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Background. Neonatal chylothorax is a rare condition, but has a high mortality. Study Objectives. To analyse the outcomes of a series of neonates with chylothorax and review the literature to determine best practice. Design. A case series review and a literature review using electronic databases including the key words neonates and chylothorax. Results. Six cases of neonatal chylothorax were identified during a ten-year period, two had congenital chylothoraces and four iatrogenic chylothoraces after thoracic surgery or chest instrumentation. The neonates were ventilated for a median of 30 (range 13–125) days with a median maximum daily pleural fluid output of 218 (range 86–310) ml/kg/day. All the neonates were given medium-chain triglyceride (MCT) feeds which stabilised pleural fluid output in four and reduced it in another. Octreotide was used in three neonates, but the dosage used had no significant effect on pleural output. Two neonates required surgical intervention. The literature review demonstrated MCT feeds can reduce or stabilise pleural fluid output, but highlighted variable use of octreotide and inconsistent dosing regimens and outcomes. No consensus regarding indications for surgical intervention was identified. Summary and Conclusion. Neonatal chylothorax is uncommon, but affected neonates require high healthcare utilisation.
Systemic AL (light chain) amyloidosis is a rare protein misfolding disorder associated with plasma cell dyscrasia affecting various organs leading to organ dysfunction and failure. The Amyloidosis Forum is a public–private partnership between the Amyloidosis Research Consortium and the US Food and Drug Administration Center for Drug Evaluation and Research with the goal of accelerating the development of effective treatments for AL amyloidosis. In recognition of this goal, 6 individual working groups were formed to identify and/or provide recommendations related to various aspects of patient-relevant clinical trial endpoints. This review summarizes the methods, findings, and recommendations of the Health-Related Quality of Life (HRQOL) Working Group. The HRQOL Working Group sought to identify existing patient-reported outcome (PRO) assessments of HRQOL for use in clinical trials and practice deemed relevant across a broad spectrum of patients with AL amyloidosis. A systematic review of the AL amyloidosis literature identified 1) additional signs/symptoms not currently part of an existing conceptual model, and 2) relevant PRO instruments used to measure HRQOL. The Working Group mapped content from each identified instrument to areas of impact in the conceptual model to determine which instrument(s) provide coverage of relevant concepts. The SF-36v2
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Health Survey (SF-36v2; QualityMetric Incorporated, LLC) and Patient-Reported Outcomes Measurement Information System-29 Profile (PROMIS-29; HealthMeasures) were identified as instruments relevant to patients with AL amyloidosis. Existing evidence of reliability and validity was evaluated with a recommendation for future work focused on estimating clinically meaningful within-patient change thresholds for these instruments. For sponsors, the context of use—including specific research objectives, trial population, and investigational product under study—should inherently drive selection of the appropriate PRO instrument and endpoint definitions to detect meaningful change and enable patient-focused drug development.
s257was observed as to what 'disease activity' conceptualized on the PGA-DA; patients better understood the PGI-S. Some patients experienced joint stiffness throughout the day and not only in the morning. Recall preference was split amongst the sample; however, some patients were unable to accurately recall their symptoms over 7 days, while all patients could recall over 24 hours. ConClusions: All the PRO measures are suitable for SLE patients to self-report symptom severity except the PGA-DA; the PGI-S is a preferable global assessment. A 24-hour recall period would provide a more accurate representation of symptom experience than a 7-day recall period. Patients highlighted a need for new SLE therapies.
A471reported but studies are often in small samples of a specific condition and/or country. This study presents a cross-national comparison of age and gender effects on scores of the SF-12v2®, a widely used HRQL measure, using data from a large multinational survey. Methods: Data collected in 2013 and 2014 from participants (18 to 80 years
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