Tinzaparin Versus Dalteparin for Peri-Procedure Thromboembolic Prophylaxis in Patients with Dialysis Dependent Renal Disease.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 3136-3136
Author(s):  
Marc Rodger ◽  
Tim Ramsay ◽  
Martin Mackinnon ◽  
Margit Westphal ◽  
Melissa Spero ◽  
...  
Keyword(s):  
Research Funding ◽  
Primary Outcome ◽  
Study Drug ◽  
Dalteparin Sodium ◽  
Invasive Procedures ◽  
Bridging Therapy ◽  
Prophylactic Dose ◽  
Significant Difference ◽  
The Mean ◽  
Therapeutic Doses

Abstract Abstract 3136 Poster Board III-73 Background Low-molecular-weight heparins (LMWHs) can be administered in fixed subcutaneous doses permitting predictable parenteral anticoagulation in an out-of-hospital setting. The renal clearance of LMWHs leads to uncertainty about the safety of use of LMWHs in patients on hemodialysis given the potential for bioaccumulation of anticoagulant effect which may lead to bleeding. Methods We conducted a randomized open label trial to investigate the feasibility and safety of a perioperative anticoagulation protocol in hemodialysis patients comparing two LMWHs preparations as outpatient bridging therapy when warfarin is interrupted for invasive procedures. The primary objective of the study was to compare if tinzaparin and dalteparin differentially bioaccumulate in hemodialysis (HD) patients after three therapeutic doses. Warfarin therapy was discontinued in these patients 5 to 6 days prior to surgery. Patients were randomized to either 3 doses of tinzaparin (175 IU/kg/day) or dalteparin (200 IU/kg/day) with two dialyses in the interval between the first dose of study drug and inavsive procedure. Primary outcome was pre-dialysis anti-Xa levels 20-24hrs post third therapeutic LMWH dose to determine if these LMWHs accumulated in HD patients, and if they differentially accumulated. After procedure, patients received a daily prophylactic dose of tinzaparin (4500 IU) and dalteparin (5000 IU) for 3 to 4 days along with dialysis. Post- procedure pre- and post- dialysis anti-Xa levels were also monitored. Results Of 29 eligible and consenting patients, 17 patients (58.6%) received tinzaparin sodium and 12 patients (41.4%) received dalteparin sodium. Two patients were withdrawn prior to invasive procedure and the invasive procedures were canceled (1 for important bio-accumulation in the dalteparin arm; 1 for major bleed in the tinzaparin arm). Primary outcome anti-Xa samples were not analyzable in 2 patients (1 contaminated with heparin; 1 drawn too early). There was important and significant bioaccumulation in both study drug arms. The mean primary outcome pre-dialysis anti-Xa level 20-24hrs post third tinzaparin dose was 0.40 IU/ml (SD= 0.21). The mean primary outcome pre-dialysis anti-Xa level 20-24hrs post third dalteparin dose was 0.57 IU/ml (SD= 0.43). There was no statistically significant difference between the two study drug groups (student's t-test (p value = 0.33). Postoperatively 16/21 (76%) of patients tested had undetectable pre-dialysis anti-Xa levels 20-24hrs post prophylactic dose, with the remaining 5 patients having a mean anti-Xa level of 0.23 IU/ml (SD = 0.25). During subsequent follow-up, 2 patients experienced serious adverse events (one non-STEMI in patient who died of sepsis (tinzaparin group) and one upper extremity DVT (dalteparin group)). Conclusions Tinzaparin and Dalteparin significantly accumulate at therapeutic doses in HD patients with no statistically significant difference detected in accumulation between the drugs. Neither drug importantly accumulates at prophylactic doses in HD patients. “Bridging therapy” with LMWHs at therapeutic doses in patients on hemodialysis who require temporary interruption of warfarin for invasive procedures is risky and of uncertain risk benefit. Disclosures Rodger: Bayer: Research Funding; Leo Pharma: Research Funding; Pfizer: Research Funding; Boehringer Ingelheim: Membership on an entity's Board of Directors or advisory committees; Biomerieux: Research Funding; GTC Therapeutics: Research Funding.

scholarly journals Comparison between Prophylactic versus Therapeutic Doses of Low-Molecular-Weight Heparin in Severely Ill Coronavirus Disease 2019 Patients in Relation to Disease Progression and Outcome

2020 ◽  
Vol 3 (4) ◽  
pp. 162-169
Author(s):  
Ahmed Elmelhat ◽  
Essam Elbourai ◽  
Hany Dewedar ◽  
Taghrid Elgergawi ◽  
Maryam Alkhanbouli ◽  
...  
Keyword(s):  
Therapeutic Dose ◽  
Inflammatory Markers ◽  
Study Groups ◽  
Prophylactic Dose ◽  
Enoxaparin Group ◽  
Significant Difference ◽  
The Mean ◽  
Group 2 ◽  
Therapeutic Doses ◽  
Group 1

