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Graft-Versus-Host Disease and Graft-Versus-Leukemia Responses

The ability of allogeneic hematopoietic cell transplantation (HCT) to cure certain hematologic malignancies is widely recognized. An important therapeutic aspect of HCT in eradicating malignant cells is the graft-versus-leukemia (GVL) effect. The importance of the GVL effect in allogeneic HCT has been recognized since the earliest experiments in stem cell transplantation. Forty years ago, Barnes and colleagues noted that leukemic mice treated with a subtherapeutic dose…

Cord Blood Transplantation

Cord Blood as a Stem Cell Source It is well established that ethnic minorities have a low likelihood of matching with an unrelated hematopoietic stem cell donor. In recent years, there has been increasing interest in alternative stem cell donors given the genetic heterogeneity of our population coupled with diminishing family size. Cord blood (CB) represents an alternative donor type for hematopoietic cell transplantation (HCT) and…

Haploidentical Hematopoietic Stem Cell Transplantation

Introduction Allogeneic hematopoietic stem cell transplantation (alloHSCT) is an effective treatment for a wide range of hematologic malignancies, nonmalignant hematologic diseases, and immunologic disorders. Sources of donor stem cells for alloHSCT include human leukocyte antigen (HLA)-matched siblings (MSD), HLA-matched unrelated adult donors (MUD) matched at least at HLA-A, -B, -C, -DRB1, HLA-mismatched unrelated donors, unrelated umbilical cord blood, and HLA half-matched related donors, mismatched at 2…

Unrelated Donor Hematopoietic Cell Transplantation

The outcomes of unrelated donor hematopoietic cell transplantation (HCT) have greatly improved as a result of a better understanding of the diversity of human leukocyte antigen (HLA) and killer-cell immunoglobulin-like receptor (KIR) genes. The HLA and KIR genetic systems regulate the transplantation barrier. Clinical outcomes after unrelated donor transplantation can be achieved with donor matching for the highly polymorphic HLA loci and through consideration of donor…

Indications, Outcomes, and Donor Selection for Allogeneic Hematopoietic Cell Transplantation for Hematologic Malignancies in Adults

Background Early reports of leukemia-free survival, following human leukocyte antigen (HLA)-identical sibling hematopoietic cell transplantation (HCT) for refractory acute leukemia, was reported in the 1970s. Since then, allogeneic HCT has evolved into an effective and increasingly safe, curative, and intent therapeutic modality for hematologic malignancies. The indications and applicability of HCT for specific diseases change over time, especially as non-transplant treatment options and our understanding of…

Principles of Cell-Based Genetic Therapies

The use of gene transfer to treat human diseases has now been demonstrated to be efficacious in a growing number of indications. Proof-of-principle successes in several monogenic diseases—both hematologic and non-hematologic—have been published and widely publicized in the past decade. The previous occurrence of serious adverse events in some trials related to insertional mutagenesis has stimulated the development of safer vector systems and the improved efficiency…

Graft Engineering and Cell Processing

It has been more than 70 years since allogeneic hematopoietic stem cells (HSC) were first transplanted to human recipients in the form of bone marrow. Since that time, the source of HSC has widened to include autologous bone marrow, growth factor mobilized peripheral blood progenitor cells (PBPCs), umbilical cord blood, and more. In parallel, there has been the development of other cellular therapies to treat a…

Practical Aspects of Hematopoietic Stem Cell Harvesting and Mobilization

Hematopoietic stem cell (HSC) products for autologous or allogeneic transplantation are available from bone marrow (BM), peripheral blood, or umbilical cord blood (UCB) sources. Bone marrow was the original source of cells for transplantation because of the ease and reliability of collecting adequate numbers of cells for transplantation, and it remains the standard with which other sources of HSCs are compared. Peripheral blood stem cell (PBSC)…

Therapy-Related Late Effects of Hematologic Malignancies

The past several decades have seen a marked improvement in survival for patients with hematologic malignancies. The 5-year survival is as follows: leukemia, 66%; Hodgkin lymphoma (HL), 89%; non-Hodgkin lymphoma (NHL), 75%; and multiple myeloma (MM), 54%. As a consequence, the population of long-term cancer survivors continues to grow. As of January 1, 2019, there were 451,700 leukemia survivors living in the United States, 234,890 HL…

Palliative Care

Palliative care for seriously ill children and adults is provided by an interdisciplinary team of clinicians who focus on communication about values and goals of care and ameliorating suffering from physical, social, spiritual, existential, and psychological concerns. Members of the interdisciplinary palliative care team include registered and advanced practice nurses, physician assistants, social workers, pharmacists, chaplains, and adult and pediatric physicians, many of whom are ABIM…

