Assessment of hepatic involvement by two-dimensional shear wave elastography in paediatric patients with cystic fibrosis.

AIM: This study aimed to investigate parenchymal changes in the liver in paediatric patients with cystic fibrosis (CF) and to analyse diagnostic performance of two-dimensional shear wave elastography (2D-SWE) for the detection of hepatic involvement. METHODS: Patients with CF treated and followed at our centre were evaluated prospectively. All patients underwent liver tissue stiffness (TS) measurements by 2D-SWE, in addition to routine clinical assessments, laboratory work-up and abdominal ultrasound imaging. Data from patients with CF were compared with healthy control subjects. RESULTS: This study included 39 patients with CF and 37 healthy controls. Patients had a mean body weight of 29.9 (16.6-55) kg, mean age of 9 (5-17) years, mean height of 130 (107-172) cm and a mean body mass index of 16.1 (12.8-21.4) kg/m2 . Average SWE values of the liver were 1.02 (0.70-1.60) m/s in patients with CF (n = 39) and 0.89 (0.60-1.35) m/s in healthy controls (n = 37). Cystic fibrosis patients had significantly increased tissue stifness by liver elastography compared to controls (P = 0.005). CONCLUSION: Parenchymal liver changes may occur early in cystic fibrosis, which cannot be detected by conventional ultrasonography but may be demonstrated by 2D-SWE. Based on this cross-sectional study, 2D-SWE may be a promising, simple and non-invasive modality for objective monitoring of patients with cystic fibrosis who require lifelong follow-up, by providing numerical data for tissue stiffness early in the disease.

The cumulative effects of MEFV gene polymorphisms and mutations in patients with inflammatory bowel diseases.

OBJECTIVE: To determine the cumulative effects of Mediterranean fever gene polymorphisms and mutations in patients with inflammatory bowel diseases. METHODS: The case-control study was conducted from January, 2012, to January, 2016,at Cukurova University, Turkey, and comprised patients diagnosed with inflammatory bowel diseases and followed up at the Children Gastroenterology Department. By using molecular methods, 12 Mediterranean fevergene variants most frequently observed in the country were examined in all the diagnosed cases. The results were compared with age-matched healthy population data from the Genetic Diseases Diagnosis and Treatment Centre. Data was analysed using Graph Pad Prism. RESULTS: Of the 151 subjects, 46(30.4%) were cases and 105(69.5%) were controls. Among the cases, there were 23(50%) subjects with a mean age of 14.8±3 years who had ulcerative colitis, and 23(50%) with mean age 14.5±3.2 years who had Crohn's disease. The mean age of the controls was 16.4±3.2 years (p=0.716). Patients with ulcerative colitishad high frequencies of C allele in D102D T>C variant, G allele in G138G A>G variant, A allele in A165A C>A variant and A allele in R202Q G>A variant. Those with Crohn's disease frequently had wild type of R202Q G>A variant. Also, D102D T>C / R314R C>T haplotype was common at a certain level in the UC group. CONCLUSIONS: Mediterranean fever gene variant was more frequently found in cases with ulcerative colitis compared to the controls.

The contribution of diet preference to the disease course in children with familial Mediterranean fever: a cross-sectional study.

OBJECTIVES: Familial Mediterranean fever (FMF) is characterized by recurrent, self-limiting attacks of fever and serositis. Nutrition is very important in the management of chronic diseases. Previous studies suggested that salty and fatty diet cause inflammation, therefore we aimed to investigate the effects of dietary self-efficacy and behavior about low-salt or low-fat diet on disease course in children with FMF. MATERIAL AND METHODS: This cross-sectional study included patients aged between 10-18 years, diagnosed in our department and admitted between June 2019 and September 2019. Demographic and clinical properties were obtained from the medical files of the patients. Children's Dietary Self-Efficacy Scale (CDSS) and Health Behavior Questionnaire (HBQ) - Diet Behavior Scale (DBS) were performed for dietary self-efficacy and behavior about preferring low-salt or low-fat diet. Clinical features were compared between patients, which were grouped according to the sum of these two scales, with a cut-off score of 5. RESULTS: The mean age of 74 FMF patients (44 females, 34 males), included in the study, was 14.6 ±2.82 years. Median CDSS and DBS scores of the patients were 5 (minimum -6, maximum 14) and 0 (minimum -10, maximum 12), respectively. According to the sum of these two scales, 39 (52.7%) patients who had scored at least 5, had a statistically higher rate of complete response to colchicine. The remaining clinical parameters were similar between these two groups. CONCLUSIONS: Low-salt or low-fat diet may be an adjuvant modification in the management of children with FMF. Further studies are needed to clarify the role of low-salt or low-fat diet in FMF pathogenesis.

