scholarly journals Multidisciplinary approach to screening and management of children with Fabry disease: practice at a Tertiary Children’s Hospital in China

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Qian Shen ◽  
Jialu Liu ◽  
Jing Chen ◽  
Shuizheng Zhou ◽  
Yi Wang ◽  
...  
Keyword(s):  
High Risk ◽  
Fabry Disease ◽  
Multidisciplinary Approach ◽  
Tubular Dysfunction ◽  
Renal Tubular Dysfunction ◽  
Risk Profiles ◽  
Family Screening ◽  
Children's Hospital ◽  
Triple Test ◽  
Children’S Hospital

Abstract Background Fabry disease (FD) remains poorly recognized, especially in children in China. Considering the diversity and nonspecific clinical manifestations accompanying with life-threatening aspect of this disease, methods to improve effective screening and management of the suspects are needed. This study aims to explore how it can be done effectively from a multidisciplinary perspective for children with FD at a tertiary children’s hospital in China. Methods A multidisciplinary team (MDT) of pediatric FD experts was launched at Children’s Hospital of Fudan University. Children with high-risk characteristics were referred by the MDT screening team using the dried blood spot (DBS) triple-test (α-galactosidase A, globotriaosylsphingosine, GLA gene). For newborns who were undergoing genetic testing in the hospital, the GLA gene was listed as a routine analysis gene. Evaluation, family screening, and genetic counselling were implemented after screening by the MDT management team. Results Before the establishment of the MDT, no case was diagnosed with FD in the hospital. However, twelve months following the MDT program's implementation, thirty-five children with high-risk profiles were referred for screening by DBS triple-test, with a yield of diagnosis of 14.3% (5/35). These 5 diagnosed children were referred due to a high-risk profile of pain accompanied by dermatological angiokeratoma and hypohidrosis (n = 2), pain accompanied by abnormal liver function (n = 1), pain only (n = 1), and unexplained renal tubular dysfunction (n = 1). Two neonates were detected early with GLA mutations in the hospital, with a yield of detection of 0.14% (2/1420). Furthermore, another 3 children diagnosed with FD were referred from other hospitals. Family screening of these 10 diagnosed children indicated that 9 boys inherited it from their mothers and 1 girl inherited it from her father. Four of them started to receive enzyme replacement therapy. Conclusion Screening and management of children with FD is effective based on a defined screening protocol and a multidisciplinary approach. We should pay more attention to the high-risk profiles of pain, angiokeratoma, decreased sweating, and unexplained chronic kidney disease in children.

Analysis of the Effects of Applying Federal Diagnosis-Related Grouping (DRG) Guidelines to a Population of High-Risk Newborn Infants

PEDIATRICS ◽  
1985 ◽  
Vol 76 (1) ◽  
pp. 104-109
Author(s):  
Ronald L. Poland ◽  
Robert O. Bollinger ◽  
Mary P. Bedard ◽  
Sanford N. Cohen
Keyword(s):  
High Risk ◽  
Length Of Stay ◽  
Geometric Mean ◽  
Tertiary Care ◽  
Newborn Infants ◽  
Frequency Distributions ◽  
Federal Register ◽  
Children's Hospital ◽  
Lengths Of Stay ◽  
Children’S Hospital

Length of stay data collected for high-risk newborn infants admitted to a tertiary care children's hospital neonatal unit over a 6-year period were compared with mean and outlier lengths of stay published in the Federal Register as part of a proposed system for prospective payment of hospital cost by diagnosis-related groupings (DRGs). We found that the classification system for newborns markedly underestimated the number of days required for the treatment of these infants. The use of the geometric mean instead of the arithmetic mean as the measure of central tendency was a significant contributor to the discrepancy, especially in those sub-groups with bimodal frequency distributions of lengths of stay. Another contributor to the discrepancy was the lack of inborn patients in the children's hospital cohort. The system of prospective payments, as outlined, does not take into account several factors that have a strong influence on length of stay such as birth weight (which requires more than three divisions to serve as an effective predictor), surgery, outborn status, and ventilation. Implementation of the system described in the Federal Register would severely discourage tertiary care referral hospitals from providing neonatal intensive care.

