Abstract from Alnylam:
“Results from 112 patients, of which 104 have acute intermittent porphyria (AIP), showed a mean of 9.5 acute attacks reported in the prior year with severe neuropathic pain as the cardinal feature in 100% of attacks, along with other symptoms including nausea or vomiting (>80%), fatigue (77%), and weakness (79%).
Approximately 64% of patients reported experiencing porphyria symptoms between attacks, with about 46% experiencing symptoms daily. Patients also reported a diminished quality of life and significant healthcare utilization, with a mean of 4.6 overnight hospitalizations per year (range 0-70) and a mean hospital stay duration of 6.6 days (range 1-60).
Of the attacks reported on study, approximately 76% of them required treatment with heme or at a healthcare facility.”