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  • 1
    Online Resource
    Online Resource
    Field methods for sampling tree height for tropical forest biomass estimation
    Wiley ; 2018
    In:  Methods in Ecology and Evolution Vol. 9, No. 5 ( 2018-05), p. 1179-1189
    Details
    In: Methods in Ecology and Evolution, Wiley, Vol. 9, No. 5 ( 2018-05), p. 1179-1189
    Abstract: Quantifying the relationship between tree diameter and height is a key component of efforts to estimate biomass and carbon stocks in tropical forests. Although substantial site‐to‐site variation in height–diameter allometries has been documented, the time consuming nature of measuring all tree heights in an inventory plot means that most studies do not include height, or else use generic pan‐tropical or regional allometric equations to estimate height. Using a pan‐tropical dataset of 73 plots where at least 150 trees had in‐field ground‐based height measurements, we examined how the number of trees sampled affects the performance of locally derived height–diameter allometries, and evaluated the performance of different methods for sampling trees for height measurement. Using cross‐validation, we found that allometries constructed with just 20 locally measured values could often predict tree height with lower error than regional or climate‐based allometries (mean reduction in prediction error = 0.46 m). The predictive performance of locally derived allometries improved with sample size, but with diminishing returns in performance gains when more than 40 trees were sampled. Estimates of stand‐level biomass produced using local allometries to estimate tree height show no over‐ or under‐estimation bias when compared with biomass estimates using field measured heights. We evaluated five strategies to sample trees for height measurement, and found that sampling strategies that included measuring the heights of the ten largest diameter trees in a plot outperformed (in terms of resulting in local height–diameter models with low height prediction error) entirely random or diameter size‐class stratified approaches. Our results indicate that even limited sampling of heights can be used to refine height–diameter allometries. We recommend aiming for a conservative threshold of sampling 50 trees per location for height measurement, and including the ten trees with the largest diameter in this sample.
    Type of Medium: Online Resource
    ISSN: 2041-210X , 2041-210X
    URL: Issue
    URL: Article
    DOI: 10.1111/mee3.2018.9.issue-5
    DOI: 10.1111/2041-210X.12962
    Language: English
    Publisher: Wiley
    Publication Date: 2018
    detail.hit.zdb_id: 2528492-7
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  • 2
    Online Resource
    Online Resource
    Comparative Treatment Outcomes for Idiopathic Subglottic Stenosis: 5‐Year Update
    Wiley ; 2023
    In:  Otolaryngology–Head and Neck Surgery Vol. 168, No. 6 ( 2023-06), p. 1570-1575
    Details
    In: Otolaryngology–Head and Neck Surgery, Wiley, Vol. 168, No. 6 ( 2023-06), p. 1570-1575
    Abstract: The North American Airway Collaborative (NoAAC) previously published a 3‐year multi‐institutional prospective cohort study showing variation in treatment effectiveness between 3 primary surgical techniques for idiopathic subglottic stenosis (iSGS). In this report, we update these findings to include 5 years of data evaluating treatment effectiveness. Patients in the NoAAC cohort were re‐enrolled for 2 additional years and followed using the prespecified published protocol. Consistent with prior data, prospective observation of 487 iSGS patients for 5 years showed treatment effectiveness differed by modality. Cricotracheal resection maintained the lowest rate of recurrent operation (5%), followed by endoscopic resection with adjuvant medical therapy (30%) and endoscopic dilation (50%). These data support the initial observations and continue to provide value to providers and patients navigating longitudinal decision‐making. Level of evidence: 2—prospective cohort study.
