Background: The success of clinical trials of new therapies for sickle cell disease (SCD) will depend on the recruitment and retention of a large and diverse group of people with SCD (PwS). ...Qualitative studies have reported barriers to participation in clinical trials, including the potential impact on health, unmanageable study demands, limited knowledge of trials, and lack of trust in healthcare(Patterson CA et al. J Pediatr Hematol Oncol 2015; Lee LH et al. Blood Adv 2021). The Learning and Insights into Sickle Cell Trial Experiences (LISTEN) Survey was developed to provide a robust and comprehensive understanding of the global barriers and motivators to participation in clinical trials for PwS. Aim: To present interim findings from the LISTEN Survey regarding participation and experiences of PwS in clinical trials, barriers and motivators to participation, and differences in responses from PwS compared with perspectives of healthcare professionals (HCPs) treating PwS. Methods: Between October 6, 2022 and June 22, 2023, a broad range of adults (≥18 years) with SCD and HCPs involved in the treatment and/or clinical research of SCD in 17 countries were asked to complete corresponding quantitative surveys. PwS were asked whether they had participated in a clinical trial and, if so, whether their experience had met their expectations. PwS were asked to rate on a 7-point scale (from not at all to extremely important) and rank (from most to least important) the importance of specified factors (grouped into five categories) when deciding whether to participate in a clinical trial for SCD. HCPs provided their perspectives on the importance of these factors to PwS. The results presented here include the total proportion of respondents who rated factors as extremely or very important and the proportion who ranked factors first or second. Results: Overall, 1028 PwS (57% female) with a median age (interquartile range) of 30 years (24-37) from 16 of the 17 countries and 361 HCPs (67% hematologists and/or SCD specialists) from 17 countries completed the survey. In total, 35% of PwS had been invited to participate in a clinical trial for SCD. In PwS who had taken part in a clinical trial (24%; n=249/1028), compared with their expectations, 36% reported a better experience, 36% the same as expected experience, 9% a worse experience, and 20% did not know what to expect. Extremely or very important factors that motivated PwS to participate in a clinical trial included the potential to better manage their symptoms (52%), the opportunity to try a new treatment that might work better (51%), and to increase their knowledge of SCD (51%; Figure 1). An important barrier to participation was the potential to experience different side effects (51%). In the trial information category, PwS ranked (first or second of five) safety measures (57%) and how the treatment works (49%) as the most important factors; these were considered to be significantly more important than who is leading (27%) or funding (24%) the trial; p<0.001 for all combinations. In the further considerations category, the majority of PwS ranked (first or second of five) speaking to other PwS involved in the trial (51%) and experts running the trial (50%) as the most important factors. HCPs overstated the potential practical barriers for PwS compared with responses from PwS, including missing school/work (50% vs 42%; p=0.009), the effort required to start the trial treatment (45% vs 32%; p<0.001), and travel requirements (52% vs 36%; p<0.001). HCPs understated the importance of wider clinical trial outcomes as motivators for PwS compared with responses from PwS, including supporting new treatment developments that may benefit them (41% vs 51%; p=0.001) or other PwS (38% vs 49%; p<0.001; Figure 2). Conclusions: Improving access and recruitment into clinical trials in SCD will require clear communication of the potential benefits to individuals and the wider SCD community, as well as potential safety and side effects. Findings from the LISTEN Survey highlight that those directly involved in the trial should deliver these messages, including other PwS who are involved in the trial. Given the disconnect between PwS and HCPs, shared decision-making may also improve understanding and increase participation in clinical trials. Further analyses of the survey results will be important to identify the differences in responses between regions, age groups, and other subpopulations.
Rooted in the patient experience, this Double Take video breaks down the pathophysiology and new treatments for sickle cell disease, including gene therapy.
