RESUMO
As outcomes after ITx improve, greater emphasis is needed on HRQOL. The primary aims of this study were to (i) assess the feasibility of measuring HRQOL in pediatric ITx recipients, (ii) measure HRQOL using validated instruments, and (iii) compare HRQOL in ITx recipients to healthy normal (NL) children. The CHQ and Pediatric Quality of Life (PedsQL4.0) instruments were administered to both patients and parents at outpatient visits. All 24 eligible patients were enrolled. The median age at study enrollment was 6.0 yr (range: 2-18 yr), and the median time from transplant to study enrollment was 2.8 yr (range: 0.5-11.8 yr). The majority of subjects were male (58%), Latino (58%), and liver-inclusive (92%) recipients. For CHQ and PedsQL4.0, parental responses were significantly lower in multiple categories including physical health and social functioning compared to healthy norms. Patient responses were not different from NL using CHQ but using PedsQL4.0 were significantly lower in the school functioning subcategory and psychosocial health summary score. HRQOL as reported by children and families after ITx is significantly lower in multiple categories compared to NL.
Assuntos
Nível de Saúde , Intestinos/transplante , Qualidade de Vida , Adolescente , Criança , Pré-Escolar , Feminino , Humanos , Masculino , Pais/psicologia , Autoavaliação (Psicologia) , Inquéritos e QuestionáriosRESUMO
BACKGROUND AND PURPOSE: We have previously shown that treatment with zinc plus cyclo-(His-Pro) (CHP) significantly stimulated synthesis of the insulin degrading enzyme and lowered plasma insulin and blood glucose levels, alongside improving oral glucose tolerance in genetically type 2 diabetic Goto-Kakizaki (G-K) rats and in aged obese Sprague-Dawley (S-D) rats. Thus, we postulated that zinc plus CHP (ZC) treatment might also improve body weight control in these rats. We therefore determined the effects of ZC treatment on body weights in both genetically diabetic, mature G-K rats and non-diabetic, obese S-D rats. EXPERIMENTAL APPROACH: G-K rats aged 1.5-10 months and non-diabetic overweight or obese S-D rats aged 6-18 months were treated with 0-6 mg CHP plus 0-10 mg zinc L(-1) drinking water for 2-4 weeks, and changes in weight, serum leptin and adiponectin levels, food and water intakes were measured. KEY RESULTS: The optimal dose of CHP (in combination with zinc) to reduce weight and plasma leptin levels and to increase plasma adiponectin levels was close to 0.1 mg kg(-1) day(-1), in either mature G-K rats and aged overweight or obese S-D rats. Food and water intake significantly decreased in ZC treated rats in both aged S-D rats and mature G-K rats, but not in young S-D and G-K rats. CONCLUSIONS AND IMPLICATIONS: ZC treatment improved weight control and may be a possible treatment for overweight and obesity.
Assuntos
Peso Corporal/efeitos dos fármacos , Diabetes Mellitus Tipo 2/tratamento farmacológico , Peptídeos Cíclicos/farmacologia , Piperazinas/farmacologia , Zinco/farmacologia , Adiponectina/sangue , Administração Oral , Fatores Etários , Animais , Diabetes Mellitus Experimental/tratamento farmacológico , Diabetes Mellitus Experimental/fisiopatologia , Diabetes Mellitus Tipo 2/fisiopatologia , Quimioterapia Combinada , Feminino , Leptina/sangue , Masculino , Obesidade/tratamento farmacológico , Peptídeos Cíclicos/administração & dosagem , Piperazinas/administração & dosagem , Ratos , Ratos Sprague-Dawley , Zinco/administração & dosagemRESUMO
Posttransplant de novo autoimmune hepatitis (d-AIH) is increasingly described as a long-term complication after pediatric liver transplantation (LT). d-AIH is characterized by graft dysfunction, the development of autoimmune antibodies and histologic evidence of hepatitis in liver transplant recipients without previous history of autoimmune liver disease. This study is a matched case-control, univariate analysis aimed at identifying risk factors for the development of d-AIH and evaluating response to treatment. From 1984 to 2003, 619 children received 788 LTs at a single center. Forty-one patients developed d-AIH and were matched with controls for year of LT, age at time of LT and diagnosis. The following variables were insignificant in the development of d-AIH: age, gender, race, initial diagnosis, ischemia time, graft type, Epstein-Barr virus and cytomegalovirus status, HLA typing and primary immunosuppression. Compared to controls, d-AIH patients were less likely to be on monotherapy immunosuppression or weaned off prednisone at the time of diagnosis. The d-AIH group relative to the controls had statistically significant greater numbers of rejection episodes. d-AIH was treated with prednisone and/or MMF in 39 of 41 patients and lead to significant improvements in liver function tests. Thirty-nine patients are alive at a mean of 4.0 years follow-up after diagnosis. Three have required retransplantation.
