1.1.5.2 Transplantation Therapy

1.1.5.2 Transplantation Therapy

1.1.5.2.1 Bone Marrow Transplantation (BMT)

The primary pathogenesis of aplastic anemia lies in primary and secondary defects in the quantity and/or quality of hematopoietic stem cells. Allogeneic bone marrow transplantation (allo-BMT) is the best method for reconstructing hematopoietic function in severe aplastic anemia (SAA) and eliminating qualitative defects in hematopoietic stem cells. For SAA patients receiving HLA-matched sibling donors, irradiation-based pre-treatment regimens should be avoided, and CTX+ATG protocols are more suitable, with long-term survival rates after transplantation reaching around 70%.

1.1.5.2.2 Peripheral Blood Stem Cell Transplantation

Adequate quantities of hematopoietic stem cells are isolated from peripheral blood for autologous transplantation. In the 1990s, hematopoietic stimulants such as G-CSF and GM-CSF were widely used in clinical practice, revealing their strong mobilizing effects and relatively few side effects, which made allogeneic hematopoietic stem cell transplantation feasible.

1.1.5.2.3 Umbilical Cord Blood Transplantation

Umbilical cord blood contains more early-stage stem cells than bone marrow, making it another ideal source for hematopoietic stem cell transplantation. The content of highly proliferative colony-forming units (HPP-CSF) and the proportion of CD34⁺CD38⁻ cells forming colonies in umbilical cord blood are higher than in bone marrow, though research on umbilical cord blood lymphocytes suggests that these lymphocytes are not fully mature. T lymphocytes stimulated by foreign antigens exhibit lower cytotoxic activity compared to adult T cells, and continuous stimulation can lead to tolerance toward foreign antigens. Some T helper cells also express CD45RA and CD38, but these T cells lack auxiliary functions and primarily serve immunosuppressive roles.

1.2 Traditional Chinese Medicine’s Understanding of Aplastic Anemia

The TCM classification of aplastic anemia falls under categories such as "blood deficiency," "blood depletion," "deficiency-induced fatigue," and "deficiency-related damage." Traditional Chinese Medicine has a long history of understanding aplastic anemia, with many similarities to modern medicine. TCM holds that "the kidney governs the bones, and the marrow resides within the bones," and "marrow and blood share a common origin," indicating a close relationship between the kidneys and bone marrow hematopoiesis. Additionally, TCM believes that "blood is the essence derived from food and water, generated by the spleen," and "the middle burner receives qi and extracts the essence, which transforms into red blood cells" [14], suggesting that the spleen also plays a role in hematopoiesis. Therefore, syndrome differentiation and treatment should focus on the kidneys and spleen, with the kidneys being particularly closely related.

Clinically, since the 1950s, TCM treatment for aplastic anemia has generally been divided into three stages: before the 1960s, the main approach was tonifying the heart and spleen or replenishing qi and nourishing blood; in the 1970s, based on the previous methods, the approach of strengthening the spleen and tonifying the kidneys emerged; since the 1980s, the primary method has almost always been tonifying the kidneys, supplemented by replenishing qi and nourishing blood, among others. Since then, numerous empirical formulas focusing on tonifying the kidneys have appeared domestically. Although the specific drug compositions vary, the underlying principles of these formulas all fall within the aforementioned scope.

Regarding the syndrome differentiation and classification of aplastic anemia, the Chinese Committee of Integrated Traditional and Western Medicine Hematology held an academic conference in Suzhou in 1979 [15], where aplastic anemia was classified into acute labor marrow depletion type (equivalent to acute aplastic anemia), yin deficiency type, yang deficiency type, and both yin and yang deficiency type. The latter three corresponded to chronic aplastic anemia. However, through practical experience, it was found that labeling the condition as "acute labor marrow depletion" did not reflect the interconnections among qi, blood, yin, yang, and the zang-fu organs, making it difficult to guide clinical prescription and medication. As for the latter three types, only yin and yang were mentioned without specifying the zang-fu organs, which also failed to provide clear guidance for clinical prescription and medication. Consequently, at the National Conference on Integrated Traditional and Western Medicine Hematology held in Dalian in 1989, the issue of classifying acute and chronic aplastic anemia was revisited. It was concluded that the pathogenesis of aplastic anemia is most closely related to the kidneys, so the disease was reclassified into three types—kidney yin deficiency, kidney yang deficiency, and both kidney yin and yang deficiency—with the kidneys as the central focus, which would better guide syndrome differentiation and treatment.