cometadata/datacite-titles-descriptions-related-identifiers huggingface dataset:
DOI: 10.6084/m9.figshare.5127052.v1
title: Supplementary Material for: Neurally Adjusted Ventilatory Assist in Preterm Neonates with Acute Respiratory Failure

description: <b><i>Background:</i></b> Neurally adjusted ventilatory assist (NAVA) is a novel mode of ventilation that has been demonstrated to improve infant-ventilator interaction, compared to the conventional modes in retrospective and short-term studies. <b><i>Objectives:</i></b> To prospectively evaluate the physiologic effects of NAVA in comparison with pressure-regulated volume control (PRVC) in two nonrandomized 12-hour periods. <b><i>Methods:</i></b> We studied 14 consecutive intubated preterm neonates receiving mechanical ventilation for acute respiratory failure. Peak airway pressure (Paw<sub>peak</sub>), diaphragm electrical activity (EAdi), tidal volume (V<sub>T</sub>), mechanical (RR<sub>mec</sub>) and neural (RR<sub>neu</sub>) respiratory rates, neural apneas, and the capillary arterialized blood gases were measured. The RR<sub>mec</sub>-to-RR<sub>neu</sub> ratio (MNR) and the asynchrony index were also calculated. The amount of fentanyl administered was recorded. <b><i>Results:</i></b> Paw<sub>peak</sub> and V<sub>T</sub> were greater in PRVC (p &lt; 0.01). Blood gases and RR<sub>mec</sub> were not different between modes, while RR<sub>neu</sub> and the EAdi swings were greater in NAVA (p = 0.02 and p &lt; 0.001, respectively). MNR and the asynchrony index were remarkably lower in NAVA than in PRVC (p = 0.03 and p &lt; 0.001, respectively). 1,841 neural apneas were observed during PRVC, with none in NAVA. Less fentanyl was administered during NAVA, as opposed to PRVC (p &lt; 0.01). <b><i>Conclusions:</i></b> In acutely ill preterm neonates, NAVA can be safely and efficiently applied for 12 consecutive hours. Compared to PRVC, NAVA is well tolerated with fewer sedatives.
DOI: 10.6084/m9.figshare.5127055
title: Supplementary Material for: Dystroglycan in Human Fetal Membranes Decreases in Later Gestation and with Spontaneous Membrane Rupture

description: <b><i>Background/Aims:</i></b> The transmembrane protein dystroglycan (DG) is known to anchor the cell membrane to the extracellular matrix, and is susceptible to cleavage by matrix metalloproteinases. This study tested the hypothesis that changes in DG abundance in fetal membranes (FM) occur late in gestation, with spontaneous rupture of membranes (SROM), and during labor. <b><i>Methods:</i></b> FM were collected from a prospective cohort consisting of four groups of patients (term labor, term unlabored, preterm labor, and preterm unlabored). FM were subjected to immunohistochemical staining using antibodies specific for α- and β-DG subunits, and staining intensity was graded by a blinded pathologist. <b><i>Results:</i></b> α- and β-DG staining was significantly decreased at term and after SROM (p &lt; 0.05), but not in the presence of labor. <b><i>Conclusions:</i></b> Decreased DG intensity was seen in FM of patients at term and with SROM, but no change was observed with labor.
DOI: 10.6084/m9.figshare.5127058
title: Supplementary Material for: Menstrual Cycle Recovery in Patients with Anorexia Nervosa: The Importance of Insulin-Like Growth Factor 1

