The first consideration should be allogeneic hematopoietic stem cell transplantation.

The first consideration should be allogeneic hematopoietic stem cell transplantation.
Six. Efficacy criteria for AA
(1) Complete remission: Disappearance of anemia and bleeding symptoms, with hemoglobin levels reaching 120 g/L in males and 110 g/L in females, neutrophil count reaching 1.5 × 10^9/L, platelet count reaching 100 × 10^9/L, and no relapse during follow-up for more than 1 year.
(2) Partial remission: Disappearance of anemia and bleeding symptoms, with hemoglobin levels reaching 120 g/L in males and 100 g/L in females, white blood cell count reaching 3.5 × 10^9/L, and a certain degree of increase in platelet count; stable condition or continued improvement during 3 months of follow-up.
(3) Significant improvement: Marked improvement in anemia and bleeding symptoms, no need for blood transfusions, hemoglobin level increasing by more than 30 g/L compared to the baseline within 1 month of treatment, and maintaining this level for 3 months. Patients meeting any of the above three efficacy criteria must not require blood transfusions within 3 months.
(4) No effect: Despite adequate treatment, there is no significant improvement in symptoms or routine blood tests.
Seven. Professor Pei Zhengxue’s clinical experience in treating aplastic anemia
This disease can be classified under the categories of “Xulao,” “Xusun,” “Xuezheng,” and “Xue Xu” in traditional Chinese medicine, closely related to the spleen and kidney. The pathogenesis is mainly due to kidney deficiency, spleen deficiency, blood heat causing erratic blood flow, and qi deficiency leading to inability to control blood.
(---) Integrated Chinese and Western medicine treatment 1. Key points for integrated diagnosis
Includes medical history, symptoms, and laboratory tests. Medical history: congenital deficiency, commonly seen in children under 10 years old; acquired damage, such as drug-induced injury from chemotherapy drugs, acetaminophen, chloramphenicol, etc. Symptoms: anemia symptoms, such as dizziness, fatigue, insomnia, and excessive dreaming, which fall under the category of “Guipi Tang syndrome.” Bleeding symptoms include petechiae, nosebleeds, and menorrhagia in women. Fever symptoms include severe infections and anemic low-grade fever. Laboratory tests: complete blood count shows pancytopenia, with reticulocyte count <1%. Bone marrow examination reveals hypoplasia, with decreased megakaryocytes when proliferation is active, and bone marrow biopsy shows fatty infiltration.
2. TCM syndrome differentiation and treatment
(1) The kidney governs bone marrow, while the spleen controls blood. Aplastic anemia is caused by dysfunction of red marrow hematopoiesis, characterized by a decrease in all three blood cell lines in peripheral blood. Professor Pei Zhengxue believes that, based on the pathogenesis of aplastic anemia, to improve the bone marrow picture, treatment should focus on the kidney; however, given the clinical manifestations of aplastic anemia—peripheral blood cell reduction, often accompanied by pallor, loss of appetite, fatigue, weakness, shortness of breath, reluctance to speak, palpitations, forgetfulness, insomnia, and excessive dreaming—indicating both spleen and kidney deficiency, treatment should focus on the spleen, which aligns with the principle of “the kidney governs the innate, while the spleen governs the acquired.”
(2) Tonifying both the spleen and kidney is the primary treatment. “Formed blood cannot be quickly generated; it is the intangible qi that must be urgently strengthened.” The reduction in red blood cells, white blood cells, and platelets in aplastic anemia all represent a deficiency in formed blood components, i.e., “formed blood.” To generate “formed blood,” one must urgently tonify the “intangible qi.” Professor Pei Zhengxue believes that the fundamental aspect of treating aplastic anemia is to tonify qi. The “Lanzhou Formula” uses numerous qi-tonifying herbs, each playing a role in critical situations, clinically demonstrating the TCM principle that “qi is the commander of blood”—tonifying qi is equivalent to tonifying blood. Professor Pei Zhengxue also believes that when conditions are stable, treatment should focus on the spleen and kidney, with the spleen and kidney complementing each other according to the specific syndrome; when conditions are urgent, treatment should focus on stopping bleeding, clearing heat and detoxifying, draining fire and cooling blood. Clearing heat and detoxifying means eliminating inflammation and infection, while draining fire means stopping bleeding. In addition, promoting blood circulation and removing blood stasis should be combined, addressing both root and branch, with clear priorities and orderly procedures. Based on clinical experience, Professor Pei Zhengxue notes that, from a functional perspective, white blood cells are yang, while platelets are yin; therefore, strengthening yang can “increase white blood cells,” and nourishing yin can “increase platelets.” Red blood cells are formed blood, so the key to “increasing red blood cells” lies in tonifying qi and nourishing blood. Although this view is merely based on simple empirical observations, it does contain a core of clinical practice and represents the essence of integrated TCM and Western medicine syndrome differentiation.
