scholarly journals The STArgardt Remofuscin Treatment Trial (STARTT): design and baseline characteristics of enrolled Stargardt patients

2021 ◽  
Vol 1 ◽  
pp. 96
Author(s):  
Patty P.A. Dhooge ◽  
Philipp T. Möller ◽  
Camiel J.F. Boon ◽  
Andrew J. Lotery ◽  
Philipp Herrmann ◽  
...  
Keyword(s):  
Visual Acuity ◽  
Visual Function ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Reading Speed ◽  
Primary Outcome Measure ◽  
Treatment Trial ◽  
Patient Reported ◽  
Baseline Characteristics ◽  
Sd Oct

Background: This report describes the study design and baseline characteristics of patients with Stargardt disease (STGD1) enrolled in the STArgardt Remofuscin Treatment Trial (STARTT). Methods: In total, 87 patients with genetically confirmed STGD1 were randomized in a double-masked, placebo-controlled proof of concept trial to evaluate the safety and efficacy of 20 milligram oral remofuscin for 24 months. The primary outcome measure is change in mean quantitative autofluorescence value of an 8-segment ring centred on the fovea (qAF8). Secondary efficacy variables are best corrected visual acuity (BCVA), low-luminance visual acuity (LLVA), mesopic microperimetry (mMP),  spectral domain optical coherence tomography (SD-OCT), reading speed on Radner reading charts, and patient-reported visual function as assessed by the National Eye Institute Visual Functioning Questionnaire 25 (NEI VFQ-25) and Functional Reading Independence (FRI) Index. Results: Mean age of participants was 35±11 years with 49 (56%) female. Median qAF8 value was 438 Units (range 210-729). Median BCVA and LLVA in decimal units were 0.50 (range 0.13-0.80) and 0.20 (range 0.06-0.63), respectively. The median of the mean retinal sensitivity with mMP was 20.4 dB (range 0.0-28.8). SD-OCT showed median central subfield retinal thickness of 142 µm (range 72-265) and median macular volume of 1.65 mm3 (range 1.13-2.19). Compared to persons without vision impairment, both reading performance and patient-reported visual function were significantly lower (p<0.001, one sample t-test). Mean reading speed was 108±39 words/minute with logRAD-score of 0.45±0.28. Mean VFQ-25 composite score was 72±13. Mean FRI Index score 2.8±0.6. Conclusions: This trial design may serve as reference for future clinical trials as it explores the utility of qAF8 as primary outcome measure. The baseline data represent the largest, multi-national, STGD1 cohort to date that underwent standardized qAF imaging, reading speed assessment and vision-related quality of life measures which all contribute to the characterization of STGD1. EudraCT registration: 2018-001496-20 (09/05/2019)

scholarly journals Beyond visual acuity: Patient-relevant assessment measures of visual function in retinal diseases

2021 ◽  
pp. 112067212199062
Author(s):  
María Cinta Puell ◽  
Inés Contreras ◽  
Isabel Pinilla ◽  
José Juan Escobar ◽  
Antonio Soler-García ◽  
...  
Keyword(s):  
Visual Acuity ◽  
Clinical Practice ◽  
Outcome Measures ◽  
Visual Function ◽  
Outcome Measure ◽  
Expert Panel ◽  
Reading Speed ◽  
Evidence Synthesis ◽  
Age Related Macular Degeneration ◽  
Patient Reported

Purpose: To identify patient-reported outcomes (PROs) and other clinical outcome measures (contrast sensitivity (CS), low-luminance visual acuity (LLVA) and reading acuity or reading speed (RA-RS)), relevant to patients with age-related macular degeneration (AMD) or diabetic retinopathy (DR), which would be recommended for use in clinical practice. Methods: The RAND/UCLA Appropriateness Method, based on the synthesis of the scientific evidence and the collective judgment of an expert panel using the two-round Delphi method, was applied. The evidence synthesis was performed by searching for articles on outcome measures for AMD and/or DR published between 2005 and 2018 in English or Spanish. The expert panel consisted of 14 Spanish ophthalmologists, who rated the recommendation degree for each outcome measure on a scale of 1 (extremely irrelevant) to 9 (maximum relevance). The recommended outcome measures were established according to the panel median score and the level of the panelists’ agreement. Results: Through the evidence search, 33 PRO-specific questionnaires (21 for visual function, six for AMD, three for DR, one for AMD and DR) and two treatment satisfaction questionnaires (one on AMD and one on DR) were identified. In addition, 21 methods were found for measuring CS, five for LLVA, and nine for RA-RS. According to the panel ratings, 11 of the 64 outcome measures evaluated for AMD, and seven of the 61 evaluated for DR were recommended. Conclusion: The AMD and DR outcome measures recommended will help ophthalmologists choose the outcome measure most appropriate for their patients. Furthermore, the use of PROs will contribute to shifting clinical practice towards patient-centered medicine.

