Paroxysmal nocturnal haemoglobinuria (PNH) is a rare blood disorder where the body’s immune system attacks red blood cells, causing them to break down too quickly. Researchers surveyed 113 Chinese patients with PNH to see how doctors were treating them and what problems they faced. They found that patients with more severe disease had higher risks of dangerous blood clots and experienced significant fatigue and anemia. Most patients were treated with blood thinners, steroids, or other medications, but doctors weren’t yet using newer treatments that target the root cause of the disease. This study shows there’s room to improve how PNH is managed in China.

The Quick Take

  • What they studied: How doctors in China treat patients with PNH (a rare blood disease) and what health problems these patients experience
  • Who participated: 113 patients with PNH in China, ages 16 and older, along with their doctors who reported on their care
  • Key finding: About half of the patients had severe disease with high levels of a marker called LDH. These sicker patients were much more likely to develop dangerous blood clots (50% versus 23%) and experienced more serious symptoms
  • What it means for you: If you or someone you know has PNH in China, this research suggests that current treatments help manage symptoms like fatigue and anemia, but newer medicines that address the root cause of the disease may offer better protection against blood clots. Talk to your doctor about all available treatment options.

The Research Details

Researchers conducted a survey between July and November 2022 where they asked doctors in China about their patients with PNH. Each doctor reported on up to 10 patients they had recently seen. The doctors provided information about their patients’ symptoms, test results, and what medicines they were taking. The researchers then compared patients with mild disease to those with severe disease to see if there were differences in their symptoms and treatments.

This type of study is called a ‘cross-sectional’ study, which means researchers took a snapshot of how things were at one point in time, rather than following patients over months or years. The information came from real-world medical practice, not from a controlled experiment, so it shows what actually happens in hospitals and clinics.

Understanding how doctors currently treat PNH in China is important because it shows what’s working and what might need improvement. By looking at real patients in real clinics, researchers can see if newer medicines are being used and whether patients are getting the best possible care. This information helps doctors make better decisions about treatment and helps identify gaps in care that need to be filled.

This study has some strengths: it included real patients from actual medical practice, and doctors reported on consecutive patients they saw (not just selected ones). However, the study has limitations: it only included 113 patients, which is a relatively small number for drawing broad conclusions; it was a snapshot in time rather than following patients over time; and it relied on doctors’ reports rather than independent verification of all information. The study was conducted in 2022, so treatment options may have changed since then.

What the Results Show

The study found that nearly half of the 113 patients (49.5%) had high levels of a marker called LDH, which indicates more severe disease. These patients with high LDH had noticeably worse symptoms and more complications than those with lower LDH levels.

The most common symptoms across all patients were extreme tiredness (fatigue) in 93% of patients and anemia (low red blood cell count) in 74% of patients. About 39% of all patients experienced hemolytic crises, which are episodes where red blood cells break down very rapidly, causing serious symptoms.

Blood clots were a major concern, especially in patients with high LDH. Half of the high-LDH patients (50%) experienced blood clots compared to only 23% of patients with lower LDH levels. This difference was statistically significant, meaning it’s unlikely to be due to chance.

Most patients were treated with blood thinners (40%), steroids like prednisone (31%), or an immunosuppressant drug called cyclosporine (27%). Patients with more severe disease were more likely to receive a blood thinner called rivaroxaban and vitamin B12 supplements.

The study found that patients reported moderate levels of fatigue on a standardized scale, indicating that tiredness was a significant quality-of-life issue even with current treatments. Doctors perceived the disease as more severe in high-LDH patients, suggesting they recognized the greater health burden in this group. The research also showed that current treatment approaches in China focused mainly on managing symptoms like anemia and preventing hemolytic crises, rather than targeting the underlying cause of the disease (uncontrolled complement activation).

This study provides a snapshot of PNH treatment in China at a specific time (2022). It shows that newer complement inhibitor medications, which were recently approved in China, were not yet widely used in this patient population. Previous research in other countries has shown that newer targeted treatments can reduce hemolysis and blood clot risk more effectively than traditional approaches. This suggests that Chinese patients may benefit from broader access to and use of these newer medications.

The study included only 113 patients, which is a relatively small sample size that may not represent all PNH patients in China. The data came from doctors’ reports rather than independent medical records, which could introduce reporting errors. The study was conducted at one point in time, so it doesn’t show how patients change over time or how treatment outcomes develop. The study didn’t include information about patient outcomes after treatment, so we don’t know how well the current treatments actually work. Additionally, the study was completed in 2022, and treatment options and practices may have changed since then.

The Bottom Line

If you have PNH in China: (1) Work closely with your hematologist to monitor your LDH levels and symptoms, as these indicate disease severity. (2) Ask your doctor about all available treatment options, including newer complement inhibitor medications if appropriate for your situation. (3) Take blood thinners as prescribed if you’re at risk for blood clots. (4) Report symptoms like unusual fatigue, shortness of breath, or signs of blood clots (swelling, pain) immediately to your doctor. Confidence level: Moderate - based on real-world data but with a small sample size.

This research is most relevant for: patients with PNH in China, their families and caregivers, hematologists and doctors treating PNH patients, and healthcare policymakers in China. People without PNH don’t need to apply these findings, but they may be interested in understanding rare blood diseases. Patients with PNH in other countries may find this helpful for understanding treatment approaches, though their local options may differ.

Improvements in symptoms and reduction in blood clot risk typically take weeks to months with medication adjustments. Fatigue may improve gradually over 4-12 weeks as anemia is better controlled. Blood clot prevention requires ongoing medication and monitoring. Newer targeted treatments may show benefits within weeks to months, but long-term benefits develop over 6-12 months of consistent use.

Want to Apply This Research?

  • Track weekly fatigue levels (1-10 scale), any symptoms of hemolytic crisis (dark urine, severe fatigue, shortness of breath), and signs of blood clots (leg swelling, chest pain, shortness of breath). Also log which medications you’re taking and any side effects.
  • Use the app to set reminders for taking blood thinners and other medications on schedule. Log your energy levels throughout the day to identify patterns and discuss with your doctor. Record any unusual symptoms immediately so you can report them at your next appointment.
  • Create a monthly summary view showing trends in fatigue, symptom frequency, and medication adherence. Share this data with your healthcare provider at each visit. Set alerts for concerning symptoms that warrant immediate medical attention, such as signs of blood clots or severe hemolytic episodes.

This research describes how PNH is currently treated in China and should not be used to diagnose or treat any condition. PNH is a serious medical condition requiring specialized care from a hematologist. If you have symptoms of PNH (severe fatigue, dark urine, shortness of breath, or unexplained blood clots), consult a qualified healthcare provider immediately. Treatment decisions should be made in consultation with your doctor based on your individual health status, not on this research summary. This study represents data from 2022 and treatment options may have changed. Always follow your doctor’s recommendations for your specific situation.