9.1.8. Drugs for other blood disorders
Restrictions:
Restricted to use under the advice of the national paroxysmal nocturnal haemoglobinuria (PNH) service as monotherapy for the treatment of adult and paediatric patients 12 years of age or older with a weight of 40 kg and above with PNH:
- In patients with haemolysis with clinical symptom(s) indicative of high disease activity.
- In patients who are clinically stable after having been treated with a complement component 5 (C5) inhibitor for at least the past 6 months.
Restrictions:
Restricted to use under the advice of the national paroxysmal nocturnal haemoglobinuria (PNH) service as add-on to ravulizumab or eculizumab for the treatment of adult patients with PNH who have residual haemolytic anaemia.
Restrictions:
Restricted to specialist use for the treatment of severe and moderately severe haemophilia B (congenital factor IX deficiency) in adult patients without a history of factor IX inhibitors.
Restrictions:
Restricted to use under the advice of the national paroxysmal nocturnal haemoglobinuria (PNH) service as monotherapy in the treatment of adult patients with PNH who have haemolytic anaemia.
Restrictions:
Restricted to specialist use under the advice of the national PNH Service for patients with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least 3 months.
Restrictions:
Restricted to specialist use for the following indications:
- Treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) on the advice of the national PNH service:
- in patients with haemolysis with clinical symptom(s) indicative of high disease activity
- in patients who are clinically stable after having been treated with eculizumab for at least the past 6 months
- Treatment of patients with atypical haemolytic uremic syndrome (aHUS) under the advice of the National Renal Complement Therapeutics Service (based in Newcastle) who are complement inhibitor treatment-naïve or have received eculizumab for at least 3 months and have evidence of response to eculizumab.
Prescribing Notes:
Non-formulary indications as not recommended by SMC (non-submission):
- Treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin 4 (AQP4) antibody-positive
- Add-on to standard therapy for the treatment of adult patients with generalized myasthenia gravis (gMG) who are antiacetylcholine receptor (AChR) antibody-positive


