Glycogen storage disease type Ib (GSD-Ib) is a rare inborn error of glycogen metabolism uniquely associated with neutropenia and neutrophil dysfunction, causing severe infections, inflammatory bowel disease (IBD), and impaired wound healing. Recently, kidney sodium-glucose co-transporter-2 (SGLT2) inhibitors such as empagliflozin known to reduce plasma levels of 1,5-anhydroglucitol (1,5-AG) and its toxic derivatives in neutrophils, have been described as a new treatment option in case reports of patients with GSD-Ib from Europe and Asia. We report our experience with an 11-year-old girl with GSD-Ib presenting with short fasting hypoglycemia, neutropenia with neutrophil dysfunction, recurrent infections, suboptimal growth, iron-deficiency anemia, and IBD. Treatment with daily empagliflozin improved neutrophil counts and function with a significant reduction in G-CSF needs. Significant improvement in IBD has led to weight gain with improved nutritional markers and improved fasting tolerance. Reduction of maximum empagliflozin dose was needed due to arthralgia. No other significant side effects of empagliflozin were observed. This report uniquely highlights the novel use of untargeted metabolomics profiling for monitoring plasma levels of 1,5-AG to assess empagliflozin dose responsiveness and guide dietary management and G-CSF therapy. Clinical improvement correlated to rapid normalization of 1,5-AG levels in plasma sustained after dose reduction. In conclusion, empagliflozin appeared to be a safe treatment option for GSD-Ib-associated neutropenia and neutrophil dysfunction. Global untargeted metabolomics is an efficient method to assess biochemical responsiveness to treatment.

Pain management is challenging for patients with sickle cell disease (SCD) who present in vaso‐occlusive crisis (VOC). Opioid therapy is highly effective, nevertheless undesirable side effects can hinder their effectiveness. Regional anesthesia with deposition of perineural anesthetic offers nociceptive blockade, local vasodilatation, and reduces the inflammatory response. Among pediatric patients, continuous peripheral nerve block (CPNB) for perioperative adjunctive analgesia is safe. Herein, we describe the trajectory of a cohort of pediatric SCD patients with opioid‐refractory upper‐extremity VOC following placement of CPNBs for analgesia; highlighting reduced opioid consumption, improved pain scores, and decreased length of hospitalization.

ObjectivesThe objective of this study was to assess communication at the bedside in the emergency room between residents and their patients in order to identify common communication gaps. We also intended to evaluate whether residents for whom English is a second language (ESL residents) communicate less effectively.MethodsA scorable checklist was developed in order to assess and identify communication gaps between the residents and their patients. Medical students observed the internal medicine and family medicine residents while they admitted patients to the medical service in the Emergency Room. Before this, medical students were trained for two weeks with a senior internist. The role of the medical student was not revealed; rather they were self-described as observers of the admission process.ResultsOver an 8 week period, 71 observations were made of 27 medicine residents. 71 patient intakes were observed, evaluating 27 residents. In 52.1% of these interactions, the residents used medical acronyms when communicating with the patients. During 66.2% of interactions, technical medical terms or expressions were used during the history taking and in only 27.6% of those cases were the terms explained at least partially. Teach back technique was not observed in any of the interactions evaluated. Data was also analyzed based on whether the doctors were ESL residents or native English speakers. ESL residents tended to use significantly more technical language than the native English speakers, but the native English speakers tended to use more acronyms.ConclusionsHow much patients understand of what their doctor says is called “health literacy.” Resident physicians often overestimate their patients’ health literacy, and this leads to communication gaps which have the potential to result in poorer health outcomes for the patients. The checklist developed for this pilot study assessed how well residents tailor their communication to their patients’ health literacy. Our assessment revealed much room for improvement. This checklist can be used as a tool to teach future residents how to better assess and take into consideration their patients’ health literacy level and as a result communicate with patients more effectively.