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2 Minute Medicine Rewind June 9, 2025
1. Respiratory outcomes for preterm infants with a patent ductus arteriosus (PDA) undergoing transcatheter closure compared to surgical ligation did not differ.
Evidence Rating Level: 2 (Good)
Transcatheter closure of PDAs has seen increased use in recent years. However, indications and the optimal timing of this procedure as well as comparisons with traditional management options remains unclear at this time. This retrospective cohort study therefore sought to compare respiratory outcomes of PDA closure via a transcatheter approach or surgical approach in extremely preterm infants. Preterm infants with gestational age between 22 weeks and less than 29 weeks from the Neonatal Research Network’s Generic Database (NRN GDB) with a diagnosis of PDA were included in this study if they underwent transcatheter closure (n = 202, median [IQR] gestational age, 25.4 [24.1-27.1] weeks; 56% female) or surgical ligation (n = 359, median [IQR] gestational age, 24.9 [24.0-25.9] weeks; 52% female). The primary outcome was total days of mechanical ventilation with secondary outcomes including days of positive pressure ventilation and length of hospital stay. Total days of mechanical ventilation was comparable between children receiving transcatheter closure vs surgical ligation (46 [30-68] days vs 46 [21-73] days). When adjusting for several factors including center, birth year and gestational age, there were similarly no significant differences in total days of mechanical ventilation between both groups (adjusted median difference, −2.65 [95% CI, −8.36 to 3.07]; P = .36). Similarly, there were no significant differences in length of hospital stay and total days of positive pressure ventilation support. Overall, this study found that respiratory outcomes did not differ in extremely preterm infants with a PDA undergoing transcatheter closure versus surgical ligation.
Use of Common Psychiatric Medications and Risk and Prognosis of Amyotrophic Lateral Sclerosis
1. Prediagnostic use of anxiolytics, hypnotics, sedatives and antidepressants were associated with a higher risk of amyotrophic lateral sclerosis (ALS).
Evidence Rating Level: 2 (Good)
While ALS has been historically recognized as a disease affecting the motor neurons, psychiatric symptoms have also been shown to be a manifestation of ALS. Studies investigating the relationship between psychiatric medication use and the risk of developing ALS are limited. This case-control study therefore sought to investigate the association between the use of anxiolytics, hypnotics, sedatives and antidepressants and the risk of developing ALS as well as the progression of ALS. 1057 case participants and 5281 controls (mean[SD] age, 67.5[11.5] years; 53.% male) were included from the Swedish MND Quality Registry between January 2015 and July 2023. Following multivariable adjustment, having at least 2 prescriptions of psychiatric medications was associated with an increased risk of developing ALS when the medications were prescribed 0-1, 1-5 and greater than 5 years before development of ALS (prescribed hypnotics and sedatives 0-1 years befor index date: odds ratio [OR], 6.10; 95% CI, 3.77-9.88; prescribed anxiolytics 1-5 years before index date: OR, 1.60; 95% CI, 1.15-2.23; prescribed antidepressants >5 years before index date: OR, 1.21; 95% CI, 1.02-1.44). Following multivariable adjustment, prescribed use of anxiolytics and antidepressants were associated with an increased risk of mortality (hazard ratio [HR], 1.52; 95% CI, 1.12-2.05 and HR, 1.72; 95% CI, 1.30-2.29, respectively). Overall, this study found that prediagnostic prescribed use of anxiolytics, hypnotics, sedatives and antidepressants were associated with higher risk of developing ALS.
1. The use of dapagliflozin in patients with metabolic dysfunction-associated steatohepatitis (MASH) resulted in a higher proportion of improvement of MASH compared to placebo.
Evidence Rating Level: 1 (Excellent)
Therapeutic options for MASH remain limited, with current management strategies focusing on the treatment of coexisting conditions such as type 2 diabetes mellitus (T2DM). While sodium-glucose cotransporter 2 (SGLT2) inhibitors are widely used in the management of T2DM and heart failure, their effectiveness in MASH remains unknown. This randomized controlled trial therefore sought to investigate the efficacy and safety of the SGLT2 inhibitor, dapagliflozin, in MASH. 154 patients (mean[SD] age, 35.1[10.2] years; 85% male) over the age of 18 with biopsy-confirmed MASH from 6 centres in China were randomized to receive dapagliflozin (n = 74) or placebo (n = 76). The primary outcome was MASH improvement (defined as a decrease in non-alcoholic fatty liver disease activity score [NAS] of at least 2 points). MASH improvement was observed in 53% of patients in the dapagliflozin group versus 30% in the placebo group (risk ratio 1.73 (95% CI 1.16 to 2.58); P=0.006), with a mean difference in NAS between the groups of -1.39 ((−1.99 to −0.79); P<0.001). The rate of adverse events was comparable between groups (56% in the dapagliflozin group compared to 64% in the placebo group). Overall, this study found that use of dapagliflozin patients with MASH resulted in a greater proportion of patients experiencing improvement of MASH compared to those receiving placebo.
