Challenges in handling a civilian mass pediatric disaster during flood relief in a partially inundated armed forces medical facility.

BACKGROUND: Armed forces hospitals are often called upon to provide medical aid to civilians during natural calamities. Though children are often the most vulnerable segment of population in these events, research that addresses their unique needs and the role of armed forces hospitals remains sparse. OBJECTIVES: We examined pediatric morbidity and mortality at a flooded armed forces hospital. Factors that affected outcomes were identified. METHODS: 158 patients were evacuated en masse from a children's hospital in northern India that was submerged by flood to an adjacent partially inundated armed forces hospital specializing in military medicine and adult trauma. The children were provided case-based clinical care as per existing disaster management protocol. Geoclimatic vulnerability factors, morbidity/mortality, and medical and logistical challenges for future intervention were investigated. RESULTS: One pediatrician who provided initial triage was joined by two others after 48 hours. A limited load of adult patients permitted more resources for the children, majority (49 percent) of whom were neonates. Intensive care was necessitated for 32 (20.2 percent) cases, with half managed in adult ICU. Overall in-hospital mortality was 5.7 percent. Experienced staff, cross-specialty multitasking, and innovative and noncensorious leadership were identified as assets amidst resources compromised by flooding. Clear delineation of primary caregiver role of pediatrician at outset, pediatric emergency care training, pediatric triage, resource allocation for thermoregulation, oxygen therapy and ventilation, earmarking centers for transfer of cases, and safe transportation to the centers were identified as areas meriting further attention. CONCLUSION: Armed forces hospitals in vulnerable geoclimatic zones must address pediatric concerns in disaster management plans.

The diagnostic utility of rheumatoid factor and anticitrullinated protein antibody for rheumatoid arthritis in the Indian population.

Background: There is lack of Indian data on diagnostic utility of rheumatoid factor (RF) and anti-citrullinated protein antibody (ACPA) for diagnosis of rheumatoid arthritis (RA) and prevalence of these antibodies in patients with RA and the healthy population. The study was aimed to assess the diagnostic utility and prevalence of RF and ACPA at different titers in the Indian scenario. Method: All the patients of RA fulfilling the European League Against Rheumatism/American College of Rheumatology (EULAR/ACR) 2010 classification criteria and age and gender-matched healthy controls were included in the study. RF and ACPA were measured by nephelometry and the enzyme-linked immunosorbent assay (ELISA) method, respectively. Result: Of 803 patients (291 men and 512 women) included, the RF was positive in 566 (70.5%) study patients. The ACPA was positive in 527 (71.7%) patients of 735 of them. Among 408 healthy controls, 45 (11%) were RF positive and 19 (4.7%) were ACPA positive.At the positive cutoff level, the RF had a specificity of 87.6% (95% confidence interval [CI] = 84.4-90.8; positive likelihood ratio [LR+] 5.7). Specificity at 2 and 3 times above the upper limit of normal (ULN) increased to 96.2% (95% CI = 94.3-98.1; LR+ 15.7) and 97.1% (95% CI = 95.5-98.7; LR+ 17.1), respectively.The specificity of ACPA at the positive cutoff level was 94.4% (95% CI = 92.2-96.6; LR+ 12.7), which increased to 98% (95% CI = 96.6-99.4), at 2xULN level. The likelihood ratio for ACPA at all cutoff levels measured was more than 10. Conclusion: The sensitivity and specificity of RF and ACPA in our study population are comparable with those of other studies. ACPA at lower titers may have sufficient diagnostic utility for RA in an appropriate clinical setting.

Lessons Learnt From a Greenfield Hangar-Based 1,000-Bedded Temporary Hospital in India.

During the COVID-19 pandemic, the total number of hospital beds in the National Capital Region (NCR) of Delhi was 54,321 (roughly 300 beds per one lakh population), which was inadequate for the patients. Therefore, the Indian government initiated the construction of a 1,000-bedded greenfield hangar-based hospital to bridge the healthcare gap. As a result, Intensive Care Unit (ICU) beds in the facility augmented the COVID-19 care ICU beds in the city by 11%. The authors were involved in the planning, developing, and initiating the functioning of 1,000-bedded Dedicated COVID-19 Hospital (DCH). The hospital was conceptualized, built, and operationalized in 12 days only. Lessons learned from this experience would be of benefit should similar situations arise in future. Coordinating structural designing early with the entire project team-from facility administrators and medical practitioners to architects, consultants, and contractors-can result in a structure that better matches the facility's long-term needs and often saves construction time and costs. This article enumerates various challenges faced and the way they were addressed. This hangar-based hospital can be rapidly constructed and deployed on a massive scale. While structural integrity is essential, the planning team was particularly aware of the patient-centric modality of healthcare. Many modifications were carried out in the structure based on patient inputs. Informal discussions with discharged patients and relatives revealed that the human-centric approach was the mainstay of the therapy.

