D1.308 - Case report: challenges in a long-term prophylaxis in hereditary angioedema (HAE)
Case report
Background: Long-term prophylaxis (LTP) in HAE-C1INH prevents attacks and thus reduces the burden of the disease. LTP should be individualized taking into consideration several aspects (i.e. quality of life, availability of health care, number and severity of attacks, etc.). Currently there are three medications recommended for the first-line long-term prophylactic treatment in HAE-C1INH: plasma-derived C1-INH, Berotralstat and Lanadelumab. Both Berotralstat and Lanadelumab affect plasma kallikrein activity, plasma-derived C1-INH is a substitution of defective serine protease inhibitor.
Methods: Retrospective analysis of medical records in a 41-year-old female with HAE-C1INH-Type2 (HAE-2) focusing on number of attacks per year.
Results: The patient was diagnosed with HAE-2 at the age of 14. Due to high frequency of attacks (23-62 attacks per year) she was several years on the LTP with tranexamic acid or attenuated androgens with little effect. At the age of 35 she switched to Berotralstat 150mg per day taking it 4 consecutive years with only mild reduction of number of attacks (22-33 attacks per year). When she was 39 we started LTP with Lanadelumab 300mg every 2 weeks but the frequency of attacks was not significantly reduced. Finally, LTP with subcutaneously administered plasma-derived C1-INH led to significant reduction of number of attacks and the patient experienced only 2 attacks during last year. Neither of those two attacks was severe.
Conclusion: There is currently no evidence that any of LTP options in HAE-C1INH should be preferred over each other. In our case report we present a patient with high activity of HAE and poor response to LTP with either Berotralstat or Lanadelumab. The disease activity was finally reduced by plasma-derived C1-INH. There are probably individual differences among patients considering therapeutic response to LTP. The choice of LTP should thus be made by shared decision between physician and patient.