Is Henoch-Schönlein purpura a susceptibility factor for functional gastrointestinal disorders in children?

Henoch-Schönlein purpura (HSP), the most common childhood vasculitis is characterized by non-thrombocytopenic palpable purpura, arthritis/arthralgia, abdominal pain and renal involvement. Functional gastrointestinal disorders (FGIDs) are heterogeneous disease spectrum with unclear etiology and include the most common subtypes: functional dyspepsia, irritable bowel syndrome (IBS), functional abdominal pain and functional constipation. Formerly, FGIDs were known as non-organic disorders; however, recent advances revealed that low-grade inflammation may also play a role. We aimed to clarify whether HSP predisposes to FGIDs in pediatric population. Seventy-four children with HSP, diagnosed at least 6 months before the study and 78 healthy controls were enrolled to the study. Patients with red flag signs for organic GI disorders were excluded. Rome IV criteria were utilized for FGIDs diagnosis. We compared the frequencies of FGIDs between HSP patients and healthy subjects. We also examined the parameters including age, abdominal pain, arthralgia, bloody stool, renal involvement and treatment with corticosteroids and laboratory results at HSP diagnosis such as erythrocyte sedimentation rate, C-reactive protein, hemoglobin, leukocytes and platelet counts among patients with and without FGIDs. Overall FGIDs and IBS frequency were 35.1% (n = 26) and 10.8% (n = 8) in HSP patients, 19.2% (n = 15) and 2.6% (n = 2) in healthy controls, respectively. Disease characteristics and laboratory parameters at disease onset were similar between HSP patients with and without FGIDs. Overall FGIDs rate, particularly IBS were statistically higher in HSP patients. We speculate that children with preceding HSP may be predisposed to FGIDs. Since the FGIDs pathogenesis is still remains unclear, further studies are needed to confirm this hypothesis and clarify the etiology. Physicians also should pay attention to FGIDs in HSP patients with ongoing abdominal pain and thus prevent this comorbidity with dietary and psychologic measures.

Hyperimmunoglobulinemia D syndrome with recurrent perianal abscess successfully treated with canakinumab.

Hyperimmunoglobulinaemia D syndrome is an autoinflammatory disease usually representing recurrent episodes of fever, arthralgia/arthritis, cervical lymphadenopathy, vomiting, diarrhoea, abdominal pain and skin rashes lasting 3-7 days every 4-8 weeks since their infancy. Recent reports suggested a link between perianal fistulae/abscess and severe colitis with hyperimmunoglobulinaemia D syndrome resembling an inflammatory bowel disease phenotype. Herein, we report an 18-month-old patient with recurrent attacks of fever and pharyngitis lasting 2-3 days every 10-15 days since the first two weeks of life. Inflammatory attacks were accompanied by diarrhoea, oral aphthous ulcers, cervical lymphadenopathy, maculopapular rash, severe leukocytosis and perianal fistulae/abscess. After the initiation of canakinumab, the patient was clinically improved with complete healing of perianal fistulas/abscesses. In conclusion, hyperimmunoglobulinaemia D syndrome should be considered in differential diagnosis of inflammatory bowel disease and recurrent perianal abscess/fistula in a patient with inflammatory attacks.

The Effect of Enteral Nutrition Support Rich in TGF-ß in the Treatment of Inflammatory Bowel Disease in Childhood.