Pediatric Dystonic Storm: A Hospital-Based Study.

OBJECTIVE: Pediatric dystonic storm is an underrecognized entity. We aimed to evaluate the profiles of children presenting with dystonic storm in a referral hospital. Management schema and treatment responsiveness of this uncommonly reported entity were analyzed. METHODS: Retrospective review of all children (up to 18 years) hospitalized with dystonic storm over 39 months in the aforementioned facility. RESULTS: Twenty-three children whose ages ranged from 2 years 2 months to 14 years 4 months years (median: 6 years 11 months) (males: 13, females: 11) presented with dystonic storm. The annual incidence was 0.4 per 1,000 fresh admissions with an event rate of 0.9 per 1,000 for all admissions. All had Dystonia Severity Action Plan grades 4/5 with identifiable trigger in 13 (50%). Underlying dystonic disorder preexisted in 10 (43.4%); 8 of these had cerebral palsy. Polypharmacotherapy with >4 drugs out of trihexyphenidyl, tetrabenazine, clonazepam, gabapentin, levodopa-carbidopa, trichlorophos, and melatonin was needed. Supportive care and adequate sedation helped in symptom control. All children were managed with midazolam infusion over 2-10 days (median: 5 days). Mechanical ventilation was resorted to in 6 children (3-22 days). Vecuronium and propofol were used in 3/23 (13%) and 4/23 (17%) children, respectively. Deep brain stimulation was curative in 1 child. Hospitalization ranged from 5 to 31 (median: 11) days. Although there were no deaths, rhabdomyolysis was noted in 1 child. Postdischarge, 6 (26%) children relapsed. CONCLUSIONS: Dystonic storm is a medical emergency mandating aggressive multimodal management. Supportive care, antidystonic drugs, and early elective ventilation alongside adequate sedation with benzodiazepines ameliorate complications. Relapses of dystonic storm are not uncommon.

Comparison of a Voiding Diary With Clinical Management Tool As an Outpatient Screening Tool for Childhood Functional Voiding Disorders.

OBJECTIVE: To study the agreement of questionnaire-based assessment with voiding diary for differentiating primary mono-symptomatic nocturnal enuresis from voiding disorder in children. METHOD: Children 5-12 years old with bedwetting after exclusion of secondary enuresis were enrolled and parents filled a clinical management tool (CMT) questionnaire and a 48-hours voiding diary. Point prevalence and agreement of classification as primary mono-symptomatic nocturnal enuresis or voiding disorder were compared. RESULTS: Of 1276 children screened, 143 (11.2%) reported enuresis. Of 100 (82 males) children finally analyzed, constipation and positive family history occurred in 14% and 37%, respectively. Questionnaire-based assessment and voiding diary identified 65% and 71%, respectively as voiding disorder [Cohen's kappa 0.542 (95%CI: 0.367-0.717)]. Discordance of classification was noted in 20%. Voiding diary identified additional 7% cases of voiding disorder. CONCLUSIONS: While CMT and voiding diary have moderate agreement, voiding diary should be used for cases screened negative by a questionnaire-based tool.

Challenges in handling a civilian mass pediatric disaster during flood relief in a partially inundated armed forces medical facility.

BACKGROUND: Armed forces hospitals are often called upon to provide medical aid to civilians during natural calamities. Though children are often the most vulnerable segment of population in these events, research that addresses their unique needs and the role of armed forces hospitals remains sparse. OBJECTIVES: We examined pediatric morbidity and mortality at a flooded armed forces hospital. Factors that affected outcomes were identified. METHODS: 158 patients were evacuated en masse from a children's hospital in northern India that was submerged by flood to an adjacent partially inundated armed forces hospital specializing in military medicine and adult trauma. The children were provided case-based clinical care as per existing disaster management protocol. Geoclimatic vulnerability factors, morbidity/mortality, and medical and logistical challenges for future intervention were investigated. RESULTS: One pediatrician who provided initial triage was joined by two others after 48 hours. A limited load of adult patients permitted more resources for the children, majority (49 percent) of whom were neonates. Intensive care was necessitated for 32 (20.2 percent) cases, with half managed in adult ICU. Overall in-hospital mortality was 5.7 percent. Experienced staff, cross-specialty multitasking, and innovative and noncensorious leadership were identified as assets amidst resources compromised by flooding. Clear delineation of primary caregiver role of pediatrician at outset, pediatric emergency care training, pediatric triage, resource allocation for thermoregulation, oxygen therapy and ventilation, earmarking centers for transfer of cases, and safe transportation to the centers were identified as areas meriting further attention. CONCLUSION: Armed forces hospitals in vulnerable geoclimatic zones must address pediatric concerns in disaster management plans.

