Fatigue and weaknessBruising and bleeding — even difficulty controlling minor bleedsBlood in the urine or dark, tea-colored urine that’s most noticeable in the morning but clears as the day progressesSmall red dots on the skin that indicate bleeding below the skin’s surfaceRecurring infections or feverHeadache, abdominal pain, or back painBlood clots
The good news is, there are steps you can take to manage these symptoms. Start with these strategies.
1. Follow your treatment plan.
“With good care and the right medicines, your PNH can be managed,” says Robert Alan Brodsky, MD, a professor of medicine and the director of the division of hematology at Johns Hopkins Medicine in Baltimore. The monoclonal antibody medicines eculizumab (Soliris) and ravulizumab-cwvz (Ultomiris) were the first drugs approved by the Food & Drug Administration (FDA) to specifically treat PNH, and they’ve made a big difference with the disease. “Most people can be rendered asymptomatic or minimally symptomatic with these medications,” Dr. Brodsky says. What’s more, these drugs help slash the risk of blood clots — an otherwise life-threatening issue — in people with PNH. In May 2021, the FDA approved another medication to treat PNH: pegcetacoplan (Empaveli), a targeted C3 inhibitor. “Most patients with PNH do extremely well on treatment with eculizumab or ravulizumab,” says Shruti Chaturvedi, MBBS, an assistant professor of medicine at Johns Hopkins Medicine in Baltimore. “For the small number of patients that PNH is not fully controlled by eculizumab, pegcetacoplan can improve blood levels and reduce the need for blood transfusions.” which means you need expert care. PNH is a rare disease and complicated to treat, so it’s crucial to find a doctor who’s experienced in and familiar with the illness, according to the Aplastic Anemia & MDS International Foundation (AAMDS). Some handle this by seeing their primary care physician or hematologist and having them regularly consult with the PNH specialist, who the patient may only see from time to time, such as every six months. You can find an arrangement that works best for you, depending on the severity of your condition. The AAMDS Foundation offers a Patient Travel Assistance Fund to help cover travel costs associated with an in-person appointment with a PNH expert.
3. Track your signs and symptoms.
Keeping a log of how you feel day to day and informing your doctor of any changes or unexpected developments can help keep your PNH and accompanying symptoms under control. Be sure to reach out to your doctor if you have sudden or severe pain in your belly, back, head, or elsewhere, which may be a sign of a blood clot. Ask your doctor what else you specifically should keep an eye out for, based on your individual situation. Judith, 70, was diagnosed with PNH in 2006 and makes sure she keeps meticulous medical records, including her own symptom diary. Doing so enables her to spot correlations between how she feels and changes in her blood and other test results. “I always ask for my medical reports,” she says. “This gives me an objective way of measuring my own sense of well-being. And I’m not the only one who does this. One doctor told me that many people come to him with Excel spreadsheets!” Having the symptoms listed along with the raw data “helps with my anxiety about having PNH,” she says.
4. Manage your stress.
When you have PNH, feeling stressed or overwhelmed can lead to disease flares (attacks of hemoglobinuria), so it’s important to take steps to keep stress at bay. Stress management is personal and can take several forms. Sometimes it means asking friends and family for help with daily chores or adjusting what you expect from yourself. For example, do you really need to unload the dishwasher every morning? Make the bed? Clean the coffeepot? Other methods of stress management include taking time to relax, doing activities or hobbies you enjoy, and connecting with friends and family.
6. Carry a medical alert card
Accidents happen, and surgery can be extra risky for people with PNH. Hazards include serious or potentially uncontrolled bleeding, the need for extra platelets, an increased risk of blood clots, and accelerated hemolysis (destruction of red blood cells). It’s a good idea to keep a card in your wallet or purse or wear medical alert jewelry. Make sure it includes key information about your PNH, the medications you take, and emergency contact numbers for your doctors and loved ones.
