Assessment on hereditary angioedema burden of illness in Brazil: A patient perspective

2019 ◽  
Vol 40 (3) ◽  
pp. 193-197 ◽  
Author(s):  
Anderson Abdon Barbosa ◽  
Raquel de Oliveira Martins ◽  
Renata Martins ◽  
Anete Sevciovic Grumach
Keyword(s):  
Hereditary Angioedema ◽  
Burden Of Illness ◽  
Family Members ◽  
The United States ◽  
Consent Form ◽  
South American ◽  
Similar Data ◽  
Autosomal Dominant Disorder ◽  
Life Threatening ◽  
Subcutaneous Tissues

Background: Hereditary angioedema (HAE) is an autosomal dominant disorder that affects ∼1 in 50,000 individuals. The disorder is characterized by spontaneous and gradual episodes of edema in subcutaneous tissues or mucosal membrane that may endanger the patient's life. Previous studies that concern HAE treatment and burden of illness in the United States and Europe suggest an improvement in the control of HAE. However, no similar data are available in South American patients. The purpose of the present study was to evaluate the burden of disease and compliance to therapy of patients with HAE in Brazil. Methods: We developed a structured questionnaire to assess the variables that influence the diagnosis and treatment of Brazilian patients with HAE. The questionnaire was evaluated by allergists with expertise in HAE and was reformatted before applying it. The consent form was included before survey access; approval from the ethic committee of Faculdade de Medicina do ABC (Santo André, São Paulo, Brazil) was ensured. The Brazilian association of patients with HAE distributed the survey by e-mail to associated patients all over the country, and responses were accepted within a period of 3 months after signing a consent form. Results: Ninety patients (67 women, 23 men; average age, 36.5 years) responded; the main findings were the following: 53 of 90 (64%) had no need of visiting emergency departments in the past 6 months; 71 of 90 (79%) reported life-threatening fear due to their diagnosis; 73 of 90 (83%) had other family members affected, but 54 of 73 of these affected relatives did not look for specific HAE treatment; and 26 of 90 (29%) reported death due to asphyxia or throat swelling in family members. Conclusion: Patients with HAE report understanding how severe their diagnosis represent, but they did not ponder how important their commitment to treatment may decrease the constant fear brought by the disease in its possible swelling crisis. Family data supported this conclusion.

Caregivers’ role in managing hereditary angioedema and perceptions of treatment-related burden

2021 ◽  
Vol 42 (3) ◽  
pp. S11-S16 ◽  
Author(s):  
Timothy J. Craig ◽  
Aleena Banerji ◽  
Marc A. Riedl ◽  
Jessica M. Best ◽  
Jinky Rosselli ◽  
...  
Keyword(s):  
Hereditary Angioedema ◽  
Genetic Disease ◽  
Online Survey ◽  
Scientific Literature ◽  
Upper Airway ◽  
The United States ◽  
Care Recipient ◽  
Psychosocial Impacts ◽  
Life Threatening ◽  
Familial Caregivers

Hereditary angioedema (HAE) is a rare genetic disease that results in recurrent, debilitating, and potentially life-threatening swelling episodes in the extremities, genitals, gastrointestinal tract, and upper airway. Patients can experience significant burdens related to their disease. Informal or familial caregivers often support patients with HAE and likely share in the disease-related burdens, although there are limited HAE caregiver‐focused reports in the scientific literature. In the United States, we conducted an online survey of adults caring for an individual with HAE to better understand their experiences with the disease and identify psychosocial impacts of providing care for a patient with HAE. Thirty caregivers provided responses to the survey. Most caregivers were family members of the care recipient and many had HAE themselves. Caregivers reported participating in a number of medical-related tasks and experiencing some burdens as a result of caring for a person with HAE.

Hereditary Angioedema: Management of Laryngeal Attacks

2011 ◽  
Vol 25 (6) ◽  
pp. 379-382 ◽  
Author(s):  
Sandra C. Christiansen ◽  
Bruce L. Zuraw
Keyword(s):  
Hereditary Angioedema ◽  
Treatment Options ◽  
The United States ◽  
Fresh Frozen Plasma ◽  
C1 Inhibitor ◽  
Acute Therapy ◽  
Fresh Frozen ◽  
Life Threatening ◽  
Frozen Plasma ◽  
Future Management

