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1.
Artigo em Inglês | MEDLINE | ID: mdl-38924098

RESUMO

BACKGROUND: Low neighborhood income is linked with increased hospitalizations for central line-associated bloodstream infections (CLABSIs) in pediatric short bowel syndrome (SBS). We assessed whether this relationship varies by hospital center. METHODS: We performed a retrospective cohort study using the Pediatric Health Information System (2018-2023) database for patients <18 years old with SBS (N = 1210) at 24 hospitals in the United States. Using 2015 US Census data, we determined the estimated median household income of each patient's zip code. Hospital-level neighborhood income was defined as the median of the estimated median household income among patients at each hospital. We applied an extension of Cox regression to assess risk for CLABSI hospitalization. RESULTS: Among 1210 children with 5255 hospitalizations, most were <1 year on initial admission (53%), male (58%), and publicly insured (69%). Hospitals serving low-income neighborhoods served more female (46% vs 39%), Black (29% vs 22%), and Hispanic (22% vs 16%) patients with public insurance (72% vs 65%) residing in the southern United States (47% vs 21%). In univariate analysis, low hospital-level neighborhood income was associated with increased risk of CLABSI hospitalization (rate ratio [RR], 1.48; 95% CI, 1.21-1.83; P < 0.001). These findings persisted in multivariate analysis (RR, 1.43; 95% CI, 1.10-1.84; P < 0.01) after adjusting for race, ethnicity, insurance, region, and patient-level neighborhood income. CONCLUSION: Hospitals serving predominantly low-income neighborhoods bear a heavier burden of CLABSI hospitalizations for all their patients across the socioeconomic spectrum. Hospital initiatives focused on CLABSI prevention may be pivotal in addressing this disparity.

2.
Pediatr Transplant ; 28(3): e14744, 2024 May.
Artigo em Inglês | MEDLINE | ID: mdl-38566341

RESUMO

BACKGROUND: There is limited data in the literature about pediatric kidney transplant (KT) following gut transplant (GT). The purpose of this study is to highlight the technical challenges and outcomes of KT in pediatric gut recipients who developed kidney failure (KF). METHODS: A retrospective single-center study of pediatric GT recipients from January 2000 to December 2019 was performed. In total, 14 (7%) out of 206 pediatric GT recipients developed KF and were listed for KT. Ten patients underwent kidney after gut transplant (KAGT), three patients underwent simultaneous kidney and re-do gut transplant (SKAGT), and one patient died on the KT waitlist. RESULTS: 1-, 5-, and 10-year kidney graft survival was 100%, 91%, and 78%, respectively. 1-, 5-, and 10-year GT graft survival was 100%, 77%, and 77%, respectively. 1-, 5-, and 10-year patient survival was 100%, 91%, and 91%, respectively. CONCLUSION: Despite the technical complexity, KAGT and SKAGT for pediatric GT recipients that develop KF can be performed with favorable outcomes.


Assuntos
Transplante de Rim , Humanos , Criança , Estudos Retrospectivos , Transplantados , Sobrevivência de Enxerto
3.
Pediatr Transplant ; 28(3): e14756, 2024 May.
Artigo em Inglês | MEDLINE | ID: mdl-38623905

RESUMO

INTRODUCTION: Intestinal failure, defined as the loss of gastrointestinal function to the point where nutrition cannot be maintained by enteral intake alone, presents numerous challenges in children, not least the timing of consideration of intestine transplantation. OBJECTIVES: To describe the evolution of care of infants and children with intestinal failure including parenteral nutrition, intestine transplantation, and contemporary intestinal failure care. METHODS: The review is based on the authors' experience supported by an in-depth review of the published literature. RESULTS: The history of parenteral nutrition, including out-patient (home) administration, and intestine transplantation are reviewed along with the complications of intestinal failure that may become indications for consideration of intestine transplantation. Current management strategies for children with intestinal failure are discussed along with changes in need for intestine transplantation, recognizing the difficulty in generalizing recommendations due to the high level of heterogeneity of intestinal pathology and residual bowel anatomy and function. DISCUSSION: Advances in the medical and surgical care of children with intestinal failure have resulted in improved transplant-free survival and a significant fall in demand for transplantation. Despite these improvements a number of children continue to fail rehabilitative care and require intestine transplantation as life-saving therapy or when the burden on ongoing parenteral nutrition becomes too great to bear.