Background and Objective: Malnutrition is a major complication of inflammatory bowel disease (IBD). Our aim of the study was to examine the effects of Modulen IBD supplementation, which was administered to IBD patients without limiting their daily diet in addition to medical treatment, on the clinical, laboratory, anthropometric values, and disease activities of these patients. Materials and Methods: Seventy three children with IBD were evaluated retrospectively. The cases were classified as those who had Crohn disease receiving (CD-M; n = 16) or not receiving Modulen IBD (CD; n = 19) and those who had ulcerative colitis receiving (UC-M; n = 13) or not receiving Modulen IBD (UC; n = 25). Disease activities, laboratory values, remission rates, and anthropometric measurements of the groups were compared. In addition to IBD treatment, Modulen IBD in which half of the daily calorie requirement was provided was given for eight weeks. Results: In the third month of treatment, 14 (88%) patients were in remission in CD-M group and eight (42%) patients were in remission in CD group. The height and weight z scores, which were low at the time of diagnosis, improved in the first week in CD-M group. Inflammatory parameters (UC) were significantly lower in the UC-M group compared to the UC group in first and third months. In the third month, eight (62%) patients in the UC-M group and four (16%) in the UC group were remitted clinically and in terms of laboratory values. Conclusions: TGF-ß-rich enteral nutrition support in children with IBD is an easy, effective, and reliable approach. It was shown that TGF-ß-rich enteral nutritional supplementation enabled the disease to enter the remission earlier, and contributed to the early recovery of weight and height scores.

Trientine-induced Rhabdomyolysis in an Adolescent with Wilson's Disease.

BACKGROUND: Drugs are very important in the etiology of nontraumatic rhabdomyolysis. CASE DESCRIPTIONS: A 16-year-old male patient with Wilson's disease was admitted for myoclonic contractions. Oral trientine was started for neurological problems and tremor on the hands due to D-penicillamine 1 month ago. Patient was oligoanuric, and his creatine kinase level was 15197 U/L. Rhabdomyolysis was associated with trientine, and trientine treatment was stopped. Hemodiafiltration was performed. The patient began to urinate on the 24th day. CONCLUSION: This is the first pediatric patient with rhabdomyolysis induced by trientine. Drugs used should be questioned carefully in patients with rhabdomyolysis. HOW TO CITE THIS ARTICLE: Aslan N, Yavuz S, Yildizdas D, Horoz OO, Coban Y, Tumgor G, et al. Trientine-induced Rhabdomyolysis in an Adolescent with Wilson's Disease. Indian J Crit Care Med 2019;23(10):489-490.

Frequency of Celiac Disease in Children with Peptic Ulcers.

AIM: The aim of the present study is to investigate the frequency of celiac disease in children with peptic ulcers and to compare it with that of non-celiac peptic ulcers in terms of clinical and laboratory values. METHODS: Upper gastrointestinal endoscopy was performed in 1769 patients at the Department of Pediatric Gastroenterology, The Faculty of Medicine, Cukurova University, Turkey, between January 2012 and January 2017. These cases consisted of subjects presenting with various GIS symptoms and indicated for endoscopy (with chronic diarrhea, delayed growth and development, abdominal pains, GIS bleeding, etc.). The levels of immunoglobulin A (IgA) serum anti-tissue transglutaminase antibodies, IgA anti-endomysial antibodies (EMA), and IgA serum were estimated in the patients with peptic ulcers. RESULTS: Celiac disease was diagnosed with serology, endoscopy, and histopathology in 250 (14%) of all cases undergoing endoscopy. Peptic ulcers were diagnosed in 74 patients (4.2%) of all cases undergoing endoscopy. tTGA and EMA (+) levels were determined in 22 (29%) of the 74 patients with peptic ulcers, and then the presence of peptic ulcers was investigated in the upper gastrointestinal system using gastrointestinal endoscopy, followed by histopathological confirmation of celiac disease. HP infection was present in 14 (63%) of the patients with celiac disease and in 23 (44%) of non-celiac peptic ulcers; the difference was not statistically significant (p = 0.12). In the total ulcer group, 10.8% (8/74) of patients with celiac peptic ulcers were negative for HP infection, whereas 21% (8/37) of HP-negative patients with ulcers had celiac disease. CONCLUSION: There exists a high risk of celiac disease in children with peptic ulcers. We thus recommend celiac disease to be investigated, particularly in HP-negative patients with peptic ulcers but with no history of NSAID use.

