Life expectancy and risk factors for early death in patients with severe thalassemia syndromes in South India.

In spite of advances in chelation therapy and screening of blood, mortality associated with the most common life-threatening noncommunicable disease of children in India, transfusion-dependent thalassemia (TDT), remains poorly defined. This study aims at estimating death rates and mortality risk factors associated with TDT. The clinical records of 1087 patients from 5 thalassemia centers in India were retrospectively analyzed from 2011 to 2018. Median patient age was 8.5 years, with 107 patients older than 18 years; 656 patients were male and 431 were female. Demographic details and clinical parameters were analyzed at presentation and at last visit. With 41 recorded deaths, actuarial survival at 26.9 years was 50%, and under-5 mortality was 7 times higher than in the general population. Patients with transfusion-transmitted infections (TTIs) had 3.4 times higher risk for death (P = .031). Serum ferritin higher than 4000 ng/dL had 4.6 times higher risk for mortality compared with ferritin lower than 1000 ng/dL (P = .00063). A hemoglobin drop lower than 2 g/dL per week had 7.7 times higher mortality risk compared with a drop of less than 1 g/dL per week (P < .0001). Social determinants (sex, economic status, and distance from center), splenectomy, and even cardiac complications were not associated with higher mortality risk. Main causes of death were infection, iron overload, TTIs, and allo-immunization. Patients who received more than 4 years of adequate care had more than 66% mortality risk reduction (P < .0001). TDT in India continues to result in high mortality. Ineffective transfusion, TTIs, and chelation continue to be the most significant risk factors. Comprehensive care in dedicated day care centers from early age is likely to improve outcomes.

Persistent heterogeneity in diabetes technology reimbursement for children with type 1 diabetes: The SWEET perspective.

BACKGROUND: Frequent use of modern diabetes technologies increases the chance for optimal type 1 diabetes (T1D) control. Limited reimbursement influences the access of patients with T1D to these modalities and could worsen their prognosis. We aimed to describe the situation of reimbursement for insulins, glucometers, insulin pumps (CSII) and continuous glucose monitoring (CGM) for children with T1D in European countries participating in the SWEET Project and to compare data from EU countries with data from our previous study in 2009. METHODS: The study was conducted between March 2017 and August 2017. First, we approached diabetes technology companies with a survey to map the reimbursement of insulins and diabetic devices. The data collected from these companies were then validated by members of the SWEET consortium. RESULTS: We collected data from 29 European countries, whereas all types of insulins are mostly fully covered, heterogeneity was observed regarding the reimbursement of strips for glucometers (from 90 strips/month to no limit). CSII is readily available in 20 of 29 countries. Seven countries reported significant quota issues or obstacles for CSII prescription, and two countries had no CSII reimbursement. CGM is at least partially reimbursed in 17 of 29 countries. The comparison with the 2009 study showed an increasing availability of CSII and CGM across the EU. CONCLUSIONS: Although innovative diabetes technology is available, a large proportion of children with T1D still do not benefit from it due to its limited reimbursement.

Information Technology-Assisted Treatment Planning and Performance Assessment for Severe Thalassemia Care in Low- and Middle-Income Countries: Observational Study.

BACKGROUND: Successful models of information and communication technology (ICT) applied to cost-effective delivery of quality care in low- and middle-income countries (LMIC) are an increasing necessity. Severe thalassemia is one of the most common life-threatening noncommunicable diseases of children globally. OBJECTIVE: The aim was to study the impact of ICT on quality of care for severe thalassemia patients in LMIC. METHODS: A total of 1110 patients with severe thalassemia from five centers in India were followed over a 1-year period. The impact of consistent use of a Web-based platform designed to assist comprehensive management of severe thalassemia (ThalCare) on key indicators of quality of care such as minimum (pretransfusion) hemoglobin, serum ferritin, liver size, and spleen size were assessed. RESULTS: Overall improvements in initial hemoglobin, ferritin, and liver and spleen size were significant (P<.001 for each). For four centers, the improvement in mean pretransfusion hemoglobin level was statistically significant (P<.001). Four of five centers achieved reduction in mean ferritin levels, with two displaying a significant drop in ferritin (P=.004 and P<.001). One of the five centers did not record liver and spleen size on palpation, but of the remaining four centers, two witnessed a large drop in liver and spleen size (P<.01), one witnessed moderate drop (P=.05 for liver; P=.03 for spleen size), while the fourth witnessed a moderate increase in liver size (P=.08) and insignificant change in spleen size (P=.12). CONCLUSIONS: Implementation of computer-assisted treatment planning and performance assessment consistently and positively impacted indexes reflecting effective delivery of care to patients suffering from severe thalassemia in LMIC.

