Update on the management of vasoproliferative tumour. / Actualización sobre el manejo del tumor vasoproliferativo.

CASE REPORT: Here we report a 19-year-old female patient who presented a vasoproliferative tumour. It caused complications, such as epiretinal membrane, macular oedema, vitreous haemorrhage, and exudative retinal detachment. The patient was treated with 3 injections of intravitreal bevacizumab, an intravitreal dexamethasone implant, tocilizumab, and double freeze-thaw cryotherapy. DISCUSSION: Therapeutic options are: observation, if it is small, if it is a peripheral lesion, and if there seems to be no threat to vision. If it requires treatment, laser photocoagulation, intravitreal bevacizumab, trans-conjunctival cryotherapy, transpupillary thermotherapy, photodynamic therapy, brachytherapy plaques and surgery are the different options available. Recently, tocilizumab and intravitreal dexamethasone implants have been reported to be beneficial.

The Medication Level Variability Index (MLVI) Predicts Poor Liver Transplant Outcomes: A Prospective Multi-Site Study.

Nonadherence to immunosuppressant medications is a leading cause of poor long-term outcomes in transplant recipients. The Medication Level Variability Index (MLVI) provides a vehicle for transplant outcome risk-stratification through continuous assessment of adherence. The MALT (Medication Adherence in children who had a Liver Transplant) prospective multi-site study evaluated whether MLVI predicts late acute rejection (LAR). Four hundred pediatric (1-17-year-old) liver transplant recipients were enrolled and followed for 2 years. The a-priori hypothesis was that a higher MLVI predicts LAR. Predefined secondary analyses evaluated other outcomes such as liver enzyme levels, and sensitivity analyses compared adolescents to pre-adolescents. In the primary analysis sample of 379 participants, a higher prerejection MLVI predicted LAR (mean prerejection MLVI with LAR: 2.4 [3.6 standard deviation] versus without LAR, 1.6 [1.1]; p = 0.026). Fifty-three percent of the adolescents with MLVI>2 in year 1 had LAR by the end of year 2, as compared with 6% of those with year 1 MLVI≤2. A higher MLVI was significantly associated with all secondary outcomes. MLVI, a marker of medication adherence that uses clinically derived information, predicts LAR in pediatric liver transplant recipients.

Posttransplant metabolic syndrome in the withdrawal of immunosuppression in Pediatric Liver Transplant Recipients (WISP-R) pilot trial.

Posttransplant metabolic syndrome (PTMS)-obesity, hypertension, elevated triglycerides, low HDL and glucose intolerance-is a major contributor to morbidity after adult liver transplant. This analysis of the Withdrawal of Immunosuppression in Pediatric Liver Transplant Recipients (WISP-R) pilot trial is the first prospective study of PTMS after pediatric liver transplant. Twenty children were enrolled in WISP-R, at median age 8.5 years (IQR 6.4-10.8), and weaned from calcineurin-inhibitor monotherapy. The 12 children who tolerated complete immunosuppression withdrawal were compared to matched historical controls. At baseline, 45% of WISP-R subjects and 58% of controls had at least one component of PTMS. Calcineurin-inhibitor withdrawal in the WISP-R subjects did not impact the prevalence of PTMS components compared to controls. At 5 years, despite weaning off of immunosuppression, 92% of the 12 tolerant WISP-R subjects had at least one PTMS component and 58% had at least two; 33% were overweight or obese, 50% had dyslipidemia, 33% glucose intolerance and 42% systolic hypertension. Overweight/obesity increased the risk of hypertension in all children. Compared to controls, WISP-R tolerant subjects had similar GFR at baseline but did have higher GFR at 2, 3 and 4 years. Further study of PTMS and immunosuppression withdrawal after pediatric liver transplant is warranted.

Health status in young adults two decades after pediatric liver transplantation.

