Science made simple
What did scientists want to find out?
PNH is a rare disease. Scientists are still learning how the symptoms of PNH affect people’s daily lives.
In this study, the researchers measured the level of fatiguefatigueExtreme tiredness or exhaustion. Fatigue is one of the most common symptoms of PNH. that people with PNH experienced and compared it against other bone marrow diseases. The people in the study had one or more bone marrow failure diseases: myelodysplastic syndrome (MDS)Myelodysplastic syndromes (MDS), aplastic anemia (AA)Aplastic anemiaA rare blood disease that is a known risk factor for PNH. Aplastic anemia occurs when a person’s bone marrow does not make enough new blood cells. Some scientists believe that aplastic anemia weakens the body’s bone marrow, which then affects its ability to produce healthy red blood cells. People with PNH can share symptoms with those that have aplastic anemia, such as low blood cell counts. and PNHParoxysmal nocturnal hemoglobinuria (PNH)A rare disease in which the body attacks and destroys its own red blood cells, leading to fatigue, weakness, headaches and other symptoms, including chest pain and abdominal pain.. These 3 diseases have similar effects on the body, including fatigue.
How, where and when did they look for information to analyze?
The AA and MDS International Foundation — AAMDS for short — is a nonprofit group that supports people with bone marrow disorders and their families. They surveyed people with bone marrow disorders about how often they experienced certain symptoms.
Researchers used surveys from 303 people, and focused on the topics of fatigue, pain and emotional challenges, including depression, anxiety and stress.
Participants completed 3 surveys, which were pulled from various disease areas to measure different symptoms associated with these conditions:
The Functional Assessment of Cancer Therapy – Anemia, or FACT-An for short, which measures anemia symptoms that stem from cancer therapy
The brief pain inventory, or BPI, which measures the severity of pain and its impact on daily activities
The Depression, Anxiety and Stress Scale, or DASS-21, which measures depression, anxiety and stress
Many elements can contribute to fatigue, and there are many different ways to manage it. The researchers asked participants how often they used certain strategies, like:
- prescription stimulants
- over‐the-counter supplements
- eating healthily
- taking naps
- counseling/support groups
- preserving energy
Then, the researchers rated how helpful these strategies had been over the course of a month.
What did the researchers learn?
The researchers used descriptive statistics to measure the levels of fatigue, pain and emotional challenges taken from the surveys. Descriptive statistics are a way of reporting information in a way that is simple and easy to understand.
The researchers found that:
There were no significant differences among the groups studied.
Fatigue for the groups studied was considered severe.
Scores for stress were normal.
Scores for pain and depression were mild.
Scores for anxiety were moderate.
Quality of life
Across all groups studied, the average score for quality of life was 68 out of a maximum of 104, which is lower than average scores for a healthy population.
Strategies for fatigue
The 3 strategies most people found helpful for fatigue were preserving energy, physical activity, and naps.
What did the researchers conclude from the study?
Many individuals with rare bone marrow disorders experience severe fatigue
Strategies like rest, naps and regular exercise helped many, but may not work well for everyone
More study is needed to learn whether these and other strategies help to significantly lower fatigue
- Title: Fatigue, symptom burden, and health‐related quality of life in patients with myelodysplastic syndrome, aplastic anemia, and paroxysmal nocturnal hemoglobinuria
- Journal: Cancer Medicine, February 2019; 8(2):543-553
- Written by: Escalante, C. P., Chisolm, S., Song, J., Richardson, M., Salkeld, E., Aoki, E., & Garcia-Manero, G.
Science made simple
Read more about PNH discoveries and research
- Understanding the disease burden of PNH
- The effects of PNH in children versus in adults
- People with PNH can develop blood clots, even if they don’t have significant hemolysis
- PNH may be caused by a different genetic mutation
Detecting PNH in bone marrow samples
instead of blood samples
Please note: The information on this page is meant to be informational only and is not intended to replace medical advice. Always talk to your healthcare provider about any questions you may have on PNH, its symptoms or treatment.