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16th December 2021
In patients with sickle cell disease, an early awareness of the risks associated with COVID-19 infection and acceptance of virtual appointments, resulted in a significant reduction in morbidity and mortality among those with the condition. This was the finding of a study by researchers from the Department of Haematology and Medical Oncology, Emory University School of Medicine and Georgia Comprehensive Sickle Cell Centre at Grady Health System, Atlanta, US presented at ASH 2021.
The Grady Comprehensive Sickle Cell (SC) Center is the largest adult sickle cell centre in the US with the first 24/7 acute care unit for the management of sickle cell vaso-occlusive events (VOE). In 2019, the centre provided 3077 outpatient appointments for patients with sickle cell disease (SCD) and 3695 acute care visits. However, the arrival of the COVID-19 pandemic led to a huge drop in the number of both outpatient and acute care visits. But how the virus impacted on patients who were registered with the centre was the subject of the study presented by the Atlanta researchers at the ASH Conference 2021. The team made use of the Grady centre’s database, which was used for tracking the outcomes of registered patients during the COVID-19 pandemic and to date, represents the largest single-centre study on COVID-19 in people with SCD between March 2020 and March 2021.
From a total of 1343 patients in the database, 55 patients with average age of 28 years (51% female) contracted COVID-19. Among the 55 who tested positive for COVID-19, only 16 required treatment and of whom, 2 died.
In terms of COVID-19 symptoms, 58% experienced pain as the main symptom, followed by cough and fever (40%), dyspnoea (31%), and pneumonia with chest x-ray evidence (25%). Two patients developed acute respiratory distress syndrome (ARDS) and were intubated. Interestingly, the two deaths occurred early in the course of the pandemic in June and July 2020 when 20 total cases were diagnosed and there were no deaths recorded between October 2020 and March 2021. Due to the COVID-19 restrictions, the Grady centre quickly adopted virtual visits to deliver healthcare to their patients.
Commenting on their findings, the authors considered that the early adoption of virtual visits aided in protecting patients against COVID-19 as witnessed by the absence of any deaths during second peak in the winter of 2021. The authors felt that this indicated how patient’s diligence and awareness to stay home during the pandemic, proved to be crucial in reducing morbidity and mortality. They concluded that the option of virtual visits for healthcare delivery was key and should be utilised further in sickle cell care.
El Rassi F et al. COVID-19 Infection and Outcomes at a Comprehensive Sickle Cell Center. ASH Conference 2021
14th October 2021
NICE has given the green light to crizanlizumab for the management of sickle cell crises in people with sickle cell disease. The term sickle cell disease describes a group of inherited red blood cell disorders that affect haemoglobin and which, according to the World Health Organization, approximately 5% of the world’s population carries trait genes for such disorders. Sickle cell disease affects around 1 in 500 African American children and 1 in 36,000 Hispanic American children and is characterised by a change in the shape of red blood cells which become more ‘sickle-like”, reducing their flexibility of the cells. These sickle-like red blood cells can lead to recurrent and unpredictable blockage of small blood vessels producing ischaemic pain, referred to as vaso-occlusion (VOC) or sickle cell crises. In addition, activated and adherent leukocytes are the likely drivers of VOC in collecting venules and this process appears to be initiated by a transmembrane protein, P-selectin. Studies have shown that blockage of P-selectin appears to improve blood flow and thus reduce the risk of VOC and sickle cell–related pain crises.
The monoclonal antibody crizanlizumab binds to P-selectin blocking its action. The approval by NICE was based on data from the SUSTAIN trial. This double-blind, randomised, placebo-controlled, Phase II trial, assigned 198 patients to either a low-dose crizanlizumab (2.5 mg per kilogram of body weight), a high-dose crizanlizumab (5.0 mg per kilogram), or placebo and which were administered intravenously 14 times over a period of 52 weeks. The primary outcome was the annual rate of sickle cell–related pain crises with high-dose crizanlizumab versus placebo. For the study, this was defined as acute episodes of pain caused by a VOC that resulted in a visit to a medical facility and treatment with pain relief medication. The results showed that the median rate of crises per year was 1.63 with high-dose crizanlizumab versus 2.98 with placebo (p = 0.01). In addition, the median time to the first crisis was significantly longer with high-dose crizanlizumab than with placebo (4.07 vs. 1.38 months, p = 0.001),as was the median time to the second crisis (10.32 vs. 5.09 months, p=0.02). In addition, the overall incidence of serious adverse event was comparable across the three arms.
NICE recognised that a limitation of the trial was the absence of longer term data on crizanlizumab such as mortality or among those who did not seek medical advice on VOCs. There was also no data on the prolonged treatment benefit and what happens when patients stop taking crizanlizumab.
While the appraisal document concluded that “Crizanlizumab is not recommended for routine use in the NHS“, the drug could be used where specific criteria are met. Thus, guidance notes that “crizanlizumab is recommended as an option for preventing recurrent sickle cell crises (vaso-occlusive crises) in people aged 16 or over with sickle cell disease only if the conditions in the managed access agreement are followed.“
Source. NICE 2021