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Audit of the Use of Regular Haem Arginate Infusions in Patients with Acute Porphyria to Prevent Recurrent Symptoms

  • Joanne T. MarsdenEmail author
  • Simon Guppy
  • Penelope Stein
  • Timothy M. Cox
  • Michael Badminton
  • Tricia Gardiner
  • Julian H. Barth
  • M. Felicity Stewart
  • David C. Rees
Research Report
Part of the JIMD Reports book series (JIMD, volume 22)

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.

Keywords

Iron Overload GnRH Agonist Transferrin Saturation Recurrent Attack Serum Ferritin Concentration 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.

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Copyright information

© SSIEM and Springer-Verlag Berlin Heidelberg 2015

Authors and Affiliations

  • Joanne T. Marsden
    • 1
    Email author
  • Simon Guppy
    • 2
  • Penelope Stein
    • 3
  • Timothy M. Cox
    • 3
    • 4
  • Michael Badminton
    • 5
  • Tricia Gardiner
    • 5
  • Julian H. Barth
    • 6
  • M. Felicity Stewart
    • 7
  • David C. Rees
    • 2
  1. 1.Department of Clinical BiochemistryKing’s College Hospital, NHS Foundation TrustLondonUK
  2. 2.Haematological MedicineKing’s College HospitalLondonUK
  3. 3.Department of MedicineAddenbrooke’s HospitalCambridgeUK
  4. 4.University of CambridgeCambridgeUK
  5. 5.Department of Medical Biochemistry and ImmunologyUniversity Hospital of WalesCardiffUK
  6. 6.Department of Clinical ChemistryLeeds General InfirmaryLeedsUK
  7. 7.Department of Clinical BiochemistryUniversity of Manchester, Manchester Academic Health Sciences Centre, Salford Royal NHS Foundation TrustSalfordUK

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