Assuntos
Rejeição de Enxerto/patologia , Hepatite Autoimune/epidemiologia , Transplante de Fígado/efeitos adversos , Complicações Pós-Operatórias/epidemiologia , Adolescente , Biópsia , Criança , Quimioterapia Combinada , Rejeição de Enxerto/epidemiologia , Hepatite Autoimune/patologia , Teste de Histocompatibilidade , Humanos , Imunossupressores/uso terapêutico , Transplante de Fígado/imunologia , Estudos Retrospectivos , Fatores de RiscoRESUMO
BACKGROUND: This study sought to describe the long-term nutritional outcomes of children after intestinal transplant (SBT). METHODS: Between 1991 and March 2005, 30 children received 33 SBT at a single center. Eligibility criteria included patient and graft survival >6 months. Weight, height, albumin, prealbumin, zinc (Zn), and essential fatty acid (EFA) levels were reviewed retrospectively. RESULTS: The 19 patients who met inclusion criteria had a median age at SBT of 2.9 years. The majority of patients were male, Latino, transplanted for necrotizing enterocolitis and received combined liver-SBT. All patients were weaned off total parenteral nutrition to elemental formula at a mean of 39 days post-SBT. Seventeen of 19 patients were Zn deficient and four patients were EFA deficient post-SBT. CONCLUSIONS: Pre-SBT most subjects were significantly deficient in anthropometric and biochemical parameters. Post-SBT the mean Z score for weight and height improved significantly at year 1, then leveled off in year 2. Serum protein levels improved from pre-SBT, yet remained low-normal. Zn deficiency was seen frequently after SBT and is under investigation. Children who developed EFA deficiency were on the same formula, receiving inadequate EFA supplementation. Successful SBT was associated with growth and maintenance of serum nutritional parameters but not with significant catch-up growth.
Assuntos
Intestino Delgado/transplante , Fenômenos Fisiológicos da Nutrição , Transplante Homólogo/fisiologia , Adolescente , Criança , Pré-Escolar , Estudos de Coortes , Ácidos Graxos Essenciais/sangue , Seguimentos , Sobrevivência de Enxerto , Humanos , Seleção de Pacientes , Estudos Retrospectivos , Resultado do TratamentoRESUMO
BACKGROUND: Minimally invasive esophagomyotomy, consisting of a laparoscopic or thoracoscopic approach, has become a preferred surgical treatment for adults with achalasia. This multicenter study reports on the clinical status of children who have undergone minimally invasive esophagomyotomy for achalasia. METHODS: Symptomatology for achalasia was assessed in 22 pediatric patients who underwent minimally invasive esophagomyotomy for achalasia between 1995 and 2000. All patients were evaluated for duration of hospitalization, postoperative resumption of feeds, postoperative complications, and symptomatic relief. Participants were assigned pre-and postoperative symptom severity scores ranging from 0 (no symptoms) to 3 (severe). RESULTS: The median age of the 10 females and 12 males at time of surgery was 11.3 years +/- 3.4 (standard deviation). Transabdominal laparoscopic esophagomyotomy with fundoplication was performed in 18 patients, and thoracoscopic esophagomyotomy without fundoplication was performed in 4. Two patients required conversion from transabdominal laparoscopic esophagomyotomy to open esophagomyotomy because of intraoperative esophageal perforation. The mean duration of postsurgical follow-up was 17 +/- 16 (standard deviation) months (range, 1-54 months). Mean duration of hospitalization (days +/- standard error or mean) was less for transabdominal laparoscopic esophagomyotomy than for converted open esophagomyotomy (2.7 +/- 0.3 vs. 9.0 +/- 3.0 days; P < 0.05) or for thoracoscopic esophagomyotomy (4.8 +/- 1.7 days; P = not significant). Mean time to resumption of soft feedings (days +/- standard error or mean) occurred sooner after transabdominal laparoscopic esophagomyotomy than after converted open esophagomyotomy (2.0 +/- 0.2 vs. 5.5 +/- 0.5 days; P < 0.001) or after thoracoscopic esophagomyotomy (4.0 +/- 1.3 days; P = not significant). Patients experienced significant pre-to postoperative improvement in mean severity score with regard to dysphagia (2.6 vs. 0.4; P < 0.001) and regurgitation (1.7 vs. 0.2; P < 0.001). CONCLUSIONS: Minimally invasive esophagomyotomy can provide excellent symptomatic relief from dysphagia and regurgitation for children with achalasia.
