Hematopoietic Cell Transplantation Nursing CE Course

ion, differentiation, and apoptosis. As individuals age, HSC function declines, leading to a weakened immune response, increased susceptibility to infections, and reduced regenerative capacity. Aging is also associated with a gradual decline in blood volume, decreased plasma protein levels, and diminished lymphoid progenitors, contributing to an increased risk of age-related hematologic disorders. As the aging process progresses, there is a noticeable reduction in RBC and WBC production, while platelet levels generally remain stable. Lymphocytes become less responsive to antigens over time, antibody production decreases, and immune response time slows, making older adults more vulnerable to infections. The immune response in older adults tends to be less robust, often resulting in lower-than-expected WBCs, even during active infections. Hemoglobin levels also decline beginning around the fourth decade of life, with this trend continuing into older adulthood. Beyond age-related changes, abnormal bone marrow pathology can result from genetic mutations, environmental exposures, infections, malignancies, or autoimmune conditions. These disruptions can manifest as either bone marrow failure disorders, where blood cell production is insufficient, or proliferative disorders, where unregulated abnormal cell growth occurs. Both conditions can severely impact oxygen transport, immune defense, and blood clotting, increasing the risk of anemia, recurrent infections, and bleeding complications. Understanding these changes is essential for identifying and managing hematologic disorders in normal aging and pathologic bone marrow conditions (Ignatavicius et al., 2020; Zhang et al., 2023).

Bone Marrow Failure Disorders

Bone marrow failure occurs when the bone marrow’s capacity to generate RBCs, WBCs, and platelets is significantly compromised. These disorders fall into two categories: acquired and inherited. Acquired bone marrow failure results from external influences or somatic mutations that disrupt normal hematopoiesis. These disruptions may stem from autoimmune processes, infections, malignancies, or environmental toxins. One of the most well-known examples is aplastic anemia (AA), which is marked by hypocellular bone marrow and pancytopenia, a reduction in all blood cell types. AA can be acquired through viral infections (e.g., Epstein-Barr virus, hepatitis), exposure to toxic chemicals (e.g., benzene, chemotherapy), or autoimmune destruction of HSCs. Inherited forms arise from germline mutations, either passed down from parents or emerging spontaneously. In addition to symptoms commonly seen in AA, such as fatigue, increased bleeding, and recurrent infections, these disorders often present with unique extramedullary manifestations specific to each syndrome. Fanconi anemia (FA), for example, is an inherited disorder caused by defects in DNA repair mechanisms, leading to progressive bone marrow failure and a significantly heightened risk of malignancies (Deng & McReynolds, 2024; Moore & Krishnan, 2023).

Another key disorder associated with bone marrow failure is myelodysplastic syndrome (MDS), which occurs when HSCs acquire genetic mutations that impair normal blood cell production. In MDS, the bone marrow produces dysplastic (abnormally shaped) cells that fail to function properly, leading to cytopenias (low levels of RBCs, WBCs, or platelets) and increased risk of transformation to acute myeloid leukemia (AML). AML is a rapidly progressing blood cancer characterized by the unregulated proliferation of myeloblasts, immature WBCs that overwhelm the bone marrow and impair normal hematopoiesis. Bone marrow failure disorders, particularly MDS and AML, are more commonly seen in older adults and may be associated with previous chemotherapy, radiation exposure, or environmental toxins. Understanding the underlying mechanisms of bone marrow failure is crucial for early diagnosis, risk assessment, and treatment strategies aimed at improving patient outcomes (Deng & McReynolds, 2024; Moore & Krishnan, 2023).

Myeloproliferative Neoplasms and Leukemias

Myeloproliferative neoplasms (MPNs) are marked by the overproduction of one or more types of mature myeloid blood cells. MPNs are driven primarily by genetic mutations that lead to the uncontrolled growth of hematopoietic cells. Unlike MDS, where blood cells are often dysfunctional, MPNs produce functional, mature blood cells but in excessive amounts, increasing the risk of complications such as thrombosis, bleeding, and organ enlargement (e.g., splenomegaly). Over time, some MPNs may progress to secondary myelofibrosis or AML. The most common types of MPNs include polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF). These conditions are frequently linked to mutations in the Janus kinase 2 (JAK2) gene, which encodes the JAK2 protein—a key component of cell signaling pathways that regulate blood cell production. PV leads to an overproduction of RBCs, resulting in increased blood viscosity and a heightened risk of thrombosis. ET is characterized by excessive platelet production, which can cause abnormal clotting or, paradoxically, an increased risk of bleeding. PMF involves the progressive scarring of the bone marrow, impairing normal blood cell production and leading to extramedullary hematopoiesis (Thapa et al., 2023).

