Cardiac manifestations of MIS-C: cardiac magnetic resonance and speckle-tracking data.

Background: Cardiac involvement is central in MIS-C and represents the main cause of morbidity. In this study, we aimed to assess myocardial damage in patients with MIS-C using cardiac magnetic resonance (CMR) during the acute phase, as well as left ventricular and atrial longitudinal strain on admission, at discharge, and after 3 months. Methods: We performed a single-center prospective cohort study and case-control study. Between September 2020 and February 2022, we enrolled 39 patients hospitalized for MIS-C at our center. We performed left ventricular and atrial longitudinal 2D strain analysis on admission and during follow-up; echocardiographic data were compared to a matched control population. Patients above 4 years old with increased troponin underwent CMR. Results: Of 24 patients (mean age: 8.2 ± 4.9 years) who underwent CMR, 14 (58%) presented myocardial edema and 6 (25%) late gadolinium enhancement (LGE). LGE was associated with older age (p < 0.01), increased BMI (p = 0.03), increased ferritin levels (p < 0.001), lower left ventricular (LV) ejection fraction (p < 0.001), LV longitudinal strain (p = 0.004), left atrial (LA) strain (p = 0.05), and prolonged hospital stay (p = 0.02). On admission, LV ejection fraction, LV longitudinal strain, and LA strain were impaired, but each improved gradually over time; LVEF was the fastest to recover, while global LV longitudinal strain was still impaired as compared to controls after 3 months (p = 0.01). Conclusion: Our study demonstrates that myocardial injury is present in a quarter of MIS-C patients, and impaired LA and LV myocardial deformation persist for at least several weeks after the acute phase. CMR and LV/LA strain could help us to individualize follow-up of MIS-C patients.

Pediatric en bloc kidney transplantation into pediatric recipients: the French experience.

Chronic shortage of available donor organs has led to re-evaluation of the use of en bloc kidney transplants. Although excellent results have been reported in adult patients, experience in pediatric patients remains limited because of potential early complications and poor long-term graft outcome. We report 14 pediatric en bloc renal transplantations into 14 pediatric recipients, performed between 1990 and 2007 in France. We retrospectively analyzed demographic data, postoperative complications, and graft function with a median follow-up of five yr. Donor age ranged from four to 54 months. Complications were vascular graft thrombosis in four patients, leading to graft loss in two cases, and to excellent long-term graft function in the two others. Two hemorrhagic complications resulted in death in one case and in graft loss in the other. Six acute rejection episodes occurred in four patients. Median glomerular filtration rate at three months, one, five, and 10 yr was 90.8, 106, 87.8, and 66.1 mL/1.73 m(2) /min. We believe that en bloc transplantation may be an option for children with end-stage kidney disease.

A multicenter randomized controlled trial of a 3-L/kg/min versus 2-L/kg/min high-flow nasal cannula flow rate in young infants with severe viral bronchiolitis (TRAMONTANE 2).

PURPOSE: High-flow nasal cannula (HFNC) therapy is increasingly proposed as first-line respiratory support for infants with acute viral bronchiolitis (AVB). Most teams use 2 L/kg/min, but no study compared different flow rates in this setting. We hypothesized that 3 L/kg/min would be more efficient for the initial management of these patients. METHODS: A randomized controlled trial was performed in 16 pediatric intensive care units (PICUs) to compare these two flow rates in infants up to 6 months old with moderate to severe AVB and treated with HFNC. The primary endpoint was the percentage of failure within 48 h of randomization, using prespecified criteria of worsening respiratory distress and discomfort. RESULTS: From November 2016 to March 2017, 142 infants were allocated to the 2-L/kg/min (2L) flow rate and 144 to the 3-L/kg/min (3L) flow rate. Failure rate was comparable between groups: 38.7% (2L) vs. 38.9% (3L; p = 0.98). Worsening respiratory distress was the most common cause of failure in both groups: 49% (2L) vs. 39% (3L; p = 0.45). In the 3L group, discomfort was more frequent (43% vs. 16%, p = 0.002) and PICU stays were longer (6.4 vs. 5.3 days, p = 0.048). The intubation rates [2.8% (2L) vs. 6.9% (3L), p = 0.17] and durations of invasive [0.2 (2L) vs. 0.5 (3L) days, p = 0.10] and noninvasive [1.4 (2L) vs. 1.6 (3L) days, p = 0.97] ventilation were comparable. No patient had air leak syndrome or died. CONCLUSION: In young infants with AVB supported with HFNC, 3 L/kg/min did not reduce the risk of failure compared with 2 L/kg/min. This clinical trial was recorded on the National Library of Medicine registry (NCT02824744).