Transcobalamin deficiency: vitamin B12 deficiency with normal serum B12 levels.

Transcobalamin (TC) deficiency is a rare autosomal recessive inborn error of cobalamin transport which clinically manifests in early infancy. We describe a child with TC deficiency who presented with classical clinical and lab stigmata of inborn error of vitamin B12 metabolism except normal serum B12 levels. He was started on empirical parenteral cobalamin supplements at 2 months of age; however, the definitive diagnosis could only be established at 6 years of age when a genetic evaluation revealed homozygous nonsense variation in exon 8 of the TCN2 gene (chr22:g.31019043C>T).

Hemolytic uremic syndrome in a developing country: Consensus guidelines.

BACKGROUND: Hemolytic uremic syndrome (HUS) is a leading cause of acute kidney injury in children. Although international guidelines emphasize comprehensive evaluation and treatment with eculizumab, access to diagnostic and therapeutic facilities is limited in most developing countries. The burden of Shiga toxin-associated HUS in India is unclear; school-going children show high prevalence of anti-factor H (FH) antibodies. The aim of the consensus meeting was to formulate guidelines for the diagnosis and management of HUS in children, specific to the needs of the country. METHODS: Four workgroups performed literature review and graded research studies addressing (i) investigations, biopsy, genetics, and differential diagnosis; (ii) Shiga toxin, pneumococcal, and infection-associated HUS; (iii) atypical HUS; and (iv) complement blockade. Consensus statements developed by the workgroups were discussed during a consensus meeting in March 2017. RESULTS: An algorithm for classification and evaluation was developed. The management of Shiga toxin-associated HUS is supportive; prompt plasma exchanges (PEX) is the chief therapy in patients with atypical HUS. Experts recommend that patients with anti-FH-associated HUS be managed with a combination of PEX and immunosuppressive medications. Indications for eculizumab include incomplete remission with plasma therapy, life-threatening features, complications of PEX or vascular access, inherited defects in complement regulation, and recurrence of HUS in allografts. Priorities for capacity building in regional and national laboratories are highlighted. CONCLUSIONS: Limited diagnostic capabilities and lack of access to eculizumab prevent the implementation of international guidelines for HUS in most developing countries. We propose practice guidelines for India, which will perhaps be applicable to other developing countries.

Can microRNA profiles predict corticosteroid responsiveness in childhood nephrotic syndrome? A study protocol.

INTRODUCTION: In last few years, several studies have revealed the remarkable stability of extracellular microRNAs (miRNAs) circulating in the blood or excreted in the urine and underscored their key importance as biomarkers of certain diseases. Since miRNA in urinary sediment is relatively stable and easily quantified, it has the potential to be developed as a biomarker for disease diagnosis and monitoring. Identification of serum and urinary levels of certain miRNAs may assist in the diagnosis and assessment of disease activity in patients with nephrotic syndrome (NS). The global expression profile of miRNAs in childhood NS in Indian population remains unknown. Hence, further research is warranted in this area. This study seeks to prospectively evaluate whether a multipronged multiomics approach concentrating on microRNA expression profiles in children with NS vis-a-vis normal healthy children is discriminant enough to predict steroid responsiveness in childhood NS. METHODS AND ANALYSIS: In this prospective multicentric cohort study, subjects will be recruited from general paediatric and paediatric nephrology outpatient departments (OPDs) in tertiary care level referral hospitals. Age-matched and sex-matched healthy individuals with normal renal function (as assessed by normal serum creatinine and normal ultrasound of kidneys, ureter and bladder) in 1:1 ratio between study and control groups will be recruited from among the healthy siblings of children presenting to the OPDs. Differential microRNA expression profiles in urine and serum samples of children with steroid-sensitive NS (SSNS) and steroid-resistant NS (SRNS) with healthy children will be compared in a two-phased manner: a biomarker discovery phase involving pooled samples across SSNS, SRNS and healthy siblings analysed in triplicate using next-generation sequencing, slide microarray and quantitative reverse transcriptase PCR (qRT-PCR) arrays covering human miRNome followed by a validation phase with customised qRT-PCR primers based on the concordance in the discovery phase differential expression profiles and bioinformatics analysis. ETHICS AND DISSEMINATION: The study is funded after dueInstitutional Ethics Committee (IEC) clearance, and results will be available as open access.