Background: Patient perspectives have shifted towards greater expectations of autonomy and personalized care, leading to growing demands for partnerships between patients, healthcare providers ...(HCPs), and industry. Such collaborations provide a mechanism to understand the unique needs of patient communities, which is particularly important for rare diseases, such as hereditary hemolytic anemias (HHAs). The Patient Vision Project (initiated and sponsored by Agios Pharmaceuticals) was launched in November 2021, aiming to establish a set of principles for defining industry standards in engagement with multiple stakeholders, including (but not limited to) HCPs, caregivers and patients with HHA. Methods: The Project consisted of 4 phases: In-depth discovery (company-wide survey, and one-to-one interviews with patient advocates, HCPs and company leaders), with the goal to understand perspectives on patient-centricity and areas of strength and opportunity to optimize patient engagementThematic analysis of discovery findings and recommendations to align on company commitment to develop a patient engagement model1-Day interactive workshop (patients, HCPs, HHA experts and Agios representatives) for feedback on modelDevelopment of an implementation plan The workshop included 31 participants (patients, HCPs, HHA experts and Agios representatives) divided into breakout groups to define traits of a patient ally, identify universal ally behaviors, discuss ways to support core areas for collaboration and commitment to patients, and provide input to industry on patient needs and expectations (Figure). Results: During the discovery phase, 116 of 152 (76%) Agios respondents completed the survey, and 7 Agios leaders and 8 HHA experts (HCPs, patients, and patient advocates) were interviewed. Responses to questions pertaining to patient-centricity emphasized patient partnership/prioritization, with 42% of the 152 responses favoring ongoing patient engagement to understand their needs, transparent communication, and identification of collaboration opportunities. On questions related to company engagement with patients, 93% felt connected to patients because of their work, 86% felt they added value, and 86% felt the company had a clear approach for patient engagement. Interviewees emphasized the importance for company leadership to prioritize patient-centricity, as well as the value of hearing the patient voice and meeting their needs, while being transparently connected to their lives, experiences, and priorities. Findings from the discovery phase led to the concept of a “patient ally”, along with 4 universal behaviors to define allyship: (1) Listening actively and openly; (2) Collaborating and communicating; (3) Fostering mutual respect and trust; (4) Honoring individuality while maintaining perspective. 3 core areas for demonstrating collaboration and commitment were also defined: (1) Providing credible education; (2) Facilitating community connection; and (3) Delivering support. Implementation of key learnings consisted of: Establishing an internal culture based on the patient ally mindsetCreating a cross-functional working group to establish meaningful strategies and aligned approaches based on actionable insights from patients and HCPsFounding a “Red Cell Revolution™” group of patients, caregivers and HCPs across three HHA indications (PK deficiency, Thalassemia and Sickle Cell Disease) and Agios representatives to define and collaborate on shared issues within the HHA communityMeasurement of patient engagement activities to monitor the intended positive impact on the patient community Conclusion: Inception of the Patient Vision Project stemmed from a growing awareness of the importance of the patient voice-understanding how patients want to be engaged-and a vision to create a transparent mechanism that supports industry partnerships with patients to improve the drug development process. Evaluating patient engagement from industry, HCPs, and patient perspectives enabled the strategic alignment and shaping of a vision for effective partnership. In addition to providing a path forward to the participants of the Project, this initiative challenges industry standards and provides a blueprint to other companies with a common goal of understanding patient needs to ultimately reduce disease burden and improve patient outcomes.
Sickle cell disease (SCD) is a genetic disorder, characterized by hemolytic anemia and vaso‐occlusive crises (VOCs). Data on the global SCD impact on quality of life (QoL) from the patient viewpoint ...are limited. The international Sickle Cell World Assessment Survey (SWAY) aimed to provide insights into patient‐reported impact of SCD on QoL. This cross‐sectional survey of SCD patients enrolled by healthcare professionals and advocacy groups assessed disease impact on daily life, education and work, symptoms, treatment goals, and disease management. Opinions were captured using a Likert scale of 1‐7 for some questions; 5‐7 indicated “high severity/impact.” Two thousand one hundred and forty five patients (mean age 24.7 years standard deviation (SD) = 13.1, 39% ≤18 years, 52% female) were surveyed from 16 countries (six geographical regions). A substantial proportion of patients reported that SCD caused a high negative impact on emotions (60%) and school achievement (51%) and a reduction in work hours (53%). A mean of 5.3 VOCs (SD = 6.8) was reported over the 12 months prior to survey (median 3.0 interquartile range 2.0‐6.0); 24% were managed at home and 76% required healthcare services. Other than VOCs, fatigue was the most commonly reported symptom in the month before survey (65%), graded “high severity” by 67% of patients. Depression and anxiety were reported by 39% and 38% of patients, respectively. The most common patient treatment goal was improving QoL (55%). Findings from SWAY reaffirm that SCD confers a significant burden on patients, epitomized by the high impact on patientsʼ QoL and emotional wellbeing, and the high prevalence of self‐reported VOCs and other symptoms.