DOZ047.70: Prevalence of abnormal triple endoscopy findings for tracheoesophageal fistula patients in a multidisciplinary aerodigestive clinic

2019 ◽  
Vol 32 (Supplement_1) ◽  
Author(s):  
S Chowdhuri ◽  
B Osterbauer ◽  
M Bansal ◽  
C Hochstim ◽  
V Bhardwaj
Keyword(s):  
Los Angeles ◽  
Tracheoesophageal Fistula ◽  
Multidisciplinary Approach ◽  
Growth Failure ◽  
Interdisciplinary Approach ◽  
Type I ◽  
Abnormal Finding ◽  
Complex Needs ◽  
Children's Hospital ◽  
Children’S Hospital

Abstract Introduction Tracheoesophageal fistula (TEF) is a rare congenital defect involving malformations of the esophagus and trachea, which can be life-threatening if left untreated. Children who have undergone TEF repair often present with symptoms such as cough, vomiting, and growth failure. Although the variety of these symptoms implies a necessity for a multidisciplinary approach to care of TEF repair patients, many are still followed by a single discipline such as gastroenterology or pulmonology. The purpose of this study is to describe the experience of Children's Hospital Los Angeles Aerodigestive team in evaluating TEF patients using an interdisciplinary approach. In particular, we aim to explore the findings obtained during triple endoscopy in order to confirm the necessity of care by multiple specialties, ideally in the setting of an Aerodigestive Clinic. Methods Consent was obtained for all children (birth to 18 years) seen in the Children's Hospital Los Angeles (CHLA) Aerodigestive (AERO) Clinic between June 2016 and August 2018. All patients with a diagnosis of TEF were included in the study and data collected included: age, sex, presenting diagnoses and symptoms, and triple endoscopy findings. Results Of the first 109 patients in the AERO Clinic, 18 had TEF. Of these, 7 (39%) were female with a mean age of 3.4 years (SD 3), and 44% were Hispanic. Ten TEF patients underwent triple endoscopy and all had at least one abnormal finding. Six were diagnosed with laryngeal cleft (LC); four type I, one type II, and one was unspecified. Other abnormal findings included: bronchomalacia (7), tracheomalacia (8), esophagitis (4), gastritis (2), and duodenitis (1). Additionally, 7 had an abnormal bronchoalveolar lavage (BAL) finding, with 6 culture positive results. Conclusion TEF patients can present with feeding and respiratory difficulties months to years after their surgical repair. The triple endoscopy findings of TEF patients presented in this study illustrate that symptoms may arise from the gastrointestinal or respiratory tract. Our experience with TEF patients at CHLA indicates that a multidisciplinary approach to evaluation and management is necessary to serve the complex needs of this population.

scholarly journals Safety and tolerability of Meningococcus B vaccine in patients with chronical medical conditions (CMC)

2019 ◽  
Vol 45 (1) ◽  
Author(s):  
L. Nicolosi ◽  
C. Rizzo ◽  
G. Castelli Gattinara ◽  
N. Mirante ◽  
E. Bellelli ◽  
...  
Keyword(s):  
High Risk ◽  
Adverse Events ◽  
Clinical Importance ◽  
European Medicines Agency ◽  
Vaccine Administration ◽  
Children's Hospital ◽  
Children’S Hospital ◽  
Control And Prevention ◽  
Low Incidence ◽  
High Risk Children

