In an effort to improve clinical research, treatment, and outcomes for patients with sickle cell disease (SCD), the American Society of Hematology Research Collaborative (ASH RC) undertook a study to determine perceptions and factors involved in clinical trial participation among patients with SCD. Results of this study were recently reported in the journal Blood Advances.

In performing this study, ASH RC collaborated with the Center for Information & Study on Clinical Research Participation to provide 8 community-based, weekend workshops throughout the US during 2019. These workshops had multiple goals, such as to enhance understanding of life with SCD as a patient or caregiver, to assess satisfaction with existing treatment options, and to discuss various aspects of clinical research and outcomes. This study report focused on views of members of the SCD community regarding treatment needs and clinical research.

The workshops were based on an adapted community-based participatory research approach. Workshops were distributed across the US in locations based on geographic diversity and whether there was a sufficiently large population of patients with SCD. Transportation, food, and childcare assistance and gifts were provided for participants to facilitate involvement in the study.

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Patients were recruited from 4 populations within the SCD community, including parents of children with SCD, adolescents with SCD, young adults with SCD, and adults with SCD who were 40 years of age or older. Questions regarding clinical research involved participants’ perceptions of clinical research and its risks and benefits, their participation in clinical trials, and other related topics.

The top symptoms that participants reported would be targeted by an ideal therapy were acute pain/SCD crisis (30%), general pain (18%), and fatigue (12%). Participants highlighted frustrations with insufficient access to treatment, with affordability as a key issue. Additionally, participants expressed that it takes considerable time to determine the right medication for the patient and that long-term side effects associated with treatment were also a concern.

Most patients either strongly agreed (73%) or agreed (18%) that a cure was their top priority. However, some patients either disagreed (9%) or strongly disagreed (1%) on prioritization of a cure, noting that quality of life improvements may be more likely through treatments directed at symptoms.

Most participants expressed a willingness to be involved with clinical trials under certain conditions, however, many reportedly had not been asked to enroll in a clinical trial yet. Most participants had limited familiarity with clinical research. Participants also expressed a desire for more research opportunities because SCD is often an exclusion criterion for trials. Negative experiences in the clinic when in need of care also impacted some participants’ interest in involvement with clinical research. Most participants also expressed some level of willingness to share their health information for the betterment of research and outcomes.

“The ASH RC will continue to engage those living with SCD and their support networks to update these findings and ensure the voice of the community is incorporated in building and maintaining a network of clinical trials constructed with a shared vision for community-centric research,” the study authors wrote in their report.

Disclosures: Some authors have declared affiliations with or received grant support from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.


Lee LH, Whisenton LH, Benger J, Lanzkron S. A community-centered approach to sickle cell disease and clinical trials participation: an evaluation of perceptions, facilitators, and barriers. Blood Adv. Published online September 20, 2021. doi:10.1182/bloodadvances.2020003434