description: <b><i>Background:</i></b> Follow-up visits of patients recovering from anorexia nervosa (AN) have shown that some patients do not resume menstrual cycles despite returning to the normal weight for their age and height. <b><i>Aim:</i></b> To verify whether leptin, insulin-like growth factor 1 (IGF-1) or another hormonal marker could be a good predictor of the return of menses. <b><i>Patients and Methods:</i></b> This prospective study included female adolescents diagnosed with AN or eating disorders not otherwise specified (EDNOS) and who were being treated in an ambulatory care unit during nutritional recovery. Body mass index and leptin, luteinizing hormone, estradiol and IGF-1 levels of these patients were evaluated. Blood samples were collected in the 1st (T1), 5th (T2), 10th (T3), 15th (T4) and 20th (T5) weeks of treatment. The hormone levels during nutritional recovery and at the time of the resumption of menses were analyzed. <b><i>Results:</i></b> The hormonal profiles improved after nutritional recovery, with IGF-1 correlating the most with the resumption of menses and nutritional recovery (p = 0.0001). At the resumption of menstruation, the patients showed IGF-1 levels &gt;342.8 ng/ml. <b><i>Conclusion:</i></b> IGF-1 was the best predictor of the return of menses in female adolescents with AN or EDNOS.
DOI: 10.6084/m9.figshare.5127061.v1
title: Supplementary Material for: Heparin Use in Hemodialysis Patients following Gastrointestinal Bleeding

description: <b><i>Background:</i></b> Heparin is commonly given during hemodialysis (HD). Patients undergoing HD have a high rate of gastrointestinal bleeding (GIB). It is unclear whether or when it is safe to give heparin after acute GIB. We describe the patterns and safety of heparin use with outpatient HD following an acute GIB. <b><i>Methods:</i></b> We identified patients aged ≥67 who, from 2004-2008, experienced GIB requiring hospitalization within 2 days of receiving maintenance HD with heparin. We used Cox regression to estimate the risk of recurrent GIB and death associated with receiving heparin the day they resumed outpatient HD post-GIB. <b><i>Results:</i></b> Of the 1,342 patients who had GIB, 1,158 (86%) received heparin at a median dose of 4,000 units with their first outpatient HD session after discharge from GIB. On average, their post-GIB doses were slightly lower than their pre-GIB doses (mean change: -214 ± 3,266 units, p &lt; 0.02). However, only 27% of patients had a decrease in their dose, while 21% had their dose increased. We did not find an increased risk of death or recurrent GIB associated with using heparin post-GIB (HR; 95% confidence interval (CI), for death: 1.01; 0.69-1.48; for recurrent GIB: 0.78; 0.39-1.57). <b><i>Conclusions:</i></b> The vast majority of these high-risk patients received heparin on the very first day they resumed outpatient HD post-GIB, and the majority at unchanged doses to those received pre-GIB. Even if the practice was not associated with increased risks of death or re-bleeding, it highlights an area for possible system-based improvement to the care for patients on HD.
DOI: 10.6084/m9.figshare.5127037
title: Supplementary Material for: Consensus Development from the 5th Asia-Pacific Primary Liver Cancer Expert Meeting (APPLE 2014)

description: A key mission of the Asia-Pacific Primary Liver Cancer Expert (APPLE) Association is to ensure a coherent view for management of hepatocellular carcinoma (HCC) and to advance new treatment for this difficult disease. At the 5th APPLE meeting, held in July 2014 in Taipei, Taiwan, an APPLE consensus development program was established to facilitate discussion among experts in the Asia-Pacific region on pertinent issues for HCC management, including (1) surgery for intermediate/advanced-stage disease, (2) prevention of HCC recurrence after curative treatment, (3) optimizing imaging diagnosis, (4) radiotherapy: current practice and future clinical trials, and (5) the role of cytotoxic chemotherapy. A pre-congress questionnaire was undertaken with the consensus development committee members to help understand the current practice patterns for HCC in the Asia-Pacific region and to identify issues relating to optimal patient care and further clinical trials for which consensus needs to be developed. In this report, the results of the questionnaire are presented, and the pertinent issues identified by each consensus group for further discussion and consensus development are summarized.
DOI: 10.6084/m9.figshare.5127043
title: Supplementary Material for: Use of Transarterial Chemoembolization (TACE) and Sorafenib in Patients with Unresectable Hepatocellular Carcinoma: US Regional Analysis of the GIDEON Registry

