Evaluating the impact of CRBN mutations on response to immunomodulatory drugs and novel CRBN-binding agents in myeloma.

Immunomodulatory drug (IMiD) resistance is a key clinical challenge in myeloma treatment. Previous data suggests almost one third of myeloma patients acquire mutations in the key IMiD effector cereblon by the time they are pomalidomide refractory. Some events, including stop codons/frameshift mutations and copy loss, having clearly explicable effects on cereblon function.

[Diabetes mellitus and obesity: risk optimization before surgical interventions].

Background: The prevalence of obesity has increased significantly in recent years and is a causal risk factor for the development of type 2 diabetes. Moreover, chronic degenerative joint diseases are also triggered by obesity.

Weight Loss: Both obesity-related secondary diseases-type 2 diabetes and chronic degenerative joint disease-can be prevented or at least delayed by lifestyle intervention aimed at weight reduction.

Depression in children with abdominal pain in the emergency department.

Objective: This cross-sectional study examined depression and associated impairment in youth presenting to a pediatric emergency department (PED) with abdominal pain.

Methods: Participants were 11-17 years old, presenting to a PED with idiopathic abdominal pain. Participants completed demographics, pain, pain-related impairment, and depression surveys.

Assessing Experiences With Trofinetide for Rett Syndrome: Interviews With Caregivers of Participants in Clinical Trials.

Purpose: Rett syndrome (RTT) is a rare neurodevelopmental disorder that mainly affects girls and women. Trofinetide is approved for the treatment of RTT in adults and children aged ≥2 years. To gain insight into experiences with RTT and effects of trofinetide treatment at different stages of RTT, interviews with caregivers of individuals with RTT were conducted upon their exit from the open-label trofinetide trials.