<b><i>Introduction:</i></b> The predominant coagulation abnormalities in patients with coronavirus disease 2019 (COVID-19) suggest a hypercoagulable state and are consistent with uncontrolled clinical observations of an increased risk of venous thromboembolism. <b><i>Aim and Objectives:</i></b> To compare the effect of prophylactic versus therapeutic doses of enoxaparin in the treatment of severe cases of COVID-19 infection. <b><i>Materials and Methods:</i></b> This was a retrospective observational study conducted at Latifa hospital, Dubai. Fifty-nine patients enrolled from March to June 2020 and divided into 2 groups: patients who received the prophylactic dose of enoxaparin (group 1) and patients who received the therapeutic dose of enoxaparin (group 2). <b><i>Results:</i></b> The mean age of all cases was 47.2 ± 10.4 years, while the mean weight was 76.4 ± 13.4 kg. Males represented 79.7% of cases. Blood group “O” was the most frequent blood group (40.9%). None of the cases were smokers or using alcohol. Bronchial asthma, lung diseases, diabetes mellitus, hypertension, CKD, cardiac disease, thyroid disease, and immunodeficiency were present in 1.7, 1.7, 39, 27.1, 5.1, 1.7, 5.1, and 1.7% respectively. There was no significant difference between both study groups regarding personal and medical characteristics, except for hypertension where 35.9% of group 2 (therapeutic) cases were hypertensive compared to 10% of group 1 cases (prophylactic). There was a significant difference between both study groups regarding inflammatory markers improvement duration, duration of MV and O<sub>2</sub> support duration, with longer duration among (therapeutic) group 2 cases compared to group 1 cases (prophylactic). There was a highly significant difference between both study groups regarding ICU admission, as 64% of group 1 cases were admitted compared to 25% of group 1 cases. Similarly, 38.5% of group 2 cases needed MV compared to only 10% of group 1 cases, which was statistically significant. There was no significant difference between both groups regarding bleeding tendency and mortality (<i>p</i> value 0.54). <b><i>Conclusion:</i></b> Our results showed that use of prophylactic dose of enoxaparin might have some benefits compared to the therapeutic dose in terms of less duration of ICU and hospital stay, duration of oxygen support, need and duration of MV, and normalization of inflammatory markers. However, there was no significant difference between the 2 regimens regarding the mortality.

scholarly journals Factor H (FH) Is Released Slowly and Continuously from Human Endothelial Cells (ECs): FH Is Not Packaged in Weibel-Palade Bodies or Secreted in Response to EC Stimulation

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 4175-4175
Author(s):  
Sarah E Sartain ◽  
Nancy A Turner ◽  
Hui Shiu-Ki ◽  
Charles G. Minard ◽  
Joel L Moake
Keyword(s):  
Research Funding ◽  
Complement Components ◽  
Alexa Fluor ◽  
Time Points ◽  
Significant Difference ◽  
The Mean ◽  
Von Willebrand ◽  
Fund Research

