The Impact Of Obesity And Deprivation On HRQOL Gains Following Hip And Knee Replacements

VA L U E I N H E A LT H

life (r= -0.134).  Conclusions: The quality of life for patients with rare endocrine diseases is low especially regarding vitality, energy or fatigue and general health. The direct medical costs are significant and negatively correlated with the quality of life. PSY92 Disease-Specific Patient-Reported Outcome Instruments In Sickle Cell Disease: A Systematic Literature Review Gupta J, Ghildiyal A DRG Abacus, Gurgaon, India

Objectives: The US Food and Drug Administration (FDA) convened patient-focused drug development meetings to identify those symptoms/effects of sickle cell disease (SCD) on daily living, which are most important to patients. Drug developers are now expected to present direct evidence of the benefit of SCD therapies on these symptoms/effects. Such evidence has to be generated by measuring standardised patient-reported outcomes (PROs). This review aimed to identify and summarize published data on available SCD-specific PRO instruments.  Methods: MEDLINE® and EMBASE® databases were searched to identify studies assessing SCD-specific PROs, published in English and in the last 15 years.  Results: The literature search identified 26 studies (sample size, N= 12 to N= 561) assessing 13 SCD-specific PRO instruments. Most studies were conducted in the US (22 studies) followed by the UK (3 studies). Equal number of studies (13 studies) included either adult or paediatric (< 18 years) patients. Of the identified instruments, nine were used in adults and four in children or adolescents. The Pediatric Quality of Life Inventory™ Sickle Cell Disease Module (PedsQL-SCD) was most frequently used (8 studies), followed by the Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me; 4 studies) and the Sickle Cell Pain Burden Interview-Youth (SCPBI-Y; 3 studies). Pain was the most frequently assessed concept, included in eight of the 13 identified instruments. Other key symptoms/concepts assessed included cognition, fatigue, functional ability, self-efficacy, sleep, stiffness, emotional and social impairment/ stigma, and quality of life. Eight instruments were developed in the last 5 years. Validation studies were available for 10 instruments.  Conclusions: Several SCDspecific PRO instruments are available for selection as endpoint in clinical studies to demonstrate the effectiveness of innovative interventions in target population. Many scales are recently developed and their utility in clinical studies is yet to be ascertained. PSY93 Validity of Change In Recall Period For The Norfolk QOL Patient Reported Outcome (PRO) Measure von Maltzahn R1, Benjamin K2, Murphy R2, Lipman K3, d la Cruz M2 1GSK, Uxbridge, UK, 2ICON, Gaithersburg, MD, USA, 3ICON plc, Dublin, Ireland

Objectives: Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) is a rare degenerative disease with a progression pattern similar to that observed in diabetic neuropathy (Vinik et al., 2014). The Norfolk QOL-DN was developed to capture patients’ perceptions of the effects of diabetic neuropathy and has been validated in TTR-FAP patients. Based on regulatory feedback the recall period of the measure was changed from 4- to 1-week. The objective of this research was to determine the validity and acceptability of the one-week recall period.  Methods: Individual qualitative telephone interviews were conducted with patients diagnosed with TTR-FAP. Interviews were conducted by trained interviewers between May 2016 and June 2016. At the start of the interview, subjects completed the Norfolk QOL-DN while “thinking aloud,” or talking about the questionnaire instructions and items and their response to them. Participants were also asked questions about the ease of completing the Norfolk QOL-DN, focusing on the relevance of the one-week recall period. Interviews were audio-recorded and transcribed and analysed using qualitative thematic and content analysis.  Results: Twenty participants were interviewed; 55% male; mean age 61.1 years. No participants had significant difficulties understanding how to answer the Norfolk QOL-DN questions using a one-week recall period. All participants had experienced at least one of the symptoms covered by the instrument in the past week. The most prevalent type of symptom was neuropathy-related; All participants experienced at least one neuropathy symptom within the one-week time period. Generic functioning and HRQoL items were somewhat less sensitive as participants had adjusted their lives to their disability, not because they had problems with the recall period.  Conclusions: Participants in this study were able to understand and respond to the Norfolk QOL-DN items using the one-week recall period. Additional work exploring the item relevance and performance within TTR FAP would add to the rigour of this measure within this population. PSY94 The Impact Of Systemic Juvenile Idiopathic Arthritis (SJIA) On A Caregiver’s Productivity And The Child’s Schooling Shenoi S1, Horneff G2, Cidon M3, Ramanan A4, Kimura Y5, Quartier P6, Foeldvari I7, Zeft A8, Lomax K9, Gregson J10, Mckenna SJ11, Abma T12, Campbell S13, Weiss J14, Marinsek N15, Patel D13, Wulffraat N16 1Seattle Children’s Hospital, Seattle, WA, USA, 2Asklepios Kliniken, Sankt Augustin, Germany, 3Stanford University, Palo Alto, CA, USA, 4University Hospital Bristol, Bristol, UK, 5Hackensack University Medical Center, NJ, NJ, USA, 6Hôpital Necker, Paris, France, 7Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg, Germany, 8Pediatrics Rheumatology, Cleveland Clinic, Cleveland, OH, USA, 9Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA, 10Novartis Pharma AG, Basel, Switzerland, 11Novartis Business Services, Dublin, Ireland, 12VU University medical center, Amsterdam, The Netherlands, 13Navigant Consulting, Inc., London, UK, 14Navigant Consulting Inc, London, UK, 15Navigant Consulting, London, UK, 16Wilhelmina Kinderziekenhuis, Utrecht, The Netherlands

