ABSTRACT
Background: Modulator therapy has improved nutritional status in individuals with cystic fibrosis (CF), which is associated with favorable outcomes. Because of the high metabolic demands of CF, nutritional recommendations include energy intake of 110% to 200% of daily estimated needs for healthy individuals. With changes in energy balance after initiation of modulator therapy, these recommendations may no longer be appropriate for some people with CFand may lead to excessiveweight gain. Overweight and obesity are being reported, and nutrition concerns now include dietary quality. Dietary quality in relation to growth in young children starting lumacaftor/ivacaftor therapy has not been examined over a 24-week period and may provide new data for future nutrition guidance for individuals with CF. Method(s): The purpose of this observational study was to determine the effect of lumacaftor/ivacaftor treatment on growth and diet in medicationnaive children. Subjects aged 2 to 5 with D508/D508 mutations were recruited from the United States and Canada. Length/height, weight, and body mass index (BMI) were measured in triplicate and averaged. Z-scores were calculated using Centers for Disease Control and Prevention reference data. Dietary data were captured using 3-day weighted food records after study visits. The Healthy Eating Index (HEI) was generated using the U.S. Department of Agriculture scoring system for each recorded day and averaged. Outcomes were assessed before treatment (baseline) and 12 and 24 weeks after beginning medication. Mixed longitudinal models were used for analysis over time. Result(s): Participants (mean age 2.9 +/- 1.4, 50% female) who completed food records for at least their baseline visit plus one other visit (n = 14) had significant increases inweight-for-age z-score (WAZ) 12 (0.6 +/- 1.7, p = 0.02) and 24 (0.21 +/- 1.8, p = 0.001) weeks after therapy. There was no significant change in height-for-age (HAZ), BMI-for-age (BMIZ), or head circumference- for-age (HCZ) z-score at 12 or 24 weeks. Although not statistically significant, percentage estimated energy requirement (%EER) decreased at 12 (-7 +/- 90%, p = 0.54) and 24 (-27 +/- 90%, p = 0.08) weeks. HEI total score did not change over the 24 weeks, although vegetables and greens and beans HEI subgroup scores decreased significantly from baseline to 24 weeks (-0.73 +/- 2.2, p = 0.02;-0.68 +/- 2.1, p = 0.02, respectively). Pooled visit correlation between total vegetables and WAZ indicated a positive association (r = 0.35, p = 0.04). Conclusion(s): WAZ increased significantly over 24 weeks of lumacaftor/ ivacaftor therapy and was positively correlated with total vegetable intake, suggesting that participants with greater WAZ scores consumed more vegetables, although over the course of the study, total vegetable intake and intake of greens and beans decreased, and WAZ increased. %EER decreased over the course of the study, but not statistically significantly so, probably because of variability in energy intake within this small study sample with some COVID-19 interruptions. In summary, WAZ of children aged 2 to 5 with D508/D508 mutations increased, with no significant changes in HAZ, BMIZ, or HCZ, and they consumed fewer total vegetables and greens and beans after 24 weeks of lumacaftor/ivacaftor therapy. Acknowledgements: Supported by Vertex Pharmaceutics Inc. and the National Center for Advancing Translational Sciences, National Institutes of Health, through Grant UL1TR001878.Copyright © 2022, European Cystic Fibrosis Society. All rights reserved
ABSTRACT
Background: NASPGHAN guidelines for fellowship training in pediatric gastroenterology (GI) highlight the importance of multidisciplinary treatment across GI conditions. Specific required medical knowledge for pediatric GI fellows includes (1) understanding of the biopsychosocial model and brain-gut axis involvement in functional GI disorders (FGIDs) and (2) familiarity with the role of psychological evaluation and interventions within the multidisciplinary management of FGIDs. Pediatric psychologists are well-suited to provide this training as education of interdisciplinary professionals is a defining competency in pediatric psychology. While pediatric GI psychologists may be informally involved in the training of GI fellows through shared multidisciplinary patient care, we sought to develop a didactic series to formalize a GI Psychology curriculum consistent with clinical training guidelines and the expressed interests of fellows at our institution. Method(s): GI psychologists and GI chief fellows at Children's Hospital of Philadelphia (CHOP) developed an 8-lecture didactic series to be incorporated into an existing weekly didactic meeting for all GI fellows. GI psychologists presented a topic quarterly over the course of 2 training years (FY20-FY21). Topics for the inaugural 2-year series were: Introduction to GI Psychology, Giving the Positive Functional Diagnosis, Cognitive Behavioral Therapy for Functional Abdominal Pain Disorders, Behavioral Interventions for Constipation