The Autism Impact Measure (AIM): Meaningful Change Thresholds and Core Symptom Changes Over One Year from an Online Survey in the U.S.
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SUBMITTER: Silkey M
PROVIDER: S-EPMC10465376 | biostudies-literature | 2023 Sep
REPOSITORIES: biostudies-literature
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The Autism Impact Measure (AIM): Meaningful Change Thresholds and Core Symptom Changes Over One Year from an Online Survey in the U.S.
Silkey Mariabeth M Durán-Pacheco Gonzalo G Johnson Michelle M Liu Chuang C Clinch Susanne S Law Kiely K Loss Georg G
Journal of autism and developmental disorders 20220704 9
Validated outcome measures with the capacity to reflect meaningful change are key to assessing potential interventions for autism spectrum disorder (ASD). We derive clinically meaningful change thresholds (MCTs) of the Autism Impact Measure (AIM) and identify factors associated with meaningful change. Baseline and 12-months follow-up survey of caregivers of 2,761 children with ASD aged 3-17 years from the U.S. Simons Foundation Powering Autism Research for Knowledge (SPARK) cohort were analyzed. ...[more]
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Project description:The Autism impact measure (AIM) is a caregiver-reported questionnaire assessing autism symptom frequency and impact in children, previously shown to have good test-retest reliability, convergent validity and structural validity. This study extended previous work by exploring the AIM's ability to discriminate between 'known-groups' of children, and estimating thresholds for clinically important responses. Data were collected online and electronically on computer and mobile devices; hence, it was also possible to confirm other psychometric properties of the AIM in this format. This study provides confirmatory and additional psychometric validation of the AIM. The AIM offers a valid, quick and inexpensive method for caregivers to report core symptoms of autism spectrum disorder (ASD) including communication deficits, difficulties with social interactions and repetitive behaviors.
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Project description:The Autism Impact Measure (AIM) was designed specifically for treatment-outcome assessment in children with ASD, focusing on treatment-relevant aspects of symptom presentation and efficient detection of short-term improvement. The AIM demonstrated strong reliability and validity in initial psychometric studies. The current study evaluated the AIM's sensitivity to change across well-established treatments. The sample included 471 children with ASD (ages 2-14) participating in one of six treatments. The AIM was administered at baseline and 6-week intervals and a battery of domain-specific concurrent measures was also administered. A longitudinal repeated measures design examined the degree to which: (a) AIM domain scores changed over time in response to treatment and (b) change in AIM domains was associated with change in measures of similar constructs. Results across growth curve models indicated that AIM domains are sensitive to change in symptoms across treatment. Across all models, symptoms decreased over time, with some deceleration in rate of improvement. For all AIM domains except Repetitive Behavior, symptoms improved as a function of treatment group. Correlations of change between AIM and other measures varied across domains (from 0.01-0.43 across measures). This was the first large-scale study to systematically evaluate sensitivity to change in a measure of core ASD symptoms. The results provide support for the AIM's ability to detect short-term improvement across symptom domains and indicate that AIM domains are sensitive to change overall and as a function of different treatment conditions. The brief repeated assessment window also highlights the AIM's utility for detecting improvements across short-term treatments. Autism Res 2020, 13: 1867-1879. © 2020 International Society for Autism Research and Wiley Periodicals LLC LAY SUMMARY: Good measures are important for assessing outcomes in children with autism. However, there are few tools for tracking short-term changes in autism symptoms. This study tested a new measure, the Autism Impact Measure (AIM), in a large group of children with autism. The results showed that the AIM appears to be a valid and accurate tool for measuring autism symptoms. The AIM may be a helpful tool for researchers and clinicians interested in tracking short-term improvements in autism symptoms.
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Project description:ObjectivesThe Nasal Polyposis Symptom Diary (NPSD) is a novel and short patient-reported outcome (PRO) tool specifically developed to assess important and relevant symptoms reported by patients with chronic rhinosinusitis with nasal polyposis (CRSwNP). We evaluated the psychometric properties of 4 predefined NPSD-derived scores intended to support symptom-improvement assessments of investigational therapies for inclusion in product labeling.MethodsFive hundred eighteen patients with severe CRSwNP from a Phase III clinical trial (NCT03401229) completed the NPSD, comprising 11 items: 8 symptom-specific, 2 symptom-impact, and 1 optional medication-compliance. The psychometric characteristics of 3 single-item symptom scores (Nasal Blockage Score [NBS], Nasal Congestion Score [NCS], and Difficulty with Sense of Smell Score [DSS]) and a Total Symptom Score (TSS, summary of the 8 symptom-specific items) were evaluated for reliability, validity, and ability to detect change. Within-patient meaningful change thresholds (MCTs) were established using anchor- and distribution-based methods. Comparative PROs included the 22-item Sino-Nasal Outcome Test (SNOT-22) and Patient Global Impression of Severity (PGI-S).ResultsThe TSS exhibited strong internal consistency (Cronbach α = .88) and test-retest reliability (intraclass correlation coefficient >.80). Correlation between the TSS and SNOT-22 total score indicated good convergent validity (r = .70). All 4 NPSD scores demonstrated known-groups validity (significant differences among subgroups of patients with predetermined disease severity levels based on PGI-S categories) and were sensitive to detect change in patients' clinical status (significant differences among subgroups of patients with reported changes between 2 time-points in PGI-S and Patient Global Impression of Change scores). MCTs for improvement were established at 1.0 point for NBS, NCS, and DSS, and 4.0 points for TSS.ConclusionThese findings support the reliability, validity, and suitability of the 4 NPSD-derived scores for evaluating treatment effect on CRSwNP symptoms and their use in clinical trials with predetermined MCTs for improvement.