Objectives: To evaluate the impact of SJIA on the caregiver’s work productivity and the child’s schooling.  Methods: As part of an international study to assess the burden of SJIA on the caregiver and patient, from a caregiver’s perspective, caregiver’s work productivity (using the work productivity and activity impairment questionnaire (WPAI) and impact on patients schooling (using tailored questions)

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were assessed. Caregivers indicated change in work commitments due to their child’s SJIA and how frequently it caused them to miss work or for their child or siblings to miss school.  Results: Sixty-one biologic treated patients participated (12 anakinra, 25 canakinumab, 24 tocilizumab). Mean age at diagnosis and survey completion was 6.4 and 11.3 years, respectively. 77% of caregivers were employed (full or part time), however 36% had reduced their number of working hours or stopped working due to their child’s SJIA. SJIA related appointments were the main cause of a caregiver missing work, in the past two months (57%). Employed caregivers had missed a mean 2.8 hours of work in the past seven days due to their child’s SJIA, equating to approximately 25 working days annually. Caregiver’s mean absenteeism score based on WPAI, was 10% and mean presenteeism score (reduced on-the-job effectiveness) was 11%, equivalent to a productivity loss of 27.5 work days. In the two months prior to survey completion, a mean 2.9 school days were missed by patients due to their SJIA. Assuming a 5-day school week and 36 school weeks per year, this equated to 10% yearly schooling loss. SJIA caused patients’ siblings to miss a mean 0.3 days of school in the past two months.  Conclusions: SJIA families experience reduced school and work productivity. There is a need for effective therapeutic interventions which are not burdensome and limit disruption on SJIA families’ lives. PSY96 The Impact Of Obesity And Deprivation On HRQOL Gains Following Hip And Knee Replacements Martin RL Open Access Consulting, London, UK

