scholarly journals Proposal for a new therapeutic high dosage of Pidotimod in children with Periodic fever, aphthous stomatitis, pharyngitis, adenitis (PFAPA) syndrome: a randomized controlled study.

2020 ◽  
Author(s):  
Sara Manti ◽  
Federica Filosco ◽  
Giuseppe Fabio Parisi ◽  
Giuseppe Germano Finocchiaro ◽  
Maria Papale ◽  
...  
Keyword(s):  
Adverse Events ◽  
Periodic Fever ◽  
Phase 1 ◽  
Aphthous Stomatitis ◽  
Controlled Study ◽  
Serious Adverse Events ◽  
Group A ◽  
Pfapa Syndrome ◽  
Decision Group ◽  
Group B

Abstract Background. Despite to PFAPA syndrome is considered a benign and self-limited condition in childhood its impact on patients and families can be remarkable in many cases. Currently, the therapeutic options for managing are non-specific and no consensus exists about the best treatment to use. Pidotimod has been suggested as a new potential treatment in PFAPA syndrome for its immunodulatory effects. We conducted a preliminary, prospective, controlled, open, cross-over trial to assess the efficacy and the safety of Pidotimod in the treatment of children with PFAPA syndrome. Methods. 22 children with PFAPA syndrome were randomly allocated to treatment with pidotimod (with 2 vials of 400mg daily) in combination with betamethasone 0.5-1 mg on need, based on parents/caregivers' decision (group A) or betamethasone 0.5-1mg on need, based on parents/caregivers' decision (group B). Each treatment period was for 3 months (Phase 1), after that patients were switched to the other arm for other 3 months (Phase 2). Efficacy was expressed in terms of number of episodes of fever, pharyngitis, or aphthous stomatitis, as well as the additional use of betamethasone on need. Safety and tolerability of the Pidotimod were evaluated on the basis of the number and type of adverse events (AEs) recorded during the treatment.Results. Patients receiving Pidotimod and use betametasone showed a significant decrease in frequency of fevers (p=0.002); number of episodes of pharyngitis (p=0.049); aphthous stomatitis (p=0.036) as well as the betamethasone use on need (p=0.007). Overall, 19/22 (86.4%) showed benefits from Pidotimod administration. The safety profile of Pidotimod was excellent as no serious adverse events have been reported in the treated groups.Conclusions. We firstly showed that high dosage of Pidotimod could be an effective and safe to reduce the PFAPA attacks in children.

scholarly journals Proposal for a new therapeutic high dosage of Pidotimod in children with Periodic fever, aphthous stomatitis, pharyngitis, adenitis (PFAPA) syndrome: an interventional study.

2020 ◽  
Author(s):  
Sara Manti ◽  
Federica Filosco ◽  
Giuseppe Fabio Parisi ◽  
Giuseppe Germano Finocchiaro ◽  
Maria Papale ◽  
...  
Keyword(s):  
Adverse Events ◽  
Periodic Fever ◽  
Phase 1 ◽  
Aphthous Stomatitis ◽  
Interventional Study ◽  
Serious Adverse Events ◽  
Group A ◽  
Pfapa Syndrome ◽  
Decision Group ◽  
Group B

Abstract Background. Despite to PFAPA syndrome is considered a benign and self-limited condition in childhood its impact on patients and families can be remarkable in many cases. Currently, the therapeutic options for managing are non-specific and no consensus exists about the best treatment to use. Pidotimod has been suggested as a new potential treatment in PFAPA syndrome for its immunodulatory effects. We conducted a preliminary, prospective, controlled, open, cross-over trial to assess the efficacy and the safety of Pidotimod in the treatment of children with PFAPA syndrome. Methods. 22 children with PFAPA syndrome were randomly allocated to treatment with pidotimod (with 2 vials of 400mg daily) in combination with betamethasone 0.5-1 mg on need, based on parents/caregivers' decision (group A) or betamethasone 0.5-1mg on need, based on parents/caregivers' decision (group B). Each treatment period was for 3 months (Phase 1), after that patients were switched to the other arm for other 3 months (Phase 2). Efficacy was expressed in terms of number of episodes of fever, pharyngitis, or aphthous stomatitis, as well as the additional use of betamethasone on need. Safety and tolerability of the Pidotimod were evaluated on the basis of the number and type of adverse events (AEs) recorded during the treatment.Results. Patients receiving Pidotimod and use betametasone showed a significant decrease in frequency of fevers (p=0.002); number of episodes of pharyngitis (p=0.049); aphthous stomatitis (p=0.036) as well as the betamethasone use on need (p=0.007). Overall, 19/22 (86.4%) showed benefits from Pidotimod administration. The safety profile of Pidotimod was excellent as no serious adverse events have been reported in the treated groups.Conclusions. We firstly showed that high dosage of Pidotimod could be an effective and safe to reduce the PFAPA attacks in children.

scholarly journals Proposal for a new therapeutic high dosage of Pidotimod in children with Periodic fever, aphthous stomatitis, pharyngitis, adenitis (PFAPA) syndrome: an observational study.