Abstract Introduction Ultra-large von Willebrand factor (ULVWF) strings are synthesized in ECs, packaged in Weibel-Palade Bodies (WPBs), and secreted by stimulated ECs. Complement components studied to date [C3, factor (F) B, FD, FP, FH, FI, C5] are released slowly and continuously from human umbilical vein endothelial cell (HUVEC) cytoplasm and are not packaged in WPBs (PLoS One. 2013;8(3):e59372). In contrast, a recent report (Blood. 2014;123(1):121-5) contended that FH co-localizes with VWF in the WPBs. If this were so, it could have therapeutic importance for the treatment of atypical hemolytic uremic syndrome (aHUS) resulting from deficiency of FH because it might be possible to increase circulating FH levels transiently by administration of the WPB secretagogue, des-amino-D-arginine vasopressin (DDAVP). Hypothesis FH is not co-localized with VWF in WBPs, but rather is released slowly and continuously from EC cytoplasm regardless of cell stimulation. Methods Immunofluorescent Microscopy HUVECs were stimulated with histamine and stained with rabbit anti-VWF plus secondary donkey anti-rabbit Alexa Fluor IgG-488. The cells were then fixed and stained with goat-anti FH plus secondary chicken anti-goat Alexa Fluor IgG-647. The nuclei were detected with DAPI. In vitro VWF and FH from HUVECs HUVECs either were, or were not, stimulated with histamine. Supernatant was collected a variety of times over 7 hrs and assayed for VWF and FH antigen levels by ELISA. VWF assay antibodies (polyclonal): (1) capture, rabbit anti-human VWF (Ramco); (2) detection, goat anti-human VWF (Bethyl) and rabbit anti-goat IgG-HRP (Invitrogen). FH assay antibodies: (1) capture, polyclonal goat anti-human FH (Advanced Research Technologies); (2) detection, monoclonal mouse anti-human FH (Pierce, Thermo Scientific) and polyclonal goat anti-mouse IgG-HRP (Invitrogen). In vivo VWF and FH Plasma samples were obtained from 6 pediatric patients with von Willebrand disease (VWD) being tested for EC release of WPB VWF in response to DDAVP. For each patient, 1 sample was obtained prior to DDAVP administration, and 2 other samples were obtained 1 and 4 hours later. VWF levels were measured in each sample using standard clinical laboratory procedure at an affiliated hospital. FH antigen levels were quantified by ELISA, as above. Results Using non-overlapping spectral secondary detection antibody pairs, VWF was seen in clusters in HUVEC WPBs (Fig. 1A). In contrast, FH was distributed throughout the HUVEC cytoplasm (Fig. 1B). The VWF and FH images did not overlap, indicating that VWF and FH did not co-localize in the WPBs. Fig 1. Immunofluorescent images of HUVECs stained for VWF and FH. Fig 1. Immunofluorescent images of HUVECs stained for VWF and FH. Histamine addition to HUVECs in vitro resulted in ~ 4-fold increases in VWF secreted from HUVEC WPBs at 30 min and 1 hour, and 2-fold increases at 3 hours (Fig 2A). In contrast, FH release was slow and continuous, regardless of histamine stimulation, suggesting that FH is located in EC cytoplasm and is not stored in WPBs (Fig. 2B). Fig 2. In vitro VWF and FH release from ECs under non-stimulated and histamine-stimulated conditions. Fig 2. In vitro VWF and FH release from ECs under non-stimulated and histamine-stimulated conditions. In all 6 patient samples, VWF antigen increased significantly at 1-hour post-DDAVP administration (Fig. 3A). In contrast, FH antigen levels did not change significantly at hour 1 or hour 4, compared to hour 0, indicating that FH is not co-localized and secreted along with VWF from the WPBs of stimulated ECs in vivo (Fig. 3B). Fig 3. (A) Mean responses of VWF antigen to DDAVP, in vivo, with 95% CIs.The mean response was significantly greater 1-hour and 4-hours post-DDAVP compared with baseline (P=0.0085 and 0.0079, respectively). After 1-hour post-DDAVP, the mean response was 201% greater (95% CI: 129%, 314%) than baseline. After 4-hours, the mean response was 174% greater (95% CI: 123%, 247%) than baseline. (B) Responses of FH to DDAVP, in vivo. There was no statistically significant difference in FH response between time points (P=0.77). Fig 3. (A) Mean responses of VWF antigen to DDAVP, in vivo, with 95% CIs.The mean response was significantly greater 1-hour and 4-hours post-DDAVP compared with baseline (P=0.0085 and 0.0079, respectively). After 1-hour post-DDAVP, the mean response was 201% greater (95% CI: 129%, 314%) than baseline. After 4-hours, the mean response was 174% greater (95% CI: 123%, 247%) than baseline. (B) Responses of FH to DDAVP, in vivo. There was no statistically significant difference in FH response between time points (P=0.77). Conclusions We used immunofluorescent microscopy and ELISA assays on samples obtained in vitro and in vivo to demonstrate that FH is not packaged in, or secreted from, the WPBs of stimulated human ECs. FH is, therefore, similar to all other complement components studied to date in that it is released slowly and continuously from ECs and is not influenced by cell stimulation. DDAVP is unlikely to be a viable treatment option for patients with aHUS secondary to deficiency or inhibition of FH. Disclosures Sartain: Hemostasis and Thrombosis Research Society: Research Funding. Turner:Mary R Gibson Foundation: Research Funding; Hinkson Memorial Fund : Research Funding. Moake:Mary R Gibson Foundation: Research Funding; Hinkson Memorial Fund: Research Funding.

scholarly journals A Cross-Sectional Study on Subjective Fever Assessment in Children by Palpation: Are Fathers as Reliable as Mothers?

2020 ◽  
Vol 2020 ◽  
pp. 1-5
Author(s):  
Ehud Rosenbloom ◽  
Crysta Balis ◽  
Dustin Jacobson ◽  
Melanie Conway ◽  
Ji Cheng ◽  
...  
Keyword(s):  
Primary Outcome ◽  
Pediatric Patients ◽  
Cross Sectional Study ◽  
Primary Outcome Measure ◽  
Sectional Study ◽  
Cross Sectional ◽  
Predictive Values ◽  
Mothers And Fathers ◽  
Significant Difference ◽  
The Mean

