Mapping the Modified Ashworth Scale and Physician’s Global Assessment to Preference-Based Health Utilities in Adults with Lower Limb Spasticity

VA L U E I N H E A LT H

relevant PROMs were selected for further evaluation (4 MS-specific, 1 generic). The measures reviewed were: MS International Quality of Life questionnaire (MusiQoL), MS Quality of Life Instrument (MSQOL-54), MS Impact Scale (MSIS-29), MS Walking Scale (MSWS-12), and the PROMIS item banks. While some of the identified measures have shown responsiveness in clinical trials, none meets all FDA requirements on PROMs.  Conclusions: Based on the preliminary conceptual model, physical functioning aspects of MS are proximal to the disease and thus a potential target for future PRO labeling claims. No existing PRO measure appears likely to support an FDA labeling claim in their current format. The next phase of the project is the conduct of clinician interviews and concept elicitation focus groups to further revise and refine the conceptual model. PND54 Pro Labeling in Products Approved by the European Medicines Agency (EMA) for Narcolepsy Perrier L1, Jurado D1, Anfray C1, Acquadro C2 1Mapi Research Trust, Lyon, France, 2Mapi, Lyon, France

Objectives: Narcolepsy is a rare neurological disease, which prevalence is estimated between 1/3,300 and 1/5,000 (Orphanet), characterized by excessive daytime sleepiness (EDS), sleep paralysis, and hypnagogic hallucinations. Additionally, narcolepsy can occur with cataplexy, a sudden loss of muscle tone triggered by positive or negative emotions, in 60-70% of narcoleptic patients. The objectives of this study were to identify 1) the medicines approved for the treatment of narcolepsy by the European Medicines Agency (EMA); 2) which of them had a patient-reported outcomes (PRO) labeling in their summary of product characteristics (SmPC), and 3) if the PRO measures were used as primary efficacy variables.  Methods: The EMA website was explored to identify all medicines approved for narcolepsy. The PROLABELS database, through the ePROVIDE platform, was used for PRO labeling identification. All corresponding SmPCs and assessment reports (AR) were reviewed for endpoint positioning.  Results: The EMA approved only two products for narcolepsy, i.e., pitolisant and sodium oxybate. The review of the SmPCs and of the ARs of both products showed that two primary efficacy criteria, i.e., change in the number of cataplexy attacks and in excessive daytime sleepiness, were assessed using PRO measures. The number of cataplexy attacks was obtained from diary cards filled in by the patients. The Epworth Sleepiness Scale (ESS) score was used to assess efficacy on excessive daytime sleepiness. The ESS is a self-administered questionnaire providing a measure of a person’s general level of daytime sleepiness, or their average sleep propensity in daily life. The ESS asks people to rate, on a 4-point scale (0 – 3), their usual chances of dozing off or falling asleep in eight different situations or activities (e.g., sitting, reading, watching TV, etc.).  Conclusions: PRO assessment play a major role in the evaluation of products for the treatment of narcolepsy approved by the EMA. PND55 Use of Biological Based Therapies and Assessment of Health Related Quality of Life Among Thalassaemia Patients Ismail wI1, Hassali MA2, Farooqui M3, Saleem F4, Roslan MN1, Mohd Azahar NM1 of Health Sciences, University Technology MARA, Penang, Malaysia, 2Universiti Sains Malaysia, Penang, Malaysia, 3Qassim University, Qassim, Saudi Arabia, 4University of Balochistan, Quetta, Pakistan