A phase III trial of intramuscular recombinant interferon beta as treatment for exacerbating-remitting multiple sclerosis: design and conduct of study and baseline characteristics of patients

1995 ◽  
Vol 1 (2) ◽  
pp. 118-135 ◽  
Author(s):  
LD Jacobs ◽  
DL Cookfair ◽  
RA Rudick ◽  
RM Herndon ◽  
J R Richert ◽  
...  
Keyword(s):  
Primary Outcome ◽  
Interferon Beta ◽  
Outcome Measure ◽  
Primary Outcome Measure ◽  
Phase Iii ◽  
Secondary Outcome ◽  
Recombinant Interferon ◽  
Study Population ◽  
The Mean ◽  
Baseline Characteristics

The design and conduct of a randomized, double-blinded, placebo-controlled, multicenter, phase III study of recombinant interferon beta-1a (IFN-β-1a) as treatment for exacerbating-remitting MS are described, as are baseline characteristics of the study population. The purpose of the study was to determine if 6.0 × 106 IU (30 μg) of IFN-β-1a, administered by weekly intramuscular (i.m.) injections, was effective in delaying the onset of sustained disability. The primary outcome measure was time to onset of treatment failure, defined as a worsening on the Kurtzke Expanded Disability Status Scale (EDSS) of greater than or equal to 1.0 point compared with baseline, persisting for at least 6 months. An intent-to-treat design was used. The primary outcome measure was analyzed using the Mantel-Cox log-rank statistic and Kaplan-Meier survival curves. Secondary outcomes included quantitative measures of upper and lower extremity function, neuropsychological test performance, functional and quality of life assessments and several measures derived from annual brain MRI studies. Entry criteria included prestudy exacerbation rates of at least 0.67 per year and EDSS scores of 1.0–3.5. A total of 301 MS patients were randomly assigned to receive weekly i.m. injections of IFN-β-1a or placebo. The average age of the study population at entry was 37 years; 92% were Caucasian and 73% were women. The mean prestudy disease duration was 6.5 years, mean prestudy exacerbation rate was 1.2 per year and the mean EDSS score was 2.3. The randomization yielded well-balanced treatment arms. Various aspects of the study are discussed, including: (1) the decision to focus study design on sustained disability; (2) the rationale for the treatment regimen; (3) measures taken to assure the reliability of the primary outcome measure; and (4) a description of the secondary outcome measures.

Is oocyte maturation rate associated with triptorelin dose used for triggering final oocyte maturation in patients at high risk for severe ovarian hyperstimulation syndrome?

2019 ◽  
Vol 34 (9) ◽  
pp. 1770-1777 ◽  
Author(s):  
G T Lainas ◽  
T G Lainas ◽  
I A Sfontouris ◽  
K Chatzimeletiou ◽  
C A Venetis ◽  
...  
Keyword(s):  
High Risk ◽  
Oocyte Maturation ◽  
Ovarian Stimulation ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Primary Outcome Measure ◽  
Final Oocyte Maturation ◽  
Maturation Rate ◽  
Baseline Characteristics ◽  
Severe Ohss

