Hepatitis-Associated Aplastic Anemia with Dr. Roma Rajput

Leigh Clark:    Hi everyone, this is Podcast for Patients with the Aplastic Anemia and MDS International Foundation. I'm Leigh Clark, director of patient services. Our podcast is brought to you with special thanks to the generous support from our patients, families and caregivers, just like you and our corporate sponsors. Thank you to everyone for supporting the series.
    Today we're going to be talking about hepatitis-associated aplastic anemia with Roma Rajput, who is with the FDA she is a clinical reviewer there and also, she's a research collaborator with NH, HHLBI at the National Institutes of Health. Welcome, Dr. Rajput.
Dr. Roma Rajput:    Thank you for having me.
Leigh Clark:    Thank you so much for joining us. So, what is Hepatitis-associated aplastic anemia?
Dr. Roma Rajput:    So, hepatitis-associated aplastic anemia is a rare form of acquired aplastic anemia in which bone marrow failure develops several weeks to months, even up to a year after an acute episode of hepatitis. Patients often have a story of abdominal pain and jaundice and are found to have marked elevations in their liver enzymes. Patients get tested for viruses and other known causes of hepatitis, which are almost always negative. Often the hepatitis is mild, however, cases of severe liver injury requiring emergent liver transplant have been reported in the literature. Patients will present several weeks to months after the initial episode of hepatitis with signs of bone marrow failure. This includes fatigue and shortness of breath due to anemia, easy bleeding and bruising due to low platelet count, and infections due to low white blood cell count.
    Blood counts are generally available from the time of liver injury since these patients are um, seen by health care professionals and they often reveal near normal blood counts.
Leigh Clark:    How does it differ from non-hepatitis immune aplastic anemia?
Dr. Roma Rajput:    This is a good question. We know that both hepatitis-associated and non-hepatitis-associated aplastic anemia are immune-mediated diseases. In both cases, T-cells destroy their own bone marrow. We also know that both groups have high levels of interfering Gama and tumor necrosis factor alpha, which are markers of inflammation and cell destruction when they're diagnosed. Our group at NHLBI recently presented an abstract at the American Society of Hematology Conference attempting to answer this very question. We compared the hepatitis and non-hepatitis groups based on their clinical characteristics and presence of mutations in their cells. Our preliminary results show that hepatitis-associated aplastic anemia individuals have a significantly lower number of PNH cells and significantly lower number of somatic mutations, both of which are quite common in the non-hepatitis group. We're trying to validate our results with a larger cohort of patients at this time.
Leigh Clark:    And what are the treatment options?
Dr. Roma Rajput:    So hepatitis-associated and non-hepatitis-acquired aplastic anemia are treated exactly the same. The affected individuals will receive treatment based on the current treatment guidelines for acquired aplastic anemia and this is with either immuno-suppressive therapy or bone marrow transplant.
Leigh Clark:    What are the risk of PNH developing in hepatitis-associated uh, patients versus non hepatitis-associated patients?
Dr. Roma Rajput:    This is a good question. We know that approximately 30-40% of acquired aplastic anemia patients will have PNH cells. But only 10% or so will require treatment due to the breakdown of the red blood cells or large clone that increases their risk for blood clots. Based on our NIH study, given that less patients are likely to have coexisting PNH cells, the risk of clinically significant PNH developing is most likely the same if not less.
Leigh Clark:    Is there anything else that you would like patients to know about hepatitis-associated aplastic anemia?
Dr. Roma Rajput:    I want patients to know that both hepatitis-associated aplastic anemia and non-hepatitis-associated aplastic anemia are immune-mediated diseases. Both respond to immuno-suppressive therapy and can be cured by hematopoietic stem cell transplant. While the treatment remains the same, there are certain distinct differences between the two populations. Further research understanding these differences may provide insight on how to better manage this unique subset within acquired aplastic anemia.
Leigh Clark:    Well, thank you so much, Dr. Rajput for sharing your time and your expertise with all of us today uh, during this podcast. And if you'd like to find out more about aplastic anemia and treatments, please visit our website at aamds.org. You can also gain information by following us on Facebook, Instagram and X. Or please do feel free to give us a call on our help line at 800-747-2820. This concludes our podcast.
Dr. Roma Rajput:    Thank you for having me.