Learning Lab NEJM Visual Abstracts

In a trial involving 215 patients with a median age of 16 years, fingolimod was superior to interferon beta-1a in reducing relapses of multiple sclerosis and the accumulation of new lesions on MRI over a 2-year period. Seizures occurred in 5.6% of patients in the fingolimod group.

In a randomized trial, patients with chest pain underwent a standard diagnostic evaluation with or without coronary CT angiography. The group assigned to coronary CT angiography had a lower rate of coronary heart disease death or nonfatal myocardial infarction at five years.

The antiinflammatory small molecule ibudilast was tested in a phase 2 trial in patients with progressive multiple sclerosis. The rate of brain atrophy over 96 weeks was lower with ibudilast than with placebo. Side effects with ibudilast included GI symptoms and depression.

Patients discharged from the hospital carry an increased risk of venous thromboembolism after discharge.  A randomized trial of outpatient rivaroxaban for six weeks after discharge from the hospital had no significant effect on the risk of venous thromboembolism compared to placebo.

In a randomized trial, 706 patients with an acute myocardial infarction and cardiogenic shock were assigned to either culprit-lesion-only PCI or immediate multivessel PCI. At one year, there was no difference in mortality rates between the two groups.

In a randomized trial involving 8014 patients with out-of-hospital cardiac arrest, the use of epinephrine resulted in a significantly higher rate of 30-day survival than placebo but not in the rate of survival with a favorable neurologic outcome.

The FDA has recommended a streamlined trial design for assessing drugs’ safety and efficacy in patients with multidrug-resistant HIV infection, and ibalizumab has recently undergone such a trial. But clinicians and patients should recognize this design’s limitations.

In this double-blind, randomized trial in Bangladesh, maternal vitamin D supplementation from midpregnancy until birth or 6 months post partum did not improve fetal or infant growth.

A randomized trial showed that overall survival with sunitinib alone was noninferior to overall survival with sunitinib after nephrectomy among patients with metastatic renal-cell carcinoma.

In a randomized trial involving patients who were hypotensive after trauma, 30- day mortality was 33% among patients who received standard crystalloid-based resuscitation as compared with 23% among patients who received fresh frozen plasma in addition to standard measures.

In this international, randomized trial, patients with minor stroke or TIA who received clopidogrel plus aspirin had a lower risk of major ischemic events but a higher risk of major hemorrhage at 90 days than those who received aspirin alone.

In a trial, the median metastasis-free survival among men with nonmetastatic, castration-resistant prostate cancer and a short PSA doubling time was 36.6 months with enzalutamide and 14.7 months with placebo. Falls and heart problems were more common with enzalutamide.

At 8 years of follow-up, premenopausal women with breast cancer had higher rates of disease-free and overall survival with the addition of ovarian suppression to antiestrogen therapy and a higher rate of hormonal side effects than with tamoxifen alone.

Among patients with hepatocellular carcinoma whose disease had progressed during receipt of sorafenib or other systemic therapy, median overall survival was 10.2 months with cabozantinib and 8.0 months with placebo. High-grade adverse events were as previously noted for the drug.

Patients undergoing major abdominal surgery received restrictive or liberal intravenous fluids during surgery and up to 24 hours thereafter. The restrictive regimen did not improve disability-free survival and resulted in increased acute kidney injury.

In a randomized trial of patients with primary biliary cholangitis, bezafibrate and ursodeoxycholic acid resulted in a higher rate of complete biochemical response than ursodeoxycholic acid alone. Bezafibrate was associated with increases in creatinine and myalgias.

The addition of pembrolizumab to chemotherapy for metastatic lung cancer without EGFR or ALK mutations resulted in better progression-free and overall survival than chemotherapy alone. Immune-related adverse effects were more common with the combination.

This trial compared extracorporeal membrane oxygenation with non-ECMO ventilator care in patients with severe ARDS. There was no significant betweengroup difference in 60-day mortality. Interpretation was made difficult by crossovers from control to ECMO treatment.

Inhaled glucocorticoid plus β-agonist in a single inhaler was compared with maintenance inhaled glucocorticoid for exacerbation risk among patients with mild asthma. Combination therapy was noninferior to maintenance therapy.

A blinded trial involving patients with resected stage III melanoma showed that adjuvant pembrolizumab every 3 weeks for a year was associated with a 1-year recurrence-free survival rate of 75%, as compared with 61% for placebo.

Verubecestat, an orally administered inhibitor of BACE-1, reduces amyloid concentration in the cerebrospinal fluid. In a randomized, 78-week trial involving patients with mild or moderate Alzheimer’s disease, the drug did not slow cognitive decline as compared with placebo.