Abnormal hematopoietic cell proliferation is also a defining feature of leukemias. Acute leukemias—including AML and acute lymphoblastic leukemia (ALL)—arise from mutations in hematopoietic stem or progenitor cells, leading to uncontrolled growth of immature (blast) cells. These malignant cells rapidly accumulate in the bone marrow, crowding out normal cells and resulting in anemia, increased infection risk, bleeding, and organ infiltration. In contrast, chronic leukemias, such as chronic myeloid leukemia (CML) and chronic lymphocytic leukemia (CLL), involve the overproduction of more mature but functionally defective blood cells. CML is driven primarily by the breakpoint cluster region-Abelson murine leukemia (BCR-ABL) fusion gene. The BCR-ABL genetic abnormality occurs when segments of chromosome 9, which harbor the ABL1 gene, exchange with segments of chromosome 22, containing the BCR gene. This translocation creates a hybrid gene commonly linked to CML and is also known as the “Philadelphia chromosome” because it was first identified in Philadelphia. The resulting fusion gene produces an abnormal protein that instructs blood cells to make too much tyrosine kinase, a protein that drives the uncontrolled cell growth seen in cancer (Chennamadhavuni et al., 2023).

Bone Marrow Infiltrative and Autoimmune Disorders

Nonhematologic malignancies, such as metastatic cancers (e.g., breast, prostate, lung), can infiltrate the bone marrow, disrupting normal hematopoiesis and leading to cytopenias. Multiple myeloma (MM), a plasma cell malignancy, results in excessive monoclonal antibody production, bone marrow suppression, and osteolytic bone lesions. Additionally, autoimmune diseases like systemic lupus erythematosus (SLE) and rheumatoid arthritis (RA) can lead to immune-mediated destruction of hematopoietic cells, further exacerbating bone marrow dysfunction. Overall, abnormal bone marrow pathology encompasses a wide spectrum of disorders that impair hematopoietic function, leading to significant morbidity and mortality (Giordano et al., 2024).

Transplant Types

An HCT is a procedure in which healthy stem cells are infused into a patient to replace damaged or diseased bone marrow. Initially, allogeneic transplants were the only type available, but advancements in medicine have led to the development of autologous and syngeneic transplants. An allogeneic transplant involves receiving HSCs from a genetically matched or partially matched donor, which can be a sibling, unrelated donor, or umbilical cord donor. Human leukocyte antigen (HLA) matching, which will be discussed in greater detail later in this module, is critical to reduce the risk of graft-versus-host disease (GVHD) and improve transplant success. GVHD is a potentially serious complication of transplant where the donor’s immune cells attack the recipient’s tissues. Allogeneic transplant is most commonly used for hematologic malignancies such as leukemias and lymphomas, bone marrow failure syndromes, and immune disorders. With an autologous HCT, the patient’s own hematopoietic cells are harvested before receiving high-dose chemotherapy or radiation therapy. After treatment, the stored cells are reinfused to help restore bone marrow function. Autologous HCT is commonly used for MM, certain lymphomas, and autoimmune disorders, as it eliminates the risk of immune rejection or GVHD. Syngeneic HCT is a rare type of transplant in which stem cells are donated by an identical twin. Since the donor and recipient share the exact same genetic makeup, there is no risk of GVHD or immune rejection (American Cancer Society [ACS], 2023; Ignatavicius et al., 2020; Negrin, 2025b).

HCT is commonly referred to as hematopoietic stem cell transplant (HSCT) or bone marrow transplant (BMT). While these terms are sometimes used interchangeably, they have distinct meanings based on the type and source of hematopoietic cells used in transplantation. HCT is the broadest term, encompassing the transplantation of any hematopoietic cell, including stem cells, progenitor cells, and more mature hematopoietic cells. HCT refers to the overall process of replacing diseased or damaged bone marrow with healthy hematopoietic cells and can involve bone marrow, peripheral blood stem cells (PBSCs), or umbilical cord blood as the cell source. HSCT specifically refers to the transplantation of HSCs, regardless of whether they come from bone marrow, peripheral blood, or umbilical cord blood. BMT is a specific type of HSCT where stem cells are directly extracted from the donor’s bone marrow, typically from the pelvis (iliac crest), under anesthesia. Historically, BMT was the standard method for hematopoietic transplantation, but PBSCT has largely replaced it due to its less invasive stem cell collection and faster engraftment. Engraftment is the process by which transplanted cells grow and produce new blood cells. With PBSCT, the donor is given a WBC growth factor to stimulate increased production of circulating HSCs. These cells are then collected directly from the bloodstream using an apheresis machine, which is a specialized device that separates the blood into its different components, such as plasma, platelets, WBCs, and RBCs. Although less common, BMT is still preferred to treat some conditions, such as AA, severe immune deficiencies, and certain leukemias, as it carries a lower risk of chronic GVHD. The best type of transplant and stem cell source is determined based on the patient’s diagnosis, age, overall health, and diagnostic test results. Factors such as disease type, availability of a suitable donor, and risk of complications like GVHD play a crucial role in selecting a transplant type. Table 1 outlines the different types of transplants in each category and some of their most common indications (ACS, 2023; Aplastic Anemia and MDS International Foundation, n.d.; Kelkar & Banerjee, 2021; Saad et al., 2020).