Seronegative autoimmune hepatitis in childhood.

Comprehensive guidelines on seropositive autoimmune hepatitis have been published for both adults and children, although these guidelines comprise only limited knowledge about seronegative autoimmune hepatitis. Autoimmune hepatitis presents as an acute or chronic progressive disease and poor outcomes are inevitable if left untreated. The absence of autoantibody positivity, hypergammaglobulinemia and lack of comprehensive algorithms makes seronegative autoimmune hepatitis a mysterious disease. In general, seronegative autoimmune hepatitis often presents with acute hepatitis, and its treatment and prognosis similar to seropositive autoimmune hepatitis. The present review focuses on the known characteristics of seronegative autoimmune hepatitis in childhood, and those of which current knowledge is vague.

The Effects of Protein Intake on Clinical Outcome in Pediatric Intensive Care Units.

OBJECTIVE: Factors such as increased metabolic needs and inadequate calorie and protein intake increase the risk of malnutrition in critically ill children admitted to the pediatric intensive care unit. This study aimed to determine the risk of malnutrition and associated clinical outcomes. MATERIALS AND METHODS: Data from all patients aged 1 month to 18 years in 4 pediatric intensive care units in Adana, Turkey, were prospectively collected. Patient anthropometric data, the duration of mechanical ventilation, the length of stay in pediatric intensive care unit, 60-day mortality, nutritional status, and calorie and protein intake were recorded. RESULTS: A total of 111 patients were included in the study. There was a significant difference between survivor and non-survivor patients in terms of calorie and protein intake 48 hours after admission and protein intake on the seventh day after admission (P = .001, P = .000, and P = .003, respectively). No significant correlation was found between the length of pediatric intensive care unit stay, sepsis, and calorie intake in the first week. It was found that 1 g/kg/ day increase in protein intake on the seventh day of intensive care hospitalization decreased the risk of mechanical ventilation by 0.49 times (P = .035; sensitivity: 83.3%; specificity: 34.5%). CONCLUSION: Nutritional status should be evaluated fully in patients admitted to the pediatric intensive care unit and early detected malnutrition should be monitored closely to determine the need for early intervention. The risk of developing malnutrition is high in critically ill children. Providing the necessary energy and protein intake with nutritional therapy affects the clinical course in children with critical illness. Protein intake causes prolongation of mechanical ventilation time, delaying clinical recovery.

The frequency of Duchenne muscular dystrophy/Becker muscular dystrophy and Pompe disease in children with isolated transaminase elevation: results from the observational VICTORIA study.

Introduction: Elevated transaminases and/or creatine phosphokinase can indicate underlying muscle disease. Therefore, this study aims to determine the frequency of Duchenne muscular dystrophy/Becker muscular dystrophy (DMD/BMD) in male children and Pompe disease (PD) in male and female children with isolated hypertransaminasemia. Methods: This multi-center, prospective study enrolled patients aged 3-216 months with serum alanine transaminase (ALT) and/or aspartate transaminase (AST) levels >2× the upper limit of normal (ULN) for ≥3 months. Patients with a known history of liver or muscle disease or physical examination findings suggestive of liver disease were excluded. Patients were screened for creatinine phosphokinase (CPK) levels, and molecular genetic tests for DMD/BMD in male patients and enzyme analysis for PD in male and female patients with elevated CPK levels were performed. Genetic analyses confirmed PD. Demographic, clinical, and laboratory characteristics of the patients were analyzed. Results: Overall, 589 patients [66.8% male, mean age of 63.4 months (standard deviation: 60.5)] were included. In total, 251 patients (188 male and 63 female) had CPK levels above the ULN. Of the patients assessed, 47% (85/182) of male patients were diagnosed with DMD/BMD and 1% (3/228) of male and female patients were diagnosed with PD. The median ALT, AST, and CPK levels were statistically significantly higher, and the questioned neurological symptoms and previously unnoticed examination findings were more common in DMD/BMD patients than those without DMD/BMD or PD (p < 0.001). Discussion: Questioning neurological symptoms, conducting a complete physical examination, and testing for CPK levels in patients with isolated hypertransaminasemia will prevent costly and time-consuming investigations for liver diseases and will lead to the diagnosis of occult neuromuscular diseases. Trial Registration: Clinicaltrials.gov NCT04120168.

