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338 Diffusion Basis Spectrum Imaging (DBSI) Prognosticates Outcomes for Cervical Spondylotic Myelopathy after Surgery
- Justin Zhang, Saad Javeed, Jacob K. Greenberg, Dinal Jayasekera, Christopher F. Dibble, Jacob Blum, Rachel Jakes, Peng Sun, Sheng-Kwei Song, Wilson Z. Ray
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- Journal:
- Journal of Clinical and Translational Science / Volume 6 / Issue s1 / April 2022
- Published online by Cambridge University Press:
- 19 April 2022, p. 62
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OBJECTIVES/GOALS: Diffusion basis spectrum imaging (DBSI) allows for detailed evaluation of white matter microstructural changes present in cervical spondylotic myelopathy (CSM). Our goal is to utilize multidimensional clinical and quantitative imaging data to characterize disease severity and predict long-term outcomes in CSM patients undergoing surgery. METHODS/STUDY POPULATION: A single-center prospective cohort study enrolled fifty CSM patients who underwent surgical decompression and twenty healthy controls from 2018-2021. All patients underwent diffusion tensor imaging (DTI), DBSI, and complete clinical evaluations at baseline and 2-years follow-up. Primary outcome measures were the modified Japanese Orthopedic Association score (mild [mJOA 15-17], moderate [mJOA 12-14], severe [mJOA 0-11]) and SF-36 Physical and Mental Component Summaries (PCS and MCS). At 2-years follow-up, improvement was assessed via established MCID thresholds. A supervised machine learning classification model was used to predict treatment outcomes. The highest-performing algorithm was a linear support vector machine. Leave-one-out cross-validation was utilized to test model performance. RESULTS/ANTICIPATED RESULTS: A total of 70 patients – 20 controls, 25 mild, and 25 moderate/severe CSM patients – were enrolled. Baseline clinical and DTI/DBSI measures were significantly different between groups. DBSI Axial and Radial Diffusivity were significantly correlated with baseline mJOA and mJOA recovery, respectively (r=-0.33, p<0.01; r=-0.36, p=0.02). When predicting baseline disease severity (mJOA classification), DTI metrics alone performed with 38.7% accuracy (AUC: 72.2), compared to 95.2% accuracy (AUC: 98.9) with DBSI metrics alone. When predicting improvement after surgery (change in mJOA), clinical variables alone performed with 33.3% accuracy (AUC: 0.40). When combining DTI or DBSI parameters with key clinical covariates, model accuracy improved to 66.7% (AUC: 0.65) and 88.1% (AUC: 0.95) accuracy, respectively. DISCUSSION/SIGNIFICANCE: DBSI metrics correlate with baseline disease severity and outcome measures at 2-years follow-up. Our results suggest that DBSI may serve as a valid non-invasive imaging biomarker for CSM disease severity and potential for postoperative improvement.
VP157 What Is The Response To Immuno-Oncology By Health Technology Assessment Agencies?
- Judith Rubinstein, Boaz Adler, Jake Blumenthal, Ruth Chang, S. Yin Ho, Ashley Jaksa, Briana Lurie, Anson Pontynen, Emily Rubinstein, Rachel Sliman
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 33 / Issue S1 / 2017
- Published online by Cambridge University Press:
- 12 January 2018, pp. 220-221
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INTRODUCTION:
Immunotherapies are a relatively new innovative class of drug that have garnered excitement in the fight against cancer. In 2011, the immunotherapy drug, ipilimumab, was approved. Since then, four additional drugs have gained approval. This analysis evaluates the initial reception of immunotherapies by Health Technology Assessment (HTA) agencies.
