9.6 Metabolic disorders

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First line drugs

Second line drugs

Specialist drugs

Secondary care drugs

Traffic light status (TLS) explained:

Green: Routine prescribing within licensed indication
Amber 1: specialist recommendation followed by GP initiation and continuation
Amber 2: specialist or GP initiation in line with local guideline after 1st line failure followed by GP continuation
Amber 3: specialist initiation and stabilisation followed by GP continuation
Amber SCG: specialist initiation and stabilisation followed by GP continuation in line with an agreed shared care guideline
Red: Hospital or specialist prescribing only
Double Red: These medicines have been evaluated and rejected by MKPAG and are NOT approved for use within MK. They are not recommended for use because of lack of clinical effectiveness, cost effectiveness or safety.

9.6 Metabolic disorders

9.6.1 Acute porphyrias

Haem derivatives

Haem arginate

Normosang® solution for infusion 25mg/ml

Note:

Many drugs can precipitate porphyria crisis. The BNF contains some guidance but please refer to Haematologists or Medicines Information Centre at Milton Keynes Hospital for advice on prescribing for patients with acute porphyria.
Haemarginate (Normosang^®) is kept in the Pharmacy Emergency Cupboard fridge for the treatment of acute porphyric crisis under the supervision of a Consultant Haematologist.

9.6.2 Carnitine deficiency

Carnitine derivatives

Levocarnitine (formerly Carnitine)

Oral liquid 100mg per 1ml = 1g/10ml, 10% solution
Oral liquid 300mg per 1ml = 1.5g/5ml, 30% Paediatric solution

Injection 200mg per 1ml = 1g in 5ml soultion

Note:

Specialist advice is necessary before prescribing any of these.

9.6.3 Fabry's disease - not treated at MKUH

9.6.4 Gaucher's disease - not treated at MKUH

9.6.5 Homocystinuria - not treated at MKUH

9.6.6 Mucopolysaccharidosis - not treated at MKUH

9.6.7 Nephropathic cystinosis- not treated at MKUH

9.6.8 Niemann-Pick type C disease- not treated at MKUH

9.6.9 Pompe disease - not treated at MKUH

9.6.10 Tyrosinaemia type I - not treated at MKUH

9.6.11 Urea cycle disorders

For further information on the emergency management of urea cycle disorders consult the British Inherited Metabolic Disease Group (BIMDG) website at www.bimdg.org.uk

9.6.12 Wilson's disease

Penicillamine

Tablet 125mg

Antidotes and chelators

Trientine dihydrochloride

Capsule 250mg, 300mg

Notes:

Use as per Clinical Commissioning Policy: Trientine for Wilson disease (all ages)
NHSE Funded; use must be registered on the Blueteq system
Only available via an agreed Shared Care Agreement with the approved specialist centre

Traffic light status (TLS) explained:

Green: Routine prescribing within licensed indication
Amber 1: specialist recommendation followed by GP initiation and continuation
Amber 2: specialist or GP initiation in line with local guideline after 1st line failure followed by GP continuation
Amber 3: specialist initiation and stabilisation followed by GP continuation
Amber SCG: specialist initiation and stabilisation followed by GP continuation in line with an agreed shared care guideline
Red: Hospital or specialist prescribing only
Double Red: These medicines have been evaluated and rejected by MKPAG and are NOT approved for use within MK. They are not recommended for use because of lack of clinical effectiveness, cost effectiveness or safety.