- D1.342 - Case Report: Off-Label Dosage of Berotralstat in Hereditary Angioedema

Background:

Several option for long-term prophylaxis (LTP) in hereditary angioedema (HAE) are currently available. Berotralstat, a plasma kallikrein inhibitor, was the first available oral treatment option for HAE. The recommended dose for adults and adolescents aged 12 years and older weighing ≥ 40 kg is 150 mg of berotralstat once daily.

Case report:

Here we report the case of a 56-year-old patient with a low body weight of 49 kg, who tapered down the berotralstat doses to an intake of 4 times per week, without break-through attacks. A further reduction of the intake resulted in break-through attacks, with the need of icatibant treatment. Upon return to the intake of 4 times per week, he continued to be free of attacks.

Conclusions:

Despite not recommended by the treating physicians, the tapering down of the berotralstat dosing to a 4 times per week regimen has been working well for a HAE patient with very low body weight.