Bước đầu đánh giá hiệu quả điều trị của azacitidine kết hợp venetoclax ở người bệnh bạch cầu cấp dòng tủy tái phát hoặc kháng trị tại bệnh viện Truyền máu Huyết học=Initial evaluation of the treatment effectiveness of azacitidine combined with venetoclax in relapsed/refractory acute myeloid leukemia at BTH hospital

0 Người đánh giá. Xếp hạng trung bình 0

Tác giả: Nguyễn Thùy Trang Bùi, Ngọc Ngân Linh Ngô, Chí Dũng Phù

Ngôn ngữ: vie

Ký hiệu phân loại:

Thông tin xuất bản: Tạp chí Y học Việt Nam (Tổng hội Y học Việt Nam), 2024

Mô tả vật lý: tr.160-168

Bộ sưu tập: Metadata

ID: 478228

Thêm vào giỏ

Description of the clinical features, pathogenesis and initially evaluation of the efficacy and complications of azacitidine combined with venetoclax regimen in relapse/refactory acute myeloid leukemia. Methods: A retrospective case series study of 11 patients who were diagnose relapse or refactory acute myeloid leukemia at Blood Transfusion and Hematology hospital. Results: The rate of achieving CR/CRi increased with the number of treatment cycles, from 45.5% (n = 5) to 72.7% (n = 8) after 2 cycles and to 81.8% (n = 9) at the end of the treatment. In 11 case studied, 2 cases (18,2%) failed treatment with azacitidine and venetoclaxt. With a median follow-up of 8.7 months (0.8 months–28.1 months), the median PFS was 8.2 months, and the median OS was not reached at the end of the study, OS -1 year was estimated 55.6%. We found that treatment-related adverse events were mainly hematological toxicity. With the majority of patients, 81.8% (n = 9), having grade III or IV neutopenia, the rate of infection and neutropenic fever is also as high as 63.6% (n = 7). However, we did not record any deaths during treatment with azacitidine and venetoclax. Conclusion: The combination treatment regimen of azacitidine and venetoclax achieves a high treatment response in relapsed or refractory myeloid leukemia patients with good tolerability and is suitable even in subjects with poor general conditions. However, the duration of response is not prolonged, so more advanced treatments such as cell transplantation are still needed to improve patient survival outcomes. When treated with azacitidine and venetoclax regimens, hematological toxicity is common, especially the risk of severe neutopenia, so prevention, early detection, and early treatment of infections are the keys to reducing the risk of death during treatment.Mô tả đặc điểm lâm sàng, sinh bệnh học và bước đầu đánh giá hiệu quả, biến chứng của phác đồ azacitidine kết hợp venetoclax ở người bệnh bạch cầu cấp dòng tủy tái phát, kháng trị. Phương pháp nghiên cứu: Thiết kế nghiên cứu hồi cứu – mô tả hàng loạt ca. Đối tượng nghiên cứu: 11 người bệnh bạch cầu cấp dòng tủy tái phát sớm, hoặc kháng trị tại Bệnh viện Truyền máu Huyết Học TP. Hồ Chí Minh được điều trị với phác đồ kết hợp azacitidine và venetoclax. Thời gian nghiên cứu: 6/2021 đến hết ngày 01/06/2024. Kết quả: Tỷ lệ đạt CR/CRi tăng lên theo số chu kì điều trị, từ 45,5% (n=5) lên 72,7% (n=8) sau 2 chu kì và lên 81,8% (n=9) tại thời điểm kết thúc điều trị. Trong 11 ca nghiên cứu, có 2 (18,2%) thất bại với điều trị azacitidine và venetoclax. Với thời gian theo dõi trung vị 8,7 tháng (0,8 tháng – 28,1 tháng), thời gian PFS trung vị là 8,2 tháng, thời gian OS trung vị là không đạt được khi kết thúc nghiên cứu, xác suất OS – 1 năm ước tính đạt 55,6%. Chúng tôi nhận thấy, biến cố bất lợi liên quan tới điều trị chủ yếu là độc tính huyết học, có 81,8% (n=9) người bệnh bị giảm bạch cầu hạt mức độ III/IV, vì vậy tỷ lệ nhiễm trùng và sốt giảm bạch cầu cũng cao tới 63,6% (n=7), tuy nhiên chúng tôi không ghi nhận trường hợp nào tử vong trong thời gian điều trị với azacitidine và venetoclax. Kết luận: phác đồ điều trị kết hợp azacitidine và venetoclax đạt được hiệu quả đáp ứng điều trị cao ở người bệnh bạch cầu cấp dòng tủy nguyên phát tái phát/kháng trị, với khả năng dung nạp tốt, phù hợp ở cả đối tượng tổng trạng kém. Tuy nhiên thời gian duy trì đáp ứng là không kéo dài, vì vậy vẫn cần các phương pháp điều trị hiệu quả hơn như ghép tế bào để cải thiện kết cục sống còn của người bệnh. Khi điều trị phác đồ azacitidine và venetoclax, độc tính về huyết học là thường gặp, nhất là nguy cơ giảm bạch cầu hạt mức độ nặng, vì vậy dự phòng, phát hiện sớm và điều trị tích cực nhiễm trùng là chìa khóa giúp giảm nguy cơ tử vong trong điều trị.

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