Background. Fever is common in pediatric patients. Often, parents rely solely on palpation when assessing their child’s fever. The objective of the current study was to determine the accuracy of parents in detecting their child’s fever by palpation. Methods. A prospective cross-sectional study was conducted at the emergency department (ED) of a tertiary pediatric hospital. Infants and children, 0–4 years of age, presenting to the ED with both parents were included. Parents were separately asked if their child had a fever and, if so, were asked to assess the temperature by palpation. A nurse obtained the rectal temperature. The primary outcome measure was the accuracy of fathers and mothers in detecting fever. Results. A total of 170 children with their parents were enrolled. The mean ages of the children, mothers, and fathers were 18.9 (SD 0.8) months, 31.1 (SD 6.4) years, and 33.7 (SD 6.9) years, respectively. No statistically significant difference was found between mothers and fathers in the ability to assess fever by palpation (OR 0.65, 95% CI 0.39,−1.08). Sensitivities for detecting fever by palpation for mothers and father were 86.4% and 88.2%, respectively (specificity among mothers: 54.2% and specificity among fathers: 43.1%). The overall negative and positive predictive values were 65.9% (95% CI 55%–75.7%) and 75.7% (95% CI 69.9%–80.8%), respectively. Conclusions. Mothers and fathers do not differ in their ability to accurately assess their child’s fever by palpation. The low positive and negative predictive values indicate that if temperature was not measured, physicians cannot rely on parents’ reports.

Effectiveness and tolerability of oral versus subcutaneous methotrexate in patients with rheumatoid arthritis

Rheumatology ◽  
2021 ◽  
Author(s):  
Janne Heuvelmans ◽  
Nathan den Broeder ◽  
Geke A H van den Elsen ◽  
Alfons A den Broeder ◽  
Bart J F van den Bemt
Keyword(s):  
Rheumatoid Arthritis ◽  
Retrospective Cohort ◽  
Primary Outcome ◽  
Real Life ◽  
Treat To Target ◽  
Oral Methotrexate ◽  
Real Life Setting ◽  
Significant Difference ◽  
The Mean ◽  
Secondary Outcomes

Abstract Objectives The aim of this study was to compare the effectiveness and tolerability between oral methotrexate (MTX) and subcutaneous MTX in a large group of rheumatoid arthritis (RA) patients in a real-life setting. Methods In this retrospective cohort study, adult patients with clinical diagnosis of RA who started MTX treatment (monotherapy or combined with hydroxychloroquine), either started with oral or subcutaneous MTX. The primary outcome was superiority testing of between group difference in change in DAS28CRP between baseline and 3–6 months, and subsequent non inferiority testing (NI margin 0.6) analyses in case of non-superiority. Secondary outcomes included MTX dose, side effects, laboratory abnormalities, and use of comedication. Results 640 RA patients were included: 259 started with oral MTX and 381 with subcutaneous. There was no significant difference in ΔDAS28CRP, after adjusting for confounding, 0.13 (95%-CI: -0.14, 0.40), and oral MTX strategy was non inferior to subcutaneous. The mean MTX dose was slightly lower for the oral strategy (18.0 SD6.9 vs 19.9 SD8.2, p= 0.002), which was accompanied by a lower cumulative incidence of adverse events (41% vs 52%, p= 0.005). No differences were seen in use of other comedication. Conclusions Starting with oral MTX in RA in a real-life setting is non inferior to a subcutaneous MTX treatment with regard to disease activity control, at least when used in dosages up to 25 mg and on a background of HCQ cotreatment and a treat-to-target approach. In addition, tolerability was better. This supports the strategy of starting with oral MTX.

scholarly journals A Prospective Study Evaluating IOP Changes after Switching from a Therapy with Prostaglandin Eye Drops Containing Preservatives to Nonpreserved Tafluprost in Glaucoma Patients

2012 ◽  
Vol 2012 ◽  
pp. 1-6 ◽  
Author(s):  
Stefano Ranno ◽  
Matteo Sacchi ◽  
Cinzia Brancato ◽  
Daniela Gilardi ◽  
Andrea Lembo ◽  
...  
Keyword(s):  
Primary Outcome ◽  
Open Angle Glaucoma ◽  
Hypotensive Effect ◽  
Eye Drops ◽  
Lowering Effect ◽  
Significant Difference ◽  
Ocular Discomfort ◽  
The Mean ◽  
A Prospective Study ◽  
Time Point

Purpose. To compare the ocular hypotensive effect of tafluprost with prostaglandin analogues (PGAs) in glaucoma patients.Methods. 89 primary open-angle glaucoma patients treated with bimatoprost, latanoprost, or travoprost for at least 3 months complaining for ocular discomfort were switched to tafluprost. IOP was assessed at baseline and 3 months after switching the therapy by daily curve. Primary outcome was to compare the mean daily IOP of tafluprost with PGAs.Results. The mean daily IOP was16±2.1and16.6±2.0 mm Hg at baseline and after switching to tafluprost, respectively (P>0.05). When analysis was carried out between tafluprost and each previous PGAs, the comparison between latanoprost and tafluprost and travoprost and tafluprost did not show any statistically significant difference in mean daily IOP and at each time point. The comparison between bimatoprost and tafluprost showed a statistically significant difference in mean daily IOP (P<0.05) and at each time point (P<0.05).Conclusions. After 3 months of switching tafluprost showed an overall IOP lowering effect similar to others PGAs. When each PGA was compared with tafluprost, bimatoprost showed to provide a statistically significant additional IOP lowering effect.

