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01.12.2014 | Research article | Ausgabe 1/2014 Open Access

BMC Gastroenterology 1/2014

Abdominal attacks and treatment in hereditary angioedema with C1-inhibitor deficiency

BMC Gastroenterology > Ausgabe 1/2014
Eitan Rubinstein, Leslie E Stolz, Albert L Sheffer, Chris Stevens, Athos Bousvaros
Wichtige Hinweise

Electronic supplementary material

The online version of this article (doi:10.​1186/​1471-230X-14-71) contains supplementary material, which is available to authorized users.

Competing interests

Dyax Corp designed and provided funding for the studies described in this article. Dr. Rubinstein has no conflicts to declare. Dr. Bousvaros reports personal fees from Dyax Corp, personal fees from Millennium pharmaceuticals, grants and personal fees from Merck pharmaceuticals, personal fees from Up to Date, personal fees from Imedex, non-financial support from Prometheus, non-financial support from Nutricia, personal fees from Cubist, outside the submitted work. Dr. Sheffer reports serving as a principle investigator for Dyax Corp. Leslie Stolz, PhD and Joseph Biedenkapp, PhD are full-time employees of Dyax Corp. Chris Stevens, MD is a consultant for Dyax Corp. The authors declare that they have no competing interests.

Authors’ contributions

ER participated in the analysis and interpretation of data and reviewed and revised the article; LS participated in the conception and design, analysis and interpretation of data and reviewed and revised the article; AS participated in the conception and design, analysis and interpretation of data and reviewed and revised the article; CS participated in the conception and design, analysis and interpretation of data and reviewed and revised the article; AB participated in the analysis and interpretation of data and reviewed and revised the article; all authors approved the final article as submitted.



Hereditary angioedema (HAE) is characterized by unpredictable attacks of debilitating subcutaneous and mucosal edema. Gastrointestinal attacks are painful, of sudden onset and often mistaken for acute abdomen leading to unnecessary surgery. The purpose of this study was to analyze symptom presentation of gastrointestinal angioedema in pediatric and adult HAE patients.


Information collected during the clinical development of ecallantide for treatment of acute HAE attacks included affected anatomic location, accompanying symptoms, medical history, and pain assessments. Efficacy endpoints included Treatment Outcome Score (TOS, maximum score = 100; minimally important difference = 30), a point-in-time measure of treatment response, and time to treatment response.


Forty-nine percent of 521 HAE attacks only involved abdominal symptoms. The most commonly reported abdominal symptoms were distension (77%), cramping (73%) and nausea (67%). The most common pain descriptors were tender, tiring-exhausting, aching, cramping and sickening. White blood cell counts were elevated (>10 × 109/L) in 23% of attacks (mean ± SD: 15.1 ± 11.27 × 109/L). A high proportion of patients reported a history of abdominal surgery, including appendectomy (23%), cholecystectomy (16.4%), and hysterectomy (8.2%). Mean TOS at 4 hours post ecallantide was 77±33 versus 29±65 for placebo. Median time to significant symptom resolution was 165 minutes (95% CI 136, 167) for ecallantide versus >4 hours (95% CI 161, >4 hours) for placebo. Anaphylactic reactions occurred in 6 of the 149 treated patients.


HAE should be considered in the differential diagnosis of patients with recurrent discrete episodes of severe, unexplained crampy abdominal pain associated with nausea.

Trials registration

The data used in the analysis were gathered across multiple clinical trials conducted during the clinical development program for ecallantide. All of the studies were conducted using Good Clinical Practices (GCP) and in accordance with the ethical principles that have their origins in the Declaration of Helsinki. Each site that participated in the clinical trials obtained the appropriate IRB or Ethics Committee approval prior to enrolling any patients. All patients provided written informed consent prior to undergoing any study-related procedures. Pediatric patients provided written assent and their parents or guardians gave written informed consent.
The following trials have been registered at http://​www.​clinicaltrials.​gov: EDEMA2 (identifier NCT01826916); EDEMA3 (identifier NCT00262080); EDEMA4 (identifier NCT00457015); and DX-88/19 (identifier NCT00456508).
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