Abstract STUDY QUESTION Are oocyte maturation rates different among 0.1, 0.2 and 0.4 mg triptorelin used for triggering final oocyte maturation in patients at high risk for ovarian hyperstimulation syndrome (OHSS) undergoing ICSI? SUMMARY ANSWER A dose of 0.1 mg triptorelin results in similar oocyte maturation rates compared to higher doses of 0.2 and 0.4 mg in patients at high risk for OHSS undergoing ICSI. WHAT IS KNOWN ALREADY The GnRH agonist triptorelin is widely used instead of hCG for triggering final oocyte maturation, in order to eliminate the risk of severe OHSS in patients undergoing ovarian stimulation for IVF/ICSI. However, limited data are currently available regarding its optimal dose use for this purpose in patients at high risk for OHSS. STUDY DESIGN, SIZE, DURATION A retrospective study was performed between November 2015 and July 2017 in 131 infertile patients at high risk for severe OHSS undergoing ovarian stimulation for ICSI. High risk for severe OHSS was defined as the presence of at least 19 follicles ≥11 mm in diameter on the day of triggering final oocyte maturation. PARTICIPANTS/MATERIALS, SETTING, METHODS Ovarian stimulation was performed with recombinant FSH and GnRH antagonists. Patients received 0.1 (n = 42), 0.2 (n = 46) or 0.4 mg (n = 43) triptorelin for triggering final oocyte maturation. Hormonal evaluation of FSH, LH, estradiol (E2) and progesterone (PRG) was carried out on the day of triggering final oocyte maturation, 8 and 36 hours post triggering and 3, 5, 7, and 10 days after triptorelin administration. During this period, all patients were assessed for symptoms and signs indicative of severe OHSS development. Primary outcome measure was oocyte maturation rate, defined as the number of metaphase II (MII) oocytes divided by the number of cumulus-oocyte-complexes retrieved per patient. Results are expressed as median (interquartile range). MAIN RESULTS AND THE ROLE OF CHANCE No significant differences in patient baseline characteristics were observed among the 0.1 mg, the 0.2 mg and the 0.4 mg groups. Regarding the primary outcome measure, no differences were observed in oocyte maturation rate among the three groups compared [82.6% (17.8%) versus 83.3% (18.8%) versus 85.1% (17.2%), respectively, P = 0.686]. In addition, no significant differences were present among the 0.1 mg, 0.2 mg and 0.4 mg groups, regarding the number of mature (MII) oocytes [21 (13) versus 20 (6) versus 20 (11), respectively; P = 0.582], the number of oocytes retrieved [25.5 (13) versus 24.5 (11) versus 23 (12), respectively; P = 0.452], oocyte retrieval rate [81.0% (17.7%) versus 76.5% (23.5%) versus 75.0% (22.5), respectively; P = 0.088], the number of fertilized (two pronuclei) oocytes [12.5 (9) versus 14.5 (7) versus 14.0 (8), respectively; P = 0.985], fertilization rate [71.7% (22%) versus 77.1% (19.1%) versus 76.6% (23.3%), respectively; P = 0.525] and duration of luteal phase [7 (1) versus 8 (2) versus 7 (1) days, respectively; P = 0.632]. Moreover, no significant differences were present among the three triptorelin groups regarding serum levels of LH, FSH, E2 and PRG at any of the time points assessed following triggering of final oocyte maturation. LIMITATIONS, REASONS FOR CAUTION This is a retrospective study, and although there were no differences in the baseline characteristics of the three groups compared, the presence of bias cannot be excluded. WIDER IMPLICATIONS OF THE FINDINGS Based on the results of the current study, it appears that triggering final oocyte maturation with a lower (0.1 mg) or a higher dose (0.4 mg) of triptorelin, as compared to the most commonly used dose of 0.2 mg, does not confer any benefit in terms of oocyte maturation rate in patients at high risk for severe OHSS. STUDY FUNDING/COMPETING INTEREST(S) No external funding was obtained for this study. There are no conflicts of interest.

scholarly journals Pneumatic Vitreolysis With Intravitreal Air for Focal Vitreomacular Traction

2020 ◽  
pp. 247412642096264
Author(s):  
Mark E. Seamone ◽  
Uriel Rubin ◽  
Parampal S. Grewal ◽  
Mark Greve
Keyword(s):  
Visual Acuity ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Case Series ◽  
Primary Outcome Measure ◽  
Air Injection ◽  
Vitreomacular Traction ◽  
Corrected Visual Acuity ◽  
Visual Improvement ◽  
Phakic Eyes

Purpose: To determine whether pneumatic vitreolysis with intravitreal air is effective for focal vitreomacular traction (VMT). Methods: We conducted a retrospective consecutive case series of 20 eyes from 19 individuals with focal VMT who underwent pneumatic vitreolysis with intravitreal air (January 2017 to November 2018). We analyzed patients via spectral-domain optical coherence tomography before intravitreal air injection and at 1 month. The primary outcome measure was release of VMT. Results: We observed release of VMT in 55% of individuals. An analysis limited to phakic eyes demonstrated release of VMT in 69%, and 65% developed improved best-corrected visual acuity. Individuals with persistent VMT and visual improvement had a significant reduction in angle of vitreoretinal insertion ( P < .01), area under VMT ( P < .05), and subfoveal cyst area ( P < .05). Conclusions: Intravitreal air is an effective treatment for focal VMT. In individuals with persistent VMT, visual-acuity improvement was associated with a reduction in overall VMT.