description: <b><i>Background: </i></b>Global Investigation of Therapeutic Decisions in Hepatocellular Carcinoma and of Treatment with Sorafenib (GIDEON) is a worldwide, prospective, non-interventional study to evaluate the safety of sorafenib in a variety of patient subsets. <b><i>Methods: </i></b>Eligible patients had unresectable hepatocellular carcinoma for whom the decision had been made to treat with sorafenib. Treatment strategies were instituted at the physician's discretion. Patient and disease characteristics, treatment practices, incidences of adverse events (AEs), and overall survival were collected. <b><i>Results: </i></b>In the United States, 563 patients were evaluable for safety. Subgroup analysis was performed for patients who underwent transarterial chemoembolization (TACE) prior to the initiation of sorafenib (group A, n=158), after the initiation of sorafenib only (group B, n=29), both (group C, n=38), or did not undergo TACE (n=318). Patient demographics were similar across the groups. In group A, 29% had Child-Pugh score B or C at diagnosis, and 19% had Barcelona Clinic Liver Cancer tumor stage C or D. In group B, 48% had Child-Pugh score B or C at study entry, and 31% had BCLC stage C or D. The majority of patients in all groups initially received full-dose sorafenib. Incidences of grade ≥3 drug-related AEs were 30%, 17%, and 16% in groups A, B, and C, respectively, and 22% in patients who did not undergo TACE. No new safety signals emerged. <b><i>Conclusions: </i></b>The results from GIDEON reaffirm that sorafenib can be safely used in the context of TACE.
DOI: 10.6084/m9.figshare.5127046
title: Supplementary Material for: Time from Nephrectomy as a Prognostic Factor in Metastatic Renal Cell Carcinoma Patients Receiving Targeted Therapies: Overall Results from a Large Cohort of Patients

description: <b><i>Objective:</i></b> To investigate whether time from nephrectomy (Nx) to the diagnosis of metastatic disease may be an independent prognostic factor in metastatic renal cell carcinoma (mRCC) patients treated with targeted therapies (TTs). <b><i>Subjects and Methods:</i></b> All patients who underwent Nx and at least 1 TT were considered. The patients were divided into two groups based on time from Nx [&gt;1 year (Nx &gt;1) and &lt;1 year (Nx &lt;1)] and a third group for cytoreductive Nx (cNx). Median overall survival (OS) represented the primary outcome. <b><i>Results:</i></b> A total of 297 patients met the inclusion criteria. The time from Nx was &gt;1 year in 47%, &lt;1 year in 26% and concomitant with the diagnosis of metastatic disease in 27% of the cases (i.e. cNx). The median OS was 40.6 months (95% CI 30.5-50.7) for the Nx &gt;1 group, 24.3 months (95% CI 17.7-31) for the Nx &lt;1 group and 16.2 months (95% CI 11.2-21.3) for the cNx group (p &lt; 0.05 for all comparisons). On multivariate analysis, time from Nx resulted to be an independent prognostic factor (Nx &lt;1 vs. cNx: HR = 0.62, 95% CI 0.42-0.90, p = 0.13; Nx &gt;1 vs. cNx: HR = 0.43, 95% CI 0.31-0.61, p &lt; 0.001). <b><i>Conclusion:</i></b> We report that time from Nx is an independent prognostic factor for OS in patients affected by mRCC treated with TTs. © 2014 S. Karger AG, Basel
DOI: 10.6084/m9.figshare.5127058.v1
title: Supplementary Material for: Menstrual Cycle Recovery in Patients with Anorexia Nervosa: The Importance of Insulin-Like Growth Factor 1

