TY - JOUR
T1 - Burden of disease, treatment utilization, and the impact on education and employment in patients with sickle cell disease
T2 - A comparative analysis of high- and low- to middle-income countries for the international Sickle Cell World Assessment Survey
AU - Osunkwo, Ifeyinwa
AU - James, John
AU - El-Rassi, Fuad
AU - Nero, Alecia
AU - Minniti, Caterina P.
AU - Trimnell, Cassandra
AU - Paulose, Jincy
AU - Ramscar, Nicholas
AU - Bailey, Tom
AU - Rajkovic-Hooley, Olivera
AU - Andemariam, Biree
N1 - Funding Information:
I.O. reports consultancy for Agios, Cheisi, Cyclerion, FORMA Therapeutics, Global Blood Therapeutics, HCP Live, Novartis, and Takeda; speakers' bureau for Emmaus, Global Blood Therapeutics, and Novartis; advisory board for Acceleron, Cyclerion, Cheisi, Emmaus, FORMA therapeutics, Global Blood Therapeutics, Novartis, and Novo‐Nordisk; grants from Centers for Disease Control (CDC), Department for Public Health (DPH), Health Resources and Services Administration (HRSA), Patient Centered Outcomes Research Institute (PCORI), and NC; Data and Safety Monitoring Board (DSMB) membership for Micella Biopharma; and Editor‐In‐Chief for . J.J. reports employment by the Sickle Cell Society and honoraria from Novartis. F.E.‐R. reports research funding from Cyclerion, Novartis, and Pfizer; and advisory board participation for Novartis, Global Blood Therapeutics, and bluebird bio. A.N. reports consultancy/expert testimony for bluebird bio, Global Blood Therapeutics, and Novartis. C.P.M. reports consultancy for Agios, bluebird bio, Emmaus, Forma Therapeutics, Global Blood Therapeutics, Novartis, Roche, and Sanguine. C.T. reports consultancy/expert testimony for Novartis, Cyclerion, and Global Blood Therapeutics. J.P. is an employee of Novartis Pharmaceuticals Corporation. N.R. is an employee of Novartis Pharma AG. O.R.‐H. and T.B. are employees of Adelphi Real World, which received payment from Novartis Pharmaceuticals as part of this research. B.A. reports consultancy or membership on an advisory committee for Agios, Aruvant, bluebird bio, CRISPR/Vertex, Cyclerion, Emmaus, Forma Therapeutics, Global Blood Therapeutics, Hemanext, Novartis, Novo Nordisk, Sanofi Genzyme, and Terumo BCT; and research funding from Forma Therapeutics, Global Blood Therapeutics, Hemanext, Imara, and Novartis. Hematology News
Funding Information:
We would like to thank the patients, caregivers, and HCPs who have participated in the SWAY survey, and Adelphi Real World for executing the survey, collating the data, and running the analyses. We would like to thank Daniel Blumrich (Novartis Pharmaceuticals Corporation) for his substantial contribution to the conceptualization of SWAY, the defining of its objectives and design, and for his role in coordinating the running of the survey. We would also like to thank Biba Tinga (Sickle Cell Disease Association of Canada), Yolande Adjibi (Fédération SOS Globi), Elvie Ingoli (Interessengemeinschaft Sichelzellkrankheit und Thalassämie), Elmas Citak and Chantelle Rodgers (IXL Sickle Cell Awareness), Matthew Neal (Sickle Cell Society), and Carolyn Rowley (Cayenne Wellness Center) for their contribution to patient recruitment. We also thank all staff who contributed to patient recruitment from the Italian Association of Pediatric Hematology Oncology, Sickle Cell Disease Association of America, Sickle Cell 101, and Red Moon. This survey was sponsored by Novartis Pharmaceuticals Corporation. Medical writing assistance was provided by Alex Smith PhD, from Mudskipper Business Ltd, funded by Novartis Pharmaceuticals Corporation.
Funding Information:
This survey was sponsored by Novartis Pharmaceuticals Corporation. Medical writing assistance was provided by Alex Smith PhD, from Mudskipper Business Ltd, funded by Novartis Pharmaceuticals Corporation.
Publisher Copyright:
© 2022 Novartis Pharmaceuticals. American Journal of Hematology published by Wiley Periodicals LLC.
PY - 2022/8
Y1 - 2022/8
N2 - The international Sickle Cell World Assessment Survey (SWAY) reported a high impact of sickle cell disease (SCD) on patients' daily lives globally. In this study, we analyzed whether the reported burden differed between patients from the USA (n = 384) and other high-income (HI; n = 820) or low- to middle-income (LMI; n = 941) countries. We assessed symptoms and complications, incidence/management of vaso-occlusive crises (VOCs), treatment utilization/satisfaction, and the impact of SCD on education/employment. Certain symptoms (bone aches, insomnia, and joint stiffness) and complications (swollen/painful fingers/toes, gallstones, vision problems, blood clots, and asthma) were reported proportionally more by patients in the USA than in the HI/LMI countries. Self-reported VOCs were more common (mean [SD]: 7.1 [5.7] vs. 5.5 [8.9] and 4.4 [4.6] in the previous 12 months) and were managed more often by hospitalization (52% vs. 24% and 32%) in the USA than the HI and LMI countries. A higher proportion of patients from the USA than the HI/LMI countries reported a negative impact of SCD on their employment/schooling. Although high overall satisfaction with current treatments was reported globally, most patients indicated a strong desire for alternative pain medications. There are likely several reasons for the relatively high patient-reported burden in the USA group compared with the HI/LMI countries, including an older population and differences in newborn screening programs and pediatric/adult transition of care. It is clear that there is an urgent need for improved understanding and management of SCD globally, not just in the USA.
AB - The international Sickle Cell World Assessment Survey (SWAY) reported a high impact of sickle cell disease (SCD) on patients' daily lives globally. In this study, we analyzed whether the reported burden differed between patients from the USA (n = 384) and other high-income (HI; n = 820) or low- to middle-income (LMI; n = 941) countries. We assessed symptoms and complications, incidence/management of vaso-occlusive crises (VOCs), treatment utilization/satisfaction, and the impact of SCD on education/employment. Certain symptoms (bone aches, insomnia, and joint stiffness) and complications (swollen/painful fingers/toes, gallstones, vision problems, blood clots, and asthma) were reported proportionally more by patients in the USA than in the HI/LMI countries. Self-reported VOCs were more common (mean [SD]: 7.1 [5.7] vs. 5.5 [8.9] and 4.4 [4.6] in the previous 12 months) and were managed more often by hospitalization (52% vs. 24% and 32%) in the USA than the HI and LMI countries. A higher proportion of patients from the USA than the HI/LMI countries reported a negative impact of SCD on their employment/schooling. Although high overall satisfaction with current treatments was reported globally, most patients indicated a strong desire for alternative pain medications. There are likely several reasons for the relatively high patient-reported burden in the USA group compared with the HI/LMI countries, including an older population and differences in newborn screening programs and pediatric/adult transition of care. It is clear that there is an urgent need for improved understanding and management of SCD globally, not just in the USA.
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U2 - 10.1002/ajh.26576
DO - 10.1002/ajh.26576
M3 - Article
C2 - 35472249
AN - SCOPUS:85132116282
SN - 0361-8609
VL - 97
SP - 1055
EP - 1064
JO - American Journal of Hematology
JF - American Journal of Hematology
IS - 8
ER -