Objectives: Obesity and deprivation have been shown to negatively impact HRQoL. This research aimed to investigate the relationship between gains in HRQoL resulting from hip and knee replacements and obesity and deprivation at the local National Health Service (NHS) Clinical Commissioning Group (CCG) level.  Methods: The following CCG data was extracted from the 2016 focus pack online tool: pre-treatment EQ-5D score for patients undergoing hip and knee replacements, the EQ-5D health gain achieved post-surgery, the deprivation index and proportion of people classified as obese within each CCG. Linear regression analyses were conducted using Microsoft Excel 2013® to determine the relationship between baseline and gains in EQ-5D and obesity and deprivation.  Results: There were 208 CCGs with data available, for which a positive correlation was observed between the deprivation index score and the level of obesity (R2=  0.20; p< 0.001). When the HRQoL impact of these factors in reference to hip and knee replacements was analysed, it showed that at baseline prior to surgery EQ-5D scores were negatively correlated with both obesity (hip: R2=  0.16; p< 0.001; knee: R2= 0.04; p< 0.01) and deprivation (hip: R2=  0.26; p< 0.001; knee: R2=  0.33; p< 0.001). Interestingly, the impact of these factors on the HRQoL gain following surgery was statistically significant for obesity (hip: R2=  0.06; p< 0.001; knee: R2= 0.05; p< 0.01) but not for deprivation (hip: R2= 0.0044; p= 0.34; knee: R2=  0.012; p= 0.11).  Conclusions: Both population obesity and deprivation are negatively correlated with poorer baseline EQ-5D scores in patients undergoing hip and knee replacement surgery, while obesity but not deprivation appears to significantly decrease HRQoL gains achieved through these surgeries. More research is needed to understand the complex interactions of obesity and deprivation with HRQoL gains from major surgeries. PSY97 Health Related Quality Of Life For Systemic Juvenile Idiopathic Arthritis (SJIA) Patients And Caregivers On Biologic Therapy, From A Caregiver’s Perspective Shenoi S1, Horneff G2, Cidon M3, Ramanan A4, Kimura Y5, Quartier P6, Foeldvari I7, Zeft A8, Lomax K9, Gregson J10, Mckenna SJ11, Abma T12, Campbell S13, Weiss J14, Marinsek N15, Patel D13, Wulffraat N16 1Seattle Children’s Hospital, Seattle, WA, USA, 2Asklepios Kliniken, Sankt Augustin, Germany, 3Stanford University, Palo Alto, CA, USA, 4University Hospital Bristol, Bristol, UK, 5Hackensack University Medical Center, NJ, NJ, USA, 6Hôpital Necker, Paris, France, 7Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg, Germany, 8Pediatrics Rheumatology, Cleveland Clinic, Cleveland, OH, USA, 9Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA, 10Novartis Pharma AG, Basel, Switzerland, 11Novartis Business Services, Dublin, Ireland, 12VU University medical center, Amsterdam, The Netherlands, 13Navigant Consulting, Inc., London, UK, 14Navigant Consulting Inc, London, UK, 15Navigant Consulting, London, UK, 16Wilhelmina Kinderziekenhuis, Utrecht, The Netherlands

Objectives: To evaluate the impact of SJIA on patient and caregiver health related quality of life (HRQoL), from the caregiver’s perspective, based on an international, non-interventional study. Methods: This study assessed the physical, mental and stress impact associated with SJIA on the caregiver and patient, from the caregiver’s perspective, using the following validated outcome measures; Child Health Questionnaire Parent-Form 50 (CHQ-PF50) for the child and 36-Item Short-Form Health Survey (SF-36v2) for the caregiver. The physical and mental component scores were compared to US population norms. Eligible patients had received biologic treatment for ≥ 2 months.  Results: Sixty-one biologic treated children were included. The mean (±SD: standard deviation) CHQ PF-50 physical (PhS) and psychosocial (PsS) summary scores in children with SJIA compared to the US normative population were significantly lower, by a large (> 0.8 SD) and moderately large (> 0.5 SD) effect size, (PhS: 40.0±18.2 vs. 53.0±8.8; PsS: 46.6±11.3 vs. 51.2±9.1), respectively. Caregivers displayed a mean SF-36v2 physical component score higher than a normative US population, although their mean mental component score was significantly lower than the US normative population, by a small effect size (> 0.3 SD), (MCS 46.2±10.7 vs. 50.0+10). The most frequent and high cause of stress for SJIA caregivers were: ‘worry about the long-term impact of their child’s SJIA’ (45%); ‘uncertainty about the future of their child, family and themselves, due to their child’s SJIA’ (28%); ‘uncertainty about their child’s ability to become fully independent as an adult’ (27%) and ‘feeling helpless about their child’s SJIA’ (24%).  Conclusions: SJIA has a significant impact on both the child and caregivers’ HRQoL. SJIA patients’ physical HRQoL was severely impaired and their psychosocial health impaired to a small degree. Caregivers were physically healthy but displayed mental HRQoL impairment.