Abstract Background Invasive meningococcal disease is a serious global health threat in the world; in 2016, the European Centre for Disease Control and Prevention reported 3280 confirmed cases (including 304 deaths) of Invasive Meningococcal Diseases in Europe. In Italy, in 2017 were reported 200 cases 41% of which due to menB serogroup. From January 2013 the European Medicines Agency (EMA) has authorized the marketing of the meningococcal B vaccine 4CMenB. Methods The study aimed to evaluate and complement the safety profile of 4CMenB in high risk children accessing the vaccine service of the Bambino Gesù Children’s Hospital. All individuals aged six weeks or more receiving the meningococcal 4CMenB (Bexsero®) vaccine that approached the vaccine Centre at the Bambino Gesù Children’s Hospital in Rome, were asked to participate. All parents or caregivers of vaccinated individuals in the study period, were recruited and requested to answer to a questionnaire on adverse events following immunization (AEFI) observed after 7 days, starting from the date of vaccination. Results During the study period (October 2016–October 2017), we collected 157 completed questionnaires (out of 200 distributed). Of those 132 were first doses and 25 were booster administered doses. The median age of the study population was 4.5 years (range 0.29 to 26.8 years), the majority of subjects were high-risk individuals (64%) with chronic health conditions. Overall, 311 adverse events were reported in the 7 days after vaccine administration. In particular 147 events (47%) after administration of first dose and 58 (19%) after the booster doses. A large majority of those events, were of little clinical importance and concentrated in the 24 h after vaccine administration. No hospitalizations or Emergency Department access were reported. Conclusions Results of our study demonstrated that the Bexsero® vaccine is almost well tolerated, with a low incidence of severe AEFIs. Our results also shown that the occurrence of AEFIs is similar within healthy and high risk children.

scholarly journals The Impact of Breastfeeding on the Childhood Acute Leukemia Risk Among Children Attending Alexandria University Children's Hospital

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 3733-3733
Author(s):  
Mohamed NZ Massoud ◽  
Hoda MA Hassab ◽  
Aida M M Ali ◽  
Doha N Mohamed
Keyword(s):  
High Risk ◽  
Acute Leukemia ◽  
Breast Feeding ◽  
Bottle Feeding ◽  
Risk Level ◽  
Children's Hospital ◽  
Children’S Hospital ◽  
Duration Of Breastfeeding ◽  
The Impact ◽  
Breast Fed

Abstract Objective: The present study was conducted to investigate the impact of breastfeeding on the risk of development of acute leukemia among children Admitted to Alexandria University Children's Hospital. METHODS: A case control study included 134 ALL&AML cases & 134 matched controls for age & sex from the same family relatives to evaluate socioeconomic & genetic causes of the disease. The mothers were interviewed for the completion of interview format that included: duration & patterns of breastfeeding. RESULTS: lower proportion of acute leukemia cases 59.7% were exclusively breast-fed babies as compared to 89.6% of the control subjects. However, higher proportions of index children were either predominant breast-fed 19.4% or complementary-fed 10.4%. Nearly 10.4% were bottle-fed babies as compared to none of their controls. The differences between cases & controls as regards pattern of breast feeding were statistically significant where p<0.001. As regards duration of breastfeeding 75.4% of index children were breast-fed for more than 6 months as compared to vast majority of their controls (97.8%). The difference between cases & controls was statistically significant where p <0.001. The association also was statistically significant when 2 groups (standard & high risk) were considered as regards pattern of breastfeeding where x2=13.055, p =0.004. The multivariate logistic regression analysis indicated that bottle feeding had the odds of 7.76 of being at high risk level for acute leukemia (OR=7.76, 95% CI 1.9-33.8). CONCLUSION: Exclusive breast-feeding and breast-feeding for one year are protective against acute childhood leukemia. Table 1. Bivariate and multivariate analysis for pattern & duration of breastfeeding of the studied children with acute leukemia and their controls. Pattern and duration of breastfeeding Bi variant variable Multivariate variable Type of feeding during the first 6 month of life. Cases (n=134) Control (n=134) Significance OR (95% CI) Significance No. % No. % X2 = 37.6 P>0.0001* 1 Exclusive BF 80 59.7 120 89.6 Predominant BF 26 19.4 13 9.7 1.67 (1.26 - 2.2)* X2 = 9.4 P=0.002* Complementary feeding 14 10.4 1 0.7 21 (2.8 - 436.4)* X2 = 16.13 P>0.0001* Bottle feeding 14 10.4 0 0 2.5 (2.1 - 2.9)* X2 = 19.12 P>0.0001* Duration of breastfeeding 0-≤ 3 month 2 1.7 0 0 X2 =15.331 MC P=0.0001* AN- FEP=0.18 =13.06 P=0.0003* X2=0.38 P=0.537 3-≤ 6 month 17 14.2 3 2.2 7.6 (2.1 - 33.8)* 6-12 month 16 13.3 17 12.7 1.26 0.6-2.8 More than 12 month 85 70.8 114 85.1 1 - X2: Chi-Square test FEP: Fisher's Exact test -NA-: Not Applicable *significant at P≤0.0 Disclosures No relevant conflicts of interest to declare.

