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New data at ASCAT congress suggest significant burden of sickle cell disease (SCD) on patients, caregivers and healthcare systems

New data at ASCAT congress suggest significant burden of sickle cell disease (SCD) on patients, caregivers and healthcare systems


UK study showed living with SCD reduces productivity and decreases potential future income by more than £500,000 for an already underserved patient population


Additional data demonstrated the importance of appropriate treatment measures to increase haemoglobin levels and reduce hospitalisations in people with SCD



LONDON, October 21, 2022 – Global Blood Therapeutics (GBT), a part of Pfizer, today announced results from a new UK study on productivity-loss and lifetime-income-loss for patients with sickle cell disease (SCD), that estimates those who cannot work or die of their disease lost more than £500,000 of potential future income over their lifetime. In addition, a retrospective analysis of over 6,000 patients with SCD in the UK, demonstrated that those with more severe anaemia (as measured by lower haemoglobin [Hb] levels) were more likely to be hospitalised than those with less severe anaemia (as measured by higher Hb levels).  These data, as well as UK findings from the Sickle Cell Health Awareness, Perspectives, and Experiences (SHAPE) Survey, which further confirmed the physical, emotional and societal burden of SCD on quality of life for people with SCD and their caregivers, were presented at the Annual Scientific Conference on Sickle Cell and Thalassemia (ASCAT) congress, held 20-22 October 2022, in London, UK.


“Sickle cell disease places a significant burden on all aspects of the lives of people living with this condition, also impacting their caregivers and healthcare systems. These results from the UK, show that effective disease management to prevent hospitalisations and improve quality of life is vital. However, it is also important to understand that ongoing medical appointments, acute crises and the day-to-day reality of living with SCD, means people are often left unable to work, resulting in the loss of potential income, through no fault of their own,” said Kim Smith-Whitley, Site Head, Advisor of Scientific & Clinical Affairs at Global Blood Therapeutics. “The data presented at ASCAT reminds us of the need to work together to make a positive change for those living with SCD and those who care for them.”


SCD affects approximately 15,000 people in the UK.1 People living with SCD experience progressive, serious complications and morbidities, including organ damage, which lead to decreased quality of life and early mortality.2 Furthermore, economic disadvantages and health inequalities experienced by many patients with SCD can have negative societal impacts in areas such as access to healthcare, education and employment.3-9


“The studies presented at ASCAT confirm what the Sickle Cell Society has been aware of for a long-time – people living with SCD are impacted by serious health inequalities and living with this debilitating condition can have a major impact on people’s earning potential. This loss of income can further perpetuate the health, financial and educational inequalities across generations of families living with SCD,” said John James OBE, Chief Executive, Sickle Cell Society, UK “We will be using this data to support our ongoing work to help secure improvements to the care of people with SCD in the UK, in the hope this will lead to better outcomes for patients and their families.”


Societal Burden of Sickle Cell Disease in the UK

In the first UK study (9272 patients aged > 12 years) to show empirical estimates for productivity loss (PL) and loss of future income (LFI), people living with SCD were found to lose the equivalent of >3 weeks’ average 2021 UK weekly earnings (£586) per year due to PL. In total, (GBP) £510,315 LFI was estimated over a lifetime, for each patient with an LFI event, equivalent to £30,546 per patient in the cohort. Of the 555 LFI events, the majority 425 (76.6%) were deaths, occurring at a median age of 61 years. Unemployment accounted for 129 (23.2%) events occurring at a median age of 39 years.


“Routine healthcare appointments, acute crises and organ damage combine to result in productivity loss, while it is estimated that premature mortality and long-term unemployment may lead to people losing over £500,000 of potential future income. This enormous financial impact needs addressing as a moral and public health priority by healthcare providers and governments around the world,” said Martin Besser, Consultant in Haematology, MRCP Addenbrooke’s Hospital, Cambridge UK.



SHAPE was administered as an online survey in 10 countries to understand the impact of SCD and quality of life (QOL) of patients and caregivers. The SHAPE data presented at ASCAT provides results from the UK compared to the other countries; of 919 patients and 207 caregivers interviewed globally, 151 patients and 30 caregivers were from the UK. The symptoms more frequently reported by > 60% patients in the UK compared to the global data were low mood/feeling down/depressed (74% UK vs 62% total), poor sleep/insomnia (72% UK vs 60% total), headache (71% UK vs 60% total), poor mobility/movement (70% UK vs 46% total), and joint stiffness (62% UK vs 50% total). More than half of the caregivers in the UK felt that caring for someone with SCD affected their earning potential (57% vs 54% total) and the ability to attend and succeed at school or work (53% vs 56% total). These UK results align to the global data in confirming the significant impact SCD has on quality of life for those living with the disease, as well as their caregivers.


