Explaining the most important trials in respiratory medicine
89 trials across 39 topics. Every citation is checked against PubMed. Pick a therapy to see what its evidence actually showed, or start typing to search.
| Topic | Therapy group | Therapy | Effect | Trial | Finding |
|---|---|---|---|---|---|
| Asthma | Biologics | Benralizumab | ABRA 2025 | A single dose of benralizumab at the time of an eosinophilic exacerbation beat prednisolone | |
| Asthma | Biologics | Depemokimab | SWIFT 2024 | Two injections a year roughly halved exacerbations in eosinophilic severe asthma | |
| Asthma | Inhaled therapy | Albuterol/budesonide as needed | MANDALA 2022 | Adding budesonide to the albuterol reliever cut severe exacerbations by a quarter | |
| Asthma | Biologics | Tezepelumab | NAVIGATOR 2021 | Tezepelumab halved exacerbations in severe asthma, including in patients with low eosinophils | |
| Asthma | Inhaled therapy | Budesonide/formoterol as needed | SYGMA 1 2018 | As-needed ICS/formoterol beat reliever alone on symptoms and exacerbations, but lost to daily ICS on symptom control | |
| Asthma | Inhaled therapy | Budesonide/formoterol as needed | SYGMA 2 2018 | As-needed ICS/formoterol matched daily ICS for exacerbations on a quarter of the steroid dose | |
| Asthma | Oral therapy | Azithromycin | AMAZES 2017 | Azithromycin cut asthma exacerbations by 41% and improved quality of life, at the cost of diarrhoea | |
| Asthma | Inhaled therapy | Tiotropium | PrimoTinA-asthma 2012 | Adding tiotropium to ICS/LABA gave modest bronchodilation and 21% fewer severe exacerbations | |
| COPD | Biologics | Mepolizumab | MATINEE 2025 | Mepolizumab reduced exacerbations by 21% in eosinophilic COPD on triple therapy, without improving symptoms | |
| COPD | Oxygen | Long-term oxygen therapy | REDOX 2024 | 24 hours a day of oxygen was no better than 15 in severe hypoxaemia | |
| COPD | Biologics | Dupilumab | BOREAS 2023 | Dupilumab cut exacerbations by 30% in eosinophilic COPD already on triple therapy | |
| COPD | Inhaled therapy | Ensifentrine | ENHANCE 2023 | Ensifentrine improved lung function in both replicate trials and reduced exacerbations, with placebo-level tolerability | |
| COPD | Inhaled therapy | Budesonide/glycopyrrolate/formoterol | ETHOS 2020 | Triple therapy beat both dual therapies, and the lower steroid dose worked as well as the higher | |
| COPD | Inhaled therapy | Fluticasone furoate/umeclidinium/vilanterol | IMPACT 2018 | Triple therapy reduced exacerbations versus either dual therapy, at the cost of more pneumonia | |
| COPD | Inhaled therapy | Indacaterol/glycopyrronium | SUNSET 2018 | Stopping inhaled steroids cost a little lung function but no extra exacerbations, except in eosinophilic patients | |
| COPD | Inhaled therapy | Beclometasone/formoterol/glycopyrronium | TRIBUTE 2018 | Extrafine triple therapy modestly reduced exacerbations versus dual bronchodilation, without excess pneumonia | |
| COPD | Inhaled therapy | Indacaterol/glycopyrronium | FLAME 2016 | Dual bronchodilation beat LABA/ICS for exacerbation prevention, with less pneumonia | |
| COPD | Oxygen | Supplemental oxygen | LOTT 2016 | Oxygen for moderate desaturation did nothing for survival, admissions, or quality of life | |
| COPD | Oral therapy | Roflumilast | REACT 2015 | Roflumilast missed its primary endpoint by a whisker and hit it on the sensitivity analysis | |
| COPD | Oral therapy | Azithromycin | MACRO 2011 | Daily azithromycin for a year cut exacerbations and improved quality of life, at the cost of hearing loss and resistance concerns | |
| COPD | Inhaled therapy | Tiotropium | POET-COPD 2011 | Tiotropium prevented exacerbations better than salmeterol | |
| COPD | Inhaled therapy | Salmeterol/fluticasone | TORCH 2007 | Salmeterol/fluticasone narrowly missed a mortality benefit but reduced exacerbations, at the cost of more pneumonia | |