scholarly journals Impact of Available Therapies on Survival of Very Elderly Myelodysplastic Syndrome (MDS) Patients (Age above 75 yrs)

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 4611-4611
Author(s):  
Uma Borate ◽  
Vipin Lohiya ◽  
Garrett Sherwood ◽  
Bradford E Jackson ◽  
Harry P. Erba
Keyword(s):  
Research Funding ◽  
Rank Test ◽  
Very Elderly ◽  
Data Safety ◽  
Log Rank Test ◽  
Kaplan Meier ◽  
Significant Difference ◽  
Study Population ◽  
The Mean

Abstract Objective: Very elderly MDS patients (≥75 years) have limited therapeutic options and are usually ineligible for allogeneic stem cell transplantation. We aimed to study the impact of available MDS therapies on very elderly MDS patients and their correlation with patient demographics, performance status(PS) disease characteristics and patient outcomes. Methods: We performed a retrospective analysis of MDS patients ≥75 years diagnosed and treated at the University of Alabama at Birmingham from 2008 to 2014, with a minimum followup of 12 months. We analyzed demographics, ECOG PS, karyotypic risk categories as defined by the IPSS scoring system, blast percentage, IPSS and R-IPSS scores and overall survival (OS) in this population. We stratified patients based on therapy into two groups - the hypomethylating agent (HMA) group (defined as receiving therapy with ≥ 1 cycle of HMA; Azacitidine or Decitabine or both) and the non-HMA group, which included treatment with supportive transfusions, erythropoietin stimulating agents (ESAs), lenalidomide and cytotoxic chemotherapy. We analyzed group differences for all the parameters mentioned above using chi square test for categorical variables, and t test and Mann Whitney u-test for mean and medians respectively. In addition, OS was examined using Kaplan Meier curves using the log rank test. We used univariate and multivariate analysis to examine the effects of variables of interest on OS.All results were considered statistically significant at α=0.05 level. Results: The study population included 58 patients of which 35 patients were males (60%). Median age was 78 years. Forty patients (71%) of patients had good, 6% had intermediate and 23% had poor karyotypic profiles by the IPSS scoring system. ECOG ≥2 was observed in 44% of the patients with no significant differences in both groups. Average IPSS and R-IPSS scores were 1.2 and 4.5 respectively. Median OS for the entire study population was noted to be 15.5 months (7-34m). There were 25 patients in the HMA group and 33 patients in the non-HMA group. The blast percentage was higher in HMA group (20.5% vs 9.4%) compared to non-HMA group. More patients had a good karyotypic profile in the non-HMA group when compared to HMA group (80% vs 60%). There was a statistically significant difference between the mean IPSS and R-IPSS prognostic scores in non-HMA and HMA group (0.9 vs 1.7, p=0.010 and 3.5 vs 5.5, p=0.002) respectively. There was no significant difference in median overall survival between the non-HMA and HMA group (16.5 m (7-53) vs 15.5 m (5-19) p=0.278) respectively but the mean survival rates between non-HMA and HMA group were statistically different (32.81 vs 15.85, p=0.034). According to the log rank test, a statistical difference (p=0.027) in survival estimates was observed between the two groups on Kaplan Meier curve, where the HMA group had a significantly shorter survival compared to the non-HMA group. In the univariate analysis for the entire sample, higher IPSS score; R-IPSS score, and higher blast percentage were associated with increased rate of events. Moreover, rates of events were found to be lower in patients who did not receive HMA therapy (HR - 0.45, p=0.033), however in multivariable analysis, only higher blast percentage was associated with increased rate of events (HR - 1.06 p=0.025 95% CI - 1.004-1.11). Patients in the HMA group received average of 7.8 cycles. After stopping HMA therapy, 10 patients received other therapies including cytotoxic chemotherapy, hydroxyurea and lenalidomide, 4 were enrolled in a clinical trial, 9 received supportive transfusions and ESAs while 2 died immediately afterwards. Conclusion: Our study did not find a difference in median OS between patients who received HMA therapy versus non-HMA therapy in this population of very elderly MDS patients. Patients who received HMA therapy had a higher risk karyotypic profile, increased blast percentage and higher IPSS and R-IPSS scores. The average number of HMA cycles they received was 7.8, indicating adequate therapy. However, we could not evaluate transfusion needs, hospitalizations or other quality of life measures in these 2 groups. In conclusion, further studies need to be done to better evaluate various MDS therapies and their impact on quality of life and survival in this very elderly population with a higher comorbidity burden, possibly limiting the benefit of these treatments typically seen in younger MDS patients. Disclosures Borate: Genoptix: Consultancy; Seattle Genetics: Research Funding; Gilead: Speakers Bureau; Alexion: Speakers Bureau; Novartis: Speakers Bureau; Amgen: Speakers Bureau. Erba:Millennium/Takeda: Research Funding; Jannsen (J&J): Other: Data Safety and Monitoring Committees; Ariad: Consultancy; Millennium/Takeda: Research Funding; Celgene: Consultancy, Speakers Bureau; Astellas: Research Funding; Celgene: Consultancy, Speakers Bureau; Pfizer: Consultancy; Astellas: Research Funding; Incyte: Consultancy, Speakers Bureau; Pfizer: Consultancy; Seattle Genetics: Consultancy, Research Funding; Amgen: Consultancy, Research Funding; Sunesis: Consultancy; Incyte: Consultancy, Speakers Bureau; GlycoMimetics: Other: Data Safety and Monitoring Committees; Jannsen (J&J): Other: Data Safety and Monitoring Committees; Amgen: Consultancy, Research Funding; Celator: Research Funding; Novartis: Consultancy, Speakers Bureau; Daiichi Sankyo: Consultancy; Sunesis: Consultancy; Seattle Genetics: Consultancy, Research Funding; Daiichi Sankyo: Consultancy; Ariad: Consultancy; Novartis: Consultancy, Speakers Bureau; GlycoMimetics: Other: Data Safety and Monitoring Committees; Celator: Research Funding.