 

Table 1

Types of Transplants and Indications

Allogeneic Transplantation

Common Indications: Acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), myelodysplastic syndrome (MDS) Types: Matched Sibling Donor Transplant Stem cells come from an human leukocyte antigen (HLA)–matched sibling (best possible match) Reduces the risk of graft-versus-host disease (GVHD) compared to unrelated donors Best survival outcomes among allogeneic transplants Matched Unrelated Donor Transplant The donor is an HLA-matched but unrelated person identified through a donor registry Used when no sibling match is available Higher risk of GVHD compared to sibling donors, requiring stronger immunosuppressive therapy Haploidentical (Half-Matched) Transplant The donor is a half-matched family member, using a parent, child, or sibling Expands donor availability, especially for patients without a full HLA match Requires specialized techniques like T-cell depletion or posttransplant cyclophosphamide (Cytoxan) administration to reduce GVHD risk Umbilical Cord Blood Transplant  Stem cells are collected from the umbilical cord and placenta immediately after birth and stored frozen in cord blood banks until needed More tolerant to HLA mismatches, making it useful for patients without fully matched donors Slower engraftment due to lower stem cell count, sometimes requiring double cord blood units for adults Commonly used in pediatric patients

Autologous Transplantation

Common Indications High-risk multiple myeloma (MM), Hodgkin Lymphoma (HL), non-Hodgkin Lymphoma (NHL), autoimmune disorders, and certain solid tumors such as germ cell and neuroblastoma Types Single Autologous Transplant The patient’s stem cells are collected, stored, and reinfused after treatment Tandem Autologous Transplant Two sequential autologous transplants are performed several weeks apart Increases disease control but carries a higher toxicity Autologous with Reduced-Intensity Conditioning  Uses lower-dose chemotherapy before stem cell infusion Beneficial for older or frail patients who cannot tolerate full-intensity regimens

Syngeneic Transplantation

Common Indications Leukemia, lymphoma, aplastic anemia (AA), genetic disorders No need for immunosuppressive therapy since the immune system fully recognizes the cells as “self” Extremely rare due to the low frequency of identical twin donors

(ACS, 2023; Aplastic Anemia and MDS International Foundation, n.d.; Kelkar & Banerjee, 2021; Saad et al., 2020)

Prevalence and Eligibility

The incidence of HCT has grown significantly in recent years as it has become a pivotal treatment for various conditions. According to a recent analysis of national transplant registry data by Kuwatsuka and colleagues (2024), AML accounts for 39% of allogeneic HCTs, while ALL accounts for 22%, MDS for 11%, and malignant lymphoma for 10%. Data from the Center for International Blood and Marrow Transplant Research (CIBMTR) indicates that 22,827 HCTs were performed in the United States in 2021. Among these, 5,073 (22%) involved unrelated donors, while 4,276 (19%) were from related donors. PBSCs were the most used source for transplantation, comprising 77% of related donor transplants and 80% of unrelated donor transplants. Bone marrow was more frequently utilized in related donor transplants (23%) compared to unrelated donor transplants (13%). Umbilical cord blood was used in a smaller percentage of cases, accounting for less than 1% of related transplants and 7% of unrelated transplants. These figures highlight the increasing reliance on PBSCs as the preferred stem cell source while indicating the continued, though limited, use of bone marrow and cord blood for transplantation (Health Resources & Services Administration [HRSA], 2023).

Patient Eligibility

Determining patient eligibility for HCT is a complex process involving disease-specific criteria, comprehensive health evaluations, and psychosocial assessments. Adhering to evidence-based guidelines and fostering multidisciplinary collaboration is essential for selecting appropriate candidates and maximizing the likelihood of successful transplantation while minimizing risks. The decision to proceed with HCT is influenced by the disease’s stage, prognosis, and responsiveness to prior treatments. Early referral to a transplant center is critical, as timely intervention can significantly improve patient outcomes. A thorough evaluation of overall health is necessary, as factors such as age, performance status, comorbidities, and organ function play a pivotal role. While there is no absolute age limit, older patients may face increased risks. However, select individuals over 65 years of age may still be considered based on their physical condition. Performance status, commonly measured using the Eastern Cooperative Oncology Group (ECOG) scale or Karnofsky Performance Status (KPS), helps assess a patient’s ability to tolerate the intensive transplantation process (Holmberg & Sandmaier, 2025; Kanate et al., 2020).

