scholarly journals Review of Type III Non-Hereditary Angioedema Patients in Manitoba.

2020 ◽  
Author(s):  
Kelsey Hinther ◽  
Chrystyna Kalicinsky
Keyword(s):  
Hereditary Angioedema ◽  
Prophylactic Treatment ◽  
Retrospective Chart Review ◽  
Inclusion Criteria ◽  
Effective Response ◽  
On Demand ◽  
Efficacious Treatment ◽  
Inhibitor Level ◽  
High Index Of Suspicion ◽  
And Function

Abstract Introduction: Non-hereditary angioedema with normal C1 esterase inhibitor level and function (NHAE-nC1INH) is a novel entity. The diagnosis of NHAE-nC1INH requires a high index of suspicion, given that there are no laboratory tests to confirm the diagnosis. There is limited evidence on the treatment efficacy for short- and long-term prophylaxis, and acute attacks. The aim of the study is to describe a cohort of NHAE-nC1INH in Manitoba, including an estimate of prevalence, as well as to analyze the response to plasma derived C1esterase inhibitor (pdC1INH), and a bradykinin-2 receptor antagonist (Icatibant).Methods: A retrospective chart review of patients diagnosed with normal C1 esterase level and function angioedema seen at the Clinical Immunology and Allergy Clinic at the Health Sciences Centre in Winnipeg, Manitoba, was done. Inclusion criteria included the following: (1) Reside within Manitoba (2) Aged 18 years and older (3) Angioedema without urticaria (4) Normal C1-INH antigenic level and function (5) No symptom resolution with antihistamines, epinephrine or corticosteroids. (6) No family history. Five patients were included in the study who met the above inclusion criteria. The five patients charts were reviewed. Response to pdC1INH and Icatibant were defined as time to improvement of angioedema symptoms.Results: Four out of five patients had an effective response to pdC1INH for prophylactic treatment and for on-demand treatment for angioedema episodes. pdC1INH was ineffective in 1 patient for prophylaxis and on-demand treatment; tranexamic acid was successful for prophylactic treatment in 1 patient who did not respond to pdC1INH for prophylaxis and for on-demand treatment. 3 of 5 patients have tried Icatibant. All three patients were not responsive to Icatibant.Conclusion: This study suggests that pdC1INH may be an efficacious treatment option for NHAE-nC1INH patients. Larger studies are required to better elucidate the effectiveness of on-demand and prophylactic treatment for NHAE-nC1INH.

scholarly journals Review of the Manitoba cohort of patients with hereditary angioedema with normal C1 inhibitor

2019 ◽  
Vol 15 (1) ◽  
Author(s):  
Lundy McKibbin ◽  
Colin Barber ◽  
Chrystyna Kalicinsky ◽  
Richard Warrington
Keyword(s):  
Family History ◽  
Tranexamic Acid ◽  
Hereditary Angioedema ◽  
Acute Treatment ◽  
Chart Review ◽  
Positive Family History ◽  
Retrospective Chart Review ◽  
Factor Xii ◽  
C1 Inhibitor ◽  
And Function

Abstract Background Hereditary angioedema with normal C1 inhibitor (HAE-nC1 INH) is a rare, underappreciated condition characterized by recurrent subcutaneous angioedema. The underlying pathophysiology and diagnostic criteria continues to evolve. There is a significant overlap between HAE-nC1 INH and idiopathic nonhistaminergic angioedema, ultimately this may be found to be the same condition. Characterization of cohorts suspected to have either of these conditions is warranted to help refine diagnosis, pathophysiology, and treatment response. Methods A retrospective chart review of 418 patients diagnosed with angioedema was conducted. The following inclusion criteria was used: lack of response to antihistamines, steroids, and epinephrine; normal C4, C1 inhibitor (C1 INH) level and function; lack of urticaria or pruritus; occurrence without offending drugs; and positive family history. Enzyme immunoassays for C1 INH function were performed at the Mayo Clinic. Charts meeting these criteria were reviewed for frequency and type of episodes as well as use and response to therapies. Results 6 patients met the above criteria. 3 of these completed genetic testing, none were found to have factor XII abnormalities. None had angiopoietin 1 or plasminogen gene sequencing. 5 of 6 patients were successfully treated with C1 INH or tranexamic acid for acute treatment of attacks (4 with C1 INH and 1 with tranexamic acid). 4 patients have used Icatibant with good response (typically under 40 min for near full recovery); of these, 3 required Icatibant as acute treatment after other therapies (C1 inhibitor and tranexamic acid) were ineffective. There were 9 patients who otherwise met criteria, but due to a lack of family history were classified as having idiopathic non-histaminergic angioedema. Conclusions This retrospective chart review found 6 HAE-nC1 INH patients in Manitoba. 1 responded to tranexamic acid and not C1 INH, 4 typically responded to C1 INH, and 1 responded exclusively to Icatibant. All patients—4 total—who used Icatibant responded; of these 4 patients, 3 required Icatibant after other therapies had failed.

