RESUMO
OBJECTIVES: To assess outcomes following liver transplantation for maple syrup urine disease by determining attainment and sustainability of metabolic control and apply an "ideal" outcome composite in long-term survivors. STUDY DESIGN: A single center, retrospective review collected clinical data including branched-chain amino acid (leucine, isoleucine, and valine) levels following liver transplant and determined achievement of an ideal long-term outcome profile of a first allograft stable on immunosuppression monotherapy, normal growth, and absence of common transplant-related sequelae. RESULTS: Of 77 patients meeting inclusion criteria identified, 23 were long-term (≥10-year) survivors and were additionally assessed for ideal outcome attainment. Patient and graft survival were 100% and 99%, respectively, and all patients were on an unrestricted protein intake diet. Although significant variation was noted in mean isoleucine (P < .01) and leucine (P < .05) levels postliver transplantation, no difference was seen in valine (P = .29) and overall clinical impact was likely negligible as metabolic stability was achieved and sustained beyond 3 years postliver transplantation and no metabolic crises were identified. Of 23 long-term survivors with available data, 9 (39%) achieved all composite metrics determined to define "ideal" outcomes in pediatric postliver transplantation populations. CONCLUSIONS: Liver transplant enables long-term metabolic stability for patients with maple syrup urine disease. A combination of experience and improvement in both pre- and postliver transplantation care has enabled excellent survival and minimal comorbidities following transplant.
Assuntos
Aminoácidos de Cadeia Ramificada/metabolismo , Transplante de Fígado , Doença da Urina de Xarope de Bordo/metabolismo , Doença da Urina de Xarope de Bordo/cirurgia , Adolescente , Biomarcadores/metabolismo , Criança , Pré-Escolar , Feminino , Seguimentos , Sobrevivência de Enxerto , Humanos , Lactente , Masculino , Doença da Urina de Xarope de Bordo/diagnóstico , Doença da Urina de Xarope de Bordo/mortalidade , Complicações Pós-Operatórias/diagnóstico , Complicações Pós-Operatórias/epidemiologia , Estudos Retrospectivos , Sobreviventes , Resultado do Tratamento , Adulto JovemRESUMO
OBJECTIVES: To describe characteristics of liver transplant patients with severe sepsis in the PICU. DESIGN: Retrospective descriptive analysis. SETTING: Tertiary children's hospital PICU. PATIENTS: Liver transplant recipients admitted January 2010 to July 2016 for pediatric severe sepsis. INTERVENTIONS: None. MEASUREMENTS AND MAIN RESULTS: Between January 2010 and July 2016, 173 liver transplants were performed, and 36 of these patients (21%) were admitted with severe sepsis (54 episodes total). Median age at admission was 2 years (1-6.5 yr), 47.2% were male. Bacterial infections were the most common (77.8%), followed by culture negative (12.9%) and viral infections (7.4%). Fungal infections accounted for only 1.9%. Median time from transplant for viral and culture negative infections was 18 days (8.25-39.75 d) and 25 days (9-41 d), whereas 54.5 days (17-131.25 d) for bacterial infections. Bloodstream and intra-abdominal were the most common bacterial sites (45% and 22.5%, respectively). Multidrug-resistant organisms accounted for 47.6% of bacterial sepsis. Vancomycin-resistant Enterococcus and extended-spectrum beta-lactamase producers were the most frequently identified multidrug-resistant organisms. Patients with multidrug-resistant organism sepsis demonstrated higher admission Pediatric Logistic Organ Dysfunction scores (p = 0.043) and were noted to have an odds ratio of 3.8 and 3.6 for mechanical ventilation and multiple organ dysfunction syndrome, respectively (p = 0.047 and p = 0.044). Overall mortality was 5.5% (n = 2 patients), with both deaths occurring in multidrug-resistant organism episodes. CONCLUSIONS: We report that multidrug-resistant organisms are increasingly being identified as causative pathogens for sepsis in pediatric liver transplant recipients and are associated with significantly higher odds for mechanical ventilation and higher organ failure. The emergence of multidrug-resistant organism infections in pediatric liver transplant patients has implications for patient outcomes, antibiotic stewardship, and infection prevention strategies.