Assuntos
Enteropatias , Insuficiência Intestinal , Síndrome do Intestino Curto , Transplantes , Criança , Lactente , Humanos , Intestinos , Intestino Delgado , Nutrição Parenteral , Enteropatias/cirurgia , Síndrome do Intestino Curto/cirurgia
4.
J Pediatr Gastroenterol Nutr ; 78(2): 328-338, 2024 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-38374561

RESUMO

OBJECTIVES: The Starzl Network for Excellence in Pediatric Transplantation identified optimizing immunosuppression (IS) as a priority practice improvement area for patients, families, and providers. We aimed to evaluate associations between clinical characteristics, early IS, and outcomes. METHODS: We analyzed pediatric liver transplant (LT) data from 2013 to 2018 in the United Network for Organ Sharing (UNOS) and the Society of Pediatric Liver Transplantation (SPLIT) registries. RESULTS: We included 2542 LT recipients in UNOS and 1590 in SPLIT. IS choice varied between centers with steroid induction and mycophenolate mofetil (MMF) use each ranging from 0% to 100% across centers. Clinical characteristics associated with early IS choice were inconsistent between the two data sets. T-cell depleting antibody use was associated with improved 1-year graft (hazard ratio [HR] 0.50, 95% confidence interval [CI] 0.34-0.76) and patient (HR 0.40, 95% CI 0.20-0.79) survival in UNOS but decreased 1-year patient survival (HR 4.12, 95% CI 1.31-12.93) and increased acute rejection (HR 1.58, 95% CI 1.07-2.34) in SPLIT. Non-T-cell depleting antibody use was not associated with differential risk of survival nor rejection. MMF use was associated with improved 1-year graft survival (HR 0.73, 95% CI 0.54-0.99) in UNOS only. CONCLUSIONS: Variation exists in center choice of early IS regimen. UNOS and SPLIT data provide conflicting associations between IS and outcomes in multivariable analysis. These results highlight the need for future multicenter collaborative work to identify evidence-based IS best practices.


Assuntos
Transplante de Rim , Transplante de Fígado , Criança , Humanos , Rejeição de Enxerto/prevenção & controle , Sobrevivência de Enxerto , Terapia de Imunossupressão/métodos , Imunossupressores/uso terapêutico , Ácido Micofenólico/uso terapêutico
5.
Transplant Direct ; 10(3): e1589, 2024 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-38414976

RESUMO

Background: Enhanced B-cell presentation of donor alloantigen relative to presentation of HLA-mismatched reference alloantigen is associated with acute cellular rejection (ACR), when expressed as a ratio called the antigen presenting index (API) in an exploratory cohort of liver and intestine transplant (LT and IT) recipients. Methods: To test clinical performance, we measured the API using the previously described 6-h assay in 84 LT and 54 IT recipients with median age 3.3 y (0.05-23.96). Recipients experiencing ACR within 60 d after testing were termed rejectors. Results: We first confirmed that B-cell uptake and presentation of alloantigen induced and thus reflected the alloresponse of T-helper cells, which were incubated without and with cytochalasin and primaquine to inhibit antigen uptake and presentation, respectively. Transplant recipients included 76 males and 62 females. Rejectors were tested at median 3.6 d before diagnosis. The API was higher among rejectors compared with nonrejectors (2.2 ±â€…0.2 versus 0.6 ±â€…0.04, P value = 1.7E-09). In logistic regression and receiver-operating-characteristic analysis, API ≥1.1 achieved sensitivity, specificity, and positive and negative predictive values for predicting ACR in 99 training set samples. Corresponding metrics ranged from 80% to 88% in 32 independent posttransplant samples, and 73% to 100% in 20 independent pretransplant samples. In time-to-event analysis, API ≥1.1 predicted higher incidence of late donor-specific anti-HLA antibodies after API measurements in LT recipients (P = 0.011) and graft loss in IT recipients (P = 0.008), compared with recipients with API <1.1, respectively. Conclusions: Enhanced donor antigen presentation by circulating B cells predicts rejection after liver or intestine transplantation as well as higher incidence of DSA and graft loss late after transplantation.