scholarly journals Estimates of the mean difference in orthopaedic randomized trials: obligatory yet obscure

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Lauri Raittio ◽  
Antti Launonen ◽  
Ville M. Mattila ◽  
Aleksi Reito
Keyword(s):  
Primary Outcome ◽  
Outcome Measure ◽  
Patient Reported Outcome ◽  
Ceiling Effect ◽  
Mean Difference ◽  
Discussion Section ◽  
Power Calculations ◽  
Randomized Controlled ◽  
Patient Reported ◽  
The Mean

Abstract Background Randomized controlled trials in orthopaedics are powered to mainly find large effect sizes. A possible discrepancy between the estimated and the real mean difference is a challenge for statistical inference based on p-values. We explored the justifications of the mean difference estimates used in power calculations. The assessment of distribution of observations in the primary outcome and the possibility of ceiling effects were also assessed. Methods Systematic review of the randomized controlled trials with power calculations in eight clinical orthopaedic journals published between 2016 and 2019. Trials with one continuous primary outcome and 1:1 allocation were eligible. Rationales and references for the mean difference estimate were recorded from the Methods sections. The possibility of ceiling effect was addressed by the assessment of the weighted mean and standard deviation of the primary outcome and its elaboration in the Discussion section of each RCT where available. Results 264 trials were included in this study. Of these, 108 (41 %) trials provided some rationale or reference for the mean difference estimate. The most common rationales or references for the estimate of mean difference were minimal clinical important difference (16 %), observational studies on the same subject (8 %) and the ‘clinical relevance’ of the authors (6 %). In a third of the trials, the weighted mean plus 1 standard deviation of the primary outcome reached over the best value in the patient-reported outcome measure scale, indicating the possibility of ceiling effect in the outcome. Conclusions The chosen mean difference estimates in power calculations are rarely properly justified in orthopaedic trials. In general, trials with a patient-reported outcome measure as the primary outcome do not assess or report the possibility of the ceiling effect in the primary outcome or elaborate further in the Discussion section.

scholarly journals Clinical impact of left and right axis deviations with narrow QRS complex on 3-year outcomes in a hospital-based population in Japan

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Yuta Seko ◽  
Takao Kato ◽  
Yuhei Yamaji ◽  
Yoshisumi Haruna ◽  
Eisaku Nakane ◽  
...  
Keyword(s):  
Cardiovascular Events ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Conduction Block ◽  
Primary Outcome Measure ◽  
Excess Risk ◽  
Major Adverse Cardiovascular Events ◽  
Prognostic Impact ◽  
Axis Deviation ◽  
The Right

AbstractWhile the prognostic impact of QRS axis deviation has been assessed, it has never been investigated in patients without conduction block. Thus, we evaluated the prognostic impact of QRS-axis deviation in patients without conduction block. We retrospectively analyzed 3353 patients who had undergone both scheduled transthoracic echocardiography and electrocardiography in 2013 in a hospital-based population, after excluding patients with a QRS duration of ≥ 110 ms, pacemaker placement, and an QRS-axis − 90° to − 180° (northwest axis). The study population was categorized into three groups depending on the mean frontal plane QRS axis as follows: patients with left axis deviation (N = 171), those with right axis deviation (N = 94), and those with normal axis (N = 3088). The primary outcome was a composite of all-cause death and major adverse cardiovascular events. The cumulative 3-year incidence of the primary outcome measure was significantly higher in the left axis deviation group (26.4% in the left axis deviation, 22.7% in the right axis deviation, and 18.4% in the normal axis groups, log-rank P = 0.004). After adjusting for confounders, the excess risk of primary outcome measure remained significant in the left axis deviation group (hazard ratio [HR] 1.44; 95% confidence interval [CI] 1.07–1.95; P = 0.02), while the excess risk of primary outcome measure was not significant in the right axis deviation group (HR 1.22; 95% CI 0.76–1.96; P = 0.41). Left axis deviation was associated with a higher risk of a composite of all-cause death and major adverse cardiovascular events in hospital-based patients without conduction block in Japan.

scholarly journals Computerised cognitive–behavioural therapy for adults with intellectual disability: randomised controlled trial

2017 ◽  
Vol 211 (2) ◽  
pp. 95-102 ◽  
Author(s):  
Patricia Cooney ◽  
Catherine Jackman ◽  
David Coyle ◽  
Gary O'Reilly
Keyword(s):  
Intellectual Disability ◽  
Randomised Controlled Trial ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Controlled Trial ◽  
Behavioural Therapy ◽  
Primary Outcome Measure ◽  
Cognitive Behavioural ◽  
Randomised Controlled

