Self-reported level of and factors influencing the compliance to desferrioxamine therapy in multitransfused thalassaemias.

AIM: To analyse the self-reported degree of and factors influencing the compliance to desferrioxamine (DFO) therapy in children with transfusion-dependent thalassaemia major in Malaysia. METHODS: A cross-sectional study was conducted on all children with thalassaemia major on DFO attending Likas Hospital, Sabah, in September 2008. Patients or carer-providers were interviewed to report on the degree of compliance as either highly (administering >90% of DFO), moderately (51-90%), poorly (1%-50%) or non-compliant (0%) to DFO in the preceding month. The latest serum ferritin levels were noted. RESULTS: The median (range) age at first blood transfusion of these 139 (73 males, median (range) age at interview: 9.0 years (2-16 years)) patients were 1.0 year (2 months to 10 years). The median (range) duration of regular DFO therapy was 2 years (2 months to 10 years). Forty-three (31%) of the patients reported themselves to be highly compliant, 70 (50%) moderately and 26 (19%) poorly or non-compliant. Multivariate analysis showed that a lower family income negatively affected the degree of compliance, while family support positively affected the degree of compliance to DFO. No correlation existed between self-reported degree of compliance and latest serum ferritin level (6444 ± µmol/L; P = 0.186). CONCLUSION: The self-reported compliance to DFO therapy was moderate in this cohort of patients with thalassaemia major in Malaysia. The serum ferritin level was high, possibly because of the relatively short duration of DFO therapy and compliance. Oral iron chelator should be considered to improve the compliance to iron chelation.

Use of probiotics in clinical practice with special reference to diarrheal diseases: A position statement of the Malaysian Society of Gastroenterology and Hepatology.

Probiotics comprise a large group of microorganisms, which have different properties and thus confer different benefits. The use of probiotics has shown promising results in the management of diarrheal diseases. While the availability of probiotic products has flourished in the marketplace, there is limited guidance on the selection of probiotics for clinical use. This position paper is aimed at informing clinicians about the proper selection criteria of probiotics based on current evidence on strain-specific efficacy and safety for the management of diarrheal diseases. Members of the working group discussed issues on probiotic use in clinical practice, which were then drafted into statements. Literature to support or refute the statements were gathered through a search of medical literature from 2011 to 2020. Recommendations were formulated based on the drafted statements and evidence gathered, revised as necessary, and finalized upon agreement of all members. Twelve statements and recommendations were developed covering the areas of quality control in the manufacturing of probiotics, criteria for selection of probiotics, and established evidence for use of probiotics in diarrheal diseases in adults and children. Recommendations for the use of specific probiotic strains in clinical practice were categorized as proven and probable efficacy based on strength of evidence. Robust evidence is available to support the use of probiotics for diarrheal diseases in clinical practice. Based on the results obtained, we strongly advocate the careful evaluation of products, including manufacturing practices, strain-specific evidence, and contraindications for at-risk populations when choosing probiotics for use in clinical practice.

Childhood autoimmune liver disease: indications and outcome of liver transplantation.

BACKGROUND: Graft rejection and disease recurrence are well-recognized complications of liver transplantation (LT) for autoimmune hepatitis (AIH) and autoimmune sclerosing cholangitis (AISC). We describe indications and outcome of LT for childhood AIH and AISC. PATIENTS AND METHODS: Twenty-year retrospective review of a cohort of children (n = 101) with AIH, AISC, or AIH/sclerosing cholangitis overlap syndrome from a single center. RESULTS: AIH type 1 (AIH1, n = 67) was more common than AIH type 2 (AIH2, n = 18), AISC (n = 8), or overlap syndrome (n = 8). Overall, 18 patients (18%) required LT, the indications being failure of medical therapy (n = 16) and fulminant liver failure (n = 2). Patients with AIH who required LT had a more prolonged prothrombin time at presentation compared with those who did not undergo transplantation (P = 0.01). Patients with AIH1 who received LT had a lower aspartate transaminase (P = 0.009) and alanine transaminase (P = 0.02) levels at initial diagnosis compared with those with AIH1 who did not undergo transplantation. Post-LT, 11 patients (61%) had 18 episodes of rejection, most were steroid sensitive. Disease recurrence was observed in 7 patients (39%, median duration post-LT 33 months), more common in AIH2 (80% recurrence rate), and those taking cyclosporine (71%, 5/7 patients) compared with those taking tacrolimus (18%, 2/11 patients; P < 0.05) and in 3 of 3 children who did not have maintenance steroids post-LT. The overall 5- and 7-year post-LT survival rate was 94% and 88%, respectively. CONCLUSIONS: LT is a good therapeutic option for progressive AIH and AISC, although recurrence of the primary autoimmune process limits the outcome.

Outcome of biliary atresia in Malaysia: a single-centre study.

AIM: This study aimed to determine the outcome of the operation of children with biliary atresia (BA) at a tertiary paediatric referral centre in Malaysia. METHODS: A prospective study on all patients with BA referred to the University of Malaya Medical Centre (UMMC), Kuala Lumpur, from 1996 to 2005 was conducted. Survival with native liver, liver transplantation (LT) or death at 2 years of age was determined. RESULTS: The median age at referral of the 57 patients with BA seen at University of Malaya Medical Centre was 62 days. Kasai procedure was not performed in nine patients who were all referred late (median age of referral 180 days). The median age at hepatoportoenterostomy (HPE) of the remaining 48 patients was 70 days. A total of 53 (93%) patients had type 3 BA, while only 1 (2%) patient had BA splenic malformation. At 2 years, the survival rate with native liver for the 48 patients who had HPE was 37%, while the overall survival (native liver and LT) rate was 40%. Two of the four patients who had LT survived with a liver graft at 2 years. CONCLUSIONS: The 2-year survival with native liver following corrective surgery for BA in UMMC, Malaysia, compares favourably with other international figures, but the overall survival rate was adversely affected by a lack of timely LT. The outcome of BA in Malaysia may be further improved by increasing the awareness among child-health professionals on the importance early referral for appropriate surgery in infants suspected of having BA.