    Type of Medium: Online Resource
    ISSN: 0194-5998 , 1097-6817
    URL: Issue
    URL: Article
    DOI: 10.1002/ohn.v168.6
    DOI: 10.1002/ohn.190
    Language: English
    Publisher: Wiley
    Publication Date: 2023
    detail.hit.zdb_id: 2008453-5
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  • 3
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    Cognitive training and remediation interventions for substance use disorders: a Delphi consensus study
    Wiley ; 2023
    In:  Addiction Vol. 118, No. 5 ( 2023-05), p. 935-951
    Details
    In: Addiction, Wiley, Vol. 118, No. 5 ( 2023-05), p. 935-951
    Abstract: Substance use disorders (SUD) are associated with cognitive deficits that are not always addressed in current treatments, and this hampers recovery. Cognitive training and remediation interventions are well suited to fill the gap for managing cognitive deficits in SUD. We aimed to reach consensus on recommendations for developing and applying these interventions. Design, Setting and Participants We used a Delphi approach with two sequential phases: survey development and iterative surveying of experts. This was an on‐line study. During survey development, we engaged a group of 15 experts from a working group of the International Society of Addiction Medicine (Steering Committee). During the surveying process, we engaged a larger pool of experts ( n  = 54) identified via recommendations from the Steering Committee and a systematic review. Measurements Survey with 67 items covering four key areas of intervention development: targets, intervention approaches, active ingredients and modes of delivery. Findings Across two iterative rounds (98% retention rate), the experts reached a consensus on 50 items including: (i) implicit biases, positive affect, arousal, executive functions and social processing as key targets of interventions; (ii) cognitive bias modification, contingency management, emotion regulation training and cognitive remediation as preferred approaches; (iii) practice, feedback, difficulty‐titration, bias modification, goal‐setting, strategy learning and meta‐awareness as active ingredients; and (iv) both addiction treatment work‐force and specialized neuropsychologists facilitating delivery, together with novel digital‐based delivery modalities. Conclusions Expert recommendations on cognitive training and remediation for substance use disorders highlight the relevance of targeting implicit biases, reward, emotion regulation and higher‐order cognitive skills via well‐validated intervention approaches qualified with mechanistic techniques and flexible delivery options.
    Type of Medium: Online Resource
    ISSN: 0965-2140 , 1360-0443
    URL: Issue
    URL: Article
    DOI: 10.1111/add.v118.5
    DOI: 10.1111/add.16109
    Language: English
    Publisher: Wiley
    Publication Date: 2023
    detail.hit.zdb_id: 2002997-4
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  • 4
    Online Resource
    Online Resource
    Impact of Diagnosis and Therapy on Cognitive Function in Urea Cycle Disorders
    Wiley ; 2019
    In:  Annals of Neurology Vol. 86, No. 1 ( 2019-07), p. 116-128
    Details
    In: Annals of Neurology, Wiley, Vol. 86, No. 1 ( 2019-07), p. 116-128
    Abstract: Individuals with urea cycle disorders (UCDs) often present with intellectual and developmental disabilities. The major aim of this study was to evaluate the impact of diagnostic and therapeutic interventions on cognitive outcomes in UCDs. Methods This prospective, observational, multicenter study includes data from 503 individuals with UCDs who had comprehensive neurocognitive testing with a cumulative follow‐up of 702 patient‐years. Results The mean cognitive standard deviation score (cSDS) was lower in symptomatic than in asymptomatic ( p 〈 0.001, t test) individuals with UCDs. Intellectual disability (intellectual quotient 〈 70, cSDS 〈 −2.0) was associated with the respective subtype of UCD and early disease onset, whereas height of the initial peak plasma ammonium concentration was inversely associated with neurocognitive outcomes in mitochondrial (proximal) rather than cytosolic (distal) UCDs. In ornithine transcarbamylase and argininosuccinate synthetase 1 deficiencies, we did not find evidence that monoscavenger therapy with sodium or glycerol phenylbutyrate was superior to sodium benzoate in providing cognitive protection. Early liver transplantation appears to be beneficial for UCDs. It is noteworthy that individuals with argininosuccinate synthetase 1 and argininosuccinate lyase deficiencies identified by newborn screening had better neurocognitive outcomes than those diagnosed after the manifestation of first symptoms. Interpretation Cognitive function is related to interventional and non‐interventional variables. Early detection by newborn screening and early liver transplantation appear to offer greater cognitive protection, but none of the currently used nitrogen scavengers was superior with regard to long‐term neurocognitive outcome. Further confirmation could determine these variables as important clinical indicators of neuroprotection for individuals with UCDs. ANN NEUROL 2019