| COPD | Surgery | Lung volume reduction surgery | NETT 2003 | No overall survival benefit, but a real one for upper-lobe emphysema with low exercise capacity | |
| COPD | Antibiotics | Broad-spectrum antibiotics | Anthonisen 1987 1987 | Antibiotics helped exacerbations defined by increased dyspnoea, sputum volume and purulence | |
| COPD | Oxygen | Domiciliary oxygen | MRC oxygen 1981 | Domiciliary oxygen for at least 15 hours a day improved survival in hypoxaemic cor pulmonale | |
| COPD | Oxygen | Continuous oxygen | NOTT 1980 | Continuous oxygen halved mortality compared with nocturnal-only oxygen in hypoxaemic COPD | |
| Bronchiectasis | Oral therapy | Brensocatib | ASPEN 2025 | Brensocatib cut exacerbations by about 20% — the first drug licensed specifically for bronchiectasis | |
| Bronchiectasis | Mucoactive therapy | Hypertonic saline and carbocisteine | CLEAR 2025 | Neither hypertonic saline nor carbocisteine reduced exacerbations in bronchiectasis | |
| Bronchiectasis | Inhaled antibiotics | Colistimethate sodium | PROMIS 2024 | Inhaled colistimethate reduced exacerbations in one trial and not in its replicate | |
| Bronchiectasis | Oral therapy | Azithromycin | BAT 2013 | Maintenance azithromycin roughly halved the proportion of patients having any exacerbation | |
| Bronchiectasis | Mucoactive therapy | Recombinant human DNase | rhDNase in bronchiectasis 1998 | DNase made idiopathic bronchiectasis worse, not better | |
| Cystic fibrosis | CFTR modulators | Vanzacaftor/tezacaftor/deutivacaftor | SKYLINE 2025 | The once-daily triple matched the twice-daily standard on lung function | |
| Cystic fibrosis | Mucoactive therapy | Hypertonic saline and dornase alfa withdrawal | SIMPLIFY 2023 | Patients on triple modulator therapy could stop hypertonic saline or dornase alfa without losing lung function | |
| Cystic fibrosis | CFTR modulators | Elexacaftor/tezacaftor/ivacaftor | VX17-445-102 2019 | Triple modulator therapy gave a 14-point FEV1 gain to the 90% of CF patients with a single Phe508del allele | |
| Cystic fibrosis | CFTR modulators | Ivacaftor | STRIVE 2011 | Ivacaftor produced a 10-point FEV1 gain in G551D cystic fibrosis — the first drug to correct the basic defect | |
| ILD | Prostacyclin analogues | Inhaled treprostinil | TETON-2 2026 | Inhaled treprostinil slowed FVC decline in IPF, in patients mostly already on antifibrotics | |
| ILD | Antifibrotics | Nerandomilast | FIBRONEER-ILD 2025 | Nerandomilast slowed FVC decline in progressive pulmonary fibrosis, including on top of nintedanib | |
| ILD | Antifibrotics | Nerandomilast | FIBRONEER-IPF 2025 | Nerandomilast slowed FVC decline on top of existing antifibrotics — the first genuinely additive agent in IPF | |
| ILD | Immunosuppression | Methotrexate | PREDMETH 2025 | Methotrexate matched prednisone as first-line treatment for pulmonary sarcoidosis, with a different side-effect profile | |
| ILD | Immunosuppression | Rituximab plus mycophenolate | EVER-ILD 2023 | Adding rituximab to mycophenolate improved lung function in NSIP-pattern ILD, with more viral infection | |
| ILD | Antifibrotics | Nintedanib | INBUILD 2019 | Nintedanib slowed FVC decline across progressive fibrosing ILD, whatever the underlying diagnosis | |
| ILD | Antifibrotics | Nintedanib | SENSCIS 2019 | Nintedanib slowed lung function decline in scleroderma ILD, but the result is fragile and skin disease was unchanged | |
| ILD | Antifibrotics | Pirfenidone | ASCEND 2014 | Pirfenidone slowed FVC decline and improved progression-free survival in IPF | |
| ILD | Antifibrotics | Nintedanib | INPULSIS 2014 | Nintedanib roughly halved the annual rate of FVC decline in IPF, at the cost of diarrhoea | |
