Đánh giá hiệu quả điều trị ghép tế bào gốc đồng loài trên người bệnh suy tủy xương tại Bệnh viện Truyền máu Huyết học

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Tác giả: Thiện Ngôn Huỳnh, Văn Mẫn Huỳnh

Ngôn ngữ: vie

Ký hiệu phân loại: 174.25 Innovative procedures

Thông tin xuất bản: Tạp chí Y học Việt Nam (Tổng hội Y học Việt Nam), 2022

Mô tả vật lý: 58-65

Bộ sưu tập: Metadata

ID: 500767

Thêm vào giỏ

Investigating clinical features, laboratories and pre-treatment. Determining rate of engraftment, rate of overall survival time, event-free survival time, GvHD-relapsed free survival, and complications after allogeneic stem cell transplantation in patients with acquired aplastic anemia. Objects and methods: The clinical data of 33 acquired aplastic anemia (AA) patients (16 male and 17 female) who received MSD allo- HSCT from 09/2008 to 09/2021 were analyzed retrospectively. The median age was 12 (6.5 - 40) years old. Eight patients had transfusiondependent intermediate aplastic anemia (AA), 13 patients had SAA and 12 patients had very severe aplastic anemia (VSAA). Clone PNH was 30.3%. 21 (63.3%) patients received more than 10 units of transfusions (red blood cells and/or platelets) and five (15.2%) patients failed to respond to the previous immunosuppressive therapy. The median time from diagnosis to HSCT was 2 (range: 1.4 - 3.5) months. All patients received allogeneic peripheral blood stem cell (PBSC) transplantation (allo-PBSCT). Some various conditioning regimens were used such as cyclophosphamide (CY) + horse anti-thymocyte globulin (h-ATG) (22 patients), CY + h-ATG + Fludarabine (FLU) (2 patients), CY + FLU (1 patient), CY (only one patient), CY + r-ATG + FLU (6 NB), CY + r-ATG (1 NB). Fifteen patients received prophylaxis for graft-versushost disease (GVHD) with cyclosporine (CSA) plus short-term methotrexate (MTX). Results: Engraftment was observed in 32 (96.9%) patients. The median time to neutrophil (ANC) recovery and to platelet (PLT) recovery were 13 (range: 9 - 20) days and 15 (range: 8 - 28) days, respectively. 9 (27.3%) patients developed acute GVHD (aGVHD) and 66.7% patients of them had grade II-IV aGVHD. Three patients suffered from chronic GVHD (cGVHD) which was well managed with corticoids. CMV reaction occurred in 14 (42.4%) patients and was controlled with Ganciclovir. Of th patients who had secondary graft rejection, one patient successfully received second stem cell transplantation and the other achieved partial response following with h-ATG + Cyclosporin. The median follow-up time was 27 (5.3 - 127) months. Five-year estimated overall survival (OS), event-free survival (EFS) and GvHD-free relapse-free survival (GRFS) was 96.9%, 90.5% and 66.6% respectively. Early complications after transplantation included febrile neutropenia (32 patients), severe sepsis (30.3% patients), platelet transfusion refractory (3 patients), hemorrhagic cystitis (3 patients) and death (1 patient). Chronic renal failure, hypothyroidism, cataract, and femoral head avascular necrosis were the most common late complications. Conclusion: MSD allo-HSCT is an effective therapy for patients with acquired AA. The outcome of allogeneic HSCT in patients with acquired AA at our institution was comparable to the results of the other previous studies.Nghiên cứu đặc điểm lâm sàng, sinh học và điều trị của người bệnh suy tủy trước ghép. Xác định tỷ lệ mọc mảnh ghép, tỷ lệ sống còn toàn bộ (OS), sống còn không biến cố (EFS), sống còn không tái phát - không GvHD (GRFS) sau 5 năm. Xác định tỷ lệ các biến chứng sau ghép tế bào gốc đồng loài Đối tượng và phương pháp: Mô tả hàng loạt ca, hồi cứu 33 bệnh nhân người lớn và trẻ em ( 16 nam và 17 nữ) được chẩn đoán suỷ tuỷ xương và được ghép tế bào gốc đồng loại từ 09/2008 đến 09/2021. Tuổi trung vị là 12 (6.5 - 30.5) tuổi. Chẩn đoán suy tủy độ trung bình lệ thuộc truyền máu (8 bệnh nhân), suy tủy độ nặng (13 bệnh nhân), suy tủy độ rất nặng (12 bệnh nhân). Tỷ lệ người bệnh (NB) có clone PNH kèm theo là 30.3%. 15.2% NB đã được điều trị liệu pháp miễn dịch trước ghép. 21 (63.3%) NB nhận hơn 10 đơn vị máu (hồng cầu lắng và/hoặc tiểu cầu). Thời gian trung vị từ lúc chẩn đoán đến lúc ghép là 2 (1.4 - 3.5) tháng. Tất cả bệnh nhân đều nhận ghép tế bào gốc từ máu ngoại vi. Các phác đồ điều kiện hóa được sử dụng: cyclophosphamide (CY) + anti-thymocyte globulin từ ngựa (h-ATG) (22 NB)), CY + h- ATG + Fludarabine (FLU) (2 NB), CY + FLU (1 NB), CY (1 NB), CY + r-ATG (1 NB), CY + r- ATG + FLU (6 NB). Tất cả được dự phòng bệnh mảnh ghép chống chủ (GvHD) với cyclosporine (CSA) với methotrexate (MTX). Kết quả nghiên cứu: Tỷ lệ mọc mảnh ghép là 96.9% (32/33). Thời gian trung vị hồi phục bạch cầu và hồi phục tiểu cầu lần lượt là 13 (9- 20) ngày và 15 (8 - 28) ngày. Có 9 (27.3%) NB phát triển GvHD cấp, độ II-IV chiếm 66.7% và có 3 NB có GvHD mạn giới hạn, được điều trị tốt với corticoid. Tái hoạt CMV xảy ra ở 14 NB (42.4%), được điều trị với Ganciclovir. Có 3 bệnh nhân thải ghép thứ phát, trong đó có 1 bệnh nhân được ghép lần hai thành công, một bệnh nhân còn lại đạt được đáp ứng một phần sau điều trị với ATG ngựa + Cyclosporin. Tỷ lệ sống còn toàn bộ (OS), tỷ lệ sống không sự kiện (EFS), tỷ lệ sống không tái phát - không GvHD sau 5 năm lần lượt là 96.9%, 90.5% và 66.6%. Những biến chứng sớm sau ghép bao gồm sốt giảm bạch cầu hạt (96,9%), nhiễm trùng nặng (30.3%), kháng tiểu cầu (3 NB), viêm bàng quang xuất huyết (3 NB), tử vong (01 NB) Suy thận mạn, suy giáp, đục thuỷ tinh thể, hoại tử chỏm xương đùi là những biến chứng muộn hay gặp. Kết luận: Ghép tế bào gốc đồng loài là phương pháp điều trị khá hiệu quả ở những người bệnh suy tuỷ xương. Kết quả điều trị này trong nghiên cứu của chúng tôi tương đương những nghiên cứu khác trước đây.

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