Publication Only Abstracts
PU001 Adynovi advances the advate experience by improving the quality of life
C. C. Tarniceriu1,2,*
1Hematology, Emergency County Hospital “Sf. Spiridon” Iași; 2Anatomy, University of Medicines and Pharmacy “Grigore T. Popa”, Iasi, Romania
Introduction: Haemophilia A is known as a hereditary coagulopathy that is manifested by the appearance of haemorrhagic episodes as a result of a quantitative/qualitative deficiency of the coagulation factor VIII.
Poster purpose is to present the impact of personalised therapy on the quality of life of people with haemophilia.
Methods: The review of the literature was carried out, to which data from the experience of the Haematology Clinic were added – case presentation of a patient with Haemophilia type A in whom therapy was personalised with octocog alfa and then with rurioctocog alfa pegol 45-year old male, diagnosed in childhood with Haemophilia type A severe. Clinical features: multiple haemophilic arthropathies (left major joints) – HJHS = 43, target joints – left ankle, hepatitis VCH, bleeding haemorrhoids – haemorrhagic shock (Hb – 3 g/dL), herniated disc L5-S1, poor peripheral venous access. Living conditions: continuous physical effort – welder, over 50% of the time orthostatic. Personalised therapy with octocog alfa was carried out – minimum factor VIII level target was 5%; later it was decided to increase the minimum level of factor VIII at 10% as a result of the continuous physical effort. In achieving this goal, Bayesian estimation was used within the MYPKfit. Subsequently, the same therapeutic targets were maintained under the administration of factor VIII with an extended half-life FVIII rurioctocog alfa pegol.
Results: In question is a patient with haemophilia type A severe form with significant damage to the osteoarticular system and who feels the burden of the treatment. Personalised therapy was aimed at a minimum factor VIII level that would ensure superior protection on the osteoarticular system. Under treatment with rurioctocog alfa pegol, a lower consumption of factor VIII was found (DT octocog alfa = 37.170 U.I.
Number of factor VIII administrations – 14/month versus DT rurioctocog alfa pegol = 34.016U.I-No administrations – 8/month) to maintain the minimum level of factor VIII at 10%. Also, the effectiveness of the treatment during the administration of rurioctocog alfa is increased: ABR – 24 versus ABR – 10.
Discussion/Conclusion: Ruricoctocog alfa pegol increases the quality of life of patients through increased efficacy, personalisation of therapy, and decreased treatment burden.
Keywords: Congenital type A haemophilia, octocog alfa, rurioctocog alfa pegol, personalised therapy, quality of life.
Disclosure of Interest: None declared.
PU002 Sickle cell health awareness, perspectives and experiences (SHAPE) survey: Perspectives on sickle cell disease (SCD) from physicians in India, Malaysia and Thailand
R. Gomez1*; A. Su2
1Pfizer, Singapore, Singapore; 2Ipsos Healthcare, London, UK
Introduction: The burden of SCD in India (IND), Malaysia (MLY) and Thailand (TH) is not well known. SHAPE Survey was developed to understand the experiences and unmet needs of Healthcare Professionals (HCPs) treating the condition and the experiences and unmet needs of SCD patients.
Methods: The SHAPE survey was deployed among consenting HCPs from IND, MLY and TH managing SCD patients for >1 year. Survey responses were collected by interviewing HCPs either through face-to-face meetings or online. HCPs were invited if they had clinical experience between 3 and 35 years and were managing ≥3 SCD patients.
Results: A total of 85 HCPs participated in the survey from IND (30), MLY (25) and TH (30). Participants included haematologists (28%), paediatricians (22%), paediatric haematologists (18%), GPs (12%) and other specialties (20%).
Hydroxyurea/hydroxycarbamide (72% total: 56% IND, 77% MLY and 83% TH) was observed to be the main treatment their patients are currently receiving. Around 1/3rd patients receive chronic blood transfusion (33% total: 30% IND, 33% MLY and 36% TH).
Surveyed HCPs believed that SCD greatly impacts long-term health prospects, self-esteem and mental health in younger patients. Amongst adult patients, earning potential is also impacted. IND participants reported a notable impact on overall wellbeing (77% IND, 5% MLY and 4% TH) and on optimism about future (70% IND, 15% MLY and 13% TH). Financial burden of SCD is evident across three countries, with 78% of surveyed HCPs agreeing that their SCD patients face financial difficulties and 60% agreeing that their patients of lower social economic status are more difficult to treat.
98% of surveyed HCPs agree early intervention is the key to managing SCD, though 86% agree they would like more support to educate patients about the long-term effects of SCD. More HCPs in IND (93%) consider the cost/benefit ratio to the patient and/or healthcare system before recommending a treatment versus MLY (60%) and TH (63%).
Surveyed HCPs in TH (80%) and MLY (68%) express greater confidence in knowledge versus IND (37%), suggesting need for further educational support.
Discussion/Conclusion: There is a need for greater recognition of the financial burden and impact on overall wellbeing of patients and their families living with SCD in IND, MLY and TH. Surveyed HCPs are optimistic about using novel treatment options but need additional support for educating patients.
Disclosure of Interest: R. Gomez Shareholder of: Stockholder of Pfizer Ltd, Employee of: Full time employee of Pfizer Ltd, A. Su: None declared.