Pain Management and Antiemetic Therapy in Hematologic Disorders

Relieving pain in patients with hematologic disorders requires a multifaceted approach. This chapter will provide the clinician with the tools to perform a systematic evaluation of the pain complaint and propose a rational, evidence-based, strategy. After the source and type of the pain has been identified and assessed, appropriate nonpharmacologic and pharmacologic therapies can be initiated. The approach to managing pain in hematologic disorders is largely…

Psychosocial Aspects Of Hematologic Disorders

Major advances in cancer diagnostics and therapeutics have led to increased survival and improved health outcomes for people diagnosed with hematologic malignancies. However, high levels of psychological distress (e.g., depression) and diminished positive psychological well-being factors (e.g., optimism) exist in this population for myriad reasons, including the cancer diagnosis itself, uncertain prognosis, prolonged treatment course that entails numerous hospitalizations with extended periods of isolation, and potential…

Identification and Management of Checkpoint Inhibition Toxicity

Checkpoint inhibitors revolutionized cancer therapy and the field of oncology when the first drug of this class was introduced for melanoma treatment. But their therapeutic benefit comes at a cost of immunotherapy-related adverse events (IrAEs). Checkpoint inhibitors are immunomodulatory antibodies that are used to activate the immune system to detect and destroy malignant cells. Several immune checkpoint receptors are targeted by different immunotherapy agents, such as…

Grading and Toxicity Management after Immune Effector Therapy

The growing use of novel immunotherapies has revolutionized the management of hematologic malignancies over the past several years. While historically cytotoxic chemotherapy has been the standard treatment approach, newer cellular therapies have demonstrated promising activity in patients with relapsed and refractory hematologic malignancies and have significantly improved outcomes in this setting. The increased use of these innovative therapies has highlighted a set of unique toxicities which…

Principles of Radiation Therapy for Hematologic Disease

The approach and utilization of radiation therapy (RT) for the treatment of hematologic malignancies have seen many changes throughout the years. Advances in imaging and technology have allowed for more precise delineation and targeting of disease. This, along with a reduction in radiation doses and treatment volume, have resulted in increased sparing of normal tissues and dramatically reduced short- and long-term toxicities previously associated with RT.…

Key Considerations for Managing Infections in the Compromised Host

Advances in the supportive care during the treatment of hematologic malignancies have improved the life expectancy of afflicted patients. However, this progress is increasingly at the expense of developing a wider range of infectious complications caused by drug-resistant organisms. The clinical approach to infections occurring among hematology patients involves consideration of host immune system defects, and skin or mucosal barrier disruptions that predispose patients to infection…

Immunoglobulin Light-Chain Amyloidosis (Primary Amyloidosis)

Amyloidosis is defined as the clinical syndrome associated with deposition of amyloid. Amyloid in tissue is defined by its tinctorial properties of a homogeneous, eosinophilic, hyaline material when viewed by hematoxylin and eosin staining. Amyloid stains specifically with Congo red, demonstrating a deep pink amorphous composition. Although Congo red staining is the sine qua non for the diagnosis of amyloid, many pathology laboratories prefer the use…

Waldenström Macroglobulinemia/Lymphoplasmacytic Lymphoma

Waldenström macroglobulinemia (WM) is a lymphoid neoplasm resulting from the accumulation, predominantly in the bone marrow (BM), of a clonal population of lymphocytes, lymphoplasmacytic cells, and plasma cells, which secrete a monoclonal immunoglobulin (Ig)M. WM corresponds to lymphoplasmacytic lymphoma (LPL) as defined in the World Health Organization classification system. Most cases of LPL are WM; less than 5% of cases secrete IgA, IgG, kappa, lambda, or…

Multiple Myeloma

Clonal plasma cell (PC) neoplasms are comprised of a number of interrelated and partially overlapping entities, ranging from benign to fully malignant, which are variously defined and categorized by burden of disease, presence of organ injury, anatomic location, and in some cases, unique constellations of signs, symptoms, and pathologic findings (see box on Rare Disorders Associated With Monoclonal Proteins and/or Plasma Cells , and also Chapter…

Monoclonal Gammopathy of Undetermined Significance and Smoldering Multiple Myeloma

Monoclonal gammopathy of undetermined significance (MGUS) is a premalignant clonal plasma cell disorder. It is characterized by the presence of a monoclonal (M) protein in the serum or urine that is commonly discovered during laboratory work-up of a variety disorders. MGUS is a precursor to various hematologic disorders including multiple myeloma (MM), Waldenström macroglobulinemia (WM), solitary plasmacytoma, and immunoglobulin light chain amyloidosis (AL). Smoldering MM (SMM)…