--- title: At 65, Very Severe Aplastic Anemia and PNH Led to Persistent Hemolysis—Complement Inhibition Helped Him Regain Quality of Life url: "https://www.yyhmedical.com/en/stories/vsaa-pnh-complement-inhibitor-treatment" type: Article inLanguage: en-US disease: Aplastic Anemia treatment: Clinical Trials Related to PNH datePublished: 2026-05-08 --- # At 65, Very Severe Aplastic Anemia and PNH Led to Persistent Hemolysis—Complement Inhibition Helped Him Regain Quality of Life > After being diagnosed with very severe aplastic anemia (VSAA) and later developing PNH, 65-year-old Mr. Liu faced persistent hemolysis, transfusion dependence, and limited treatment options; individualized complement inhibition and enrollment in a PNH clinical trial at Beijing GoBroad Boren Hospital gradually improved his blood counts and quality of life. Summary: Mr. Liu, 65, was found to have pancytopenia after seeking care for a fever and was diagnosed with very severe aplastic anemia (VSAA). His condition initially stabilized after immunosuppressive therapy, but about a year later his PNH clone began to expand, and he developed VSAA-PNH overlap syndrome with hemolysis, anemia, and a large gallstone that complicated treatment. Persistently low platelet counts made surgery high risk, leaving him with limited options. In 2025, he came to the Anemia Diagnosis & Treatment Center at Beijing GoBroad Boren Hospital, where the team developed an individualized plan that included complement inhibition to control hemolysis and enrollment in a PNH-related clinical trial. With treatment, he gradually became transfusion independent, his hemoglobin and platelet counts continued to improve, and his quality of life improved markedly. After years of difficult treatment, he could finally see hope again. Very severe aplastic anemia with paroxysmal nocturnal hemoglobinuria (VSAA-PNH overlap syndrome) is a high-risk hematologic overlap condition characterized by severe bone marrow failure together with persistent intravascular hemolysis. When gallstones and obstructive jaundice are also present, very low platelet counts can make surgery especially risky, creating a major treatment challenge and an unfavorable prognosis. In this case, the Anemia Diagnosis & Treatment Center at Beijing GoBroad Boren Hospital shares the management of a critically ill patient with VSAA-PNH overlap syndrome complicated by obstructive jaundice. The team's experience with individualized comprehensive treatment and a clinical trial offers a practical reference for similarly complex patients with severe hematologic disease and surgical complications. Mr. Liu (pseudonym), a 65-year-old from Tianjin, had been living a quiet, ordinary life. Then in 2023, he went to a local hospital because of a fever, and the blood test results immediately alarmed his family: all three blood cell lines were low. His white blood cell count was only 1.53 × 10^9/L, hemoglobin was 74 g/L, platelets were as low as 9 × 10^9/L, and neutrophils were just 0.18 × 10^9/L. The local hospital promptly treated him for infection, but his blood counts failed to recover. His family then took him to the hematology department of a tertiary hospital, where bone marrow aspiration and other detailed tests finally revealed the cause: very severe aplastic anemia (VSAA). He was started on intensive immunosuppressive therapy (IST), and fortunately his blood counts gradually returned to normal. But the disease was not finished with him. About a year after IST, follow-up testing detected a PNH clone—a population of abnormal blood cells associated with paroxysmal nocturnal hemoglobinuria—and the clone continued to expand. By July 2025, it had reached 91.1%. Taken together, the findings led to a diagnosis of VSAA-PNH overlap syndrome. At that point, Mr. Liu also had markedly elevated direct bilirubin, and imaging showed a gallstone about 3 cm in size that was already causing partial obstructive jaundice. Doctors advised stopping his medication that was supporting blood cell production, but once it was stopped, the aplastic anemia relapsed, his blood counts fell sharply, and he again required frequent transfusions. With platelets persistently below 20 × 10^9/L and no acute abdominal emergency, outside hospitals considered surgery too risky. His family felt desperate and unsure where to turn. ![9109f9e9-0d78-4ec6-9740-0f70a77502eb.png](https://gaobo-byh-h5.oss-accelerate.aliyuncs.com/images/20260913/7be4f72eda750d81a349771532711544_20260913000234.png "7be4f72eda750d81a349771532711544_20260913000234.png") Progression of Mr. Liu's PNH clone With few options left, Mr. Liu came to Beijing GoBroad Boren Hospital. On admission, repeat blood counts showed a white blood cell count of 5.18 × 10^9/L, hemoglobin 65 g/L, mean corpuscular volume 118.6 fL, platelets 