scholarly journals 546. Capturing Clinician’s Experiences Repurposing Drugs to Inform Future Studies During COVID-19

2020 ◽  
Vol 7 (Supplement_1) ◽  
pp. S339-S339
Author(s):  
Mili Duggal ◽  
Heather Stone ◽  
Parvesh Paul ◽  
Reema Charles ◽  
Leonard Sacks ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Case Reports ◽  
Healthcare Providers ◽  
Individual Case ◽  
Intravenous Immunoglobulins ◽  
Interferon Alfa ◽  
Case Report Form ◽  
Electronic Case Report Form ◽  
Repurposed Drugs ◽  
The University

Abstract Background CURE ID is an internet-based repository developed collaboratively by FDA and NCATS/NIH, with the support of WHO and IDSA. It encourages clinicians globally to share novel uses of existing drugs for patients with difficult-to-treat infections. It is designed to serve as a rapid communication platform for healthcare providers during an outbreak, providing for systematic case-sharing, discussion, and the latest literature. Besides case reports, CURE ID offers a discussion platform for clinicians, disease-specific clinical trials curated from clincialtrials.gov, and a newsfeed that shows relevant journal articles and news related to COVID-19 and other infectious diseases. Methods The CURE ID team extracted individual case reports on patient-level treatments and outcomes of COVID-19 infection from the published literature and gathered clinician-submitted cases through the electronic case report form. Additionally, CURE ID partnered with the University of Pennsylvania’s CORONA database to further populate the CURE ID database with published cases. Results As of submission, lopinavir-Ritonavir (n=51) was the most commonly reported drug used. The following were also reported: hydroxychloroquine (n=31), azithromycin (n=28), arbidol (n=22), interferon alfa-2B (n=18), moxifloxacin (n=18), methylprednisolone (n=17), ivermectin (n=14), lopinavir (n=12), oseltamivir (n=12). The other drugs reported were danoprevir-ritonavir, intravenous immunoglobulins, interferon, interferon alfa, and tocilizumab. CURE ID currently includes more than 150 detailed COVID case reports of 65 repurposed drugs. We expect case reporting for specific drugs to be dynamic and additional data to accrue. Updated results will be presented. Conclusion Several drugs are being repurposed to treat COVID-19. CURE ID gives clinicians an opportunity to share their treatment experiences and discuss their questions with a global community of healthcare providers. By utilizing the CURE ID platform, in conjunction with data gathered from other registries, observational studies and clinical trials, hypotheses can be generated that may inform future clinical trials and ultimately, potentially find safe and effective treatments for this deadly disease. Disclosures All Authors: No reported disclosures

PEDIATRIC NEUROLOGISTS E-MAIL/INTERNET

PEDIATRICS ◽  
1994 ◽  
Vol 94 (3) ◽  
pp. 321-321
Author(s):  
Mack KJ
Keyword(s):  
Electronic Mail ◽  
Case Reports ◽  
Individual Case ◽  
Journal Articles ◽  
Therapeutic Approaches ◽  
University Of Wisconsin ◽  
On Line ◽  
The University ◽  
E Mail ◽  
Clinical Problems

An electronic mail mailing list has been created to aid communication between pediatric neurologists. Any child neurologist or neurologist with connections to the Internet, or a private on-line service can subscribe. The mailserver serves as a forum for discussion of topics in child neurology. Topics include problem patients, therapeutic approaches to clinical problems, and discussions of recent journal articles. In addition, we hope that individual case reports may evolve into series of patients by pooling similar patients from separate institutions. Cases, questions, comments, etc. e-mailed to the mailserver are automatically forwarded to everyone on the list. Responses can either be private (to the initial sender or any other member) or public (to everyone on the list). The CHILD-NEURO mailserver is run out of the University of Wisconsin, and the service is free-of-charge. To sign up, send an e-mail message from the address you will be using to: [email protected] with no subject and SUBSCRIBE CHILDNEURO as the entire message. A welcoming message with instructions on the use of the mailserver will be sent to new subscribers. Please e-mail or phone Dr. Ken Mack ([email protected]; 608-263-9086) or Dr. Steve Leber ([email protected]) for more information, or if difficulties arise in subscribing to the list.

scholarly journals Remdesivir for Early COVID-19 Treatment of High-Risk Individuals Prior to or at Early Disease Onset—Lessons Learned