Assuntos
Acalasia Esofágica/cirurgia , Esôfago/cirurgia , Laparoscopia/métodos , Toracoscopia/métodos , Adolescente , Criança , Pré-Escolar , Feminino , Seguimentos , Fundoplicatura , Humanos , Complicações Intraoperatórias , Tempo de Internação , Masculino , Procedimentos Cirúrgicos Minimamente Invasivos , Complicações Pós-Operatórias , Índice de Gravidade de Doença , Resultado do TratamentoRESUMO
BACKGROUND: Previous studies have shown that plasma free choline concentrations are significantly decreased in many long-term home total parenteral nutrition (TPN) patients. Furthermore, low choline status has been associated with both hepatic morphologic and hepatic aminotransferase abnormalities. A preliminary pilot study suggested choline-supplemented TPN may be useful in reversal of these hepatic abnormalities. METHODS: Fifteen patients (10 M, 5 F) who had required TPN for > or =80% of their nutritional needs were randomized to receive their usual TPN (n = 8), or TPN to which 2 g choline chloride had been added (n = 7) for 24 weeks. Baseline demographic data were similar between groups. Patients had CT scans of the liver and spleen, and blood for plasma free and phospholipid-bound choline, alanine aminotransferase (ALT), aspartate aminotransferase (AST), alkaline phosphatase, gamma glutamyl transferase (GGT), bilirubin, serum lipids, complete blood count (CBC), and chemistry profile obtained at baseline, and weeks 2, 4, 6, 12, 16, 20, 24, and 34. CT scans were analyzed for Hounsfield unit (HU) densities. RESULTS: There were no significant differences in any measured parameters after 2 weeks. However, at 4 weeks, a significant difference in liver HU between groups was observed (13.3+/-5.0 HU [choline] vs 5.8+/-5.2 HU [placebo], p = .04). This significant trend continued through week 24. Recurrent hepatic steatosis and decreased HU were observed at week 34, 10 weeks after choline supplementation had been discontinued. A significant increase in the liver-spleen differential HU was also observed in the choline group (10.6+/-6.2 HU [choline] vs 1.3+/-3.3 HU [placebo], p = .01). Serum ALT decreased significantly (p = .01 to .05) in the choline group vs placebo at weeks 6,12, 20, and 24. Serum AST was significantly decreased in the choline group by week 24 (p = .02). The serum alkaline phosphatase was significantly reduced in the choline group at weeks 2, 12, 20, 24, and 34 (p = .02 to 0.07). Total bilirubin was normal in these patients and remained unchanged during the study. Serum GGT tended to decrease more in the choline group, but the greater decrease was not statistically significant. CONCLUSIONS: Choline deficiency is a significant contributor to the development of TPN-associated liver disease. The data suggest choline is a required nutrient for long-term home TPN patients.
Assuntos
Deficiência de Colina/terapia , Colina/administração & dosagem , Lipotrópicos/administração & dosagem , Fígado/patologia , Nutrição Parenteral Total/efeitos adversos , Adulto , Colina/sangue , Suplementos Nutricionais , Emulsões Gordurosas Intravenosas , Feminino , Humanos , Lipotrópicos/sangue , Fígado/enzimologia , Masculino , Necessidades Nutricionais , Baço/patologia , Tomografia Computadorizada por Raios X , Transaminases/metabolismoRESUMO
BACKGROUND: Autoimmune hepatitis (AIH) and cryptogenic chronic hepatitis (CCH) are important causes of liver failure in children, frequently necessitating orthotopic liver transplantation (OLT). The aim of this study is to review disease progression and potential differences between subgroups of children with AIH and CCH. METHODS: The medical records of 65 children diagnosed with AIH or CCH between 1980 and 1998 were evaluated. RESULTS: The median age at presentation was 9 years, 8 months (range 4 months-19 years), and the median follow-up period was 8 years (range 3 months-18 years, 10 months). Forty-one patients (63%) were female. Twenty-eight patients were Hispanic, 28 were Caucasian, 8 were African-American, and 1 was Asian. Forty-three patients (66%) were diagnosed with type 1 AIH, 8 (12%) with type 2 AIH, and 14 (22%) with CCH. Forty patients (62%) underwent OLT (51% of those with type 1 AIH, 75% of those with type 2 AIH, and 86% of those with CCH). Thirteen (33%) of the transplanted patients experienced disease recurrence. African-American patients experienced a significantly higher rate of disease recurrence post-OLT than did Hispanic patients. Seven patients (11%) died, two without OLT, and five posttransplantation. CONCLUSIONS: AIH and CCH frequently necessitate OLT in children. CCH is a more aggressive disease than Type 1 AIH among children with these disorders. Ethnicity influences the rate of disease recurrence after liver transplantation.
