Cystic fibrosis-related diabetes
gPatients should be monitored for cysticfibrosis-related
diabetes.hUseful Resources
Cysticfibrosis: diagnosis and management. National
Institute for Health and Care Excellence. NICE guideline 78.
October 2017
http://www.nice.org.uk/guidance/NG 78MUCOLYTICS
Dornase alfa
(Phosphorylated glycosylated recombinant human
deoxyribonuclease 1 (rhDNase))
lDRUG ACTIONDornase alfa is a genetically engineered
version of a naturally occurring human enzyme which
cleaves extracellular deoxyribonucleic acid (DNA).lINDICATIONS AND DOSE
Management of cystic fibrosis patients with a forced vital
capacity (FVC) of greater than 40 % of predicted to
improve pulmonary function
▶BY INHALATION OF NEBULISED SOLUTION
▶Child 5–17 years: 2500 units once daily, administered by
jet nebuliser
DOSE EQUIVALENCE AND CONVERSION
▶Dornase alfa 1000 units is equivalent to 1 mglSIDE-EFFECTSChest pain.conjunctivitis.dyspepsia.
dysphonia.dyspnoea.fever.increased risk of infection.
skin reactions
lPREGNANCYNo evidence of teratogenicity; manufacturer
advises use only if potential benefit outweighs risk.
lBREAST FEEDINGAmount probably too small to be
harmful—manufacturer advises caution.
lDIRECTIONS FOR ADMINISTRATIONDornase alfa is
administered by inhalation using a jet nebuliser, usually
once daily at least 1 hour before physiotherapy; however,
alternate-day therapy may be as effective as daily
treatment.
For use undiluted with jet nebulisers only; ultrasonic
nebulisers are unsuitable.
lPRESCRIBING AND DISPENSING INFORMATIONNot all
children benefit from treatment with dornase alfa;
improvement occurs within 2 weeks, but in more severely
affected children a trial of 6 – 12 weeks may be required.
lPATIENT AND CARER ADVICE
Medicines for Children leaflet: Dornase alfa for cystic
fibrosiswww.medicinesforchildren.org.uk/dornase-alfa-for-
cystic-fibrosislMEDICINAL FORMS
There can be variation in the licensing of different medicines
containing the same drug.
Nebuliser liquid
▶Pulmozyme(Roche Products Ltd)
Dornase alfa 1 mg per 1 mlPulmozyme 2. 5 mg nebuliser liquid 2. 5 ml
ampoules| 30 ampouleP£ 496. 43 DT = £ 496. 43Ivacaftor 22-Mar-2017
lINDICATIONS AND DOSE
Cystic fibrosis (specialist use only)
▶BY MOUTH
▶Child 6–17 years (body-weight 25 kg and above): 150 mg
every 12 hours
DOSE ADJUSTMENTS DUE TO INTERACTIONS
▶Manufacturer advises reduce dose to 150 mg twice a
week with concurrent use of potent inhibitors of
CYP 3 A 4.
▶Manufacturer advises reduce dose to 150 mg once daily
with concurrent use of moderate inhibitors of CYP 3 A 4.lCONTRA-INDICATIONSOrgan transplantation (no
information available)
lINTERACTIONS→Appendix 1 : ivacaftor
lSIDE-EFFECTS
▶Common or very commonBreast abnormalities.diarrhoea.
dizziness.ear discomfort.headache.hearing impairment
.rash.tympanic membrane hyperaemia
▶UncommonGynaecomastia
▶Frequency not knownCataract
lPREGNANCYManufacturer advises use only if potential
benefit outweighs risk—no information available.
lBREAST FEEDINGManufacturer advises use only if
potential benefit outweighs risk—no information
available.
lHEPATIC IMPAIRMENT
Dose adjustmentsMax. 150 mg once daily in moderate
impairment; in severe impairment, manufacturer
recommends use only if potential benefit outweighs risk—
starting dose 150 mg on alternate days, dosing interval
adjusted according to clinical response and tolerability.
lRENAL IMPAIRMENTCaution in severe impairment.
lMONITORING REQUIREMENTS
▶Manufacturer advises monitor liver function before
treatment, every 3 months during thefirst year of
treatment, then annually thereafter (more frequent
monitoring should be considered in patients with a history
of transaminase elevations).
▶Manufacturer advises baseline and follow-up ophthalmic
examinations in children.
lDIRECTIONS FOR ADMINISTRATIONTablets should be
taken with fat-containing food.
lPRESCRIBING AND DISPENSING INFORMATIONIvacaftor
should be prescribed by a physician experienced in the
treatment of cysticfibrosis.
lPATIENT AND CARER ADVICEPatients or carers should be
given advice on how to administer ivacaftor tablets.
Driving and skilled tasksManufacturer advises that
patients and their carers should be counselled on the
effects on driving and skilled tasks—increased risk of
dizziness.lMEDICINAL FORMS
There can be variation in the licensing of different medicines
containing the same drug.
Tablet
CAUTIONARY AND ADVISORY LABELS 25
▶Kalydeco(Vertex Pharmaceuticals (UK) Ltd)A
Ivacaftor 150 mgKalydeco 150 mg tablets| 56 tabletP
£ 14 , 000. 00186 Conditions affecting sputum viscosity BNFC 2018 – 2019
Respiratory system3