description: <b><i>Background:</i></b> Follow-up visits of patients recovering from anorexia nervosa (AN) have shown that some patients do not resume menstrual cycles despite returning to the normal weight for their age and height. <b><i>Aim:</i></b> To verify whether leptin, insulin-like growth factor 1 (IGF-1) or another hormonal marker could be a good predictor of the return of menses. <b><i>Patients and Methods:</i></b> This prospective study included female adolescents diagnosed with AN or eating disorders not otherwise specified (EDNOS) and who were being treated in an ambulatory care unit during nutritional recovery. Body mass index and leptin, luteinizing hormone, estradiol and IGF-1 levels of these patients were evaluated. Blood samples were collected in the 1st (T1), 5th (T2), 10th (T3), 15th (T4) and 20th (T5) weeks of treatment. The hormone levels during nutritional recovery and at the time of the resumption of menses were analyzed. <b><i>Results:</i></b> The hormonal profiles improved after nutritional recovery, with IGF-1 correlating the most with the resumption of menses and nutritional recovery (p = 0.0001). At the resumption of menstruation, the patients showed IGF-1 levels &gt;342.8 ng/ml. <b><i>Conclusion:</i></b> IGF-1 was the best predictor of the return of menses in female adolescents with AN or EDNOS.
DOI: 10.6084/m9.figshare.5127046.v1
title: Supplementary Material for: Time from Nephrectomy as a Prognostic Factor in Metastatic Renal Cell Carcinoma Patients Receiving Targeted Therapies: Overall Results from a Large Cohort of Patients

description: <b><i>Objective:</i></b> To investigate whether time from nephrectomy (Nx) to the diagnosis of metastatic disease may be an independent prognostic factor in metastatic renal cell carcinoma (mRCC) patients treated with targeted therapies (TTs). <b><i>Subjects and Methods:</i></b> All patients who underwent Nx and at least 1 TT were considered. The patients were divided into two groups based on time from Nx [&gt;1 year (Nx &gt;1) and &lt;1 year (Nx &lt;1)] and a third group for cytoreductive Nx (cNx). Median overall survival (OS) represented the primary outcome. <b><i>Results:</i></b> A total of 297 patients met the inclusion criteria. The time from Nx was &gt;1 year in 47%, &lt;1 year in 26% and concomitant with the diagnosis of metastatic disease in 27% of the cases (i.e. cNx). The median OS was 40.6 months (95% CI 30.5-50.7) for the Nx &gt;1 group, 24.3 months (95% CI 17.7-31) for the Nx &lt;1 group and 16.2 months (95% CI 11.2-21.3) for the cNx group (p &lt; 0.05 for all comparisons). On multivariate analysis, time from Nx resulted to be an independent prognostic factor (Nx &lt;1 vs. cNx: HR = 0.62, 95% CI 0.42-0.90, p = 0.13; Nx &gt;1 vs. cNx: HR = 0.43, 95% CI 0.31-0.61, p &lt; 0.001). <b><i>Conclusion:</i></b> We report that time from Nx is an independent prognostic factor for OS in patients affected by mRCC treated with TTs. © 2014 S. Karger AG, Basel
DOI: 10.6084/m9.figshare.5127034
title: Supplementary Material for: Copy Number Variants in Short Children Born Small for Gestational Age

description: <b><i>Background/Aims:</i></b> In addition to genome-wide association studies (GWAS), height-associated genes may be uncovered by studying individuals with extreme short or tall stature. <b><i>Methods:</i></b> Genome-wide analysis for copy number variants (CNVs), using single nucleotide polymorphism (SNP) arrays, was performed in 49 index cases born small for gestational age with persistent short stature. Segregation analysis was performed, and genes in CNVs were compared with information from GWAS, gene expression in rodents' growth plates, and published information. <b><i>Results:</i></b> CNVs were detected in 13 cases. In 5 children a known cause of short stature was found: UPD7, UPD14, a duplication of the <i>SHOX</i> enhancer region, an<i> IGF1R</i> deletion, and a 22q11.21 deletion. In the remaining 8 cases, potential pathogenic CNVs were detected, either de novo (n = 1), segregating (n = 2), or not segregating with short stature (n = 5). Bioinformatic analysis of the de novo and segregating CNVs suggested that <i>HOXD4</i>, <i>AGPS</i>, <i>PDE11A</i>, <i>OSBPL6</i>, <i>PRKRA </i>and <i>PLEKHA3</i>, and possibly <i>DGKB</i> and <i>TNFRSF11B</i> are potential candidate genes<i>.</i> A <i>SERPINA7</i> or <i>NRK</i> defect may be associated with an X-linked form of short stature. <b><i>Conclusion:</i></b> SNP arrays detected 5 known causes of short stature with prenatal onset and suggested several potential candidate genes.
DOI: 10.6084/m9.figshare.5127040
title: Supplementary Material for: Phase I Trial of Sorafenib Following Liver Transplantation in Patients with High-Risk Hepatocellular Carcinoma

