Abstract
Background: Prodromes are repetitive complexes of signs
or symptoms portending attacks of certain chronic diseases. Recent data
suggest that a large majority of patients with Hereditary Angioedema
(HAE) have experienced prodromes and able to predict the onset of
oncoming attacks. However, prodromes and their clinical significance
have not been investigated in a systematic manner and the underlying
pathophysiologic basis is unknown. A disease-specific, patient-reported
outcome (PRO) instrument suitable for evaluation of prodromes and
attacks is an unmet clinical need. We sought to examine and evaluate
prodromes and attacks of HAE and its associations, by a new validated
PRO instrument (HAE-EPA). Methods: HAE patients
participated in a preliminary survey addressing their demographics,
social and medical status. A cohort of 66 patients was asked to report
their recent experience in prodromes and attacks. Domains (i.e. body
locations) and dimentions (i.e. severity, impairment, functionality)
were pre-defined for both episodes. Robust bio-statistical methods were
used to analyze associations and correlations between both events.
Results: Significant correlations were demonstrated between the
two interrelated phenomena. Corrleations in severity were high across
all domains. Hierarchical Regression analysis demonstrated an
interaction between prodromes and the patients’ experience in illness
(i.e disease duration). The later can explain associations between
patient perception of the intensity of the prodromes and attacks.
Conclusions: By using the new instrument HAE patients could
effectively distinguish prodromes from attacks. The new validated
instrument demonstrated high discriminative ability, acceptability,
content validity/reliability, and therefore can be used for the
investigation and reporting prodromes, attacks and their relationships.