About 15,000 people in the UK have Sickle Cell Disease (SCD). The Phase 3 HOPE study recently published in Lancet Haematology showed that treatment with an oral medication – Oxbryta – resulted in rapid and lasting improvements in haemoglobin levels throughout 72 weeks of treatment for the 274 participants involved.

Professor Jo Howard, consultant haematologist at Guy’s and St. Thomas’ NHS Foundation Trust and Professor of Haemoglobinopathies at King’s College London was the lead author on the study, sponsored by Global Blood Therapeutics.

Oxbryta is an oral and once-daily therapy, which directly inhibits the destruction of haemoglobin, the root cause of the sickling and destruction of red blood cells in Sickle Cell Disease.

The study revealed that Oxbryta tablets improve haemoglobin levels and overall health in adult and adolescent patients with Sickle Cell Disease.

It also showed significant and sustained improvement in haemoglobin levels, reduction in destruction of red blood cells (haemolysis) and improved overall health status in patients treated with the medications.

Approximately 90 percent of patients treated with Oxbryta achieved a hemoglobin improvement of >1 g/dL from baseline at one or more time points during the study compared to placebo (25 percent).

These findings support the long-term use of Oxbryta to reduce anaemia and haemolysis in Sickle Cell Disease, potentially mitigating life-threatening complications of the condition.

Lead author Professor Jo Howard said: “Sickle cell disease is a devastating disease that can lead to organ damage and a shortened life expectancy and is complicated by significant disparities in access to quality care.”

“Fortunately, we have entered a new era of treatment. The HOPE Study is the longest registrational trial to date among recently approved therapies for sickle cell disease, and these results further demonstrate that by sustainably improving both the haemolysis and anaemia manifestations of the disease, Oxbryta has the potential to be a safe and effective disease-modifying treatment in patients with sickle cell disease.”

The analysis also showed that study participants treated with Oxbryta had numerically fewer vaso-occlusive crises, and were three times less likely to experience an acute anaemic episode.

Approximately 74 percent of patients taking Oxbryta also had their overall clinical status rated as “moderately improved” or “very much improved” by their clinician compared with approximately 47 percent of the placebo group, a statistically significant difference. The study also evaluated the incidence and outcomes of leg ulcers in patients with Sickle Cell Disease. Results of the analysis showed leg ulcers improved or resolved by week 72 in all patients receiving Oxbryta compared with 63 percent of patients in the placebo group. Resolution of leg ulcers was associated with increases in haemoglobin levels and decreases in haemolysis.

Ted W. Love, M.D., president and chief executive officer of Global Blood Therapeutics said: “The sickle cell disease community, which for decades has been dramatically underserved, deserves treatments that address the sickling and destruction of red blood cells due to hemoglobin polymerization – the root cause of this disease.”

“The HOPE Study represents a significant milestone in advancing the treatment of SCD, and we are building on this ground breaking trial with our commitment to increase access to Oxbryta and develop novel therapeutics that can transform SCD into a well-managed disease.”

About Sickle Cell Disease
About 15,000 people in the UK have Sickle Cell Disease (SCD). Sickle cell disease affects three million of people throughout the world, particularly among those whose ancestors are from sub-Saharan Africa. It also affects people of Hispanic, South Asian, Southern European and Middle Eastern ancestry. Sickle Cell Disease is a lifelong inherited rare blood disorder that impacts hemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body.

Due to a genetic mutation, individuals with SCD form abnormal hemoglobin known as sickle hemoglobin. Through a process called hemoglobin polymerization, red blood cells become sickled – deoxygenated, crescent-shaped and rigid. The sickling process causes hemolytic anemia (low hemoglobin due to red blood cell destruction) and blockages in capillaries and small blood vessels, which impede the flow of blood and oxygen throughout the body. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.

This research was covered by The Voice Online.

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