Bone marrow transplantation for high risk neuroblastoma at the children's hospital of philadelphia: An update

1994 ◽  
Vol 23 (4) ◽  
pp. 323-327 ◽  
Author(s):  
Audrey E. Evans ◽  
Charles S. August ◽  
Naynesh Kamani ◽  
Nancy Bunin ◽  
Joel Goldwein ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
High Risk ◽  
Marrow Transplantation ◽  
Children's Hospital ◽  
Children’S Hospital

Pediatric Treatment Guidelines for Philadelphia Positive (Ph+) Acute Lymphoblastic Leukemia (ALL): What Are They in today’s Imatinib Era?

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 4393-4393
Author(s):  
Michael Burke ◽  
Jennifer Willert ◽  
Sunil J. Desai ◽  
Richard Kadota
Keyword(s):  
High Risk ◽  
Medical Center ◽  
Lymphoblastic Leukemia ◽  
Response To Therapy ◽  
Treatment Practices ◽  
High Risk Patients ◽  
Children's Hospital ◽  
Children’S Hospital ◽  
Risk Patients ◽  
Upfront Therapy

Abstract Background: The treatment of pediatric Philadelphia positive (Ph+) leukemias in the era of the tyrosine kinase inhibitors (TKI) continues to evolve with the role of allogeneic hematopoietic cell transplantation (allo-HCT) in these high-risk patients becoming more controversial. Ph+ acute lymphoblastic leukemia (ALL) prior to imatinib in both pediatric and adult patients has often involved intensive chemotherapy, including consolidative allo-HCT. Whether treatment strategies in 2008 have changed for these Ph+ leukemias in pediatrics, from heavily allo-HCT based to TKI based medical therapy, is presently unclear. Methods: Thirty-two pediatric centers across the United States and Canada were surveyed regarding current treatment practices for Ph+ ALL in order to explore treatment practices in 2008. The survey targeted primary pediatric oncologists and bone marrow transplant physicians regarding their treatment approach for Ph+ ALL in terms of upfront therapy, utility of allo-HCT, use of TKI (including their role in the post-HCT setting) and how response to therapy was monitored. Results: Twenty-three of the thirty-two centers completed the survey to provide a completion rate of 72% (Table 1). Twenty-two of the 27 physicians (81%) reported they do not classify patients by risk group according to age and presenting WBC (e.g. low-, intermediate- or high-risk) but rather use response to therapy to identify high risk patients, initially treating all Ph+ ALL patients the same. Eighty-one percent of survey responders recommended allo-HCT in first remission, when a matched sibling donor was available, for Ph+ ALL. Regarding the use of TKI in the post-HCT setting, 13 of 27 (48%) physicians reported using imatinib as maintenance therapy post-HCT as a means to prevent relapse. All physicians reported using PCR techniques for bcr-abl of either bone marrow, peripheral blood or both to monitor treatment response with frequencies ranging from monthly to every six months. Conclusion: Treatment of pediatric Ph+ ALL appears variable and center dependent. Classifying patients into low-, intermediate- or high-risk disease based on age and presenting WBC was not shown to be standard practice but rather using treatment response to identify high-risk patients. This survey identified a trend toward less allo-HCT in 2008 for Ph+ ALL compared to years past. Despite the trend toward less HCT, the treatment consensus in 2008 for pediatric Ph+ ALL remains MSD allo-HCT when available. Use of imatinib was recognized by all survey responders as standard of care in upfront therapy for Ph+ ALL, but the use of imatinib or other TKI in the post-HCT setting as maintenance therapy remains in question. Prospective pediatric clinical trials will be necessary to determine the optimal strategy for the Ph+ diseases. Table 1. Pediatric Centers British Columbia’s Children’s Hospital Children’s Hospital of Pittsburgh Children’s Memorial Medical Center–Northwestern Cincinnati Children’s Hospital Medical Center City of Hope Columbia Presbyterian College of Phys & Surgeons Doernbecher Children’s Hospital-OHSU Duke University Medical Center Mayo Clinic Medical College of Wisconsin Nationwide Children’s Hospital Schneider Children’s Hospital St. Jude Children’s Research Hospital Stollery Children’s Hospital–Edmonton Texas Children’s Cancer Center at Baylor College of Medicine The Children’s Hospital of Philadelphia The University of Chicago Comer Children’s Hospital University of California at San Diego/Rady Children’s Hospital San Diego UCSF School of Medicine University of Florida University of Michigan–C.S. Mott Children’s Hospital University of Minnesota Children’s Hospital, Fairview Washington University–St. Louis

