Theoretical Basis

Theoretical Basis

Immune factors play a very important role in the occurrence and development of aplastic anemia. Aplastic anemia is often triggered by immune abnormalities, immediately causing extensive destruction and growth inhibition of hematopoietic cells and immune matrix cells. Currently, the main treatments for aplastic anemia in China include immunosuppressants, bone marrow transplantation, cytokines, and androgens. However, these treatments are often limited in widespread application in China due to high costs, numerous side effects, or the inability to find HLA-matched donors. Traditional Chinese medicine has conducted many years of clinical and experimental research on chronic aplastic anemia and achieved therapeutic effects. However, in the field of acute aplastic anemia, especially regarding the regulation of immune abnormalities and the removal of immune factors that damage hematopoietic cells, there is still a lack of research [25]. Therefore, we have replicated animal models and conducted experimental research on the use of traditional Chinese medicine to treat acute aplastic anemia [26].

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Research on Pei Zhengxue’s Series of Formulas and Medicines

Below is Professor Pei Zhengxue’s understanding of traditional Chinese medicine’s approach to aplastic anemia [27]:

1 Kidney Governs Bone Marrow, Spleen Governs the Periphery

Aplastic anemia is caused by dysfunction of red bone marrow hematopoiesis, characterized by a decrease in the three lineages of blood cells in the peripheral blood. The “Plain Questions · Great Discussion on Yin-Yang Correspondence” states: “The kidney governs the generation of marrow.” Based on this statement, Mr. Pei believes that to improve the bone marrow picture of patients with aplastic anemia, one must focus on treating the kidney. He thinks that Liuwei Dihuang Tang indeed has the effect of regulating bone marrow hematopoietic function, with Cornus officinalis used in large quantities—up to 30 grams—which has a remarkable effect. The “Spiritual Pivot · Chapter on Deciding Qi” says: “The middle jiao receives qi, transforms it into juice, and the resulting red substance is called blood.” The “Difficult Questions · Forty-Two Difficult Questions” states: “The spleen wraps the blood and warms the five zang organs.” These statements demonstrate that the generation and distribution of blood are closely related to the spleen. Mr. Pei believes that strengthening the spleen and benefiting qi is more effective in improving the peripheral blood picture, with Guipi Tang being the first choice. In this formula, longan meat is used in large quantities—up to 20 grams—resulting in even better efficacy. The clinical manifestations of patients with aplastic anemia often include pale complexion, loss of appetite, fatigue, shortness of breath, reluctance to speak, palpitations, forgetfulness, insomnia, and excessive dreaming—all symptoms of deficiency in both heart and spleen. There are also symptoms of kidney qi deficiency, such as dizziness, tinnitus, lower back pain, and leg weakness. For such clinical presentations, adjusting Guipi Tang and Liuwei Dihuang Tang according to individual needs is in line with the principle of unity of theory, law, prescription, and medicine. Traditional Chinese medicine holds that “the kidney governs the innate, while the spleen governs the acquired.” In treating blood diseases, Mr. Pei has come to understand the concept of “the kidney governs bone marrow, while the spleen governs the periphery,” because bone marrow gradually forms in the womb, while peripheral blood begins to develop after birth.

2 It is Difficult to Suddenly Generate Tangible Blood, but Intangible Qi Must Be Supplemented Urgently

A decrease in the three lineages of blood cells is the main characteristic of aplastic anemia. All three lineages belong to the tangible components of blood—this is what is referred to as “tangible blood.” To generate “tangible blood,” one must urgently supplement “intangible qi.” This idea originates from the “Yin-Yang Interdependence” theory in the “Inner Canon,” later developed into the notion that “qi is the commander of blood.” Through Mr. Pei’s clinical experience, this principle has been applied to the treatment of aplastic anemia, effectively addressing the root cause of the disease. Among qi-tonifying herbs, Taizishen is the first choice. Mr. Pei says: “This herb has a mild taste but strong qi, and can enter the blood.” Other commonly used herbs include Jilin ginseng, Beisha Shen, Dang Shen, and Huangqi. Among them, Jilin ginseng is expensive, so ginseng roots are often used instead. Mr. Pei says: “The roots are sharp and have strong qi, easily entering the blood.” “Qi is the root of yang.” If qi deficiency persists for a long time, it will inevitably lead to yang deficiency. Therefore, in qi-tonifying formulas, adding herbs that strengthen yang, such as Yin Yang Huo, Po Gu Zi, and Tu Si Zi, can complement each other effectively [28].

