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Introduction The ILUVIEN® (fluocinolone acetonide) Clinical Evidence in Portugal (ICE-PT) study is a retrospective, multicenter, observational study evaluating the effectiveness and safety of the FAc implant in patients with diabetic macular edema. Methods Patients included in this study had received the 0.2 µg/day fluocinolone acetonide implant for the treatment of diabetic macular edema and had measurements of visual acuity and retinal thickness assessed by optical coherence tomography for at least 12 months pre- and post-fluocinolone acetonide implant administration, with ⩾2 follow-up visits. Outcomes measured included visual acuity, central foveal thickness, and intraocular pressure. Results There was a significant increase in mean visual acuity compared with baseline at 3, 6, 9, and 12 months post-fluocinolone acetonide in both the overall study population and the pseudophakic subgroup (p less then 0.05 at all time points in both groups). A significant reduction in mean central foveal thickness compared with baseline was seen in the overall study population at 3, 6, 9, and 12 months post-fluocinolone acetonide (p less then 0.05 at all time points). At 12-month post-fluocinolone acetonide, a small but significant intraocular pressure increase of 1.0 mmHg was seen in the overall study population. Conclusion The results of this analysis show that switching from the current standard of care to the fluocinolone acetonide implant leads to beneficial effects in terms of vision and retinal structure in patients with diabetic macular edema and that patients benefited from FAc implant administration, regardless of lens status.Hemolytic uremic syndrome (HUS) may result in thrombotic central nervous system complications. We present a child with diarrhea-associated HUS who developed new-onset focal seizures secondary to cerebral sinovenous thrombosis (CSVT). Her CSVT was treated with low-molecular-weight heparin. The patient’s seizures were controlled with levetiracetam, and her HUS was managed supportively with hemodialysis. Repeat imaging nearly 6 months following presentation and initiation of anticoagulation demonstrated cerebral sinus enlargement and persistent intraluminal webbing. Anticoagulation was discontinued after 6 months, and she did not experience long-term gross neurologic sequelae. CSVT is a complication of HUS that has not been previously described. In this report, we summarize the thrombotic central nervous system complications of pediatric HUS.Introduction A lack of uniformity in the choice of outcome measurement in hemophilia care and research has led to studies with incomparable results. Chaetocin nmr We identified a need to define core outcome measures for use in research and clinical care of persons with hemophilia. Objective To move toward a core set of outcome measures for the assessment of persons with hemophilia in research and practice. Methods A modified nominal groups process was conducted with an international group of hemophilia experts, including persons with hemophilia as follows. Step 1 item generation for all potential outcome measures. Step 2 survey where respondents voted on the relative importance and usefulness of each item. Steps 3/4 2-day meeting where attendees voted for items they valued, followed by open discussion and a second round of voting. Step 5 survey where respondents selected their top five items from those with >50% agreement at the meeting. Results The highest ranked items for the pediatric core set (% agreement) are treatment satisfaction (92.7%), joint health (83.3%), a measure of access to treatment (82.5%), a measure of treatment adherence (72.5%), and generic performance based physical function (72.1%). The highest ranked items for the adult core set (% agreement) are total bleeding events (88.1%), EuroQol five dimensions (85.4%), treatment adherence (82.1%), joint health (79.1%), and number/location of bleeds per unit time (78.6%). Conclusion This process generated a list of preferred outcome measures to consider for assessment in persons with hemophilia. This information now requires refinement to define optimal core sets for use in different clinical/research contexts.Background Hemophilia gene therapy is a rapidly evolving therapeutic approach in which a number of programs are approaching clinical development completion. Objective The aim of this study was to evaluate knowledge and perceptions of a variety of health care practitioners and scientists about gene therapy for hemophilia. Methods This survey study was conducted February 1 to 18, 2019. Survey participants were members of the ISTH, European Hemophilia Consortium, European Hematology Association, or European Association for Hemophilia and Allied Disorders with valid email contacts. The online survey consisted of 36 questions covering demographic information, perceptions and knowledge of gene therapy for hemophilia, and educational preferences. Survey results were summarized using descriptive statistics. Results Of the 5117 survey recipients, 201 responded from 55 countries (4% response rate). Most respondents (66%) were physicians, and 59% were physicians directly involved in the care of people with hemophilia. Among physician respondents directly involved in hemophilia care, 35% lacked the ability to explain the science of adeno-associated viral gene therapy for hemophilia, and 40% indicated limited ability or lack of comfort answering patient questions about gene therapy for hemophilia based on clinical trial results to date. Overall, 75% of survey respondents answered 10 single-answer knowledge questions correctly, 13% incorrectly, and 12% were unsure of the correct answers. Conclusions This survey highlighted knowledge gaps and educational needs related to gene therapy for hemophilia and, along with other inputs, has informed the development of “Gene Therapy in Hemophilia An ISTH Education Initiative.”Introduction We conducted a longitudinal study in patients with pancreatic and colorectal cancer. We determined the effect of chemotherapy on extracellular vesicle tissue factor (EVTF) activity and the association of plasma EVTF activity with venous thromboembolism (VTE) and survival. Material and methods We enrolled 13 patients with pancreatic and 22 patients with colorectal cancer. Plasma samples were collected during the 85-day study period. Patients were followed for 3 months after the study period. We recorded symptomatic VTE during the study period (3 months) or asymptomatic deep vein thrombosis detected by ultrasound at day 85. We measured EVTF activity before and after chemotherapy. Results and conclusions In the pancreatic cancer group, 2 patients had elevated levels of EVTF activity. One of these patients developed symptomatic VTE and died, and the second patient did not have a VTE but died. Chemotherapy decreased EVTF activity in 2 pancreatic patients with high levels. In the colorectal cancer group, 4 patients developed VTE, but EVTF activity was not elevated in any patient and no patient died.