Incidence of mycobacterial disorders in Indian adult male service population followed for over two decades.

BACKGROUND: The comprehensive epidemiology of mycobacterial disorders is scarce from our country. The incidence of Tuberculosis (TB) and Leprosy in a cohort of military personnel followed for a long duration was evaluated in this study. METHODS: The data for this descriptive epidemiologic study was derived from the electronic medical records (EMR) data of the service personnel enrolled between 1990 and 2015. They were recruited between the ages of 17 and 18 years in good health and their morbidity data was derived from the medical records. The incidence rate (IR) was calculated as per person-years (py) using appropriate statistical methods. RESULTS: The study population includes 51,217 participants (median age 33 years, range 17-54) with a mean follow up of 12.5 years. Yearly evaluation of the data gave a cumulative follow up duration of 613,925py. A total of 530 patients developed TB, giving an IR of 86.3 per 100,000 person years (95% CI 79.2-93.9). Leprosy was diagnosed in 59 cases giving an IR of 9.6 per 100,000py (95% CI 7.4-12.3). Pulmonary (71%) and pleural (24%) locations were the most common sites of the TB infection. The data about the contribution of the mycobacterial disorders towards the mortality and the subtypes of leprosy was not available in the EMR. CONCLUSION: Low IR of mycobacterial disorders was observed in this study when compared with the previous reports. Healthy lifestyle and good socioeconomic status could explain the low IR of mycobacterial disorders in the military personnel.

Device-Associated Infection Rates in 20 Cities of India, Data Summary for 2004-2013: Findings of the International Nosocomial Infection Control Consortium.

OBJECTIVE: To report the International Nosocomial Infection Control Consortium surveillance data from 40 hospitals (20 cities) in India 2004-2013. METHODS: Surveillance using US National Healthcare Safety Network's criteria and definitions, and International Nosocomial Infection Control Consortium methodology. RESULTS: We collected data from 236,700 ICU patients for 970,713 bed-days Pooled device-associated healthcare-associated infection rates for adult and pediatric ICUs were 5.1 central line-associated bloodstream infections (CLABSIs)/1,000 central line-days, 9.4 cases of ventilator-associated pneumonia (VAPs)/1,000 mechanical ventilator-days, and 2.1 catheter-associated urinary tract infections/1,000 urinary catheter-days In neonatal ICUs (NICUs) pooled rates were 36.2 CLABSIs/1,000 central line-days and 1.9 VAPs/1,000 mechanical ventilator-days Extra length of stay in adult and pediatric ICUs was 9.5 for CLABSI, 9.1 for VAP, and 10.0 for catheter-associated urinary tract infections. Extra length of stay in NICUs was 14.7 for CLABSI and 38.7 for VAP Crude extra mortality was 16.3% for CLABSI, 22.7% for VAP, and 6.6% for catheter-associated urinary tract infections in adult and pediatric ICUs, and 1.2% for CLABSI and 8.3% for VAP in NICUs Pooled device use ratios were 0.21 for mechanical ventilator, 0.39 for central line, and 0.53 for urinary catheter in adult and pediatric ICUs; and 0.07 for mechanical ventilator and 0.06 for central line in NICUs. CONCLUSIONS: Despite a lower device use ratio in our ICUs, our device-associated healthcare-associated infection rates are higher than National Healthcare Safety Network, but lower than International Nosocomial Infection Control Consortium Report.

Equipment and Analytical Companies Meeting Continuous Challenges May 20-21 2014 Continuous Manufacturing Symposium.