| PH | Activin signalling inhibitors | Sotatercept | ZENITH 2025 | Sotatercept cut death, transplant or hospitalisation by three quarters in high-risk PAH | |
| PH | Prostacyclin analogues | Inhaled treprostinil | INCREASE 2021 | Inhaled treprostinil improved walk distance in PH due to interstitial lung disease | |
| PH | Vasodilators | Ambrisentan plus tadalafil | AMBITION 2015 | Starting two vasodilators at once halved clinical failure compared with either alone | |
| PE | Catheter intervention | Large-bore mechanical thrombectomy | PEERLESS 2025 | Mechanical thrombectomy beat catheter-directed lysis on a composite driven by less ICU use, with no mortality difference | |
| PE | Anticoagulation | Reduced-dose direct oral anticoagulant | RENOVE 2025 | Reduced-dose anticoagulation failed formal non-inferiority for recurrence but bled much less | |
| PE | Mechanical prevention | Retrievable IVC filter | PREPIC2 2015 | Adding a retrievable IVC filter to anticoagulation prevented nothing | |
| PE | Thrombolysis | Tenecteplase | PEITHO 2014 | Thrombolysis prevented decompensation in intermediate-risk PE but caused major bleeding and stroke | |
| PE | Thrombolysis | Alteplase | MAPPET-3 2002 | Alteplase reduced treatment escalation in submassive PE, but the benefit was almost entirely in that soft endpoint | |
| Pneumonia | Corticosteroids | Hydrocortisone | CAPE COD 2023 | Hydrocortisone roughly halved 28-day mortality in severe community-acquired pneumonia | |
| Pneumonia | Antibiotics | Short-course antibiotics | PneumA 2003 | Eight days of antibiotics matched fifteen for ventilator-associated pneumonia, except in non-fermenting gram-negatives | |
| COVID-19 | Antivirals | Nirmatrelvir-ritonavir | EPIC-HR 2022 | Nirmatrelvir-ritonavir cut hospitalisation or death by 89% in unvaccinated high-risk outpatients | |
| COVID-19 | Corticosteroids | Dexamethasone | RECOVERY dexamethasone 2021 | Dexamethasone reduced mortality in COVID-19 patients needing oxygen, and not in those who did not | |
| COVID-19 | Immunomodulators | Tocilizumab or sarilumab | REMAP-CAP IL-6 2021 | IL-6 receptor blockade improved organ support-free days and survival in critical COVID-19 | |
| COVID-19 | Anticoagulation | Therapeutic-dose heparin | REMAP-CAP anticoagulation 2021 | Full-dose anticoagulation did not help critically ill COVID-19 patients | |
| COVID-19 | Antivirals | Remdesivir | ACTT-1 2020 | Remdesivir shortened time to recovery by five days without a clear mortality benefit | |
| Lung cancer | Surgery | Sublobar resection | CALGB 140503 2023 | Sublobar resection matched lobectomy for small peripheral node-negative lung cancers | |
| Lung cancer | Screening | Volume CT screening | NELSON 2020 | Volume CT screening reduced lung cancer mortality with a 2.1% referral rate | |
| Lung cancer | Screening | Low-dose CT screening | NLST 2011 | Low-dose CT screening reduced lung cancer mortality — at the cost of a 96% false-positive rate | |
| Pleural disease | Pleural intervention | Conservative management | PSP 2020 | Most primary spontaneous pneumothoraces resolved without any intervention, though the non-inferiority result is fragile | |
| Pleural disease | Pleural intervention | Ambulatory device | RAMPP 2020 | Ambulatory devices sent most patients home the same day, but caused more adverse events | |
| Pleural disease | Pleural intervention | Talc poudrage | TAPPS 2020 | Talc poudrage at thoracoscopy was no better than talc slurry down a chest drain | |
| Pleural disease | Pleural intervention | Pleural manometry | Pleural manometry 2019 | Manometry during large-volume drainage did not reduce chest discomfort | |
| Pleural disease | Pleural intervention | Indwelling pleural catheter | AMPLE 2017 | Indwelling pleural catheters kept patients out of hospital more than talc pleurodesis | |
