Inhaled aztreonam lysine in the management of Pseudomonas aeruginosa in patients with cystic fibrosis: real-life effectiveness.

BACKGROUND: Inhaled antibiotics have achieved or stabilised the clinical condition of patients with cystic fibrosis (CF) and chronic Pseudomonas aeruginosa infection. We aimed to determine the effectiveness of aztreonam lysine inhaled solution (AZLI) in patients with CF and chronic P. aeruginosa infection. METHODS: A retrospective observational study was conducted on patients with CF and chronic P. aeruginosa infection who received AZLI between July 2012 and September 2018 inclusive in three Spanish hospitals in a routine clinical practice setting. The primary endpoint was the absolute change in the percentage of predicted forced expiratory volume in 1 second (FEV1) compared with the previous 12 months, at the start of AZLI treatment and 12 months after starting the drug. Other variables analysed were exacerbations, hospitalisations, type and route of antibiotics prescribed, weight and body mass index (BMI) and adverse drug reactions. RESULTS: In a cohort of 52 patients, AZLI treatment led to stabilisation of FEV1, changing from a mean (SD) value of 55.60 (21.3)% at the start of treatment to 56.8 (20.4)% after 12 months of treatment (p=0.5296) in patients who had not previously received the drug. In addition, it significantly reduced exacerbations from a median (P25; P75) of 2.0 (1.0; 3.0) in the 12 months prior to AZLI to 1.0 (1.0; 2.0) in the 12 months after treatment initiation (p=0.0350). AZLI also reduced the need for other antibiotics and prevented a decrease in BMI, with an adequate safety profile. CONCLUSIONS: AZLI achieved stabilisation of lung function measured by FEV1 in patients with CF and chronic P. aeruginosa infection, along with an adequate safety profile.

COVID-19 in Solid Organ Transplantation: A Matched Retrospective Cohort Study and Evaluation of Immunosuppression Management.

BACKGROUND: The epidemiological and clinical characteristics of solid organ transplant (SOT) patients during the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) epidemic remains unclear. We conducted a matched retrospective cohort study to compare clinical outcomes among SOT recipients with the general population and to assess immunosuppression management. METHODS: Adult SOT recipients with laboratory polymerase chain reaction-confirmed SARS-CoV-2 infection admitted to a tertiary-care hospital in Barcelona, Spain, from March 11 to April 25, 2020, were matched to controls (1:4) on the basis of sex, age, and age-adjusted Charlson's Index. Patients were followed for up to 28 days from admission or until censored. Primary endpoint was mortality at 28 days. Secondary endpoints included admission to the intensive care unit and secondary complications. Drug-drug interactions (DDI) between immunosuppressants and coronavirus disease 2019 (COVID-19) management medication were collected. RESULTS: Forty-six transplant recipients and 166 control patients were included. Mean (SD) age of transplant recipients and controls was 62.7 (12.6) and 66.0 (12.7) years, 33 (71.7%) and 122 (73.5%) were male, and median (interquartile range) Charlson's Index was 5 (3-7) and 4 (2-7), respectively. Mortality was 37.0% in SOT recipients and 22.9% in controls (P = 0.51). Thirty-three (71.7%) patients underwent transitory discontinuation of immunosuppressants due to potential or confirmed DDI. CONCLUSIONS: In conclusion, hospitalized SOT recipients with COVID-19 had a trend toward higher mortality compared with controls, although it was not statistically significant, and a notable propensity for DDI.

61.º Congreso de la SEFH. Ciclodextrina intratecal en el tratamiento de la enfermedad de Niemann Pick tipo C / No disponible

No disponible

Evaluation of the efficiency under current use of human fibrinogen concentrate in trauma patients with life-threatening hemorrhagic disorders.

The aim of the study was to assess the influence of fibrinogen concentrate on survival when it is used in trauma patients with life-threatening hemorrhagic disorders. Secondly, to evaluate when the fibrinogen concentrate administration maximizes its efficacy, and to describe what other concomitant treatment the patients received in order to control their life-threatening hemorrhage. Retrospective, observational, and multicenter study was carried out in three trauma areas between June 2012 and June 2014. The totality of trauma patients with a documented life-threatening hemorrhage who received a fibrinogen concentrate prescription was included in the study. Demographic and analytical data, admission diagnosis, treatment indication, fibrinogen concentrate dose, survival after 1 and 7 days, hospitalization time, and concomitant blood product treatment were collected. One hundred and twenty-three patients were finally included. The mean dose of fibrinogen concentrate administered was 2.87 g. The mean initial fibrinogen plasma level was 1.49 g/l, which rose to 2.26 g/l. The number of patients who survived after 24 h was 80.49%, and 69.11% after 7 days. Lower fibrinogen plasma levels are statistically associated with a higher probability of death after 7 days (P = 0.004). The most suitable threshold to recommend the fibrinogen concentrate administration has been found to be 1.5 g/dl (P = 0006, after 24 h; P = 0.032, after 7 days). Finally, the most common concomitant treatment was the erythrocytes concentrate. A statistically significant relationship between lower fibrinogen plasma levels and a higher probability of death after 7 days has been found. Our data support the threshold of 1.5 g/l as the recommended level to administer fibrinogen concentrate in trauma patients.

Intrathecal cyclodextrin in the treatment of Niemann-Pick disease type C.

CASE: A child with Niemann-Pick disease type C was started on miglustat therapy at the age of 2 years. Intrathecal administration of hydroxypropyl-ß-cyclodextrin was added 5 months later. The initial dose of 175 mg was gradually increased over the first 6 months to reach 325 mg. The drug was administered every 15 days, and the patient received 43 doses. A slight delay in progression of the disease was seen during the first year of intrathecal hydroxypropyl-ß-cyclodextrin. However, additional symptoms have emerged since that time, suggesting a lack of effectiveness of the drug. Our patient has shown no drug-related adverse events. CONCLUSIONS: Intrathecal hydroxypropyl-ß-cyclodextrin therapy is safe, but its efficacy seems questionable in a patient with the severe infantile form of Niemann-Pick disease type C.