cataracts and lens opacities - annual ophthalmology screening recommended
  • Hearing loss - audiology baseline and annual screening recommended

Reporting #

Suspected adverse reactions associated with DEFRATAJ should be reported to the local pharmacovigilance authority and to pharmacovigilance@tajpharma.com.