description: Liver transplantation offers excellent long-term survival for hepatocellular carcinoma (HCC) patients who fall within established criteria. For those outside such criteria, or with high-risk pathologic features in the explant, HCC recurrence rates are higher. We conducted a multicenter phase I trial of sorafenib in liver transplantation patients with high-risk HCC. Subjects had HCC outside the Milan criteria (pre- or post-transplant), poorly differentiated tumors, or vascular invasion. We used a standard 3+3 phase I design with a planned duration of treatment of 24 weeks. Correlative studies included the number of circulating endothelial cells (CECs), plasma biomarkers, and tumor expression of p-Erk, p-Akt, and c-Met in tissue micro-arrays. We enrolled 14 patients with a median age of 63 years. Of these, 93% were men and 71% had underlying hepatitis C virus (HCV) and 21% had HBV. The maximum tolerated dose of sorafenib was 200 mg BID. Grade 3-4 toxicities seen in &gt;10% of subjects included leukopenia (21%), elevated gamma-glutamyl transferase (21%), hypertension (14%), hand-foot syndrome (14%) and diarrhea (14%). Over a median follow-up of 953 days, one patient died and four recurred. The mean CEC number at baseline was 21 cells/4 ml for those who recurred, and 80 cells/4 ml for those who did not (p=0.10). Mean soluble vascular endothelial growth factor receptor-2 levels decreased after 1 month on sorafenib (p=0.09), but did not correlate with recurrence. There was a trend for tumor c-Met expression to correlate with increased risk of recurrence. Post-transplant sorafenib was found to be feasible and tolerable at 200 mg PO BID. The effect of post-transplant sorafenib on recurrence-free survival is potentially promising but needs further validation in a larger study.
DOI: 10.6084/m9.figshare.5127028
title: Supplementary Material for: Frontal Corpus Callosum Alterations in Progressive Supranuclear Palsy but Not in Parkinson's Disease

description: <b><i>Background:</i></b> Frontal lobe involvement is considered a clinical and magnetic resonance imaging (MRI) feature in later stages of progressive supranuclear palsy (PSP). <b><i>Objective:</i></b> Diffusion tensor imaging (DTI) was used to investigate the integrity of frontal pathways in PSP and Parkinson's disease (PD) patients. <b><i>Methods:</i></b> DTI and 3-D MRI were performed in 15 PSP patients (parkinsonism subtype: n = 8; Richardson subtype: n = 7), 15 PD patients, and 18 matched controls. DTI analysis was performed in order to identify differences along frontal white matter structures including the corpus callosum (CC) and was complemented by atlas-based volumetry and planimetry. <b><i>Results:</i></b> Significantly reduced regional fractional anisotropy was observed for PSP patients versus controls and PSP versus PD patients, respectively, in frontal areas including the area II of the CC and bilaterally in the callosal radiation. The DTI findings correlated with frontal lobe volumes. These differences were not observed between PD patients and controls. <b><i>Conclusion:</i></b> DTI identified a PSP-associated microstructural alteration pattern in the frontal lobes and in the CC area II including the corresponding bilateral callosal radiation tracts that could not be identified in both control samples, supporting the prominent PSP-associated frontal involvement as a potential neuroimaging marker.
DOI: 10.6084/m9.figshare.5127031
title: Supplementary Material for: Clinical Efficacy and Tolerability of a Novel Selective Corticosteroid in Atopic Dermatitis - Two Randomised Controlled Trials