6.
J Pediatr Gastroenterol Nutr ; 78(2): 414-427, 2024 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-38299267

RESUMO

The advent of social media has changed numerous aspects of modern life, with users developing and maintaining personal and professional relationships, following and sharing breaking news and importantly, searching for and disseminating health information and medical research. In the present paper, we reviewed available literature to outline the potential uses, pitfalls and impacts of social media for providers, scientists and institutions involved in digestive health in the domains of patient care, research and professional development. We recommend that these groups become more active participants on social media platforms to combat misinformation, advocate for patients, and curate and disseminate valuable research and educational materials. We also recommend that societies such as NASPGHAN assist its members in accessing training on effective social media use and the creation and maintenance of public-facing profiles and that academic institutions incorporate substantive social media contributions into academic promotion processes.


Assuntos
Gastroenterologia , Mídias Sociais , Criança , Humanos , Gastroenterologia/educação , Sociedades Médicas , Assistência ao Paciente , América do Norte
7.
Transplantation ; 108(4): 827-835, 2024 Apr 01.
Artigo em Inglês | MEDLINE | ID: mdl-37723639

RESUMO

Intestinal transplant (ITx) rejection lacks a reliable noninvasive biomarker and rejection surveillance relies on serial endoscopies and mucosal biopsies followed by histologic assessment. Endoscopic biopsies are also essential for identifying other ITx-related complications such as infectious, allergic, and inflammatory graft enteritis as well as post-transplant lymphoproliferative disease or graft versus host disease. In spite of its central role in ITx, published guidelines on endoscopy and biopsy are lacking and significant variability between centers in terms of timing and technical performance exists. Therefore, an international expert group convened and discussed several aspects related to the surveillance endoscopy after ITx with the aim to summarize and standardize its practice. This article summarizes these considerations on endoscopic ITx monitoring and highlights practices of surveillance and for-cause endoscopy, biopsy techniques, pathologic evaluation, potential risks and complications, outsourcing, and less-invasive monitoring techniques.


Assuntos
Rejeição de Enxerto , Enteropatias , Humanos , Rejeição de Enxerto/diagnóstico , Rejeição de Enxerto/patologia , Intestinos/transplante , Transplante Homólogo , Endoscopia Gastrointestinal/efeitos adversos , Endoscopia Gastrointestinal/métodos , Aloenxertos , Enteropatias/patologia
8.
J Pediatr ; 265: 113819, 2024 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-37940084