METHODS:The Context Matters Data Model was used to evaluate the regulatory and HTA agency decisions surrounding the five approved immunotherapies through November 2016: atezolizumab, elotuzumab, ipilimumab, nivolumab, and pembrolizumab. Thirty-three labels from Australia, Canada, Europe, and the United States, and ninety-two assessments from Agenzia Italiana del Farmaco (AIFA), Gemeinsamer Bundesausschuss (Federal Joint Committee; G-BA), Haute Autorité de Santé (French National Authority for Health; HAS), Institute for Clinical and Economic Review (ICER), Institute for Quality and Efficiency in Health Care (IQWiG), National Institute for Health and Care Excellence (NICE), Pharmaceutical Benefits Advisory Committee (PBAC), pan-Canadian Oncology Drug Review (pCODR), and Scottish Medicines Consortium (SMC) were found. Using a sample t-test and a chi-squared test, reimbursement agencies’ decisions were evaluated, and the clinical and economic factors that went into these decisions were examined.
RESULTS:Of the evaluated reviews: sixty-four were for melanoma indications, fourteen were for non-small-cell lung cancer (NSCLC) indications, and seven were for kidney cancer indications. Many of the reviews did not reach any decision, but 75 percent of HTA decisions (n = 72; p = .0000) reached were positive. Elotuzumab, approved for multiple myeloma, received a positive decision from G-BA and a negative one from SMC. There was an association between different disease conditions or drugs and the rate of positive decisions.
For reviews that had clinical reasons for their decisions, 72.9 percent (n = 59; p = .0000) had positive clinical rationales that were associated with positive decisions (p = .000). Economic rationales for decisions were more mixed, with only 48.4 percent (n = 31; p = .0000) receiving positive decisions. Positive economic evaluations were also associated with positive decisions (p = .000). Atezolizumab, approved only in the United States at the time of this writing, has yet to be reviewed by any of the HTA agencies.
CONCLUSIONS:Immunotherapies are promising new options for the treatment of cancer. Thus far, reception by HTA agencies has generally been positive.
VP175 Validating Outcome Assessments For Health Technology Assessment In Ceroid Lipofuscinosis Neuronal 2 (CLN2), An Ultra-Rare Disease
- Rachel Ballinger, Samuel Llewellyn, Jake Macey, Lina Eliasson, Thomas Butt, Mohit Jain, Andrew Olaye, Andrea West
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- Journal:
- International Journal of Technology Assessment in Health Care / Volume 33 / Issue S1 / 2017
- Published online by Cambridge University Press:
- 12 January 2018, pp. 231-232
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INTRODUCTION:
Ceroid lipofuscinosis neuronal 2 (CLN2) disease, a form of Batten disease, is a rare, degenerative neurometabolic disorder. Disease onset around 2–4 years is followed by rapid decline in motor and neurologic function and mortality in early teenage years (1). Disease burden is best captured using observer-reported outcomes. However, validation is challenging in ultra-orphan diseases, requiring flexible methods and reasonable acceptance of limitations related to participant access.
The study aim was to assess content validation of clinical trial measures (i) CLN2 Disease Based Quality of Life Assessment (Sponsor-developed), (ii) EQ-5D-5L, (iii) Pediatric Quality of Life Inventory (PedsQL); and (iv) PedsQL Family Impact Module.
METHODS:The Batten Disease Family Association recruited United Kingdom caregivers of a child with CLN2 disease (aged 3–7 years, non-participants in any CLN2 trial), to:
1. Focus groups with symptom elicitation
2. Cognitive interviews to assess measures.
RESULTS:The Focus group comprised eleven caregivers (eight female, three male) from six families. Three families were current caregivers and remainders bereaved. Symptom and disease impact elicited showed the majority of measures domains were relevant.
The interview sample comprised sixteen current caregivers (twelve female, four male) from ten families (caring for eleven children). Overall measures were relevant, easy to understand and answer. However several items were difficult to apply to children with advanced disease (for example, Euroqol, EQ-5D-5L “overall health”), when ability is lost (for example, PedsQL walking), with misinterpretation of “no difficulties” with eating where child feeds using gastrostomy (CLN2 QoL). Caregivers found it difficult to know how their uncommunicative child was feeling (PedsQL worrying, EQ-5D-5L depression). Some symptoms and impacts were missing (for example, constipation, working life).
CONCLUSIONS:The mixed-methods approach enabled content validity assessment of multiple measures. While these measures were largely relevant, adjustments could strengthen these for use in this fatal pediatric condition population and increase their acceptance within health technology assessment (HTA).