Download Deferasirox (SCG Final)

GREATER MANCHESTER INTERFACE
PRESCRIBING GROUP
On behalf of the
GREATER MANCHESTER MEDICINES
MANAGEMENT GROUP
SHARED CARE GUIDELINE for DEFERASIROX
Reference Number DEFE07 fnl
Scope: Deferasirox for paediatric patients
Issue date - May 2007
Classification
SHARED CARE GUIDELINE
Replaces - No previous version available
Author(s)/Originator(s)
Dr Andrew Will
Consultant Paediatric Haematologist, RMCH
To be read in conjunction with the Exjade® (Novartis Pharmaceuticals) patient
following documents
information leaflet and summary of product
characteristics
Authorised by
Interface Prescribing Group
Date May 2007
Review Date
May 2009
1. Introduction
Deferasirox (Exjade®) is an oral iron chelator licensed for the treatment of
chronic iron overload in patients with β thalassaemia major aged ≤ 6 years,
and the treatment of chronic iron overload due to blood transfusions when
desferrioxamine therapy is contraindicated or inadequate for patients with
other anaemias, or for patients aged 2 to 5 years. Deferasirox does not have
to be used in combination with parenteral desferrioxamine to achieve negative
iron balance in iron overloaded subjects. Deferasirox is presented as
dispersible tablets which dissolve in a small volume of water to produce a
tasteless liquid which appears to be well tolerated. Deferasirox is not
associated with blood dyscrasias thus negating the need for weekly full blood
counts. Other points to note concerning deferasirox are that it requires only a
24-hour dose interval and studies have demonstrated acceptability and
compliance with therapy.
The principal alternative to deferasirox is desferrioxamine via repeated, often
painful, subcutaneous 12-hour infusion. Thus deferasirox has obvious
advantages over this therapy.
The Guidance in this protocol should be read in conjunction with the published
information regarding deferasirox, principally the summary of product
characteristics (SPC) and the patient information leaflet (both available on-line
via http://emc.medicines.org.uk )
2. Scope
Deferasirox may be considered for shared care arrangements for the
treatment of children and adolescents aged 2 to 18 years with iron overload
secondary to repeated blood transfusions.
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3. Clinical condition being treated
Specifically, deferasirox is indicated for the treatment of chronic iron overload
due to frequent blood transfusions ( 7 ml/kg/month of packed red blood cells)
in patients with β thalassaemia major aged 6 years and older. Deferasirox is
also indicated for the treatment of chronic iron overload due to blood
transfusions when desferrioxamine therapy is contraindicated or inadequate in
the following patient groups:
- in patients with other anaemias,
- in patients aged 2 to 5 years,
- in patients with β thalassaemia major with iron overload due to infrequent
blood transfusions (<7 ml/kg/month of packed red blood cells).
Deferasirox is not licensed for use in patients less than 2 years.
Deferasirox comes in 125, 250, and 500 mg, dispersible tablets and is given in
a dose of between 5 and 30 mg/kg body weight once daily.
Deferasirox is contraindicated in patients with hypersensitivity to the active
substance or to any of the excipients.
Deferasirox is contraindicated in patients with an estimated creatinine
clearance of < 60 ml/minute.
Precautions: Deferasirox should be used with caution in patients with renal
and hepatic impairment.

Hepatic Impairment - monitor LFT’s monthly.

Renal impairment - monitor U&E’s monthly.
Deferasirox needs to be monitored by monthly assessment of liver and renal
function. This fits in well with the monthly visits to hospital for blood cross
matching and transfusion. Annual auditory and ophthalmic testing, height and
weight measurements and sexual development assessments are
recommended but this should already be part of the routine annual
assessment of children on regular transfusion regimens.
The patient has no direct responsibilities for monitoring drug therapy other
than the need to report the occurrence of possible side effects (see below).
Patients will have direct access to advice by phone from the Royal
Manchester Children’s Hospital (RMCH) Department of Haematology as well
as the contact number of their GP.
Efficacy will be assessed by the consultant Paediatric Haematologist
according to successive levels of serum ferritin.
4. Product information and treatment regimen to be used
The starting dose of deferasirox is dependent on the underlying diagnosis;
patients with β thalassaemia major start at 20 mg/kg/day and sickle cell
disease patients at 10 mg/kg/day.
Deferasirox is administered as dispersible tablets of 125, 250, and 500 mg.
The patient dissolves this in water to produce a neutral tasting, clear liquid,
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which is usually taken prior to breakfast but can be administered at any time
of day, 30 minutes prior to taking food. Apple juice or orange juice may be
used as an alternative to water.
The haematology specialist nurses based at RMCH will train patients and
carers in the preparation of the drug. Patients will also receive a leaflet
produced by Novartis and an Exjade® clear plastic tumbler with liquid measure
graduations on its side to facilitate preparation of the dispersible tablets. For
patients whose first language is not English, an interpreter will be present
during training.
5. Regimen Management
The Consultant Paediatric Haematologist and the Specialist Nursing staff at
RMCH will assume responsibility for:

Initial patient assessment to establish requirement for deferasirox

Initiating treatment with a test dose

Providing information and training for patients

Discussing benefits and potential side effects of treatment with patient

Corresponding with patient’s GP, inviting shared care for the patient

Continuing clinical assessment of the patient to ensure on-going
appropriateness of treatment

Communicating any changes to therapy promptly with the patient’s GP

Making arrangement for interpretation services where appropriate
The RMCH Paediatric Haematology Centre will provide the following
information to the patient’s GP:

The Exjade® (deferasirox) summary of product characteristics

A copy of this shared care guideline
The patient’s GP will assume responsibility for:

Notifying the consultant if willing to accept shared care

Prescribing deferasirox. Deferasirox should be supplied on a monthly
basis as a month's supply of the total number of dispersible tablets
required for 4 weeks therapy. The Consultant Paediatric Haematologist
will calculate the actual dosage and numbers of tablets required and
this information passed on to the GP prior to prescription.

Liaising with the Paediatric Haematology Department at RMCH
regarding any side effects reported by the patient to decide the
preferred course of action.
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6. Summary of cautions, contraindications, and adverse-effects
Please note that the following does not in any way replace the SPC and
should be read in conjunction with it.
CONTRAINIDICATIONS
Deferasirox is contraindicated in patients with hypersensitivity to the active
substance or to any of the excipients.
Deferasirox is contraindicated in patients with an estimated creatinine
clearance of < 60 ml/min.
ADVERSE EFFECTS - (For a comprehensive list refer to the SPC)
Very Common
Increased serum creatinine is common but usually remains within normal
limits and represents the effect of the rate of renal clearance of chelated iron.
However relevant precautions need to be taken and if the following occur at
two consecutive visits:

a non-progessive increase in serum creatinine > 50% (calculated with
reference to the mean of two pre-treatment measures)

serum creatinine above the age-adjusted upper limit of normal

the increase in serum creatinine is not attributed to any known cause
↓
the dose must be reduced by 10 mg/kg/day
Common
Headache, gastrointestinal upset, maculopapular rash, raised liver enzymes,
proteinuria.
These all tend to be mild and transient and have only led to alteration of
deferasirox therapy in a small number of patients; the rash in particular usually
lasts only a few days and does not require any alteration of dosage.
7. Special considerations
Therapy with deferasirox is only suitable for patients who have undergone the
necessary training provided by the RMCH Paediatric Haematology
Department.
Deferasirox should be kept dry and stored at room temperature.
Deferasirox should be freshly prepared immediately before use.
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8. Back-up care available to GP from Hospital, including emergency
contact procedures and help line numbers
Paediatric Haematology Centre - Nursing staff
0161 922 2396
Paediatric Haematology Centre - Medical staff
0161 922 2246
Out of hours via RMCH switchboard
0161 794 4696
9. Statement of agreements
Shared care is an agreement between the GP and the Consultant. This form
is a request by the consultant to share the suggested care pathway of your
patient. If you are unable to agree to the sharing of care and initiating the
suggested medication, please make this known to the consultant within 14
days, ideally stating the nature of your concern.
10. Written information provided to the patients
Exjade® (deferasirox, Novartis Pharmaceuticals) patient information leaflet.
Copies can be downloaded from: http://emc.medicines.org.uk
11. Supporting References
Exjade® (deferasirox) summary of product characteristics. August 2006.
Deferasirox – 'Important renal and hepatic information'. August 2006.
Deferasirox – ‘Dosing and monitoring guidelines’. October 2006.
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