News in brief: TGA approves Trikafta for CF; Deprescribing ineffective treatments for kids with bronchiolitis; Pembrolizumab promising in mesothelioma


CFTR modulator registered by the TGA

Trikafta has been registered by the TGA for the treatment of cystic fibrosis in patients aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

The TGA approval of Trikafta (elexacaftor, ivacaftor, tezacaftor fixed dose combination tablets + ivacaftor tablets) was based on the results of four global Phase 3 studies, which included multiple trial sites in Australia.

The drug is expected to benefit about 750 Australians with CF.

The PBAC was also considering an application for Trikafta to be listed on the PBS at their March meeting. The results of the PBAC’s decision are expected to be announced shortly.


Education discourages ineffective treatments for bronchiolitis in kids

Reducing non-evidence based practices in the management of children with bronchiolitis is possible, according to the results of a RCT conducted at 26 hospitals in Australia and New Zealand.

Unnecessary and potentially harmful treatments for bronchiolitis include chest radiography, salbutamol, glucocorticoids and antibiotics were targeted in an intervention aimed at de-implementation.

Intervention hospitals received a targeted education program including clinical leads, workshops, audits and feedback for nursing and medical staff in the ED while control hospitals received electronic and printed copies of the Australasian Bronchiolitis Guideline.

The study found compliance with the Guideline during the first 24 hours of hospitalisation – with no use of chest X-ray, salbutamol, glucocorticoids, antibiotics, and adrenaline – occurred in 85.1% infants treated at intervention hospitals compared to 73.0% in the control hospitals (group (p < 0.001).

Absolute improvements in compliance occurred for each of the five guideline recommendations and across the children’s time in ED and during the total hospitalisation.

“Our results show that targeted interventions designed to address factors influencing bronchiolitis management can improve the care delivered to infants with bronchiolitis and deimplement unnecessary care,” the study authors said.

JAMA Pediatrics


Pembrolizumab shows promise in previously treated mesothelioma

Patients with previously-treated advanced malignant pleural mesothelioma (MPM) may have tumour responses lasting up to a year or more following pembrolizumab therapy, a study involving Australian patients has shown.

Results from a cohort of 118 patients with MPM in the phase 2 KEYNOTE-158 study showed an objective response rate of 8% after pembrolizumab monotherapy. The duration of response ranged from  4·0  to  33·9+ months (median 14.3)  and  60% of objective responses were ongoing at 12 months.

The antitumour activity was seen irrespective of PD-L1 expression, both in patients with PD-L1-positive and PD-L1-negative tumours, and with manageable toxicity.

The study was conducted in 14 countries and included patients treated at Blacktown Hospital and Chris O’Brien Lifehouse, NSW.

Lancet Respiratory Medicine.

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