1Faculty

Objectives: To analyse the prevalence of Biologically Based Therapies (BBTs) use and evaluate the Health Related Quality of Life (HRQoL) in a group of thalassaemia patients.  Methods: The study involved 390 thalassaemia patients and was conducted at the Kedah Thalassaemia Society, Alor Setar, Malaysia. The Short form survey version 2 (SF-36) was used to determine the HRQoL.  Results: From the total of 390 patients, the use of various types of Complementary and Alternative Medicines (CAM) were reported in 313 (80.6%) of them. Among these users of CAM, a total of 268 (85.62%) was found to use BBTs, therefore they were designated as BBT users and vice versa. The common types of BBTs used by the patients are nutritional supplements such as vitamins, minerals, enzymes (n= 174) and special diet such as herbs, animal products, and juices (n= 97). From the total number of participants, 14.1% were found to be spending more than 100 Malaysian Ringgit (MYR) on a monthly basis for these therapies and the rest of the participants were not aware of their expenditure on BBTs per month. The source of BBTs coming from the patient themselves were 215 (68.7%) whereas from friends’ and family members’ recommendations were 63.9 %. Only 27.2 % of the participants had disclosed the use of BBTs. Better HRQoL was found among the BBT non-users compared to BBT users. On the scale of physical function, role (p= 0.03) and social (p= 0.03) scores showed a significant difference between the BBT users and non-users.  Conclusions: Biologically based therapies are commonly used among thalassaemia patients in Malaysia. As the results revealed that the assessment of HRQoL shows better quality of life among BBT users, therefore, the potential role of BBTs in thalassaemia care should be further investigated. PND56 Quality of Life for Patients with Neurofibromatosis Type 1 Associated Plexiform Neurofibromas (PNF) Lai J, Beaumont JL, Jensen S Northwestern University, Chicago, IL, USA

Objectives: Neurofibromatosis Type 1 plexiform neurofibromas (pNFs) are associated with a variety of symptoms and concerns that affect patients’ quality of life (QOL); yet most research limited in studying pain. This study aimed to provide a more comprehensive understanding of their QOL by reporting their scores on measures from the Quality of Life in Neurological Disorders (Neuro-QoL) and Patient-Reported Outcomes Measurement Information System (PROMIS). These measures selected based on our previous qualitative study results.  Methods: 183 adult patients with pNF (mean= 40.1 yrs; 67% female) were recruited. 45% had a diagnosis under 5 years old, and 51% had more than 20 café-au-lait spots. All completed Neuro-QOL Stigma and PROMIS Anxiety, Depression, Self-Efficacy

20 (2017) A399–A811

A727

(Manage Day Activity, Manage Emotion, Manage Medications, Manage Social Interaction, Manage Symptoms), Pain (Interference, and Intensity), Physical Function, and Social Function (Ability to Participate Social Role, and Satisfaction Roles Activities). Scores were reported using the US general population based T-score system, where mean= 50 and standard deviation (SD)= 10. A Tscore >  53 or <  47 (i.e., 1/3 SD from norm) was considered significant as it exceeded the minimal important difference (1/3 SD). For Social Function and Self-Efficacy measures, higher scores meant better functioning; while higher scores meant worse functioning on others.  Results: Patients reported similar social function (M= 50.6 and 51.4) and selfefficacy/manage symptoms (M= 49.2) to the US general population. yet significant worse than the general population on all other domains, with Anxiety being the worst (M= 58.1), followed by Depression (M= 56.2), Stigma (M= 55.9), Pain Interference (M=  55.2), Self-Efficacy/Social Interaction (M=  44.8), and Self-Efficacy/Emotion (M= 44.9), with rest between 4 and 5 Tscores worse than the norm.  Conclusions: Results of this study showed patients with pNF experienced significant inferior QOL on numerous domains. Given the incurable life-long characteristics of the pNF, interventions to help patients better cope with pNF are needed. PND57 Mapping the Modified Ashworth Scale and Physician’s Global Assessment to Preference-Based Health Utilities in Adults with Lower Limb Spasticity Hansen RN1, Lubinga SJ1, Fonseca T2, Dinet J2, Gabriel S2, Sullivan SD1 of Washington, Seattle, WA, USA, 2Ipsen Pharma, Boulogne-Billancourt, France