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Project description:Autism spectrum disorder (ASD) is a heterogeneous disease that is characterized by abnormalities in social communication and interaction as well as repetitive behaviors and restricted interests. Structural brain imaging has identified significant cortical folding alterations in ASD; however, relatively less known is whether the core symptoms are related to neuroanatomical differences. In this study, we aimed to explore core-symptom-anchored gyrification alterations and their developmental trajectories in ASD. We measured the cortical vertex-wise gyrification index (GI) in 321 patients with ASD (aged 7-39 years) and 350 typically developing (TD) subjects (aged 6-33 years) across 8 sites from the Autism Brain Imaging Data Exchange I (ABIDE I) repository and a longitudinal sample (14 ASD and 7 TD, aged 9-14 years in baseline and 12-18 years in follow-up) from ABIDE II. Compared with TD, the general ASD patients exhibited a mixed pattern of both hypo- and hyper- and different developmental trajectories of gyrification. By parsing the ASD patients into three subgroups based on the subscores of the Autism Diagnostic Interview-Revised (ADI-R) scale, we identified core-symptom-specific alterations in the reciprocal social interaction (RSI), communication abnormalities (CA), and restricted, repetitive, and stereotyped patterns of behavior (RRSB) subgroups. We also showed atypical gyrification patterns and developmental trajectories in the subgroups. Furthermore, we conducted a meta-analysis to locate the core-symptom-anchored brain regions (circuits). In summary, the current study shows that ASD is associated with abnormal cortical folding patterns. Core-symptom-based classification can find more subtle changes in gyrification. These results suggest that cortical folding pattern encodes changes in symptom dimensions, which promotes the understanding of neuroanatomical basis, and clinical utility in ASD.
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Project description:A dataset range of isocenter congruency verification tests have been examined from a statistical perspective for the purpose of establishing tolerance levels that are meaningful, based on the fundamental limitation of linear accelerator isocentricity and the demands of a high-precision stereotactic radiosurgery program. Using a laser-defined isocenter, a total of 149 individual isocenter congruency tests were examined with recorded values for ideal spatial corrections to the isocenter test tool. These spatial corrections were determined from radiation exposures recorded on an electronic portal imaging device (EPID) at various gantry, collimator, and treatment couch combinations. The limitations of establishing an ideal isocenter were quantified from each variable which contributed to uncertainty in isocenter definition. Individual contributors to uncertainty, specifically, daily positioning setup errors, gantry sag, multileaf collimator (MLC) offset, and couch walkout, were isolated from isocenter congruency measurements to determine a clinically meaningful isocenter measurement. Variations in positioning of the test tool constituted, on average, 0.38 mm magnitude of correction. Gantry sag and MLC offset contributed 0.4 and 0.16 mm, respectively. Couch walkout had an average degrading effect to isocenter of 0.72 mm. Considering the magnitude of uncertainty contributed by each uncertainty variable and the nature of their combination, an appropriate schedule action and immediate action level were determined for use in analyzing daily isocenter congruency test results in a stereotactic radiosurgery (SRS) program. The recommendations of this study for this linear accelerator include a schedule action level of 1.25 mm and an immediate action level of 1.50mm, requiring prompt correction response from clinical medical physicists before SRS or stereotactic body radiosurgery (SBRT) is administered. These absolute values were derived from considering relative data from a specific linear accelerator and, therefore, represent a means by which a numerical quantity can be used as a test threshold with relative specificity to a particular linear accelerator.
