Interviews with the Experts: Side Effects of PNH Treatment | Aplastic Anemia & MDS International Foundation Return to top.


This is the third part in a series of interviews about PNH. Read the first and second parts.

Interviews with the Experts: Side Effects of PNH Treatment

Although eculizumab (Soliris®) is known to be effective in treating PNH, it has some side effects including colds, nausea, and headaches. Are these side effects treated the same way as if caused by other conditions?

The most frequently reported side effects of eculizumab in the PNH randomized trial were: headache, nasopharyngitis, back pain, and nausea. Interestingly, adverse reactions occurred among 4 (9%) patients receiving eculizumab and 9 (21%) patients receiving placebo (inactive ingredient).

Among 193 patients with PNH treated with eculizumab on two other studies, the side effect profile was similar to that reported in the randomized study mentioned above , affecting16% of  patients enrolled, with the most common being: viral infection (2%), headache (2%), anemia (2%), and fever (2%). These side effects are typically treated with supportive measures.

Treatment with eculizumab can also lead to a potentially serious problems -- infection with the meningococcus bacteria that causes meningitis. What should patients know about this? Are alerts and antidotes needed?

Meningococcal infections have been reported in patients treated with eculizumab. Because meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early, the following steps must be followed :
•    Eculizumab is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) and all prescribers, and patients must enroll in the program
•    Treating Physicians must be aware of and compliant with the most current Advisory Committee on Immunization Practices recommendationsfor meningococcal vaccination in patients with PNH.
•    All patients should be immunized with a meningococcal vaccine at least 2 weeks prior to administering the first dose of eculizumab, unless the risks of delaying eculizumab therapy outweigh the risk of developing a meningococcal infection in which case an antibiotic can be prescribed  concomitantly with eculizumab therapy to prevent an infection during the first two weeks of therapy. It should be noted that the benefits and risks of antibiotic prophylaxis for prevention of meningococcal infections in patients receiving Soliris have not been established.
•    A group B meningococcal vaccine became available in 2014, which can also be utilized in this patient population.
Patients should be educated on, and monitored for early signs of meningococcal infections. -all patients should be provided with a Patient Safety Card, and instructed to seek medical attention if they develop the following:
•    headache with nausea or vomiting
•    headache and a fever
•    headache with a stiff neck or stiff back
•    fever
•    fever and a rash
•    confusion
•    muscle aches with flu-like symptoms
•    eyes sensitive to light

Do other types of treatments for PNH (such as prednisone) have similar of different side effects that patients should be aware of?

Prednisone is a steroid that can decrease hemolysis caused by PNH. It may also increase counts of white blood cells and platelets in some people. Prednisone may not be effective in controlling hemolysis, nor does it stop all hemolysis in a given patient. Furthermore, it has a lot of side effects, especially if taken for a long time. For those reasons combined, doctors disagree about whether or not it should be prescribed for people with PNH. Brief treatment courses with prednisone given in pulses may be helpful to control hemolysis in some patients. Large prospective randomized trials evaluating the role of prednisone in this patient population is lacking.


Jamile Shammo, MD, FACP, FASCP

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Position / Title: 
Associate Professor of Medicine
Rush University Medical Center

Dr. Shammo is an associate professor of medicine and pathology, Section of Hematology and Stem Cell Transplantation, Division of Hematology/Oncology, at Rush University Medical Center in Chicago where she spearheads the MDS/MPN/Bone marrow failure program. She is also director of the Hematology/Oncology Fellowship Program and CME Course Director in the Division of Hematology/Oncology.  After earning a medical degree with honors from Aleppo Medical School in Syria, Dr. Shammo completed internships and residencies in the departments of pathology and internal medicine at McGaw Medical Center of Northwestern University, in Evanston, and a 3-year fellowship in the Division of Hematology/Oncology at University of Chicago.  She is board certified in anatomic and clinical pathology, internal medicine, and hematology, and board eligible in oncology.  She is also a fellow of the American Society of Clinical Pathologists and American College of Physicians, and is a member of the American Society of Hematology, the American Society of Clinical Oncologists, and the American College of Physicians.  Dr. Shammo received the Department of Medicine Service and Teaching Award from Rush University Medical Center in 2003.  She has authored or co-authored over 40 publications, including abstracts, posters, book chapters, and online CME activities, as well as articles published in Blood, JCO, Clinical Lymphoma, Journal of Heart and Lung Transplantation, Cytotherapy, and American Journal of Clinical Pathology, among others. Additionally, she functions as a reviewer for several medical journals and as an editor for the Journal of Clinical Oncology.  She has designed and was involved as principal investigator for many clinical trials related to chronic myelogenous leukemia, MPN’s, PNH, and myelodysplastic syndromes.  As an invited speaker, Dr. Shammo has presented her research at national and international meetings and conferences. 

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