treatment

Last edited 11/2021 and last reviewed 03/2022

Often, there is no specific treatment and the avoidance of precipitating factors is the best action that can be taken.

Treatment of acute hepatic porphyia (AHP):

• treatment options for AHP aim to prevent attacks or manage symptoms
  • include pain management, stopping medication that could have triggered symptoms, gonadotrophin releasing hormone (GnRH) analogues for hormone-induced attacks in women, and oral or intravenous glucose for acute attacks
  • haem arginate is indicated for treating acute attacks of AHP
    • also used outside its marketing authorisation to prevent attacks
  • liver transplant may be an option for some people with recurrent severe attacks when other treatment options have not worked
  • givosiran is recommended as an option for treating acute hepatic porphyria (AHP) in adults and young people aged 12 and older, only if:
    • they have clinically confirmed severe recurrent attacks (4 attacks or more within 12 months)
    • is a small-interfering ribonucleic acid that suppresses delta-aminolevulinic acid synthase 1 production by the liver. This reduces the level of toxic precursors of porphyrin

Phlebotomy and / or chloroquine has been effective in porphyria cutanea tarda.

Beta-carotene is used to reduce sensitivity in erythropoietic protoporphyria.

Splenectomy may be used for intractable haemolytic anaemia.

Reference:

• Edel Y, Mamet R. Porphyria: What Is It and Who Should Be Evaluated?. Rambam Maimonides Med J. 2018;9(2):e0013. Published 2018 Apr 19. doi:10.5041/RMMJ.10333
• Karim Z, Lyoumi S, Nicolas G, Deybach JC, Gouya L, Puy H. Porphyrias: a 2015 update. Clin Res Hepatol Gastroenterol. 2015;39:412-425
• NICE (November 2021). Givosiran for treating acute hepatic porphyria