Background: Patient engagement is becoming increasingly important for all facets of healthcare, from drug development and approval, to ensuring equitable access and the delivery of care. It is ...imperative to bring the voice of those impacted by the actual disease into these processes: this is particularly true for rare diseases, such as hereditary hemolytic anemias (HHAs), where the disease burden is often high and coupled with a poor quality of life and complex treatment requirements. The Red Cell Revolution™ (RCR) advisory council arose organically as part of an Agios Pharmaceuticals-sponsored discovery process, during which a range of stakeholders including healthcare providers (HCPs), advocates, patients, and company leaders offered perspectives on how to optimize engagement with the disease communities. Insights uncovered as part of this process demonstrated that there are unifying health concerns and needs among those impacted by pyruvate kinase (PK) deficiency, sickle cell disease (SCD), and thalassemia, and that there is an opportunity to explore new patient-advocacy research, supported by the creation of a unique multi-stakeholder council. Objective: To apply a multi-stakeholder, rigorous patient-advocacy data collection approach to understand the unmet needs of patients, caregivers, and HCPs for three HHAs: PK deficiency, SCD, and thalassemia. Methods: The RCR (supported by Agios Pharmaceuticals) was established across three allied disease areas, with representation from patients (N=5), caregivers (N=1), advocates (N=3), clinicians (N=8), and Agios representatives (N=5) impacted by HHA. The patient advocacy research method commenced with a survey shared with all RCR members, which curated both qualitative and quantitative insights from the group. These insights subsequently underwent cluster analysis to determine the shared concerns experienced by those impacted by HHA. The data were also subjected to linguistic analysis whereby terminology used to describe the experiences of participants was ranked according to frequency of use to reveal the most prominent concerns. The results of these analyses were distilled into an agreed group vision by the RCR, and a specific research strategy was aligned on for the RCR to pursue. Results: RCR members provided detailed answers about unmet needs across three categories: 1) impact of disease; 2) local and regional community needs; and 3) international community needs. When applied, the analysis uncovered 12 common concerns expressed by all participants across the three disease areas. These are outlined in Table 1. These insights were distilled into four key topic areas: 1) emotional and physical fatigue, 2) timely care, 3) transition from pediatric to adult care, and 4) access disparities, which were then ranked according to four parameters, namely whether it was: 1) common across all three disease areas, 2) global in scope, 3) high potential for lasting impact, and 4) revolutionary (involving or causing a complete or dramatic change). This produced alignment on one key priority: emotional and physical fatigue. Conclusion: The commonality analysis deployed here demonstrates the ability of a multi-stakeholder council to determine priority areas for research to address unmet needs. The RCR will conduct an in-depth evidence audit of the existing research in this field to identify key knowledge gaps, enabling the design of a study that will answer outstanding research questions. This study will generate patient experience data with the potential to inform both the clinical setting and drug development. As a next step, the RCR is developing a patient advocacy research study to collect the necessary evidence to better understand fatigue and its impact on psychosocial quality of life measures, such as feelings of guilt.
