TABLE 3.

Long-term follow-up for patients with AIP without frequent attack^a

	Initial presentation		Routine treatment
Case no.	Neurologic porphyriab	ICU stay	AAR in the first 2 y after diagnosis	Treatment for attacks	AAR 3rd year after diagnosis till September 2022	Prophylaxis	After 1–2 y of menopause	Years with attack	AAR during most recent 2 y
2	Severe PNP	+	1	—	No attack	None	NA	<2	No attack
3	—	—	1.5	—	No attack	None	No attack	<2	No attack
4	—	—	3	HA	No attack	None	NA	<2	No attack
5	Motor paresis and confusion	—	1.5	HA	No attack	None	NA	<2	No attack
9	—	—	1	— (S)	0.32	None	No attack	5	No attack
10	Seizure due to hyponatremia	+	0.5	—	No attack	None	NA	<2	No attack
13	PRES and bilateral radial neuropathies	—	0.5	—	No attack	None	NA	<2	No attack
14	—	—	0.5	— (S)	No attack	None	NA	<2	No attack
15	Bilateral radial neuropathies	—	1	HA	No attack	None	Male	<2	No attack
16	—	—	1	—	No attack	None	No attack	<2	No attack
17	—	—	0.5	—	No attack	None	NA	<2	No attack
18	—	—	0	—	No attack	None	Male	<2	No attack
19	—	—	2	HA	No attack	None	NA	<2	No attack
20	—	—	0.5	HA	No attack	None	NA	<2	No attack
21	Seizure due to hyponatremia	+	1	HA	0.7	None	NA	5	No attack
22	—	—	0.5	HA	No attack	None	NA	<2	No attack
24	—	—	1	HA	0.56	None	NA	4	no attack
26	—	—	2	HA	4.67	None	NA	4	4c
28	—	—	0.5	— (S)	No attack	None	NA	<2	No attack

Note: +, yes; —, no.

^aDefined as <4 clinically and/or biochemically confirmed attacks per year under routine treatment for 1–2 years after AIP diagnosis.

^bSymptoms/signs of the central or peripheral nervous system.

^cFrequent 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.

Abbreviations: AAR, annualized attack rate; G, monthly subcutaneous injection with givosiran; HA, weekly i.v. infusion with daily heme arginate (3–5 mg/kg) during acute attacks; ICU, intensive care unit; NA, not applicable, not in menopause; PNP, polyneuropathy; PRES, posterior reversible encephalopathy syndrome; S, sugar supplement with i.v. glucose.