    Type of Medium: Online Resource
    ISSN: 0364-5134 , 1531-8249
    URL: Issue
    URL: Article
    DOI: 10.1002/ana.v86.1
    DOI: 10.1002/ana.25492
    Language: English
    Publisher: Wiley
    Publication Date: 2019
    detail.hit.zdb_id: 2037912-2
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  • 5
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    Online Resource
    Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy
    Wiley ; 2019
    In:  Pediatric Pulmonology Vol. 54, No. 2 ( 2019-02), p. 179-185
    Details
    In: Pediatric Pulmonology, Wiley, Vol. 54, No. 2 ( 2019-02), p. 179-185
    Abstract: Spinal Muscular Atrophy type 1 (SMA1) is a rare genetic neuromuscular disease where 75% of SMA1 patients die/require permanent‐ventilation by 13.6 months. This study assessed the health outcomes of SMA1 infants treated with AVXS‐101 gene replacement therapy. Methods Twelve genetically confirmed SMA1 infants with homozygous deletions of the SMN1 gene and two SMN2 gene copies received a one‐time intravenous proposed therapeutic dose of AVXS‐101 in an open label study conducted between December 2014 and 2017. Patients were followed for 2‐years post‐treatment for outcomes including (1) pulmonary interventions; (2) nutritional interventions; (3) swallow function; (4) hospitalization rates; and (5) motor function. Results All 12 patients completed the study. Seven infants did not require noninvasive ventilation (NIV) by study completion. Eleven patients had stable or improved swallow function, demonstrated by the ability to feed orally; 11 patients were able to speak. The mean proportion of time hospitalized was 4.4%; the mean unadjusted annualized hospitalization rate was 2.1 (range = 0, 7.6), with a mean length of stay/hospitalization of 6.7 (range = 3, 12.1) days. Eleven patients achieved full head control and sitting unassisted and two patients were walking independently. Conclusions AVXS‐101 treatment in SMA1 was associated with reduced pulmonary and nutritional support requirements, improved motor function, and decreased hospitalization rate over the follow‐up period. This contrasts with the natural history of progressive respiratory failure and reduced survival. The reduced healthcare utilization could potentially alleviate patient and caregiver burden, suggesting an overall improved quality of life following gene replacement therapy. Trial registration ClinicalTrials.gov number, NCT02122952.
    Type of Medium: Online Resource
    ISSN: 8755-6863 , 1099-0496
    URL: Issue
    URL: Article
    DOI: 10.1002/ppul.v54.2
    DOI: 10.1002/ppul.24203
    Language: English
    Publisher: Wiley
    Publication Date: 2019
    detail.hit.zdb_id: 1491904-7
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  • 6
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    Online Resource
    Longitudinal effect of eteplirsen versus historical control on ambulation in D uchenne muscular dystrophy
    Wiley ; 2016
    In:  Annals of Neurology Vol. 79, No. 2 ( 2016-02), p. 257-271
    Details
    In: Annals of Neurology, Wiley, Vol. 79, No. 2 ( 2016-02), p. 257-271
    Abstract: To continue evaluation of the long‐term efficacy and safety of eteplirsen, a phosphorodiamidate morpholino oligomer designed to skip DMD exon 51 in patients with Duchenne muscular dystrophy (DMD). Three‐year progression of eteplirsen‐treated patients was compared to matched historical controls (HC). Methods Ambulatory DMD patients who were ≥7 years old and amenable to exon 51 skipping were randomized to eteplirsen (30/50mg/kg) or placebo for 24 weeks. Thereafter, all received eteplirsen on an open‐label basis. The primary functional assessment in this study was the 6‐Minute Walk Test (6MWT). Respiratory muscle function was assessed by pulmonary function testing (PFT). Longitudinal natural history data were used for comparative analysis of 6MWT performance at baseline and months 12, 24, and 36. Patients were matched to the eteplirsen group based on age, corticosteroid use, and genotype. Results At 36 months, eteplirsen‐treated patients (n = 12) demonstrated a statistically significant advantage of 151m ( p 〈 0.01) on 6MWT and experienced a lower incidence of loss of ambulation in comparison to matched HC (n = 13) amenable to exon 51 skipping. PFT results remained relatively stable in eteplirsen‐treated patients. Eteplirsen was well tolerated. Analysis of HC confirmed the previously observed change in disease trajectory at age 7 years, and more severe progression was observed in patients with mutations amenable to exon skipping than in those not amenable. The subset of patients amenable to exon 51 skipping showed a more severe disease course than those amenable to any exon skipping. Interpretation Over 3 years of follow‐up, eteplirsen‐treated patients showed a slower rate of decline in ambulation assessed by 6MWT compared to untreated matched HC. Ann Neurol 2016;79:257–271