We conducted a cross-sectional study of patients who underwent pediatric liver transplant (LT) between 1988 and 1992 to evaluate long-term health status. Survivors completed socio-demographic, medical and Health-Related Quality of Life (HRQOL) surveys by mail including the SF-36v2, PedsQL™4.0 Generic Core Scale, PedsQL™ Cognitive Functioning Scale and PedsQL™3.0 Transplant Module. SF-36 scores were converted to SF6D-based utilities and risk factors for lower outcomes were assessed. Eighty-five of 171 patients had survived. Fifty-six were contacted with a response rate of 66%. Median age at LT was 0.86 years (IQR 0.58-3.0) and 64.3% had biliary atresia. Mean age at survey was 23.0 ± 4.4 years: 62% attended college, 68% lived with parents and 80% of those over 23 were employed. Patient health utilities were lower than norms (0.75 ± 0.12 vs. 0.82 ± 0.18, p < 0.01) and correlated with unemployment (p < 0.042), hospitalizations (p < 0.005) and lower education level (p < 0.016). Lower PedsQL™3.0 Transplant Module and PedsQL™ 4.0 Generic Core Scale scores correlated with unemployment (p = 0.006, p = 0.009) and hospitalizations (p = 0.006, p = 0.02). Pediatric transplant recipients who survive to adulthood have lower physical HRQOL, measurable transplant-related disability and lower health utility. Transplantation is life saving; however, physical and psychological sequelae continue to affect health status up to two decades later.

Liver transplantation followed by allogeneic hematopoietic stem cell transplantation for atypical mevalonic aciduria.

Mevalonic aciduria because of mutations of the gene for mevalonate kinase causes limited synthesis of isoprenoids, the effects of which are widespread. The outcome for affected children is poor. A child with severe multisystem manifestations underwent orthotopic liver transplantation at age 50 months for the indication of end-stage liver disease. This procedure corrected liver function and eliminated portal hypertension, and the patient showed substantial improvement in neurological function. However, autoinflammatory episodes continued unabated until hematopoietic stem cell transplantation was performed at 80 months. Through this complex therapy, the patient now enjoys a high quality of life without significant disability.

Blood pressure elevation in long-term survivors of pediatric liver transplantation.

As pediatric liver transplant (LT) recipients come of age, additional insight into long-term medical complications of immunosuppression is warranted. The aims of this study were to estimate the prevalence of elevated blood pressure (BP) in long-term survivors of pediatric LT using the data from the Studies in Pediatric Liver Transplantation (SPLIT) database and to identify predictive factors. Patients enrolled in the BP arm of the SPLIT cohort participated in the study. All patients were of at least 5 years but ≤10 years post-LT. Automated BP measurements were obtained at anniversary visits. BP measures were classified as normal, borderline or elevated according to standard criteria. Patients taking antihypertensive medications were classified as "elevated." Eight hundred and fifteen patients participated. The prevalence of elevated BP measurements 5 to 10 years post-LT was 17.5 to 27.5%. Of total 62.5% patients presented with at least one additional elevated BP at a later follow up visit. Multivariate analysis revealed the following parameters to be predictive of elevated BP: age at transplant, steroid use at last BP measurement and cGFR at last BP measurement. Pediatric LT patients show a high prevalence of elevated BP measurements 5 to 10 years following LT, which is related to age at LT, decreased cGFR and recent steroid use.

Risk factors for chronic anemia in pediatric orthotopic liver transplantation: analysis of data from the SPLIT registry.

Risk factors for chronic anemia in the post-transplant period have not been clearly delineated in pediatric liver transplant recipients. We analyzed data from children transplanted from 2000 to 2008 with at least two consecutive hemoglobin values from follow-up between six months and five yr post-transplant. A multivariate model was derived to determine independent risk factors associated with chronic anemia. Of 1026 children in this analysis, 242 (23.6%) were found to have chronic anemia. On multivariate analysis, GI bleeding (OR 11.83 [2.08-67.49], p = 0.0054), presence of leukopenia (OR 9.55 [95% CI 3.71-24.62], p < 0.0001), use of cyclosporine (OR 3.69, [95% CI 1.56-8.76], p = 0.0039) and corticosteroids (OR 2.90 [95% CI 1.94-4.33], p < 0.0001), and cGFR <90 mL/min/1.73 m(2) (OR 4.62 [95% CI 2.47-8.67], p < 0.0001) represented the most significant risk factors for chronic anemia. Use of antihypertensive medications (OR 1.89 [95% CI 1.23-2.91], p = 0.0039) was also significantly associated with a higher risk. In summary, chronic anemia is common in children following liver transplant. Our findings underscore the need to define the mechanisms by which these risk factors, some of which are modifiable, result in chronic anemia in pediatric liver transplant recipients.

Cognitive and academic outcomes after pediatric liver transplantation: Functional Outcomes Group (FOG) results.