3 For Chronic Conditions, Strengthen Spleen and Kidney; for Acute Conditions, Clear Heat and Cool Blood

Tonifying qi and strengthening the spleen, as well as nourishing the kidney and strengthening yang, are the fundamental principles for treating aplastic anemia. However, when infection, bleeding, or other symptoms occur, the condition is often acute, with most cases presenting as a state of intense internal fire and overwhelming heat that drives the blood (only a small number of cases involve qi deficiency that cannot control the blood). In such situations, Mr. Pei advocates clearing heat, draining fire, and cooling blood. He highly appreciates Tang Rongchuan’s view that “the heart is the emperor of fire, generating blood; blood is the soul of fire, and fire is the soul of blood. When fire rises, blood rises; when fire falls, blood falls. Knowing that blood is born from fire and that fire resides in the heart, clearing the heart means clearing fire, and clearing fire means stopping bleeding.” For acute bleeding and infection in aplastic anemia, he often chooses Sanhuang Xie Xin Tang, believing that this formula is the first choice for clearing fire and stopping bleeding in aplastic anemia. This formula is bitter and cold, directly eliminating excess fire while incorporating hemostasis into the process of clearing fire. Adding Shengdi 20 grams aims to cool the blood, enhancing its hemostatic power; frequently adding Shengshi 30–60 grams strengthens the fire-draining effect, with the explanation: “Shengshi has a mild taste and a heavy texture. Its mildness allows it to enter the qi, while its heaviness enables it to reach the blood. Only with this can the fever associated with blood disorders be effectively resolved.” Through long-term clinical observation, he has summarized the experience of “for chronic conditions, strengthen spleen and kidney; for acute conditions, clear heat and cool blood” [29].

4 Strengthening Yang to Elevate "White," Nourishing Yin to Elevate "Platelets," and Raising "Red" — The Essence Lies in Tonifying Qi and Nourishing Blood

In his long-term clinical practice, Elder Pei proposed the concept of "strengthening Yang to elevate white blood cells, nourishing Yin to elevate platelets, and tonifying Qi and nourishing blood to raise red blood cells." Although this is merely a simple empirical principle, it embodies the core of clinical practice. The "Plain Questions: On the Great Principles of Yin-Yang Correspondence" states, "Yang transforms into qi, while Yin forms substance," and Zhang Jingyue commented, "Yang is dynamic and disperses, whereas Yin is static and condenses; thus, substance is formed." From the functional attributes of white blood cells and platelets, white blood cells seem to belong to Yang, platelets to Yin, and red blood cells can be regarded as tangible blood. Therefore, the method for elevating them should naturally involve tonifying Qi and nourishing blood. Below are the commonly used medications by Elder Pei [31].

Research on Pei Zhengxue's Series of Formulas and Medications

The following are listed:

To elevate white blood cells: Cinnamon, Fuzi (processed Aconite), Ku Shen (Sophora Flavescens), Dang Shen (Codonopsis Pilosula), Po Gu Zi (Psoralea Corylifolia), Ji Xue Teng (Millettia Retusa), Huang Qi (Astragalus), American Ginseng, Star Anise.

To elevate platelets: Yu Zhu (Polygonatum Sibiricum), Huang Jing (Polygonatum Odoratum), Jujube, Sheng Di (Rehmannia Glutinosa), Ejiao (Donkey-hide Gelatin), Gui Ban Jiao (Tortoise Shell Gelatin), Lu Jiao Jiao (Deer Antler Gelatin), Lian Qiao (Forsythia Suspensa), Tu Da Huang (Rheum Palmatum).

To elevate red blood cells: Gui Pi Tang (Spleen-Nourishing Decoction), Ren Shen Yang Rong Tang (Ginseng-Nourishing and Spirit-Strengthening Decoction), Tai Zi Shen (Prince's Ginseng), Ren Shen Xu (Ginseng Root), Dang Shen (Codonopsis Pilosula), Huang Qi (Astragalus), He Shou Wu (Polygonum Multiflorum), Shan Yu Rou (Cornus Fruit), Yuan Rou (Longan Flesh), Ji Xue Teng (Millettia Retusa), Nü Zhen Zi (Ligustrum Lucidum), Han Lian Cao (Eclipta Prostrata).

Experimental Research