Research Spotlight

Kwon, J. K., N. Trexler, J. Reisch, C. M. Pfeifer, J. Ginos, J. A. Powell, J. Veltkamp, A. Anene, N. Fernandes and L. E. Chen (2020). “Correlating Abdominal Wall Thickness and Body Mass Index to Predict Usefulness of Right Lower Quadrant Ultrasound for Evaluation of Pediatric Appendicitis.” Pediatr Emerg Care 36(3): e156-e159.

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OBJECTIVES: To inform selective and efficient use of appendix ultrasound (US) beyond adult parameters of body mass index (BMI) of less than 25 kg/m, we correlate abdominal wall thickness (AWT) with age and BMI to generate parameters for male and female children. Information presented in chart format can aid in the decision to utilize US for the evaluation of appendicitis. METHODS: In this observational study, 1600 pediatric computed tomography scans of the abdomen and pelvis were analyzed to obtain measurements of AWT in the right lower quadrant. Measurements were correlated by patient age, BMI, and sex. Results and consensus-based recommendations were presented in chart format with color-coded groupings to allow for convenient referencing in the clinical setting. RESULTS: One thousand four hundred eighty-eight computed tomography scans and AWT measurements were included. All age groups with BMI of less than 25 kg/m and all male and female groups younger than 6 years regardless of BMI had median AWT of less than 4 cm resulting in strong recommendation for US. Males older than 6 years and all female age groups with BMI of greater than 30 kg/m and female older than 15 years and BMI of greater than 25 kg/m had AWT of more than 5 cm resulting in low recommendation for US. CONCLUSIONS: While the BMI cutoff standard of less than 25 kg/m for usefulness of appendix US holds in the adult population, our data expand the acceptable range in children younger than 9 years regardless of BMI and male children with BMI up to 30 kg/m. Female children younger than 15 years with a BMI up to 30 kg/m may also be amenable to right lower quadrant US based on AWT. These parameters inform selective and efficient use of US for appendix evaluation.

Kowdley, K. V., R. Vuppalanchi, C. Levy, A. Floreani, P. Andreone, N. F. LaRusso, R. Shrestha, J. Trotter, D. Goldberg, S. Rushbrook, G. M. Hirschfield, T. Schiano, Y. Jin, R. Pencek, L. MacConell, D. Shapiro and C. L. Bowlus (2020). “A randomized, placebo-controlled, phase II study of obeticholic acid for primary sclerosing cholangitis.” J Hepatol Mar 9. pii: S0168-8278(20)30160-4. [Epub ahead of print].

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BACKGROUND & AIMS: Primary sclerosing cholangitis (PSC) is a rare, cholestatic liver disease with no currently approved therapies. Obeticholic acid (OCA) is a potent farnesoid X receptor (FXR) agonist approved for the treatment of primary biliary cholangitis. We investigated the efficacy and safety of OCA in patients with PSC. METHODS: AESOP was a phase II, randomized, double-blind, placebo-controlled, dose-finding study. Eligible patients were 18 to 75 years of age with a diagnosis of PSC and serum alkaline phosphatase (ALP) >/=2xULN and total bilirubin <2.5xULN. Patients were randomized 1:1:1 to receive placebo, OCA 1.5-3 mg, or OCA 5-10 mg once daily for a 24-week double-blind phase followed by a 2-year, long-term safety extension (LTSE). Primary endpoints were change in ALP from baseline to week 24, and safety. RESULTS: The intent-to-treat population comprised 76 patients randomized to placebo (n=25), OCA 1.5-3 mg (n=25), and OCA 5-10 mg (n=26). At week 24, serum ALP was significantly reduced with OCA 5-10 mg vs. placebo: least-square (LS) mean difference = 83.4 (standard error [SE]=40.3) U/L, 95% CI: -164.28, -2.57; p=0.043. Serum ALP was not significantly reduced with OCA 1.5-3 mg vs. placebo at week 24 (LS mean [SE] difference = -78.29 [41.81] U/L, 95% CI: -162.08, 5.50; p=0.067). Total bilirubin remained comparable to baseline in all groups. The most common treatment-emergent adverse event was dose-related pruritus (placebo, 46%; OCA 1.5-3 mg, 60%; OCA 5-10 mg, 67%). Reductions in ALP were maintained during the LTSE, and no new safety signals emerged. CONCLUSIONS: Treatment with OCA 5-10 mg reduced serum ALP in patients with PSC. Mild to moderate dose-related pruritus was the most common adverse event.

Kizaki, K., S. Uchida, A. Shanmugaraj, C. C. Aquino, A. Duong, N. Simunovic, H. D. Martin and O. R. Ayeni (2020). “Deep gluteal syndrome is defined as a non-discogenic sciatic nerve disorder with entrapment in the deep gluteal space: a systematic review.” Knee Surg Sports Traumatol Arthrosc Apr 3. [Epub ahead of print].