This white paper focuses on equipment, and analytical manufacturers' perspectives, regarding the challenges of continuous pharmaceutical manufacturing across five prompt questions. In addition to valued input from several vendors, commentary was provided from experienced pharmaceutical representatives, who have installed various continuous platforms. Additionally, a small medium enterprise (SME) perspective was obtained through interviews. A range of technical challenges is outlined, including: the presence of particles, equipment scalability, fouling (and cleaning), technology derisking, specific analytical challenges, and the general requirement of improved technical training. Equipment and analytical companies can make a significant contribution to help the introduction of continuous technology. A key point is that many of these challenges exist in batch processing and are not specific to continuous processing. Backward compatibility of software is not a continuous issue per se. In many cases, there is available learning from other industries. Business models and opportunities through outsourced development partners are also highlighted. Agile smaller companies and academic groups have a key role to play in developing skills, working collaboratively in partnerships, and focusing on solving relevant industry challenges. The precompetitive space differs for vendor companies compared with large pharmaceuticals. Currently, there is no strong consensus around a dominant continuous design, partly because of business dynamics and commercial interests. A more structured common approach to process design and hardware and software standardization would be beneficial, with initial practical steps in modeling. Conclusions include a digestible systems approach, accessible and published business cases, and increased user, academic, and supplier collaboration. This mirrors US FDA direction. The concept of silos in pharmaceutical companies is a common theme throughout the white papers. In the equipment domain, this is equally prevalent among a broad range of companies, mainly focusing on discrete areas. As an example, the flow chemistry and secondary drug product communities are almost entirely disconnected. Control and Process Analytical Technologies (PAT) companies are active in both domains. The equipment actors are a very diverse group with a few major Original Equipment Manufacturers (OEM) players and a variety of SME, project providers, integrators, upstream downstream providers, and specialist PAT. In some cases, partnerships or alliances are formed to increase critical mass. This white paper has focused on small molecules; equipment associated with biopharmaceuticals is covered in a separate white paper. More specifics on equipment detail are provided in final dosage form and drug substance white papers. The equipment and analytical development from laboratory to pilot to production is important, with a variety of sensors and complexity reducing with scale. The importance of robust processing rather than overcomplex control strategy mitigation is important. A search of nonacademic literature highlights, with a few notable exceptions, a relative paucity of material. Much focuses on the economics and benefits of continuous, rather than specifics of equipment issues. The disruptive nature of continuous manufacturing represents either an opportunity or a threat for many companies, so the incentive to change equipment varies. Also, for many companies, the pharmaceutical sector is not actually the dominant sector in terms of sales. © 2014 Wiley Periodicals, Inc. and the American Pharmacists Association.

Equipment and analytical companies meeting continuous challenges. May 20-21, 2014 Continuous Manufacturing Symposium.

This white paper focuses on equipment, and analytical manufacturers' perspectives, regarding the challenges of continuous pharmaceutical manufacturing across five prompt questions. In addition to valued input from several vendors, commentary was provided from experienced pharmaceutical representatives, who have installed various continuous platforms. Additionally, a small medium enterprise (SME) perspective was obtained through interviews. A range of technical challenges is outlined, including: the presence of particles, equipment scalability, fouling (and cleaning), technology derisking, specific analytical challenges, and the general requirement of improved technical training. Equipment and analytical companies can make a significant contribution to help the introduction of continuous technology. A key point is that many of these challenges exist in batch processing and are not specific to continuous processing. Backward compatibility of software is not a continuous issue per se. In many cases, there is available learning from other industries. Business models and opportunities through outsourced development partners are also highlighted. Agile smaller companies and academic groups have a key role to play in developing skills, working collaboratively in partnerships, and focusing on solving relevant industry challenges. The precompetitive space differs for vendor companies compared with large pharmaceuticals. Currently, there is no strong consensus around a dominant continuous design, partly because of business dynamics and commercial interests. A more structured common approach to process design and hardware and software standardization would be beneficial, with initial practical steps in modeling. Conclusions include a digestible systems approach, accessible and published business cases, and increased user, academic, and supplier collaboration. This mirrors US FDA direction. The concept of silos in pharmaceutical companies is a common theme throughout the white papers. In the equipment domain, this is equally prevalent among a broad range of companies, mainly focusing on discrete areas. As an example, the flow chemistry and secondary drug product communities are almost entirely disconnected. Control and Process Analytical Technologies (PAT) companies are active in both domains. The equipment actors are a very diverse group with a few major Original Equipment Manufacturers (OEM) players and a variety of SME, project providers, integrators, upstream downstream providers, and specialist PAT. In some cases, partnerships or alliances are formed to increase critical mass. This white paper has focused on small molecules; equipment associated with biopharmaceuticals is covered in a separate white paper. More specifics on equipment detail are provided in final dosage form and drug substance white papers. The equipment and analytical development from laboratory to pilot to production is important, with a variety of sensors and complexity reducing with scale. The importance of robust processing rather than overcomplex control strategy mitigation is important. A search of nonacademic literature highlights, with a few notable exceptions, a relative paucity of material. Much focuses on the economics and benefits of continuous, rather than specifics of equipment issues. The disruptive nature of continuous manufacturing represents either an opportunity or a threat for many companies, so the incentive to change equipment varies. Also, for many companies, the pharmaceutical sector is not actually the dominant sector in terms of sales.