2020 ◽  
Author(s):  
Sara Manti ◽  
Federica Filosco ◽  
Giuseppe Fabio Parisi ◽  
Giuseppe Germano Finocchiaro ◽  
Maria Papale ◽  
...  
Keyword(s):  
Adverse Events ◽  
Periodic Fever ◽  
Phase 1 ◽  
Aphthous Stomatitis ◽  
Potential Treatment ◽  
Phase 2 ◽  
Serious Adverse Events ◽  
Group A ◽  
Pfapa Syndrome ◽  
Group B

Abstract Background. Despite to PFAPA syndrome is considered a benign and self-limited condition in childhood its impact on patients and families can be remarkable in many cases. Currently, the therapeutic options for managing are non-specific and no consensus exists about the best treatment to use. Pidotimod has been suggested as a new potential treatment in PFAPA syndrome for its immunodulatory effects. We conducted a preliminary, prospective, controlled, open, cross-over trial to assess the efficacy and the safety of Pidotimod in the treatment of children with PFAPA syndrome.Methods. 22 children with PFAPA syndrome were randomly allocated to treatment with Pidotimod (with 2 vials of 400 mg daily) in combination with betamethasone 0.5-1 mg on need (group A) or betamethasone 0.5-1 mg on need (group B). Each treatment period was for 3 months (Phase 1), after that patients were switched to the other arm for other 3 months (Phase 2). Efficacy was expressed in terms of number of episodes of fever, tonsillitis, and aphthous stomatitis, as well as the additional use of betamethasone on need. Safety and tolerability of the Pidotimod were evaluated on the basis of the number and type of adverse events (AEs) recorded during the treatment.Results. Patients receiving Pidotimod and betametasone showed a significant decrease in frequency of fevers (p = 0.002); number of episodes of tonsillitis (p = 0.049); aphthous stomatitis (p = 0.036) as well as the betamethasone use on need (p = 0.007). Overall, 19/22 (86.4%) showed benefits from Pidotimod administration. The safety profile of Pidotimod was excellent as no serious adverse events have been reported in the treated groups.Conclusions. We firstly showed that high dosage of Pidotimod is an effective and safe to reduce the PFAPA attacks in children.

scholarly journals A Phase 1 Randomized Placebo-Controlled Study to Assess the Safety, Immunogenicity and Genetic Stability of a New Potential Pandemic H7N9 Live Attenuated Influenza Vaccine in Healthy Adults

Vaccines ◽  
2020 ◽  
Vol 8 (2) ◽  
pp. 296
Author(s):  
Irina Kiseleva ◽  
Irina Isakova-Sivak ◽  
Marina Stukova ◽  
Marianna Erofeeva ◽  
Svetlana Donina ◽  
...  
Keyword(s):  
Adverse Events ◽  
Influenza Vaccine ◽  
Phase 1 ◽  
Healthy Adults ◽  
Controlled Study ◽  
Temperature Sensitive ◽  
Serious Adverse Events ◽  
Double Blind ◽  
Human Influenza Virus ◽  
Live Attenuated Influenza Vaccine

This study describes a double-blind randomized placebo-controlled phase I clinical trial in healthy adults of a new potential pandemic H7N9 live attenuated influenza vaccine (LAIV) based on the human influenza virus of Yangtze River Delta hemagglutinin lineage (ClinicalTrials.gov Identifier: NCT03739229). Two doses of H7N9 LAIV or placebo were administered intranasally to 30 and 10 subjects, respectively. The vaccine was well-tolerated and not associated with increased rates of adverse events or with any serious adverse events. Vaccine virus was detected in nasal swabs during the 6 days after vaccination or revaccination. A lower frequency of shedding was observed after the second vaccination. Twenty-five clinical viral isolates obtained after the first and second doses of vaccine retained the temperature-sensitive and cold-adapted phenotypic characteristics of LAIV. There was no confirmed transmission of the vaccine strain from vaccinees to placebo recipients. After the two H7N9 LAIV doses, an immune response was observed in 96.6% of subjects in at least one of the assays conducted.

