3 – 6 months, maintenance dose adjusted according to
serum-ferritin concentration; maximum 28 mg/kg per
day; Usual maximum 21 mg/kg
Chronic iron overload when desferrioxamine is contra-
indicated or inadequate in non-transfusion-dependent
thalassaemia syndromes (specialist use only)
▶BY MOUTH
▶Child 10–17 years:Initially 7 mg/kg once daily,
maintenance dose adjusted according to serum-ferritin
concentration and liver-iron concentration (consult
product literature); maximum 7 mg/kg per daylCAUTIONSHistory of liver cirrhosis.not recommended in
conditions which may reduce life expectancy (e.g. high-
risk myelodysplastic syndromes).platelet count less than
50x10^9 /litre.risk of gastro-intestinal ulceration and
haemorrhage.unexplained cytopenia—consider treatment
interruption
lINTERACTIONS→Appendix 1 : iron chelators
lSIDE-EFFECTS
▶Common or very commonConstipation.diarrhoea.
gastrointestinal discomfort.headache.nausea.skin
reactions.urine abnormalities.vomiting
▶UncommonAnxiety.cataract.cholelithiasis.deafness.
dizziness.fatigue.fever.gastrointestinal disorders.
gastrointestinal haemorrhage (including fatal cases).
hepatic disorders.laryngeal pain.maculopathy.oedema.
renal tubular disorders.sleep disorder
▶Rare or very rareOptic neuritis
▶Frequency not knownAcute kidney injury.alopecia.
anaemia aggravated.angioedema.hypersensitivity
vasculitis.metabolic acidosis.nephritis tubulointerstitial.
nephrolithiasis.neutropenia.pancreatitis acute.
pancytopenia.renal tubular necrosis.severe cutaneous
adverse reactions (SCARs).thrombocytopenia
lPREGNANCYManufacturer advises avoid unless
essential—toxicity inanimalstudies.
lBREAST FEEDINGManufacturer advises avoid—present in
milk inanimalstudies.
lHEPATIC IMPAIRMENTAvoid in severe impairment.
Dose adjustmentsUse with caution in moderate
impairment, reduce dose considerably then gradually
increase to max. 50 % of normal dose.
lRENAL IMPAIRMENTManufacturer advises avoid if
estimated creatinine clearance less than 60 mL/minute.
Dose adjustmentsManufacturer advises reduce dose if
serum-creatinine increased above age-appropriate limits
or creatinine clearance less than 90 mL/minute on
2 consecutive occasions—consult product literature.
lMONITORING REQUIREMENTS
▶Manufacturer advises monitoring of the following patient
parameters: baseline serum creatinine twice and
creatinine clearance once before initiation of treatment,
weekly in thefirst month after treatment initiation or
modification, then monthly thereafter; proteinuria before
treatment initiation then monthly thereafter, and other
markers of renal tubular function as needed; liver function
before treatment initiation, every 2 weeks during thefirst
month of treatment, then monthly thereafter; eye and ear
examinations before treatment and annually during
treatment; serum-ferritin concentration monthly.
▶Manufacturer advises monitor liver-iron concentration
every three months in children with non-transfusion-
dependent thalassaemia syndromes when serum ferritin is
800 micrograms/litre.
▶Manufacturer advises monitor body-weight, height, and
sexual development before treatment and then annually
thereafter.
lDIRECTIONS FOR ADMINISTRATIONForfilm-coated tablets,
manufacturer advises tablets may be crushed and
sprinkled on to soft food (yoghurt or apple sauce), then
administered immediately.
lPATIENT AND CARER ADVICEPatient or carers should be
given advice on how to administer deferasirox tablets.
Medicines for Children leaflet: Deferasirox for removing excess
ironwww.medicinesforchildren.org.uk/deferasirox-for-
removing-excess-iron
lNATIONAL FUNDING/ACCESS DECISIONS
Scottish Medicines Consortium (SMC) Decisions
TheScottish Medicines Consortiumhas advised (June 2017 )
that deferasirox (Exjade®) is accepted for restricted use
within NHS Scotland for the treatment of chronic iron
overload associated with the treatment of rare acquired or
inherited anaemias requiring recurrent blood transfusions.
It is not recommended for patients with myelodysplastic
syndromes.
Patients with myelodysplastic syndromes, the
commonest cause of transfusion-dependent anaemia,
were poorly represented in the clinical trial population and
the economic case was not demonstrated in this group.lMEDICINAL FORMS
There can be variation in the licensing of different medicines
containing the same drug.
Tablet
CAUTIONARY AND ADVISORY LABELS 25
▶Exjade(Novartis Pharmaceuticals UK Ltd)A
Deferasirox 90 mgExjade 90 mg tablets| 30 tabletP£ 126. 00
Deferasirox 180 mgExjade 180 mg tablets| 30 tabletP£ 252. 00
Deferasirox 360 mgExjade 360 mg tablets| 30 tabletP
£ 504. 00Deferiprone
lDRUG ACTIONDeferiprone is an oral iron chelator.lINDICATIONS AND DOSE
Treatment of iron overload in patients with thalassaemia
major in whom desferrioxamine is contra-indicated or is
inadequate
▶BY MOUTH
▶Child 6–17 years: 25 mg/kg 3 times a day; maximum
100 mg/kg per daylCONTRA-INDICATIONSHistory of agranulocytosis or
recurrent neutropenia
lINTERACTIONS→Appendix 1 : deferiprone
lSIDE-EFFECTS
▶Common or very commonAbdominal pain (reducing dose
and increasing gradually may improve tolerance).
agranulocytosis.appetite increased.arthralgia.diarrhoea
(reducing dose and increasing gradually may improve
tolerance).fatigue.headache.nausea (reducing dose and
increasing gradually may improve tolerance).neutropenia
.urine discolouration.vomiting (reducing dose and
increasing gradually may improve tolerance)
▶Frequency not knownSkin reactions.zinc deficiency
lCONCEPTION AND CONTRACEPTIONManufacturer advises
avoid before intended conception—teratogenic and
embryotoxic inanimalstudies. Contraception advised in
females of child-bearing potential.
lPREGNANCYManufacturer advises avoid during
pregnancy—teratogenic and embryotoxic inanimal
studies.
lBREAST FEEDINGManufacturer advises avoid—no
information available.
lHEPATIC IMPAIRMENTManufacturer advises monitor liver
function—interrupt treatment if persistent elevation in
serum alanine aminotransferase.BNFC 2018 – 2019 Iron overload 577
Blood and nutrition9