Steroid responsive encephalopathy associated with autoimmune thyroiditis (SREAT) in childhood.

AIM: Steroid responsive encephalopathy with autoimmune thyroiditis (SREAT) is a clinically and electrographically heterogeneous steroid-responsive encephalopathy associated with thyroid autoantibodies. We report an adolescent with SREAT and review PubMed literature relating to childhood. METHODS: A 14-year-old boy, without any preceding history of trauma, meningoencephalitis or seizures, was admitted in a comatose state. A similar episode of loss of consciousness 2 months prior with normal neuroimaging and electroencephalogram (EEG) had been followed by behavioral alterations. A year previously, during evaluation for increased appetite and poor weight gain, he was noted to have small goitre with thyroid-stimulating hormone (TSH) 7.26 mIU/L, T3 1.232 nmol/L, and T4 117.63 nmol/L. Routine hemogram, blood biochemistry, thyroid function tests including free hormone levels, ultrasonography thyroid and magnetic resonance imaging were normal. EEG showed diffuse slowing of all waves. Cerebrospinal fluid showed no pleocytosis and electrophoresis showed oligoclonal band. Viral studies and serum N-methyl-D-aspartate receptor antibody levels were negative. Anti-thyroid peroxidase (Anti-TPO) antibodies were raised. Intervention was with intravenous dexamethasone 4 mg every 6 h for 1 week followed by tapering schedule of oral prednisolone over 6 months. RESULTS: He regained consciousness after the second dose of dexamethasone and was discharged on day 7 in a fully conscious and ambulant state on a tapering course of low dose prednisolone for 6 months. He remains euthyroid with normal sensorium and behavior at 18 months follow-up. Only 50 cases below 18 years age were identified amongst 300 PubMed articles up to 31 July 2013. CONCLUSION: Prompt steroid therapy following early recognition by high clinical suspicion and measurement of antithyroid antibody titers can lead to a favorable prognosis in SREAT.

Outcome of severe steroid-dependent nephrotic syndrome treated with mycophenolate mofetil.

BACKGROUND: Mycophenolate mofetil (MMF) is used as a steroid-sparing agent in pediatric nephrotic syndrome (NS). However, data about its long-term efficacy and safety is limited. METHODS: We report the long-term outcome of MMF therapy in 46 NS patients who remained steroid dependent (SD) despite previous treatment with levamisole and cyclophosphamide. RESULTS: After 1 year of MMF initiation, 32 (70 %) patients had reduced steroid requirement: 12 with decreased threshold dose and 20 were able to stop steroids. At follow-up of mean 3.56 (standard deviation + 1.76) years, 25 (54 %) children required no further alternative immunosuppression (IS), having infrequent or no relapses, of which 14 stopped MMF after a mean 2.4 (standard deviation + 0.9) years; 11 are continuing on MMF for a median of 2.25 years (range 1.33-7.75 years). One patient had a psoriasis flare, and MMF was stopped. No other patient required permanent drug withdrawal due to side effects. The outcome of patients who did not require further alternate IS was significantly better than those who did, with 56 % vs. 10.5 %, respectively, being off regular medications at last follow-up. CONCLUSIONS: We conclude that MMF therapy is safe in the long term and allows >50 % of severe SDNS patients to avoid further toxic IS.

An unusual cause of delayed puberty: Berardinelli- Seip syndrome.

Berardinelli- Seip syndrome is an autosomal recessive disorder characterized by generalized lipoatrophy, extreme insulin resistance with dyslipidemia in childhood and development of diabetes in adolescence. Menstrual irregularities are commonly seen as a result of secondary polycystic ovarian syndrome. Delayed puberty as a manifestation of these abnormalities in girls has rarely been described earlier. We report one such case patient who had delayed puberty and portal hypertension as unique features amongst the characteristic phenotypes of this syndrome.