Background Hereditary angioedema (HAE) patients suffering from laryngeal attacks in the United States faced severely limited treatment options until 2008. These potentially life-threatening episodes occur in over one-half of the patients affected by HAE during their lifetimes. Acute therapy had been relegated to supportive care, intubation, and consideration of fresh frozen plasma (FFP)–-the latter with the potential for actually accelerating the speed and severity of the swelling. Methods In this article we will review the recently approved and emerging HAE treatments that have evolved from the recognition that bradykinin generation is the fundamental abnormality leading to attacks of angioedema. Results Acute therapy for laryngeal attacks will be discussed including purified plasma–derived C1 inhibitor (C1INH), recombinant C1INH, an inhibitor of plasma kallikrein (ecallantide), and a B2 receptor antagonist (icatibant). Prophylactic care has also been transformed from a reliance on attenuated androgens with their attendant side effects to C1INH replacement. Conclusion The arrival of these novel therapies promises to transform the future management of HAE.

scholarly journals Ecallantide for the Treatment of Hereditary Angiodema in Adults

2011 ◽  
Vol 5 ◽  
pp. CMC.S4434 ◽  
Author(s):  
Michael Lunn ◽  
Erin Banta
Keyword(s):  
Upper Airway ◽  
The United States ◽  
Reversible Inhibitor ◽  
Plasma Kallikrein ◽  
Autosomal Dominant Disorder ◽  
Clinical Disorder ◽  
Life Threatening ◽  
The Complement System ◽  
Submucosal Edema ◽  
Esterase Inhibitor

Hereditary angioedema (HAE) is a clinical disorder characterized by a deficiency of C1 esterase inhibitor (C1-INH). HAE has traditionally been divided into two subtypes. Unique among the inherited deficiencies of the complement system, HAE Types I and II are inherited as an autosomal dominant disorder. The generation of an HAE attack is caused by the depletion and/or consumption of C1-inhibitor manifested as subcutaneous or submucosal edema of the upper airway, face, extremities, or gastrointestinal tract mediated by bradykinin. Attacks can be severe and potentially life-threatening, particularly with laryngeal involvement and treatment of acute attacks in the United States has been severely limited. In December 2009 the FDA approved ecallantide for the treatment of acute HAE attacks. Ecallantide is a small recombinant protein acting as a potent, specific and reversible inhibitor of plasma kallikrein which binds to plasma kallikrein blocking its binding site, directly inhibiting the conversion of high molecular weight kininogen to bradykinin. Administered subcutaneously, ecallantide was demonstrated in two clinical trials, EDEMA3 and EDEMA4, to decrease the length and severity of acute HAE attacks. Although there is a small risk for anaphylaxis, which limits home administration, ecallantide is a novel, safe, effective and alternative treatment for acute HAE attacks.

scholarly journals Guidelines for management of hereditary angioedema: What is new? What is missing?

2022 ◽  
Vol 43 (1) ◽  
pp. 12-19 ◽  
Author(s):  
Cristine Radojicic
Keyword(s):  
Hereditary Angioedema ◽  
Treatment Guidelines ◽  
Task Force ◽  
Burden Of Illness ◽  
Continuous Assessment ◽  
Practice Parameters ◽  
Key Points ◽  
Dominant Disease ◽  
Subcutaneous Tissues ◽  
Evidence Based Guidelines

Background: Hereditary angioedema is an autosomal dominant disease that presents with recurrent episodic swelling of the submucosal and/or subcutaneous tissues of the cutaneous, gastrointestinal, and respiratory systems. Evaluation and treatment guidelines have been published nationally and internationally to aid the treating provider. Methods: A review of the most cited and most recent updated guidelines was undertaken to review key points and to explore real-world feasibility of incorporating them into clinical practice. The International World Allergy Organization/European Academy of Allergy and Clinical Immunology (WAO/EAACI) Guideline for the Management of Angioedema - The 2017 Revision and Update, and the consensus reports from the Hereditary Angioedema International Working Group, the Joint Task Force on Practice Parameters focused practice parameter update, and the most recently updated US HAEA Medical Advisory Board 2020 Guidelines for the Management of Hereditary Angioedema were reviewed and summarized. Results: Key points that have been consistent throughout the guidelines include recommendations for evaluation and classification of hereditary angioedema as well as evidence-based guidelines for treatment. Further attention is required on the evaluation and continuous assessment of the burden of illness and quality of life (QoL). Conclusion: The guidelines for management of hereditary angioedema provide a framework for the clinician. However, the physician-patient dialog with regard to the patient disease experience, which includes attack frequency, severity, and Qol, must be continually assessed.

scholarly journals Mercy towards decarceration: Examining the legal constraints on early release from prison

