Surgery compared to fibrinolytic therapy for symptomatic left-sided prosthetic heart valve thrombosis (SAFE-PVT): Rationale and design of a randomized controlled trial.

BACKGROUND: Left-sided mechanical prosthetic heart valve thrombosis (PVT) occurs because of suboptimal anticoagulation and is common in low-resource settings. Urgent surgery and fibrinolytic therapy (FT) are the two treatment options available for this condition. Urgent surgery is a high-risk procedure but results in successful restoration of valve function more often and is the treatment of choice in developed countries. In low-resource countries, FT is used as the default treatment strategy, though it is associated with lower success rates and a higher rate of bleeding and embolic complications. There are no randomized trials comparing the two modalities. METHODS: We performed a single center randomized controlled trial comparing urgent surgery (valve replacement or thrombectomy) with FT (low-dose, slow infusion tissue plasminogen activator, tPA) in patients with symptomatic left-sided PVT. The primary outcome was the occurrence of a complete clinical response, defined as discharge from hospital with completely restored valve function, in the absence of stroke, major bleeding or non-CNS systemic embolism. Outcome assessment was done by investigators blinded to treatment allocation. The principal safety outcome was the occurrence of a composite of in-hospital death, non-fatal stroke, non-fatal major bleed or non-CNS systemic embolism. Outcomes will be assessed both in the intention-to-treat, and in the as-treated population. We will also report outcomes at one year of follow-up. The trial has completed recruitment. CONCLUSION: This is the first randomized trial to compare urgent surgery with FT for the treatment of left-sided PVT. The results will provide evidence to help clinicians make treatment choices for these patients. (Clinical trial registration: CTRI/2017/10/010159).

Ballerina foot in heart.

A 67-year-old woman with no known risk factors for coronary artery disease presented to the outpatient department with complaints of exertional fatigue and angina New York Heart Association class III.

Congenital Diarrhea and Enteropathies in Infants: Approach to Diagnosis.

Congenital diarrhea and enteropathies (CODEs) are monogenic disorders causing early onset of intractable diarrhea. Their diagnosis and management are challenging. With the availability of commercial next generation genetic testing, we are now better able to classify and manage these disorders. The authors present their experience with 4 cases. Two patients had congenital tufting enteropathy (CTE) and 1 case each of microvillous inclusion disease (MVID) and trichohepatoenteric syndrome (THES). Age at onset varied from 3 to 38 d of life. Light microscopy and electron microscopy of duodenal and rectal endoscopic biopsies were consistent with the diagnosis. Genetic evaluation was possible in 3 cases indicating causative mutations. Two children (CTE and MVID) were alive at last follow-up. The authors suggest a stepwise approach to the diagnosis and management of these disorders in the Indian context.

Preterm White Matter Injury: A Prospective Cohort Study.

OBJECTIVE: To determine the incidence and risk factors of preterm white matter injury [WMI; periventricular-intraventricular hemorrhage (PIVH) and/or periventricular leukomalacia (PVL)]. DESIGN: Prospective cohort study. SETTING: Level-3 neonatal intensive care unit. PATIENTS: Inborn preterm neonates (n=140) delivered at <32 weeks gestation or birthweight <1500 g. METHODS: Serial cranial ultrasounds were performed at postnatal ages of 3 days (±12 hour), 7 (±1) days, 21 (±3) days and 40 (±1) weeks postmenstrual age (PMA). PIVH and PVL were graded as per Volpe and De-Vries criteria, respectively. Univariate followed by multivariate analysis was done to evaluate risk factors for PIVH and PVL. OUTCOME MEASURES: The primary outcome was the incidence of preterm WMI. The secondary outcomes were evaluation of risk factors and natural course of WMI. RESULTS: The mean (range) gestation and birth weight of enrolled neonates were 29.7 (24-36) weeks and 1143 (440-1887) g, respectively. PIVH occurred in 25 (17.8%) neonates. PVL occurred in 34 (24.3%) neonates. None of them were grade III or IV PVL. Preterm WMI (any grade PIVH and/or PVL) occurred in 52 (37.1%) neonates. Severe PIVH (grade III) and cystic PVL occurred in 7 (5%) and 5 (3.6%) neonates, respectively. On multivariate analysis, none of the presumed risk factors were associated with PIVH. However, hemodynamically significant patent ductus arteriosus, and apnea of prematurity were significantly associated with increased risk of PVL. CONCLUSIONS: Significant WMI occurred only in one-third of the cohort, which is comparable to that described in literature from the developed countries.

