PNH National Services

Indications for Treatment with Eculizumab, Ravulizumab and Pegcetacoplan

Please see the clinical trial page for clinical trial information.

Indications for treatment with Eculizumab:

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).
  • 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 Ravulizumab:

  • Thrombosis related to PNH.
  • Complications associated with haemolysis:
    • Renal failure.
    • Pulmonary hypertension.
  • 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.
  • Exceptional cases in whom ravulizumab 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 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).