Hereditary spherocytosis

Soliris is a drug that has been FDA approved to treat paroxysmal nocturnal hemoglobinuria (PNH). PNH causes the early death and impaired production of blood cells (red blood cells, white blood cells, and platelets). This early destruction of blood cells occurs because the affected cells are missing two important proteins, making them vulnerable to destruction by a part of our immune system called the complement system.^[1] Soliris works by blocking the complement system. You can learn more about Soliris by visiting the DailyMed.gov Web page on this topic. 

Currently, we are not aware of any benefits of Soliris for treating hereditary spherocytosis. The destruction of red blood cells in this condition is a result of mishapen and fragile cell membranes.^[2]

Clinical trials are medical research studies in which people participate as volunteers. They are a means of developing new treatments and medications for diseases and conditions.  Studies may also focus on improving diagnostic techniques, investigating the cause of the condition or understanding how the condition changes throughout an affected person's lifetime. There are strict rules for clinical trials, which are monitored by the National Institutes of Health (NIH) and the U.S. Food and Drug Administration.

Please note: you do not have to be willing to enroll in a clinical trial to find this information helpful.  Seeing what medications or new treatments are being tested in these studies can help you stay up to date on which new treatment options may be available in the future.  Staying in touch with the advocacy groups can also help you stay up-to-date on both research and treatment options. 

Some of the research studies at the NIH Clinical Center involve promising new treatments that may directly benefit patients. The Clinical Center does not charge patients for participation and treatment in clinical studies conducted at the NIH. 

Studies conducted off campus at other hospitals, clinics or through pharmaceutical companies do bill your insurance for part of the cost of the trial, so it is always good to ask if you will be responsible for any of the cost of the study before volunteering.
ClinicalTrials.gov (developed by the National Institutes of Health through the National Library of Medicine) provides patients, family members, and members of the public with current information on clinical research studies.

You can view the current clinical trials enrolling people with hereditary spherocytosis.   You can use the "Map" tab or "Modify Search" feature to limit your search to different states of the United States. Just click on a study to find out more information and if interested you can review the "eligibility" criteria to see if you can enroll. After you click on a study, review its "eligibility" criteria to determine its appropriateness. Use the study’s contact information to learn more. Check this site often for regular updates. 

You can also contact the Patient Recruitment and Public Liaison (PRPL) Office at the National Institutes of Health (NIH) by calling 1-800-411-1222 to speak with a specialist, who can help you determine if you are eligible for any clinical trials.