Molecular Absorbent Recirculating System therapy (MARS®) in pediatric acute liver failure: a single center experience

Background

Supportive care as a bridge to transplant or recovery remains challenging in children suffering from acute liver failure (ALF). We report our experience in children using the Molecular Absorbent Recirculating System (MARS®).

Methods

Retrospective data from children receiving therapy using MARS® from October 2009 to October 2012 were included in this single-center retrospective study. Patient characteristics, clinical presentation and complications of ALF, clinical and biological data before and after each MARS® session, technical modalities and adverse events were recorded.

Results

A total of six children underwent 17 MARS® sessions during the study period. Two adolescents were treated with the adult filter MARSFLUX® and four infants were treated with the MiniMARS® filter. The mean PEdiatric Logistic Dysfunction (PELOD) score at admission was 19 (range 11–33). All patients were mechanically ventilated, and four had acute kidney injury. The neurological course improved in one case, judged as stable in two cases and worsened in one case; data were unavailable in two cases. Mean serum ammonia levels decreased significantly following treatment with MARS® from an initial 89?±?29 to 58?±?35 mcmol/L (p?=?0.02). No other significant biological improvement was observed. Hemodynamic status improved/remained unchanged in the adolescent group, but in the infants four of the seven sessions were poorly tolerated and two sessions were aborted. Three patients died, two were successfully transplanted and one recovered without transplantation.

Conclusion

In our experience, treatment with MARS® is associated with encouraging results in adolescents, but it needs modification for very sick infants to improve tolerance.     

Islet of Langerhans isolation from pediatric and juvenile donor pancreases

Islet grafts isolated from young donors allow superior functional outcomes but are often associated with poor islet isolation yields. The objective of this study was to comparatively analyze the outcomes of islet isolation between young and older donors. We retrospectively analyzed 564 pancreas isolations performed at our institution. Isolation outcomes were compared between donors aged ≤20 years (n = 42, YD) and >20 years (n = 522, OD). Isolation procedure was identical in both groups. Prepurification percentage of embedded islets was higher in YD (44.3 ± 22.7% vs. 24.9 ± 20.9%, P < 0.001). This led to a lower recovery rate in YD (48% vs. 76%, P = 0.002) and hence lower postpurification IEQ/g pancreas in YD (2 412 ± 1 789 IEQ/g vs. 3 194 ± 1 892 IEQ/g, P = 0.01). Final yield was 180 982 ± 128 073 IEQ in YD and 244 167 ± 134 137 IEQ in OD, (P = 0.006). In vitro function was markedly, albeit nonsignificantly, higher in YD (SI: 4.5 ± 5.1 vs. 3.0 ± 5.7, P = 0.350). Proportion of transplanted preparations was similar in both groups, 38% (16/42) in YD vs. 43% (224/522) in OD, P = 0.628. In spite of isolation and purification difficulties, pancreases from young donors allowed similar islet transplantation rates as older donors. Efforts should be directed at improving islet extraction in these donors to realize their full potential for islet transplantation.     

A Centre for the Diagnosis and Treatment of Tuberculosis (CDT) in a resource-limited setting: a dragnet for patients with heart disease?

Background

Cardiovascular disease is a growing public health problem in sub-Saharan Africa. Cough and dyspnea are symptoms of both lung diseases and heart failure. This study aimed at determining the contribution of cardiac diseases versus pulmonary diseases in the etiological profile of patients presenting with cough and dyspnea in a Center for the Diagnosis and Treatment of Tuberculosis (CDT), in a semi-rural area in Cameroon.

Methods

This is a cross-sectional analysis of data from patients aged 18 years or more who consulted for cough and or dyspnea between December 2009 and December 2010 at the CDT of Lafe-Baleng, Bafoussam, Cameroon.

Results

A total of 1196 patients were received for various complaints during the study period; 348 (29.1%) of them presented with cough and or dyspnea, and were included in the study. 186 patients (53.4%; 95% CI: 48.2-58.6) had a pure cardiac disease, while 122 patients (35.1%; 95% CI: 30.2-40.2) had a pulmonary disease. The prevalence of hypertension was 50.9%, and hypertensive heart disease was the most frequent cardiac disease with a prevalence rate of 37.6%. Heart failure was diagnosed in 222 patients, representing 63.8% (95% CI: 58.9-68.9) of patients with cough and or dyspnea, and 18.6% (95% CI: 16.5-21.0) of all the patients received at the CDT of Lafe-Baleng during the study period. Compared to patients with a pulmonary disease, patients with cardiac disease were older (p < 0.001) and more likely to present with dyspnea (p < 0.001) and to have hypertension (p < 0.001).

Conclusion

We found a high prevalence of heart failure in this Centre for the Diagnosis and Treatment of Tuberculosis thus, a veritable dragnet for patients with heart disease. Our findings emphasize the urgent need to increase the access to cardiovascular care and to continuously raise the awareness of the communities on cardiovascular diseases in Cameroon.     

Psychometric properties of the French Female Sexual Function Index (FSFI)

Introduction

Few validated questionnaires are available in French to assess sexual function. The aim of this study was thus to validate a French version of the Female Sexual Function Index (FSFI) in a sample of French women.

Methods

In this prospective monocentric and cross-sectional study, an already existing French version of the FSFI, was back-translated and compared to the original version. It was then randomly distributed to 800 women attending Gynecology consultation at Nantes University Hospital in April 2012. Various statistical analyzes were used to test the psychometric properties of the French FSFI.

Results

512 questionnaires were completed. Mean FSFI summary score was 25.2. Intraclass correlation coefficients were superior to 0.75 and Cronbach’s coefficients superior to 0.8 similarly to the original version. Variance analysis revealed significant differences in summary score between premenopausal and postmenopausal women and according to the marital status. Convergent validity was excellent (100 %) and discriminant validity was satisfactory (89.5 %). The factorial structure corresponded to the original version with six retrieved dimensions.

Conclusions

Our study demonstrated similar or adequate psychometric properties of the French version of the FSFI compared to the original English version.     

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Whole MYBPC3 NGS sequencing as a molecular strategy to improve the efficiency of molecular diagnosis of patients with hypertrophic cardiomyopathy

Hypertrophic cardiomyopathy (HCM) is the most common heritable cardiomyopathy, historically believed to affect 1 of 500 people. MYBPC3 pathogenic variations are the most frequent cause of familial HCM and more than 90% of them introduce a premature termination codon. The current study aims to determine the prevalence of deep intronic MYBPC3 pathogenic variations that could lead to splice mutations. To improve molecular diagnosis, a next‐generation sequencing (NGS) workflow based on whole MYBPC3 sequencing of a cohort of 93 HCM patients, for whom no putatively causative point mutations were identified after NGS sequencing of a panel of 48 cardiomyopathy‐causing genes, was performed. Our approach led us to reconsider the molecular diagnosis of six patients of the cohort (6.5%). These HCM probands were carriers of either a new large MYBPC3 rearrangement or splice intronic variations (five cases). Four pathogenic intronic variations, including three novel ones, were detected. Among them, the prevalence of one of them (NM_000256.3:c.1927+ 600 C>T) was estimated at about 0.35% by the screening of 1,040 unrelated HCM individuals. This study suggests that deep MYBPC3 splice mutations account for a significant proportion of HCM cases (6.5% of this cohort). Consequently, NGS sequencing of MYBPC3 intronic sequences have to be performed systematically.