针对遗传性代谢障碍的异构干细胞移植:在一个机构拥有35年的经验
Hiromasa Yabe1, Takashi Koike2, Shohei Yamamoto2
1Department of Pediatrics, Tokai University School of Medicine, 143 Shimokasuya, Isehara, Kanagawa, 259 1193, Japan. yabeh@tokai.ac.jp.
International journal of hematology
|July 1, 2024
概括
自2007年以来,针对遗传代谢疾病的血造干细胞移植显示出改善的生存率和减少的移植失败,这是由于更新的调节方案和供体选择. 这些进步提高了患有粘膜多糖症和上腺核细胞衰竭等疾病的患者的治疗结果.
科学领域:
- 血液学 血液学 血液学
- 儿科血液学 儿科血液学
- 移植免疫学 移植免疫学
背景情况:
- 造血干细胞移植 (HSCT) 是遗传代谢障碍 (IMD) 的治疗选择.
- 随着时间的推移,治疗方案和资源的可用性都发生了重大变化.
- 在不同时期比较结果对于优化患者护理至关重要.
研究的目的:
- 分析和比较托卡伊大学医院在两个不同时期 (2007年以前和2007年之后) 进行的IMD的HSCT结果.
- 评估改变医疗资源和调节方案对移植成功的影响.
- 确定预测改善存活率和减少移植失败的因素.
主要方法:
- 针对IMD (粘多糖症,上腺核衰竭等) 的186个HSCT的回顾性分析. 在1986年至2021年期间进行的.
- 调节方案的比较:口服布苏尔方 (2007年之前) 与静脉注射布苏尔方,胸腹部辐射 (TAI) 与弗鲁达拉宾和梅尔法兰/三硫 (2007年后).
- 对捐赠者类型 (HLA相同的兄弟姐妹与替代捐赠者) 的分析及其对结果的影响.
主要成果:
- 总体生存率从76% (在2007年之前) 提高到97% (在2007年之后) (P=0.006).
- 无事件生存率 (EFS) 从62% (在2007年之前) 提高到85% (在2007年之后) (P=0.017).
- 主要移植缺陷发生率在2007年后下降,特别是在特定的带血移植调节方案下.
结论:
- 从2007年开始的移植时代与IMD中HSCT的优越整体存活率和EFS有关.
- 骨髓修复性调节方案也预测了改善的EFS.
- 现代的调节方案和供体选择策略显著提高了对遗传代谢障碍的HSCT疗效.
相关概念视频
Bone Marrow Sampling and Transplants
324
Bone marrow transplant is a potential cure for several diseases, including cancer and specific genetic disorders. Notably, this procedure is applicable for patients suffering from aplastic anemia, certain types of leukemia, severe combined immunodeficiency disease (SCID), Hodgkin's disease, non-Hodgkin's lymphoma, multiple myeloma, thalassemia, sickle-cell disease, and certain cancers.
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
324
iPS Cell Differentiation
2.7K
The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
2.7K
Stem Cell Culture
5.1K
Stem cell research aims to find ways to use stem cells to regenerate and repair cellular damage. Over time, most adult cells undergo the wear and tear of aging and lose their ability to divide and repair themselves. Stem cells do not display a particular morphology or function. Adult stem cells, which exist as a small subset of cells in most tissues, keep dividing and can differentiate into a number of specialized cells generally formed by that tissue. These cells enable the body to renew and...
5.1K
Stem Cell Therapy for Tissue Regeneration
4.0K
Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
Types of Stem Cells used in Stem Cell Therapy
The two main cell...
Types of Stem Cells used in Stem Cell Therapy
The two main cell...
4.0K