RESUMO

OBJECTIVE: To evaluate associations between neighborhood income and burden of hospitalizations for children with short bowel syndrome (SBS). STUDY DESIGN: We used the Pediatric Health Information System (PHIS) database to evaluate associations between neighborhood income and hospital readmissions, readmissions for central line-associated bloodstream infections (CLABSI), and hospital length of stay (LOS) for patients <18 years with SBS hospitalized between January 1, 2006, and October 1, 2015. We analyzed readmissions with recurrent event analysis and analyzed LOS with linear mixed effects modeling. We used a conceptual model to guide our multivariable analyses, adjusting for race, ethnicity, and insurance status. RESULTS: We included 4289 children with 16 347 hospitalizations from 43 institutions. Fifty-seven percent of the children were male, 21% were Black, 19% were Hispanic, and 67% had public insurance. In univariable analysis, children from low-income neighborhoods had a 38% increased risk for all-cause hospitalizations (rate ratio [RR] 1.38, 95% CI 1.10-1.72, P = .01), an 83% increased risk for CLABSI hospitalizations (RR 1.83, 95% CI 1.37-2.44, P < .001), and increased hospital LOS (ß 0.15, 95% CI 0.01-0.29, P = .04). In multivariable analysis, the association between low-income neighborhoods and elevated risk for CLABSI hospitalizations persisted (RR 1.70, 95% CI 1.23-2.35, P < .01, respectively). CONCLUSIONS: Children with SBS from low-income neighborhoods are at increased risk for hospitalizations due to CLABSI. Examination of specific household- and neighborhood-level factors contributing to this disparity may inform equity-based interventions.


Assuntos
Síndrome do Intestino Curto , Criança , Humanos , Masculino , Feminino , Síndrome do Intestino Curto/epidemiologia , Síndrome do Intestino Curto/terapia , Renda , Hospitalização , Tempo de Internação , Atenção à Saúde
9.
Curr Opin Organ Transplant ; 29(1): 43-49, 2024 Feb 01.
Artigo em Inglês | MEDLINE | ID: mdl-37823752

RESUMO

PURPOSE OF REVIEW: Advances in pediatric transplant parallel those in adult populations; however, there remain critical unique considerations and differences that require specialized knowledge and a specific skill set to optimize care afforded to the pediatric transplant candidate. We introduce general themes regarding optimization of the transplant candidate that are unique to children. RECENT FINDINGS: The pathologies leading to pediatric organ transplant candidacy differ from adults and a precise understanding of the physiologies and natural histories of such diseases is critical for optimized care. Regardless of etiology, comorbidities including malnutrition, sarcopenia, and developmental delay are seen and often require disease and organ specific approaches to management. Additionally, an understanding of the concepts of developmental immunology and their relevance to transplant is critical. SUMMARY: When looking to optimize pretransplant care, awareness of the pediatric-specific challenges by the transplant community in addition to organ- and age-specific management strategies enable the best outcomes for children awaiting solid organ transplantation.


Assuntos
Transplante de Órgãos , Adulto , Criança , Humanos , Transplante de Órgãos/efeitos adversos , Listas de Espera
11.
JPEN J Parenter Enteral Nutr ; 47(8): 1047-1055, 2023 11.
Artigo em Inglês | MEDLINE | ID: mdl-37573479

RESUMO

INTRODUCTION: The International Intestinal Failure Registry (IIFR) is an international consortium to study intestinal failure (IF) outcomes in a large contemporary pediatric cohort. We aimed to identify predictors of early (1-year) enteral autonomy. METHODS: We included IIFR pilot phase patients. IF was defined by a parenteral nutrition need for at least 60 days due to a primary gastrointestinal etiology. The primary outcome was time to enteral autonomy achievement. We built a mixed-effects Weibull accelerated failure time model with random effects by center to analyze variables associated with enteral autonomy achievement with a primary outcome of time ratio (TR). RESULTS: We included 189 patients (82% with short bowel syndrome) representing 11 international centers. Cumulative incidence of early enteral autonomy was 51.6%, and death was 6.5%. In multivariable analysis, ostomy presence (TR, 2.63; 95% CI, 1.41-4.90) was associated with increased time to enteral autonomy achievement, and Asian/Indian (TR, 0.28; 95% CI, 0.10-0.81) and Pacific Islander race (TR, 0.34; 95% CI, 0.13-0.90) were associated with decreased time to enteral autonomy achievement. In a second model in the subset with measured percentage of bowel length remaining, ostomy presence (TR, 4.21; 95% CI, 1.90-9.33) was associated with increased time to enteral autonomy achievement, whereas greater percentage of bowel remaining (TR, 0.96; 95% CI, 0.94-0.98) was associated with decreased time to enteral autonomy achievement. CONCLUSIONS: Minimizing bowel resection at initial surgery and establishing bowel continuity by ostomy reversal can effectively decrease the time to early enteral autonomy achievement in children with IF.


