Journey Overview

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder where a part of the immune system called the complement system attacks and destroys blood cells. The most common symptom is dark or blood-colored urine, which is typically seen in the morning after waking from sleep. This occurs as hemoglobin from broken down blood cells is filtered from the bloodstream and into the bladder.

PNH can significantly impact the body’s supply of red blood cells, white blood cells, and platelets. This can lead to a number of symptoms and complications. Severe cases can be debilitating, and PNH can cause life-threatening complications including blood clots.

If you or a loved one has been diagnosed with PNH, this guide can help you understand the healthcare providers you’ll work with, the questions to ask at appointments, and how PNH can be managed.

Get to Know Your Care Team

  • Hematologist/ Oncologist

    Treatment for PNH is typically overseen by a hematologist, a medical doctor with specialized training in diseases that affect the blood and bone marrow. Though PNH is not cancer, treatment may be overseen by a hematologist/oncologist, a doctor who specializes in treating cancers that begin in the blood and bone marrow.

  • Primary Care Provider

    Your primary care provider is a practitioner who you see for basic healthcare needs and preventive care—things like checkups, vaccinations, and treatment when you are sick or injured. Your primary care provider may write referrals to specialists and coordinate care among the different members of your healthcare team.

  • Social Worker

    Social workers are licensed professionals trained to help people manage the emotional and practical challenges of living with a health condition. This can include finding ways to reduce the cost of treatment, accessing patient education and resources, and coping with the mental and emotional burdens of the diagnosis.

  • Other Providers and Specialists

    PNH can affect different people in different ways, and it can affect many different areas of the body. Depending on a person’s symptoms and treatment goals, they may work with a variety of other providers and specialists. Healthcare teams may include a geneticist, gastroenterologist, cardiologist, infectious disease specialist, and an OBGYN.

Treatment for PNH

  • Medications called complement inhibitors can prevent the immune system from destroying blood cells. These medications carry a risk of serious infections that affect the brain and spinal cord.
  • Blood transfusions may be given to increase the amount of red blood cells or platelets for people who have anemia.
  • Anticoagulant medications (blood thinners) may be prescribed to treat or prevent blood clots.
  • The only potential cure is an allogenic stem cell transplant, also called a bone marrow transplant. Due to the significant risk of side effects, this approach is only recommended in cases of severe PNH where other treatments have not been successful. It also requires a compatible stem cell donor.

It’s important to know what to do if symptoms reappear, worsen, or if new symptoms appear. Talk to your healthcare providers about symptoms that require immediate medical attention, including symptoms of bleeding, blood clots, or serious infections. Have a plan for seeking emergency medical care in the event you need it.

The Conversation: Questions to Ask Your Healthcare Team

Good communication with your healthcare team will help you get the most from your appointments with your healthcare provider. Follow these steps and questions to get started.

Prepare for your appointment

These steps can help you make the most of your time with your healthcare provider:

  • Keep a symptom journal. In addition to symptoms, you can use a journal to keep track of appetite, energy levels, moods, sleep habits, and how you feel each day.
  • Write down a list of questions and topics you want to cover at your next appointment
  • Prioritize the questions and topics that are most important to discuss with your healthcare providers.

Questions about treatment:

Ask these questions for every treatment you are prescribed.:

  • How does this treatment work?
  • How will we know if the treatment is working?
  • Why do you recommend this approach?
  • What are the goals of this treatment?
  • What are the risks associated with this treatment?
  • Should I make any lifestyle changes during treatment? Changes to activities, what I eat, or other habits?
  • What are the options if this treatment doesn’t work?
  • Who can I talk to if I’m concerned about the cost of treatment?

Questions about your healthcare team:

Coordinated care between your different healthcare providers is essential to managing PNH:

  • What is the most important information I need to know about my diagnosis?
  • Are there other healthcare providers I should be working with?
  • What can I do to ensure the different members of my healthcare team are up to date on my diagnosis and treatment?
  • Can I have copies of my lab reports and pathology reports?
  • Where can I learn more about PNH?
  • What new symptoms or changes in symptoms do I need to watch for? When do I need to call you? When do I need to seek emergency care?

Your appointment is an opportunity to learn more about your diagnosis. If you have a question, ask. If something isn’t clear, ask for an explanation.

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