Management of AHP
| Therapy/management strategy . | Indication . | Specifics . | Regulatory information . |
|---|---|---|---|
| Trigger avoidance | Any symptomatic AHP patient | Review safe/unsafe porphyria drug lists and other potential triggers.a | Not applicable |
| Electrolyte repletion | Electrolyte abnormalities during acute attacks | Hyponatremia is especially common during attacks. | Not applicable |
| IV fluids | Poor oral intake and tachycardia during acute attacks | Boluses and maintenance fluids may be necessary. | Not applicable |
| Pain medications | Pain not controlled with other therapies | Opioids are often needed for acute attacks. | Not applicable |
| Glucose (IV or oral) | Acute attacks when hemin is not immediately available | IV dextrose may be a good choice for maintenance fluids. | Not applicable |
| GnRH analogs | Potentially for recurrent acute attacks associated with the menstrual cycle, if not responsive to givosiran and hemin | The efficacy, indications, appropriate duration, long-term side effects, and methods of side effect prevention have not been well-established in AHP. | Not approved for AHP management, may be used off-label |
| Hemin (Panhematin in United States, Normosang in Europe) | Treatment of acute attacks | 3-4 mg/kg IV daily for 4 days for acute attacks. Panhematin should be dissolved in albumin to prevent phlebitis. Sometimes used off-label for prophylaxis. | FDA label: treatment of recurrent attacks in women temporally associated with the menstrual cycle if carbohydrate therapy is inadequate Europe: treatment of acute attacks of hepatic porphyria |
| Givosiran (Givlaari) | Prevention of recurrent attacks | 2.5 mg/kg/mo. Usefulness for treatment of sporadic attacks, chronic symptoms, or acute attacks is unclear. | FDA label: treatment of adults with AHP EMA label: treatment of patients 12 and older with AHP NDA 6 June 2019 FDA approval 20 November 2019 |
| Therapy/management strategy . | Indication . | Specifics . | Regulatory information . |
|---|---|---|---|
| Trigger avoidance | Any symptomatic AHP patient | Review safe/unsafe porphyria drug lists and other potential triggers.a | Not applicable |
| Electrolyte repletion | Electrolyte abnormalities during acute attacks | Hyponatremia is especially common during attacks. | Not applicable |
| IV fluids | Poor oral intake and tachycardia during acute attacks | Boluses and maintenance fluids may be necessary. | Not applicable |
| Pain medications | Pain not controlled with other therapies | Opioids are often needed for acute attacks. | Not applicable |
| Glucose (IV or oral) | Acute attacks when hemin is not immediately available | IV dextrose may be a good choice for maintenance fluids. | Not applicable |
| GnRH analogs | Potentially for recurrent acute attacks associated with the menstrual cycle, if not responsive to givosiran and hemin | The efficacy, indications, appropriate duration, long-term side effects, and methods of side effect prevention have not been well-established in AHP. | Not approved for AHP management, may be used off-label |
| Hemin (Panhematin in United States, Normosang in Europe) | Treatment of acute attacks | 3-4 mg/kg IV daily for 4 days for acute attacks. Panhematin should be dissolved in albumin to prevent phlebitis. Sometimes used off-label for prophylaxis. | FDA label: treatment of recurrent attacks in women temporally associated with the menstrual cycle if carbohydrate therapy is inadequate Europe: treatment of acute attacks of hepatic porphyria |
| Givosiran (Givlaari) | Prevention of recurrent attacks | 2.5 mg/kg/mo. Usefulness for treatment of sporadic attacks, chronic symptoms, or acute attacks is unclear. | FDA label: treatment of adults with AHP EMA label: treatment of patients 12 and older with AHP NDA 6 June 2019 FDA approval 20 November 2019 |
EMA, European Medicines Agency; NDA, New Drug Application.
Two recommended websites include http://www.porphyriadrugs.com and https://www.drugs-porphyria.org.