Shining a Light on the Unseen Challenges and Opportunities in Sickle Cell Disease

By Kim Smith-Whitley, M.D., GBT EVP and Head of Research and Development

Historically, sickle cell disease (SCD) has been neglected when it comes to awareness, innovation and access to high quality care. To support the global SCD community and better understand the current challenges they face, Global Blood Therapeutics (GBT) conducted the Sickle Cell Health Awareness, Perspectives and Experiences (SHAPE) survey, a multi-national study of more than 1,300 patients, caregivers and healthcare professionals. The results were presented at the European Hematology Association Annual Congress and the Global Congress on Sickle Cell Disease in June 2022.?

The survey reveals what patients and caregivers identify as the most significant disease-related challenges in their everyday lives: fatigue (84%), pain (71%) and mental health challenges (62%) were identified as the most significant by patients. In addition, the long-term impact of SCD remains a top concern and most patients (93%) agreed that reducing their risk of long-term health issues due to SCD, such as organ damage, was important to them.

The challenges facing caregivers are intuitively recognizable, but there is not a lot of data in this area. From SHAPE, we learned that symptom burden of patients negatively impacted the life of more than half of caregivers surveyed with respect to their career and education (56%), long-term health prospects (55%) and overall well-being (53%).

The SHAPE results also show that too often there is a disconnect between SCD patients and their healthcare providers (HCPs), creating barriers to optimizing care. Nearly one-third (31%) of HCPs found it challenging to understand patients’ concerns, with 43% citing difficulties due to having a different ethnic background from their patients. Our ability to improve future outcomes and promote effective long-term management depends on regular and effective communication between patients and HCPs. Over three-quarters (83%) of providers said they would like more support to educate their patients about the long-term effects of SCD, and only 50% feel they have sufficient tools to manage this long-term damage.

Broadening the Trust Equation

As a practicing hematologist for more than 30 years, these results ring true and clear with conversations I’ve had in the clinic. And having engaged with HCPs outside of SCD specialty centers and in other countries, I can appreciate the challenges many face. The SHAPE survey told us that 83% of patients trust specialist doctors more than other HCPs to offer new treatments, and 75% feel specialist doctors address the long-term impacts of SCD.

It is clear we need to expand education and broaden the trust equation in SCD across the healthcare system. While we have long known that sickle cell experts are a key element to improving the delivery and quality of care for SCD patients, this type of care is not readily accessible to all patients with SCD. And for the SCD community, the challenges SHAPE exposed are often unseen and ignored. We view this as a fundamental health equity issue and believe the path to solutions begins with understanding the needs of the SCD community so that we can develop treatments that are not only effective but also tolerable and scalable.

SHAPE has provided insight into the global impact of SCD and the opportunities for GBT to support these communities as it expands. This includes the long-term goal of providing access to innovative therapy for millions of patients in limited-resource countries.

SHAPE was made possible by a monumental effort. Thank you to all the participants from the 10 countries represented in the SHAPE survey, the SCD expert clinicians and patient advocacy leaders on our steering committee, and to my GBT colleagues and extended teams. Learn more at gbt.com/shape.

September is Sickle Cell Awareness Month

September marks the 46th year we celebrate Sickle Cell Awareness Month. At the core of achieving the goal to transform SCD into a manageable chronic condition is driving awareness of its burdens and continuing to understand how it impacts those affected. SHAPE has allowed us to shine a bright light on the current experience of living with, treating and caring for individuals with this devastating disease. We know there is much work to be done, and we will continue to support the needs of the SCD community. Check out #SickleCellAwarenessMonth to learn more about SCD and to see how some GBTers are raising awareness.?