scholarly journals Feasibility of Non-Anesthesiologist-Administered Propofol Sedation for Emergency Endoscopic Retrograde Cholangiopancreatography

2015 ◽  
Vol 2015 ◽  
pp. 1-7 ◽  
Author(s):  
Nobuhito Ikeuchi ◽  
Takao Itoi ◽  
Takuji Gotoda ◽  
Chika Kusano ◽  
Shin Kono ◽  
...  
Keyword(s):  
Adverse Events ◽  
Endoscopic Retrograde Cholangiopancreatography ◽  
Acute Cholangitis ◽  
Careful Monitoring ◽  
Endoscopic Retrograde ◽  
Serious Adverse Events ◽  
Significant Difference ◽  
Group A ◽  
Group B ◽  
Technical Safety

Background. The safety of non-anesthesiologist-administered propofol (NAAP) sedation in emergent endoscopic retrograde cholangiopancreatography (ERCP) has not been fully clarified. Thus, the aim of this study was to assess the safety of NAAP sedation in emergent ERCP.Materials and Methods. We retrospectively analyzed 182 consecutive patients who had obstructive jaundice and who underwent ERCP under NAAP sedation. The patients were divided into Group A (with mild acute cholangitis or without acute cholangitis) and Group B (moderate or severe acute cholangitis). And technical safety and adverse events were assessed.Results. The adverse events were hypoxia (31 cases), hypotension (26 cases), and bradycardia (2 cases). There was no significant difference in the rate of each adverse event of hypoxia and bradycardia in either group. Although the rate of transient hypotension associated in Group B was higher than that in Group A, it was immediately improved with conservative treatment. Moreover, there were no patients who showed delayed awakening, or who developed other complications.Conclusions. In conclusion, NAAP sedation is feasible even in emergent ERCP. Although some transient adverse events (e.g., hypotension) were observed, no serious adverse events occurred. Thus, propofol can be used in emergent ERCP but careful monitoring is mandatory.

scholarly journals Randomized controlled study of ECP with methoxsalen as first-line treatment of patients with moderate to severe cGVHD

2019 ◽  
Vol 3 (14) ◽  
pp. 2218-2229 ◽  
Author(s):  
Madan Jagasia ◽  
Christof Scheid ◽  
Gérard Socié ◽  
Francis Ayuketang Ayuk ◽  
Johanna Tischer ◽  
...  
Keyword(s):  
Adverse Events ◽  
Phase 1 ◽  
Standard Of Care ◽  
Response Assessment ◽  
Controlled Study ◽  
First Line ◽  
Serious Adverse Events ◽  
Hematopoietic Stem ◽  
Intention To Treat ◽  
First Line Therapy

Abstract The investigation of extracorporeal photopheresis (ECP) plus standard of care (SoC) (SoC+ECP) in chronic graft-versus-host disease (cGVHD) within prospective, randomized clinical studies is limited, despite its frequent clinical use. This phase 1/pilot study was the first randomized, prospective study to investigate ECP use as first-line therapy in cGVHD, based on the 2015 National Institutes of Health (NIH) consensus criteria for diagnosis and response assessment. Adult patients with new-onset (≤3 years of hematopoietic stem cell transplantation) moderate or severe cGVHD were randomized 1:1 to 26 weeks of SoC+ECP vs SoC (corticosteroids and cyclosporine A/tacrolimus) between 2011 and 2015. The primary endpoint was overall response rate (ORR), defined as complete or partial response, at week 28 in the intention-to-treat population (ITT). Other outcomes included quality of life (QoL) measures and safety. Sixty patients were randomized; ITT included 53 patients (SoC+ECP: 29; SoC: 24). Week 28 ORR was 74.1% (SoC+ECP) and 60.9% (SoC). Investigator-assessed ORR was 56.0% (SoC+ECP) and 66.7% (SoC). Patients treated with SoC experienced a decline in QoL over the 28-week study period; QoL remained unchanged in SoC+ECP patients. Most frequent treatment-emergent adverse events (TEAEs) in SoC+ECP patients were hypertension (31.0%), cough (20.7%), dyspnea (17.2%), and fatigue (17.2%). Seventeen patients (SoC+ECP: 8; SoC: 9) experienced 35 serious adverse events (SAEs). No TEAEs or SAEs were considered related to the ECP instrument or methoxsalen. The encouraging short-term results of this study could inform the design of subsequent studies. This trial was registered at www.clinicaltrials.gov as #NCT01380535.

scholarly journals Treating gambling disorder with as needed administration of intranasal naloxone: a pilot study to evaluate acceptability, feasibility and outcomes