Evaluation of nutritional status in pediatric intensive care unit patients: the results of a multicenter, prospective study in Turkey.

Introduction: Malnutrition is defined as a pathological condition arising from deficient or imbalanced intake of nutritional elements. Factors such as increasing metabolic demands during the disease course in the hospitalized patients and inadequate calorie intake increase the risk of malnutrition. The aim of the present study is to evaluate nutritional status of patients admitted to pediatric intensive care units (PICU) in Turkey, examine the effect of nutrition on the treatment process and draw attention to the need for regulating nutritional support of patients while continuing existing therapies. Material and Method: In this prospective multicenter study, the data was collected over a period of one month from PICUs participating in the PICU Nutrition Study Group in Turkey. Anthropometric data of the patients, calorie intake, 90-day mortality, need for mechanical ventilation, length of hospital stay and length of stay in intensive care unit were recorded and the relationship between these parameters was examined. Results: Of the 614 patients included in the study, malnutrition was detected in 45.4% of the patients. Enteral feeding was initiated in 40.6% (n = 249) of the patients at day one upon admission to the intensive care unit. In the first 48 h, 86.82% (n = 533) of the patients achieved the target calorie intake, and 81.65% (n = 307) of the 376 patients remaining in the intensive care unit achieved the target calorie intake at the end of one week. The risk of mortality decreased with increasing upper mid-arm circumference and triceps skin fold thickness Z-score (OR = 0.871/0.894; p = 0.027/0.024). The risk of mortality was 2.723 times higher in patients who did not achieve the target calorie intake at first 48 h (p = 0.006) and the risk was 3.829 times higher in patients who did not achieve the target calorie intake at the end of one week (p = 0.001). The risk of mortality decreased with increasing triceps skin fold thickness Z-score (OR = 0.894; p = 0.024). Conclusion: Timely and appropriate nutritional support in critically ill patients favorably affects the clinical course. The results of the present study suggest that mortality rate is higher in patients who fail to achieve the target calorie intake at first 48 h and day seven of admission to the intensive care unit. The risk of mortality decreases with increasing triceps skin fold thickness Z-score.

Biliary atresia and congenital disorders of the extrahepatic bile ducts.

Biliary atresia (BA) and choledochal cysts are diseases of the intrahepatic and extrahepatic biliary tree. While their exact etiopathogeneses are not known, they should be treated promptly due to the potential for irreversible parenchymal liver disease. A diagnosis of BA may be easy or complicated, but should not be delayed. BA is always treated surgically, and performing the surgery before the age of 2 mo greatly increases its effectiveness and extends the time until the need for liver transplantation arises. While the more common types of choledochal cysts require surgical treatment, some can be treated with endoscopic retrograde cholangiopancreatography. Choledochal cysts may cause recurrent cholangitis and the potential for malignancy should not be ignored.

Treatment With Balloon Angioplasty of Chronic Portal Vein Thrombosis.

The therapeutic options in portal vein thrombosis cases of young age and low weight, as in this case, are limited. Interventional radiologists also have minimal experience in pediatric patients. There are no reported cases anywhere worldwide, especially in this age group. However, we think that balloon angioplasty can be safely applied in cases in which esophageal variceal bleeding cannot be controlled using traditional treatment.

Evaluation of the Liver and Pancreas by 2D Shear Wave Elastography in Pediatric Wilson's Disease.