Comorbid conditions—including cardiovascular, pulmonary, renal, and hepatic diseases—must be carefully evaluated, as they can influence transplant-related morbidity and mortality. Pulmonary function tests, for instance, help assess respiratory status, with a hemoglobin-corrected diffusion lung capacity (DLCO) greater than 50% often considered acceptable for proceeding with HCT (Holmberg & Sandmaier, 2025; Kanate et al., 2020). The HCT-comorbidity index (HCT-CI) is a validated risk assessment tool used to predict nonrelapse mortality (NRM) and overall survival in patients undergoing allogeneic HCTs. Developed by Mohamed Sorror and colleagues in 2005, the HCT-CI evaluates the impact of preexisting comorbid conditions on transplant outcomes by assigning weighted scores based on the severity of various organ system impairments. The HCT-CI provides a standard method for assessing transplant-related risks beyond traditional factors such as age and disease status, helping to guide treatment decisions by identifying patients at higher risk of complications or mortality post transplant. It also allows for personalized risk stratification for pretransplant counseling and adjustments in conditioning regimens (Sorror et al., 2005).

Beyond physical health, psychosocial factors play a crucial role in the transplant process and recovery. A comprehensive pretransplant evaluation should identify any psychosocial challenges that could interfere with a successful outcome. This includes assessing the patient’s mental health, social support systems, financial stability, and ability to adhere to posttransplant care requirements. Engaging a multidisciplinary team—including social workers, psychologists, and financial counselors—ensures patients receive the necessary support to navigate the complexities of HCT. Evaluating eligibility for HCT necessitates a collaborative effort among HCPs. Transplant coordinators, nurses, advanced practice providers, social workers, financial specialists, and healthcare providers each serve an important role in holistically evaluating the patient’s condition. This team-based approach facilitates informed decision-making, ensuring that all aspects of the patient’s health and well-being are considered, and is vital for optimizing patient outcomes and aligning treatment plans with the individual’s needs and circumstances (Holmberg & Sandmaier, 2025; Kanate et al., 2020; National Marrow Donor Program [NMDP], n.d.-b).

Donor Eligibility

The NMDP, through its Be the Match Registry, maintains an extensive database of potential donors and cord blood units, facilitating matches between patients and donors. To ensure donor and recipient safety and the best possible outcomes, the NMDP has established specific eligibility criteria. Prospective donors are generally between 18 and 40 years old, as younger donors are associated with improved transplant outcomes. Individuals must be in good health and free from conditions such as HIV, hepatitis B or C, and severe cardiovascular diseases. Certain autoimmune disorders, active infections, hematologic disorders, and recent cancer diagnoses may also disqualify potential donors. Pregnant patients can register but are deterred from donation until full postpartum recovery. Additionally, those with controlled hypertension or well-managed diabetes without significant complications may be considered eligible. A history of substance use disorder may affect eligibility, depending on the duration of sobriety and overall health status. These guidelines are designed to protect both donors and recipients, ensuring the highest likelihood of a successful transplant. For detailed information on donor eligibility and the registration process, individuals are encouraged to visit the Be the Match website (Negrin, 2025a; NMDP, n.d.-a).

Human Leukocyte Antigen

HLAs are specialized proteins found on the surface of most cells in the body, except RBCs. These proteins are encoded by genes within the major histocompatibility complex (MHC) on chromosome 6 and play a crucial role in the immune system’s ability to distinguish between self and nonself. Each individual possesses a unique set of HLA proteins, which are inherited from both parents and help the immune system differentiate between the body's cells and foreign invaders. The primary function of HLAs is to present antigenic peptides to T cells, thereby initiating an immune response when foreign substances are detected. When the immune system encounters cells lacking the individual’s specific HLA markers, it recognizes them as foreign and mounts a response to neutralize or eliminate these potential threats. This mechanism is vital for defending against infections but poses challenges in organ and tissue transplantation. It is essential to match HLA types between donors and recipients as closely as possible to reduce the risk of transplant rejection (American Association for Clinical Chemistry [AACC], 2021; Delves, 2024; Ignatavicius et al., 2020).