Elizabethkingia anophelis infection in an infant: an unusual presentation.

A 7-month-old male infant presented with history of fever for 2 weeks, multiple ecchymotic patches over face, trunk and lower limbs, and one episode of seizure. The infant had shock, respiratory failure, severe anaemia, thrombocytopenia and temporoparietal haematoma on CT scan of the head. He was managed with supportive care and broad-spectrum empiric antibiotics. Two consecutive blood cultures grew Elizabethkingia anophelis, sensitive only to piperacillin-tazobactam. The infant responded to therapy and was discharged after 2 weeks of hospital stay. Repeated coagulation studies done to rule out an underlying bleeding disorder were negative. There was no clue in favour of non-accidental trauma. We report this case to highlight the unusual clinical presentation of this emerging pathogen. Mostly reported in outbreaks from surgical and post-operative intensive care units, it was worrisome to find this infant presenting with community-acquired E. anophelis infection.

Cystic Fibrosis Presenting as Pseudo-Bartter Syndrome: An Important Diagnosis that is Missed!

Cystic fibrosis (CF), an autosomal recessive disorder, occurs due to mutations in CFTR gene resulting in impaired cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel function in various epithelia. In addition to the well-known pulmonary and pancreatic morbidities, CF is characterized by electrolyte and acid-base abnormalities- hypochloremia, hyponatremia, hypokalemia and metabolic alkalosis. These are collectively known as Pseudo-Bartter syndrome, as similar abnormalities are seen in Bartter syndrome- an inherited tubulopathy affecting thick ascending limb of loop of Henle. There may be a significant clinical overlap between the Classic Bartter syndrome, Gitelman syndrome and CF presenting as Pseudo-Bartter syndrome, especially in early childhood. This review focuses on Pseudo-Bartter syndrome in CF, its pathogenesis and differentiation from Bartter/Gitelman syndrome. Other causes of metabolic abnormalities resembling Bartter syndrome are also highlighted.

Osteomyelitis in limb amputated by amniotic band sequence.

A preterm (30+2 week) neonate with below-knee amputation (right lower limb), constriction rings and syndactyly, subsequent to amniotic band sequence, developed pus discharge from the right tibial stump. The neonate did not have clinical features of systemic sepsis. Blood culture was sterile. The pus culture, however, grew methicillin-resistant coagulase-negative Staphylococcus and bone scan was suggestive of osteomyelitis of right proximal tibial stump. Osteomyelitis was likely caused by the contiguous spread of infection from the exposed stump. Neonate was treated with intravenous antibiotics for 4 weeks and discharged on oral feeds.

Juvenile dermatomyositis with IgA nephropathy: case-based review.

Juvenile dermatomyositis (JDM) is the most common childhood idiopathic inflammatory myopathy (IIM). It is characterized by the classic skin rash in the form of Gottron papules and heliotrope rash, and symmetric proximal muscle weakness. Renal involvement in JDM is rare which includes acute kidney injury and glomerulonephritis. We report a 10-year-old boy with juvenile dermatomyositis and IgA nephropathy. Child responded dramatically to the conventional therapy with steroids and methotrexate for the primary disease, and did not require any additional treatment for his renal disease. Child's primary disease is in remission and has normal urinalysis with normal renal function at 6-month follow-up. We reviewed the literature and found 11 cases of IIMs with renal involvement. Four patients (one JDM, two polymyositis, and one dermatomyositis) had IgA nephropathy out of which three patients responded to the conventional therapy of primary disease and only one patient with polymyositis needed hiking immunosuppression targeted for renal condition. Therapy targeting the underlying disorder is usually sufficient in patients with JDM and secondary IgA nephropathy.