Abstract
The National Acute Porphyria Service (NAPS) provides acute care support and clinical advice for patients in England with active acute porphyria requiring haem arginate treatment and patients with recurrent acute attacks.
This audit examined the benefits and complications of regular haem arginate treatment started with prophylactic intent to reduce the frequency of recurrent acute attacks in a group of patients managed through NAPS. We included 22 patients (21 female and 1 male) and returned information on diagnosis, indications for prophylactic infusions, frequency and dose, analgesia, activity and employment and complications including thromboembolic disease and iron overload.
The median age at presentation with porphyria was 21 years (range 9–44), with acute abdominal pain as the predominant symptom. Patients had a median of 12 (1–400) attacks before starting prophylaxis and had received a median of 52 (0–1,350) doses of haem arginate. The median age at starting prophylaxis was 28 years (13–58) with a median delay of 4 years (0.5–37) between presentation and prophylaxis. The frequency of prophylactic haem arginate varied from 1 to 8 per month, and 67% patients were documented as having a reduction in pain frequency on prophylaxis. Only one patient developed clinically significant iron overload and required iron chelation, but the number of venous access devices required varied from 1 to 15, with each device lasting a median of 1.2 years before requiring replacement. Six patients stopped haem arginate and in three this was because their symptoms had improved. Prophylactic haem arginate appears to be beneficial in patients with recurrent acute porphyria symptoms, but maintaining central venous access may prove challenging.
Competing interests: None declared
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Communicated by: John Christodoulou, MB BS PhD FRACP FRCPA
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Synopsis
Prophylactic haem arginate treatment can be beneficial to patients with recurrent attacks of acute porphyria.
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Joanne Marsden, Simon Guppy, Penelope Stein, Timothy Cox, Michael Badminton, Tricia Gardiner, Julian Barth, M Felicity Stewart and David Rees declare that they have no conflict of interest.
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This article does not contain any studies with human or animal subjects performed by the any of the authors.
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Joanne Marsden drafted and revised the article and analysed and interpreted the data.
Simon Guppy was involved in the design of the audit, collated and analysed the data and had input into revision of the article.
Penelope Stein was involved in the design of the audit, collated data and had input into revision of the article.
Timothy Cox was involved in the design of the audit, collated data and had input into revision of the article.
Michael Badminton was involved in the design of the audit, collated data and had input into revision of the article.
Tricia Gardiner was involved in the design of the audit, collated data and had input into revision of the article.
Julian Barth was involved in the design of the audit, collated data and had input into revision of the article.
M Felicity Stewart was involved in the design of the audit, collated data and had input into revision of the article.
David Rees was involved in the design of the audit, collated and analysed data and had input into the revision of article. He is Guarantor for the article.
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Marsden, J.T. et al. (2015). Audit of the Use of Regular Haem Arginate Infusions in Patients with Acute Porphyria to Prevent Recurrent Symptoms. In: Zschocke, J., Baumgartner, M., Morava, E., Patterson, M., Rahman, S., Peters, V. (eds) JIMD Reports, Volume 22. JIMD Reports, vol 22. Springer, Berlin, Heidelberg. https://doi.org/10.1007/8904_2015_411
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DOI: https://doi.org/10.1007/8904_2015_411
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