and Encopresis, Psychosocial Adjustment in Inflammatory Bowel Disease, Behavioral Treatment of Rumination Syndrome, Somatic Symptom Disorders, and Treatment Adherence. In summer 2020, GI psychologists also presented an unplanned didactic session related to coping with secondary traumatic stress during the COVID-19 pandemic. For the next iteration of the 2-year series (FY22-FY23), GI psychologists selected a new topic of Feeding and Eating Difficulties: Role of GI Psychology to replace Introduction to GI Psychology. Introduction to GI Psychology was moved to a fellow orientation session. Fellows participated in the first three years of the program which spanned the inaugural 2-year series and 1 year of a second series (n = 12, 11, 11). Attendance at specific didactic sessions was not recorded. At the end of each training year, fellows completed an anonymous program evaluation survey via REDCap for ongoing quality improvement. Fellows rated 5 items assessing the impact of the didactics on their knowledge of the biopsychosocial approach, delivery of clinical impressions and recommendations, confidence with description of psychological goals and strategies, and recommendations for continuation of the GI Psychology didactics. Items were scored on a 5-point Likert scale ranging from Strongly Agree to Strongly Disagree. During the inaugural 2-year series, the survey also included items rating the value of each didactic topic. Each year the survey allowed for open-ended suggestions for additional topics. Result(s): The GI Psychology didactic series for GI fellows has been implemented successfully at CHOP for an inaugural 2-year series (FY20-FY21) with a second series currently in progress (FY22-FY23). Although survey response rate was low (33% FY20;9% FY21;45% FY22), 100% of the fellows completing the survey Agree/Strongly Agree the didactic series increased their knowledge of the biopsychosocial approach to managing GI conditions, informed how they deliver clinical impressions and treatment recommendations for patients/families, recommended continued GI Psychology involvement in GI fellow didactics and recommended other institutions consider Psychology involvement in GI fellow didactic education. Additionally, 80% of the fellows Agree/Strongly Agree the didactic series increased their confidence to describe common goals and strategies within psychological treatment for patients with GI conditions. Conclusion(s): We describe development of a novel GI Psychology curriculum for GI fellows at CHOP focused on core topics to enhance competency in the biopsychosocial approach across GI conditions, which was favorably evaluated by GI fellows. Fellowship training programs in pediatric GI may wish to consider a similar approach to incorporating didactic training from pediatric psychologists. Doing so may increase relevant medical knowledge and facilitate experience with and use of a multidisciplinary approach to evaluation and management across GI conditions, consistent with NASPGHAN clinical training guidelines and calls for best practice to incorporate integrated psychological care across GI conditions.
ABSTRACT
A 15-year-old male presented with acute onset bilateral lower extremity weakness. He felt a 'pop' in the left side of his lower back while turning in bed, followed by weakness and numbness in the left lower extremity that progressed to involve his right leg. Denied sick contacts, ingestion, or significant family history. Initial exam 6 hours after symptom onset demonstrated 0/5 strength in bilateral lower extremities, lax rectal tone, and absent lower extremity muscle stretch and cremasteric reflexes. MRI thoracic and lumbar spine with/without contrast within 24 hours of symptom onset was unremarkable. Nerve conduction studies done within 12 hours of onset demonstrated absent peroneal and tibial F waves, present median F wave, and normal motor and sensory responses in upper and lower extremities. Cerebrospinal fluid cell counts, glucose, and protein were normal. Serum studies including inflammatory markers, ANA, ds-DNA, copper, vitamin B12, methylmalonic acid, NMO and MOG antibodies were unremarkable. Nasopharyngeal COVID-19 testing was negative. Patient received 2 days of IVIG and 5 days of IV methylprednisolone. He required intermittent catheterization for neurogenic bladder prior to discharge to inpatient rehabilitation. Exam 2 months after presentation showed proximal left lower extremity strength of 4/5, distal left lower extremity strength of 2/5, with normal right lower extremity strength. Exam also showed hyperreflexia in bilateral lower extremities. Repeat MRI spine revealed a short segment of T2 hyperintensity involving T12-L1, mainly affecting the central gray matter with no enhancement. MRI brain and orbits was normal. Repeat MRI performed 1.5 months later showed decreased extent of signal abnormality limited to the anterior horns, greater on the left. Previously published cases with similar MR findings suggested an etiology for the patient's acute onset paralysis. Video recordings of the patient are available.