Prevalence of underweight, overweight, and obesity in children and adolescents with type 1 diabetes: Data from the international SWEET registry.

OBJECTIVE: To assess the prevalence of underweight (UW), overweight (OW), and obesity in children and adolescents with type 1 diabetes (T1D). METHODS: An international cross-sectional study including 23 026 T1D children (2-18 years, duration of diabetes ≥1 year) participating in the SWEET prospective, multicenter diabetes registry. Body mass index SD score (BMI-SDS) was calculated using the World Health Organization BMI charts. Children were categorized as UW (BMI-SDS < -2SD), OW (+1SD < BMI-SDS ≤ +2SD), and obese (OB) (BMI-SDS > +2SD). Hierarchic regression models were applied with adjustment for sex, age, and duration of diabetes. RESULTS: The prevalence of UW, OW, and obesity was: 1.4%, 22.3%, and 7.3% in males and 0.6%, 27.2%, and 6.8% in females. Adjusted BMI-SDS was significantly higher in females than in males (mean ± SEM: 0.54 ± 0.05 vs 0.40 ± 0.05, P < 0.0001). In males, BMI-SDS significantly decreased by age (P < 0.0001) in the first three age categories 0.61 ± 0.06 (2 to <10 years), 0.47 ± 0.06 (10 to <13 years), 0.34 ± 0.05 (13 to <16 years). In females, BMI-SDS showed a U-shaped distribution by age (P < 0.0001): 0.54 ± 0.04 (2 to <10 years), 0.39 ± 0.04 (10 to <13 years), 0.55 ± 0.04 (13 to <16 years). BMI-SDS increased by diabetes duration (<2 years: 0.38 ± 0.05, 2 to <5 years: 0.44 ± 0.05, and ≥5 years: 0.50 ± 0.05, P < 0.0001). Treatment modality did not affect BMI-SDS. Adjusted HbA1c was significantly higher in females than in males (8.20% ± 0.10% vs 8.06% ± 0.10%, P < 0.0001). In both genders, the association between HbA1c and BMI-SDS was U-shaped with the highest HbA1c in the UW and obesity groups. CONCLUSIONS: The high rate of OW and obesity (31.8%) emphasize the need for developing further strategies to prevent and treat excess fat accumulation in T1D.

Prevalence of thyroid dysfunction among type 2 diabetes mellitus patients.

AIMS: To determine the prevalence of Thyroid dysfunction among Type 2 Diabetes mellitus. METHODOLOGY: A cross-sectional hospital-based study is conducted to find out the prevalence of thyroid dysfunction. 713 type 2 diabetes mellitus (T2DM) subjects were enrolled in the survey. These subjects were investigated for fasting blood sugar (FBS), glycosylated haemoglobin (HbA1c), total triiodothyronine (T3), total thyroxine (T4), and thyroid-stimulating hormone (TSH). RESULTS: Prevalence of thyroid dysfunction in T2DM was found in 16.2%. Gender-specific prevalence found higher in females (25%) compared to males (10.1%) (P<0.001). Age-specific prevalence found higher in the age group ≥50 yrs. (19%) Compared to other age groups (P=0.036). Subjects with poor glycaemic control demonstrated higher prevalence (27.9%) (P=0.012). Subjects with long-standing T2DM had an increased risk for thyroid dysfunction (19.8%) though findings were not statistically significant. (P=0.42). CONCLUSION: The prevalence of thyroid dysfunction with T2DM (16.2%) was high in females compared to males and especially hypothyroidism being more typical. Failure to recognise the presence of thyroid dysfunction among T2DM patients may be a primary cause of poor management of diabetes. We recommend universal screening and regular monitoring of thyroid dysfunction in T2DM patients.

Contribution of SWEET to improve paediatric diabetes care in developing countries.

Diabetes affects many children living in developing countries. Through an informal survey, five SWEET (Better control in Pediatric and Adolescent diabeteS: Working to crEate CEnTers of Reference) centers from developing countries (Mali, Costa Rica, Argentina and two from India) share their perspective on caring for children with diabetes. Each center provides a description of the population of children with diabetes they serve, the organization of care, and the challenges encountered on a daily basis in the provision of this care. In the second part, we summarize the anticipated benefits and challenges associated with participation in SWEET. This resulting article is a testimony of the reality of managing diabetes by dynamic teams striving to achieve recommended standards of care for pediatric diabetes in an environment with limited resources.

