HSCT for Hematologic Malignancies, Solid Tumors, and Bone Marrow Failure

Patients diagnosed with hematologic malignancies—such as non-Hodgkin's lymphoma, Hodgkin's lymphoma, acute myeloid and lymphoblastic leukemia, and myelodysplastic syndromes (MDS)—as well as those with bone marrow failure syndromes (e.g., severe aplastic anemia) may require high-dose therapy followed by autologous or allogeneic hematopoietic stem cell transplantation (HSCT) to control or potentially cure their disease. Additionally, certain solid tumor patients (such as those with brain tumors or germ cell tumors) may also benefit from high-dose chemotherapy followed by autologous HSCT as a salvage therapy.

Treatment Features

In addition to autologous transplantation, allogeneic transplants may come from related or unrelated donors, with human leukocyte antigen (HLA) matching being either complete or partial (e.g., haploidentical donors). The sources of hematopoietic stem cells include bone marrow, peripheral blood stem cells, and umbilical cord blood.

Treatment Course

1. Pre-transplant evaluations include assessment of the disease status (prior treatments and progression) and comorbid conditions, such as cardiopulmonary function tests. For autologous HSCT, the quantity and storage condition of the harvested stem cells are assessed. For allogeneic HSCT, donor availability and HLA compatibility are crucial factors.
2. A suitable conditioning regimen (either reduced intensity or myeloablative) and stem cell source must be selected.
3. Most patients (excluding those with immunodeficiencies) require pre-treatment regimens involving high-dose chemotherapy and/or total body irradiation (TBI). During allogeneic transplants, prophylactic medications against acute graft-versus-host disease (GvHD) are also administered.
4. Stem cell infusion is usually performed in a laminar airflow room to maintain sterility.

Post-Transplant Care

After transplantation, patients receive supportive care, which includes blood transfusions, granulocyte colony-stimulating factor (G-CSF), antibiotics, and parenteral nutrition to aid recovery and reduce complications.

HSCT 適合血液惡性腫瘤、實體腫瘤與骨髓衰竭

患有血液惡性腫瘤（包括非何杰金氏淋巴瘤、何杰金氏淋巴瘤、急性骨髓性及淋巴性白血病，以及骨髓增生異常症候群）及骨髓衰竭（如重度再生不良性貧血） 的患者，可能需要高劑量治療後進行自體或異體造血幹細胞移植，以控制甚至治癒疾病。此外，部分實體腫瘤患者（如腦瘤與生殖細胞瘤）也可能需要高劑量治療後進行自體造血幹細胞移植來挽救病情。

治療特色

除了自體移植外，異體移植的來源主要為家人或非親屬捐贈者，包括人類白血球抗原（HLA）配對完全或不完全（例如半相合）的捐贈。使用的造血幹細胞類型包括骨髓、周邊血幹細胞及臍帶血。

治療療程

1. 移植前評估包括病患疾病狀況（先前治療及病情進展）與共病狀態：如心肺功能測試、自體移植則需評估幹細胞的數量與儲存狀態、異體移植則需評估捐贈者的可用性與HLA配對情況。
2. 需選擇合適的預處理（調理）方案（降低強度或骨髓清除性）與幹細胞類型。
3. 除免疫缺陷患者外，大部分病人皆需接受先行的處理療程（高劑量化療與/或全身照射），在異體移植期間還需使用預防急性移植物抗宿主病（GvHD）的藥物。
4. 造血幹細胞通常於層流空氣室內輸注。
5. 移植後，患者會接受輸血與支持性治療，包括粒細胞刺激因子（G-CSF）、抗生素以及周邊靜脈營養等。

Early Complications (Within 30–60 Days Post-Transplant)

• Hemorrhagic cystitis

• Endothelial injury syndromes:

  • Veno-occlusive disease (VOD)

  • Capillary leak syndrome

  • Engraftment syndrome

  • Diffuse alveolar hemorrhage

  • Transplant-associated thrombotic microangiopathy (TA-TMA)

  • Idiopathic pneumonia syndrome

• Infections

  • Bacterial infections

  • Fungal infections

  • Viral infections

• Graft-versus-host disease (GvHD)

  • Acute GvHD or chronic GvHD

Late Complications

• Infertility and gonadal dysfunction

早期併發症（移植後30–60天內）

出血性膀胱炎

血管內皮損傷：

• 肝靜脈閉塞症
• 毛細血管滲漏症候群
• 植入症候群
• 瀰漫性肺泡出血
• 移植相關血栓性微血管病變
• 特發性肺炎症候群

感染

細菌感染、黴菌感染、病毒感染

移植物抗宿主病（GvHD）

急性GvHD或慢性GvHD

晚期後遺症

生育能力與性腺功能障礙