Romiplostim N01 in the Treatment of Refractory Chemoradiotherapy-induced AA

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 relapsed/refractory chemoradiotherapy-induced aplastic anemia

Currently, there are few studies on the use of TPO-RAs after tumor radiotherapy and chemotherapy. Our center has published a study on the efficacy and safety of avatrombopag (AVA) in AA secondary to radiotherapy and chemotherapy. The study demonstrated that the ORR at 1, 3, and 6 months were 32.4%, 55.9%, and 58.8%, respectively. In a phase II/III study for refractory AA, romiplostim monotherapy achieved an ORR of 84% at week 27, which showed that romiplostim seemed to be effective and safe in patients with refractory aplastic anemia, with a recommended starting dose of 20 μg/kg once a week.

Inclusion Criteria:

1. Age ≥ 18 years old;
2. Diagnosed with relapsed/refractory chemoradiotherapy-induced aplastic anemia (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 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. PNH clone ≥ 50%;
4. Received HSCT 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.