This multicenter study examined prevalence of cognitive and academic delays in children following liver transplant (LT). One hundred and forty-four patients ages 5-7 and 2 years post-LT were recruited through the SPLIT consortium and administered the Wechsler Preschool and Primary Scale of Intelligence, 3rd Edition (WPPSI-III), the Bracken Basic Concept Scale, Revised (BBCS-R), and the Wide Range Achievement Test, 4th edition (WRAT-4). Parents and teachers completed the Behavior Rating Inventory of Executive Function (BRIEF). Participants performed significantly below test norms on intelligence quotient (IQ) and achievement measures (Mean WPPSI-III Full Scale IQ = 94.7 ± 13.5; WRAT-4 Reading = 92.7 ± 17.2; WRAT-4 Math = 93.1 ± 15.4; p < 0001). Twenty-six percent of patients (14% expected) had 'mild to moderate' IQ delays (Full Scale IQ = 71-85) and 4% (2% expected) had 'serious' delays (Full Scale IQ ≤ 70; p < 0.0001). Reading and/or math scores were weaker than IQ in 25%, suggesting learning disability, compared to 7% expected by CDC statistics (p < 0.0001). Executive deficits were noted on the BRIEF, especially by teacher report (Global Executive Composite = 58; p < 0.001). Results suggest a higher prevalence of cognitive and academic delays and learning problems in pediatric LT recipients compared to the normal population.

Pediatric health-related quality of life: Feasibility, reliability and validity of the PedsQL transplant module.

The measurement properties of the newly developed Pediatric Quality of Life Inventory (PedsQL) 3.0 Transplant Module in pediatric solid organ transplant recipients were evaluated. Participants included pediatric recipients of liver, kidney, heart and small bowel transplantation who were cared for at seven medical centers across the United States and their parents. Three hundred and thirty-eight parents of children ages 2-18 and 274 children ages 5-18 completed both the PedsQL 4.0 Generic Core Scales and the Transplant Module. Findings suggest that child self-report and parent proxy-report scales on the Transplant Module demonstrated excellent reliability (total scale score for child self-report alpha= 0.93; total scale score for parent proxy-report alpha= 0.94). Transplant-specific symptoms or problems were significantly correlated with lower generic HRQOL, supporting construct validity. Children with solid organ transplants and their parents reported statistically significant lower generic HRQOL than healthy children. Parent and child reports showed moderate to good agreement across the scales. In conclusion, the PedsQL Transplant Module demonstrated excellent initial feasibility, reliability and construct validity in pediatric patients with solid organ transplants.

Once daily calcineurin inhibitor monotherapy in pediatric liver transplantation.

This report describes a group of pediatric liver transplant recipients who have undergone once daily calcineurin inhibitor (CNI) monotherapy at Children's Memorial Hospital, Chicago, between January 1, 2001 and November 30, 2008. We defined success as normal liver enzymes at 1 year after dose change, with normal enzymes throughout all follow-up. Patients who did not meet the set criteria or had lost an organ to chronic rejection were not considered for this therapeutic strategy. There were 147 patients in our organ transplant tracking record (OTTR) who were > or = 5 years post liver transplant. Of these, 56 underwent reduced dose, once daily CNI monotherapy. Patients who met the set criteria were placed on once daily calcineurin inhibitor at half their previous dose. Fifty patients successfully achieved this dose change, while six patients failed at a mean of 3.7 +/- 3.2 months following the dosing change. The mean interval from transplant was significantly longer in those patients who were successful compared to those who failed dose change (p < 0.05). Importantly, there have been no graft losses. Reduced dose, once daily CNI monotherapy is safe in carefully selected recipients, with a longer interval post liver transplantation increasing the likelihood of success.

Linear growth patterns in prepubertal children following liver transplantation.

Factors impacting linear growth following pediatric liver transplantation (LT) are not well understood. This longitudinal analysis examines predictors of linear growth impairment in prepubertal children included in Studies of Pediatric Liver Transplantation. In 1143 children with serial measurements, mean height scores increased from -1.55 at LT to -0.87 and -0.68 at 24 and 36 months post LT with minimal subsequent catch up growth observed until 60 months. Subgroup analysis of height measurements at 24 months (n = 696), 33.8% were below 10th percentile at 24 months post LT. Multivariate analysis revealed linear growth impairment more likely in patients with metabolic disease (OR 4.4, CI: 1.83-10.59) and >18 months of steroids exposure (OR 3.02, CI: 1.39-6.55). Higher percentiles for weight (OR 0.80, CI: 0.65-0.99) and height (OR 0.62, CI: 0.51-0.77) at LT decreased risk. Less linear catch up was observed in patients with metabolic disease, non-Biliary atresia cholestatic diseases and lower weight and higher height percentiles prior to LT. Prolonged steroid exposure and elevated calculated glomerular filtration rate and gamma-Glutamyltransferase following LT were associated with less catch up growth. Linear growth impairment and incomplete linear catch up growth are common following LT and may improve by avoiding advanced growth failure before LT and steroid exposure minimization.