HLA typing, also known as tissue typing or histocompatibility testing, is a meticulous process performed to assess compatibility between a donor and a recipient to maximize transplant success and minimize immune complications. This process involves analyzing blood or buccal swab samples to identify specific HLA alleles and detect any preexisting antibodies that might target donor tissues. High-resolution HLA typing provides detailed information about the alleles present. It is a critical factor in the success of HCT, as it determines how well the components of a donor’s immune system match those of the recipient. For HCT, the most important HLA loci used to assess compatibility between donor and recipient are HLA-A, HLA-B, HLA-C, and HLA-DRB1. These are four key sites within the MHC that encode proteins crucial for immune system function. For HCT, guidelines recommend selecting donors who are matched at least at HLA-A, -B, -C, and -DRB1 loci. In cases where a fully matched related donor is unavailable, alternative options include matched unrelated donors, cord blood units, or haploidentical (half-matched) donors, often a parent or a child. The closer the match between the donor and the recipient at these sites, the lower the risk of immune complications. HLA matching is typically reported on a scale of 8 or 10, depending on whether HLA-DQB1 (another locus) is included in the evaluation. A closely matched HLA improves engraftment, reduces GVHD risk, enhances overall survival rates, and decreases the need for intensive immune suppression therapy. In addition to matching, antibody screening ensures the recipient does not have preformed antibodies against the donor’s HLAs. Table 2 provides an overview of HLA matching and types of donors for HCT (Delves, 2024; Khaddour et al., 2023; Mangum & Caywood, 2022).

Table 2

Human Leukocyte Antigen Matching and Types of Donors for Hematopoietic Cell Transplantation

 

Human Leukocyte Antigen (HLA) Matching

8/8 match Perfect match Matching at HLA-A, HLA-B, HLA-C, and HLA-DRB1 (each inherited from both parents) 10/10 match Optimal match Includes HLA-DQB1 7/8 or 9/10 match One mismatch at any of these loci, which may be acceptable depending on the clinical scenario

Types of Hematopoietic Cell Transplantation (HCT) Donors and Matching

Matched related donor Usually a sibling with a full 8/8 or 10/10 HLA match Matched unrelated donor Ideally, a full 8/8 or 10/10 match Haploidentical donor Half-matched A parent, child, or sibling with 50% HLA match (5/10) Umbilical cord blood transplant Requires fewer HLA matches (e.g., 4/6 or 6/8 match) due to the immature nature of cord blood immune cells

(Delves, 2024; Khaddour et al., 2023; Mangum & Caywood, 2022)

Despite efforts to find suitable donors, challenges persist, particularly for patients from diverse ethnic backgrounds. The probability of finding a fully matched donor varies among populations, with non-Hispanic Black individuals facing lower match rates compared to non-Hispanic White individuals. Innovative approaches, such as utilizing cryopreserved bone marrow from deceased donors, are being explored to expand the donor pool and improve match availability for all patients. Accurate HLA typing and matching are essential to minimize rejection risks and enhance transplant outcomes. Nurses play a crucial role in this process by understanding the importance of HLA compatibility, educating patients about the matching process, and monitoring for potential complications associated with HLA mismatches. Their involvement is crucial in coordinating care and providing support throughout the HCT journey (Delves, 2024; Khaddour et al., 2023; Mangum & Caywood, 2022).

Harvesting and Transplanting 

Once a compatible donor is identified, the collection of the transplant material begins—a process that varies according to the donation type. For example, patients scheduled for PBSCT undergo a three-phase procedure: mobilization (using medications to stimulate the release of stem cells into circulation), apheresis (the collection phase), and subsequent reinfusion of the harvested cells. Typically, transplant centers admit recipients to the hospital for a preparative conditioning regimen that takes approximately four to seven days before the transplant occurs. During conditioning, chemotherapy and/or radiation therapy are administered to suppress the immune system and create space in the bone marrow for the incoming cells. These days are counted as negative numbers, with the day of transplant designated as day 0; each following day is then labeled day 1, day 2, and so forth. In the first 14 days post transplant, patients require intensive monitoring for complications such as fever, hypertension, nausea, vomiting, hypovolemia, and electrolyte imbalances (Ignatavicius et al., 2020; Majhail, 2021; Negrin, 2025b).

Following infusion via a central venous catheter, the transplanted cells circulate throughout the body and eventually home to the bone marrow, where they initiate engraftment. Typically beginning between 14 and 21 days post transplant, engraftment is closely observed for signs of complications like engraftment syndrome, which may include fever and weight gain. Since the pace of engraftment varies among individuals, clinicians monitor progress daily by checking neutrophil and platelet counts. Engraftment is generally considered complete when the absolute neutrophil count (ANC) exceeds 500 cells/mm³ for three consecutive days, and platelet counts stabilize between 20,000/mm³ and 50,000/mm³ without transfusions. Although many patients are discharged from the acute care setting approximately 35 days after the transplant, they are advised to remain within a reasonable distance from the transplant center for at least 100 days post procedure. Full recovery may take several months for autologous transplants and up to two years for syngeneic or allogeneic transplants. Throughout the recovery period, HCPs continue to monitor complete blood counts and perform bone marrow biopsies to assess transplant effectiveness and manage any emerging complications (Ignatavicius et al., 2020; Majhail, 2021; Negrin, 2025b).