Cost-effectiveness of prophylaxis against on-demand treatment in boys with severe hemophilia A in Iran

2009 ◽  
Vol 25 (4) ◽  
pp. 584-587 ◽  
Author(s):  
Aliasghar Ahmad Kia Daliri ◽  
Hassan Haghparast ◽  
Jahanara Mamikhani
Keyword(s):  
Cost Effectiveness ◽  
Chart Review ◽  
Prophylactic Treatment ◽  
Bleeding Event ◽  
Retrospective Chart Review ◽  
Third Party ◽  
Bleeding Events ◽  
On Demand ◽  
Bleeding Episodes ◽  
Hemophilia Treatment Centers

Objective: The aim of this study was to assess the incremental cost-effectiveness of on-demand versus prophylactic hemophilia therapy in Iran from a third-party payers’ perspective.Methods: A retrospective chart review of twenty-five type A hemophiliacs who were treated in three hemophilia treatment centers was conducted. The patients were boys 0–9 years old receiving one of two treatments: (i) prophylaxis with concentrate at clinic; (ii) concentrate at clinic as on-demand. Fourteen boys received on-demand infusions for bleeding events, and eleven boys received infusions prophylaxis. Data were extracted from documents in the hemophilia treatment centers during a period of approximately 6 months.Results: The patients receiving prophylactic treatment had fewer bleeding events each month (mean, 0.26 versus 2.74) but used more concentrate (225.31 versus 87.20 units/kg per month). Average monthly cost per patient in the prophylaxis group was approximately 1.9 times higher than in the on-demand group. Compared with on-demand infusion, prophylaxis costs 3,201,656 Rials (€213.45) per bleeding event prevented.Conclusion: Prophylactic care markedly reduces the number of bleeding episodes, but at considerable cost.

Berotralstat (BCX7353) is a novel oral prophylactic treatment for hereditary angioedema: Review of phase II and III studies

2021 ◽  
Author(s):  
Michael E. Manning ◽  
Jay M. Kashkin
Keyword(s):  
Adverse Events ◽  
Phase Ii ◽  
Hereditary Angioedema ◽  
Prophylactic Treatment ◽  
Genetic Disorder ◽  
The Body ◽  
Treatment Burden ◽  
On Demand ◽  
Phase Ii And Iii

Background: Hereditary angioedema (HAE) is a rare genetic disorder characterized by unpredictable and potentially life-threatening episodes of swelling in various parts of the body. These attacks can be painful and debilitating, and affect apatient’s quality of life. Every patient who experiences an attack should be treated with on-demand medication to mitigateattack severity and duration. Many patients with HAE also receive long-term prophylaxis to reduce the frequency and severity of edema episodes. Although long-term prophylaxis reduces the disease burden for patients with HAE, available intravenous and subcutaneous treatments are accompanied by a significant treatment burden because of the logistical, emotional, and physical challenges posed by their long-term parenteral nature. Androgens are an effective oral prophylactic treatment; however, they are associated with significant adverse events and are not suitable for all patients. Thus, the HAE community has expressed interest in the development of alternative oral prophylactic therapies for preventing HAE attacks.Objective: Here, we review the phase II and III clinical data of berotralstat (BCX7353), which was approved by the U.S. Food and Drug Administration in December 2020.Results: Berotralstat is an oral, second-generation, synthetic, small-molecule plasma kallikrein inhibitor taken once dailyfor the prevention of HAE attacks in patients ages greater than or equal to 12 years. Results from the APeX studies (APeX-1 NCT02870972, APeX-2 NCT03485911, APeX-S NCT03472040, APex-J NCT03873116) demonstrated the efficacy of berotralstat as longterm prophylaxis for patients with HAE, which showed a reduction in the attack rate and on-demand medication usage.Berotralstat was well tolerated, and gastrointestinal treatment-emergent adverse events were generally mild and selflimited.Conclusion: Oral berotralstat is an effective and safe long-term prophylactic treatment for patients with HAE that will provide patients unable to tolerate parenteral therapies with the option of disease control. Berotralstat may be associated with reduced treatment burden compared with injectable therapies, highlighting the importance of patient preference with regard tothe administration route of their HAE prophylactic treatment.