14 × 10^9/L, neutrophils 3.38 × 10^9/L, and a reticulocyte count of 48.30 × 10^9/L. Lactate dehydrogenase (LDH) was markedly elevated at 871.6 U/L. Because he also had underlying bone marrow failure, the Anemia Diagnosis & Treatment Center developed an individualized plan that combined treatment to support hematopoiesis with complement inhibition to control hemolysis. On July 29, 2025, Mr. Liu began treatment with eculizumab, a C5 complement inhibitor. His platelet count improved, reaching 48 × 10^9/L by November 11, 2025, but when his hemoglobin dropped to its lowest levels, he still needed transfusions. Years of medical care had consumed nearly all of this ordinary family's savings. Mr. Liu's son worked away from home and used his modest income to help support the family. With limited finances and little room left in the treatment budget, Mr. Liu felt anxious and helpless. More than anything, he hoped there might be an option that was both more effective and financially manageable. ![c63e2e52-9a4b-46d5-8be2-95c2d4a2f29f.png](https://gaobo-byh-h5.oss-accelerate.aliyuncs.com/images/20260913/74ff45035885ae1910e2162a05a566ef_20260913000252.png "74ff45035885ae1910e2162a05a566ef_20260913000252.png")Specialists from the Anemia Diagnosis & Treatment Center during rounds with Mr. Liu At this difficult point, a new possibility emerged. The Anemia Diagnosis & Treatment Center at Beijing GoBroad Boren Hospital had a PNH-related clinical trial underway. When Mr. Liu heard about it, he immediately expressed strong interest in participating. After rigorous eligibility screening, he qualified and enrolled in the clinical trial on November 11, 2025, starting treatment with a new complement inhibitor. From that point, his course began to change again: he gradually became transfusion independent, his LDH level fell substantially, and hemolysis came under effective control. His hemoglobin has now risen to 94 g/L and his platelet count to 84 × 10^9/L. The fatigue and dizziness caused by anemia have resolved, his quality of life has improved significantly, and his risk of thrombosis has also been markedly reduced. ![1d3436e6-ab4d-42cb-a9d1-e2850e9466bb.png](https://gaobo-byh-h5.oss-accelerate.aliyuncs.com/images/20260913/86000cff296409da303cdf9d06372d30_20260913000308.png "86000cff296409da303cdf9d06372d30_20260913000308.png")Changes in Mr. Liu's blood counts at first admission and before and after the clinical trial "I will never forget it. The doctors and the clinical trial pulled me back when I felt I had run out of options." Whenever Mr. Liu talks about his experience, his gratitude is clear. At a recent follow-up visit, he brought two commemorative banners to thank the people who had supported him. One was for the blood bank staff, who repeatedly helped him through the difficult period when transfusions were essential. The other was for the entire Anemia Diagnosis & Treatment Center team, whose attention to every detail made him feel genuinely cared for throughout treatment. Mr. Liu speaks with deep appreciation for the Anemia Diagnosis & Treatment Center. In his words, the doctors and nurses did more than treat his illness—they gave him back the confidence and dignity to keep living. That bond between patient and care team means more to him than any words that could be written on a banner. ![c38bb24d-0ce9-4974-9108-7597bcd207e8.png](https://gaobo-byh-h5.oss-accelerate.aliyuncs.com/images/20260913/a82a4f698f0021835bc2db52d40c3fc3_20260913000327.png "a82a4f698f0021835bc2db52d40c3fc3_20260913000327.png")![711fadd8-02d8-4c4b-9026-007f66afaff0.png](https://gaobo-byh-h5.oss-accelerate.aliyuncs.com/images/20260913/facca5b2e72ea9464f40577f8c52a972_20260913000336.png "facca5b2e72ea9464f40577f8c52a972_20260913000336.png")Mr. Liu personally presents commemorative banners to express his thanks This kind of warmth and hope is not unique to one patient. Prof. Liping Jing's team keeps each patient's individual circumstances at the center of care, carefully assessing the disease and tailoring treatment to find the best possible balance between safety and effectiveness. That commitment to individualized, precision care is helping more patients like Mr. Liu find a way forward even when treatment options seem limited—and feel supported along the way. > This content is published with the authorization of the patient and their family, intended solely to share real experiences and health knowledge. It does not constitute a recommendation of any treatment plan or medical diagnostic advice. For specific treatment, please follow the guidance of a professional physician. --- Original (HTML):https://www.yyhmedical.com/en/stories/vsaa-pnh-complement-inhibitor-treatment This is a clean Markdown mirror for large language models; the original page is authoritative.