Viruses ◽  
2021 ◽  
Vol 13 (6) ◽  
pp. 963
Author(s):  
Lars Dölken ◽  
August Stich ◽  
Christoph D. Spinner
Keyword(s):  
Clinical Trials ◽  
Clinical Efficacy ◽  
Case Reports ◽  
Antiviral Treatment ◽  
Treatment Options ◽  
Disease Onset ◽  
Antiviral Agent ◽  
Individual Case ◽  
Lessons Learned ◽  
Disease Stage

After more than one year of the COVID-19 pandemic, antiviral treatment options against SARS-CoV-2 are still severely limited. High hopes that had initially been placed on antiviral drugs like remdesivir have so far not been fulfilled. While individual case reports provide striking evidence for the clinical efficacy of remdesivir in the right clinical settings, major trials failed to demonstrate this. Here, we highlight and discuss the key findings of these studies and underlying reasons for their failure. We elaborate on how such shortcomings should be prevented in future clinical trials and pandemics. We suggest in conclusion that any novel antiviral agent that enters human trials should first be tested in a post-exposure setting to provide rapid and solid evidence for its clinical efficacy before initiating further time-consuming and costly clinical trials for more advanced disease. In the COVID-19 pandemic this might have established remdesivir early on as an efficient antiviral agent at a more suitable disease stage which would have saved many lives, in particular in large outbreaks within residential care homes.

scholarly journals Erythema Migrans: Course and Outcome in Patients Treated With Rituximab

2019 ◽  
Vol 6 (7) ◽  
Author(s):  
Vera Maraspin ◽  
Petra Bogovič ◽  
Tereza Rojko ◽  
Eva Ružić-Sabljić ◽  
Franc Strle
Keyword(s):  
Antibiotic Treatment ◽  
Medical Condition ◽  
Medical Center ◽  
Case Reports ◽  
Erythema Migrans ◽  
Individual Case ◽  
Underlying Disease ◽  
Adult Patients ◽  
The University ◽  
Immunocompetent Patients

Abstract Background Information on Lyme borreliosis (LB) in patients treated with rituximab is limited to individual case reports. Methods We reviewed data on adult patients diagnosed with typical erythema migrans (EM) at the LB outpatient clinic of the University Medical Center Ljubljana, Slovenia, in the 10-year period 2008–2017. For all patients, clinical and laboratory information was acquired prospectively using a standardized questionnaire. Results Among 4230 adult patients with a diagnosis of EM, 7 patients (0.17%), 5 women and 2 men with a median age of 65 years (range, 55–66 years), were receiving rituximab for an underlying medical condition. In these 7 patients, signs of disseminated LB (43%) and the isolation rates of borreliae from blood before antibiotic treatment (40%) were unusually high compared with corresponding findings in immunocompetent patients who had EM diagnosed at the same institution (8% vs <2%, respectively). The rates of LB-associated constitutional symptoms and borrelial antibodies in serum were lower than expected (14% and 29%, respectively, in patients receiving rituximab vs 25% and 65% in immunocompetent patients). One of the 7 patients (14%) experienced treatment failure; nevertheless, the outcome of early LB 1 year after antibiotic treatment, as used for immunocompetent patients with EM, was excellent in all 7 patients. Conclusions Findings in 7 patients with EM who were receiving rituximab for underlying disease suggest that although early LB in these patients is more often disseminated than in immunocompetent patients, the outcome 1 year after antibiotic treatment, as used for immunocompetent patients, is excellent.

Herpetic Whitlow of the Hand in Infants

2020 ◽  
Author(s):  
Mohammad M. Al-Qattan ◽  
Nada G. AlQadri ◽  
Ghada AlHayaza
Keyword(s):  
Bacterial Infection ◽  
Case Reports ◽  
Case Series ◽  
Individual Case ◽  
Senior Author ◽  
Secondary Bacterial Infection ◽  
Clinical Appearance ◽  
History Of ◽  
Mode Of Transmission ◽  
Time Of Presentation

Abstract Introduction Herpetic whitlows in infants are rare. Previous authors only reported individual case reports. We present a case series of six infants. Materials and Methods This is a retrospective study of six cases of herpetic whitlows in infants seen by the senior author (MMA) over the past 23 years (1995–2017 inclusive). The following data were collected: age, sex, digit involved in the hand, mode of transmission, time of presentation to the author, clinical appearance, presence of secondary bacterial infection, presence of other lesions outside the hand, method of diagnosis, treatment, and outcome. Results All six infants initially presented with classic multiple vesicles of the digital pulp. In all cases, there was a history of active herpes labialis in the mother. Incision and drainage or deroofing of the vesicles (for diagnostic purposes) resulted in secondary bacterial infection. Conclusion The current report is the first series in the literature on herpetic whitlows in infants. We stress on the mode of transmission (from the mother) and establishing the diagnosis clinically. In these cases, no need for obtaining viral cultures or polymerase chain reaction; and no medications are required. Once the vesicles are disrupted, secondary bacterial infection is frequent and a combination of oral acyclovir and intravenous antibiotics will be required.