Ecthyma gangrenosum, a skin manifestation of Pseudomonas aeruginosa sepsis in a previously healthy child: A case report.

RATIONALE: Ecthyma gangrenosum (Eg) is a necrotic lesion that is mostly seen in immunocompromised patients. It reflects a severe sepsis, possibly caused by Pseudomonas aeruginosa (Pa). PATIENT CONCERNS: A healthy 3-year-old girl admitted to the Pediatric Emergency Department presented a sepsis-associated purpura with neurological and respiratory distress. INTERVENTIONS: An empiric antibiotherapy (anti-meningococcal) was prescribed. DIAGNOSES: Forty-eight hours after admission, blood and wound cultures were positive for Pa. As a result, the decision was made to change the antibiotic therapy.Unfortunately, on day 3, the patient died. Exhaustive immunologic tests are presently being carried out. OUTCOMES: Eg caused by Pa is uncommon in healthy children, and purpura sepsis is usually caused by Neisseria meningitides infection. LESSONS: Eg should be recognized rapidly so that the appropriate treatment can be prescribed as quickly as possible.

High flow nasal cannula (HFNC) versus nasal continuous positive airway pressure (nCPAP) for the initial respiratory management of acute viral bronchiolitis in young infants: a multicenter randomized controlled trial (TRAMONTANE study).

PURPOSE: Nasal continuous positive airway pressure (nCPAP) is currently the gold standard for respiratory support for moderate to severe acute viral bronchiolitis (AVB). Although oxygen delivery via high flow nasal cannula (HFNC) is increasingly used, evidence of its efficacy and safety is lacking in infants. METHODS: A randomized controlled trial was performed in five pediatric intensive care units (PICUs) to compare 7 cmH2O nCPAP with 2 L/kg/min oxygen therapy administered with HFNC in infants up to 6 months old with moderate to severe AVB. The primary endpoint was the percentage of failure within 24 h of randomization using prespecified criteria. To satisfy noninferiority, the failure rate of HFNC had to lie within 15% of the failure rate of nCPAP. Secondary outcomes included success rate after crossover, intubation rate, length of stay, and serious adverse events. RESULTS: From November 2014 to March 2015, 142 infants were included and equally distributed into groups. The risk difference of -19% (95% CI -35 to -3%) did not allow the conclusion of HFNC noninferiority (p = 0.707). Superiority analysis suggested a relative risk of success 1.63 (95% CI 1.02-2.63) higher with nCPAP. The success rate with the alternative respiratory support, intubation rate, durations of noninvasive and invasive ventilation, skin lesions, and length of PICU stay were comparable between groups. No patient had air leak syndrome or died. CONCLUSION: In young infants with moderate to severe AVB, initial management with HFNC did not have a failure rate similar to that of nCPAP. This clinical trial was recorded in the National Library of Medicine registry (NCT 02457013).

Transcutaneous bilirubinometry is not influenced by term or skin color in neonates.

BACKGROUND: The utility of transcutaneous bilirubin measurements (TcB) in screening for hyperbilirubinemia in preterm infants (<34 weeks) and in non-white infants remains a matter of debate. AIM: To evaluate accuracy of TcB in preterm and term infants of different ethnic backgrounds, using a second generation bilirubinometer. STUDY DESIGN: The Draeger JM-103® device was used to measure TcB. Eighty five measurements of TcB and total serum bilirubin (TSB) were retrospectively compared. Neonates were stratified into groups according to gestational age: <34 weeks (group 1, n=44) and >34 weeks (group 2, n=41), and according to ethnic origin: Caucasians (group A, n=49) and non-Caucasians (group B, n=36). Statistical analysis, using Pearson's correlation coefficient (r) and Bland-Altman analysis were performed to evaluate correlation and agreement between TSB and TcB values. Multiple linear regression was used to control for confounders for TSB values. RESULTS: Correlation between TSB and TcB was high. Pearson's correlation coefficients were over 0.9 in all groups (0.910, 0.908, 0.916 and 0.934, p<0.0001 in groups 1, 2, A, and B respectively). Bland-Altman plots showed acceptable and close limits of agreements (56.8/-57.7, 54.2/-67.2, 57.7/-55.8, and 51.3/-69.9µmol/L in groups 1, 2, A and B respectively) with a trend for TcB to overestimate TSB in groups 2 and B. Birth term and skin color were not identified as confounding factors for predicting TSB in multiple linear regression. CONCLUSIONS: TcB measurements using the Draeger JM-103® device correlate significantly with TSB, regardless of term and skin color. Transcutaneous bilirubinometry seems to be a safe and cost-effective screening method for severe hyperbilirubinemia in newborns of different terms and ethnic origins.