3. Herbal medicine as the mainstay
Lanzhou Formula and its modifications: The Lanzhou Formula consists of Rehmannia root 12g, Chinese yam 20g, Cornus fruit 20g, Moutan bark 10g, Poria 12g, Alisma 10g, Ginseng root 15g, Prince ginseng 15g, Northern ginseng 15g, Codonopsis (American ginseng) 15g, Scrophularia root 15g, Ophiopogon 10g, Schisandra 6g, Cinnamon twig 10g, White peony 10g, Ginger 6g, Jujube 4 pieces, Honey-fried licorice 6g, Floating wheat 30g. This formula uses Liuwei Dihuang Decoction to tonify kidney yin and promote bone marrow regeneration; Ginseng root, Prince ginseng, Northern ginseng, Scrophularia root, Codonopsis or American ginseng, and five types of ginseng are used to tonify qi and nourish blood. Gui Zhi Tang harmonizes Ying and Wei to balance the yin-yang imbalance of the internal organs, while Shengmai Yin benefits qi and nourishes yin. Gan Mai Da Zao Tang nourishes the heart and calms the spirit, and when the heart and spirit are calm, the blood is also calm. Modifications: For increasing white blood cells, use Fupian, Chuanwu, Caowu, Ma Qianzi, Cinnamon, Danggui, Bu Gu Zhi, Tu Si Zi, Sha Yuan Zi, Ji Xue Teng, Huang Qi, American ginseng, and deer antler. For increasing platelets, use Nü Zhen Zi, Han Lian Cao, Yu Zhu, Huang Jing, Jujube, Ejiao, Lian Qiao, Tu Da Huang, and Mu Tou Hui. For increasing red blood cells, add Guipi Tang with Ginseng root, Prince ginseng, Northern ginseng, Scrophularia root, American ginseng, He Shou Wu, Erzhi Wan, and water leeches. Professor Pei Zhengxue believes that Ginseng root has a sharp shape and strong qi, allowing it to penetrate into the blood. For clearing heat and detoxifying, use Huang Lian Detoxification Decoction plus Xin Wu Wei Disinfection Drink (Ban Zhi Lian, Bai Hua She She Cao, Xia Ku Cao, Hu Zhang, Zao Xiu). For draining fire and cooling blood, use Rhinoceros Horn–Rehmannia Decoction with modifications. For promoting blood circulation and removing blood stasis, commonly use San Qi, San Ling, E Zhu, Huang Yao Zi, Xiang Fu, Hong Hua, Dan Shen, and water leeches. For patients with liver disease, adding Xiao Chai Hu Tang is particularly effective.
4. Western medicine as auxiliary
Anti-infection, blood transfusion, and symptomatic supportive treatment.
(II) Classification-based treatment
Professor Pei Zhengxue believes that the key to treating aplastic anemia is to tonify the kidney and strengthen the spleen, as well as promote blood circulation and remove blood stasis. Strengthening the spleen focuses on the periphery, which is the manifestation; tonifying the kidney focuses on the bone marrow, which is the root; prioritizing kidney tonification while supporting with spleen strengthening. Professor Pei Zhengxue treats aplastic anemia by combining kidney tonification, spleen strengthening, and blood circulation promotion. Clinically, medications are selected based on specific circumstances: for increasing white blood cells, use Ma Qianzi, Danggui, Bu Gu Zhi, Si Zi, Sha Yuan Zi, Ji Xue Teng, Huang Qi, American ginseng, and deer antler; for increasing platelets, use Nü Zhen Zi, Han Lian Cao, Yu Zhu, Huang Jing, Sheng Di Huang, Lian Qiao, and Tu Da Huang; for increasing red blood cells, use Guipi Tang with Ginseng root, Prince ginseng, Northern ginseng, Scrophularia root, American ginseng, He Shou Wu, Erzhi Wan, and water leeches. Modern pharmacological research shows that kidney-tonifying herbs can promote the recovery of hematopoietic stem cells, stimulate bone marrow proliferation, regulate the body's immune function, inhibit excessive apoptosis of hematopoietic stem cells, and improve bone marrow hematopoietic function. Qi-tonifying and spleen-strengthening herbs also have a promoting effect on bone marrow hematopoietic cells, increasing the number of cells undergoing mitosis in the bone marrow, and when combined with kidney-tonifying herbs, they can promote and protect bone marrow hematopoietic function. Traditional Chinese medicines that promote blood circulation and remove blood stasis can improve the bone marrow hematopoietic microenvironment, regulate the body's immune function, eliminate immune damage in the bone marrow microenvironment, and promote the recovery of aplastic anemia.