Clinical trials — the personal perspective of the research physician?

2020 ◽  
Vol 75 (3) ◽  
pp. 256-263
Author(s):  
Maria Y. Egorova ◽  
Irina A. Shuvalova ◽  
Olga I. Zvonareva ◽  
Igor D. Pimenov ◽  
Olga S. Kobyakova ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Medical Care ◽  
New Technologies ◽  
Healthcare Providers ◽  
Study Drug ◽  
Well Being ◽  
Personal Perspective ◽  
Public And Private ◽  
Factors Affecting ◽  
To Receive

Background. The organization of clinical trials (CTs) requires the participation and coordination of healthcare providers, patients, public and private parties. Obstacles to the participation of any of these groups pose a risk of lowering the potential for the implementation of CTs. Researchers are a key human resource in conducting of CT. Their motivation for participation can have a significant impact on the recruitment and retention of patients, on the quality of the data collected, which determines the overall outcome of the study. Aims to assess the factors affecting the inclusion of Russian physicians-researchers in CT, and to determine their role in relations with patients-participants. Materials and methods. The study was organized as a part of the Russian multicenter face-to-face study. A survey was conducted of researchers from 10 cities of Russia (20172018). The participation in the survey for doctors was anonymous and voluntary. Results. The study involved 78 respondents. Most research doctors highly value the importance of research for science (4,84 0,39), society (4,67 0,46) and slightly lower for participating patients (4,44 0,61). The expectations of medical researchers are related to improving their financial situation and attaining new experience (n = 14; 18,18%). However, the opportunity to work with new technologies of treatment and diagnosis (n = 41; 52,56%) acted as a motivating factor. According to the questionnaire, the vast majority of research doctors (n = 29; 37,18%) believe that the main reason for patients to participate in CT is to receive quality and free medical care. The most significant obstacle to the inclusion of participants in CT was the side effects of the study drug (n = 38; 48,71%). Conclusions. The potential of clinical researchers in Russia is very high. The patient-participant acts for the research doctor as the subject of the study, and not the object, so the well-being of the patient is not indifferent to the doctor. However, the features of the functioning of our health care system form the motivation of doctors-researchers (additional earnings, professional self-development) and the way they perceive the motivation of patients (CT as an opportunity to receive quality medical care).

scholarly journals Development and design of the Hantavirus registry - HantaReg - for epidemiological studies, outbreaks and clinical studies on hantavirus disease

2021 ◽  
Author(s):  
Felix C Koehler ◽  
Linda Blomberg ◽  
Thomas Theo Brehm ◽  
Stefan Büttner ◽  
Oliver A Cornely ◽  
...  
Keyword(s):  
Data Entry ◽  
Scoring Systems ◽  
Epidemiological Studies ◽  
Incidence Rates ◽  
Case Report Form ◽  
Treatment Pathways ◽  
General Data Protection Regulation ◽  
The World ◽  
Electronic Case Report Form ◽  
Substantial Risk

Abstract Background Frequent outbreaks around the globe and endemic appearance in different parts of the world emphasize the substantial risk of hantavirus diseases. Increasing incidence rates, trends of changing distribution of hantavirus species and new insights into clinical courses of hantavirus diseases call for multinational surveillance. Furthermore, evidence-based guidelines for the management of hantavirus diseases and scoring systems, which allow stratification of patients into risk categories, are lacking. Methods Hantavirus registry (HantaReg) is a novel registry platform facilitating multinational research of hantavirus-caused diseases, such as haemorrhagic fever with renal syndrome (HFRS) and hantavirus cardiopulmonary syndrome (HCPS). HantaReg provides an electronic case report form and uses the General Data Protection Regulation compliant platform clinicalsurveys.net, which can be accessed from any internet browser in the world. Having a modular structure, the registry platform is designed to display or hide questions and items according to the documented case (e.g. patient with HFRS versus HCPS) to facilitate fast, but standardized, data entry. Information categories documented in HantaReg are demographics, pre-existing diseases, clinical presentation, diagnostic and therapeutic approaches, as well as outcome. Conclusions HantaReg is a novel, ready-to-use platform for clinical and epidemiological studies on hantavirus diseases and facilitates the documentation of the disease course associated with hantavirus infections. HantaReg is expected to promote international collaboration and contributes to improving patient care through the analysis of diagnostic and treatment pathways for hantavirus diseases, providing evidence for robust treatment recommendations. Moreover, HantaReg enables the development of prognosis-indicating scoring systems for patients with hantavirus disease.

