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.