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3026 Experiences of Sickle Cell Disease (SCD) Reported By Healthcare Professionals (HCPs) across Different Regions: International Sickle Cell World Assessment Survey (SWAY)

Program: Oral and Poster Abstracts
Session: 904. Outcomes Research—Non-Malignant Conditions: Poster II
Hematology Disease Topics & Pathways:
Sickle Cell Disease, Hemoglobinopathies, Diseases
Sunday, December 12, 2021, 6:00 PM-8:00 PM

Ifeyinwa Osunkwo, MD, MPH1, Biree Andemariam, MD2, Caterina P Minniti, MD3, Fuad El Rassi, MD4, Erfan Nur, MD, PhD5, Alecia C Nero, MD6*, Raffaella Colombatti, MD7, Mariane de Montalembert, MD, PhD8, Miguel R Abboud, MD9, Jean-Benoît Arlet, MD10*, Suman Jain, MD11*, Wasil Jastaniah, MD12*, Marimilia Pita, MD13*, Beverley Francis-Gibson14*, Cassandra Trimnell, BA15*, Laurie DeBonnett, MD16*, Tom Bailey, MSci17*, Olivera Rajkovic-Hooley, PhD17*, Baba PD Inusa, MD, MBBS18 and John James, MSc19*

1Sickle Cell Disease Enterprise at the Levine Cancer Institute/, Atrium Health - Wake Forest School of Medicine, Charlotte, NC
2New England Sickle Cell Institute at the University of Connecticut, Farmington, CT
3Albert Einstein College of Medicine, New York, NY
4Emory University School of Medicine and Georgia Comprehensive Sickle Cell Center at Grady Health System, Atlanta, GA
5Amsterdam University Medical Centers, Academic Medical Center, Amsterdam, Netherlands
6University of Texas Southwestern Medical Center, Dallas, TX
7Azienda Ospedale-Università di Padova, Padova, Italy
8Hôpital Universitaire Necker-Enfants Malades, Paris, France
9American University of Beirut Medical Center, Beirut, Lebanon
10Hôpital Européen Georges-Pompidou, Paris, France
11Thalassemia and Sickle Cell Society, Hyderabad, India
12Umm Al-Qura University, Mecca, Saudi Arabia
13Laureate University-UAM, Pediatric-Hematology, Hospital Samaritano, São Paulo, Brazil
14Sickle Cell Disease Association of America, Baltimore, MD
15Sickle Cell 101, San Jose, CA
16Novartis Pharmaceuticals Corporation, East Hanover, NJ
17Adelphi Real World, Bollington, United Kingdom
18Evelina Children's Hospital and Guy’s and St Thomas’ Hospital, London, United Kingdom
19Sickle Cell Society, London, United Kingdom

Background: SCD is an inherited blood disorder that for many patients (pts) has a high clinical burden, results in poor quality of life (QoL), and reduces life expectancy. Gaining a deeper understanding of pt and HCP experiences of SCD is important to improve pt management.

Aims: SWAY was a cross-sectional survey that assessed pt and HCP experiences of SCD. Here we focus on the experiences of HCPs from various regions on SCD symptoms and complications, impact of SCD on QoL, treatment goals and treatment satisfaction.

Methods: SWAY was developed by international SCD expert physicians, pt advocates and Novartis. HCPs completed the survey between Apr and Oct 2019. Eligible HCPs had qualified in their primary specialty by 2014 and were managing ≥10 SCD pts at the time of survey (≥5 pts per HCP in Canada; ≥2 pts in the Netherlands). Responses to questions on how much SCD impacts pt QoL, and on HCP treatment satisfaction, were ranked on a Likert scale (1–7, where 1=not at all/strongly dissatisfied, 7=a great deal/strongly satisfied; 5–7 indicated high impact/satisfaction). The data reflect only the experiences of the surveyed HCPs in each region (recruited by Adelphi Real World fieldwork). A limitation is that Asia and South America (SA) were represented by single countries (India and Brazil, respectively).

Results: SWAY was completed by 365 HCPs from 6 regions (Table). In all regions HCPs recognized the prevalence of acute and chronic pain, however acute pain was reported less frequently by HCPs in Africa than in other regions (Table). Acute chest syndrome and joint issues were among the top 5 most frequently mentioned complications by HCPs in all regions. Globally, HCPs recognized the high impact of SCD symptoms and complications on pt QoL and the high negative impact of SCD on pt emotional wellbeing (Likert score 5–7 reported by 79–100% and 71–97%, respectively).

Fewer HCPs in the Middle East (ME) reported a high impact of SCD on physical and sexual activity, compared with HCPs in other regions. Around 40% of HCPs in the ME and Asia thought SCD has a high impact on daily activities, compared with 79–90% of HCPs in other regions. In Asia, fewer HCPs reported that SCD has a high impact on pts’ education and ability to maintain a job compared with HCPs in other regions (Figure).

Hydroxyurea (HU) was among the top 3 most common therapies ever initiated and was the therapy most likely to be initiated in any age group by HCPs in almost all regions. In Africa, the most common therapy ever initiated and the therapy most likely to be initiated in any age group was opioids (Table). Fewer HCPs in North America (NA; 32%) and SA (27%) were highly satisfied with current SCD treatments, compared with HCPs in other regions (46–72%). The main reason for dissatisfaction was limited treatment options in all regions except Asia, where HCPs said they were unable to reach their treatment goals with current therapies.