Autologous Transplant

For autologous transplants, patients usually require growth factors or chemotherapeutic agents to stimulate stem cell production. In hematologic malignancies, plerixafor (Mozobil) is often used to enhance mobilization by accelerating the release of stem cells into the bloodstream, which can reduce the number of apheresis sessions needed. Once an adequate quantity of stem cells are circulating, the patient proceeds to apheresis—the process of harvesting these cells. Although autologous transplants avoid donor compatibility issues, success depends on the patient’s ability to produce a sufficient number of stem cells for collection (Ignatavicius et al., 2020; Majhail, 2021; Negrin, 2025b).

Apheresis is performed over one to five sessions, with one session per day; however, plerixafor (Mozobil) may allow for a successful harvest after just one day. During each apheresis session, blood is extracted from the patient and passed through an apheresis machine that separates the stem cells. The stem cells are then frozen to be reinfused later, and the remaining blood components are returned to circulation. Once apheresis is complete, many patients undergo additional chemotherapy or radiation as part of the conditioning regimen to eliminate any remaining cancer cells and suppress the immune system to reduce the risk of graft rejection. Once conditioning is complete, the stem cells are reinfused into the patient’s bloodstream, where they travel to the bone marrow and begin to produce new blood cells. Following the procedure, patients are closely monitored for signs of treatment toxicity, infection, or potential graft failure (Ignatavicius et al., 2020; Majhail, 2021; Negrin, 2025b).

Allogeneic Transplant

Patients receiving an allogeneic transplant will receive stem cells or bone marrow from a compatible donor. If PBSCs are used, the procedure closely mirrors that of an autologous donation, but the stem cells are sourced from a donor. During allogeneic HCT using bone marrow, the donor is taken to an operating room. Approximately 500 to 1000 mL of bone marrow is aspirated through a large-bore needle from multiple aspiration sites, such as the iliac crest or other long bones. The donated marrow is then filtered, prepared, and transplanted into the recipient. The donor will regenerate their marrow within a few weeks after donation. The patient could also receive an allogeneic transplant using cord blood. Similarly to autologous transplants, allogeneic transplant recipients may undergo a conditioning regimen with chemotherapy or radiation to prepare the body. Once conditioning is completed, stem cells are infused into the recipient’s circulatory system, where they migrate to the bone marrow and begin to produce new blood cells. Post transplant, patients are closely monitored to detect any potential complications, including infections, GVHD, and organ toxicity (Ignatavicius et al., 2020; Majhail, 2021; Negrin, 2025b).

In the case of a syngeneic transplant from an identical twin, the harvesting and infusion procedures are largely similar to those used in allogeneic transplants. However, despite the high level of compatibility, these patients remain vulnerable to the adverse effects of the conditioning regimen, such as infection, organ toxicity, pulmonary edema, and heart failure (Ignatavicius et al., 2020; Majhail, 2021; Negrin, 2025b).

 

Side Effects and Complications

Patients undergoing HCT may experience a broad range of side effects, many of which result from the high-dose chemotherapy and radiation used during the conditioning phase. Since these treatments target rapidly dividing cells, patients often develop mucositis (inflammation and ulceration of the mucosal lining), diarrhea from gastrointestinal damage, and alopecia as a result of damage to hair follicles. Typically, hair regrowth begins within two to three months after treatment ends. In addition to these acute side effects, HCT recipients face risks that vary based on factors such as the underlying disease, patient age, overall health, and the specifics of the transplant process. Common complications include GVHD, graft failure, infections, and potential injury to internal organs. Long-term risks can involve reproductive issues, secondary malignancies—often emerging three to five years post transplant—and, in severe cases, death. The likelihood of permanent infertility depends largely on the type and dosage of chemotherapy or radiation administered (Ignatavicius et al., 2020; Khaddour et al., 2023; Majhail, 2021).

Hospital stays following HCT can range from several days to months, largely depending on the patient’s recovery and the need for supportive care. Many patients require ongoing blood transfusions to maintain adequate blood counts until the transplanted marrow becomes fully functional. For instance, whole blood or packed red blood cell transfusions may be necessary to manage anemia, while platelet transfusions are used to address or prevent bleeding. Moreover, the immunosuppressive state induced by both the conditioning regimen and posttransplant medications significantly increases the risk of infection for months or even years following transplantation. To mitigate this risk, patients should receive detailed discharge instructions on infection prevention, emphasizing strict hand hygiene and other infection control practices. Prophylactic antibiotics and other supportive therapies are commonly prescribed, and regular follow-up visits are essential to monitor and manage any infectious complications that may arise (Ignatavicius et al., 2020; Khaddour et al., 2023; Majhail, 2021).