1. In patients with previously treated metastatic epidermal growth factor receptor (EGFR)-mutated non-small cell lung cancer (NSCLC), treatment with sacituzumab tirumotecan (sac-TMT) resulted in improvements in objective response rate (ORR), progression-free survival (PFS) and overall survival (OS) compared to docetaxel.
Evidence Rating Level: 1 (Excellent)
In patients with EGFR-mutated NSCLC, the first-line therapeutic option is typically EGFR tyrosine kinase inhibitors. Once disease progression occurs on both EGFR tyrosine kinase inhibitors and platinum-based chemotherapy, guidelines recommend the use of docetaxel. However, as the efficacy of docetaxel remains suboptimal, there is a need for novel treatment options. This randomized controlled trial therefore sought to compare the efficacy of sac-TMT, a novel antibody-drug conjugate, against docetaxel in patients with previously treated advanced EGFR-mutated NSCLC. 137 patients (median age, 56 years; 44% male) between the ages of 18 and 75 across 48 centres in China with EGFR-mutated NSCLC previously treated with EGFR tyrosine kinase inhibitors and platinum-based chemotherapy were randomly assigned to receive either sac-TMT (n = 91) or docetaxel (n = 46). The blind independent review committee (BIRC)-assessed ORR for the sac-TMT was 45% while the BIRC-assessed ORR for the docetaxel group was 16%, with a difference of 29% (95% CI 15% to 43%; one sided P<0.001). Additionally, the sac-TMT group had a 70% reduction in risk of disease progression or death compared to the docetaxel group (hazard ratio 0.30, 95% CI 0.20 to 0.46; one sided P<0.001). The 12 month OS for the sac-TMT group was 73% (95% CI, 62% to 81%) compared to 54% (95% CI, 39% to 67%) in the docetaxel group. Overall, this study found that use of sac-TMT in patients with previously treated advanced EGFR-mutated NSCLC resulted in benefits in ORR, PFS and OS compared to docetaxel.
1. Rectoscopy alone for evaluation of endoscopic and histologic remission of ulcerative colitis (UC) had good accuracy and is sufficient for use in clinical practice.
Evidence Rating Level: 2 (Good)
In recent years, endoscopic healing has become one of the main treatment targets in UC with studies also showing that histologic healing is associated with better outcomes. International guidelines have proposed use of rectosigmoidoscopy (RS) following initiation of pharmacological management to assess for endoscopic healing, yet acceptability of RS has been limited thus far. This prospective study therefore sought to investigate whether RS and rectoscopy could adequately measure UC activity in the proximal colon. Between January 2021 and January 2023, 80 patients (median[IQR] age, 43[30-57] years) from Amiens University Hospital were included. Endoscopic healing was defined as a Mayo endoscopic score (MES) of 0 and histological healing was defined as a Nancy index less than or equal to 1. 34 of 80 included patients had an MES of 0 on both RS and colonoscopy, while 2 patients showed endoscopic healing on RS but not colonoscopy corresponding to a к index of 0.95 (%-agree 97.5, P < .0001, 0% false positives). Similarly, the agreement between rectoscopy and colonoscopy for assessment of patients with MES of 0 were also nearly perfect with a к index of 0.83 (%-agree 91.2, P < .001, 0% false positive). The agreement between RS and colonoscopy for histological remission was nearly perfect with a к index of 0.94 (%-agree 97.3, P < .001) while the agreement between rectoscopy and colonoscopy for histological remission was similarly nearly perfect with a к index of 0.84 (%-agree 93.1, P < .001). Overall, this study found that rectoscopy alone was sufficient in clinical practice for the evaluation of endoscopic and histological remission in UC.
Image: PD
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