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Project description:Study objectivesWhile caregiver-reported sleep disturbances are common in children and adolescents with autism spectrum disorder (['), few studies have measured objective sleep in ASD compared to controls, and their findings are mixed. We investigated (1) differences in sleep architecture, specifically slow-wave sleep (SWS) and rapid eye movement (REM) sleep, between ASD and typically developing controls (TD); and (2) if any observed differences in sleep were associated with core ASD symptoms.MethodsWe used ambulatory polysomnography (PSG) in 53 participants with ASD (ages 4-18) and 66 age-matched TD in their home sleeping environment. The primary outcome measures were SWS and REM sleep. Core behavioral ASD symptoms were assessed using the Autism Diagnostic Interview-Revised (ADI-R). Spectral power bands during sleep, and additional behavioral measures, were examined in exploratory analyses.ResultsCompared to TD, participants with ASD exhibited a higher SWS ratio and lower REM sleep ratio. Within the ASD group, higher SWS was associated with more severe symptoms on the Restricted, Repetitive, and Stereotyped Behaviors subscale of the ADI-R. No association was observed between REM sleep ratio and any ASD symptom.ConclusionsIncreased SWS and reduced REM sleep ratio differentiated ASD from TD. However, only increased SWS was associated with more severe core ASD symptoms. Increased SWS may reflect neuronal immaturity specific to ASD in this age group. These findings may inform the underlying mechanisms of clinical symptoms observed in children and adolescents with ASD.
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Project description:IntroductionThe NSCLC Symptom Assessment Questionnaire (NSCLC-SAQ) was developed to assess NSCLC symptom severity in accordance with Food and Drug Administration evidentiary expectations leading to Food and Drug Administration qualification in 2018. This study evaluated the NSCLC-SAQ's measurement properties within a clinical trial.MethodsThe KEYNOTE-598 phase 3 study of participants with stage IV metastatic NSCLC with programmed death-ligand 1 tumor proportion score greater than or equal to 50% was used to assess the NSCLC-SAQ's reliability, construct validity, responsiveness, and estimate clinically meaningful within-person change. Other patient-reported outcome measures included patient global impression items of severity and change in lung cancer symptoms, and the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire core 30 and lung cancer module, LC13.ResultsParticipants (N = 560) were mostly men (70%), had a mean age of 64 years, and had Eastern Cooperative Oncology Group performance status of 1 (64%) or 0 (36%). Internal consistency at baseline (Cronbach's α = 0.74) and test-retest reliability after 3 weeks (intraclass correlation coefficient = 0.79) were satisfactory. NSCLC-SAQ items, domains, and total score correlated moderately to highly with patient-reported outcome measures capturing similar content, and the total score differentiated among patient global impression of severity groups (p < 0.001). The total score detected improvement over time and the estimated clinically meaningful within-person change threshold for improvement ranged from three to five points on the 0 to 20 scale. Few participants exhibited symptom worsening (n = 38), limiting inferences in this group.ConclusionsThe NSCLC-SAQ was found to be reliable, valid, responsive, and interpretable for assessing symptom improvement in NSCLC. Further evaluation is recommended in trial participants whose symptoms worsen over time.
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Project description:Objectives/hypothesisDupilumab, a fully human monoclonal antibody that blocks the shared interleukin (IL)-4/IL-13 receptor component, significantly improved outcomes for patients with chronic rhinosinusitis with nasal polyps (CRSwNP) in the SINUS-24 and SINUS-52 studies. This post hoc analysis evaluated dupilumab's effect on patient-reported symptoms and objective outcome measures using thresholds of clinically meaningful within-patient change from baseline.MethodsPatients with CRSwNP receiving subcutaneous dupilumab or placebo every 2 weeks in SINUS-24/SINUS-52 were analyzed. Patients recorded severity of nasal congestion (NC), loss of smell (LoS), and anterior/posterior rhinorrhea (each within range 0-3) daily. Total Symptom Score (TSS) was calculated as a composite severity score (0-9) for these symptoms. Objective measures included University of Pennsylvania Smell Identification Test (UPSIT; 0-40), nasal polyps score (NPS; 0-8), and Lund-Mackay computed tomography score (LMK-CT; 0-24). Thresholds of within-patient change in scores from baseline at weeks 24 and 52 considered clinically meaningful were ≥1.0 (NC, LoS), ≥3.0 (TSS), ≥8.0 (UPSIT), ≥1.0 (NPS), and ≥5.0 (LMK-CT).ResultsA total of 724 and 303 patients were included in the week 24 and 52 analyses, respectively. Responder rates were significantly higher with dupilumab versus placebo at week 24 for NC (64% vs. 24%), LoS (63% vs. 14%), TSS (62% vs. 15%), UPSIT (54% vs. 6%), NPS (63% vs. 14%), and LMK-CT (59% vs. 3%); all P < .0001. Results were consistent at week 52.ConclusionSignificantly greater proportions of dupilumab-treated patients with CRSwNP compared with placebo demonstrated clinically meaningful improvements in patient-reported sinonasal symptoms and objective outcomes.Level of evidence2 Laryngoscope, 132:259-264, 2022.