Assuntos
Bacteriemia/microbiologia , Farmacorresistência Bacteriana Múltipla , Artéria Hepática , Transplante de Fígado/efeitos adversos , Trombose/microbiologia , Antibacterianos/uso terapêutico , Bacteriemia/complicações , Bacteriemia/tratamento farmacológico , Criança , Pré-Escolar , Coinfecção/microbiologia , Feminino , Fungemia/complicações , Fungemia/microbiologia , Humanos , Lactente , Infecções Intra-Abdominais , Masculino , Insuficiência de Múltiplos Órgãos/microbiologia , Respiração Artificial , Estudos Retrospectivos , Viroses/complicações , Viroses/virologia , Resistência beta-LactâmicaRESUMO
BACKGROUND: Allospecific CD154+T-cytotoxic memory cells (CD154+TcM) predict acute cellular rejection after liver transplantation (LTx) or intestine transplantation (ITx) in small cohorts of children and can enhance immunosuppression management, but await validation and clinical implementation. METHODS: To establish safety and probable benefit, CD154+TcM were measured in cryopreserved samples from 214 children younger than 21 years (National Clinical Trial 1163578). Training set samples (n = 158) were tested with research-grade reagents and 122 independent validation set samples were tested with current good manufacturing practices-manufactured reagents after assay standardization and reproducibility testing. Recipient CD154+TcM induced by stimulation with donor cells were expressed as a fraction of those induced by HLA nonidentical cells in parallel cultures. The resulting immunoreactivity index (IR) if greater than 1 implies increased rejection-risk. RESULTS: Training and validation set subjects were demographically similar. Mean coefficient of test variation was less than 10% under several conditions. Logistic regression incorporating several confounding variables identified separate pretransplant and posttransplant IR thresholds for prediction of rejection in the respective training set samples. An IR of 1.1 or greater in posttransplant training samples and IR of 1.23 or greater in pretransplant training samples predicted LTx or ITx rejection in corresponding validation set samples in the 60-day postsampling period with sensitivity, specificity, positive, and negative predictive values of 84%, 80%, 64%, and 92%, respectively (area under the receiver operator characteristic curve, 0.792), and 57%, 89%, 78%, and 74%, respectively (area under the receiver operator characteristic curve, 0.848). No adverse events were encountered due to phlebotomy. CONCLUSIONS: Allospecific CD154+T-cytotoxic memory cells predict acute cellular rejection after LTx or ITx in children. Adjunctive use can enhance clinical outcomes.
Assuntos
Ligante de CD40/análise , Citometria de Fluxo , Rejeição de Enxerto/imunologia , Imunidade Celular , Testes Imunológicos/métodos , Intestinos/transplante , Transplante de Fígado/efeitos adversos , Linfócitos T Citotóxicos/imunologia , Doença Aguda , Adolescente , Área Sob a Curva , Biomarcadores/análise , Células Cultivadas , Criança , Pré-Escolar , Criopreservação , Feminino , Rejeição de Enxerto/prevenção & controle , Humanos , Imunidade Celular/efeitos dos fármacos , Memória Imunológica , Imunossupressores/uso terapêutico , Lactente , Intestinos/imunologia , Modelos Logísticos , Masculino , Análise Multivariada , Valor Preditivo dos Testes , Curva ROC , Reprodutibilidade dos Testes , Medição de Risco , Fatores de Risco , Linfócitos T Citotóxicos/efeitos dos fármacos , Fatores de Tempo , Resultado do Tratamento , Adulto JovemRESUMO
Rectal duplication cysts are rare, thought to be due to defects in embryologic development, and often associated with other structural abnormalities. Clues to the existence of a rectal cyst are mainly due to bowel compression and presence of ectopic gastric mucosa within the cyst, leading to rectal bleeding. The diagnosis of a rectal duplication cyst requires a high index of suspicion. Confirming the diagnosis can be difficult based on the location of the cyst. Efforts to confirm the diagnosis include digital rectal examination, computed tomography, magnetic resonance imaging, ultrasonography, and Meckel scan. Surgical resection is the treatment of choice, especially because of the cyst's potential for malignant transformation. Because of the cyst's proximal location to the nerves innervating the anal canal and sphincters, surgical resection can lead to fecal incontinence.
Assuntos
Constipação Intestinal/etiologia , Cistos/diagnóstico , Hemorragia Gastrointestinal/etiologia , Doenças Retais/diagnóstico , Reto/anormalidades , Criança , Diagnóstico Diferencial , Feminino , Humanos , Doenças Retais/etiologia , Reto/cirurgiaAssuntos
Falência Hepática Aguda/complicações , Falência Hepática Aguda/diagnóstico , Falência Hepática Aguda/terapia , Veia Porta/anormalidades , Malformações Vasculares/complicações , Malformações Vasculares/diagnóstico , Malformações Vasculares/terapia , Embolização Terapêutica/métodos , Infecções por Enterovirus/diagnóstico , Humanos , Hiperamonemia/diagnóstico , Hiperbilirrubinemia/diagnóstico , Recém-Nascido , Fígado/diagnóstico por imagem , Fígado/patologia , Masculino , Tomografia Computadorizada por Raios X , Resultado do Tratamento , UltrassonografiaRESUMO
OBJECTIVE: To assess clinical and neurocognitive function in children who have undergone liver transplantation for classical maple syrup urine disease (MSUD). STUDY DESIGN: A total of 35 patients with classical MSUD (age 9.9 ± 7.9 years) underwent liver transplantation between 2004 and 2009. Six patients donated their liver to recipients without MSUD ("domino" transplant). We analyzed clinical outcomes for our cohort and 17 additional cases from the national United Network for Organ Sharing registry; 33 patients completed IQ and adaptive testing before transplantation, and 14 completed testing 1 year later. RESULTS: Patient and graft survival were 100% at 4.5 ± 2.2 years of follow-up. Liver function was normal in all patients. Branched-chain amino acid levels were corrected within hours after surgery and remained stable, with leucine tolerance increasing more than 10-fold. All domino transplant recipients were alive and well with normal branched-chain amino acid homeostasis at the time of this report. Patient and graft survival for all 54 patients with MSUD undergoing liver transplantation in the United States during this period were 98% and 96%, respectively. One-third of our patients were mentally impaired (IQ ≤ 70) before transplantation, with no statistically significant change 1 year later. CONCLUSION: Liver transplantation is an effective long-term treatment for classical MSUD and may arrest brain damage, but will not reverse it.