ASH's commitment to SCD: building on 10 years of progress

TO THE EDITOR:

ASH’s commitment to SCD care and research

The American Society of Hematology (ASH), the largest professional society representing hematologists, has long recognized the urgent global health challenges posed by sickle cell disease (SCD), a prevalent genetic disorder that affects millions and disproportionately impacts populations with limited comprehensive health care access.

ASH Sickle Cell Disease Summit: a call to action

In 2015, ASH launched its first disease-focused strategic investment in SCD, convening a summit of >80 researchers, clinicians, and other stakeholders to prioritize actions for change. The Summit produced the first State of Sickle Cell Disease Report (2016), which has since guided ASH’s multipronged strategy to address critical research, care delivery, workforce development, and policy gaps. Now, a decade later, ASH is reflecting on its role as convener, collaborator, funder, and catalyst in the SCD community. This commentary provides a descriptive overview of ASH’s progress across the domains established in the National Academies of Sciences, Engineering, and Medicine (NASEM) 2020 report “Addressing Sickle Cell Disease: A Blueprint for Action” and highlights both achievements and ongoing opportunities for future impact.

Data infrastructure

The aim of NASEM strategy A is to establish a national system to collect and link data to characterize the burden of disease, outcomes, and the needs of those with SCD across the life span.

Robust data are essential for understanding disease burden and accelerating discovery. ASH and stakeholders identified the need to establish a comprehensive data infrastructure to assess clinical needs, evaluate care patterns, and measure outcomes. In addition, a priority stated in ASH’s State of Sickle Cell Disease 2016 report was to increase participation of individuals with SCD in clinical research, recognizing that data collection benefits from stakeholder involvement in its use.

In 2018, ASH launched the ASH Research Collaborative (ASH RC) Data Hub, one of the largest sources of aggregated, community-informed SCD real-world data in the United States. In addition to the Data Hub, the ASH RC hosts a SCD Research Network that, from the outset, included individuals living with SCD, their caregivers, community, and faith-based organizations in its design through a series of engagement workshops held across the country.¹ The Research Network develops and executes multicenter clinical studies and SCD community members, including individuals living with SCD, serve on the ASH RC committees and advisory boards, and provide valuable input and leadership.

Figure 1 reveals the locations of the 50 ASH Research Network and Council sites across the United States, as compared with the geographic distribution of individuals living with SCD. Collectively, these sites provide care for ∼37 800 individuals with SCD, accounting for more than one-third of the total US population affected by the disease.²

Figure 1.

ASH Research Collaborative SCD research network site map, overlaid on geographic distribution of people with SCD. Adapted from El Rassi² with permission. Adapted from Hassell³ with permission.

Evidence-based, accessible care

The aim of NASEM strategy C is to strengthen the evidence base for interventions and disease management and implement widespread efforts to monitor the quality of SCD care.

The need for accessible, quality SCD care has been unmet for decades, leading to wide clinical practice variation and few evidence-based quality metrics to use. The lack of a comprehensive system to standardize and monitor quality SCD care across different settings means that disparities and poor clinical outcomes often go undocumented and unaddressed.

To address the need for evidence-based guidance in SCD care, ASH convened a diverse panel of experts and patient representatives in 2016 to develop 5 comprehensive clinical guidelines, covering key domains such as organ complications, transfusion, cerebrovascular disease, pain, and stem cell transplantation. ASH created decision aids, point-of-care tools, educational resources, and patient materials with partners such as the Centers for Disease Control and Prevention, ensuring evidence translates into practice. Globally, ASH expanded access to SCD care through the Consortium on Newborn Screening in Africa (CONSA), launched in 2020 across 7 countries (Figure 2). CONSA has screened >160 000 newborns, diagnosed >2000 children, and introduced standard care and early interventions to improve survival in regions where most children with SCD die.⁴

Figure 2.

Consortium on newborn screening in Africa site map. Adapted from Hematology.org⁴ with permission.

