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Chapter 43 - Liver Transplantation in Children: Indications and Surgical Aspects
- from Section V - Other Considerations and Issues in Pediatric Hepatology
- Edited by Frederick J. Suchy, Ronald J. Sokol, William F. Balistreri
- Edited in association with Jorge A. Bezerra, Cara L. Mack, Benjamin L. Shneider
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- Book:
- Liver Disease in Children
- Published online:
- 19 January 2021
- Print publication:
- 18 March 2021, pp 801-815
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Summary
Liver transplantation is the standard of care for end-stage liver disease in children and the vast majority of transplant recipients experience successful outcomes. Better preoperative care, enhanced surgical techniques, and improved immunosuppression strategies have led to the appropriate expectation of good short- and long-term outcomes. Increased indications for liver transplant have led to an ever-increasing number of liver transplant candidates and evaluation of past policies and practices has necessitated changes in allocation policy. Collaboration has also led to better outcomes. Fortunately, continued efforts to expand the use of the donor pool through varied surgical techniques and advanced technology allows more children to benefit from this life-saving and enhancing procedure.
2092 A multicenter study of fecal microbiota transplantation for Clostridium difficile infection in children
- Maribeth R. Nicholson, Erin Alexander, Mark Bartlett, Penny Becker, Zev Davidovics, Elizabeth E. Knackstedt, Michael Docktor, Michael Dole, Grace Felix, Jonathan Gisser, Suchitra Hourigan, Kyle Jensen, Jess Kaplan, Judith Kelsen, Melissa Kennedy, Sahil Khanna, McKenzie Leier, Jeffery Lewis, Ashley Lodarek, Sonia Michail, Paul Mitchell, Maria Oliva‐Hemker, Tiffany Patton, Karen Queliza, Namita Singh, Aliza Solomon, David Suskind, Steven Werlin, Richard Kellermayer, Stacy Kahn
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- Journal:
- Journal of Clinical and Translational Science / Volume 2 / Issue S1 / June 2018
- Published online by Cambridge University Press:
- 21 November 2018, p. 64
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- Article
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OBJECTIVES/SPECIFIC AIMS: Clostridium difficile infection (CDI) is the most common cause of antibiotic-associated diarrhea and an increasingly common infection in children in both hospital and community settings. Between 20% and 30% of pediatric patients will have a recurrence of symptoms in the days to weeks following an initial infection. Multiple recurrences have been successfully treated with fecal microbiota transplantation (FMT), though the body of evidence in pediatric patients is limited primarily to case reports and case series. The goal of our study was to better understand practices, success, and safety of FMT in children as well as identify risk factors associated with a failed FMT in our pediatric patients. METHODS/STUDY POPULATION: This multicenter retrospective analysis included 373 patients who underwent FMT for CDI between January 1, 2006 and January 1, 2017 from 18 pediatric centers. Demographics, baseline characteristics, FMT practices, C. difficile outcomes, and post-FMT complications were collected through chart abstraction. Successful FMT was defined as no recurrence of CDI within 60 days after FMT. Of the 373 patients in the cohort, 342 had known outcome data at two months post-FMT and were included in the primary analysis evaluating risk factors for recurrence post-FMT. An additional six patients who underwent FMT for refractory CDI were excluded from the primary analysis. Unadjusted analysis was performed using Wilcoxon rank-sum test, Pearson χ2 test, or Fisher exact test where appropriate. Stepwise logistic regression was utilized to determine independent predictors of success. RESULTS/ANTICIPATED RESULTS: The median age of included patients was 10 years (IQR; 3.0, 15.0) and 50% of patients were female. The majority of the cohort was White (89.0%). Comorbidities included 120 patients with inflammatory bowel disease (IBD) and 14 patients who had undergone a solid organ or stem cell transplantation. Of the 336 patients with known outcomes at two months, 272 (81%) had a successful outcome. In the 64 (19%) patients that did have a recurrence, 35 underwent repeat FMT which was successful in 20 of the 35 (57%). The overall success rate of FMT in preventing further episodes of CDI in the cohort with known outcome data was 87%. Unadjusted predictors of a primary FMT response are summarized. Based on stepwise logistic regression modeling, the use of fresh stool, FMT delivery via colonoscopy, the lack of a feeding tube, and a lower number of CDI episodes before undergoing FMT were independently associated with a successful outcome. There were 20 adverse events in the cohort assessed to be related to FMT, 6 of which were felt to be severe. There were no deaths assessed to be related to FMT in the cohort. DISCUSSION/SIGNIFICANCE OF IMPACT: The overall success of FMT in pediatric patients with recurrent or severe CDI is 81% after a single FMT. Children without a feeding tube, who receive an early FMT, FMT with fresh stool, or FMT via colonoscopy are less likely to have a recurrence of CDI in the 2 months following FMT. This is the first large study of FMT for CDI in a pediatric cohort. These findings, if confirmed by additional prospective studies, will support alterations in the practice of FMT in children.
