Symptoms and Complications of Sickle Cell Disease Analyzed By Patient-Reported Genotype, Sex and Country/Region: Results from the International Sickle Cell World Assessment Survey (SWAY)

Inusa, Baba

Oral and Poster Abstracts
114. Hemoglobinopathies, Excluding Thalassemia—Clinical: Poster I

sickle cell disease, Diseases, Hemoglobinopathies

Ifeyinwa Osunkwo, MD, MPH¹^*, John James, MSc²^*, Biree Andemariam, MD³, Fuad A El Rassi, MD⁴, Beverley Francis-Gibson, MA⁵^*, Alecia C Nero, MD⁶^*, Caterina P. Minniti, MD⁷, Cassandra Trimnell, BA⁸^*, Miguel R Abboud, MD⁹, Jean-Benoît Arlet, MD¹⁰^*, Raffaella Colombatti, MD, PhD¹¹, Mariane de Montalembert, MD, PhD¹²^*, Suman Jain, MD¹³^*, Wasil Jastaniah, MD¹⁴^*, Erfan Nur, MD, PhD¹⁵, Marimilia Pita, MD¹⁶^*, Nicholas Ramscar, MBBS¹⁷^*, Tom Bailey, MSci¹⁸^*, Olivera Rajkovic-Hooley, PhD¹⁸^* and Baba PD Inusa, MD, MBBS¹⁹

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

Background: Sickle cell disease (SCD) is a group of blood disorders with a range of clinical manifestations across multiple organ systems, largely driven by hemolytic anemia and vaso-occlusion. Recurrent vaso-occlusive crises (VOCs) can lead to organ damage and long-term complications, and the symptoms and complications of SCD can have a significant negative effect on a patient’s quality of life. The symptoms and complications of SCD were assessed in the international Sickle Cell World Assessment Survey (SWAY); results for the overall patient population (Osunkwo et al. ASH 2019) and incidence of VOCs by country (Osunkwo et al. EHA 2020) have previously been reported.

Aims: To assess the most common symptoms (not including VOCs) and complications reported in SWAY by patient-reported genotype, sex and country/region.

Methods: SWAY was developed by international SCD experts, patient advocacy groups and Novartis. Patients with SCD aged ≥6 years were asked to complete the survey between April and October 2019 (completion by proxy [parent/guardian/caregiver] was required for patients aged 6–11 years, optionally available for older patients). Symptoms (experienced in the month preceding survey) and complications (ever experienced) were self-reported by patients. Opinions on the severity of symptoms were captured using a 1–7 Likert scale (1=not severe, 7=worst imaginable; 5–7 indicated high severity). Genotype was self-reported and not independently verified.

Results: 2145 patients (mean age 25 years; standard deviation, 13.1) from 16 countries across six regions were included in SWAY; 52% of patients were female. Patients’ self-reported genotypes were HbSS (n=1042 [49%]), HbSC (n=446 [21%]), HbSβ⁺ (n=60 [3%]), HbSβ⁰ (n=54 [3%]) and other (n=30 [1%]); 513 (24%) patients were unaware of their genotype.

At the time of survey completion, the most commonly reported symptom in the past month for all patient-reported genotypes was fatigue/tiredness. Fever, joint issues and infections were the most commonly reported complications across all patient-reported genotypes. The top 3 most commonly reported symptoms in the past month across both sexes were fatigue/tiredness, bone aches and headache (Figure 1). The proportion of females who rated these symptoms as being of high severity was 71%, 70% and 59%, respectively, slightly higher than the proportions reported by males (61%, 62% and 49%). Fever (females, 65%; males, 62%), joint issues (females, 61%; males, 51%) and infections (females, 59%; males, 50%) were the three most commonly reported complications ever experienced when analyzed by sex. The most common high-severity symptoms varied when analyzed by region (Figure 2). The most commonly reported symptom in the past month was fatigue/tiredness for patients in North America (71%), South America (77%), Europe (85%) and Asia (63%). The most commonly reported symptoms for patients in other regions were bone aches (Middle East, 67%) and headaches (Africa, 58%). Within regions, there was variation between countries in the symptoms reported by patients in the past month. For example, 64% of patients from France reported bone aches compared with 27% of patients from Italy. Fever, joint issues and/or infections were consistently ranked in the top 5 most common complications ever experienced by patients in all regions. There was variation within regions in commonly reported complications ever experienced; for example, 42% of patients from Ghana reported joint issues compared with 17% of patients from Nigeria.