Background: Patients with sickle cell disease (SCD) suffer a significant disease burden that affects their psychosocial well-being. Digital cognitive-behavioral therapy (CBT) has been utilized in ...other patient populations and shown to have clinical benefits. Although evidence-based, non-pharmacological interventions for pain management are widely used in other populations, this is not well studied in SCD. There are currently no large-scale, adequately powered clinical trials that evaluate the effectiveness and dissemination potential of digital behavioral pain management interventions for adults with SCD. Objective: The primary goal was to compare the effectiveness of two mobile-phone-delivered programs: 1) digital CBT program tailored for adults with SCD (CBT); or 2) pain and SCD education (Education) for reducing pain symptoms. The secondary goal was to assess whether baseline depression symptoms moderated the effect of these interventions on pain outcomes. Methods: Cognitive Behavioral Therapy and Real-Time Pain Management Intervention for Sickle Cell via Mobile Application (CaRISMA) is a multisite, randomized, pragmatic, comparative effectiveness trial conducted at seven comprehensive sickle cell centers and several community-based organizations in the U.S. The study enrolled adults with SCD who reported chronic pain or using short or long-acting opioids daily. Participants were randomized in a 1:1 ratio to receive either the CBT or Education programs. Both programs utilized identical Facebook Messenger chatbot apps, only the content differed. All intervention participants received health coach support involving weekly phone calls or text messages for a duration of 12 weeks. Participants completed follow up assessments at 3 and 6 months, and daily e-diary entries of 0-10 pain numerical rating scale, mood, and opioid use. The primary outcome was the 6-month change in the PROMIS pain interference. Secondary outcomes included average daily pain intensity for a 2-week period at each time point, change in mean % body area affected by pain (measured by a ‘paintable’ body image within the mobile app), PHQ-9 depression, GAD-7 anxiety, Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) quality of life (social functioning and emotional impact), and Sickle Cell Self-Efficacy Scale (SCSES). Generalized linear mixed models were used to compare changes in 6-month outcomes between study arms after accounting for design variables (study site and baseline depression). Results: Of the 574 participants screened for eligibility, 359 (63%) were consented and randomized (178 to CBT and 181 to Education). Seventy-five percent of participants connected with the chatbot but only 47% completed at least one CBT/education lesson. However, 80% of participants had at least one text message, phone or video session with a health coach. At 6-months, there was a significant decline in pain interference within each arm (CBT -2.13, 95%CI (-3.42, -0.84) and Education -2.66, 95%CI (-3.97, -1.36)); however, this decline did not differ between arms (p=0.57). There was not a significant 6-month change in daily pain intensity for either arm, however, for % body area covered in pain, both CBT and education conferred a similar relative decrease, 19.2% and 17.1%, respectively. There were significant 6-month improvements within-arms for PHQ-9, GAD-7, and both ASCQ-Me measures, but no between-arm differences emerged. (Table 1). Of these, only the 6-month change in ASCQ-Me emotional impact approached a significant difference between arms with digital CBT conferring slightly greater improvement in scores, 3.51 (95%CI: 2.29, 4.73) compared to education,1.79 (95%CI: .55, 3.04; p=.05). Baseline PHQ depression score (>= 10 vs < 10) did not moderate the effect of treatments on pain interference (p=0.52). Conclusions: Preliminary trial findings suggest that both digital CBT and Education, with health coach support, are effective approaches for management of SCD pain and mental health symptoms. Most participants connected with a health coach and may have derived benefit from this support, however, poor engagement with the digital CBT and education component of the study may have limited the study's ability to detect between arm differences. Secondary analyses will examine the effect of intervention engagement on treatment outcomes.