BMJ Open ◽  
2019 ◽  
Vol 9 (8) ◽  
pp. e023728 ◽  
Author(s):  
Sari Castrén ◽  
Niklas Mäkelä ◽  
Janne Haikola ◽  
Anne H Salonen ◽  
Roger Crystal ◽  
...  
Keyword(s):  
Depressive Symptoms ◽  
Pilot Study ◽  
Alcohol Use ◽  
Adverse Events ◽  
Gambling Disorder ◽  
Gambling Behaviour ◽  
Serious Adverse Events ◽  
Opioid Receptor Antagonists ◽  
Group A ◽  
Group B

Background and aimThere is growing interest in the use of medication-assisted treatments for gambling disorder (GD). Opioid receptor antagonists are hypothesised to blunt the craving associated with gambling. This study was designed to assess the feasibility of using an intranasal naloxone spray to treat GD.DesignAn 8-week, open-label, uncontrolled pilot study.SettingA single study site in the capital region of Finland.SubjectsTwenty problem gamblers (nine men) were randomised into two groups. Group A (n=10) took one dose into one nostril (2 mg naloxone), as needed, with a maximum of 4 doses/day (max. 8 mg/day). Group B (n=10) took one dose into each nostril (4 mg naloxone) as needed, with a maximum of 4 doses/day (max. 16 mg/day).InterventionNaloxone hydrochloride nasal spray.MeasuresAcceptability and feasibility of the intervention were assessed. Use of study medication, adverse events, gambling frequency and gambling expenditure were recorded in a mobile diary. Problem gambling: South Oaks Gambling Screen (SOGS), depressive symptoms: Beck Depression Inventory (BDI) and alcohol use: Alcohol Use Disorders Identification Test were recorded.ResultsStudy completion rate was 90%. Acceptability and feasibility scores were high. Group B used intranasal naloxone more frequently than group A, and consequently used more naloxone. No serious adverse events were reported. The postintervention SOGS scores were lower (median=4 (IQR=3.75) versus preintervention scores (median=12 (IQR=4.75)). Depressive symptoms were reduced during the trial (preintervention BDI median=9, IQR=9 vs postintervention BDI median=6, IQR=6).ConclusionsThe acceptability and feasibility of using intranasal naloxone were high, and no serious adverse events were reported. Preliminary results suggest mixed results in terms of gambling behaviour (ie, reduced frequency but not expenditure) and decreased depressive symptoms.Trial registration numberEudraCT2016-001828-56

scholarly journals Human Safety, Tolerability, and Pharmacokinetics of a Novel Broad-Spectrum Oral Antiviral Compound, Molnupiravir, with Activity Against SARS-CoV-2

2020 ◽  
Author(s):  
Wendy P. Painter ◽  
Wayne Holman ◽  
Jim A. Bush ◽  
Firas Almazedi ◽  
Hamzah Malik ◽  
...  
Keyword(s):  
Severe Acute Respiratory Syndrome ◽  
Adverse Events ◽  
Clinical Laboratory ◽  
Phase 1 ◽  
Influenza Viruses ◽  
Controlled Study ◽  
Serious Adverse Events ◽  
Double Blind ◽  
Antiviral Compound ◽  
Multiple Doses

AbstractMolnupiravir, EIDD-2801/MK-4482, the prodrug of the ribonucleoside analog ß-d-N4-hydroxycytidine (NHC), has activity against a number of RNA viruses including severe acute respiratory syndrome coronavirus 2, severe acute respiratory syndrome coronavirus, Middle East respiratory syndrome coronavirus, seasonal and pandemic influenza viruses, and respiratory syncytial virus.Single and multiple doses of molnupiravir were evaluated in this first-in-human, phase 1, randomized, double-blind, placebo-controlled study in healthy volunteers, which included evaluation of the effect of food on pharmacokinetics.EIDD-1931 appeared rapidly in plasma, with a median time of maximum observed concentration of 1.00 to 1.75 hours, and declined with a geometric half-life of approximately 1 hour, with a slower elimination phase apparent following multiple doses or higher single doses (7.1 hours at the highest dose tested). Mean maximum observed concentration and area under the concentration versus time curve increased in a dose-proportional manner, and there was no accumulation following multiple doses. When administered in a fed state, there was a decrease in the rate of absorption, but no decrease in overall exposure.Molnupiravir was well tolerated. Fewer than half of subjects reported an adverse event, the incidence of adverse events was higher following administration of placebo, and 93.3% of adverse events were mild. One discontinued early due to rash. There were no serious adverse events and there were no clinically significant findings in clinical laboratory, vital signs, or electrocardiography. Plasma exposures exceeded expected efficacious doses based on scaling from animal models; therefore, dose escalations were discontinued before a maximum tolerated dose was reached.Clinical trial identifierThis study was registered at ClinicalTrials.gov with the identifier NCT04392219.