scholarly journals Emollient bath additives for the treatment of childhood eczema (BATHE): multicentre pragmatic parallel group randomised controlled trial of clinical and cost effectiveness

BMJ ◽  
2018 ◽  
pp. k1332 ◽  
Author(s):  
Miriam Santer ◽  
Matthew J Ridd ◽  
Nick A Francis ◽  
Beth Stuart ◽  
Kate Rumsby ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Cost Effectiveness ◽  
Topical Corticosteroid ◽  
Primary Outcome ◽  
Control Group ◽  
Significant Difference ◽  
The Mean ◽  
Secondary Outcomes ◽  
Bath Additives

AbstractObjectivesTo determine the clinical effectiveness and cost effectiveness of including emollient bath additives in the management of eczema in children.DesignPragmatic randomised open label superiority trial with two parallel groups.Setting96 general practices in Wales and western and southern England.Participants483 children aged 1 to 11 years, fulfilling UK diagnostic criteria for atopic dermatitis. Children with very mild eczema and children who bathed less than once weekly were excluded.InterventionsParticipants in the intervention group were prescribed emollient bath additives by their usual clinical team to be used regularly for 12 months. The control group were asked to use no bath additives for 12 months. Both groups continued with standard eczema management, including leave-on emollients, and caregivers were given standardised advice on how to wash participants.Main outcome measuresThe primary outcome was eczema control measured by the patient oriented eczema measure (POEM, scores 0-7 mild, 8-16 moderate, 17-28 severe) weekly for 16 weeks. Secondary outcomes were eczema severity over one year (monthly POEM score from baseline to 52 weeks), number of eczema exacerbations resulting in primary healthcare consultation, disease specific quality of life (dermatitis family impact), generic quality of life (child health utility-9D), utilisation of resources, and type and quantity of topical corticosteroid or topical calcineurin inhibitors prescribed.Results483 children were randomised and one child was withdrawn, leaving 482 children in the trial: 51% were girls (244/482), 84% were of white ethnicity (447/470), and the mean age was 5 years. 96% (461/482) of participants completed at least one post-baseline POEM, so were included in the analysis, and 77% (370/482) completed questionnaires for more than 80% of the time points for the primary outcome (12/16 weekly questionnaires to 16 weeks). The mean baseline POEM score was 9.5 (SD 5.7) in the bath additives group and 10.1 (SD 5.8) in the no bath additives group. The mean POEM score over the 16 week period was 7.5 (SD. 6.0) in the bath additives group and 8.4 (SD 6.0) in the no bath additives group. No statistically significant difference was found in weekly POEM scores between groups over 16 weeks. After controlling for baseline severity and confounders (ethnicity, topical corticosteroid use, soap substitute use) and allowing for clustering of participants within centres and responses within participants over time, POEM scores in the no bath additives group were 0.41 points higher than in the bath additives group (95% confidence interval −0.27 to 1.10), below the published minimal clinically important difference for POEM of 3 points. The groups did not differ in secondary outcomes, economic outcomes, or adverse effects.ConclusionsThis trial found no evidence of clinical benefit from including emollient bath additives in the standard management of eczema in children. Further research is needed into optimal regimens for leave-on emollient and soap substitutes.Trial registrationCurrent Controlled Trials ISRCTN84102309.

