Is there comparative data supporting efficacy for Kalbitor (ecallantide) vs Firazyr (icatibant) for ACE inhibitor-induced angioedema?

Comment by InpharmD Researcher

A comprehensive literature search did not yield direct comparative data between Kalbitor (ecallantide) and Firazyr (icatibant) for the treatment of ACE inhibitor (ACEi)-induced angioedema. While ecallantide has been studied in ACEi-induced angioedema patients, data does not demonstrate significant benefit. In contrast, icatibant has been associated with faster symptom resolution and quicker onset of relief compared to placebo, along with a higher percentage of complete resolution (see Table 1). However, due to the lack of comparative studies, the optimal agent remains unclear.

Background

A 2018 systematic review evaluated the efficacy of icatibant, ecallantide, and tranexamic acid (TA) for angiotensin-converting enzyme inhibitor (ACEi) induced and idiopathic angioedema (non-hereditary AE). This systematic review included 61 studies with 38 describing treatment in the acute setting, although the majority of evidence was case reports. There were no direct comparisons between icatibant, ecallantide, or TA. For ACEi-induced AE, two randomized controlled studies of ecallantide do not suggest a significant benefit based on response rate versus placebo (10%-16% response rate). Icatibant may have similar efficacy to C1NH and fresh frozen plasma (FFP) with an average response time of less than 2 hours, but the majority of included studies were not controlled and of lower quality. Data for TA in ACEi-induced AE was not available. [1]

For idiopathic AE, a single study on TA use observed a 54% response rate (13 to 24 patients). Data for icatibant and ecallantide are limited to response times. Icatibant reported an initial response time of 20 to 45 minutes. For complete response, icatibant reported a range between 45 minutes to 26.6 hours while ecallantide complete response was <1 hour. However, only one case report was available for ecallantide while icatibant presented various case reports and two cohorts. Prophylactic treatment of idiopathic AE using TA found improvement of symptoms in 73% of patients, based on six studies, and 16% achieved complete absence of symptoms. Prophylaxis with icatibant and ecallantide was unexplored. Based on these results, the authors prefer icatibant over ecallantide for ACEi-induced or idiopathic AE while TA may be used as a prophylaxis agent against idiopathic AE. However, with the lack of direct comparative data, the optimal agent of the three remains uncertain. [1]

Although direct comparative data is not provided, a 2016 review discusses management strategies for angioedema due to ACEi therapy, including use of ecallantide and icatibant. While ecallantide has been studied in ACEi-induced angioedema patients, there is a lack of data demonstrating significant benefits. Both a pilot study and a larger multicenter trial showed no statistically significant differences in resolution times between the ecallantide and placebo groups, leading to the conclusion that ecallantide is not currently recommended for ACEi-induced angioedema. Conversely, the authors emphasize that icatibant has shown promise in the management of ACEi-induced angioedema. In a phase 2 trial (Table 1), icatibant was associated with faster symptom resolution (median 8 hours vs. 27.1 hours with placebo) and quicker onset of relief (2 hours vs. 11.7 hours), with a higher percentage of complete resolution in the icatibant group. However, concerns remain about its cost-effectiveness and its efficacy in more severe cases or in non-white populations. Despite these concerns, it is suggested that icatibant may potentially be considered for off-label use in life-threatening orolaryngeal ACEi-induced angioedema. [2]

Another 2016 review article examined various pharmacologic interventions for ACEi-induced angioedema, including icatibant and ecallantide. Although the underlying mechanism of ACEI-induced angioedema is not fully understood, it is believed to be related to excessive bradykinin production, which these interventions aim to reduce or antagonize its activity. Currently, both ecallantide and icatibant are Food and Drug Administration (FDA) approved for the treatment of hereditary angioedema, but lack formal approval for ACEI-induced angioedema. However, icatibant has demonstrated positive effects in several case reports where symptom resolution had occurred rapidly (median, 30 minutes; interquartile range [IQR] 27.5 to 70 minutes) with complete resolution occurring at 5 hours (IQR, 4 to 7 hours). Conversely, a small placebo-controlled Phase II study assessing the safety and efficacy of ecallantide in patients with moderate to severe ACEI-induced angioedema found no significant difference in the primary outcome, defined as number of patients meeting discharge criteria (ecallantide vs. placebo: 31% vs. 21%; 95% confidence interval [CI] -14% to 34%). Nevertheless, the study revealed that ecallantide was well tolerated and may increase the proportion of patients responding to nonconventional therapies. Overall, the authors concluded that icatibant has shown benefits in managing symptoms of ACEI-induced angioedema while trials of ecallantide have shown no advantages over conventional therapies and therefore should be avoided. Of note, there were no direct comparative studies evaluating the safety and efficacy of ecallantide versus icatibant for ACEi-induced angioedema. [3]