Assuntos
Insuficiência Intestinal , Síndrome do Intestino Curto , Humanos , Criança , Estudos Retrospectivos , Intestino Delgado/cirurgia , Intestinos , Síndrome do Intestino Curto/cirurgia
12.
JPEN J Parenter Enteral Nutr ; 47(4): 511-518, 2023 05.
Artigo em Inglês | MEDLINE | ID: mdl-36932925

RESUMO

BACKGROUND: We aimed to evaluate costs from transplant to discharge in children who had undergone intestine transplant. METHODS: We performed a cross-sectional observational study of pediatric intestine transplant recipients from 2004 through 2020, utilizing the Pediatric Health Information System database. Standardized costs were applied to all charges and converted to 2021 US dollars. We analyzed the association of cost from transplant to discharge with age, sex, race and ethnicity, length of stay, insurance type, transplant year, short bowel syndrome diagnosis, liver-containing graft, hospitalization status, and immunosuppressive regimen. Predictors with a P value <0.20 in univariable analysis were included in a multivariable model, which was reduced using backwards selection with a P value of 0.05. RESULTS: We identified 376 intestinal transplant recipients across nine centers (median age, 2 years; 44% female). Most patients had short bowel syndrome (294; 78%). The liver was included in 218 transplants (58%). Median posttransplant cost was $263,724 (interquartile range [IQR], $179,564-$384,147), and length of stay was 51.5 days (IQR, 34-77). In the final model, increased cost from transplant to hospital discharge was associated with liver-containing graft (+$31,805; P = 0.028), T-cell-depleting antibody use (+$77,004; P < 0.001), and mycophenolate mofetil use (+$50,514; P = 0.012) while controlling for insurance type and length of stay. A 60-day posttransplant hospital stay would cost an estimated $272,533. CONCLUSIONS: Intestine transplant has high immediate cost and long length of stay that varies by center, graft type, and immunosuppression regimen. Future work will examine the cost-effectiveness of various management strategies before and after transplant.


Assuntos
Sistemas de Informação em Saúde , Síndrome do Intestino Curto , Criança , Humanos , Feminino , Pré-Escolar , Masculino , Síndrome do Intestino Curto/cirurgia , Estudos Transversais , Imunossupressores/uso terapêutico , Intestinos/transplante
13.
J Pediatr Gastroenterol Nutr ; 76(5): 634-639, 2023 05 01.
Artigo em Inglês | MEDLINE | ID: mdl-36735593

RESUMO

OBJECTIVES: The objectives of this study was to describe variation in induction regimen, identify predictors of induction immunosuppression (IS) choice, and examine the impact of induction IS regimen on length of stay (LOS) and total perioperative costs in pediatric liver transplant recipients. METHODS: We analyzed liver transplant utilization data in the Pediatric Health Information System database. Patients were divided into 3 induction IS groups: (1) steroids only, (2) T-cell depleting antibody (TDA), and (3) non-TDA. We identified predictors of induction IS regimen and examined associations between each outcome and choice of induction IS. RESULTS: We analyzed 4905 liver transplant recipients (50% female, 80% under age 13 years, 42% non-Hispanic White). Most patients (3162, 64%) received steroids only induction, and about twice as many patients received a non-TDA regimen (1093, 22%) versus a TDA regimen (650, 13%). Median total perioperative costs were highest for the TDA group [$146,438 (interquartile range $113,461-$195,575)] versus the non-TDA group [$129,307 ($102,632-$173,953)] and the steroids only group ($127,049 ($98,814-$181,053)]. Compared to steroids only induction, TDA was associated with increased LOS (+2 days, P = 0.017) with no difference in cost. Non-TDA induction was associated with a decreased LOS (-3 days, P < 0.001) and increased cost (+$42,542; P < 0.001) independent of LOS. CONCLUSIONS: Compared to a steroids only induction IS regimen, non-TDA induction was associated with increased total perioperative costs, even after adjustments for LOS. Future work will combine cost and outcome data to provide decision-making support in pediatric liver transplant recipients.