1University

Objectives: The relationship between severity of lower limb spasticity and quality of life has not been previously characterized. We sought to quantify this relationship among adults with lower limb spasticity from stroke or traumatic brain injury.  Methods: Data on 381 patients from a randomized controlled trial (NCT01249404) and its open-label extension (NCT01251367) were included in our analyses. We fit random intercept linear regression models using the EQ-5D and SF-6D (converted from SF-36) index scores as dependent variables and the Modified Ashworth Scale (MAS) or Physician’s Global Assessment (PGA) as the primary independent variables. All candidate models included age and sex, and tested possible combinations of other covariates using a 10-fold (9 training, 1 prediction) crossvalidation procedure. Model selection was based on minimizing the mean squared error (MSE).  Results: The four sets of models each converged successfully, with age, sex, prior toxin use, physiotherapy use, other spasticity medication use, and baseline pain level included in all of the final models except the SF-6D and MAS model (physiotherapy was dropped). The MSE of both the EQ-5D models of MAS and PGA was 0.054. The MSE of the SF-6D models was substantially lower at 0.016 and 0.014, respectively. The MAS:SF-6D model produced utility predictions of 0.64, 0.63, 0.63, 0.62, 0.62, and 0.61 for MAS levels 0, 1, 1+, 2, 3, and 4 respectively. The PGA:SF-6D model produced utility predictions of 0.61 (non-response) and 0.63 (response). Most of the utility predictions had overlapping 95% confidence intervals.  Conclusions: The SF-6D models resulted in more accurate predictions of health-related quality of life compared to the EQ-5D models. However, the differences in predicted utility values between the levels of both MAS and PGA do not appear to be meaningfully different for spasticity patients. PND58 The Impact of Disability on Activities of Daily Living, Productivity and Independency in Patients with Multiple Sclerosis- Findings from a 2016 Study in Europe and the United States Ma E1, Wu N1, Yeh W1, Jones E2, Thomas NP1 1Genentech, Inc., South San Francisco, CA, USA, 2Adelphi Real World, Manchester, UK

Objectives: Multiple sclerosis (MS) is a chronic disease of the central nervous system, and a leading cause of disability in young adults of working age. The main objective of this study is to compare MS patients’ productivity, ability to perform daily self-care activities and independency by level of disability progression.  Methods: This study used data from the patient record form of the 2016 Adelphi Multiple Sclerosis Disease Specific Program, a cross-sectional study collecting data from 487 neurologists and 5402 patients in France, Germany, Italy, Spain, United Kingdom, and United States between November 2015 and March 2016. Patients with current EDSS score < 3, 3-5.5, and > 5.5 were categorized as with mild, moderate, and severe disability, respectively. Outcomes of interest, including activities of daily living (ADL) and instrumental activities of daily living (IADL) were compared across groups. Productivity and independency were examined by whether a patient was able to work full time and whether he/she had a caregiver, respectively.  Results: A total of 2,695, 1,568, and 871 MS patients were identified for the mild, moderate, and severe group, respectively. Among these groups, differences were observed in mean age (37.2, 46.0, and 51.7, p< 0.0001) and percent of females (68.2%, 57.5%, and 54.8%, p< 0.0001). As disability varied from mild, moderate to severe, fewer patients are being treated on a disease modifying treatment (79.4%, 69.3% vs. 48.7%, p< 0.0001), a higher proportion of patients needed assistance with ADL (3.3%, 26.9%, 63.9%, p< 0.001) and IADL. (12.6%, 44.1%, 72.0%, p< 0.001), less likely to work full time (57.1%, 28.5%, 7.6%, p< 0.001), and have a higher reliance on a professional caregiver (1.0%, 7.4%, 26.6%, p< 0.001) and a family caregiver (14.7%, 39.2%, 59.7%, p< 0.001).  Conclusions: These findings highlight the increasing burden associated with disability progression in MS patients, which limit their ability to perform self-care activities, work full time, and live independently. PND59 Impact of Disease Activity Measures on Health Utilities in PPMS Daigl M1, Jhuti GS1, McDougall F2, Bennett I1 Roche Ltd, Basel, Switzerland, 2F. Hoffmann-La Roche Ltd, Welwyn Garden City, UK

1F. Hoffmann-La

Objectives: To evaluate the impact of disease activity measures on health utilities in PPMS patients.  Methods: In the ORATORIO phase III clinical study 732 PPMS patients were randomized in a 2:1 ratio to treatment with ocrelizumab 600mg or