Woman with rare lupus-PNH combo safely treated with PiaSky
Case is 7th to record co-occurring conditions, 1st managed with PiaSky
Doctors for the first time used PiaSky (crovalimab-akkz) to control paroxysmal nocturnal hemoglobinuria (PNH) in a woman with the autoimmune disease systemic lupus erythematosus (SLE), according to a case study.
The study’s authors said the case is the seventh documented instance of PNH occurring in someone with underlying SLE, and “to the best of our knowledge … the first reported case of SLE complicated by PNH that was successfully and safely treated with [PiaSky].”
Their paper, “Paroxysmal Nocturnal Hemoglobinuria in Patients with Systemic Lupus Erythematosus: Diagnostic Challenges and Improvement with C5 Inhibitor Treatment,” was published in Internal Medicine.
PNH develops when the body starts producing blood cells that lack certain key markers. Without these markers, the immune system mistakenly identifies these blood cells as an infectious threat, and a group of immune proteins called the complement cascade becomes activated to destroy the blood cells. This ultimately leads to low red blood cell counts and anemia.
SLE is an autoimmune disorder caused by antibodies that attack healthy cells in tissues across various parts of the body.
Woman’s anemia persists despite stable SLE activity
Scientists in Japan described the case of a woman who had been diagnosed with SLE in her late 20s. For several years, she had managed her disease with standard immunosuppressant therapies. But when she was 34, the woman started to develop unexplained anemia that progressively worsened over time, even though markers of SLE activity were stable.
Lab tests indicated that the woman had hemolytic anemia, or anemia due to blood cells being destroyed. Since SLE often co-occurs with other autoimmune conditions, clinicians initially suspected that she might be experiencing a new autoimmune reaction, so they performed a Coombs test, a laboratory assessment that detects antibodies that attack red blood cells. While the test was negative, it prompted them to conduct further diagnostic testing.
Flow cytometry, a test that can look at markers on the surface of individual cells, revealed the pattern of abnormal blood cell markers that characterize PNH, cementing a PNH diagnosis.
“This case underscores a key clinical consideration,” the scientists noted. “In patients with SLE who develop hemolytic anemia, especially when a direct Coombs test is negative, PNH should be promptly ruled out.”
The woman started PiaSky treatment according to the approved dosing schedule. PiaSky is an infusion therapy that works to block activation of the complement cascade. It is sold by Roche, which was not involved in the case report.
Shortly after starting the therapy, the woman’s anemia resolved. The researchers reported that she tolerated the therapy well, with no major safety issues.
The researchers said the case suggests that PiaSky may be safely deployed to treat PNH in patients with underlying SLE, although they emphasized that further studies are necessary to determine the optimal care for patients with this rare combination of disorders.
“To better understand the clinical and immunological features of PNH in the context of SLE and to assess the safety and efficacy of complement inhibition in this setting, further accumulation of similar cases is warranted,” the scientists concluded.
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