Assessment of Safety and Efficacy of a New Intravenous Immunoglobulin (IGIV3I 10% Grifols) In Subjects with Chronic Immune Thrombocytopenia

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 4669-4669
Author(s):  
Ali Khojasteh ◽  
Giraldo Kato ◽  
Nehal Parikh ◽  
Tammuella Singleton ◽  
Cameron K Tebbi ◽  
...  
Keyword(s):  
Platelet Count ◽  
Research Funding ◽  
Clinical Laboratory ◽  
Vital Signs ◽  
Study Drug ◽  
Threshold Value ◽  
Bleeding Diathesis ◽  
Haemorrhagic Diathesis ◽  
Duration Of Response ◽  
The Mean

Abstract Abstract 4669 Two similarly designed prospective, uncontrolled, open-label clinical trials were planned to assess the safety and efficacy of a new intravenous immunoglobulin (IVIG), IGIV3I 10% Grifols, in subjects with chronic immune thrombocytopenia (ITP) in the USA, Canada and Europe (Spain, Russia and the United Kingdom). Subjects were candidates to be enrolled if they had a diagnosis of chronic ITP and a baseline platelet count ≤ 20 × 109/l. Eligible subjects received treatment with IGIV3I 10% Grifols 1g/kg for 2 consecutive days or 0.4 g/kg for 5 consecutive days. The primary efficacy endpoint was the response rate, defined as the proportion of subjects reaching or exceeding the threshold value of 50 × 109/l on or before day 8 (American study) or on day 30 (European study) where day 1 is the day of the first infusion. Secondary efficacy endpoints were the time to response, defined as the number of days elapsed from day 1 to achievement of the threshold value; duration of response, defined as the number of consecutive days with a platelet count known to be ≥50 × 109/l; the maximum (peak) platelet count and regression of haemorrhagic diathesis for those subjects presenting with bleeding signs at baseline. Safety endpoints included adverse events (AEs), physical examinations, vital signs and clinical laboratory parameters monitoring. A total of 27 subjects (18 adults and 9 pediatrics) have been enrolled, i.e. administered with at least one infusion of the product at any dose, in the study. Twenty-four (24) subjects (89%) were considered responders. This proportion was higher in pediatric subjects (9/9, 100%) than in adult subjects (15/18, 83%). The mean time to response was 1.6 days (Standard Deviation (SD) 0.9); the mean duration of response was 14.0 days (SD 12.1) and the mean maximum platelet count was 263 × 109/l (SD 195.6). Twenty-three (23) subjects (all the pediatric subjects 9/9 and 14/18 adults) presented with some sign of bleeding at baseline. All subjects (23/23, 100%) experienced an improvement in the haemorrhagic diathesis, regardless of some of them being considered non-responders according to the platelet count criterion. A total of 92 AEs potentially related to study drug were reported. The most common of these were headache (25 events), nausea (8 events), pyrexia (7 events) and chills (6 events). Only 1 AE was reported as definitely related to study drug, an event of palmar erythema. Two serious AEs (SAE) potentially related to the study drug were reported in 2 subjects. One of them was an unexpected laboratory alteration which consisted in leukopenia and decreased hemoglobin and advised to maintain the subject hospitalized for further observation during the weekend. The reaction was transient, without complications or other clinical symptoms and total recovery of the normal laboratory values. The second SAE was an event of thrombosis in the right humeral vein, in a patient with a number of predisposing medical conditions. At the time of discharge the patient's overall condition was satisfactory. Analysis of AEs, clinical laboratory values, physical assessments and vital signs did not indicate any safety concerns for subjects receiving the study drug and are in line with those expected for the ITP population treated with IVIG. Both primary and secondary efficacy endpoints show a good response to the product in terms of rapid elevation of platelet counts to an hemostatic level in line with what is expected for an IVIG product. Moreover, the improvement of the baseline bleeding diathesis even in patients considered as non-responders according to the platelet count criterion is highly suggestive of the clinical efficacy of the product. Overall, these results indicate that treatment with the new IGIV3I 10% Grifols is safe and efficacious for rapidly increasing platelet counts in chronic ITP subjects and improving their bleeding diathesis. Disclosures: Khojasteh: Grifols S.A.: Research Funding. Kato:Grifols S.A.: Research Funding. Parikh:Grifols S.A.: Research Funding. Singleton:Grifols S.A.: Research Funding. Tebbi:Grifols S.A.: Research Funding. Cromwell:Grifols S.A.: Research Funding. Fu:Grifols S.A.: Research Funding. Kessler:Grifols S.A.: Research Funding. Letzer:Grifols S.A.: Research Funding. Ritchie:Grifols S.A.: Research Funding. Sexauer:Grifols S.A.: Research Funding. Saxena:Grifols S.A.: Research Funding. Sirpal:Grifols S.A.: Research Funding. Torres:Grifols S.A.: Research Funding. Loriya:Grifols S.A.: Research Funding. Kovaleva:Grifols S.A.: Research Funding. Sandoval:Grifols S.A.: Research Funding. Sanz:Grifols S.A.: Research Funding. Julià:Grifols S.A.: Research Funding. Montañés:Grifols S.A.: Employment. Navarro:Grifols S.A.: Employment.