Acute liver failure in children.

The management of children with acute liver failure mandates a multidisciplinary approach and intense monitoring. In recent years, considerable progress has been made in developing specific and supportive medical measures, but clinical studies have mainly concerned adult patients. There are no specific medical therapies, except for a few metabolic diseases presenting with acute liver failure. Liver transplantation still remains the only definitive therapy in most instances. Recent clinical studies suggest that hepatocyte transplantation may be useful for bridging patients to liver transplantation, for providing metabolic support during liver failure and for replacing liver transplantation in certain metabolic liver diseases.

Long-term results of rhGH treatment in children with renal failure: experience of the French Society of Pediatric Nephrology.

Few publications have described the long-term effects of recombinant human growth hormone (rhGH) in uremic patients. This study reports the results of rhGH therapy at the end of treatment and at adult age in 178 French patients. At enrollment, 63 patients were under conservative treatment (CT), 40 under hemodialysis (HD), and 75 had a functioning renal transplant (RT). Under rhGH treatment, height velocities (HV) significantly increased in all patients, but the effect was significantly better in the CT group. The HV gain (HV under rhGH-HV before treatment) was similar in all three groups. Increases in HV allowed height standard deviation scores (SDS) catch up, and this effect persisted over a 5-year period. SDS height at the completion of treatment was significantly related to group (best in CT) and response to treatment during the first year. Data on adult height was available for 102 patients. Mean adult height was 162.2 cm in men and 152.9 cm in women, and 46% were > -2 SDS for height. Adult height SDS was correlated with height SDS and spontaneous HV before treatment and effect of treatment. Analysis of adult height in the 49 patients who entered the protocol with a height SDS between -2 and -3 (the current recommendation for rhGH use) revealed that 65% had an adult-height SDS >-2. These adult heights were significantly better if compared with historical cohorts of patients not treated by rhGH; rhGH significantly improves the adult-height prognosis of uremic patients suffering from growth retardation. Early rhGH administration during CT gives better height SDS at both the end of rhGH therapy and in adulthood.

A novel Wilms' tumor 1 gene mutation in a child with severe renal dysfunction and persistent renal blastema.

The Wilms' tumor suppressor gene WT1 is an important regulator of development. Mutations in this gene have been associated with Wilms' tumor, Frasier syndrome, and Denys-Drash syndrome, as well as isolated glomerular disease. Here we report the case of a 4-month-old girl, who presented with end-stage renal disease, thrombopenia, anemia, and cardiac hypertrophy accompanied by severe hypertension. Histological analysis of kidney biopsies revealed a massive and diffuse nephroblastomatosis with a dramatic reduction in the number of glomeruli. Although no normal cortical nephrons could be detected, medullary organization was nearly normal. Sequence analysis demonstrated a heterozygous nonsense mutation in exon 9 of WT1, which leads to a truncation of the WT1 protein at the beginning of zinc finger 3. Given the requirement of WT1 for normal development of the kidney and heart, these data raise the hypothesis that the mutation identified was responsible for the severe phenotype observed in our patient.

Rituximab treatment for severe steroid- or cyclosporine-dependent nephrotic syndrome: a multicentric series of 22 cases.

Several case reports suggest that rituximab (RTX) could be effective in steroid-dependent nephrotic syndrome, but RTX efficacy has not yet been studied in a series of patients. Safety and efficacy of RTX were assessed in a multicenter series of 22 patients aged 6.3-22 years with severe steroid-dependent nephrotic syndrome or steroid-resistant but cyclosporin-sensitive idiopathic nephrotic syndrome. Patients were treated with two to four infusions of RTX. Seven patients were nephrotic at the time of RTX treatment. Peripheral B cells were depleted in all subjects. Remission was induced in three of the seven proteinuric patients. One or more immunosuppressive (IS) treatments could be withdrawn in 19 patients (85%), with no relapse of proteinuria and without increasing other IS drugs. RTX was effective in all patients when administered during a proteinuria-free period in association with other IS agents. When relapses occurred, they were always associated with an increase in CD19 cell count. Adverse effects were observed in 45% of cases, but most of them were mild and transient. This study suggests that RTX could be an effective treatment for severe steroid-dependent nephrotic syndrome.