Assuntos
Imunossupressores , Transplante de Fígado , Humanos , Feminino , Criança , Adolescente , Masculino , Imunossupressores/uso terapêutico , Rejeição de Enxerto , Terapia de Imunossupressão , Soro Antilinfocitário , Esteroides , Transplantados
14.
J Pediatr Pharmacol Ther ; 28(1): 55-62, 2023.
Artigo em Inglês | MEDLINE | ID: mdl-36777976

RESUMO

OBJECTIVE: Sepsis causes morbidity and mortality in pediatric patients, but timely antibiotic administration can improve sepsis outcomes. The pharmacy department can affect the time from order to delivery of antibiotics. By evaluating the pharmacy process, this study aimed to decrease the time from antibiotic order to delivery to within 45 minutes. METHODS: All antibiotic orders placed following a positive sepsis screen for acute care patients at a freestanding children's hospital from April 1, 2019, to December 31, 2019, were reviewed. Lean Six Sigma methodology including process mapping was used to identify and implement improvements, including educational interventions for providers. Outcome measures included time from antibiotic order placement to delivery and to administration. Additional assessment of process measures included evaluation of order priority, PowerPlan (an internally created order set) use, and delivery method. RESULTS: Ninety-eight antibiotic orders for 85 patients were evaluated. In an individual chart of antibiotic delivery time, a trend towards faster delivery time was observed after interventions. Stat orders (40.5 minutes [IQR, 19.5-48]) were delivered more quickly than routine orders (51 minutes [IQR, 45-65]; p < 0.001). Orders using the PowerPlan (20.5 minutes [IQR, 18.5-38]) were delivered more quickly than those that did not (47 minutes [IQR, 34-64]; p < 0.01). Shorter time to administration was observed with pneumatic tube delivery (41 minutes [IQR, 20-50]) than with direct delivery to a health care provider (51 minutes [IQR, 31-83]; p < 0.05) or to the automated dispensing cabinet's refrigerator (47 minutes [IQR, 41-62]; p < 0.0001). CONCLUSIONS: Multifactorial coordinated interventions within the pharmacy department improve medication delivery time for pediatric sepsis antibiotic orders.

15.
Pediatr Transplant ; 27(4): e14488, 2023 06.
Artigo em Inglês | MEDLINE | ID: mdl-36808684

RESUMO

INTRODUCTION: Alpha 1 antitrypsin deficiency (A1ATD) accounts for 21% of all pediatric liver transplants due to metabolic disease in the western world. Donor heterozygosity has been evaluated in adults but not to a recipient with A1ATD. METHODS: The data of patient were retrospectively analyzed and a literature review performed. RESULTS: We present a unique case of living related donation from a A1ATD heterozygote female to a child for decompensated cirrhosis due to A1ATD. In the immediate postoperative period, the child had low-alpha 1 antitrypsin levels, but these normalized by 3 months posttransplant. He is currently 19 months post-transplant with no evidence of recurrent disease. CONCLUSION: Our case provides initial evidence that A1ATD heterozygote donors may be safely used for pediatric patients with A1ATD, thus expanding the donor pool.