Workforce development

The aim of NASEM strategy D is to increase the number of qualified health professionals providing sickle cell disease care.

A longstanding, intractable challenge for those with SCD is the shortage of adult providers able to manage their complex needs.⁵ Consequently, adult SCD care is fraught with frequent care gaps and limited multidisciplinary infrastructure, leading to increased acute care utilization and suboptimal outcomes.

This challenge requires multiple stakeholders. ASH has made progress by highlighting this issue and catalyzing change. Since 2019, the ASH SCD Centers Workshop has convened multidisciplinary teams from 58 hospitals across North America, fostering collaboration and innovation in the development of comprehensive care centers. These workshops became a resource for peer learning, operational problem-solving, and strategic planning, thus building a broader network of sustainable care infrastructure. As part of its core mission, ASH has also developed a portfolio of training and research awards designed to foster and empower career aspirations of future hematologists.⁶ These programs have supported a new generation of SCD-focused clinicians and researchers, expanding capacity both domestically and globally.

Research and development

The aim of NASEM strategy H is to establish and fund a research agenda to inform effective programs and policies across the life span.

Despite notable advances in SCD research in recent years, a bold and coordinated research investment is needed to develop targeted interventions that can transform outcomes and improve quality of life. Critical areas such as transition to adult care, chronic complication management, mental health, quality of life, and the impact of structural racism and social determinants of health remain poorly studied in SCD. Without a coordinated plan, inconsistent approaches that fail to address health inequities will persist.

Therefore, ASH outlined a comprehensive set of research priorities to guide progress, which will be updated every 5 years.⁷ This roadmap identifies critical unanswered questions and strategic opportunities, aiming to accelerate breakthroughs to benefit the global SCD collective. ASH calls on the broader SCD network to embrace collaborative models to advance these priorities through inclusive, impactful, and stakeholder-aligned goals. Simultaneously, ASH has invested >$18 million to support >130 SCD-focused initiatives through fellowships, research grants, and physician training programs, thereby strengthening the pipeline of clinicians, investigators, and institutional leaders focused on these research priorities and the overall quality of SCD care.⁸

Public education, awareness, and counseling

The aim of NASEM strategy E is to improve SCD awareness and strengthen advocacy efforts through targeted education and strategic partnerships.

SCD remains poorly understood by the general public, health professionals, and policymakers, leading to underrecognition of SCD’s burden, increased stigma, and inadequate patient and family support. Lack of awareness can perpetuate bias in health care settings, where patients often encounter skepticism about their pain and treatment needs, and limit the political will and resource allocation to improve care. Fragmented advocacy efforts further dilute the visibility and impact of the SCD community’s voice. Without coordinated education campaigns and strong partnerships among patient groups, providers, policymakers, and the media, SCD will continue to be marginalized.

In the past decade, ASH has paired hematology experts with federal, state, and community advocacy efforts to promote SCD as a national health priority. It has hosted 7 US Congressional briefings, helped secure passage of the 2018 Sickle Cell Disease Surveillance, Prevention, and Treatment Act (the first federal SCD data program) and advanced global awareness through briefings at the World Health Assembly, United Nations, and leadership of the World SCD Coalition. A cornerstone of these efforts is the Sickle Cell Disease Coalition, launched in 2016 with >100 partners globally, which amplifies the community’s voice, fosters collaboration, and provides regular meetings and educational opportunities to build a sustainable SCD network.

Conclusion

Although ASH has helped catalyze progress in SCD, much work remains. To align its programs and partnerships, ASH established the Center for Sickle Cell Disease Initiatives in 2024, recognizing that lasting change requires transparency, trust, and community collaboration. This update reviews achievements and reaffirms ASH’s commitment to shared action. We invite all stakeholders to build the systems, workforce, and evidence basis needed to ensure uniform high-quality care. By sharing resources, supporting research, and contributing expertise, partners can help shape the future of SCD care with improved outcomes, quality of life, and innovation.

Conflict-of-interest disclosure: The authors declare no competing financial interests.