Incarceration ◽  
2020 ◽  
Vol 2 (1) ◽  
pp. 263266632097780
Author(s):  
Alexandra Cox ◽  
Dwayne Betts
Keyword(s):  
United States ◽  
Criminal Justice ◽  
Criminal Justice System ◽  
Justice System ◽  
Family Members ◽  
The United States ◽  
Early Release ◽  
The Us ◽  
Legal Constraints ◽  
Release From Prison

There are close to seven million people under correctional supervision in the United States, both in prison and in the community. The US criminal justice system is widely regarded as an inherently unmerciful institution by scholars and policymakers but also by people who have spent time in prison and their family members; it is deeply punitive, racist, expansive and damaging in its reach. In this article, we probe the meanings of mercy for the institution of parole.

scholarly journals The Global Registry for Hereditary Angioedema due to C1-Inhibitor Deficiency

2021 ◽  
Author(s):  
Andrea Zanichelli ◽  
Henriette Farkas ◽  
Laurance Bouillet ◽  
Noemi Bara ◽  
Anastasios E. Germenis ◽  
...  
Keyword(s):  
Hereditary Angioedema ◽  
Rare Condition ◽  
C1 Inhibitor ◽  
C1 Inhibitor Deficiency ◽  
Therapeutic Decisions ◽  
Life Threatening ◽  
Enhance Patient Care ◽  
Electronic Support ◽  
The Impact ◽  
Global Registry

AbstractHereditary angioedema (HAE) is a rare condition, mostly due to genetic deficiency of complement C1 inhibitor (C1-INH). The rarity of HAE impedes extensive data collection and assessment of the impact of certain factors known to affect the course of this disabling and life-threatening disease. Establishing a global registry could assist to overcome such issues and provides valuable patient data from different countries. The HAE Global Registry is a disease-specific registry, with web-based electronic support, where data are provided by physicians and patients through a dedicated application. We collected data between January 1, 2018, and August 31, 2020. Data on 1297 patients from 29 centers in 5 European countries were collected. At least one attack was recorded for 497 patients during the study period. Overall, 1182 patients were diagnosed with HAE type 1 and 115 with type 2. At the time of database lock, 389 patients were taking long-term prophylactic medication, 217 of which were on danazol. Most recorded attacks affected the abdomen, were generally moderate in severity, and occurred in patients who were not on prophylactic treatment (70.6%, 6244/8848). The median duration of attacks was 780 min (IQR 290–1740) in patients on prophylactic medication and 780 min (IQR 300–1920) in patients not on continuous prophylactic medication. In conclusion, the establishment of a registry for C1-INH-HAE allowed collection of a large amount of data that may help to better understand the clinical characteristics of this disease. This information may enhance patient care and guide future therapeutic decisions.

scholarly journals Assessment and management of disease burden and quality of life in patients with hereditary angioedema: a consensus report

2021 ◽  
Vol 17 (1) ◽  
Author(s):  
Konrad Bork ◽  
John T. Anderson ◽  
Teresa Caballero ◽  
Timothy Craig ◽  
Douglas T. Johnston ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Hereditary Angioedema ◽  
Clinical Management ◽  
Disease Burden ◽  
The United States ◽  
Consensus Meeting ◽  
Consensus Statements ◽  
Consensus Report

Abstract Background Hereditary angioedema (HAE) is a rare disease characterized by unpredictable, potentially life-threatening attacks, resulting in significant physical and emotional burdens for patients and families. To optimize care for patients with HAE, an individualized management plan should be considered in partnership with the physician, requiring comprehensive assessment of the patient’s frequency and severity of attacks, disease burden, and therapeutic control. Although several guidelines and consensus papers have been published concerning the diagnosis and treatment of HAE, there has been limited specific clinical guidance on the assessment of disease burden and quality of life (QoL) in this patient population. Practical guidance is critical in supporting effective long-term clinical management of HAE and improving patient outcomes. The objective of this review is to provide evidence-based guidelines for an individualized assessment of disease burden and QoL in patients with HAE. Methods A consensus meeting was held on February 29, 2020, consisting of 9 HAE experts from the United States and Europe with extensive clinical experience in the treatment of HAE. Consensus statements were developed based on a preliminary literature review and discussions from the consensus meeting. Results Final statements reflect the consensus of the expert panel and include the assessment of attack severity, evaluation of disease burden, and long-term clinical management of HAE caused by C1-esterase inhibitor deficiency. Patient-reported outcome measures for assessing HAE attack severity and frequency are available and valuable tools; however, attack frequency and severity are insufficient markers of disease severity unless they are evaluated in the broader context of the effect on an individual patient’s QoL. QoL assessments should be individualized for each patient and minimally, they should address the interference of HAE with work, school, social, family, and physical activity, along with access to and burden of HAE treatment. Advances in HAE therapies offer the opportunity for comprehensive, individualized treatment plans, allowing patients to achieve minimal attack burden with reduced disease and treatment burden. Conclusion This consensus report builds on existing guidelines by expanding the assessment of disease burden and QoL measures for patients with HAE.