References:

[1] van den Elzen M, Go MFCL, Knulst AC, Blankestijn MA, van Os-Medendorp H, Otten HG. Efficacy of Treatment of Non-hereditary Angioedema. Clin Rev Allergy Immunol. 2018;54(3):412-431. doi:10.1007/s12016-016-8585-0
[2] Caballero T, Pedrosa M. Angioedema due to ace inhibitors. Curr Treat Options Allergy. 2016;3(4):401-415. doi:10.1007/s40521-016-0099-8
[3] Scalese MJ, Reinaker TS. Pharmacologic management of angioedema induced by angiotensin-converting enzyme inhibitors. Am J Health Syst Pharm. 2016;73(12):873-879. doi:10.2146/ajhp150482

Literature Review

A search of the published medical literature revealed 2 studies investigating the researchable question:

Is there comparative data supporting efficacy for Kalbitor (ecallantide) vs Firazyr (icatibant) for ACE inhibitor-induced angioedema?

Level of evidence

C - Multiple studies with limitations or conflicting results  Read more→



Please see Tables 1-2 for your response.


A Randomized Trial of Icatibant in ACE-Inhibitor–Induced Angioedema

Design

Double-blind, double-dummy, randomized, phase 2

N= 27

Objective

To report the results of a phase 2 study of icatibant, as compared with standard combination therapy consisting of a glucocorticoid and an antihistamine, in patients with ACE-inhibitor–induced angioedema of the upper aerodigestive tract

Study Groups

Icabitant (n= 13)

Standard therapy (n= 14)

Inclusion Criteria

Age 18 to 95 years, receiving ACE inhibitors and presented to the emergency department with ACEi-induced angioedema affecting the upper aerodigestive tract

Exclusion Criteria

Angioedema due to other causes, history of angioedema before the initiation of ACE-inhibitor therapy, acute urticaria, unstable angina, acute myocardial ischemia, acute heart failure with a New York Heart Association class of III or IV, pregnancy/lactation

Methods

Patients were randomized (1:1) within 10 hours of symptom onset to receive icatibant 30 mg subcutaneous in the abdominal wall or standard therapy consisting of prednisolone IV 500 mg plus clemastine 2 mg.

Patients received an intravenous and subcutaneous normal saline (0.9%) placebo to mask therapy. The intensity of six symptoms (pain, shortness of breath, dysphagia, change in voice, sensation of a foreign body, and feeling of pressure) was assessed before treatment at multiple intervals up to 48 hours using a visual analog scale (VAS) ranging from 1 to 10. Then a composite score was determined using the average of the six symptoms. Angioedema was also assessed at the lips/cheeks, tongue, oropharynx, and hypopharynx/larynx. 

Rescue therapy via icatibant 30 mg and prednisolone 500 mg were allowed if the symptoms did not reduce six hours after treatment initiation. 

Duration

48 hours

Follow-up: 14 days after hospital admission

Outcome Measures

Primary: time to complete resolution of edema after administration of study treatment

Secondary: complete resolution of edema at 4 hours of treatment, time to onset of symptom relief, patients who did not respond to treatment as defined by requiring rescue therapy

Baseline Characteristics

 

Icatibant (n= 13)

Standard therapy (n= 14)

 

Age, years

62.4 ± 9.7 69.4 ± 16.6  

Female

4 (31%) 6 (43%)  

ACE inhibitor

Benazepril

Captopril

Captopril-hydrochlorothiazide

Enalapril

Lisinopril

Ramipril

 

1 (8%)

1 (8%)

1 (8%)

5 (38%)

0

5 (38%)

 

0

1 (7%)

0

2 (14%)

3 (21%)

8 (57%)

 

Previous episode of ACE-inhibitor-induced angioedema

5 (38%) 5 (36%)  

