bluuebunny/crossref_metadata_2025_split huggingface dataset:
Mobilization and Collection of HSC
Authors: Worel Nina , Bilgin Yavuz M. , Wuchter Patrick 
Published: April 2024

AbstractThe intravenous infusion of patient’s own HSC (autologous SCT) to restore BM damage is the basic principle of high-dose chemotherapy, since otherwise the patient would expect long-lasting aplasia with life-threatening infections. Therefore, a sufficient collection of HSC before application of high-dose therapy is mandatory. Since HSC expresses CD34 on their surface, the number of CD34+ cells in the transplant material is considered as an indicator of the HSC content.The aim of infusion of HSC from a donor (allogeneic SCT) is to restore BM damage and to treat the patient’s disease. It represents a permanent cellular immunotherapy by adding a graft versus tumor effect in malignant diseases.
Short- and Long-Term Controls After HCT
Authors: Suárez-Lledó Maria , Rovira Montserrat 
Published: April 2024

AbstractPatients undergoing HCT (mainly allo-HCT) have a risk of developing complications related to pre-, peri-, and post-HCT. The resulting morbidity of the HCT process makes it necessary for patients to adopt a healthy lifestyle that promotes health and contemplate preventive measures for the detection and treatment of possible complications.
The Roles of the HCT Nurse
Authors: Kenyon Michelle , Murray John 
Published: April 2024

AbstractHaematopoietic cell transplantation (HCT) is undoubtedly one of the most challenging and complex forms of treatment for malignant and non-malignant blood disorders as well as autoimmune disease. As a result, nursing in the field of HCT and cellular therapy offers a wealth of opportunities to make a positive difference to patient experience through high-quality specialist nursing care and the unique role of nursing within the transplant MDT. In this section, we will describe the nursing roles that surround the patient pathway and offer an overview the particular aspects of patient care that they encompass.
Neutropenic Fever
Authors: Mikulska Malgorzata 
Published: April 2024

AbstractFever during neutropenia is almost universal after an HCT. In neutropenic HCT recipients, clinicians are faced with a unique combination of issues: (1) high incidence of bacterial bloodstream infections, (2) high mortality in case of infections due to Gram-negative bacteria unless effective antibiotic treatment is provided promptly, and (3) numerous causes of fever other than bacterial infection.
Invasive Fungal Diseases
Authors: Maertens Johan A. 
Published: April 2024

AbstractInvasive fungal diseases are potentially life-threatening infectious complications following HCT and CAR-T cell treatment. The successful introduction of azole-based antifungal prophylaxis has dramatically changed the fungal epidemiology and resistance patterns in many institutions. Timely and accurate diagnosis remains a challenge, especially for mould infections, and relies nowadays heavily on the detection of fungal biomarkers, fungal DNA, and sensitive imaging. Various antifungal strategies are in place, and recommendations regarding drugs of choice are available.
Viral Infections
Authors: Ljungman Per , Piñana Jose Luis , Cesaro Simone , de la Cámara Rafael 
Published: April 2024

AbstractViral infections are important and possibly serious complications to cellular therapies especially allogeneic hematopoietic stem cell transplantation. The most important virus infections are caused by the herpesviruses, adenovirus, and community acquired respiratory viruses including SARS-CoV-2, but also other more rare infections require attention. This chapter discusses some of these infections and their management
Other Life-Threatening Infections
Authors: Martino Rodrigo 
Published: April 2024

AbstractToxoplasma gondii is a protozoan that commonly infects animals and birds. Primary T. gondii infection in humans and other mammals is usually asymptomatic but leads to lifelong latent infection. Transmission to humans occurs by ingesting tissue cysts from undercooked meat or oocysts (released in the feces of cats). Latent cysts can give rise during immunosuppression to a severe localized reactivation producing, for example, toxoplasma encephalitis or chorioretinitis, with dissemination being common (Martino, et al.Clin Infect Dis 2000;31:1188–95; Martino et al. Clin Infect Dis 2005;40:67–78; Tomblyn et al., Biol Blood Marrow Transplant 2009;15:1143–23; Martino, et al., Clin Infect Dis 1996;23:419–20.).Very detailed guidelines for the management and diagnosis of toxoplasmosis following HCT have been recently finalized for the European Conference of Infections in Leukemia 9th Meeting (ECIL-9) conference and are being considered for publication as of June 2023 (Aerts et al., Under peer-review, 2023).
Bleeding and Thrombotic Complications
Authors: Shah Rahul , Savani Bipin N. , Chaturvedi Shruti 
Published: April 2024

AbstractBleeding and thrombotic complications are an important cause of morbidity and mortality in patients undergoing hematopoietic cell transplantation (HCT). The major thrombotic complications include venous thromboembolism (VTE) including catheter-related thrombosis (CRT), sinusoidal obstruction syndrome (SOS), and transplant-associated thrombotic microangiopathy (TA-TMA), while bleeding commonly involves the gastrointestinal or respiratory tracts and is most common in thrombocytopenic patients or those with graft-versus-host disease (GVHD). HCT is associated with multiple risk factors for both thrombosis and bleeding including the underlying malignancy, thrombocytopenia, high-dose myeloablative chemotherapy (MAC) and immune-modulatory drugs, GVHD, infections, indwelling vascular catheters, and prolonged immobilization (Chiu and Lazo-Langner 2023; Gerber et al. 2008; Chaturvedi et al. 2016; Nadir and Brenner 2007). In addition, HCT is also associated with alterations in the coagulation system with activation of endothelium-dependent coagulation factors, increase in von Willebrand factor (vWF) and platelet adhesion, increased thrombin generation, decreased antithrombin levels, and decreased levels of anticoagulant proteins such as protein C (Vannucchi et al. 1994). Collectively, major patient-, disease-, and therapy-related factors contribute to hemostatic complications in HCT patients. Thrombotic and bleeding complications in HCT are discussed separately in the following section.
Graft Failure
Authors: Valcárcel David , Sánchez-Ortega Isabel , Sureda Anna 
Published: April 2024