BackgroundDespite the evidence base for computer-assisted cognitive–behavioural therapy (CBT) in the general population, it has not yet been adapted for use with adults who have an intellectual disability.AimsTo evaluate the utility of a CBT computer game for adults who have an intellectual disability.MethodA 2 × 3 (group × time) randomised controlled trial design was used. Fifty-two adults with mild to moderate intellectual disability and anxiety or depression were randomly allocated to two groups: computerised CBT (cCBT) or psychiatric treatment as usual (TAU), and assessed at pre-treatment, post-treatment and 3-month follow-up. Forty-nine participants were included in the final analysis.ResultsA significant group x time interaction was observed on the primary outcome measure of anxiety (Glasgow Anxiety Scale for people with an Intellectual Disability), favouring cCBT over TAU, but not on the primary outcome measure of depression (Glasgow Depression Scale for people with a Learning Disability). A medium effect size for anxiety symptoms was observed at post-treatment and a large effect size was observed after follow-up. Reliability of Change Indices indicated that the intervention produced clinically significant change in the cCBT group in comparison with TAU.ConclusionsAs the first application of cCBT for adults with intellectual disability, this intervention appears to be a useful treatment option to reduce anxiety symptoms in this population.

scholarly journals Telephone-guided self-help for mental health difficulties in neurological conditions: a randomised pilot trial

2021 ◽  
pp. archdischild-2019-318577
Author(s):  
Sophie D Bennett ◽  
Isobel Heyman ◽  
Anna E Coughtrey ◽  
Sophia Varadkar ◽  
Terence Stephenson ◽  
...  
Keyword(s):  
Mental Health ◽  
Young People ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Mental Health Problems ◽  
Pilot Trial ◽  
Primary Outcome Measure ◽  
Self Help ◽  
Mental Health Difficulties ◽  
Neurological Conditions

ObjectiveThis study aimed to conduct a randomised pilot trial to assess the feasibility of a randomised controlled trial (RCT) to investigate the effect of telephone-guided self-help for the treatment of mental health difficulties in children with neurological conditions.DesignPreliminary RCT. The primary outcome measure was the Strengths and Difficulties Questionnaire.SettingNeurology clinics in a national tertiary paediatric hospital.PatientsYoung people attending neurology clinics who met criteria for mental health difficulties according to the Development and Wellbeing Assessment.Interventions12 weeks of telephone-guided self-help based on a modular approach to psychological therapy for children delivered to children and/or their parents (n=17; eight males; mean age 12.04 years, SD=3.34) or a waiting list for telephone-guided self-help with no additional intervention over 12 weeks (n=17; nine males; mean age 10.53 years, SD=3.14).Results124 participants completed the DAWBA, and 34 children and young people were entered into the trial. 65% of those randomised to the intervention arm completed the full intervention, and the intervention was acceptable to those completing it. However, there were significant problems related to lack of data completion (38% data loss for primary outcome measure), choice of control comparator and outcome measures. Due to significant loss of data at follow-up, the effect size findings are considered unreliable.ConclusionsFurther feasibility work should be conducted to improve data completeness before progression to a definitive trial of guided self-help for mental health problems in children with neurological conditions can be recommended.Trial registration numberISRCTN21184717.

scholarly journals COMPARISON RESULTS OF SURGICAL TREATMENT LOCALIZED AND EXTENDED UTERINE CANCER

2010 ◽  
Vol 1 (3) ◽  
pp. 56-61
Author(s):  
I I Ushakov ◽  
E A Artoshina ◽  
P G Brousov ◽  
I V Nazvantsev ◽  
S A Levakov ◽  
...  
Keyword(s):  
Surgical Treatment ◽  
High Risk ◽  
Adjuvant Radiotherapy ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Uterine Cancer ◽  
Primary Outcome Measure ◽  
Pelvic Lymphadenectomy ◽  
Peritoneal Washing ◽  
Comparison Results

We selected 145 patients with endometrial carcinoma who had been treated with standard surgery (hysterectomy and BSO, peritoneal washing, palpation pelvic and para-aortic nodes) and with complete systematic pelvic lymphadenectomy (n=30 patients) or combined pelvic and para-aortic lymphadenectomy (n=30). Patients at intermediate or high risk of recurrence were offered adjuvant radiotherapy. The primary outcome measure was results of the surgical therapy.

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