Scores for baseline severity of symptoms

Composite investigator-assessed symptom score

Composite investigator-assessed angioedema score

Composite patient-assessed VAS score

 

1.1 ± 0.2

1.1 ± 0.2

2.9 ± 0.6

 

1.2 ± 0.2

1.1 ± 0.2

3.5 ± 0.6

 

Angioedema location

Lips

Cheeks

Tongue

Pharynx and soft palate

Larynx

Floor of mouth

Face

 

3 (23%)

3 (23%)

8 (62%)

4 (31%)

6 (46%)

6 (46%)

3 (23%)

 

4 (29%)

2 (14%)

10 (71%)

5 (36%)

7 (50%)

9 (64%)

3 (21%)

 

Results

Endpoint

Icatibant (n= 13)

Standard therapy (n= 14)

p-value

Median time to complete resolution of edema, hours (interquartile range [IQR])

8.0 (3.0 to 16.0)

27.1 (20.3 to 48.0)

0.002

Patients with complete resolution of edema at 4 hours after treatment

5 (38%)

0

0.02

Median time to onset of symptom relief, hours (95% confidence interval [CI])

According to composite investigator-assessed symptom score

According to composite patient-assessed VAS score

According to composite investigator-assessed angioedema score

 

2.0 (1.0 to 8.1)

2.0 (2.0 to 6.3)

2.0 (2.0 to 12.0)

 

11.7 (8.0 to 18.0)

7.9 (1.2 to 11.8)

12.0 (11.3 to not estimatable)

 

0.03

0.36

0.003

Requiring rescue therapy

0

3

 

Any adverse events

Drug-related adverse events

Serious adverse events

Injection-site reaction

Redness

Swelling

Pain

Itching

Sensation of warmth

1 (7%)

1 (7%)

0

--

12 (80%)

8 (53%)

7 (47%)

4 (27%)

4 (27%)

4 (27%)

1 (7%)

1 (7%)

--

4 (27%)

3 (20%)

2 (13%)

1 (7%)

0

 

Study Author Conclusions

Among patients with ACE-inhibitor–induced angioedema, the time to complete resolution of edema was significantly shorter with icatibant than with combination therapy with a glucocorticoid and an antihistamine.

InpharmD Researcher Critique

The study was conducted in Germany which may not reflect the U.S. healthcare landscape. Approximately 36% of patients in both groups had a previous history of ACEi-angioedema, representing a portion of the population that may not be treatment-naive. Lastly, the small patient population makes it difficult to determine the magnitude of effects as seen by the wide range of confidence intervals and interquartile range.
References:

Baş M, Greve J, Stelter K, et al. A randomized trial of icatibant in ACE-inhibitor-induced angioedema. N Engl J Med. 2015;372(5):418-425. doi:10.1056/NEJMoa1312524

Randomized Trial of Icatibant for Angiotensin-Converting Enzyme Inhibitor-Induced Upper Airway Angioedema

Design

Multicenter, phase 3, randomized, double-blind clinical trial

N= 121

Objective

To evaluate the efficacy of icatibant in subjects with angiotensin-converting enzyme inhibitors (ACE-I)-induced angioedema

Study Groups

Icatibant (n= 61)

Placebo (n= 60)

Inclusion Criteria

Age ≥ 18 years; currently being treated with an ACE-I; presented with ACE-I–induced angioedema of the head and/or neck; moderately severe ACE-I–induced angioedema < 12 hours' duration

Exclusion Criteria

Diagnosis of angioedema of other etiology (hereditary angioedema, acquired angioedema, or allergic angioedema); family history of recurrent angioedema; history of angioedema attacks before starting ACE-I treatment; anaphylaxis, trauma, abscess or infection or associated disease; local inflammation; local tumor; postoperative or postradiogenic edema; salivary gland disorders; non-ACE-I drug-induced angioedema; acute urticaria; vascular condition that was a contraindication to study participation in the investigator's judgment; requiring immediate airway intervention; evident clinical response to antihistamines, corticosteroids, or epinephrine

Methods

Patients were randomized (1:1) to receive icatibant 30 mg or placebo administered as a single subcutaneous injection. Severity of the ACE-I–induced angioedema attack was determined by the patient's worst severity rating at baseline among 4 clinical domains (difficulty breathing, difficulty swallowing, voice changes, and tongue swelling) assessed by the enrolling physician. Antihistamines, corticosteroids, and epinephrine were allowed at any time before or after study drug administration