Cost-Effectiveness Analysis of Fecal Microbiota Transplantation for Recurrent Clostridium difficile Infection
- Raghu U. Varier, Eman Biltaji, Kenneth J. Smith, Mark S. Roberts, M. Kyle Jensen, Joanne LaFleur, Richard E. Nelson
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- Journal:
- Infection Control & Hospital Epidemiology / Volume 36 / Issue 4 / April 2015
- Published online by Cambridge University Press:
- 02 January 2015, pp. 438-444
- Print publication:
- April 2015
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OBJECTIVE
Clostridium difficile infection (CDI) places a high burden on the US healthcare system. Recurrent CDI (RCDI) occurs frequently. Recently proposed guidelines from the American College of Gastroenterology (ACG) and the American Gastroenterology Association (AGA) include fecal microbiota transplantation (FMT) as a therapeutic option for RCDI. The purpose of this study was to estimate the cost-effectiveness of FMT compared with vancomycin for the treatment of RCDI in adults, specifically following guidelines proposed by the ACG and AGA.
DESIGNWe constructed a decision-analytic computer simulation using inputs from the published literature to compare the standard approach using tapered vancomycin to FMT for RCDI from the third-party payer perspective. Our effectiveness measure was quality-adjusted life years (QALYs). Because simulated patients were followed for 90 days, discounting was not necessary. One-way and probabilistic sensitivity analyses were performed.
RESULTSBase-case analysis showed that FMT was less costly ($1,669 vs $3,788) and more effective (0.242 QALYs vs 0.235 QALYs) than vancomycin for RCDI. One-way sensitivity analyses showed that FMT was the dominant strategy (both less expensive and more effective) if cure rates for FMT and vancomycin were ≥70% and <91%, respectively, and if the cost of FMT was <$3,206. Probabilistic sensitivity analysis, varying all parameters simultaneously, showed that FMT was the dominant strategy over 10, 000 second-order Monte Carlo simulations.
CONCLUSIONSOur results suggest that FMT may be a cost-saving intervention in managing RCDI. Implementation of FMT for RCDI may help decrease the economic burden to the healthcare system.
Infect Control Hosp Epidemiol 2014;00(0): 1–7
Chapter 43 - Liver transplantation in children: indications and surgical aspects
- from Section V - Other considerations and issues in pediatric hepatology
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- By M. Kyle Jensen, University of Utah, Primary Children’s Medical Center, Salt Lake City, UT, USA, Maria H. Alonso, Department of Pediatrics, Northwestern University, Chicago, and Director of Hepatology, Department of Gastroenterology, Hepatology and Nutrition, Children’s Memorial Hospital, Chicago, IL, USA, Jaimie D. Nathan, Department of Surgery, University of Cincinnati College of Medicine, Cincinnati, OH, USA, Frederick C. Ryckman, Pediatric Surgery Training Program, Pediatric Liver Care Center, Cincinnati Children’s Hospital, Cincinnati, OH, USA, Gregory M. Tiao, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, USA, William F. Balistreri, University of Cincinnati College of Medicine
- Edited by Frederick J. Suchy, University of Colorado Medical Center, Ronald J. Sokol, University of Colorado Medical Center, William F. Balistreri
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- Book:
- Liver Disease in Children
- Published online:
- 05 March 2014
- Print publication:
- 20 February 2014, pp 760-772
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Summary
Introduction
Liver transplantation has become the standard of care for end-stage liver disease in children and successful outcomes are now achieved in the vast majority of transplant recipients. Progressive improvement has occurred through better preoperative care of patients with liver disease, improved operative techniques that has allowed the donor pool to expand, and improved immunosuppression strategies to prevent rejection while avoiding complications of over-immunosuppression. The success of the past, however, has also bred unique challenges for the future. With the increasing number of liver transplant candidates, improved donor awareness and organ availability must occur. A delicate balance between the risks assumed by living donors and the needs of their children must be struck. The increasing numbers of surviving patients present unique challenges and complications related to lifelong immunosuppression. The future success of pediatric liver transplantation will require appreciation of the increasingly complex care needs of this population and a national focus on donor organ shortages.
The evaluation process
Collective experience suggests that the progression of chronic liver disease is not linear, but rather exponential, suggesting that early warning signs of hepatic compromise, such as deteriorating synthetic function or refractory nutritional failure should lead to prompt evaluation. In children with acute liver failure (ALF) or rapidly progressive decompensation of chronic disease, aggressive critical care intervention is essential to maintain all other physiologic systems until a suitable donor organ becomes available.