Fibrocalculous pancreatic diabetes in adult.

Fibrocalculous pancreatic diabetes has distinctive features like younger age at onset, presence of large intraductal calculi, aggressive course of the disease, and proneness for pancreatic cancer. Pancreatic calculi are the hallmark for the diagnosis. We report a 32-year-old male patient, a known case of diabetes since 2 years, presented with recurrent pain abdomen, malabsorption, and neuropathic symptoms. The diagnosis was established on the basis of clinical examination, biochemical and radiological investigations. He was prescribed two doses of premix insulin and pancreatic enzyme supplements for relief of abdominal pain and steatorrhea.

Laparoscopic Mitrofanoff continent catheterisable stoma in children with spina bifida.

BACKGROUND: In 1980, Mitrofanoff described the creation of an appendicovesicostomy for continent urinary diversion. This procedure greatly facilitates clean intermittent catheterisation in patients with neurogenic bladder. The purpose of our study was to determine the clinical efficacy of the laparoscopic Mitrofanoff catheterisable stoma for children and adolescents with spina bifida. MATERIALS AND METHODS: Review of hospital records revealed that 11 children with spina bifida underwent a laparoscopic Mitrofanoff procedure with at least 1-year of follow-up. A four-port transperitoneal laparoscopic approach was used to create a Mitrofanoff appendicovesicostomy. The child was followed-up in the urology clinic at 6 weeks, 3 months, 6 months, 1-year, and then semiannually after that. Questionnaires were administered to determine, from the children's perspective, the level of satisfaction with catheterisation and the psychosocial implications of catheterisation before and after the creation of the Mitrofanoff continent catheterisable stoma. RESULTS: Of the 11 children, six were female, and five were male. The mean age at presentation to Paediatric urological services was 11 × 3.22 years. Overall the mean operative time was 144.09 × 17.00 min. Mean estimated blood loss was 37.36 × 11.44 cc. None of the cases needed conversion to open. Patient satisfaction with their catheterisation was measured at 2.18 × 0.98 preoperatively, Post-operatively, this improved to 4.27 × 0.46. Statistical analysis using paired t-test showed significance with P < 001. CONCLUSIONS: Laparoscopic Mitrofanoff catheterisable stoma is feasible in children with spina bifida and is associated with reasonable outcome with early recovery, resumption of normal activities and excellent cosmesis.

Preliminary experience with laparoscopic Foley's YV plasty for ureteropelvic junction obstruction in children.

INTRODUCTION: Laparoscopic dismembered pyeloplasty is an acceptable option for ureteropelvic junction (UPJ) obstruction in the paediatric population. We compared our results of laparoscopic dismembered and non-dismembered Foley's YV pyeloplasty. MATERIALS AND METHODS: Children presenting with hydronephrosis secondary to UPJ obstruction formed the study group. Foley's YV plasty was planned whenever it was observed that a tension free dismembered pyeloplasty was not possible in spite of all possible manoeuvres. Children were followed up for urinary infection, and renogram was repeated after 3 months. RESULTS: During the study period, 108 children (63 male and 45 female) with a mean age of 4.94 ± 2.78 years underwent laparoscopic dismembered pyeloplasty and the remaining 11 children (5 male and 6 female) with a mean age of 4.00 ± 1.776 years underwent laparoscopic Foley's YV plasty. There were no major peri-operative complications noted and conversion to open was not necessary in any child. Renogram done at 3 months post-operatively showed good drainage and improvement of renal function. CONCLUSIONS: Laparoscopic Foley's YV pyeloplasty is a safe and effective technique in appropriately selected cases of primary UPJ obstruction in children.

Laparoscopic mitrofanoff appendicovesicostomy: Our experience in children.

INTRODUCTION: The Mitrofanoff principle was originally described as a method to provide an alternative means to access the bladder. It creates a conduit to the bladder through which patients with a sensitive, absent, or traumatized urethra can perform clean intermittent catheterization (CIC) easily. We report our experience with complete laparoscopic Mitrofanoff appendicovesicostomy to promote a catheterizable abdominal stoma. MATERIALS AND METHODS: A 4-port transperitoneal approach was used to create a complete laparoscopic Mitrofanoff appendicovesicostomy. RESULTS: Six children with a mean age of 12.8 years (range 9-16 years) underwent laparoscopic Mitrofanoff appendicovesicostomy. Mean operative time was 139.6 min and Mean estimated blood loss was 46 cc. No cases of urinary leaks were noted. There have been no cases of either stomal stenosis or appendicovesical stenosis noted. CONCLUSIONS: Pure laparoscopic Mitrofanoff appendicovesicostomy is feasible and is associated with reasonable outcome with early recovery, resumption of normal activities and excellent cosmesis.