BACKGROUND: The primary aim of the study was to demonstrate parenchymal changes in the liver and pancreas related to copper accumulation using ultrasound in pediatric patients with Wilson's disease and secondly, to investigate the effectiveness of two-dimensional shear wave elastography in the diagnosis of involvement of these organs. METHODS: Patients with Wilson's disease (n = 25) who were treated and followed at our center were evaluated prospectively. In addition to routine clinical assessments, eye examination, laboratory analyses, and abdominal ultrasound imaging, all patients underwent tissue stiffness measurements from the liver and pancreas (head, body and tail) by two-dimensional shear wave elastography. The data obtained from the WD patients were compared with those of age- and sex-matched healthy controls (n = 37). RESULTS: Liver elastography measurements showed significantly increased tissue stiffness in the patient group than in control subjects (P < .001). While there was no significant difference between the groups in the tissue thickness of pancreatic head, body, and tail, tissue stiffness was significantly reduced in the patient group (P < .001). Disease duration was significantly associated and moderately correlated with liver tissue stiffness (r = 0.417, P = .038) but not significantly associated with pancreatic tissue stiffness. CONCLUSION: In the early stages of Wilson's disease, parenchymal changes occur in the liver and pancreas, which cannot be detected by conventional ultrasonography imaging but may be demonstrated by two-dimensional shear wave elastography. Ultrasound elastography is an easy to use, non-invasive, and promising method that provides numerical data on the early changes in tissue stiffness, allowing for objective monitoring of Wilson's disease patients who require lifelong follow-up.

Comparison of Isocaloric and Hypercaloric Diet in Undernourished Patients.

OBJECTIVE: To determine efficacies of fiber-containing isocaloric and hypercaloric enteral supplements generally used to treat undernutrition. STUDY DESIGN: Retrospective cohort study. PLACE AND DURATION OF STUDY: Department of Pediatrics, Sanliurfa Halfeti State Hospital, Turkey from September 2019 to June 2020. METHODOLOGY: Pediatric patients aged 1-19 years were diagnosed solely with primary undernutrition, were given fiber containing isocaloric or hypercaloric enteral supplements for six months based on their energy requirements. A comparative analysis of anthropometrical data was made with each formula. The analysis included baseline weight, height, BMI of patients, and important micro-nutrient levels at three and six months after intervention. RESULTS: BMI, weight and height z-scores (p <0.001) were improved over six months. There were no differences in BMI and weight scores except for a significant improvement in height between baseline and third month, which was observed in patients who received hypercaloric formula unlike isocolaric formula. There was a two-tailed improvement in blood biochemistry values of both groups. CONCLUSION: Both isocaloric and hypercaloric supplementation had positive effects on anthropometry and blood biochemistry. These results show that both formulae are highly beneficial for children with primary undernutrition. Key Words: Pediatrics, Body mass index, Therapeutics, Gastroenterology, Undernutrition.

Evaluation of pancreatic functions in cases of primary and secondary malnutrition.

BACKGROUND: This study assessed pancreatic functions by investigating fecal elastase-1 (FE-1) levels in stool specimens in children with primary and secondary malnutrition. METHODS: A total of 139 malnourished children who were hospitalized and followed up at a tertiary care pediatrics clinic and 23 healthy children with no known systemic disease or malnutrition were included in this study. Malnourished patients were divided into four groups according to underlying diagnosis including primary malnutrition (N.=51), cystic fibrosis (N.=44), celiac disease (N.=12) and secondary malnutrition (N.=32; remaining patients with various diagnoses). Patient's demographic characteristics and laboratory data were investigated. FE-1 levels of the patients and healthy subjects were evaluated. RESULTS: FE-1 levels in patients with cystic fibrosis, primary malnutrition, and celiac disease, and other patients with secondary malnutrition were significantly lower than those in the control group. CONCLUSIONS: Pancreatic enzymes are used due to pancreatic failure in cases of cystic fibrosis, and patients benefit considerably from treatment. This study shows that pancreatic failure may also occur in cases of primary and secondary malnutrition apart from cystic fibrosis, emphasizing the likelihood of pancreatic enzyme support to be useful in terms of pancreatic failure developing secondarily in cases of primary malnutrition.