Assuntos
Hepatite Autoimune/cirurgia , Hepatite Crônica/cirurgia , Transplante de Fígado , Adolescente , Adulto , Criança , Pré-Escolar , Etnicidade , Feminino , Hepatite Autoimune/classificação , Humanos , Lactente , Masculino , Recidiva , Resultado do TratamentoRESUMO
We report a case of Alstrom syndrome with evidence of extensive hepatic disease diagnosed at five years of age, who subsequently developed acute liver failure and died at eight years of age. Such a case, with the patient dying before the age of ten, has not been described before. The biochemical findings during our patient's liver failure raised the question of a possible mitochondrial function defect in this syndrome. Further investigation of this possibility is needed.
Assuntos
Hepatopatias/fisiopatologia , Falência Hepática Aguda/fisiopatologia , Anormalidades Múltiplas/fisiopatologia , Idade de Início , Criança , Diabetes Mellitus/congênito , Coagulação Intravascular Disseminada , Nanismo , Evolução Fatal , Feminino , Perda Auditiva Neurossensorial , Encefalopatia Hepática , Humanos , Hiperamonemia , Hepatopatias/complicações , Falência Hepática Aguda/complicações , Falência Hepática Aguda/diagnóstico , Mitocôndrias Hepáticas , Obesidade , Degeneração Retiniana/congênito , Síndrome , Transtornos da Visão/congênitoRESUMO
Previous studies have already shown that prostate extract (PE) has antidiabetic activity when given to animals and humans. In this study, we explore whether this antidiabetic activity is related to the high concentrations of zinc, cyclo (his-pro) (CHP), and the prostaglandin precursor, arachidonic acid (AA), in prostate tissue. When streptozotocin-induced diabetic rats were given drinking water containing 10 mg/L zinc and 100 mg/L PE for 3 weeks, fasting blood glucose levels and glucose clearance rates, but not plasma insulin levels, were significantly lower than at pretreatment. In subsequent experiments, blood glucose levels in rats given PE for 3 weeks were significantly lower than in rats given distilled water or 10 mg/L zinc alone. However, in rats given 100 mg/L CHP with zinc, blood glucose levels were also lower than in rats given PE alone. Time-course studies in diabetic rats given drinking water containing 20 mg/L Zn, 20 mg/L L-histidine, and 10 mg/L CHP showed that blood glucose levels dropped 209 +/- 53 mg/dL in 1 day and stayed low for 2 weeks. When CHP was replaced with 100 mg AA/L, blood glucose levels dropped 230 +/- 64 mg/dL in 5 days, but returned to the original values 11 days later. Growth rate improved and water consumption decreased significantly in CHP- and AA-treated diabetic rats. High intake of L-histidine and testosterone increased blood glucose concentrations in diabetic rats. To determine optimal dosages of CHP and AA, we gave rats drinking water containing 10 mg/L Zn and 0.5 mg/L L-histidine with various concentrations of CHP or AA. The most effective doses for reducing blood glucose levels were 0.32 mg CHP/kg/day and 11 mg AA/kg/day. These data suggest that the active antidiabetic ingredients in the PE are CHP, zinc, and AA or its precursors.
Assuntos
Diabetes Mellitus Experimental/tratamento farmacológico , Hipoglicemiantes/uso terapêutico , Próstata/química , Animais , Antioxidantes/uso terapêutico , Ácido Araquidônico/uso terapêutico , Glicemia/efeitos dos fármacos , Extratos Celulares/uso terapêutico , Diabetes Mellitus Experimental/induzido quimicamente , Diabetes Mellitus Experimental/metabolismo , Modelos Animais de Doenças , Cães , Sinergismo Farmacológico , Insulina/fisiologia , Masculino , Peptídeos Cíclicos/uso terapêutico , Piperazinas/uso terapêutico , Ratos , Estreptozocina , Zinco/uso terapêuticoRESUMO
BACKGROUND: Clinical and hematological profile of chronic anemia in children after orthotopic liver transplantation (OLT) is unknown. METHODS: We prospectively studied children after orthotopic liver transplantation (OLT) with hemoglobin levels < 2 standard deviation of age appropriate mean for > 6 months. Investigations included hemogram, reticulocyte count, peripheral blood smear, serum vitamin B-12, folic acid levels, iron studies, Coomb's tests, serum erythropoietin (EPO) levels, and stool and urine tests for occult blood. RESULTS: Fifty-six participants (22 male and 34 female, mean age 82.9 months, range 20-232, mean post-OLT duration 48.8 months, range 6-132) were studied. The causes of anemia were idiopathic (32), iron deficiency (4), viral infections (2, HIV=1, parvovirus=1), and lymphoproliferative disease (2). Fifteen participants showed spontaneous recovery within 1-6 months. Thirty-one children with idiopathic anemia had low or normal EPO levels (mean 7.33 mmicro/L, range <2.5 to 15.9, normal 4-24). When outliers (iron deficiency=4, HIV disease= 1) were excluded, there was no statistical correlation between hematocrits and EPO levels. Serum vitamin B-12 levels (n=52) were elevated (normal 110-930 pg/ml) (mean=1,186 pg/ml) in 32 (61.5%) and were significantly higher in those with abnormal liver function tests. CONCLUSION: Anemia is a common problem in children after OLT. More than half the participants had anemia of unknown etiology with an inappropriate EPO response for the degree of anemia. The normal negative correlation between hematocrit and EPO was lost in these children. The observation regarding serum vitamin B-12 levels requires further study.