Staged buccal mucosa urethroplasty in reoperative hypospadias.

INTRODUCTION: Repeated attempts at surgical repair of serious complications involving either the partial or complete breakdown of the hypospadias repair are less likely to succeed because the penis is densely scarred, or significantly shortened, and the skin over the penis is immobile and hypovascular. Buccal mucosa (BM) has become the preferred material for reconstruction, whenever a child with skin-deficient hypospadias needs reoperation. We report the results of our surgical experience with staged reoperation using BM, in the repair of hypospadias in children with complications after multiple failed repairs. MATERIALS AND METHODS: Children needing reoperation for hypospadias underwent a staged repair using buccal mucosa. The complications were noted. RESULTS: Twenty-one children aged 3 - 16 years underwent this staged repair during the period May 2000 - April 2010. Two of these 21 children had a failed first stage. One child developed a urethro-cutaneous fistula following the second stage, which was corrected in an additional stage. CONCLUSIONS: The use of the buccal mucosa graft for urethral reconstruction in a child with hypospadias, needing a reoperation, is a successful method, with a low incidence of complications.

Laparoscopic hemi/partial nephrectomy in children with ureteral duplication anomalies.

INTRODUCTION: Recent advances in laparoscopic surgery as well as increasing experience with these techniques have led to the selection of laparoscopic surgery for hemi/partial nephroureterectomy in children with a non/poorly functioning moiety in a duplex kidney. There is very little data on the long term follow-up of such children. We report our experience of laparoscopic hemi-nephroureterectomy in children with duplex moiety. MATERIALS AND METHODS: We retrospectively reviewed the case records of children undergoing laparoscopic partial/hemi nephroureterectomy. Demographic data, age, weight, diagnosis, type of operation, operative time, concomitant and subsequent procedures, blood loss, use of drains, analgesic requirement, length of hospitalization and complications were recorded. RESULTS: 29 children (21 girls and 8 boys) underwent laparoscopic hemi/partial nephrectomy. The median operative time was 94 min (range 68-146 min). The mean blood loss was 25 ml and no children required perioperative blood transfusion. CONCLUSIONS: Laparoscopic hemi/partial nephrectomy in children is safe, associated with decreased morbidity and shorter hospitalization. Long term follow-up is necessary to study long term outcomes.

Type 1 diabetes mellitus with ketoacidosis in infancy.

Type 1 diabetes mellitus is considered a common form of diabetes mellitus in young people. Type 1 diabetes in infants is rare. However, the condition is rare in infants. Type 1 diabetes has not been reported in the literature in 45 days old child of an Indian population. Type 1 diabetes typically begins between the ages of 7 and 13 years, but 1-3% of patients are under 1 year of age. This communication describes a case of type 1 diabetes in a 45 days old male child which presented as diabetic ketoacidosis. It was effectively managed with continuous intravenous regular insulin infusion. The present report is made because of the rarity of the condition in the early age group of Indian children.

Familial early onset of type-2 diabetes mellitus and its complications.

BACKGROUND: Globally, the prevalence of chronic, non-communicable diseases is increasing at an alarming rate. Furthermore, approximately 197 million people worldwide have impaired glucose tolerance. Consequently, diabetes is rapidly emerging as a global health problem that threatens to assume a pandemic level by 2030. In Indian population, genetic predisposition to trigger diabetes at an early age as compared to western counterpart has been focused very much. AIM: To gain further insight into the positive correlation between the diabetes and family history was the objective of this study. MATERIALS AND METHODS: Patients attending the Diabetes Centre, K.L.E.S Dr. Prabhakar. Kore Hospital and Medical Research Centre; J. N. Medical College; KLE University Belgaum, Karnataka- India, were recruited, diagnosed and analyzed as per WHO criteria. RESULTS: The prevalence of diabetes was higher among patients with diabetic mother (25.6%) compared to patients with diabetic father (21.2%) and there was early onset of type -2 diabetes among patients having both parents with diabetic when compared to other patients. CONCLUSION: Based on the present observation, it would be appropriate to emphasize again that a strong family history for diabetes, would signal at an early age, the onset of diabetes perhaps with its complications.