Improving pts’ QoL was among the top 3 treatment goals for 51–84% of HCPs across all regions. For HCPs in NA and the ME, the most important goal when treating vaso-occlusive crises was to improve QoL; in SA, Europe and Asia it was to avoid organ damage; and in Africa it was to eliminate pain completely.

Discussion: The top 5 most frequent SCD symptoms and complications that HCPs reported were similar across all regions. There were regional differences in HCP experiences of how SCD impacts aspects of pts’ daily life, with fewer HCPs in the ME reporting a high impact on physical and sexual activity, and fewer HCPs in Asia and the ME reporting a high impact on daily activities compared with other regions. This may be due to cultural variations, with pts in these regions being less comfortable discussing these topics with HCPs. There was a difference in the reported impact of SCD on school and work between HCPs in Asia and other regions, which could be due to varying expectations regarding school/work productivity. HU was one of the top 3 most common treatments ever initiated by HCPs for pts of any age, except in Africa, which may be due to an educational knowledge gap about HU, high cost, or poor access in this region. HCPs in almost all regions, except Asia, were dissatisfied with current SCD treatments because of limited therapeutic options, indicating a global unmet need for additional treatment choices. Improving QoL was the most important treatment goal for HCPs in all regions, demonstrating the high negative impact that SCD has on pt QoL and the ongoing need for methods to address this.

Disclosures: Osunkwo: Novartis Pharmaceuticals: Consultancy, Honoraria, Speakers Bureau; FORMA Therapeutics: Consultancy, Membership on an entity's Board of Directors or advisory committees; Global Blood Therapeutics: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Chiesi: Consultancy, Membership on an entity's Board of Directors or advisory committees; Acceleron: Consultancy, Membership on an entity's Board of Directors or advisory committees; Cyclerion: Consultancy, Membership on an entity's Board of Directors or advisory committees; Emmaus: Consultancy, Speakers Bureau; Takeda: Consultancy; Agios: Consultancy, Membership on an entity's Board of Directors or advisory committees; Novo Nordisc: Consultancy, Membership on an entity's Board of Directors or advisory committees; CDC: Research Funding; HRSA: Research Funding; PCORI: Research Funding. Minniti: Roche: Consultancy, Honoraria; NovoNordisk: Consultancy, Honoraria; Novartis: Consultancy, Honoraria; GBT: Consultancy, Research Funding. El Rassi: Cyclerion: Research Funding; Novartis: Research Funding; Pfizer: Research Funding; Novartis: Membership on an entity's Board of Directors or advisory committees; Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees; bluebird bio: Membership on an entity's Board of Directors or advisory committees. Nur: Roche: Speakers Bureau; Celgene: Speakers Bureau; Novartis: Research Funding, Speakers Bureau. Nero: Global Blood Therapeutics: Consultancy; Editas Medicine: Consultancy; bluebird bio: Consultancy; Novartis: Consultancy. Colombatti: Global Blood Therapeutics: Research Funding; Addmedica: Consultancy; Forma Therapeutics: Consultancy; Novartis: Consultancy; NovoNordisk: Consultancy; BlueBirdBio: Consultancy; Global Blood Therapeutics: Consultancy; BlueBirdBio: Research Funding. de Montalembert: Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees; Addmedica: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; bluebird bio: Membership on an entity's Board of Directors or advisory committees; Vertex: Membership on an entity's Board of Directors or advisory committees. Abboud: Jazz Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Research Support and Advisory Board, Research Funding; AstraZeneca: Membership on an entity's Board of Directors or advisory committees, Other: Reserach support and advisory board , Research Funding; GBT: Other: Research Support, Research Funding; Vertex Pharmaceuticals: Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: DSMB. Arlet: Addmedica: Research Funding; Pfizer: Honoraria; Novartis company: Consultancy, Honoraria, Research Funding. Jastaniah: Novartis: Consultancy, Honoraria, Research Funding. Pita: GLOBAL ALLIANCE OF SCD: Membership on an entity's Board of Directors or advisory committees; LUA VERMELHA SCD ASSOCIATION: Membership on an entity's Board of Directors or advisory committees. Francis-Gibson: Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees; Sickle Cell Disease Association of America: Current Employment; ASH: Membership on an entity's Board of Directors or advisory committees; Alliance for Regenerative Medicine Foundation for Cell and Gene Medicine: Membership on an entity's Board of Directors or advisory committees; Global Alliance of SCD Organizations: Membership on an entity's Board of Directors or advisory committees. Trimnell: Novartis: Consultancy; Cyclerion: Consultancy; Global Blood Therapeutics: Consultancy. DeBonnett: Novartis Pharmaceuticals Corporation: Current Employment. Bailey: Novartis Pharmaceuticals: Other: I am an employee of Adelphi Real World, which received payment from Novartis Pharmaceuticals for this research. Rajkovic-Hooley: Novartis Pharmaceuticals: Other: I am an employee of Adelphi Real World, which received payment from Novartis Pharmaceuticals for this research. James: GBT: Honoraria; Novartis: Honoraria.

*signifies non-member of ASH