Plasma exchange helps child with treatment-resistant aHUS
Case report highlights importance of early recognition, intervention
Written by |
Plasma exchange helped ease severe bluish discoloration and pain in the fingers and toes of a 3-year-old boy with atypical hemolytic uremic syndrome (aHUS) that persisted despite treatment with Soliris (eculizumab).
During the procedure, the liquid part of the blood, or plasma, is removed and replaced by that of a healthy donor to eliminate disease-driving antibodies.
While Soliris, together with dialysis, improved the boy’s kidney function, the persistent breakdown of red blood cells (hemolysis) and worsening symptoms in his fingers and toes prompted the decision to start plasma exchange. Dialysis is used to remove waste and excessive fluid from the blood when the kidneys fail.
According to researchers, early recognition of treatment-resistant hemolysis and blood flow complications is essential for timely therapeutic intervention.
“Adjunctive use of therapeutic plasma exchange may help remove [disease-causing] antibodies and complement components, thereby stabilizing disease activity,” they wrote.
The case was described in “Therapeutic Plasma Exchange for Eculizumab-refractory Peripheral Cyanosis in Pediatric Anti-factor H Atypical Hemolytic Uremic Syndrome: A Rare Case Report,” published in the Journal of Transfusion and Immunohematology.
Boy improved after 4 plasma exchange sessions
aHUS is caused by abnormal activity of the complement pathway, a part of the immune system, which results in blood clotting in small blood vessels, known as thrombotic microangiopathy (TMA). The disease is marked by hemolytic anemia, when red blood cells — those that transport oxygen in the bloodstream — are destroyed more quickly than they are produced; thrombocytopenia, or low platelet counts, which are key to blood clotting; and acute kidney failure.
The disease can be caused by self-reactive antibodies that block the activity of complement-regulating proteins, such as factor H, ultimately resulting in uncontrolled complement activation.
Here, researchers in India described the case of a 3-year-old boy with anti-complement factor H antibodies who presented with cyanosis (bluish color due to lack of oxygen in blood) in the fingers and toes, as well as signs of TMA.
The boy initially presented with reddish spots on the face and decreased urine output, with initial lab tests indicating signs of hemolytic anemia and thrombocytopenia. He was referred to the researchers’ hospital, where physical examination revealed he also had irritability, pallor, and jaundice (a yellowish discoloration of the skin and whites of the eyes).
In such scenarios, therapeutic plasma exchange may serve as an effective adjunctive treatment to control ongoing hemolysis and improve clinical outcomes.
Further tests also showed signs of kidney damage, including elevated levels of creatinine and urea, and elevated D-dimer, an indicator of a blood clotting disorder. The boy also had high levels of antibodies against complement factor H and normal levels of ADAMTS13, which excluded thrombotic thrombocytopenic purpura, another type of TMA.
The boy was started on Soliris and dialysis. Although his kidney function improved, hemolysis continued, and cyanosis progressively worsened. The medical team then decided to start therapeutic plasma exchange on the sixth day after admission.
After four plasma exchange sessions, markers of red blood cell destruction decreased, and hemolysis resolved. The child’s cyanosis and associated pain also gradually reduced. He was discharged in stable condition with normal blood and biochemical tests.
According to the researchers, “this case highlights an uncommon but severe extrarenal [not in the kidneys] manifestation of aHUS in the pediatric population,” noting that reduced blood flow to the fingers and cyanosis may persist even after timely initiation of Soliris.
“In such scenarios, therapeutic plasma exchange may serve as an effective adjunctive treatment to control ongoing hemolysis and improve clinical outcomes,” the scientists added.
Leave a comment
Fill in the required fields to post. Your email address will not be published.