Assuntos
Anemia/etiologia , Transplante de Fígado/efeitos adversos , Adolescente , Anemia/sangue , Criança , Pré-Escolar , Estudos Transversais , Eritropoetina/sangue , Feminino , Ferritinas/sangue , Ácido Fólico/sangue , Hemoglobinas/análise , Humanos , Ferro/sangue , Masculino , Estudos Retrospectivos , Vitamina B 12/sangueRESUMO
Alagille syndrome (AGS) is frequently associated with growth failure, which has been attributed to concurrent congenital anomalies, cholestasis, and malabsorption and/or malnutrition. However, the underlying cause of the growth failure is not well understood. Our objective is to analyze the growth pattern in 26 patients with AGS and the possible effect that orthotopic liver transplantation (OLT) may have on this pattern. The standardized height, weight, and growth velocity of 26 pair-matched patients with AGS were compared. Thirteen patients underwent OLT. Repeated-measure ANOVA methods were used for the statistical analysis. The overall mean standardized height (z score) was -2.92 in the OLT group versus -1.88 in the non-OLT group (P =.03). The overall mean standardized weight was -1. 21 in the non-OLT group and -1.67 in the OLT group (P =.23). In 15 patients, birth weight was 2.82 +/- 0.4 kg, for a mean standardized weight of -0.95, and weight at diagnosis was 4.53 +/- 2.12 kg, for a mean standardized weight of -1.56. Bone age was delayed in the 9 patients who underwent bone-age analysis. Growth hormone therapy administered to 2 patients did not improve growth. Patients with AGS had growth failure secondary to other factors in addition to liver disease. Growth failure beginning in the prenatal period supports a genetic basis for this feature. Growth improvement up to normal levels should not be expected as a benefit of OLT in these patients. Growth failure as a primary indication for OLT should be cautiously examined in patients with AGS.
Assuntos
Síndrome de Alagille/fisiopatologia , Síndrome de Alagille/cirurgia , Desenvolvimento Infantil , Transplante de Fígado , Determinação da Idade pelo Esqueleto , Síndrome de Alagille/patologia , Estatura , Peso Corporal , Osso e Ossos/fisiopatologia , Criança , Pré-Escolar , Feminino , Crescimento , Humanos , Lactente , Recém-Nascido , Masculino , Resultado do TratamentoRESUMO
BACKGROUND: Posttransplant lymphoproliferative disease (PTLD) is a serious complication associated with the use of chronic immunosuppression for solid organ transplantation. This study represents a retrospective analysis of UCLA's experience with PTLD in all pediatric liver transplant recipients between 1984-1997. We assessed the clinical presentation, risk factors, incidence density, immunological characteristics, management, and outcome of patients who developed PTLD when receiving either primary cyclosporin A (CsA) or tacrolimus. METHODS: A total of 251 children received primary CsA therapy of which 70 required OKT3 for steroid resistant rejection and 29 required tacrolimus rescue for OKT3 resistance and/or chronic rejection. One hundred forty one children received tacrolimus as primary therapy. Sixty patients who survived for less than 6 months after transplantation were excluded from the study. RESULTS: The total incidence density (ID) rate of PTLD was 1.8+/-0.4 per 100 patient-years (30/392). The overall ID rate of PTLD in the CsA group was 0.93+/-0.2 per 100 patient-years (15/251). Within this group of primary CsA-treated patients, the ID rate of PTLD was 0.49+/-0.1 without OKT3 or tacrolimus, 0.67+/-0.2 with OKT3, and 6.42+/-1.1 with tacrolimus rescue. The overall PTLD ID rate in the primary tacrolimus-treated patients was 4.86+/-1.2 per 100 person-years (15/141). There was a 5-fold increase in the ID rate of PTLD in the primary tacrolimus group when compared to the comparable, primary CsA group (P<0.001). The mean time to PTLD was 5-fold longer (49.7+/-20.7 months) in the CsA group when compared to the CsA/tacrolimus rescue group (9.8+/-3 months, P<0.05) or the tacrolimus primary group (12.6+/-5.1 months, P<0.05). Five patients had monoclonal disease in the CsA group, but only one in the tacrolimus group (P<0.05). Clinical presentations with enlarged lymph nodes, fevers, malaise, anorexia, weight loss, hypoalbuminemia, and gastrointestinal blood loss were common. Mortality was 20%, three patients died in each group. CONCLUSION: The use of primary tacrolimus therapy was associated with a significant 5-fold higher rate of PTLD when compared to those treated with primary cyclosporine. Early diagnosis, decrease and/or discontinuation of potent immunosuppressive agents may contribute to decrease morbidity and mortality of this entity.
