Romiplostim N01 in Chemoradiotherapy-induced Aplastic Anemia

Romiplostim N01 in the Treatment of Refractory Chemoradiotherapy-induced Aplastic Anemia: a Single-center, Prospective, Open-label Study

This study aimed to explore the efficacy and safety of romiplostim N01 in the treatment of chemoradiotherapy-induced aplastic anemia (AA)

Inclusion Criteria:

1. Age ≥ 18 years old;
2. Diagnosed with relapsed/refractory chemoradiotherapy-induced AA. Relapsed/ refractory was defined as patients who had no response to at least 3 months of cyclosporine or thrombopoietin receptor agonists (TPO-RAs) such as eltrombopag;
3. At least one of the following conditions must be met: hemoglobin < 90 g/ L, platelet count < 30×109/L, neutrophil count < 1.0×109/L;
4. With baseline liver and kidney functions <2 upper limit of normal (ULN);
5. Without active infection that cannot be controlled by standard treatment;
6. Signed the informed consent;
7. ECOG score ≤ 2;

Exclusion Criteria:

1. Had other primary or secondary bone marrow failure (BMF) diseases, such as Fanconi anemia, congenital keratinization disorder, etc.;
2. With evidence of clonal hematological bone marrow diseases (MDS, AML) in cytogenetics;
3. Paroxysmal nocturnal hemoglobinuria (PNH) clone ≥ 50%;
4. Received hematopoietic stem cell transplantation before enrollment;
5. Received ATG treatment before enrollment;
6. Severe bleeding or infection that cannot be controlled by standard treatment;
7. Allergic or intolerant to romiplostim N01;
8. Active infections of HIV, HCV, or HBV, liver cirrhosis, or portal hypertension
9. History of malignant tumors within 5 years;
10. History of arterial or venous thrombosis;
11. Pregnant or lactating patients;
12. Participated in other clinical trials within 3 months.