| Popis: |
Hereditary angioedema (HAE) is a severe and disabling condition characterized by recurrent episodes of subcutaneous or mucosal swelling in the skin, respiratory and gastrointestinal tracts. HAE due to C1-esterase inhibitor deficiency (C1-INH-HAE) is the most prevalent subtype. The present Iberian study compared C1-INH-HAE treatment guidelines/norms published between 2010 and 2022 to identify main differences regarding therapeutic approaches for on-demand treatment and short- and long-term prophylaxis (LTP). Globally, HAE guidelines evolved with the availability of new treatments and with a change in the management paradigm towards an individualized, patient-centered approach where quality of life (QoL) is central. In line with that, an increasing trend was observed towards home-based treatment, as it potentially facilitates timely interventions, provides greater flexibility and convenience, and is associated with increased QoL, enabling patients to lead more normal lives. LTP is the indication with most innovations over the years, concurrently with the availability of new therapies and awareness of patients’ needs. Several prophylactic therapies with a high level of evidence became available, although formal head-to-head comparisons are lacking. The treatment goals became more ambitious, from a reduction in the frequency, severity, and duration of attacks to achieving total disease control and normalization of patients' lives at present. Changes in terminology, such as the introduction of designations as ‘first line”, and the introduction of patient-reported outcome measures (PROM) to assess patients’ perceptions of their self-experienced QoL and well-being, are also relevant and addressed in the document, together with still unmet needs in the management of C1-INH-HAE. |