Conclusions: Fatigue/tiredness was the most commonly reported symptom in SWAY when analyzed by both patient-reported genotype and by sex. It was also the most commonly reported symptom in nearly all of the regions analyzed. The complications that patients had ever experienced were largely consistent across all analysis groups, with fever, joint issues and infections the most commonly reported. Females reported complications and symptoms at a higher rate than males.

Disclosures: Osunkwo: Data and Safety Monitoring Board (DSMB) membership for Micella Biopharma: Membership on an entity's Board of Directors or advisory committees; Patient Centered Outcomes Research Institute (PCORI): Research Funding; Health Resources and Services Administration (HRSA): Research Funding; FORMA Therapeutics: 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; Acceleron: Membership on an entity's Board of Directors or advisory committees; Terumo: Consultancy; Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Pfizer: Consultancy, Membership on an entity's Board of Directors or advisory committees. James: Novartis: Honoraria; Sickle Cell Society: Current Employment. Andemariam: bluebird bio: Consultancy, Membership on an entity's Board of Directors or advisory committees; Emmaus: Membership on an entity's Board of Directors or advisory committees; Vertex: Honoraria; Imara: Research Funding; Hemanext: Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Pfizer: 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; NovoNordisk: Consultancy, Membership on an entity's Board of Directors or advisory committees; Roche: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Terumo BCT: Consultancy, Membership on an entity's Board of Directors or advisory committees; Sanofi Genzyme: Consultancy, Membership on an entity's Board of Directors or advisory committees; Guidepoint: Honoraria; Accordant: Membership on an entity's Board of Directors or advisory committees; CHNCT: Consultancy; CRISPR/Vertex: 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. El Rassi: 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; Novartis: Membership on an entity's Board of Directors or advisory committees, Research Funding; Pfizer: Research Funding; Cyclerion: Research Funding. Francis-Gibson: Sickle Cell Disease Association of America: Current Employment. Nero: Bluebird bio: Consultancy; Novartis: Consultancy. Minniti: CLS Bering: Consultancy; Emmaus: Consultancy, Research Funding; Roche: Consultancy, Research Funding; TauTona: Consultancy, Research Funding; Bluebird bio: Consultancy, Research Funding; Global Blood Therapeutics: Consultancy, Research Funding; Novartis: Consultancy, Research Funding. Trimnell: Cyclerion: Consultancy; Novartis: Consultancy; Global Blood Therapeutics: Consultancy. Abboud: Global Blood Therapeutics: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; AstraZeneca: Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Consultancy, Honoraria, Research Funding; Crispr Therapeutics: Membership on an entity's Board of Directors or advisory committees; Novo Nordisk: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Amgen: Other: Travel support; Eli Lilly: Research Funding; Modus Pharmaceuticals: Research Funding. Arlet: Novartis: Consultancy, Honoraria. Colombatti: Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees; Addmedica: 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. de Montalembert: Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees; Bluebird bio: Honoraria, Membership on an entity's Board of Directors or advisory committees; Vertex: 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. Jain: Thalassemia and Sickle Cell Society: Other: Chief Medical Research Officer and Secretary. Jastaniah: Novartis: Consultancy, Honoraria. Nur: Novartis: Consultancy. Ramscar: Novartis Pharma AG: Current Employment. Bailey: Novartis: Other: Employee of Adelphi Real World, which has received consultancy fees from Novartis; Adelphi Real World: Current Employment. Rajkovic-Hooley: Novartis: Other: Employee of Adelphi Real World, which has received consultancy fees from Novartis; Adelphi Real World: Current Employment. Inusa: Global Blood Therapeutics: Honoraria, Other: Steering committee participation, Research Funding, Speakers Bureau; Novartis: Honoraria, Other: Steering committee participation, Research Funding, Speakers Bureau; Bluebird bio: Research Funding; AstraZeneca: Honoraria, Other: Steering committee participation, Research Funding, Speakers Bureau; Vertex: Research Funding.

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^*signifies non-member of ASH

808 Symptoms and Complications of Sickle Cell Disease Analyzed By Patient-Reported Genotype, Sex and Country/Region: Results from the International Sickle Cell World Assessment Survey (SWAY)