Graft-Versus-Host Disease

GVHD is a serious complication of HCT that occurs when donor T cells mistakenly recognize the recipient’s tissues as foreign and launch an immune attack. Patients are at the greatest risk for GVHD if they undergo an allogeneic transplant, but it is still a possibility in autologous transplantation if a strong immune response is initiated. Approximately 25% to 50% of allogeneic transplant recipients experience some degree of GVHD, with about 15% dying from complications. GVHD is generally categorized as acute or chronic. Acute GVHD usually manifests within the first three months post transplant, with early symptoms including a mild skin rash, liver dysfunction, and gastrointestinal symptoms. In contrast, chronic GVHD may develop months or even years later, potentially affecting multiple organ systems. Around 30% to 70% of allogeneic transplant recipients develop chronic GVHD. Patients with chronic GVHD might experience musculoskeletal pain; respiratory symptoms such as shortness of breath; ocular dryness; skin changes, including rashes and jaundice; and gastrointestinal issues like xerostomia, mucositis, nausea, vomiting, and diarrhea. Interestingly, a mild form of acute GVHD in cancer patients can be beneficial, as it may indicate that the transplant is working to destroy the malignant cells. Statistically, cancer patients with mild GVHD have a decreased recurrence rate (Ignatavicius et al., 2020; Khaddour et al., 2023; National Organization for Rare Disorders [NORD], 2024).

Prevention of GVHD is key. Patients are typically treated with immunosuppressive medications such as cyclosporine (Neoral), tacrolimus (Prograf), methotrexate (Reditrex), corticosteroids, mycophenolate mofetil (Cellcept), or antithymocyte globulin (Atgam). While these agents reduce the risk of GVHD, they also dampen the immune system, further increasing the patient’s vulnerability to infections. GVHD can be severe and life-threatening and must be recognized and treated promptly to reduce morbidity and mortality. Effective management requires careful titration of immunosuppressive therapy to minimize its severity while still allowing for proper donor cell engraftment. Recent advances include the FDA’s approval of ibrutinib (Imbruvica), in 2017, for treating chronic GVHD after other therapies have failed and abatacept (Orencia), in 2021, for preventing acute GVHD, marking significant progress in the field (Khaddour et al., 2023; NORD, 2024).

Graft Failure 

Graft rejection, or graft failure, occurs when the bone marrow fails to regain proper function after the infusion of HSCs or when no functional improvement is observed following the transplant. This complication is more common in settings with significant HLA mismatch—such as with cord blood or haploidentical donors—and less common with autologous transplants or those from fully matched sibling donors. Contributing factors to graft failure include residual host immune responses against donor cells, an insufficient number of infused cells, damage incurred during cell collection or cryopreservation, an inadequate conditioning regimen, and infections. The recipient should be monitored for chimerism to ensure proper engraftment and a successful transplant. Chimerism refers to the presence of donor-derived cells within the recipient’s blood. It is typically performed by assessing markers such as CD33, which identifies granulocytes, and CD3, which marks T cells, to ensure that the majority of cells in circulation originate from the donor. Numerous studies have demonstrated that effective chimerism is linked to lower relapse rates and improved survival in patients undergoing allogeneic transplantation (Ignatavicius et al., 2020; Khaddour et al., 2023).

Organ Injury and Toxicity

Treatments used in conjunction with HCT may cause organ injury or toxicity. The lungs, liver, kidneys, and bones are at the highest risk of damage. Organ injury and toxicity following HCT can include hepatic sinusoidal obstruction syndrome (SOS, also called veno-occlusive disease [VOD]), renal failure, and pulmonary toxicity. SOS/VOD occurs due to biliary obstruction from damage to the hepatic sinusoids caused by chemotherapy; symptoms usually appear within six weeks of HCT. Chronic kidney disease (CKD) can result from treatment with total body irradiation before transplant but may not present until three to six months later. Pulmonary complications can occur in response to the patient’s immunocompromised state, raising the risk of bacterial, fungal, and viral lung infections. Avascular necrosis (AVN) is a common bone injury observed after HCT in which bone tissue dies due to insufficient blood supply. AVN often results in severe pain and joint dysfunction. While it can affect several joints, the hip is most commonly involved (Khaddour et al., 2023; Negrin, 2025b).

