TABLE 2.
Long-term management of porphyric attacksa and treatment outcome for patients with AIP experiencing frequentb attacks
| Initial presentation | Routine treatment | Prophylaxis | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Case no. | Neurologic porphyriac | ICU stay | AAR (1st 2 y) | Treatment for acute attacks | AAR (before prophylaxis) | Drug | Year of AIP before prophylaxis | Duration of prophylaxis (year) | AAR after prophylaxis | Year since discontinuation | AAR after menopause | AAR (recent 2 y) |
| 1 | Mild PNP | — | >6 | HA | >6 | HA | 12 | 9 | 4 | >1 | No attack | No attack |
| 6 | Severe painful PNP | — | 5.5 | HA | 6.45 | HA | 2 | 5 | 0.75 | ongoing | Frequent | 1 |
| 7 | — | — | 6.5 | HA | 6.5 | HA | 2 | 15 | 2 | >1 | 1 | No attack |
| 8 | — | — | 10 | HA | HA:12, G:38d | HA, G | 2 | HA:4, G:3 | HA:1.3, G: 10d | Ongoing | NA | 9 |
| 11 | — | — | 6 | HA | 6 | HA | 1 | 5 | 0.8 | >10 | NA | No attack |
| 12 | Coma due to severe hyponatremia | + | 5 | HA | 5 | HA | 2 | 16 | 2.1 | Ongoing | NA | 0.5 |
| 23 | Mild PNP | — | 12.5 | HA | 12.5 | HA | 2 | 7 | 11d | ongoing | NA | 10.5d |
| 25 | - | — | 5 | HA | 4.3 | G | 2 | 3.25 | 1.7d | >1e | NA | No attack |
| 29 | - | — | 8 | HA | 8 | HA | 2 | 10+ | <2 | >1 | No attack | No attack |
| 27 | Severe painful PNP, seizure | + | 10 | HA | >6f | — | — | — | — | — | — | — |
Note: +, yes; —, no.
Attacks requiring hospitalization for heme arginate infusion for porphyric attacks.
Defined as 4 or more clinically and/or biochemically confirmed attacks per year under routine treatment for 1–2 years after AIP diagnosis.
Symptoms/signs of the central or peripheral nervous system.
Frequent mild porphyric attacks with abdominal pain, which could be treated with i.v. glucose administration and/or pain killer with non-narcotic agents, and extra heme arginate infusion was not required.
Discontinued givosiran due to pregnancy and remission after delivery.
Patient passed away in 2006 due to septic shock and frequent severe attacks of >6 per year.
Abbreviations: AAR, annualized attack rate; AIP, acute intermittent porphyria; G, monthly subcutaneous injection with givosiran; HA, weekly i.v. infusion with heme arginate (3–5 mg/kg) for prophylaxis or daily heme arginate (3–5 mg/kg) during acute attacks; ICU, intensive care unit; NA, not applicable, not in menopause; PNP, polyneuropathy.