| Pleural disease | Intrapleural therapy | Alteplase plus DNase | MIST2 2011 | Combined t-PA and DNase improved drainage and cut surgical referral; neither drug worked alone | |
| Sleep medicine | Weight-loss pharmacotherapy | Tirzepatide | SURMOUNT-OSA 2024 | Tirzepatide halved the apnoea-hypopnoea index in obese patients with OSA | |
| Sleep medicine | Positive airway pressure | CPAP | SAVE 2016 | CPAP did not prevent cardiovascular events, though it treated the apnoea and improved symptoms | |
| Sleep medicine | Positive airway pressure | Adaptive servo-ventilation | SERVE-HF 2015 | Adaptive servo-ventilation abolished the central apnoeas and increased mortality | |
| Sleep medicine | Neurostimulation | Upper-airway stimulation | STAR 2014 | Hypoglossal nerve stimulation cut the AHI by two thirds — in an uncontrolled cohort | |
| Sleep medicine | Positive airway pressure | CPAP | Barbe 2012 2012 | CPAP did not significantly reduce incident hypertension or cardiovascular events in non-sleepy OSA | |
| Smoking cessation | Nicotine delivery | E-cigarettes | TEC 2019 | E-cigarettes nearly doubled one-year quit rates compared with nicotine replacement | |
| Smoking cessation | Behavioural intervention | Smoking cessation programme | Lung Health Study 2005 | A cessation programme that worked for one in five participants still reduced all-cause mortality over 14.5 years | |
| ARDS | Corticosteroids | Dexamethasone | DEXA-ARDS 2020 | Dexamethasone reduced ventilator days and mortality in established moderate-to-severe ARDS | |
| ARDS | Oxygen | Conservative oxygen targets | LOCO2 2020 | Targeting a low PaO2 in ARDS did not help and raised safety concerns | |
| ARDS | Sedation and paralysis | Cisatracurium | ROSE 2019 | Routine early paralysis added nothing when the comparator was lighter sedation | |
| ARDS | Extracorporeal support | Veno-venous ECMO | EOLIA 2018 | Immediate ECMO did not significantly beat conventional ventilation with ECMO held in reserve | |
| ARDS | Ventilator strategy | Recruitment manoeuvres with titrated PEEP | ART 2017 | Aggressive recruitment with titrated PEEP increased mortality | |
| ARDS | Ventilator strategy | Prone positioning | PROSEVA 2013 | Prone positioning halved mortality in severe ARDS | |
| ARDS | Sedation and paralysis | Cisatracurium | ACURASYS 2010 | Early cisatracurium improved adjusted 90-day survival in severe ARDS | |
| ARDS | Extracorporeal support | Referral to an ECMO centre | CESAR 2009 | Referral to an ECMO centre improved 6-month survival without disability in severe respiratory failure | |
| ARDS | Ventilator strategy | Low tidal volume ventilation | ARMA 2000 | Ventilating at 6 mL/kg instead of 12 cut ARDS mortality from 40% to 31% | |
| Ventilation & weaning | Weaning strategy | Pressure-support spontaneous breathing trial | TIP-EX 2022 | Pressure support and T-piece breathing trials produced the same ventilator-free days | |
| Ventilation & weaning | Oxygen delivery | High-flow nasal oxygen | FLORALI 2015 | High-flow oxygen did not reduce intubation but was associated with lower 90-day mortality | |
| Ventilation & weaning | Non-invasive ventilation | CPAP or bilevel NIV | 3CPO 2008 | Non-invasive ventilation made patients with pulmonary oedema feel better faster without saving lives | |
| Ventilation & weaning | Weaning strategy | Paired sedation interruption and breathing trial | Wake up and breathe 2008 | Pairing daily sedation interruption with a breathing trial got patients off ventilators sooner and improved survival | |
| Ventilation & weaning | Non-invasive ventilation | Non-invasive ventilation | Brochard 1995 1995 | Non-invasive ventilation cut intubation, length of stay and mortality in COPD exacerbations |
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