Indications for Treatment with Eculizumab, Ravulizumab and Pegcetacoplan
Please see the clinical trial page for clinical trial information.
Indications for treatment with Eculizumab/Ravulizumab:
Patients fulfilling any of the following categories:
- Thrombosis related to PNH.
- Complications associated with haemolysis:
- Renal failure.
- Pulmonary hypertension.
- Pregnancy (and for at least 3 months post-partum) – ECULIZUMAB ONLY.
- Haemolytic (LDH >1.5xULN) symptomatic PNH with either of the following:
- With anaemia (Hb <90g/dL) or
- With agreement with Joint Service colleagues at MDT.
- Exceptional cases in whom eculizumab is considered appropriate (not fulfilling the above criteria) will be approved through discussion between the 2 Nationally Commissioned PNH Services and the National Commissioners.
Indications for treatment with Iptacopan:
- Haemolytic (LDH >1.5xULN) symptomatic PNH with either of the following:
- With anaemia (Hb <90g/dL) or
- With the agreement with Joint Service colleagues at MDT.
- Complications associated with haemolysis:
- Renal failure.
- Pulmonary hypertension.
N.B – Current indications (NICE), patients with thrombosis related to PNH without haemolysis are not eligible for Iptacopan.
Indications for treatment with Pegcetacoplan:
- Established on Ravulizumab or Eculizumab for 3 months.
- Anaemia (Hb < normal range).
- Agreement of treating PNH physicians at National centre (Leeds, Kings or Monklands).