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According to a story from Physician’s Weekly, paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease that can inflict a range of symptoms, one of which is fatigue. While the disease does have treatment options, new tools are needed in order to measure the effectiveness of treatment. One tool that could be used to measure fatigue in people living with the disease is the Functional Assessment of Chronic Illness Therapy Fatigue scale (FACIT-fatigue). A recent study sought to utilize the tool in the disease for the first time.

Findings

The research drew on data from the PNH registry which included information from 423 patients. These were patients that had begun to be treated with eculizumab within a 28-day period after being enrolled in the patient registry. The researchers hoped to learn how much of a change on the scale was necessary to conclude that a patient had experienced significant improvement in their fatigue.

The results found that a five-point change on the FACIT-fatigue scale was sufficient to produce a Clinically Important Change (CIC) in fatigue for PNH patients. At six months of treatment, 65% of patients saw a CIC on FACIT-fatigue. The scientists recommend that fatigue in these patients should be monitored regularly as a routine component of disease management.

About Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disease. This life-threatening illness is most characterized by the destruction of red blood cells by a part of the innate immune system called the complement system. Paroxysmal nocturnal hemoglobinuria is an acquired disease, meaning that it is not caused by genetic abnormalities. It can appear on its own or alongside other blood diseases, such as myelodysplastic syndromes or aplastic anemia. The disease occurs because of a defect in a protein that appears on the surface of red blood cells called DAF. Symptoms of the disease include red urine (especially in the morning), palpitations, blood clots, fatigue, shortness of breath, pain when swallowing, abdominal pain, and erectile dysfunction. Blood clots are a primary cause of death for patients with paroxysmal nocturnal hemoglobinuria. This disease is best treated with either eculizumab or, more recently, ravulizumab where it is available. To learn more about paroxysmal nocturnal hemoglobinuria, click here.