scholarly journals Human Safety, Tolerability, and Pharmacokinetics of Molnupiravir, a Novel Broad-Spectrum Oral Antiviral Agent with Activity Against SARS-CoV-2

2021 ◽  
Author(s):  
Wendy P. Painter ◽  
Wayne Holman ◽  
Jim A. Bush ◽  
Firas Almazedi ◽  
Hamzah Malik ◽  
...  
Keyword(s):  
Severe Acute Respiratory Syndrome ◽  
Adverse Events ◽  
Clinical Laboratory ◽  
Phase 1 ◽  
Influenza Viruses ◽  
Controlled Study ◽  
Serious Adverse Events ◽  
Double Blind ◽  
Time Curve ◽  
Multiple Doses

Molnupiravir, EIDD-2801/MK-4482, the prodrug of the active antiviral ribonucleoside analog ß-d-N4-hydroxycytidine (NHC; EIDD-1931), has activity against a number of RNA viruses including severe acute respiratory syndrome coronavirus 2, severe acute respiratory syndrome coronavirus, Middle East respiratory syndrome coronavirus, and seasonal and pandemic influenza viruses. Single and multiple doses of molnupiravir were evaluated in this first-in-human, phase 1, randomized, double-blind, placebo-controlled study in healthy volunteers, which included evaluation of the effect of food on pharmacokinetics. EIDD-1931 appeared rapidly in plasma, with a median time of maximum observed concentration of 1.00 to 1.75 hours, and declined with a geometric half-life of approximately 1 hour, with a slower elimination phase apparent following multiple doses or higher single doses (7.1 hours at the highest dose tested). Mean maximum observed concentration and area under the concentration versus time curve increased in a dose-proportional manner, and there was no accumulation following multiple doses. When administered in a fed state, there was a decrease in the rate of absorption, but no decrease in overall exposure. Molnupiravir was well tolerated. Fewer than half of subjects reported an adverse event, the incidence of adverse events was higher following administration of placebo, and 93.3% of adverse events were mild. One discontinued early due to rash. There were no serious adverse events and there were no clinically significant findings in clinical laboratory, vital signs, or electrocardiography. Plasma exposures exceeded expected efficacious doses based on scaling from animal models; therefore, dose escalations were discontinued before a maximum tolerated dose was reached.

scholarly journals The Effect of Cardiometry Guided Fluid Management on Outcome of Patients Presented for Intracranial Surgeries: Randomized Controlled Study

2021 ◽  
pp. 16-25
Author(s):  
Amgd Shaaban El-Sheikh ◽  
Sameh Abdelkhalik Ismael ◽  
Nagat Sayed El-Shmaa ◽  
Soheir Mostafa Soliman
Keyword(s):  
Adverse Events ◽  
Hospital Stay ◽  
Fluid Therapy ◽  
Fluid Management ◽  
Controlled Study ◽  
Number Of Patients ◽  
Neurosurgical Patients ◽  
Group A ◽  
Group B ◽  
Hospital Stay Duration

Background: Fluid management in neurosurgical patients is critical and important during the perioperative period. Electrical cardiometry (EC) is a new noninvasive technique for measuring cardiac output (COP). EC works based on the application of a high frequency transthoracic current and the analysis of variations of voltage in each heartbeat. The aim of this work is to compare the fluid management of intracranial surgeries using EC routine parameters. Methods: This is a prospective randomized, double-blinded controlled study was carried out on 70 patients of both genders aged > 21 years old, ASA physical status II or III, GCS 15 scheduled for elective craniotomy. Patients were divided into two equal groups at random; group A: standard management, group B: EC guided management. The primary outcome was the duration of intensive care unit (ICU) stay. Results: The ICU and hospital stay duration were significantly decreased in group B compared to group A. The mean total amount of infused volume of crystalloid solutions was significantly decreased in group B compared to group A. Hemodynamics, and number of patients received colloid, blood, vasopressor, and inotropes were insignificantly different between both groups. There was a significant increase in optic nerve sheath diameter in group A compared to group B at PACU and 24 h. Adverse events were comparable between both groups except encephalodema, which was significantly higher in group A. Conclusions: EC is an effective tool in COP measurement and a novel guide for fluid therapy as EC guided fluid therapy group was significantly decreased in ICU and hospital stay duration and the total amount of crystalloid with fewer adverse events.

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