photo of doctor talking to patient

If you have PNH, you will find that your condition requires constant management. But it’s important to be able to confront it head-on. If you do this, you will be better able to manage your symptoms and live the life that you want. Here are some ways to get in control:

Pick a doctor that you trust. It’s very important that you find a doctor who has experience with PNH. Since it’s such a rare condition, this may be hard to find. The Aplastic Anemia and MDS International Foundation has a list of doctors who specialize in PNH. Your local hospital or medical center may also have suggestions as well. Some good questions to ask at your first visit include:

  • What is PNH?
  • What causes PNH?
  • How is PNH diagnosed?
  • How will you monitor my PNH to make sure it doesn’t get worse?
  • How will you manage my PNH?

It’s also important that you keep your medical provider aware of all your symptoms. Keep a diary of when they start, how often they happen, and how severe they are. This way, they can better help you manage them. 

Make an agenda for each doctor visit. You may not be able to cover everything at each provider appointment. It’s a good idea to write down the top three things you want to discuss, so you can focus on them. Bring a list of current prescription and over-the-counter medicines, vitamins, or supplements you use, as well as notes on symptoms and side effects. You may also want to bring your transfusion records. Don’t shy away from asking questions, either: Doctors expect them. It shows that you are involved in your care and want to stay informed.

Make sure you get the answers that you need. A diagnosis of PNH is complicated. To make it even trickier, your doctor may sometimes use language that you don’t understand. It may help to bring a friend or loved one with you to your visit. One way to help be sure you understand what your doctor has told you is to repeat back to them what you think they said, in your own words. This way, your doctor can explain it to you again if something isn’t clear.

Keep health information organized. It can be hard to keep track of your medical records while you manage your PNH. But you will need to, so that you can be prepared for medical appointments and advocate for your health. This information helps both you and your medical team understand how your disease is progressing and how well your treatment is working. This should include:

  • Names of all your health care providers, their contact information, and the role they play in your care
  • Doctor visit summaries and notes
  • Hospital discharge summaries
  • Pharmacy printouts of any prescribed medication information
  • Test results like bloodwork or urine tests so you can track any changes
  • Insurance forms
  • Legal documents like a living will and medical power of attorney

You can store all of this on your computer, an app, or in an old-fashioned notebook – whatever is easiest for you.

Know your treatment options. Talk to your doctor about the pros and cons of each treatment. Make sure you know what to expect, including side effects. Questions to ask include:

  • What are all my treatment options?
  • Is there a particular treatment option you recommend for me? If so, why?
  • Is this a new or experimental treatment?
  • How will I know if this treatment is working?
  • Where will I get the treatment?
  • What are your tips to stick with the treatment plan?
  • How much of my treatment will be covered by insurance?
  • Has this drug been specifically FDA-approved to treat PNH?
  • Will this drug interact with any other medications or supplements that I use?

Learn from others. It’s normal to be frightened by a diagnosis of PHN. But there’s more information, more research, and more treatment options than ever before. Case in point: while it used to be that people with PNH only lived an average of 15 to 20 years, research suggests that life expectancy has been steadily going up over the last 2 decades. Here are some ways to stay informed:

Talk to other patients. It helps to speak to other people who live with PNH. They may have tried a treatment your doctor recommended or know of clinical trials you haven’t heard of. The Aplastic Anemia and MDS International Foundation offers virtual monthly support groups. You can get more information at their website.

Attend a virtual conference. Groups like the Aplastic Anemia and MDS International Foundation often sponsor conferences on rare conditions such as PNH.

Keep up to date with clinical trials. The PNH Global Alliance website has a list of clinical trials with notes on which ones are looking for people to take part.

Show Sources

Photo Credit: Tetra Images / Getty Images


European Society for Blood and Marrow Transplantation: “Understanding PNH.”

Aplastic Anemia and MDS International Foundation: “PNH-Paroxysmal Nocturnal Hemoglobinuria,” “Questions to Ask Your Healthcare Team,” “Use Doctor’s Visits Wisely.”

Agency for Healthcare Research and Quality: “Use the Teach Back Method: Tool #5.”

National Institute on Aging: “How to Prepare for a Doctor’s Appointment.”

Johns Hopkins Medicine: “Medical Records: Getting Organized.”