Endoscopic sleeve gastroplasty: suturing the gastric fundus does not confer benefit

Endoscopy ◽  
2020 ◽  
Author(s):  
Jad Farha ◽  
Christopher McGowan ◽  
Abdellah Hedjoudje ◽  
Mohamad I. Itani ◽  
Shahem Abbarh ◽  
...  
Keyword(s):  
Weight Loss ◽  
Primary Outcome ◽  
Gastric Fundus ◽  
Serious Adverse Event ◽  
Endoscopic Sleeve Gastroplasty ◽  
Significant Difference ◽  
Sleeve Gastroplasty ◽  
The Mean ◽  
Procedure Duration ◽  
Percentage Excess Weight Loss

Abstract Background There is heterogeneity regarding the technical aspects of endoscopic sleeve gastroplasty (ESG), such as applying fundal sutures. Our aim was to determine whether ESG with fundal suturing (ESG-FS) affects weight loss and the serious adverse event (SAE) rate when compared with ESG with no fundal suturing (ESG-NFS). Methods We conducted a two-center retrospective analysis of 247 patients who underwent ESG with or without fundal suturing. The primary outcome was percentage excess weight loss (%EWL) at 3, 6, and 12 months post-ESG. The secondary outcomes included the SAE rate and procedure duration. Results At 3, 6, and 12-months, ESG-NFS had a significantly greater mean %EWL compared with ESG-FS (38.4 % [standard deviation (SD) 15.3 %] vs. 31.2 % [SD 13.9 %], P = 0.001; 54.7 % [SD 19.2 %] vs. 37.7 % [SD 17.3 %], P < 0.001; 65.3 % [SD 21.1 %] vs. 40.6 % [SD 23.5 %], P < 0.001, respectively). There was no statistically significant difference in the SAE rates for ESG-NFS (n = 2; 2.0 %) and ESG-FS (n = 4; 2.6 %; P > 0.99). The mean procedure time was significantly shorter in the ESG-NFS group at 59.1 minutes (SD 32.7) vs. 93.0 minutes (35.5; P < 0.001), and a lower mean number of sutures were used, with 5.7 (SD 1.1) vs. 8.4 (SD 1.6; P < 0.001). Conclusion ESG-NFS demonstrated greater efficacy and shorter procedure duration. Therefore, fundal suturing should not be performed.

scholarly journals Efficacy and safety of subthreshold micropulse laser compared with threshold conventional laser in central serous chorioretinopathy

Eye ◽  
2019 ◽  
Vol 34 (9) ◽  
pp. 1592-1599 ◽  
Author(s):  
Zuhua Sun ◽  
Ying Huang ◽  
Chaochao Nie ◽  
Zhijie Wang ◽  
Junqing Pei ◽  
...  
Keyword(s):  
Visual Acuity ◽  
Central Serous Chorioretinopathy ◽  
Primary Outcome ◽  
Spot Size ◽  
Efficacy And Safety ◽  
Micropulse Laser ◽  
Significant Difference ◽  
Subthreshold Micropulse Laser ◽  
The Mean ◽  
Conventional Laser

Abstract Purpose To compare the efficacy and safety of subthreshold micropulse laser (SML) with threshold conventional laser (TCL) in central serous chorioretinopathy (CSC). Methods Prospective, randomized, double-masked, non-inferiority, 12-week clinical trial. Patients were randomly assigned 1:1 to SML group or TCL group. Patients in the SML group were treated with 577 nm micropulse laser. The spot size was 160 µm, the duty cycle was 5% and exposure time was 0.2 s. The power was 50% threshold tested. Patients in the TCL group were treated with 577 nm continuous laser. The power was 100% threshold tested. The primary outcome was the mean change in best-corrected visual acuity (BCVA) at week 12, with a non-inferiority limit of five letters on the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity charts. Results Eighty-eight patients were enroled. Seventy-seven patients were male. Forty-four patients were in SML group and 44 in TCL group. At week 12, SML was equivalent to TCL with a gain of 6.23 ± 8.59 and 6.61 ± 6.35 letters, respectively, (SML–TCL difference: −0.38 letters; 95% confidence interval (CI):−3.58–2.81; Pnon-inferiority = 0.0026). There was no statistically significant difference between the two groups (t = 0.240, P = 0.811). At week 12, the proportion of patients whose SRF had been totally absorbed was 63.63 and 81.82% respectively for SML and TCL groups. There was no statistically significant difference between the two groups (χ2 = 3.67, P = 0.056). Conclusions Both SML and TCL can improve visual acuity in CSC. SML was non-inferior to TCL in the improvement of BCVA.

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