Assuntos
Imunossupressores/efeitos adversos , Transplante de Fígado/efeitos adversos , Transtornos Linfoproliferativos/etiologia , Adolescente , Fatores Etários , Criança , Pré-Escolar , Ciclosporina/efeitos adversos , Herpesvirus Humano 4/isolamento & purificação , Humanos , Incidência , Lactente , Transtornos Linfoproliferativos/epidemiologia , Transtornos Linfoproliferativos/terapia , Muromonab-CD3/efeitos adversos , Estudos Retrospectivos , Fatores de Risco , Tacrolimo/efeitos adversosRESUMO
The vast majority of patients with celiac disease respond to a gluten-free diet; yet, a small number of refractory patients do not respond and have persistent malabsorption and residual mucosal abnormalities of the small intestine. The histologic features of refractory/unclassified sprue have been published as case reports, often without long-term follow up, and no clear histologic picture has emerged. We present the results of a long-term study of the clinical and histologic features of 10 patients with refractory/unclassified sprue. The histologic features of small bowel biopsies in this group of patients were compared with those of 10 patients with responsive celiac disease and with 10 patients without malabsorption who had normal duodenal biopsies. Five of the 10 refractory patients ultimately developed collagenous sprue as a distinct histologic marker of refractory disease. Additional distinctive findings found in small bowel biopsies in the refractory group were subcryptal chronic inflammation (10 of 10) and marked mucosal thinning in three patients. Other nonspecific findings included acute inflammation and gastric metaplasia. One patient with collagenous sprue developed a B-cell lymphoma of the ileum, and in general collagenous sprue was associated with a poor prognosis. Two of five patients died whereas two others require total parenteral nutrition for survival. Pathologists evaluating small bowel biopsies in the setting of malabsorption should be aware of the subtle histologic changes described here that may portend a refractory course.
Assuntos
Doença Celíaca/patologia , Adulto , Idoso , Biópsia , Doença Celíaca/complicações , Doença Celíaca/dietoterapia , Doença Celíaca/metabolismo , Doença Crônica , Colágeno/metabolismo , Colo/patologia , Enterite/patologia , Humanos , Neoplasias do Íleo/complicações , Mucosa Intestinal/patologia , Intestino Delgado/patologia , Estudos Longitudinais , Linfoma de Células B/complicações , Metaplasia , Pessoa de Meia-Idade , Nutrição Parenteral Total , Estômago/patologia , Falha de TratamentoRESUMO
BACKGROUND: Achalasia is rare in children. Recently, injection of botulinum toxin into the lower esophageal sphincter has been studied as an alternative to esophageal pneumatic dilatation or surgical myotomy as treatment for achalasia. In the current study, the effects of botulinum toxin were investigated in the largest known series of children with achalasia. METHODS: Treatment for achalasia was assessed in 23 pediatric patients who received botulinum toxin from June 1995 through November 1998. Those who continued to receive botulinum toxin and did not subsequently undergo pneumatic dilatation or surgery were considered repeat responders. Results were compared with those of published studies evaluating the use of botulinum toxin in adults with achalasia. RESULTS: Nineteen patients initially responded to botulinum toxin. Mean duration of effect was 4.2 months +/- 4.0 (SD). At the end of the study period, three were repeat responders, three experienced dysphagia but did not receive pneumatic dilatation or surgery, three underwent pneumatic dilatation, eight underwent surgery, three underwent pneumatic dilatation with subsequent surgery, and three awaited surgery. Meta-analysis shows that, in the current study group, the data point expressing time of follow-up evaluation versus percentage of patients needing one injection session without additional procedures (botulinum toxin injection, pneumatic dilatation, or surgery) falls within the curve for those in studies on adult patients receiving botulinum toxin for achalasia. CONCLUSIONS: Botulinum toxin effectively initiates the resolution of symptoms associated with achalasia in children. However, one half of patients are expected to need an additional procedure approximately 7 months after one injection session. The authors recommend that botulinum toxin be used only for children with achalasia who are poor candidates for either pneumatic dilatation or surgery.