Assuntos
Heterozigoto , Transplante de Fígado , Doadores Vivos , Deficiência de alfa 1-Antitripsina , alfa 1-Antitripsina , Obtenção de Tecidos e Órgãos , Deficiência de alfa 1-Antitripsina/genética , Deficiência de alfa 1-Antitripsina/cirurgia , Humanos , Feminino , Criança , Masculino , alfa 1-Antitripsina/análise , alfa 1-Antitripsina/genética , Fígado/química , Fígado/patologia
16.
J Pediatr Gastroenterol Nutr ; 76(5): 672-683, 2023 05 01.
Artigo em Inglês | MEDLINE | ID: mdl-36800275

RESUMO

Iron deficiency (ID) is the most common nutritional deficiency affecting children undergoing intestinal rehabilitation (IR). Patients may be asymptomatic or present with nonspecific symptoms including fatigue, irritability, and dizziness. The diagnosis of ID in this population can be complicated by the coexistence of systemic inflammation or other nutritional deficiencies which may mimic ID. Many routinely available laboratory tests lack specificity and no consensus on screening is available. Success in oral and enteral treatment is impeded by poor tolerance of iron formulations in a population already challenged with intolerance. Newer parenteral iron formulations exhibit excellent safety profiles, but their role in repletion in this population remains unclear. The following report, compiled by a multidisciplinary group of providers caring for children undergoing IR and representing the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition Special Interest Group for Intestinal Rehabilitation, seeks to address these challenges. After discussing iron physiology and population-specific pathophysiology, we make recommendations on iron intake, iron status assessment, and evaluation for alternative causes of anemia. We then provide recommendations on iron supplementation and treatment of ID anemia specific to this nutritionally vulnerable population.


Assuntos
Anemia Ferropriva , Anemia , Deficiências de Ferro , Humanos , Criança , Opinião Pública , Ferro/uso terapêutico , Anemia Ferropriva/diagnóstico , Anemia Ferropriva/tratamento farmacológico , Anemia Ferropriva/etiologia , Anemia/etiologia
17.
Mol Genet Metab Rep ; 31: 100866, 2022 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-35782613

RESUMO

Classical phenylketonuria (PKU) presents a unique challenge for women of child-bearing age. In the context of pregnancy, poorly controlled hyperphenylalaninemia can result in a devastating constellation of outcomes for the baby referred to as the maternal PKU Syndrome. We present the case of a woman with classical PKU unable to maintain a restricted diet and refractory to pharmacological therapies. She elected to undergo a domino liver transplant, receiving an organ from a donor with classical branched chain ketoacid dehydrogenase deficiency (maple syrup urine disease). Plasma phenylalanine concentrations normalized within a few days after transplant and remained so on an unrestricted diet during the first year of follow-up. The patient reports subjective improvements in mood, energy level, and overall quality of life. In the appropriate clinical setting, liver transplant should be considered to provide metabolic stability for PKU patients, particularly women of childbearing age.

18.
JPEN J Parenter Enteral Nutr ; 46(7): 1585-1592, 2022 09.
Artigo em Inglês | MEDLINE | ID: mdl-35616293

RESUMO

BACKGROUND: Newer intravenous lipid emulsions (ILEs), such as fish oil-based intravenous lipid emulsions (FO-ILEs) and soybean oil, medium-chain triglycerides, olive oil, and fish oil-based intravenous lipid emulsions (SMOF-ILEs), provide alternatives to soybean oil-based intravenous lipid emulsions (SO-ILEs). We explored current ILE practice patterns among intestinal rehabilitation and transplant centers. METHODS: A survey was developed addressing ILE availability, ILE preference in clinical scenarios, and factors influencing ILE choice. This survey was reviewed locally and by the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition Intestinal Rehabilitation Special Interest Group, the Intestinal Rehabilitation and Transplant Association scientific committee, and the American Society of Parenteral and Enteral Nutrition pediatric intestinal failure section research committee. We recruited providers nationally and internationally from centers with and without intestinal transplant programs. RESULTS: We included 34 complete responses, 29 from the United States. Sixteen centers performed intestinal transplants. All centers had access to SMOF-ILEs, 85% had access to FO-ILEs, and 91% had access to SO-ILEs. In new patients, 85% use SMOF-ILEs as the first choice ILE. In those with new intestinal failure-associated liver disease (IFALD), FO-ILE was preferred to SMOF-ILE (56% vs 38%). In those developing IFALD on SMOF-ILE, 65% switched to FO-ILE, whereas 24% remained on SMOF-ILE. CONCLUSIONS: Centers have routine access to alternative ILEs, and these are quickly replacing SO-ILEs in all circumstances. Future work should focus on how this shift in practice affects outcomes to provide decision support in specific clinical scenarios.


