Selumetinib is approved for the treatment of inoperable plexiform neurofibromas (PN) in patients with neurofibromatosis type 1 (NF1). However, its efficacy in treating NF1-associated diffuse neurofibromas (NF1-DN) or optic pathway gliomas (NF1-OPG) remains unclear. We evaluated the efficacy and safety of selumetinib in these subgroups.

This was a sub-analysis of a Korean phase II open-label trial focusing on non-target treatment effects on NF1-DN and NF1-OPG. A total of 88 pediatric and adult patients with NF1-PN (59 children and 29 adults) in this trial had been treated for at least 2 years (~ 26 cycles, 28-day cycle) with oral selumetinib (20 or 25 mg/m², or 50 mg/dose every 12 h). Tumor volume, quality of life (QoL), and visual acuity were assessed.

Among the 88 included patients, NF1-DN was diagnosed in 25 (28%), and NF1-OPG in 3 (3%). All NF1-DN patients exhibited disfigurement, two experienced pain, and a partial response (PR; ≥20% tumor reduction at a single time) was achieved in 9 of these cases (36%). The median time to PR was 6 cycles (range, 6–12), and the median time to best response was 18 cycles (range, 6–26), with a median volume change of − 11.9% (range, − 55.4% to + 36.3%). Confirmed PR (cPR; PR sustained for 6 cycles) was observed in 6 NF1-DN patients (24%), stable disease (SD) was observed in 9 of these patients (36%), and progressive disease (PD) in 10 cases (40%). In a paired comparison, cPR was significantly lower for NF1-DN than for NF1-PN (24% vs. 88%, P 0.001), and the median best volume reduction was also smaller (− 11.9% vs. −42.1%, P 0.001). For the 3 NF1-OPG patients, visual impairment was present in all cases at baseline. One patient achieved PR at cycle 12 (− 36.

Researchers from UCL (University College London) and the ESRF (The European Synchrotron) have produced the first three-dimensional map of the heart’s electrical wiring in Tetralogy of Fallot, one of the most common congenital heart problems, revealing anatomical features that may explain why many patients develop heart conduction disorders in this condition.

The research, part of the Human Organ Atlas international collaboration, can be used for surgical training and lead to even better outcomes for patients. The research appears in JTCVS Structural and Endovascular.

Congenital heart disease affects around 1% of the population worldwide. In many cases, babies must undergo life-saving heart surgery shortly after birth. Although survival rates are now high, many patients develop complications later in life, particularly abnormal heart rhythms or contraction patterns. Surgeons have long known that these problems can arise when the heart’s delicate electrical conduction system, which is invisible during surgery, is disturbed.

Current therapies offer limited benefit for patients with previously treated metastatic pancreatic ductal adenocarcinoma (mPDAC). Aberrant activation of the RAS pathway is the key driver of PDAC, with oncogenic RAS mutations present in more than 90% of cases. Daraxonrasib is an oral RAS(ON) multiselective, tri-complex inhibitor of the active guanosine triphosphate–bound state of mutant and wild-type RAS.

This cohort study compares real-world overall survival between sequential and concurrent radiotherapy and immunotherapy among patients with newly diagnosed advanced non–small cell lung cancer.