Duration

Enrollment: December 2013 to August 2015

Follow-up: 8 hours after drug administration

Outcome Measures

Primary: time to meeting discharge criteria (time from study drug administration to earliest time that difficulty breathing and difficulty swallowing absent [rating of 0 out of 4], and voice change and tongue swelling mild or absent [0 or 1])

Secondary: time to onset of symptom relief (TOSR; time from study drug administration to earliest time at which symptoms of at least moderate severity [pretreatment rating ≥ 2] improved by a minimum of 1 severity grade, and mild or absent [pretreatment rating of 0 or 1] symptoms again assessed as mild or absent); occurrence of airway intervention; admission to hospital

Baseline Characteristics

 

Icatibant (n= 61)

Placebo (n= 60)

 

Age, years

60.9 ± 12.1 61.8 ± 13.4  

Male

34 (55.7%) 25 (41.7%)  

Black or African American

41 (67.2%) 43 (71.7%)  

Body mass index, kg/m2

33.5 ± 8.9 30.5 ± 7.4  

Attack severity

Moderate

Severe or very severe

 

45 (73.8%)

16 (26.2%)

 

42 (70%)

18 (30%)

 

ACE-I taken

Lisinopril

Ramipril

Lisinopril and hydrochlorothiazide

Enalapril

Perindopril

Other

 

40 (65.6%)

6 (9.8%)

5 (8.2%)

4 (6.6%)

3 (4.9%)

3 (4.9%)

 

44 (73.3%)

2 (3.3%)

3 (5%)

1 (1.7%)

2 (3.3%)

6 (10%)

 

ACE-I treatment started within 90 days of the attack

16 (26.7%)

15 (24.6%)

 

Conventional medications administered

Antihistamines

Corticosteroids

Epinephrine

 

55 (90.2%)

52 (85.2%)

49 (80.3%)

 

55 (91.7%)

53 (88.3%)

51 (85%)

 

Median time from attack onset to conventional medication administration, hours (IQR)

3.4 (1.8 to 5.8)

3.6 (2.5 to 5.1)

 

Median time from conventional medication to study drug administration, hours (IQR)

3.7 (2 to 5.4)

3.1 (1.6 to 4.4)

 

Median time from attack onset to study drug administration, hours (IQR)

7.9 (5.5 to 9.7)

7.8 (5.6 to 9.4)

 

IQR, interquartile range

Results

Endpoint

Icatibant (n= 61)

Placebo (n= 60)

p-value

Median time to meet discharge criteria, hours (IQR)

4 (2 to 6) 4 (1 to 6) 0.63

Median TOSR, hours (IQR)

2 (0.6 to 3.1) 1.6 (0.5 to 3.9) 0.57

Admitted to hospital, n

34 32 Not disclosed

Admitted to intensive care unit, n

14 16 Not disclosed

Endotracheal intubation, n

1 0 Not disclosed

Adverse Events

Common Adverse Events: any treatment-related event (18.3% vs. 13.8%), injection site reaction (65% vs. 31%), erythema (51.7% vs. 22.4%), swelling (28.3% vs. 22.4%), cutaneous pain (16.7% vs. 12.1%), burning sensation (25% vs. 12.1%), itching (21.7% vs. 10.3%), warm sensation (26.7% vs. 13.8%)

Serious Adverse Events: any serious event (3.3% vs. 1.7%)

Percentage that Discontinued due to Adverse Events: No fatal events occurred.

Study Author Conclusions

Icatibant was no more efficacious than placebo in at least moderately severe ACE-I-induced angioedema of the upper airway.

InpharmD Researcher Critique

There was noted to be a significantly higher proportion of patients weighing 75 kg or more in the icatibant group compared to the placebo group, and weight has been observed to influence angioedema outcomes in some reports. Overall, the results of this study may be somewhat generalizable to patients with severe ACE-I-induced angioedema due to the inclusion of such patients. 
References:

Sinert R, Levy P, Bernstein JA, et al. Randomized Trial of Icatibant for Angiotensin-Converting Enzyme Inhibitor-Induced Upper Airway Angioedema. J Allergy Clin Immunol Pract. 2017;5(5):1402-1409.e3. doi:10.1016/j.jaip.2017.03.003