关键词: haplo-identical hematopoietic real world stem cell transplant

来  源:   DOI:10.7759/cureus.59744   PDF(Pubmed)

Abstract:
Hematopoietic stem cell transplant (HSCT) is potentially, the sole curative option for many malignant and non-malignant hematological disorders. Finding a human leukocyte antigen (HLA) compatible donor remains one of the limiting factors, hampering the utilization of HSCT. However, the introduction of post-transplant cyclophosphamide (PTCy) has improved the outcomes of haploidentical transplants making it a suitable option for patients lacking HLA-compatible donors. We collected data from 44 patients who underwent haplo-identical allogeneic stem cell transplants at the Armed Forces Bone Marrow Transplant Center/National Institute of Blood and Marrow Transplant (AFBMTC/NIBMT) from the year 2015 to 2022. The diseases were divided into three categories, i.e., bone marrow failure (BMF) syndromes, hematological malignancies (HM) and miscellaneous (Misc) groups. Median age at transplant was 18 (01-39) years. Transplant indications included aplastic anemia (AA) in 21 (47.7%) cases, 15 (34.1%) HM, and eight (18.2%) cases falling in the Misc groups. A maximum number of graft failures occurred in the BMF group; primary graft failure in 07 (33.3%) cases and secondary graft failure in four (19%) cases, (p-value < 0.05). Acute graft versus host disease (aGVHD) grade II-IV occurred in nine (20.5%) cases while chronic graft versus host disease (cGVHD) occurred in 10 (22.7%) cases. Cytomegalovirus (CMV) reactivation was seen in 31 (70.5%) cases. Maximum CMV reactivation was seen in HM group 13 (86.6%) cases, (p-value < 0.05) as compared to BMF (71.4%) and Misc groups (37.5%). Post-transplant cyclophosphamide (PTCy) based regimens, early neutrophil engraftment, and patients with GVHD had better survival outcomes (p-value < 0.05) overall survival (OS), and relapse-free survival (RFS). and GVHD-free relapse-free survival (GFRS) were significantly better in cases with early neutrophil engraftment. OS of the study cohort was 50% while disease-free survival (DFS) and GFRS were 45.5% and 36.4%, respectively.
摘要:
造血干细胞移植(HSCT)是潜在的,许多恶性和非恶性血液病的唯一治疗选择。寻找人类白细胞抗原(HLA)相容的供体仍然是限制因素之一,阻碍了HSCT的使用。然而,移植后环磷酰胺(PTCy)的引入改善了单倍体移植的结局,使其成为缺乏HLA相容供体的患者的合适选择.我们收集了从2015年到2022年在武装部队骨髓移植中心/国家血液和骨髓移植研究所(AFBMTC/NIBMT)接受单倍同同种异体干细胞移植的44例患者的数据。这些疾病分为三类,即,骨髓衰竭(BMF)综合征,血液恶性肿瘤(HM)和杂项(Misc)组。移植时的中位年龄为18(01-39)岁。移植适应症包括再生障碍性贫血(AA)21例(47.7%),15(34.1%)HM,8例(18.2%)落在杂项组。最大数量的移植物失败发生在BMF组;原发性移植物失败在07例(33.3%)和继发性移植物失败在4例(19%)。(p值<0.05)。急性移植物抗宿主病(aGVHD)II-IV级发生在9例(20.5%)中,而慢性移植物抗宿主病(cGVHD)发生在10例(22.7%)中。巨细胞病毒(CMV)再激活31例(70.5%)。最大CMV再激活见于HM组13例(86.6%),与BMF(71.4%)和Misc组(37.5%)相比,(p值<0.05)。基于移植后环磷酰胺(PTCy)的方案,早期中性粒细胞植入,GVHD患者的生存结果更好(p值<0.05),总生存期(OS),和无复发生存期(RFS)。在早期中性粒细胞植入的病例中,无GVHD无复发生存率(GFRS)明显更好。研究队列的OS为50%,而无病生存率(DFS)和GFRS分别为45.5%和36.4%,分别。
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