Efficiency of azathioprine monotherapy for maintenance treatment of autoimmune hepatitis in children.

INTRODUCTION: Autoimmune hepatitis (AIH) is a common pediatric liver disease and long-term remission is usually maintained with low dose prednisolone and azathioprine (AZA). The aim of this study is to evaluate the efficiency of AZA monotherapy for maintenance treatment of children with AIH. MATERIALS AND METHODS: This study was a retrospective analysis of the 55 children with AIH. Patients were divided into two groups: combination therapy (CT) and AZA group based on maintenance therapy. Results of these two different maintenance treatments were compared in children with AIH. RESULTS: The mean age of the children was 10.67 ± 4.30 years (61.8% females) with a mean follow-up period of 46.8 ± 33.6 months. For maintenance treatment, 39 (70.9%) patients received AZA and 16 (29.1%) patients received CT. Relapse was observed in nine (19.6%) cases in the follow-up period; two were in the CT group (2/16; 12.5%) and seven (7/39; 17.9%) were in the AZA group (P = 0.620). In AZA group, the duration of remission was 22.2 ± 6.1 months and that was longer than CT group (P = 0.025). CONCLUSION: Our study suggests that AZA monotherapy is an effective and safe therapy for maintaining remission in children with AIH. AZA monotherapy may be used for maintenance treatment of children with AIH, except in cases of overlap syndrome and also to avoid side effects of long-term used steroids and to improve treatment compliance in proper cases.

Turkish Translation, Validation, and Reliability Analysis of Pediatric Eosinophilic Esophagitis Symptom Severity Module Version 2.0.

The Paediatric Eosinophilic Esophagitis Symptom Severity Modules Version 2.0 (T-PEESv2.0) was developed in English as a valid, reliable questionnaire for follow up. This work aimed to develop a Turkish version of T-PEESv2.0 via translation and cultural adaptation and then to test its validation and reliability. Methods: The PEESv2.0 was translated into Turkish by standardized procedural steps completed in cooperation with the Mapi Research Trust. The final version of the questionnaire was submitted to eosinophilic oesophagitis patients or their parents at 2 times point separated by 1 week. An age-matched control group was used to test the discriminant validity. Construct validity was tested using the Wilcoxon test, and internal consistency was tested using Cronbach's alpha. Test-retest reliability was measured with Cohen's kappa and intraclass correlation coefficient. Results: One hundred twenty-eight participants (70 patients, 58 parents) were enrolled. Fifty-eight (39.1%) of them completed T-PEESv2.0-parent by proxy and 70 (54.7%) were T-PEESv2.0. The Cronbach's alpha coefficient and intraclass correlation coefficient for test-retest reliability were >0.70 for both questionnaires and for all domain (frequency and severity) and total scores. For discriminant validity analysis, subscale (frequency and domain) and total scores of the patient group were compared with those of the control group. The subscale and total scores were significantly different between the groups (P < 0.05). Conclusion: T-PEESv2.0 appeared to be valid and reliable, ready to be introduced as a clinical and research tool for the assessment of patients with eosinophilic oesophagitis.

Late-onset graft-versus-host disease after pediatric living-related liver transplantation for Langerhans cell histiocytosis.

GVHD is the most common and well-known cause of morbidity and mortality following allogeneic BM transplantation. The GVHD following OLT is an uncommon complication but has a high mortality and poses a major diagnostic and therapeutic challenge. We herein discussed a 12-month-old girl with multi-system LCH, who developed end-stage liver disease despite intensive chemotherapy. She underwent ABO-compatible liver transplantation at 28 months while in remission from LCH. The donor was her 26-yr-old father. Post-operative course was uneventful. The GVHD manifested with skin rash and BM suppression on post-transplant day 94 and confirmed by both microchimerism and skin biopsy. Prednisolone, basiliximab, and ATG were administered immediately but the bone marrow suppression was not improved and the patient died because of Candida sepsis at six-month post-transplant. GVHD after OLT should be keep in mind in patients with rash and BM suppression after liver transplantation. In LDLT, a patient who carries risk factors should investigated for optimal HLA matching.