Simulation perspective on new latent safety threats in high-risk patient care scenarios during the COVID-19 pandemic

2020 ◽  
pp. bmjstel-2020-000687
Author(s):  
Melissa Love Benbow ◽  
Shruti Kant ◽  
Heidi Werner ◽  
Nicolaus Glomb
Keyword(s):  
High Risk ◽  
San Francisco ◽  
High Risk Patient ◽  
Protective Equipment ◽  
Children's Hospital ◽  
Children’S Hospital ◽  
Inclusive Approach ◽  
Communication Challenges ◽  
Primary Focus ◽  
Simulation Practice

The Code Simulation team at University of California, San Francisco (UCSF) Benioff Children’s Hospital-San Francisco is presenting a perspective on COVID-19 related simulation in a paediatric emergency department (PED) setting. The primary focus was personal protective equipment (PPE) usage in the setting of new latent safety threats in high-risk scenarios in relation to the COVID-19 pandemic. We addressed communication challenges and trialled new workflows in relation to the COVID-19 pandemic. The perspective details the objectives, themes and lessons learnt during this process. The simulation practice occurred multiple times over multiple days with an interpersonal, interdisciplinary and inclusive approach. The results of this work were implemented into practice in the PED at UCSF Benioff Children’s Hospital-San Francisco setting and influenced hospital-wide education on PPE usage during the acute phase of the COVID-19 pandemic.

scholarly journals Lessons Learned from a Collaborative to Develop a Sustainable Simulation-Based Training Program in Neonatal Resuscitation: Simulating Success

Children ◽  
2021 ◽  
Vol 8 (1) ◽  
pp. 39
Author(s):  
Nandini Arul ◽  
Irfan Ahmad ◽  
Justin Hamilton ◽  
Rachelle Sey ◽  
Patricia Tillson ◽  
...  
Keyword(s):  
Multidisciplinary Approach ◽  
Neonatal Resuscitation ◽  
Individual Performance ◽  
Lessons Learned ◽  
Hospital System ◽  
In Situ Simulation ◽  
Children's Hospital ◽  
Resuscitation Training ◽  
Simulation Based ◽  
Children’S Hospital

Newborn resuscitation requires a multidisciplinary team effort to deliver safe, effective and efficient care. California Perinatal Quality Care Collaborative’s Simulating Success program was designed to help hospitals implement on-site simulation-based neonatal resuscitation training programs. Partnering with the Center for Advanced Pediatric and Perinatal Education at Stanford, Simulating Success engaged hospitals over a 15 month period, including three months of preparatory training and 12 months of implementation. The experience of the first cohort (Children’s Hospital of Orange County (CHOC), Sharp Mary Birch Hospital for Women and Newborns (SMB) and Valley Children’s Hospital (VCH)), with their site-specific needs and aims, showed that a multidisciplinary approach with a sound understanding of simulation methodology can lead to a dynamic simulation program. All sites increased staff participation. CHOC reduced latent safety threats measured during team exercises from 4.5 to two per simulation while improving debriefing skills. SMB achieved 100% staff participation by identifying unit-specific hurdles within in situ simulation. VCH improved staff confidence level in responding to neonatal codes and proved feasibility of expanding simulation across their hospital system. A multidisciplinary approach to quality improvement in neonatal resuscitation fosters engagement, enables focus on patient safety rather than individual performance, and leads to identification of system issues.

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