| S-EPMC9299704 | biostudies-literature
Project description:PurposeMeaningful thresholds are needed to interpret patient-reported outcome measure (PROM) results. This paper introduces a new method, based on item response theory (IRT), to estimate such thresholds. The performance of the method is examined in simulated datasets and two real datasets, and compared with other methods.MethodsThe IRT method involves fitting an IRT model to the PROM items and an anchor item indicating the criterion state of interest. The difficulty parameter of the anchor item represents the meaningful threshold on the latent trait. The latent threshold is then linked to the corresponding expected PROM score. We simulated 4500 item response datasets to a 10-item PROM, and an anchor item. The datasets varied with respect to the mean and standard deviation of the latent trait, and the reliability of the anchor item. The real datasets consisted of a depression scale with a clinical depression diagnosis as anchor variable and a pain scale with a patient acceptable symptom state (PASS) question as anchor variable.ResultsThe new IRT method recovered the true thresholds accurately across the simulated datasets. The other methods, except one, produced biased threshold estimates if the state prevalence was smaller or greater than 0.5. The adjusted predictive modeling method matched the new IRT method (also in the real datasets) but showed some residual bias if the prevalence was smaller than 0.3 or greater than 0.7.ConclusionsThe new IRT method perfectly recovers meaningful (interpretational) thresholds for multi-item questionnaires, provided that the data satisfy the assumptions for IRT analysis.
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Project description:ImportanceDefining meaningful improvement using the Total Vitiligo Area Scoring Index (T-VASI) and the Facial VASI (F-VASI) aids interpretation of findings from clinical trials evaluating vitiligo treatments; however, clear and clinically meaningful thresholds have not yet been established.ObjectiveTo assess concept validity and measurement performance of the T-VASI and F-VASI in patients with nonsegmental vitiligo and to identify meaningful change thresholds.Design, settings, and participantsThis mixed-methods study consisted of a secondary analysis of a phase 2 multicenter double-blind dose-ranging randomized clinical trial and embedded qualitative interviews conducted at 35 sites in Canada, France, Japan, and the US. The secondary analysis included the trial's adult patients with nonsegmental vitiligo (T-VASI ≥5 and F-VASI ≥0.5 at baseline). Psychometric performance of the T-VASI and F-VASI and thresholds for meaningful change were evaluated using clinician- and patient-reported information. The trial's embedded interviews were used to qualitatively assess content validity and patient perceptions of meaningful repigmentation. Data analyses were performed from March to July 2023.InterventionParticipants were randomized to 6-, 11-, or 22-mg/day upadacitinib or placebo for 24 weeks.Main outcomes and measuresPsychometric performance of the T-VASI and F-VASI and thresholds for meaningful changed plus content validity and patient perceptions of meaningful repigmentation. Measurement instruments included the T-VASI, F-VASI, Vitiligo Noticeability Scale, Total-Patient Global Vitiligo Assessment, Face-Patient Global Vitiligo Assessment, Total-Physician Global Vitiligo Assessment (PhGVA-T), Face-Physician Global Vitiligo Assessment (PhGVA-F), Patient's Global Impression of Change-Vitiligo, Physician's Global Impression of Change-Vitiligo (PhGIC-V), Vitiligo Quality-of-Life Instrument, Dermatology Life Quality Index, the Hospital Anxiety and Depression Scale, and transcribed verbatim interviews with patients.ResultsThe psychometric analysis included 164 participants (mean [SD] age, 46 years; 103 [63%] females) and the qualitative analysis included 14 participants (mean [SD] age, 48.8 [12.2] years; 9 females [64%] and 5 males [36%]). Intraclass correlation coefficients were 0.98 for T-VASI and 0.99 for F-VASI in patients with clinically stable vitiligo between baseline and week 4, supporting test-retest reliability. At baseline and week 24, correlations were moderate to strong between T-VASI and PhGVA-T (r = 0.63-0.65) and between F-VASI and PhGVA-F (r = 0.65-0.71). Average baseline and week-24 VASI scores decreased with repigmentation (ie, increasing PhGVA scores). Least-square mean VASI scores increased with greater repigmentation as measured by the PhGIC-V. Least-square mean VASI scores also differed between patients with improved PhGIC-V and those with no change or worsened V-PhGIC scores. Using a multiple anchor approach, improvements of 30% in T-VASI and 50% in F-VASI scores reflected meaningful repigmentation between baseline and week 24.Conclusion and relevanceThis mixed-methods study found that the T-VASI and F-VASI are reliable, valid, able to differentiate between clinically distinct groups, and responsive in patients with nonsegmental vitiligo. The thresholds for meaningful change were lower than those historically used in clinical trials, suggesting that T-VASI 50 and F-VASI 75 are conservative estimates and reflect improvements that would be meaningful in patients with nonsegmental vitiligo.Trial registrationClinicalTrials.gov Identifier: NCT04927975.
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