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URPOSE: Clinicians are not confident in diagnosing deep gluteal syndrome (DGS) because of the ambiguity of the DGS disease definition and DGS diagnostic pathway. The purpose of this systematic review was to identify the DGS disease definition, and also to define a general DGS diagnostic pathway. METHODS: A systematic search was performed using four electronic databases: PubMed, MEDLINE, EMBASE, and Google Scholar. In eligibility criteria, studies in which cases were explicitly diagnosed with DGS were included, whereas review articles and commentary papers were excluded. Data are presented descriptively. RESULTS: The initial literature search yielded 359 articles, of which 14 studies met the eligibility criteria, pooling 853 patients with clinically diagnosed with DGS. In this review, it was discovered that the DGS disease definition was composed of three parts: (1) non-discogenic, (2) sciatic nerve disorder, and (3) nerve entrapment in the deep gluteal space. In the diagnosis of DGS, we found five diagnostic procedures: (1) history taking, (2) physical examination, (3) imaging tests, (4) response-to-injection, and (5) nerve-specific tests (electromyography). History taking (e.g. posterior hip pain, radicular pain, and difficulty sitting for 30 min), physical examination (e.g. tenderness in deep gluteal space, pertinent positive results with seated piriformis test, and positive Pace sign), and imaging tests (e.g. pelvic radiographs, spine and pelvic magnetic resonance imaging (MRI)) were generally performed in cases clinically diagnosed with DGS. CONCLUSION: Existing literature suggests the DGS disease definition as being a non-discogenic sciatic nerve disorder with entrapment in the deep gluteal space. Also, the general diagnostic pathway for DGS was composed of history taking (posterior hip pain, radicular pain, and difficulty sitting for 30 min), physical examination (tenderness in deep gluteal space, positive seated piriformis test, and positive Pace sign), and imaging tests (pelvic radiographs, pelvic MRI, and spine MRI). This review helps clinicians diagnose DGS with more confidence. LEVEL OF EVIDENCE: IV.

Kirkpatrick, J. N., R. Grimm, A. M. Johri, B. J. Kimura, S. Kort, A. J. Labovitz, M. Lanspa, S. Phillip, S. Raza, K. Thorson and J. Turner (2020). “Recommendations for Echocardiography Laboratories Participating in Cardiac Point of Care Cardiac Ultrasound (POCUS) and Critical Care Echocardiography Training: Report from the American Society of Echocardiography.” J Am Soc Echocardiogr 33(4): 409-422.e404.

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Cardiac point of care ultrasound provides rapid bedside diagnosis of important cardiovascular pathology and is performed by a growing number of users in a variety of clinical settings. Echocardiographers and sonographers may be asked to play a role in training practitioners in cardiac ultrasound who come from disciplines outside of the cardiovascular field. These trainees’ backgrounds, needs, objectives, and available time can create challenges and opportunities for echocardiography laboratories. Furthermore, the presence of additional learners in the echocardiography laboratory will require additional resources. This document is the product of an American Society of Echocardiography writing group composed of representatives from cardiology, critical care medicine, emergency medicine, and cardiac anesthesiology and others, assembled to provide expert guidance. The following recommendations are intended as practical resource to assist echocardiography laboratories to train partners in the provision of high quality cardiac ultrasound: 1: Identify Trainee Needs; 2: Employ Educational Resources; 3: In General, Avoid Certifying Global Competency; 4: Count the Cost in Echocardiography Laboratory Resources; 5. Advocate for Resources to Meet Extra Needs.(Excerpt from text, p. 409; no abstract available.)

Kirby, T., D. C. Walters, M. Brown, E. Jansen, G. S. Salomons, C. Turgeon, P. Rinaldo, E. Arning, P. Ashcraft, T. Bottiglieri, J. B. Roullet and K. M. Gibson (2020). “Post-mortem tissue analyses in a patient with succinic semialdehyde dehydrogenase deficiency (SSADHD). I. Metabolomic outcomes.” Metab Brain Dis Mar 14. [Epub ahead of print].

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Metabolomic characterization of post-mortem tissues (frontal and parietal cortices, pons, cerebellum, hippocampus, cerebral cortex, liver and kidney) derived from a 37 y.o. male patient with succinic semialdehyde dehydrogenase deficiency (SSADHD) was performed in conjunction with four parallel series of control tissues. Amino acids, acylcarnitines, guanidino- species (guanidinoacetic acid, creatine, creatinine) and GABA-related intermediates were quantified using UPLC and mass spectrometric methods that included isotopically labeled internal standards. Amino acid analyses revealed significant elevation of aspartic acid and depletion of glutamine in patient tissues. Evidence for disruption of short-chain fatty acid metabolism, manifest as altered C4OH, C5, C5:1, C5DC (dicarboxylic) and C12OH carnitines, was observed. Creatine and guanidinoacetic acids were decreased and elevated, respectively. GABA-associated metabolites (total GABA, gamma-hydroxybutyric acid, succinic semialdehyde, 4-guanidinobutyrate, 4,5-dihydroxyhexanoic acid and homocarnosine) were significantly increased in patient tissues, including liver and kidney. The data support disruption of fat, creatine and amino acid metabolism as a component of the pathophysiology of SSADHD, and underscore the observation that metabolites measured in patient physiological fluids provide an unreliable reflection of brain metabolism.

Correction to: Treatment of Psoriasis with Secukinumab in Challenging Patient Scenarios: A Review of the Available Evidence.

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Upon publication, it was noted that five of the on-line supplementary figures had incorrect figure: figure legend associations. These were supplementary Figs. 6, 7, 14, 15, and 23.