In a randomized trial involving patients with acute stroke, the incidence of revascularization was higher with tenecteplase than with alteplase for intravenous thrombolysis before endovascular thrombectomy. Cerebral hemorrhage occurred at the same rate in each group.

Despite the benefits of rotavirus vaccination, concerns about intussusception are an important consideration. In this study, the risk of intussusception associated with rotavirus vaccination was assessed in African countries in which the vaccine had recently been rolled out.

In a randomized trial, 366 patients with advanced heart failure received a centrifugal- or axial-flow LVAD. At 2 years, the centrifugal-flow LVAD was superior with regard to survival free of disabling stroke or reoperation to replace or remove a malfunctioning device.

This randomized, controlled trial showed that, among patients with malignant pleural effusion, the insertion of talc into the pleural space through an indwelling catheter resulted in a higher frequency of successful pleurodesis than placebo.

Among patients with MCL, many of whom were at high risk, ibrutinib plus venetoclax led to a complete response in 62% at week 16. Among patients with a response, response was ongoing in 78% at 15 months. Low-grade diarrhea, fatigue, and nausea or vomiting were side effects.

Globally, cryptococcal meningitis is a leading cause of mortality among people with AIDS, despite the availability of effective amphotericin B–based therapy. In this trial in sub-Saharan Africa, the efficacy of two simpler treatment regimens was assessed.

Preventing mother-to-child transmission of hepatitis B virus is an opportunity to prevent lifelong infection and associated complications. This randomized, placebo-controlled trial in Thailand assessed tenofovir to prevent HBV infection in infants.

Whether hydrocortisone reduces mortality among patients with septic shock is unclear. Patients with septic shock undergoing mechanical ventilation were assigned to receive an infusion of hydrocortisone or placebo. Hydrocortisone did not result in lower 90-day mortality.

In this trial in patients with stroke who had a mismatch between sizes of early infarction and a hypoperfused brain region, endovascular thrombectomy 6 to 16 hours after stroke onset was associated with a favorable shift in the distribution of disability scores at 90 days.

In a randomized trial, 398 patients with heart failure and atrial fibrillation were assigned to either catheter ablation or medical therapy. Catheter ablation significantly reduced the primary outcome of death from any cause or hospitalization for worsening heart failure.

In a randomized trial, solanezumab, a humanized monoclonal antibody against soluble amyloid, did not slow cognitive decline over a period of 80 weeks in patients with mild Alzheimer’s disease and with PET or CSF biomarkers of amyloid-related disease.

The addition of HIPEC with cisplatin to cytoreductive surgery in stage III ovarian cancer resulted in recurrence-free survival that was longer by 3.5 months and overall survival that was longer by nearly a year than surgery alone and did not result in higher rates of side effects.

In this longer-term follow-up of a placebo-controlled, randomized trial of early inhaled budesonide for the prevention of bronchopulmonary dysplasia in extremely preterm infants, budesonide had no significant effect on rates of neurodevelopmental disability at 2 years.

Scleroderma is a life-threatening autoimmune disease in need of more effective treatment. A randomized trial of myeloablative therapy followed by autologous CD34+ hematopoietic stem-cell transplantation showed outcomes that were superior to those with monthly cyclophosphamide.

An AAV5 vector with a functional factor VIII gene was tested in men with hemophilia A. Men receiving the high dose had a factor VIII level above 5 IU per deciliter at 1 year; most had a normal level. Toxic effects were minimal, and bleeding episodes nearly eliminated.

Among patients who had multivessel coronary disease and acute myocardial infarction with cardiogenic shock, the 30-day risk of death or renal-replacement therapy was lower among those who underwent PCI of the culprit lesion only than among those who underwent multivessel PCI.

Patients with proximal deep-vein thrombosis were assigned to undergo anticoagulation either alone or combined with pharmacomechanical thrombolysis. After 6 to 24 months, there was no significant between-group difference in the incidence of the post-thrombotic syndrome.

Patients with homozygous Phe508del cystic fibrosis were assigned to receive combination tezacaftor–ivacaftor or placebo for 24 weeks. The combination resulted in an FEV1 that was 4 percentage points higher and a pulmonary- exacerbation rate that was 35% lower than with placebo.

The addition of bevacizumab to lomustine in patients with recurrent glioblastoma failed to increase overall survival but was associated with a small increase in progression-free survival.

Patients with early-stage COPD were assigned to usual care plus tiotropium or 4 placebo. Tiotropium resulted in better FEV1 values. The annual decline in the 5 prebronchodilator FEV1 was similar in the two groups, but a benefit from 6 tiotropium was seen in postbronchodilator FEV1.

Alectinib, a potent ALK tyrosine kinase inhibitor, was more effective and somewhat less toxic than crizotinib when used as primary therapy in patients with ALK-positive non–small-cell lung cancer. It also reduced the risk of CNS relapse.