In addition to the spleen-deficiency symptoms caused by qi and blood deficiency—such as pale complexion, dizziness, poor appetite, and fatigue—patients with aplastic anemia may also exhibit tinnitus, mental fatigue, impotence, and nocturnal emission, indicating kidney deficiency. In treatment, Professor Pei Zhengxue emphasizes strengthening the spleen and tonifying the kidney, alternating between the two and adapting flexibly. For children and young adults, whose vital energy is generally intact, the focus is on strengthening the spleen with kidney tonification as a supplement; for elderly patients, whose vital energy is mostly depleted, the focus shifts to tonifying the kidney with spleen strengthening as a supplement. Additionally, there are cases where newly diagnosed patients require stronger spleen strengthening, while long-term patients require stronger kidney tonification; abnormalities in peripheral blood counts emphasize spleen strengthening, while abnormalities in bone marrow counts emphasize kidney tonification. It is important to remember that tonifying qi does not mean neglecting blood nourishment, and nourishing blood does not mean neglecting qi; when tonifying the kidney, one should pay attention to the mutual dependence of yin and yang, warming yang without forgetting to nourish yin, and nourishing yin without forgetting to warm yang. Long-term qi and blood deficiency in patients with aplastic anemia will inevitably lead to blood stasis, and if the blood stasis is not removed, new blood will not be generated, further exacerbating blood deficiency and causing bleeding. Clinically, in addition to symptoms of qi and blood deficiency, there may also be pulse涩, tongue with blood stasis, or blood stasis in teeth and nose. Therefore, when treating patients with aplastic anemia who have been ill for a long time and show signs of blood stasis, Professor Pei Zhengxue combines blood circulation promotion with kidney tonification and spleen strengthening, resulting in remarkable therapeutic effects. Among them, spleen strengthening primarily uses Guipi Tang, with longan meat added in large quantities—15 to 25g; kidney tonification primarily uses Jin Gui Shen Qi Tang, with Cornus fruit added in large quantities—15 to 30g. If there is facial flushing and five-heart heat, add Erzhi Wan; if there is cold limbs and weak lower back and knees, switch to You Gui Wan. Aplastic anemia is essentially a deficiency condition, but even “extremely deficient” individuals may have “real” manifestations—some patients present with fever, nasal issues, dental problems, and skin blood stasis, indicating real syndromes. For fever, if the pathogen resides in the qi layer, use Ren Shen Bai Hu Tang with modifications; if it enters the Ying layer, use Qing Ying Tang with modifications; for nasal, dental, and subcutaneous blood stasis, adhere to the principle of “draining the heart is draining fire, and draining fire is stopping bleeding,” adding Huang Lian, Huang Qin, and Da Huang as appropriate. However, once the fever subsides and the bleeding stops, the focus should shift to tonifying the spleen and kidney. For patients with concurrent blood stasis and kidney deficiency, Professor Pei Zhengxue often uses a self-formulated formula called “Chuan He Ji,” containing: Danggui 10g, Chuan Xiong 6g, Sheng Di 12g, Xian He Cao 15g, He Shou Wu 15g, Tu Da Huang 15g, Ji Xue Teng 15g, Dan Shen 15g, Hong Hua 6g, Black Soybean 30g, Cornus fruit 20g, Longan meat 15g, Nü Zhen Zi 15g, Goji berry 15g, Bu Gu Zhi 15g, Rou Cong Rong 10g, Ma Qianzi 1 piece. In this formula, Danggui, Chuan Xiong, Ji Xue Teng, Dan Shen, and Hong Hua promote blood circulation and remove blood stasis; Sheng Di, He Shou Wu, Cornus fruit, Nü Zhen Zi, Goji berry, and Bu Gu Zhi nourish yin and tonify the kidney; Xian He Cao and Tu Da Huang stop bleeding and replenish deficiency; the combination of these herbs strengthens both the spleen and kidney, and Ma Qianzi 1 piece, which Professor Pei Zhengxue believes can dispel wind and unblock meridians, improving the immune status of patients with aplastic anemia. Adding Ma Qianzi 1 piece (fried to remove toxicity) to the formula doubles the therapeutic effect.