Assuntos
Toxinas Botulínicas/uso terapêutico , Acalasia Esofágica/tratamento farmacológico , Adolescente , Adulto , Toxinas Botulínicas/administração & dosagem , Criança , Esôfago/efeitos dos fármacos , Feminino , Humanos , Injeções , Masculino , Resultado do TratamentoRESUMO
BACKGROUND: Little is known about parathyroid gland function in patients receiving total parenteral nutrition (TPN). OBJECTIVE: Our objective was to determine whether parathyroid gland function is abnormal in TPN recipients. DESIGN: Six patients with a mean (+/-1 SD) age of 45.5 +/- 8.0 y who had been receiving TPN for 18.7 +/- 2. 8 y underwent bone biopsy, bone mass measurements with dual-energy X-ray absorptiometry, and dynamic tests of parathyroid gland function. Diurnal variations in blood ionized calcium (iCa(2+)) and serum parathyroid hormone (PTH) concentrations were also assessed. Results were compared with those of healthy volunteers. RESULTS: Bone mass and bone formation were subnormal in all patients. Basal serum PTH concentrations were moderately higher in the TPN recipients than in healthy volunteers, and values obtained every 30 min over 24 h were significantly higher (P < 0.001) in TPN recipients (5.0 +/- 0.9 pmol/L) than in healthy volunteers (2.6 +/- 0.6 pmol/L). The percentage increase in serum PTH during citrate-induced hypocalcemia was lower in the TPN recipients, consistent with secondary hyperparathyroidism. Evening infusions of calcium-containing TPN eliminated the nocturnal rise in serum PTH, increased the amplitude of change for iCa(2+) and PTH over 24 h, increased the orderliness of change for iCa(2+) and PTH as measured by approximate entropy (ApEn), and enhanced the synchrony of change between iCa(2+) and PTH. Treatment for 10 d with calcium-free TPN restored the nocturnal rise in serum PTH and increased ApEn for PTH. ApEn for iCa(2+) remained low, suggesting that a component of nutrient solutions, but not calcium per se, enhances the regularity of PTH release in TPN recipients. CONCLUSION: Parathyroid gland function is abnormal in long-term TPN recipients, which may contribute to disturbances in bone metabolism.
Assuntos
Cálcio/sangue , Glândulas Paratireoides/fisiopatologia , Hormônio Paratireóideo/sangue , Nutrição Parenteral Total , Adulto , Densidade Óssea , Remodelação Óssea , Cálcio/urina , Ritmo Circadiano , Citratos , Feminino , Colo do Fêmur , Humanos , Vértebras Lombares , Masculino , Pessoa de Meia-Idade , Glândulas Paratireoides/metabolismo , Nutrição Parenteral Total/efeitos adversos , Admissão do Paciente , Citrato de Sódio , Fatores de Tempo , Vitamina D/análogos & derivados , Vitamina D/sangueRESUMO
BACKGROUND: Alagille syndrome is one of the most common inherited disorders that cause chronic liver disease in children. Early reports suggested a benign course in these patients. Subsequent reports showed significant morbidity and mortality. This study was designed to analyze the long-term clinical course in Alagille syndrome. METHODS: The records of children with Alagille syndrome seen during a 20-year period were reviewed. RESULTS: Forty-three patients were identified. Liver disease was diagnosed before 12 months of age in 95%. The frequencies of renal anomalies (50%) and intracranial hemorrhage (12%) were significant. The high incidence of chronic otitis media (35%) has not been reported previously. One patient had a renal transplant. Vascular compromise as a pathologic mechanism for some characteristics of the syndrome is also suggested by the presence of small bowel stenosis and atresia, tracheal and bronchial stenosis, renal artery stenosis, middle aortic syndrome, and avascular necrosis of the humeral and femoral heads. Twenty (47%) patients underwent liver transplantation. Five of six who underwent Kasai procedure required liver transplantation. Twelve died (28%), five after liver transplantation. One patient died of intracranial bleeding. Sixteen (37%) without liver transplantation and 15 (35%) who underwent liver transplantation are alive. CONCLUSIONS: Some patients with early-onset and more severe liver disease can benefit from liver transplantation. Careful and complete assessment should be made of infants with a cholestatic syndrome, to avoid misdiagnosis and unnecessary Kasai procedures. Our observation of vascular compromise in various organ systems suggests that notch signaling pathway defects affect angiogenesis in Alagille syndrome.
