Dataset Information

Pediatric asthma: An unmet need for more effective, focused treatments.

ABSTRACT:

Background

Despite remarkable advances in our understanding of asthma, there are still several unmet needs associated with the management of pediatric asthma.

Methods

A two-day, face-to-face meeting was held in London, United Kingdom, on October 28 and 29, 2017, involving a group of international expert clinicians and scientists in asthma management to discuss the challenges and unmet needs that remain to be addressed in pediatric asthma.

Results

These unmet needs include a lack of clinical efficacy and safety evidence, and limited availability of non-steroid-based alternative therapies in patients <6 years of age. An increased focus on children is needed in the context of clinical practice guidelines for asthma; current pediatric practice relies mostly on extrapolations from adult recommendations. Furthermore, no uniform definition of pediatric asthma exists, which hampers timely and robust diagnosis of the condition in affected patients.

Conclusions

There is a need for a uniform definition of pediatric asthma, clearly distinguishable from adult asthma. Furthermore, guidelines which provide specific treatment recommendations for the management of pediatric asthma are also needed. Clinical trials and real-world evidence studies assessing anti-immunoglobulin E (IgE) therapies and other monoclonal antibodies in children <6 years of age with asthma may provide further information regarding the most appropriate treatment options in these vulnerable patients. Early intervention with anti-IgE and non-steroid-based alternative therapies may delay disease progression, leading to improved clinical outcomes.

SUBMITTER: Papadopoulos NG

PROVIDER: S-EPMC7380053 | biostudies-literature | 2019 Feb

REPOSITORIES: biostudies-literature

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Publications

Pediatric asthma: An unmet need for more effective, focused treatments.

Papadopoulos Nikolaos G NG Čustović Adnan A Cabana Michael D MD Dell Sharon D SD Deschildre Antoine A Hedlin Gunilla G Hossny Elham E Le Souëf Peter P Matricardi Paolo M PM Nieto Antonio A Phipatanakul Wanda W Pitrez Paulo M PM Pohunek Petr P Gavornikova Marcela M Jaumont Xavier X Price David B DB

Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology 20181115 1

<h4>Background</h4>Despite remarkable advances in our understanding of asthma, there are still several unmet needs associated with the management of pediatric asthma.<h4>Methods</h4>A two-day, face-to-face meeting was held in London, United Kingdom, on October 28 and 29, 2017, involving a group of international expert clinicians and scientists in asthma management to discuss the challenges and unmet needs that remain to be addressed in pediatric asthma.<h4>Results</h4>These unmet needs include a ...[more]

PMID: 30312503

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Project description:BackgroundHereditary angioedema (HAE) is a rare disease characterized by unpredictable, recurring subcutaneous or submucosal swelling. Without effective therapy, HAE can negatively impact patients' quality of life. Management of HAE includes on-demand treatment of attacks and short- and long-term prophylaxis (LTP) to prevent attacks. Newer therapies may be more tolerable and effective in managing HAE; however, therapies such as androgens are still widely used in some countries owing to their relative ease of access and adequate disease control for some patients. This study evaluated the characteristics, treatment patterns, clinical outcomes, and healthcare resource utilization of a multinational cohort of patients with HAE, with a focus on understanding reasons for recommending or discontinuing available therapies.MethodsA retrospective chart review was conducted at 12 centers in six countries and included data from patients with HAE type 1 or 2 who were ≥ 12 years of age at their first clinical visit. The relationship between LTP use and attack rates was evaluated using a multivariable Poisson regression model. Data were collected between March 2018 and July 2019.ResultsData from 225 patients were collected (62.7% female, 86.2% White, 90.2% type 1); 64.4% of patients had their first HAE-related visit to the center prior to or during 2014. Treatment patterns varied between countries. Overall, 85.8% of patients were prescribed on-demand treatment and 53.8% were prescribed LTP, most commonly the androgen danazol (53.7% of patients who used LTP). Plasma-derived C1 inhibitor (Cinryze®) was used by 29.8% of patients for LTP. Patients who received LTP had a significantly lower rate of HAE attacks than patients who did not receive any LTP (incidence rate ratio (95% confidence interval) 0.90 (0.84-0.96)). Androgens were the most commonly discontinued therapy (51.3%), with low tolerability cited as the most frequent reason for discontinuation (50.0%).ConclusionsOverall, findings from this study support the use of LTP in the prevention of HAE attacks; a lower rate of attacks was observed with LTP compared with no LTP. However, the type of LTP used varied between countries, with tolerability and accessibility to specific treatments playing important roles in management decision-making.

Dataset Information

Pediatric asthma: An unmet need for more effective, focused treatments.

Background

Methods

Results

Conclusions

Publications

Pediatric asthma: An unmet need for more effective, focused treatments.

Similar Datasets

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