Hereditary angioedema: On-demand treatment of angioedema attacks

2020 ◽  
Vol 41 (6) ◽  
pp. S26-S29
Author(s):  
Sandra C. Christiansen ◽  
Bruce L. Zuraw
Keyword(s):  
Vascular Permeability ◽  
Hereditary Angioedema ◽  
Acute Treatment ◽  
Prophylactic Treatment ◽  
Unmet Needs ◽  
Burden Of Illness ◽  
Early Administration ◽  
On Demand ◽  
The Past ◽  
Normal Life

The availability of effective acute treatment for angioedema has been fundamental in reducing the burden of illness for patients with hereditary angioedema (HAE). In building on the foundation of scientific advances that elucidate the pathomechanism(s) of attacks related to vascular permeability, novel targeted on-demand treatments have been developed and approved. These therapies have provided the means to arrest episodes of swelling, which, in the past, had the potential to inexorably lead to morbidity, and even mortality, for patients with HAE. Access to these medications, along with an emphasis on early administration and guidance that all attacks are candidates for treatment, has shifted the management paradigm for HAE. Although unmet needs remain, these acute therapies, coupled with advances in prophylactic treatment, have furthered the goal for all patients with HAE to live a normal life.

Hereditary angioedema in three brothers with normal C1 esterase inhibitor level and function

2003 ◽  
Vol 111 (2) ◽  
pp. S323
Author(s):  
S. Gupta ◽  
F. Wu ◽  
V.C. Gowda ◽  
P. Muniyappa ◽  
W.B. Klaustermeyer
Keyword(s):  
Hereditary Angioedema ◽  
C1 Esterase Inhibitor ◽  
Inhibitor Level ◽  
And Function ◽  
Esterase Inhibitor

scholarly journals Measurement of C1-Inhibitor function alone is sufficient for diagnosis of hereditary angioedema

2021 ◽  
pp. jclinpath-2021-207538
Author(s):  
Sorena Kiani-Alikhan ◽  
Elizabeth Walker ◽  
Alaco Hickey ◽  
Sofia Grigoriadou ◽  
Matt Buckland ◽  
...  
Keyword(s):  
Hereditary Angioedema ◽  
Laboratory Diagnosis ◽  
C1 Inhibitor ◽  
Laboratory Service ◽  
Number Of Patients ◽  
Centre Of Excellence ◽  
Inhibitor Level ◽  
Laboratory Investigations ◽  
And Function ◽  
The Cost

The World Allergy Organisiation/European Academy of Allergy and Clinical Immunology (WAO/EAACI) 2017/2018 guidelines recommend measuring complement4 levels, followed by C1-inhibitor level and function for diagnosis of hereditary angioedema (HAE). We analysed 6 months’ worth of data generated in our laboratory which is a specialist regional immunology service and also provides laboratory service for the Barts Health immunology department, which is a GA2LEN/HAEi-Angioedema Centre of Excellence and Reference (ACARE) and hence, investigates a large number of patients for HAE. We found that an efficient and sensitive approach for laboratory diagnosis of HAE is to only test the C1-inhibitor function. This approach had a sensitivity of 100% and reduced the cost of laboratory investigations for HAE diagnosis by 45%.

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