Iptacopan combined with immunosuppressive therapy for aplastic anemia-paroxysmal nocturnal hemoglobinuria syndrome: A case report.
- 1The First Clinical Medical College, Shandong University of Traditional Chinese Medicine, Jinan, Shandong, China.
- 2Department of Hematology, Affiliated Hospital of Shandong University of Traditional Chinese Medicine, Jinan, Shandong, China.
Abstract
Rationale
Aplastic anemia-paroxysmal nocturnal hemoglobinuria (AA-PNH) syndrome is a rare acquired hematopoietic stem cell disorder. Patients who respond poorly to conventional immunosuppressive therapy (IST) and corticosteroids face limited therapeutic options and a poor prognosis. This article reports on the use of iptacopan combined with IST for AA-PNH syndrome.Patient concerns
A previously healthy 24-year-old Han Chinese male presented with dizziness and fatigue and was found to have pancytopenia. He was initially treated with cyclosporine. However, 2 years after discontinuation, he developed acute hemolysis. After treatment with iptacopan, the patient's hemolysis was quickly controlled, but he still required blood transfusions.Diagnoses
The patient was initially diagnosed with aplastic anemia through bone marrow examination, along with a paroxysmal nocturnal hemoglobinuria clone. After recurrent acute hemolysis, reexamination of the bone marrow and flow cytometry revealed partial deficiency of cluster of differentiation (CD) 55/CD59, and fluorescently labeled aerolysin-negative cells accounted for more than 90% of granulocytes and monocytes. Eventually, the diagnosis of AA-PNH syndrome was made.Interventions
After acute hemolysis was initially managed with steroids and IST, the patient was started on iptacopan to achieve rapid hemolysis control. Given persistent transfusion dependence due to bone marrow hypoplasia, immunosuppressants and hematopoietic stimulants (danazol and eltrombopag) were added while continuing iptacopan. The hematopoietic stimulants were discontinued after 3 months due to abnormal liver function tests, and the patient was maintained on iptacopan combined with IST.Outcomes
Iptacopan rapidly and effectively controlled hemolysis, and no breakthrough hemolysis, extravascular hemolysis, or drug-related adverse events were observed. Critically, iptacopan served as a bridge to control hemolysis, while the subsequent addition of IST (tacrolimus/cyclosporine) addressed the underlying autoimmune bone marrow failure. After combined treatment with iptacopan and IST, the patient's blood cell count gradually recovered, hemoglobin returned to normal levels, and blood transfusion was no longer required.Lessons
This case suggests that iptacopan combined with IST may represent a potentially effective and safe therapeutic option for AA-PNH syndrome that is refractory to conventional IST and corticosteroids. Especially for AA-PNH patients with impaired bone marrow function and continuous dependence on blood transfusion, iptacopan combined with IST may be an effective and well-tolerated treatment regimen.
Related
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- Abdominal Magnetic Resonance Imaging (MRI) Findings Prompting the Diagnosis of Paroxysmal Nocturnal Hemoglobinuria: A Case Report.
- FACIT-fatigue score and treatment quality assessment for paroxysmal nocturnal hemoglobinuria patients treated with Eculizumab in Türkiye
- QUALIDADE DE VIDA E REPERCUSSÕES PSICOLÓGICAS APÓS TRATAMENTO COM IPTACOPAN EM PACIENTE COM HEMOGLOBINÚRIA PAROXÍSTICA NOTURNA: RELATO DE CASO
- Distinct Pharmacodynamic Complement Biomarker Signatures Define Iptacopan and Pegcetacoplan Treatment in C3 Glomerulopathy.
BibTeX
@article{liu2026iptacopan,
title = {{Iptacopan combined with immunosuppressive therapy for aplastic anemia-paroxysmal nocturnal hemoglobinuria syndrome: A case report.}},
author = {Xiaoqian Liu and Jie Xu and Yaqin Luo and Yingying Wang and Jianping Li and Linying Li and Zhaoxia Liu},
journal = {Medicine},
year = {2026},
month = oct,
doi = {10.1097/md.0000000000051162},
url = {https://europepmc.org/article/MED/42888561},
note = {CC BY},
}
RIS
TY - JOUR
TI - Iptacopan combined with immunosuppressive therapy for aplastic anemia-paroxysmal nocturnal hemoglobinuria syndrome: A case report.
AU - Xiaoqian Liu
AU - Jie Xu
AU - Yaqin Luo
AU - Yingying Wang
AU - Jianping Li
AU - Linying Li
AU - Zhaoxia Liu
PY - 2026
DA - 2026/10/01
JO - Medicine
DO - 10.1097/md.0000000000051162
UR - https://europepmc.org/article/MED/42888561
AB - Rationale Aplastic anemia-paroxysmal nocturnal hemoglobinuria (AA-PNH) syndrome is a rare acquired hematopoietic stem cell disorder. Patients who respond poorly to conventional immunosuppressive therapy (IST) and corticosteroids face limited therapeutic options and a poor prognosis. This article reports on the use of iptacopan combined with IST for AA-PNH syndrome.Patient concerns A previously healthy 24-year-old Han Chinese male presented with dizziness and fatigue and was found to have pancytopenia. He was initially treated with cyclosporine. However, 2 years after discontinuation, he developed acute hemolysis. After treatment with iptacopan, the patient's hemolysis was quickly controlled, but he still required blood transfusions.Diagnoses The patient was initially diagnosed with aplastic anemia through bone marrow examination, along with a paroxysmal nocturnal hemoglobinuria clone. After recurrent acute hemolysis, reexamination of the bone marrow and flow cytometry revealed partial deficiency of cluster of differentiation (CD) 55/CD59, and fluorescently labeled aerolysin-negative cells accounted for more than 90% of granulocytes and monocytes. Eventually, the diagnosis of AA-PNH syndrome was made.Interventions After acute hemolysis was initially managed with steroids and IST, the patient was started on iptacopan to achieve rapid hemolysis control. Given persistent transfusion dependence due to bone marrow hypoplasia, immunosuppressants and hematopoietic stimulants (danazol and eltrombopag) were added while continuing iptacopan. The hematopoietic stimulants were discontinued after 3 months due to abnormal liver function tests, and the patient was maintained on iptacopan combined with IST.Outcomes Iptacopan rapidly and effectively controlled hemolysis, and no breakthrough hemolysis, extravascular hemolysis, or drug-related adverse events were observed. Critically, iptacopan served as a bridge to control hemolysis, while the subsequent addition of IST (tacrolimus/cyclosporine) addressed the underlying autoimmune bone marrow failure. After combined treatment with iptacopan and IST, the patient's blood cell count gradually recovered, hemoglobin returned to normal levels, and blood transfusion was no longer required.Lessons This case suggests that iptacopan combined with IST may represent a potentially effective and safe therapeutic option for AA-PNH syndrome that is refractory to conventional IST and corticosteroids. Especially for AA-PNH patients with impaired bone marrow function and continuous dependence on blood transfusion, iptacopan combined with IST may be an effective and well-tolerated treatment regimen.
SN - 0304-5412
SN - 1536-5964
ER -