Assuntos
Síndrome de Alagille/epidemiologia , Adolescente , Síndrome de Alagille/genética , Síndrome de Alagille/patologia , Biópsia , Osso e Ossos/anormalidades , Criança , Pré-Escolar , Humanos , Lactente , Rim/anormalidades , Fígado/patologia , Estudos Retrospectivos , Resultado do Tratamento , Doenças Vasculares/congênitoRESUMO
BACKGROUND: Low plasma free choline concentration has been associated with elevated serum hepatic aminotransferase concentrations and hepatic steatosis in adults who need home parenteral nutrition (HPN). We sought to determine if plasma free choline is similarly reduced in children who need home total parenteral nutrition (TPN). METHODS: We compared the plasma free choline concentration in 21 children who required long-term HPN with 31 normal controls. Patients had received HPN for 75 +/- 13 (SD) months (range 3-206 months). All control children ingested a normal, mixed, nonvegetarian diet. RESULTS: The mean plasma free choline concentration in the children receiving HPN was significantly lower than normal children (6.6 +/- 4.3 vs 8.0 +/- 2.3 nmol/mL, p = .002). Plasma free choline concentration was correlated with age (r = -0.43, p = .049). Using multiple linear regression analysis for age, sex, and squared age (considered in order to account for possible nonlinearity between choline and age), HPN children showed a steady and significant decline in plasma free choline concentration with increased age at the rate of 0.03 nmol/mL per month. Plasma lipid bound choline concentration did not vary with age. No relationship was seen between either plasma free and lipid bound choline concentration and amount of daily IV lipid infusion. A significant negative correlation was observed between plasma free choline concentration and aspartate aminotransferase (AST) and alanine aminostransferase (ALT) (r = -0.72, p = .04 and r = -0.80, p = .02, respectively). CONCLUSION: Our data support the notion that patients who need long-term HPN without significant enteral feeding have a significant risk for the development of choline deficiency with its associated hepatic dysfunction.
Assuntos
Colina/sangue , Nutrição Parenteral no Domicílio , Adolescente , Alanina Transaminase/sangue , Aspartato Aminotransferases/sangue , Estudos de Casos e Controles , Criança , Pré-Escolar , Feminino , Humanos , Modelos Lineares , Lipídeos/sangue , Masculino , Fatores de TempoAssuntos
Infecções por Citomegalovirus/complicações , Diarreia Infantil/etiologia , Enterocolite/complicações , Antivirais/uso terapêutico , Infecções por Citomegalovirus/diagnóstico , Infecções por Citomegalovirus/tratamento farmacológico , Enterocolite/tratamento farmacológico , Enterocolite/microbiologia , Enterocolite/patologia , Ganciclovir/uso terapêutico , Humanos , Imunocompetência , Lactente , Mucosa Intestinal/patologia , MasculinoRESUMO
BACKGROUND: Posttransplant lymphoproliferative disease (PTLD) is a serious complication associated with the use of immunosuppression after transplantation. In a retrospective study the clinical features of PTLD located primarily in the gastrointestinal tract were analyzed. METHODS: Three hundred ninety-two consecutive pediatric patients who underwent orthotopic liver transplantation (OLT) during a 13-year period with a survival of more than 6 months were reviewed. Two immunosuppression protocols were used: cyclosporin A, or tacrolimus-based primary therapy. Twenty-nine randomly selected liver transplant recipients without PTLD were used for comparison of signs and symptoms of gastrointestinal PTLD. RESULTS: Among the 30 patients identified with PTLD, 9 had gastrointestinal PTLD. The overall incidence density of PTLD was 1.8 per 100 patient-years (30/392). Nine patients (30%) had involvement of the gastrointestinal tract, whereas 7 (23%) had the gastrointestinal tract as the only involved site. When compared with a cohort of liver transplant recipients without PTLD, only gastrointestinal bleeding, weight loss, hypoalbuminemia, and protein-losing enteropathy were signs most likely associated with gastrointestinal PTLD. Hypoalbuminemia was the most sensitive sign of gastrointestinal PTLD. The lower tract (ileum and colon) was the most common site of involvement. CONCLUSIONS: gastrointestinal involvement is common and occurs in 30% of all patients with PTLD. It may be the only affected organ in a subgroup of patients. Hypoalbuminemia, gastrointestinal bleeding, and weight loss are features that are characteristic of gastrointestinal PTLD. Patients with aggressive gastrointestinal signs and symptoms should undergo upper and lower gastrointestinal tract endoscopy with biopsy, to establish the diagnosis.