Adolescent health-related quality of life following liver and kidney transplantation.

UNLABELLED: Health perceptions of adolescent transplant patients should be considered in providing appropriate healthcare. OBJECTIVES: (i) quantify health-related quality of life (HRQOL) in adolescent liver and kidney transplant patients, (ii) compare caregiver ratings of their children's HRQOL to adolescent self-reports and (iii) examine the relationship between HRQOL and disease-specific disability (DSD). Adolescent liver (n = 51) and kidney (n = 26) transplant recipients and caregivers were surveyed using the CHQ-CF87 and CHQ-PF50. DSD scores were calculated for each patient. The response rate was >70%. Adolescent's psychological and physical health was similar to a healthy population, but general health poorer (p = 0.0006). Caretakers reported lower physical functioning and general health (p = 0.0001) but similar psychological health to a normative population. All caregivers expressed negative emotional impact of their child's health on themselves and family activities (p = 0.0001). Positive correlations were found between liver transplant recipients and caregivers: perceptions of behavior (ICC = 0.55, p < 0.001), mental health (ICC = 0.56, p < 0.001), self-esteem (ICC = 0.68, p

Safety of percutaneous liver biopsy in infants less than three months old.

OBJECTIVES: To evaluate the safety, outcomes, and complications of percutaneous liver biopsies (PLB) in infants aged 0 to 3 months. METHODS: We retrospectively reviewed the hospital records of all infants less than 3 months old who underwent PLB at Children's Memorial Hospital between July 1, 1997 and June 30, 2004 for complications surrounding the procedure and risk factors that might lead to complications. RESULTS: Sixty-six PLBs were performed in 63 infants. Most patients tolerated the procedure without complications. Twelve complications were recorded, for an overall complication rate of 18%. Of these, five were directly related to the procedure, and seven were sedation related. Three patients experienced a drop in hemoglobin greater than 2 gm/dL, one patient developed a bile leak, and one developed a skin hematoma. Seven patients had respiratory difficulty related to sedation, which manifested as increased work of breathing or decreased respiratory rate with depression in pulse oximetry. CONCLUSION: We conclude that PLB in young infants is associated with a somewhat higher risk of complications than in older children, particularly complications related to sedation.

Octreotide therapy for control of acute gastrointestinal bleeding in children.

OBJECTIVES: To evaluate the outcomes of octreotide use in children with gastrointestinal bleeding with or without portal hypertension. METHODS: The medical charts of children who received continuous octreotide infusion for the indication of gastrointestinal bleeding from January 1995 to December 2000 were retrospectively reviewed. RESULTS: Twenty-one children with portal hypertension received octreotide infusions (1-2 microg/kg/h) during 35 gastrointestinal bleeding episodes. The duration of infusion ranged from 19 hours to 7 days. Bleeding ceased in 71% of children. Post-treatment rebleeding occurred in 52%, and the mortality was 19%. Response to octreotide, rebleeding, and mortality did not differ significantly between patients with or without intrinsic liver disease. Twelve children with gastrointestinal bleeding unrelated to portal hypertension received octreotide during 14 episodes. The duration of octreotide ranged from 3 hours to 36 days. Fifty percent of children had resolution of bleeding during the infusion. Post-treatment rebleeding occurred in 29%, and the mortality was 50%. No major adverse events were observed in either group. CONCLUSIONS: A high rate of bleeding control was achieved with octreotide during acute gastrointestinal bleeding in children with portal hypertension. However, controlled studies are needed for more definitive description of its clinical effects. The role of octreotide is less clear in gastrointestinal bleeding unrelated to portal hypertension.

Timed Pediatric Risk of Mortality Scores predict outcomes in pediatric liver transplant recipients.