1. Tonifying the kidney and strengthening the spleen
   “The Plain Questions · Great Discussion on the Correspondence Between Yin and Yang” states: “The kidney generates bone marrow.” “The Plain Questions · Discussion on the Flow of Vital Energy to Heaven” states: “Bone marrow is solid, and both qi and blood originate from it.” “Zhang’s Medical Compendium” states: “The source of blood lies in the kidney.” “The Spirit Pivot · Decision on Vital Energy” states: “The middle burner receives qi and extracts juice, which transforms into red blood, thus becoming blood.” “The Forty-Two Difficulties of the Classic of Difficulties” states: “The spleen wraps blood and warms the five viscera.” These statements demonstrate that the generation and distribution of blood are closely related to the spleen. “The Plain Questions · Great Discussion on the Correspondence Between Yin and Yang” states: “For those with insufficient form, warm them with qi; for those with insufficient essence, supplement them with taste.” While Professor Pei Zhengxue is strengthening kidney essence, he also emphasizes spleen-strengthening herbs, which aligns with the principle of “formal blood cannot be quickly generated; intangible qi must be urgently strengthened.” Based on over 50 years of clinical experience, Professor Pei Zhengxue believes that tonifying the kidney can regulate bone marrow hematopoietic function. Clinically, he often chooses Rehmannia, Cornus fruit, Chinese yam, Tu Si Zi, Goji berry, Nü Zhen Zi, Rou Cong Rong, and deer antler. Patients with kidney deficiency often experience dizziness, tinnitus, lower back and knee pain, and memory loss, especially needing Cornus fruit, with dosages up to 30g. Spleen-strengthening herbs often include Ginseng root, Codonopsis, Northern ginseng, and Prince ginseng. Patients often suffer from pallor, fatigue, loss of appetite, insomnia, and excessive dreaming, so Guipi Tang and Buzhong Yiqi Tang are commonly used, with longan meat added in large quantities—up to 30g. Spleen-strengthening herbs tend to improve peripheral blood circulation. When qi deficiency leads to loss of blood control, blood spills out of the vessels, so treatment should focus on benefiting qi and cooling blood, often using Ginseng root, Codonopsis, Northern ginseng, Prince ginseng, Xian He Cao, Tu Da Huang, Ji Xue Teng, Sheng Yi Zheng Ren, Huang Qi, Shan Zhi Zi, Sheng Di Huang, Mudan Pi, Dan Shen, and Lian Qiao.
2. Promoting blood circulation and removing blood stasis
   Aplastic anemia occurs due to the failure of hematopoietic stem cells in the bone marrow. The kidney governs bones, stores essence, and generates marrow, serving as the innate foundation; the spleen governs transformation and metabolism, providing the source of qi and blood, serving as the acquired foundation; “essence and blood share the same origin,” so tonifying the kidney and filling the essence is the fundamental treatment for aplastic anemia, while strengthening the spleen and benefiting qi are also indispensable. Professor Pei Zhengxue believes that “if blood stasis is not removed, new blood will not be generated” and “long-term illness leads to blockage of the meridians.” Blood stasis remains stagnant for a long time, depriving the organs of nutrients and warmth, exacerbating organ deficiency, which in turn further leads to blood stasis formation. This vicious cycle of deficiency leading to stasis and stasis leading to deficiency further aggravates the condition of aplastic anemia, so promoting blood circulation and removing blood stasis is also a method for treating aplastic anemia. Commonly used herbs include Danggui, Chuan Xiong, Hong Hua, Mudan Pi, Dan Shen, Ji Xue Teng, and San Qi, along with water leeches to break up blood stasis. Professor Pei Zhengxue believes that this medicine can significantly accelerate patient recovery and promote self-healing.
3. Clearing heat and detoxifying
   “When evil forces gather, qi must be deficient.” Patients with aplastic anemia have weakened vital energy and are most susceptible to external pathogens. Professor Pei Zhengxue often adds wind-dispelling herbs to his treatment of aplastic anemia, such as Qiang Huo, Du Huo, and Fang Feng, because “wind is the master of all diseases—it can create life, but it can also destroy it.” When external wind-heat or wind-cold pathogens enter the body and transform into heat, they can burn the meridians, causing tooth, muscle, and purpura problems, indicating that heat is forcing blood to flow. Treatment should then focus on clearing heat and detoxifying, draining fire and cooling blood, often using San Huang Xie Xin Tang and Rhinoceros Horn–Rehmannia Decoction. Professor Pei Zhengxue believes that draining the heart is draining fire, and draining fire is stopping bleeding, often using honeysuckle, Lian Qiao, dandelion, white snake tongue grass, bai jiang cao, mudan pi, dan shen, zicao, qian cao, xian he cao, yi mu cao, white peony, nü zhen zi, sheng di huang, ze lan, xiang fu zi, da huang, huang qin, and huang lian.