description: Topical corticosteroids remain the first-line therapy for atopic dermatitis (AD). Hence, we investigated the efficacy and safety profile of a novel selective corticosteroid, GW870086. We performed 2 randomised, double-blind, controlled studies with 25 AD patients and 20 healthy subjects. The changes in the Three-Item Severity (TIS) score and the skin thickness were the primary end points, respectively. The adjusted TIS score (day 22) shows that the novel corticosteroid resulted in a non-significant, but dose-dependent reduction compared to placebo (GW870086 0.2% vs. placebo = -0.38, GW870086 2% vs. placebo = -0.89). Significant skin thinning was observed in the second study on days 14 and 21 when patients were treated with the comparator but not with the novel corticosteroid compared to placebo. The clinical efficacy of the new selective corticosteroid was not superior to placebo, although a dose-dependent improvement upon treatment was noticed without the onset of skin thinning.
DOI: 10.6084/m9.figshare.5127040.v1
title: Supplementary Material for: Phase I Trial of Sorafenib Following Liver Transplantation in Patients with High-Risk Hepatocellular Carcinoma

description: Liver transplantation offers excellent long-term survival for hepatocellular carcinoma (HCC) patients who fall within established criteria. For those outside such criteria, or with high-risk pathologic features in the explant, HCC recurrence rates are higher. We conducted a multicenter phase I trial of sorafenib in liver transplantation patients with high-risk HCC. Subjects had HCC outside the Milan criteria (pre- or post-transplant), poorly differentiated tumors, or vascular invasion. We used a standard 3+3 phase I design with a planned duration of treatment of 24 weeks. Correlative studies included the number of circulating endothelial cells (CECs), plasma biomarkers, and tumor expression of p-Erk, p-Akt, and c-Met in tissue micro-arrays. We enrolled 14 patients with a median age of 63 years. Of these, 93% were men and 71% had underlying hepatitis C virus (HCV) and 21% had HBV. The maximum tolerated dose of sorafenib was 200 mg BID. Grade 3-4 toxicities seen in &gt;10% of subjects included leukopenia (21%), elevated gamma-glutamyl transferase (21%), hypertension (14%), hand-foot syndrome (14%) and diarrhea (14%). Over a median follow-up of 953 days, one patient died and four recurred. The mean CEC number at baseline was 21 cells/4 ml for those who recurred, and 80 cells/4 ml for those who did not (p=0.10). Mean soluble vascular endothelial growth factor receptor-2 levels decreased after 1 month on sorafenib (p=0.09), but did not correlate with recurrence. There was a trend for tumor c-Met expression to correlate with increased risk of recurrence. Post-transplant sorafenib was found to be feasible and tolerable at 200 mg PO BID. The effect of post-transplant sorafenib on recurrence-free survival is potentially promising but needs further validation in a larger study.
DOI: 10.6084/m9.figshare.5127025
title: Supplementary Material for: Addition of Local Hepatic Therapy to Sorafenib in Patients with Advanced Hepatocellular Carcinoma (Stage BCLC C)

description: <b><i>Background/Aims:</i></b> For most patients with hepatocellular carcinoma (HCC), diagnosis is invariably done only in the advanced stages of the disease. For advanced, non-metastatic stage, standard therapy is transarterial chemoembolization (TACE). For metastatic disease, the recommended therapy is systemic treatment with sorafenib. In this study, we evaluated the benefit of an additional local hepatic treatment for patients with advanced metastatic disease. <b><i>Methods:</i></b> In a retrospective study, we assessed the overall survival (OS), time to progression (TTP), and disease control rate (DCR) in 37 patients with metastasized HCC treated with sorafenib. Sixteen patients received additional local therapy, while 21 patients received only sorafenib. <b><i>Results:</i></b> Median OS of patients with combined therapy was significantly higher with 25 months (95% CI: 13.7-36.3 months) as compared to 11 months (95% CI: 6.2-15.8 months) in patients treated with sorafenib alone. TTP was 7 months (95% CI: 5.3-8.7 months) compared to 5 months (95% CI: 3-7 months) and DCR was 87 versus 72% after 3 months and 31 versus 22% after 9 months. <b><i>Conclusion:</i></b> These data suggest that control of the liver tumor burden by local therapy in combination with sorafenib might prove beneficial for metastasized HCC. Randomised studies are needed to confirm this exploratory finding. © 2014 S. Karger AG, Basel