Risks to Donors

Donors involved in HCTs generally face low risks, but there are still potential side effects and complications to consider. For those donating PBSCs, the mobilization process may lead to temporary symptoms such as bone pain, fatigue, headaches, fever, or, in some cases, spleen enlargement. Monitoring during apheresis, particularly when harvesting PBSCs, is essential. The intravenous catheter used for the cell collection process can sometimes become clotted despite the use of anticoagulants. Additionally, donors are at risk of hypocalcemia due to anticoagulants, with symptoms such as numbness, tingling in the extremities, muscle cramps, and even seizures. If these symptoms occur, donors may need calcium supplements. The procedure itself can cause a drop in the donor’s intracellular fluid volume, leading to hypotension. Vital signs should be checked every hour during treatment, and donors should be advised to change positions slowly to avoid dizziness or fainting (ACS, 2020; Ignatavicius et al., 2020; Majhail, 2021).

Donating bone marrow presents risks associated with anesthesia, postoperative pain, and potential complications at the collection sites, including bleeding or infection. Close monitoring is required post procedure. Anesthesia used during the marrow extraction process necessitates appropriate monitoring of the donor’s vital signs, airway, breathing, and neurologic function. Dressings at the aspiration sites should be assessed for signs of bleeding, and fluid volume status should also be closely observed. Pain at multiple aspiration sites should be managed using both pharmacologic and nonpharmacologic methods, such as offering analgesics and repositioning to relieve pressure. Preoperative intravenous fluids should be administered, and donor education on postdonation care, such as infection control procedures and signs of potential complications, is crucial. While recovery varies from donor to donor, most experience relief from fatigue within two to three days, and return to full strength typically occurs within four weeks. While serious risks remain rare, donors may also experience psychological stress associated with the donation process. A thorough pretransplant evaluation and rigorous screening are typically conducted to mitigate risks and ensure donor safety (ACS, 2020; Ignatavicius et al., 2020; Majhail, 2021).

Posttransplantation Care

Posttransplant care in HCT is multifaceted and requires HCPs to integrate clinical expertise, vigilant monitoring, effective symptom management, and compassionate support. In the immediate posttransplant phase, patients are highly vulnerable due to the effects of high-dose chemotherapy or radiation and the consequential immunosuppression. Nurses play a critical role in observing for complications, managing side effects, and providing holistic care to promote recovery. One of the primary responsibilities of nurses is the close monitoring of vital signs and laboratory values. Regular assessments are essential to detect early signs of complications such as infections, bleeding, and electrolyte imbalances. Patients are at increased risk for sepsis, and any indication of fever, tachycardia, or hypotension must be addressed immediately. Nurses also track daily weights, fluid balance, and intake/output to detect dehydration or fluid overload, both of which are common after transplantation (Giaccone et al., 2020; Ignatavicius et al., 2020; Negrin, 2025b).

Managing the side effects of conditioning regimens is another key aspect of posttransplant care. Mucositis, for example, is a frequent complication that can lead to severe pain, impaired oral intake, weight loss, and an increased risk of infection. Nurses implement oral care protocols—including frequent rinsing with prescribed solutions and pain management strategies—to alleviate discomfort and prevent further complications. Similarly, nausea, vomiting, and diarrhea are managed through antiemetic therapies, dietary modifications, and hydration support. Another critical area of focus is the prevention and early detection of GVHD. Nurses monitor for early signs of GVHD, such as skin rashes, liver dysfunction, and gastrointestinal disturbances, and promptly report any abnormalities to the transplant team. Education regarding the importance of adherence to immunosuppressive medications is also vital to help prevent or manage GVHD (Giaccone et al., 2020; Ignatavicius et al., 2020; Negrin, 2025b).

Infection control remains a top priority during the posttransplant period. Due to chronic immunosuppression, patients require to strictly adhere to infection prevention protocols. Nurses enforce hand hygiene, maintain sterile environments, and educate both patients and their families about practices to minimize exposure to pathogens. The use of prophylactic antibiotics, antifungals, and antivirals is common, and nurses must be alert for any adverse reactions or signs of breakthrough infections. Effective pain management and supportive care are essential components of posttransplant nursing care. Patients may experience significant discomfort from the conditioning regimen and procedural aftereffects. Nurses employ both pharmacologic interventions and nonpharmacologic strategies—such as repositioning, relaxation techniques, and the application of cold or warm compresses—to alleviate pain and improve comfort (Giaccone et al., 2020; Ignatavicius et al., 2020; Negrin, 2025b).

Psychosocial support is equally important. The transplant process can be emotionally draining, and patients often experience anxiety, depression, or feelings of isolation. Nurses provide empathetic support, facilitate communication between patients and the health care team, and offer referrals to counseling or support groups when needed. Education on recognizing signs of complications and guidance for postdischarge care further empower patients to participate actively in their recovery. A comprehensive approach to posttransplant care is essential to mitigate complications, promote engraftment, and ensure the best possible outcomes for patients throughout their recovery journey (Giaccone et al., 2020; Ignatavicius et al., 2020; Negrin, 2025b).

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