16 December 2019

Progress is being made in the care of sickle cell disease patients at all levels, the annual meeting of the American Society of Hematology has heard.

A group of studies presented last week at the ASH annual meeting in Orlando, Florida, highlighted brand-new research findings and improved treatment.

Dr Jeffrey Shearstone and colleagues at Syros Pharmaceuticals reported on a new foetal haemoglobin repressor protein called nuclear factor IX, which they believe could help correct the red blood cell abnormalities in the disease.

Nuclear factor IX has a clear role in helping red blood cells switch from producing foetal haemoglobin to adult haemoglobin, representing a potential target for new drugs that would help people living with sickle cell disease make new healthy red blood cells.

A second study, in Africa, looked at the effectiveness of oral arginine supplements for pain relief in people with sickle cell disease. Children with the condition were given oral arginine supplements during vaso-occlusive crises. They reported less pain, needed less pain medication, and were discharged from hospital earlier than those given a placebo.

The trial, based in Nigeria, was led by Dr Richard Onalo of the University of Abuja, Nigeria, and involved 68 children.

He said: “Arginine is a cheap, readily available over-the-counter supplement. Oral arginine is a safe and useful product in patients experiencing painful crises. The supplement adds to the pain killing ability of conventional drugs and may lead to faster resolution of a crisis and a shorter hospital stay.”

Dr Julie Panepinto, of the Medical College of Wisconsin, USA, commented on these and other sickle cell disease trials presented at the meeting.

She said: “These studies together illustrate how progress is being made all the way from the laboratory bench to dissemination and implementation of better treatments and systems-level quality improvements.

“Researchers are moving the needle with studies both here in the US and around the world.”


Source: ASH December 2019