“The results of the UK SHAPE survey confirm that effective disease management is essential to improve quality of life for people with SCD, helping them to maximise their potential in life and work. It is also important that caregivers are given the support they need, particularly as caring for someone with SCD impacts their own ability to work or attend school and also impacts their earning potential,” said Dr. Baba Inusa, professor and consultant of paediatric haematology, Guy’s and St Thomas’ NHS Foundation Trust, London and chair of the National Haemoglobinopathy Panel in England.


Hospitalisations and Annual Bed Days for Patients with SCD and Varying Levels of Anaemia

The study aimed to quantify differences in all-cause hospitalisations and annual bed days in patients with SCD and differing levels of anaemia. Results showed patients with less severe anaemia (as measured by higher levels of Hb) had progressively reduced hospitalisations and annual bed days compared to patients with more severe anaemia (lower levels of Hb). Of 6,018 patients with recorded Hb, 72% had a history of Hb >10.5 g/dL, 44% had 8 to 10.5 g/dL, 15% had 6 to 8 g/dL, and 3% had <6 g/dL. The annual rate of all-cause hospitalisations decreased as Hb increased: 5.8 in patients with Hb <6 g/dL, 4.1 for 6 to 8 g/dL, 3.3 for 8 to 10.5 g/dL, and 1.7 for >10.5 g/dL. Hospitalisations were lower for patients with a history of Hb >10.5 g/dL than for those with Hb <6 g/dL (P<0.001).  Hospitalisations related to end organ damage (EOD) were also lower for those who had a history of Hb >10.5 g/dL compared with those who had a history of Hb <6 g/dL (0.4 and 2.4 hospitalisations per patient interval per year, respectively; P<0.001).


“We know that anaemia in SCD is associated with an increased risk of complications such as leg ulcers and end-organ damage (e.g. pulmonary hypertension, renal insufficiency) that often require hospitalisation, which is a key driver of healthcare costs and burden of illness. These results suggest hospitalisations were reduced in people with higher levels of haemoglobin. Put simply, those with more severe anaemia are more likely to end up in hospital, highlighting the need for appropriate treatment measures to optimise haemoglobin levels in patients with SCD to reduce hospitalisations,” said Professor Paul Telfer, Clinical Professor of Haemoglobin Disorders and Haematology, Queen Mary, University of London.


About Sickle Cell Disease 
It is estimated that more than 100,000 people in the United States,10 approximately 52,000 people in Europe,11 up to 100,000 people in Brazil,12 and millions of people throughout the world have sickle cell disease (SCD).10 SCD occurs particularly among those whose ancestors are from sub-Saharan Africa; Spanish-speaking regions in the Western Hemisphere (South America, the Caribbean, and Central America); Saudi Arabia; India; and Mediterranean countries such as Turkey, Greece, and Italy.10 SCD is a lifelong inherited blood disorder that impacts haemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body.13 Due to a genetic mutation, individuals with SCD form abnormal haemoglobin known as sickle haemoglobin. When sickle haemoglobin becomes deoxygenated, it polymerises to form rods, which deforms the red blood cells into sickled – crescent-shaped, rigid – cells.2,14,15 The recurrent sickling process causes destruction of the red blood cells, haemolysis and anaemia (low haemoglobin due to red blood cell destruction), which drives vascular inflammation contributing to blockages in capillaries and small blood vessels (vaso-occlusion) that impede the flow of blood and oxygen delivery throughout the body. Episodes of painful vascular occlusions are commonly referred to as vaso-occlusive crises (VOCs). The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.2, 14-17 Complications of SCD begin in early childhood and can include neurocognitive impairment, acute chest syndrome, and silent and overt stroke, among other serious issues.18  Early intervention and treatment of SCD have shown potential to modify the course of this disease, reduce symptoms and events, prevent long-term organ damage, and extend life expectancy.2




About Global Blood Therapeutics

Global Blood Therapeutics (GBT) is a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities, starting with sickle cell disease (SCD). Founded in 2011, GBT is delivering on its goal to transform the treatment and care of SCD, a lifelong, inherited blood disorder. As of October 5, 2022, GBT is a subsidiary of Pfizer.



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Claudia Nabaie (media Europe)
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Last Updated: 24-Oct-2022