Shame and blame: Secondary stigma among families of convicted sex offenders

2021 ◽  
pp. 174889582110173
Author(s):  
Douglas Evans ◽  
Adam Trahan ◽  
Kaleigh Laird
Keyword(s):  
United States ◽  
Criminal Justice ◽  
Sex Offenders ◽  
Justice System ◽  
Sex Offender ◽  
Family Members ◽  
The United States ◽  
Formerly Incarcerated ◽  
Research Attention ◽  
Sex Offender Registry

The detriment of incarceration experienced by the formerly incarcerated has been increasingly explored in the literature on reentry. A tangential but equally concerning issue that has recently received more research attention is the effect on family members of the incarcerated. The stigma of a criminal conviction is most apparent among families of convicted sex offenders, who experience consequences parallel to those of their convicted relative. Drawing from interviews with 30 individuals with a family member incarcerated for a sex offence in the United States, this study explores manifestations of stigma due to familial association. The findings suggest that families face negative treatment from social networks and criminal justice officials, engage in self-blame and that the media’s control over the narrative exacerbates family members’ experiences. Given the pervasiveness of criminal justice system contact, the rapid growth of the sex offender registry in the United States, and the millions of family members peripherally affected by one or both, justice system reforms are needed to ensure that family members are shielded from the harms of incarceration and registration.

scholarly journals THU0442 GOUT AND HEART FAILURE IN THE US: A NATIONAL PERSPECTIVE

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 458.2-458
Author(s):  
G. Singh ◽  
M. Sehgal ◽  
A. Mithal
Keyword(s):  
Risk Factors ◽  
Heart Failure ◽  
Confidence Intervals ◽  
Burden Of Illness ◽  
Average Length ◽  
Private Insurance ◽  
Significant Risk ◽  
Case Fatality ◽  
The United States ◽  
The Us

Background:Heart failure (HF) is the eighth leading cause of death in the US, with a 38% increase in the number of deaths due to HF from 2011 to 2017 (1). Gout and hyperuricemia have previously been recognized as significant risk factors for heart failure (2), but there is little nationwide data on the clinical and economic consequences of these comorbidities.Objectives:To study heart failure hospitalizations in patients with gout in the United States (US) and estimate their clinical and economic impact.Methods:The Nationwide Inpatient Sample (NIS) is a stratified random sample of all US community hospitals. It is the only US national hospital database with information on all patients, regardless of payer, including persons covered by Medicare, Medicaid, private insurance, and the uninsured. We examined all inpatient hospitalizations in the NIS in 2017, the most recent year of available data, with a primary or secondary diagnosis of gout and heart failure. Over 69,800 ICD 10 diagnoses were collapsed into a smaller number of clinically meaningful categories, consistent with the CDC Clinical Classification Software.Results:There were 35.8 million all-cause hospitalizations in patients in the US in 2017. Of these, 351,735 hospitalizations occurred for acute and/or chronic heart failure in patients with gout. These patients had a mean age of 73.3 years (95% confidence intervals 73.1 – 73.5 years) and were more likely to be male (63.4%). The average length of hospitalization was 6.1 days (95% confidence intervals 6.0 to 6.2 days) with a case fatality rate of 3.5% (95% confidence intervals 3.4% – 3.7%). The average cost of each hospitalization was $63,992 (95% confidence intervals $61,908 - $66,075), with a total annual national cost estimate of $22.8 billion (95% confidence intervals $21.7 billion - $24.0 billion).Conclusion:While gout and hyperuricemia have long been recognized as potential risk factors for heart failure, the aging of the US population is projected to significantly increase the burden of illness and costs of care of these comorbidities (1). This calls for an increased awareness and management of serious co-morbid conditions in patients with gout.References:[1]Sidney, S., Go, A. S., Jaffe, M. G., Solomon, M. D., Ambrosy, A. P., & Rana, J. S. (2019). Association Between Aging of the US Population and Heart Disease Mortality From 2011 to 2017. JAMA Cardiology. doi:10.1001/jamacardio.2019.4187[2]Krishnan E. Gout and the risk for incident heart failure and systolic dysfunction. BMJ Open 2012;2:e000282.doi:10.1136/bmjopen-2011-000282Disclosure of Interests: :Gurkirpal Singh Grant/research support from: Horizon Therapeutics, Maanek Sehgal: None declared, Alka Mithal: None declared

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