scholarly journals Efficacy and safety of intravenous immunoglobulins for the treatment of viral encephalitis: a systematic literature review

2021 ◽  
Author(s):  
Judith N. Wagner ◽  
Annette Leibetseder ◽  
Anna Troescher ◽  
Juergen Panholzer ◽  
Tim J. von Oertzen
Keyword(s):  
Systematic Review ◽  
Viral Encephalitis ◽  
Viral Infections ◽  
Case Reports ◽  
Ivig Therapy ◽  
Intravenous Immunoglobulins ◽  
Efficacy And Safety ◽  
Serious Adverse Events ◽  
Optimal Dosing ◽  
Cochrane Database

Abstract Background For most viral encephalitides, therapy is merely supportive. Intravenous immunoglobulins (IVIG) have been used as a prophylactic and therapeutic approach. We conduct a systematic review on the safety and efficacy of IVIG in viral encephalitis. Methods We conducted a systematic review assessing PubMed, Cochrane Database, Biosis Previews and the ClinicalTrials.gov website to identify all reports on patients with viral encephalitis treated with IVIG as of May 31, 2019. The main outcomes assessed were therapeutic efficacy and safety. For an increased homogeneity of the population, atypical viral infections were excluded, as were reports on prophylactic IVIG use, intrathecal application of immunoglobulins, or use of antibody-enriched IVIG-preparations. Data were extracted from published studies. Descriptive statistics were used. Results We included a total of 44 studies (39 case reports). The case reports cover a total of 53 patients. Our search retrieved two prospective and three retrospective studies. These show heterogeneous results as to the efficacy of IVIG therapy. Only one study reports a significant association between IVIG-use and death (odds ratio 0.032; 95% confidence interval 0.0033–0.3024; p = 0.0027). None of the studies report significant differences in the number of serious adverse events. Conclusion Data on the efficacy of IVIG-therapy is heterogeneous. While it seems generally safe, evident superiority compared to supportive treatment has not been demonstrated so far. Future trials should also investigate the optimal dosing and timing of IVIG and their benefit in the immunosuppressed.

scholarly journals What Is New in the Treatment of Smoldering Multiple Myeloma?

2021 ◽  
Vol 10 (3) ◽  
pp. 421
Author(s):  
Niccolo’ Bolli ◽  
Nicola Sgherza ◽  
Paola Curci ◽  
Rita Rizzi ◽  
Vanda Strafella ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Clinical Trials ◽  
High Risk ◽  
Early Treatment ◽  
Individual Case ◽  
Symptomatic Disease ◽  
Plasma Cell Neoplasm ◽  
Smoldering Multiple Myeloma ◽  
Wide Range ◽  
Critical Issues

Smoldering multiple myeloma (SMM), an asymptomatic plasma cell neoplasm, is currently diagnosed according to the updated IMWG criteria, which reflect an intermediate tumor mass between monoclonal gammopathy of undetermined significance (MGUS) and active MM. However, SMM is a heterogeneous entity and individual case may go from an “MGUS-like” behavior to “early MM” with rapid transformation into symptomatic disease. This wide range of clinical outcomes poses challenges for prognostication and management of individual patients. However, initial studies showed a benefit in terms of progression or even survival for early treatment of high-risk SMM patients. While outside of clinical trials the conventional approach to SMM generally remains that of close observation, these studies raised the question of whether early treatment should be offered in high-risk patients, prompting evaluation of several different therapeutic approaches with different goals. While delay of progression to MM with a non-toxic treatment is clearly achievable by early treatment, a convincing survival benefit still needs to be proven by independent studies. Furthermore, if SMM is to be considered less biologically complex than MM, early treatment may offer the chance of cure that is currently not within reach of any active MM treatment. In this paper, we present updated results of completed or ongoing clinical trials in SMM treatment, highlighting areas of uncertainty and critical issues that will need to be addressed in the near future before the “watch and wait” paradigm in SMM is abandoned in favor of early treatment.

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