Enzyme replacement therapy desensitization in a child with infantile onset Pompe disease.

BACKGROUND: Enzyme replacement therapy significantly reduces morbidity and mortality in patients with Pompe disease. Development of hypersensitivity reactions to enzyme replacement therapy is common and can adversely affect disease outcomes when treatment is halted or delayed. OBJECTIVE: Our institution reports a case of successful alglucosidase alfa enzyme replacement therapy desensitisation in a 9-year-old girl with infantile onset Pompe disease. METHODS: A desensitisation protocol was tailored to our patient with the help of a multidisciplinary team including the allergist, geneticist, nurses and pharmacists. RESULTS: For our patient, desensitisation was successful using a multi-step three-fold dose escalation protocol. CONCLUSIONS: Desensitisation is possible in individuals with hypersensitivity reactions to enzyme replacement. Desensitisation protocols need to be tailored according to the patient's needs and responses to find a protocol that is safe, effective and simple.

Critically Ill Patients with COVID-19: A Narrative Review on Prone Position.

INTRODUCTION: Prone position is known to improve mortality in patients with acute respiratory distress syndrome (ARDS). The impact of prone position in critically ill patients with coronavirus disease of 2019 (COVID-19) remains to be determined. In this review, we describe the mechanisms of action of prone position, systematically appraise the current experience of prone position in COVID-19 patients, and highlight unique considerations for prone position practices during this pandemic. METHODS: For our systematic review, we searched PubMed, Scopus and EMBASE from January 1, 2020, to April 16, 2020. After completion of our search, we became aware of four relevant publications during article preparation that were published in May and June 2020, and these studies were reviewed for eligibility and inclusion. We included all studies reporting clinical characteristics of patients admitted to the hospital with COVID-19 disease who received respiratory support with high-flow nasal cannula, or noninvasive or mechanical ventilation and reported the use of prone position. The full text of eligible articles was reviewed, and data regarding study design, patient characteristics, interventions and outcomes were extracted. RESULTS: We found seven studies (total 1899 patients) describing prone position in COVID-19. Prone position has been increasingly used in non-intubated patients with COVID-19; studies show high tolerance and improvement in oxygenation and lung recruitment. Published studies lacked a description of important clinical outcomes (e.g., mortality, duration of mechanical ventilation). CONCLUSIONS: Based on the findings of our review, we recommend prone position in patients with moderate to severe COVID-19 ARDS as per existing guidelines. A trial of prone position should be considered for non-intubated COVID-19 patients with hypoxemic respiratory failure, as long as this does not result in a delay in intubation.

Extracorporeal Membrane Oxygenation for Severe Respiratory Failure During Respiratory Epidemics and Pandemics: A Narrative Review.

INTRODUCTION: Epidemics and pandemics from zoonotic respiratory viruses, such as the 2019 novel coronavirus, can lead to significant global intensive care burden as patients progress to acute respiratory distress syndrome (ARDS). A subset of these patients developed refractory hypoxaemia despite maximal conventional mechanical ventilation and required extracorporeal membrane oxygenation (ECMO). This review focuses on considerations for ventilatory strategies, infection control and patient selection related to ECMO for ARDS in a pandemic. We also summarise the experiences with ECMO in previous respiratory pandemics. METHODS: A review of pertinent studies was conducted via a search using MEDLINE, EMBASE and Google Scholar. References of articles were also examined to identify other relevant publications. RESULTS: Since the H1N1 Influenza pandemic in 2009, the use of ECMO for ARDS continues to grow despite limitations in evidence for survival benefit. There is emerging evidence to suggest that lung protective ventilation for ARDS can be further optimised while receiving ECMO so as to minimise ventilator-induced lung injury and subsequent contributions to multi-organ failure. Efforts to improve outcomes should also encompass appropriate infection control measures to reduce co-infections and prevent nosocomial transmission of novel respiratory viruses. Patient selection for ECMO in a pandemic can be challenging. We discuss important ethical considerations and predictive scoring systems that may assist clinical decision-making to optimise resource allocation. CONCLUSION: The role of ECMO in managing ARDS during respiratory pandemics continues to grow. This is supported by efforts to redefine optimal ventilatory strategies, reinforce infection control measures and enhance patient selection.