Assuntos
Enteropatias , Insuficiência Intestinal , Hepatopatias , Falência Hepática , Emulsões Gordurosas Intravenosas/uso terapêutico , Óleos de Peixe/uso terapêutico , Humanos , Enteropatias/tratamento farmacológico , Hepatopatias/terapia , Azeite de Oliva , Óleo de Soja/uso terapêutico
19.
Paediatr Drugs ; 24(4): 365-376, 2022 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-35604536

RESUMO

Pediatric intestinal transplant serves as the only definitive treatment for children with irreversible intestinal failure. Successful intestinal transplant hinges upon appropriate management of immunosuppression. The indications for intestinal transplant have changed over time. Immunosuppression regimens can be divided into induction and maintenance phases along with treatment of acute rejection. Intestinal transplant induction now often includes antithymocyte globulin or basiliximab in addition to corticosteroids. Maintenance regimens continue to be dominated by tacrolimus, with additional agents used to either decrease goal tacrolimus levels to limit toxicity or as an adjunct in sensitized patients. Careful monitoring can help to limit serious complications, such as rejection, infection, and malignancy. Future work will aim to decrease variation in practice and identify methods to determine optimal immunosuppression for a particular patient. Furthermore, there is a need for non-invasive monitoring of the intestinal graft and functional assessments of immunosuppression.


Assuntos
Imunossupressores , Tacrolimo , Basiliximab , Criança , Rejeição de Enxerto/prevenção & controle , Humanos , Terapia de Imunossupressão , Imunossupressores/efeitos adversos , Tacrolimo/efeitos adversos
20.
Pediatr Transplant ; 26(4): e14256, 2022 06.
Artigo em Inglês | MEDLINE | ID: mdl-35187765

RESUMO

BACKGROUND: Sarcopenia has been associated with poor surgical outcomes but has not been studied in pediatric intestinal transplantation. We aimed to determine sarcopenia prevalence in intestinal transplant recipients and the association of sarcopenia with outcomes. METHODS: We performed a cross-sectional retrospective chart review of intestinal transplant recipients from 2000-present. We estimated total psoas muscle area (tPMA) at L3-L4 and L4-L5 from computed tomography scans prior to or in the immediate peri-operative period. Sarcopenia was defined by tPMA below the 5th percentile for age and sex. We built a Cox-proportional hazards model to determine the association between sarcopenia and patient and graft survival. RESULTS: Of the 56 intestinal transplant recipients included, 36 (64%) were sarcopenic. Graft survival was 79% at one year and 59% at five years. Overall patient survival was 86% at one year and 76% at five years. Peri-transplant sarcopenia was associated with improved graft survival (Hazard ratio 0.42, 95% confidence interval: 0.20-0.88) but not overall survival (Hazard ratio 0.47, 95% confidence interval: 0.19-1.20). CONCLUSIONS: In this first report of sarcopenia in pediatric intestinal transplant, we found a high sarcopenia prevalence without an association with worse outcomes. The potential improved graft survival in sarcopenic patients along with underlying mechanisms warrant further exploration.


Assuntos
Sarcopenia , Criança , Estudos Transversais , Humanos , Prevalência , Estudos Retrospectivos , Sarcopenia/complicações , Sarcopenia/diagnóstico , Sarcopenia/epidemiologia , Transplantados
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