Appropriateness, endoscopic findings and contributive yield of pediatric gastrointestinal endoscopy.

AIM: To determine the predictability of the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) and American Society for Gastrointestinal Endoscopy (ASGE) guideline with regard to appropriate endoscopic practice in children, positive endoscopic findings and contributive yield in clinical practice. METHODS: This was a descriptive, retrospective analysis, conducted at the Department of Paediatrics, University Malaya Medical Centre, Malaysia. All children who had esophagogastroduodenoscopy (EGD) and colonoscopy from January 2008 to June 2011 were included. An endoscopy was considered appropriate when its indication complied with the NASPGHAN and ASGE guideline. All endoscopic findings were classified as either positive (presence of any endoscopic or histologic abnormality) or negative (no or minor abnormality, normal histology); effecting a positive contributive (a change in therapeutic decisions or prognostic consequences) or non-contributive yield (no therapeutic or prognostic consequences). RESULTS: Overall, 76% of the 345 procedures (231 EGD alone, 26 colonoscopy alone, 44 combined EGD and colonoscopy) performed in 301 children (median age 7.0 years, range 3 months to 18 years) had a positive endoscopic finding. Based on the NASPGHAN and ASGE guideline, 99.7% of the procedures performed were considered as appropriate. The only inappropriate procedure (0.3%) was in a child who had EGD for assessment of the healing of gastric ulcer following therapy in the absence of any symptoms. The overall positive contributive yield for a change in diagnosis and/or management was 44%. The presence of a positive endoscopic finding was more likely to effect a change in the therapeutic plan than an alteration of the initial diagnosis. A total of 20 (5.8%) adverse events were noted, most were minor and none was fatal. CONCLUSION: The NASPGHAN and ASGE guideline is more likely to predict a positive endoscopic finding but is less sensitive to effect a change in the initial clinical diagnosis or the subsequent therapeutic plan.

Impact on parents during hospitalisation for acute diarrhoea in young children.

INTRODUCTION: This study aimed to determine the emotional impact on parents of young children who require hospitalisation for acute diarrhoea (AD), and the disruption of daily activities experienced and costs incurred by them. METHODS: A prospective study was conducted on children below two years of age with AD admitted to two urban hospitals in Malaysia. Parents were interviewed on the emotional impact and disruption of daily activities experienced by them, as well as the extra costs incurred as a result of the hospitalisation of their child. RESULTS: The parents of 85 children (median age 13 months; boys n = 58, 68%; girls n = 27, 32%) were recruited for the study. The proportions of parents who reported that they were very worried about the symptoms of diarrhoea, vomiting and fever in their child were 82%, 83% and 78%, respectively. Parents also reported being upset, helpless, mentally and physically exhausted, and having experienced loss of sleep and disruption of daily routine during the hospitalisation of their child (median four days). The median extra cost (including out-of-pocket cost and loss of income) incurred by parents as a result of the hospitalisation was USD 252.86, which constituted 16% of the combined monthly family income. CONCLUSION: Although short, AD-related hospitalisation in young children has considerable emotional impact and creates significant distress, in addition to causing significant financial burden for parents.

Rotavirus genotypes in Malaysia and universal rotavirus vaccination.

Group A rotavirus (RV-A) genotypes isolated in Malaysia was studied to estimate the effectiveness of a universal RV-A vaccination in Malaysia. A simple mathematical model was used, with input from a two-year, two-center, prospective study on hospitalization of RV-A gastroenteritis (RVGE) in young children, published data on RV-A hospitalizations and genotypes, mortality on childhood GE and published genotype-specific efficacy data on two RV-A vaccines. Assuming a 95% vaccine coverage, the overall projected effectiveness was 75.7 to 88.1% for Rotateq and 78.7 to 90.6% for Rotarix® against RVGE-related hospitalizations. The projected annual reduction in RVGE-related deaths was 27 to 32 deaths (from 34 deaths) for Rotateq and 28 to 32 deaths annually for Rotarix. A universal RV-A vaccine is efficacious in reducing RVGE-related hospitalizations and mortality in Malaysia.

Clinical features differentiating biliary atresia from other causes of neonatal cholestasis.

INTRODUCTION: This study determined any clinical features which may help to differentiate biliary atresia (BA) from other causes of neonatal cholestasis (NC). MATERIALS AND METHODS: A prospective and observational study was conducted on consecutive infants with NC referred to the University of Malaya Medical Centre, Malaysia, between November 1996 and May 2004. RESULTS: The 3 most common causes of cholestasis among the 146 infants with NC studied were idiopathic neonatal hepatitis (n = 63, 43%), BA (n = 35, 24%) and congenital cytomegalovirus hepatitis (n = 13, 9%). Common clinical features at presentation were jaundice (100%), hepatomegaly (95%), splenomegaly (52%) and pale stools (47%). Three clinical features noted to be sensitive for BA were the presence of acholic or variably acholic stools on admission, a liver which was firm/hard in consistency and a palpable liver of ≥4 cm (sensitivity of 77%, 80% and 94%, respectively), but the corresponding specificity was poor (51%, 65% and 39%, respectively). The stools of 2 children with BA were pigmented initially but became acholic subsequently. CONCLUSIONS: We did not find any single clinical feature with sufficient sensitivity and specificity to differentiate BA from other causes of NC. Repeated inspection of stools colour is necessary as occasionally, patients with BA may have initial pigmented stools. Biochemical assessment and imaging studies are important in the assessment of any infant with NC.