More reliable methods are needed to identify children at risk for poor outcomes following liver transplantation. The Pediatric Risk of Mortality (PRISM) Score is a physiology-based scoring system used to quantify risk of mortality in pediatric intensive care unit (ICU) populations. We evaluated the PRISM Score as a predictor of outcomes including survival in the pediatric liver transplant (LT) population. We retrospectively reviewed the records of 67 consecutive LTs performed between August 1997 and February 2000 at an urban, tertiary children's hospital in Chicago, IL, USA. Four PRISM Scores were calculated to determine which periods were most meaningful. A Classic PRISM Score was calculated during first 24 h of ICU admission, and three PRISM Scores were timed with the patient's transplant: a pre-LT PRISM Score (24 h prior to transplant whether in ICU or not), a 24-h post-LT PRISM Score and a 48-h post-LT PRISM Score. These PRISM Scores and other predictors including transplant number, UNOS status and PELD Score were compared with outcomes including survival using univariate methods. The pre-LT, the 24- and the 48-h PRISM Score were associated with the post-LT number of ventilated days (p < 0.05), ICU days (p < 0.05) and with 1-yr survival (p < 0.04). The PRISM Scores were not related to the post-LT hospital length of stay (LOS) or to 1-yr re-transplantation. The PELD Score correlated with the post-LT hospital LOS, but was not associated with mortality or with the ICU LOS. A patient's UNOS status and Classic PRISM Score were not associated with any of the outcomes measured. PRISM Scores are valid predictors of outcome including survival in pediatric LT recipients. These findings help to demonstrate the importance in this population of a patient's general physiologic condition and its influence on the overall hospital course and survival.

Functional outcomes of pediatric liver transplantation.

UNLABELLED: The functional status and health-related quality of life (HRQOL) of children who survive liver transplantation (LT) have not been well documented. The purpose of this study was to determine the functional status and HRQOL in this population using a validated measure for children, the Child Health Questionnaire-Parent Form 50 (CHQ-PF50). METHODS: The CHQ-PF50 instrument was completed by the parents of 55 children who agreed to participate in a mailing survey. Subscale scores for the sample were compared with those of a published normal population (n = 391). RESULTS: Study sample characteristics were: 87% Caucasian, 54.5% female, mean age at survey was 9.6 years (range, 5-17 years). Responding caregivers were 95% biologic parents and 93% female. Compared with the normal population, LT recipients had lower subscale scores for general health perceptions (P < 0.0005), emotional impact on parents (<0.0005) and disruption of family activities (0.0005). The mean physical summary score of the LT recipients was lower than that of the normal population 48.1 +/- 12.1 (P = 0.005), but the mean psychosocial summary score was similar 48.8 +/- 11.9 (P = 0.156). Within the LT population, the original diagnosis (biliary atresia vs. other), type of LT (living donor vs. cadaveric), age at LT, z score for height, and hospital days did not significantly influence any of the subscale scores. CONCLUSIONS: Children who have survived LT have functional outcomes in the physical domain that are lower than those of normal children. Self-esteem and mental health in this group appeared normal. The parents in this sample experienced more emotional stress and disruption of family activities than did parents in a normal population.

Living donor liver transplantation for children with liver failure and concurrent multiple organ system failure.

Liver transplantation for pediatric patients in liver failure and multiple organ system failure (MOSF) often results in poor patient survival. Progression of organ failure occurs while awaiting a cadaveric allograft. Therefore, we considered living donor liver transplantation (LDLT) in this critically ill group of children and report our initial results with comparison to a similar group who received cadaveric donation (CAD). A retrospective chart review was performed on all pediatric liver transplant recipients who met criteria for MOSF at the time of transplantation. Data collection involved pretransplantation patient profiles, as well as postoperative complications and patient survival. Eight patients in MOSF received living donor transplants and 11 patients received a cadaveric allograft. Mean wait time was 3.5 days in the LDLT group and 6.5 days in the CAD group. Pretransplantation patient profiles and postoperative complications were similar between groups. Mean cold ischemia times were 3.8 hours in the LDLT group and 7.9 hours in the CAD group (P = .0002). Thirty-day and 6-month survival rates of the LDLT group were 88% and 63% compared with 45% and 27% in the CAD group, respectively. Living donor transplant recipients in MOSF had decreased wait times to transplantation, as well as decreased cold ischemia times, compared with cadaveric transplant recipients. Patients in the LDLT group had markedly improved survival compared with the CAD group. Timely transplantation before worsening organ failure may account for these findings.

Síndrome de Wünderlich: angiomiolipoma renal multicéntrico / Wünderlich's syndrome: multicentric renal angiomyolipoma

Presentamos un caso de hemorragia renal espontánea o de síndrome de Wünderlich secundario a angiomiolipoma. Constituye una urgencia vital. Las manifestaciones clínicas y la utilización de la tomografía computarizada (TC) son la base para obtener el diagnóstico definitivo y orientar la actitud terapéutica hacia una cirugía conservadora, siempre que sea posible, aunque en nuestro caso el tratamiento fue la nefrectomía (AU)

Apendicitis izquierda / Left-sided appendicitis

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