   (III) Treatment principles
   Professor Pei Zhengxue treats aplastic anemia by focusing on the kidney governing bone marrow and the spleen governing the periphery; formal blood cannot be quickly generated, while intangible qi must be urgently strengthened; when conditions are stable, strengthen the spleen and tonify the kidney; when conditions are urgent, drain fire and cool blood; strengthen yang to “increase white blood cells,” nourish yin to “increase platelets” and “increase red blood cells”—four aspects of syndrome differentiation and prescription formulation, achieving good clinical results.
4. The kidney governs bone marrow, while the spleen governs the periphery
   Aplastic anemia is caused by dysfunction of bone marrow hematopoiesis, characterized by a decrease in all three blood cell lines in peripheral blood. “The Plain Questions · Great Discussion on the Correspondence Between Yin and Yang” states: “The kidney governs bone marrow and generates marrow.” Based on this statement, Professor Pei Zhengxue believes that to improve the bone marrow picture of aplastic anemia patients, treatment should focus on the kidney. He believes that Liuwei Dihuang Decoction indeed has the effect of regulating bone marrow hematopoietic function, with Cornus fruit dosage potentially reaching 30g, showing significant effects. “The Spirit Pivot · Decision on Vital Energy” states: “The middle burner receives qi and extracts juice, which transforms into red blood, thus becoming blood.” “The Forty-Two Difficulties of the Classic of Difficulties” states: “The spleen… wraps blood and warms the five viscera.” These statements demonstrate that the generation and distribution of blood are closely related to the spleen. Professor Pei Zhengxue believes that spleen-strengthening and qi-benefiting methods are more effective at improving the peripheral blood picture, with Guipi Tang being the preferred choice, and longan meat added in large quantities—up to 30g—for better results. Clinical manifestations of aplastic anemia often include pallor, loss of appetite, fatigue, shortness of breath, reluctance to speak, palpitations, forgetfulness, insomnia, and excessive dreaming—symptoms of both spleen and kidney deficiency; dizziness, tinnitus, lower back pain, and leg fatigue are also common—symptoms of kidney qi deficiency. For such clinical presentations, using Guipi Tang and modifying it with Liuwei Dihuang is consistent with the principles of harmony among law, regulation, formula, and medicine. Traditional Chinese medicine holds that “the kidney governs the innate, while the spleen governs the acquired,” and in treating blood diseases, Professor Pei Zhengxue has realized that “the kidney governs bone marrow, while the spleen governs the periphery”—using kidney tonification to regulate bone marrow hematopoietic function and spleen strengthening to improve the peripheral blood picture.
5. Formal blood cannot be quickly generated, while intangible qi must be urgently strengthened
   A decrease in all three blood cell lines is the main characteristic of aplastic anemia. All three cell types are formed components of blood, which is referred to as “formal blood.” To generate “formal blood,” one must urgently tonify “intangible qi,” a concept rooted in the theory of “mutual dependence of yin and yang,” later developed into the idea that “qi is the commander of blood.” Through Professor Pei Zhengxue’s clinical practice, this principle has been applied to treat aplastic anemia, with each medication containing this idea. Among qi-tonifying herbs, Prince ginseng is the first choice, as Professor Pei Zhengxue says, “this herb has a mild taste but strong qi, capable of penetrating into the blood.” Other commonly used herbs include Jilin ginseng, Northern ginseng, Codonopsis, and Huang Qi. Among them, Jilin ginseng is expensive, so Ginseng root is often used instead, with Professor Pei Zhengxue saying, “Ginseng root has a sharp shape and strong qi, able to penetrate directly into the blood.” “Qi is the root of yang,” and prolonged qi deficiency inevitably leads to yang deficiency, so in qi-tonifying herbs, adding Epimedium, Bu Gu Zhi, and Tu Si Zi—yang-enhancing herbs—can complement each other effectively.
6. When conditions are stable, strengthen the spleen and tonify the kidney; when conditions are urgent, drain fire and cool blood
   Strengthening the spleen and tonifying the kidney, along with reinforcing yang, are the fundamental methods for treating aplastic anemia. However, when infections, bleeding, or other symptoms occur,