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An Audit of the Use of Gonadorelin Analogues to Prevent Recurrent Acute Symptoms in Patients with Acute Porphyria in the United Kingdom

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JIMD Reports, Volume 36


Severe recurrent acute attacks of porphyria have traditionally been treated with either prophylactic human haemin or gonadorelin analogues (GnA) in females. Evidence on the most effective treatment for this patient subgroup is lacking. This audit surveyed the use of prophylactic GnA in the UK.

Twenty female patients (who experienced between 2 and 45 acute attacks of porphyria requiring hospitalisation and treatment with human haemin prior to GnA prophylaxis) were included in the audit. Data was retrospectively collected based on patient history and case review.

Twenty three treatment courses were given lasting a median period of 12 months. Monthly subcutaneous Goserelin was most commonly used. In three patients in whom timing with the menstrual cycle was not considered, an acute attack occurred after initiation of the first dose. The majority of patients experienced oestrogen deficiency symptoms during treatment. Fifty percent of the prescribed courses of GnA resulted in a degree of clinical benefit. This successfully treated group experienced between 3 and 20 acute attacks prior to and between 0 and 6 acute attacks during GnA treatment.

The audit revealed large variation in practice in the United Kingdom regarding indication, duration of treatment, specific drug used and management of side effects. In view of the limited treatment options available for this cohort and the mixed outcome successes reported, we believe it is reasonable for porphyria specialists to continue offering GnA treatment to women with severe recurrent debilitating acute attacks of porphyria associated with the menstrual cycle, and we propose best practice guidelines to standardise management.

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Correspondence to Danja Schulenburg-Brand .

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Communicated by: Robert Steiner


Take Home Message/Synopsis

Although no trial data exists, prophylactic GnA may be a reasonable alternative to prophylactic human haemin in women with severe recurrent attacks of acute porphyria, particularly where attacks are associated with the menstrual cycle.

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Conflict of Interest

Danja Schulenburg-Brand, Tricia Gardiner, Simon Guppy, David C. Rees, Penelope Stein, Julian Barth, M. Felicity Stewart and Michael Badminton declare that they have no conflict of interest.

Informed Consent

Not applicable as this was an audit.

Animal Rights

Not applicable.

This article does not contain any studies with human or animal subjects performed by the any of the authors.

Author Contributions

Danja Schulenburg-Brand: data analysis and authored the paper.

Tricia Gardiner: audit design, data collection and review of paper.

Simon Guppy, David Rees, Penelope Stein, Julian Barth, Felicity Stewart: review of audit design, data collection and review of paper.

Mike Badminton: audit design, data collection and co-authored the paper.

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Schulenburg-Brand, D. et al. (2017). An Audit of the Use of Gonadorelin Analogues to Prevent Recurrent Acute Symptoms in Patients with Acute Porphyria in the United Kingdom. In: Morava, E., Baumgartner, M., Patterson, M., Rahman, S., Zschocke, J., Peters, V. (eds) JIMD Reports, Volume 36. JIMD Reports, vol 36. Springer, Berlin, Heidelberg.

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