Introduction: Adults living with sickle cell disease (SCD) most frequently seek medical care due to pain. To determine the most efficacious treatment plan for patients presenting with pain, providers ...must first accurately assess and diagnose the pain. Unfortunately, the current approaches for assessing pain are inadequate. Combined with medical provider biases, patients can often have their pain symptoms misinterpreted, ignored, or blatantly dismissed. To address this issue, we partnered with stakeholders, human-centered designers, and software engineers to design a novel pain assessment tool, called Painimation. Painimation allows patients to communicate their pain quality, intensity, and location using abstract animations and a paintable body image. Painimation has been validated in a general population with chronic pain, but there is limited data validating this approach in SCD. Preliminary data on the use of Painimation by adults with SCD (N = 67) found that those who described their pain using the “throbbing” animation had less severe pain symptoms than those endorsing the “shooting” animation. Objective: To replicate and extend prior findings by determining whether pain animations and body image data are associated with pain outcomes in a large cohort of adults with SCD. We hypothesized that the presence of shooting pain and greater body surface areas affected by pain would be associated with more severe pain outcomes and mental health symptoms. Methods: We performed a secondary analysis on baseline data from the “Cognitive Behavioral Therapy and Real-Time Pain Management Intervention for Sickle Cell via Mobile Application (CaRISMA)” Trial-a multisite randomized, controlled trial in adults with SCD and chronic pain. Eligible patients were randomized 1:1 to either digital cognitive behavioral therapy or digital education; in addition, both arms received at least 12 weeks of health coach support. At baseline, participants completed a battery of questionnaires and tracked their pain intensity (0-10) by the Visual Analog Scale (VAS) and mood daily via a mobile app. The Painimation app presents a front and back 2-dimensional body image that is paintable to indicate areas affected by pain. Users choose from abstract animations intended to represent different pain qualities; the intensity of the animations can be adjusted, and up to three can be selected. For the purpose of the analyses, participants were categorized into “Shooting Pain” vs “No Shooting Pain” and “Throbbing with Shooting and Stabbing” vs “Throbbing Alone,” based on our prior study. Participants were also split into groups based on whether the proportion of the body image painted was less than the median (<9.8% vs >=9.8%). Baseline characteristics and demographics were compared between groups, and multivariable regressions were used to estimate covariate-adjusted associations with the following outcomes at baseline: daily pain, Patient Reported Outcomes Measurement Information System (PROMIS) pain interference scale, Pain Catastrophizing Scale (PCS), Current Opioid Misuse Measure (COMM-9), and Adults Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) pain severity and frequency. Results: The trial enrolled 359 adults, mean age 36.3 (SD = 10.5), 66% female, 93% Black race. The “Shooting” painimation and greater body image scores were associated with all outcomes in univariate analyses (all p<0.01; Table 1) except for the proportion of “happy” mood days and anxiety scores. After controlling for age, depression, anxiety, % body image, and site, the shooting animations were independently associated with greater daily pain intensity (beta = 0.64; p = 0.046). After controlling for age, depression, and site, greater body image score was associated with daily pain intensity (beta = 1.18; p < 0.001), pain interference (beta = 2.87; p < 0.001), ASCQ-Me pain frequency (beta = 3.91; p =< 0.005), and ASCQ-Me pain severity (beta = 4.50; p = 0.002). Conclusion: Both the “shooting” animation and body image measures were associated with more severe pain outcomes. This study demonstrates that animations and body image data can be used to assess pain severity in SCD, more objectively than the 0-10 numeric VAS scale. Future studies should explore whether pain location and specific animation selected are associated with pain etiology, and determine whether this approach can differentiate different types of pain in SCD.
Depressive symptoms are prevalent in individuals living with sickle cell disease (SCD) and may exacerbate pain. This study examines whether higher depressive symptoms are associated with pain ...outcomes, pain catastrophizing, interference and potential opioid misuse in a large cohort of adults with SCD. The study utilized baseline data from the 'CaRISMA' trial, which involved 357 SCD adults with chronic pain. Baseline assessments included pain intensity, daily mood, the Patient Health Questionnaire (PHQ), the Generalized Anxiety Disorders scale, PROMIS Pain Interference, Pain Catastrophizing Scale, the Adult Sickle Cell Quality of Life Measurement Information System and the Current Opioid Misuse Measure. Participants were categorized into 'high' or 'low' depression groups based on PHQ scores. Higher depressive symptoms were significantly associated with increased daily pain intensity, negative daily mood, higher pain interference and catastrophizing, poorer quality of life and a higher likelihood of opioid misuse (all p < 0.01). SCD patients with more severe depressive symptoms experienced poorer pain outcomes, lower quality of life and increased risk of opioid misuse. Longitudinal data from this trial will determine whether addressing depressive symptoms may potentially reduce pain frequency and severity in SCD.
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.
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Osunkwo: Novartis Pharmaceuticals: Consultancy, Honoraria, Speakers Bureau; FORMA Therapeutics: Consultancy; Global Blood Therapeutics: Consultancy, Speakers Bureau; Chiesi: Consultancy; Acceleron: Consultancy; Cyclerion: Consultancy; Emmaus: Consultancy. Minniti: Roche: Consultancy, Honoraria; NovoNordisk: Consultancy, Honoraria; Novartis: Consultancy, Honoraria; GBT: Consultancy, Research Funding. 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.
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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