    Type of Medium: Online Resource
    ISSN: 0364-5134 , 1531-8249
    URL: Issue
    URL: Article
    DOI: 10.1002/ana.v79.2
    DOI: 10.1002/ana.24555
    Language: English
    Publisher: Wiley
    Publication Date: 2016
    detail.hit.zdb_id: 2037912-2
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  • 7
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    Online Resource
    Reliability and validity of active‐seated: An outcome in dystrophinopathy
    Wiley ; 2015
    In:  Muscle & Nerve Vol. 52, No. 3 ( 2015-09), p. 356-362
    Details
    In: Muscle & Nerve, Wiley, Vol. 52, No. 3 ( 2015-09), p. 356-362
    Abstract: Traditional upper extremity measures typically focus on distal abilities and do not quantify the unique progression of decline in dystrophinopathy. We designed ACTIVE‐seated to meet this need. Our objective was to establish the tool's validity and reliability. Methods ACTIVE‐seated uses the Microsoft Kinect gaming interface to quantify functional reaching ability while playing a custom‐designed game. A skeletal tracking algorithm was used to determine the furthest arm excursion in all planes in 61 subjects with dystrophinopathy and 16 controls. Results Total reachable area was scaled based on arm length to standardize comparisons across subjects and accommodate growth. ACTIVE‐seated discriminately ranked subjects from normal controls and by Brooke level ( P   〈  0.001). Scores were highly correlated with parent reports of daily activities and mobility ( P   〈  0.05). Test–retest reliability of ACTIVE‐seated was excellent (ICC = 0.97, P   〈  0.0001). Conclusions Initial evaluation of reliability and validity suggests that ACTIVE‐seated shows promise as a clinical and research outcome for individuals with dystrophinopathy. Muscle Nerve 52:356–362, 2015
    Type of Medium: Online Resource
    ISSN: 0148-639X , 1097-4598
    URL: Issue
    URL: Article
    DOI: 10.1002/mus.v52.3
    DOI: 10.1002/mus.24557
    RVK:
    XA 10000
    Language: English
    Publisher: Wiley
    Publication Date: 2015
    detail.hit.zdb_id: 1476641-3
    SSG: 12
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  • 8
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    Multicenter expanded access program for access to investigational products for amyotrophic lateral sclerosis
    Wiley ; 2024
    In:  Muscle & Nerve
    Details
    In: Muscle & Nerve, Wiley
    Abstract: Expanded access (EA) is a Food and Drug Administration‐regulated pathway to provide access to investigational products (IPs) to individuals with serious diseases who are ineligible for clinical trials. The aim of this report is to share the design and operations of a multicenter, multidrug EA program for amyotrophic lateral sclerosis (ALS) across nine US centers. Methods A central coordination center was established to design and conduct the program. Templated documents and processes were developed to streamline study design, regulatory submissions, and clinical operations across protocols. The program included three protocols and provided access to IPs that were being tested in respective regimens of the HEALEY ALS Platform Trial (verdiperstat, CNM‐Au8, and pridopidine). Clinical and safety data were collected in all EA protocols (EAPs). The program cohorts comprised participants who were not eligible for the platform trial, including participants at advanced stages of disease progression and with long disease duration. Results A total of 85 participants were screened across the 3 EAPs from July 2021 to September 2022. The screen failure rate was 3.5%. Enrollment for the regimens of the platform trial was completed as planned and results informed the duration of the corresponding EAP. The verdiperstat EAP was concluded in December 2022. Mean duration of participation in the verdiperstat EAP was 5.8 ± 4.1 months. The CNM‐Au8 and pridopidine EAPs are ongoing. Discussion Multicenter EAPs conducted in parallel to randomized clinical trials for ALS can successfully enroll participants who do not qualify for clinical trials.
    Type of Medium: Online Resource
    ISSN: 0148-639X , 1097-4598
    URL: Article
    DOI: 10.1002/mus.28169
    RVK:
    XA 10000
    Language: English
    Publisher: Wiley
    Publication Date: 2024
    detail.hit.zdb_id: 1476641-3
    SSG: 12
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