Core Outcome Set for IgE-mediated food allergy clinical trials and observational studies of interventions: International Delphi consensus study 'COMFA'.

BACKGROUND: IgE-mediated food allergy (FA) is a global health concern with substantial individual and societal implications. While diverse intervention strategies have been researched, inconsistencies in reported outcomes limit evaluations of FA treatments. To streamline evaluations and promote consistent reporting, the Core Outcome Measures for Food Allergy (COMFA) initiative aimed to establish a Core Outcome Set (COS) for FA clinical trials and observational studies of interventions. METHODS: The project involved a review of published clinical trials, trial protocols and qualitative literature. Outcomes found as a result of review were categorized and classified, informing a two-round online-modified Delphi process followed by hybrid consensus meeting to finalize the COS. RESULTS: The literature review, taxonomy mapping and iterative discussions with diverse COMFA group yielded an initial list of 39 outcomes. The iterative online and in-person meetings reduced the list to 13 outcomes for voting in the formal Delphi process. One more outcome was added based on participant suggestions after the first Delphi round. A total of 778 participants from 52 countries participated, with 442 participating in both Delphi rounds. No outcome met a priori criteria for inclusion, and one was excluded as a result of the Delphi. Thirteen outcomes were brought to the hybrid consensus meeting as a result of Delphi and two outcomes, 'allergic symptoms' and 'quality of life' achieved consensus for inclusion as 'core' outcomes. CONCLUSION: In addition to the mandatory reporting of adverse events for FA clinical trials or observational studies of interventions, allergic symptoms and quality of life should be measured as core outcomes. Future work by COMFA will define how best to measure these core outcomes.

Resolution of lobe collapse in a child with cystic fibrosis with Mycobacterium abscessus using serial intrabronchial rhDNase.

An eight-year-old girl with cysticfibrosis (CF) developed a left upper lobe collapse failing to resolve withinitial conventional antibiotic treatment, mucolytics and intensifiedphysiotherapy. Mycobacterium abscessus was isolated from her sputum. Bronchoscopy revealed thick viscousmucus plugging of the left upper lobe bronchus with complete obliteration.Three bronchoscopies with saline lavage and Dornase alfa, a rhDNase, at the endof each procedure resulted in removal of this mucus plug and the re-inflationof the affected lobe, with clinical and radiological resolution. The use of flexible bronchoscopy as a 'secondary' treatment with 0.9% saline lavage and instillation of rhDNase isdescribed sparsely in the literature. This is the first reported successfultherapeutic resolution of a lung collapse in a CF patient with Mycobacteriumabscessus, with sequential therapeutic bronchoscopies with instillation ofDornase alfa. This should be considered for lobar collapse in CF not respondingto the standard therapeutic regime.

Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quo.

INTRODUCTION: Severe asthma is a rare disease in children, for which three biologicals, anti-immunoglobulin E, anti-interleukin-5 and anti-IL4RA antibodies, are available in European countries. While global guidelines exist on who should receive biologicals, knowledge is lacking on how those guidelines are implemented in real life and which unmet needs exist in the field. In this survey, we aimed to investigate the status quo and identify open questions in biological therapy of childhood asthma across Europe. METHODS: Structured interviews regarding experience with biologicals, regulations on access to the different treatment options, drug selection, therapy success and discontinuation of therapy were performed. Content analysis was used to analyse data. RESULTS: We interviewed 37 experts from 25 European countries and Turkey and found a considerable range in the number of children treated with biologicals per centre. All participating countries provide public access to at least one biological. Most countries allow different medical disciplines to prescribe biologicals to children with asthma, and only a few restrict therapy to specialised centres. We observed significant variation in the time point at which treatment success is assessed, in therapy duration and in the success rate of discontinuation. Most participating centres intend to apply a personalised medicine approach in the future to match patients a priori to available biologicals. CONCLUSION: Substantial differences exist in the management of childhood severe asthma across Europe, and the need for further studies on biomarkers supporting selection of biologicals, on criteria to assess therapy response and on how/when to end therapy in stable patients is evident.

Outcomes for clinical trials of food allergy treatments.

OBJECTIVE: Food allergy is a common condition that can have a significant impact on the quality of life of affected individuals and their caregivers. Recent years have witnessed an increased effort to identify new treatments for food allergy. Here, we review the need to identify core outcomes for measurement in clinical trials of food allergy treatments. DATA SOURCES: We reviewed the literature regarding core outcome set development, the important role that these play in prioritizing patient-relevant outcomes, and the potential for core outcomes to accelerate the path to product marketing by allowing prompt and reliable evidence synthesis after trial publication. STUDY SELECTIONS: We reviewed recent clinical trials of food allergy treatments to understand which outcomes have previously been measured, and also reviewed available core outcome set initiatives for other allergic conditions to understand which other outcomes might be explored in future trials. RESULTS: Clinical trials of food allergy treatments have largely focused on outcomes that are relevant to investigators and commercial investors, especially the threshold of reactivity and immunologic changes. Future trials should consider addressing patient-important outcomes and should report the experiences of both adult and child participants and their caregivers. CONCLUSION: There is a pressing need for core outcome set development for food allergy treatment trials.

Anaphylaxis secondary to Parietaria judaica (wall pellitory).

Aeroallergens are an unusual cause of anaphylaxis with sparse reports in the literature. We describe a case of suspected anaphylaxis to Parietaria judaica pollen and the difficulties encountered in confirming this diagnosis.