AbstractThe current definition for hematological recovery includes neutrophil recovery, defined as the first of three consecutive days with an absolute neutrophil count ≥0.5 × 109/L and platelet recovery which is defined as a platelet count of ≥20 × 109/L in the absence of platelet transfusion for 7 consecutive days.
Early Complications of Endothelial Origin
Authors: Carreras Enric , Diaz-Ricart M. , Jodele S. , Penack O. , Vasu S. 
Published: April 2024

AbstractDuring HCT, the vascular endothelium is affected by conditioning, IS agents, inflammatory molecules released by injured cells and tissues, endotoxins translocated across injured mucosal barriers, the complex process of engraftment, and in allo-HCT immune alloreactivity. This endothelial damage can affect the entire vascular endothelium or that of specific organs and be the triggering event for several of the early complications grouped under denomination vascular endothelial syndromes of HCT.
Acute Graft-Versus-Host Disease
Authors: Holler Ernst , Greinix Hildegard , Zeiser Robert 
Published: April 2024

AbstractAcute graft-versus-host disease (GvHD) remains a major course of short term (100 days and 1 yr) mortality and morbidity after allogeneic stem cell transplantation. The pathophysiology of GvHD is described as a 3 step process starting with initial tissue damage by conditioning followed by host antigen presenting cell activation by damage and pathogen associated molecular patterns and finally resulting in activation of alloreactive T cells and proinflmmatory cytokines inducing target cell apoptosis. This activating cycle elicits multiple regulatory mechanisms and cells such as regulatory T cells and myeloid derived suppressor cells. Besides the disturbed balance between immune activation and immune tolerance, a disturbed capacity of tissue repair contributes to clincal damage.
Chronic Graft-Versus-Host Disease
Authors: Wolff Daniel , Peric Zinaida , Lawitschka Anita 
Published: April 2024

AbstractChronic GVHD (cGVHD) is the most relevant cause of late non-relapse morbidity and subsequent mortality (approximately 25%) following allo-HCT (Grube et al. 2016). Its incidence is approximately 50% among all patients following allo-HCT and has increased during the last two decades due to increasing patient age and increasing use of unrelated and/or mismatched donors, RIC regimens, PBSC with application of standard GVHD prophylaxis (calcineurin inhibitor [CNI] + MMF or MTX) only (Arai et al. 2015). While the incidence of cGVHD is lower (6–40%) in children, its incidence rises to 60% as age increases (Baird et al. 2010; Sobkowiak-Sobierajska et al. 2022).
Haemorrhagic Cystitis and Renal Dysfunction
Authors: Cesaro Simone 
Published: April 2024

AbstractHaemorrhagic cystitis is a frequent complication after haematopoietic cell transplantation. Supportive measures (analgesics, antispasmodics, blood product transfusions and hyperhydration) are the main mode of treatment, with or without antivirals or experimental approaches (hyperbaric oxygen therapy, fibrin-glue or platelet-rich plasma application, adoptive immunotherapy).
Monitoring Measurable Residual Disease in ALL and AML
Authors: Bader Peter , Kreyenberg Hermann , Ossenkoppele Gert 
Published: April 2024

AbstractIn ALL evaluation of molecular treatment response, assessment of minimal residual disease, nowadays named measurable residual disease (MRD), is a substantial independent predictor of outcome, as proven by randomized studies (Conter et al. 2010; Gökbuget et al. 2012; Bassan and Spinelli 2015). Consequently, MRD is implemented in virtually all clinical protocols in order to supplement or to redefine multifactorial risk stratification with optional customized treatment intensity. The detection of leukemic cells below the limit of classical cytomorphology is feasible by either disease-specific alterations of the immune phenotype or unique genetic features. Several competing and complementing MRD methods have been developed with preference application according to clinical protocols (Van der Velden et al. 2007; van Dongen et al. 2015).
Delayed Transfer of Immune Cells or the Art of Donor Lymphocyte Infusion (DLI) 2.0
Authors: Falkenburg J. H. Frederik , Schmid Christoph , Kolb Hans Joachim , Kuball Jürgen 
Published: April 2024

AbstractIn the context of an allogeneic hematopoietic cell transplantation (HCT), the interplay between host and donor immune cells is considered to be the primary mechanism responsible for graft-versus-leukemia (GVL) reactivity and also able to mediate graft-versus-host disease (GVHD) (Schmid et al. 2021). The tissue specificity of the immune response determines the balance between GVL and GVHD, as well as tropism of GVHD. The main population for success and failure of HCT and DLIs originates from αβT cells. Other subsets are also key modulators of efficacy. For example, NK cells most likely provide acute control of leukemia and of infections like CMV. However, NK cells become rapidly educated over time (Orr and Lanier 2010) and lose their antileukemia activity. Thus, donor transfer of NK cells is obsolete and needs additional, for example, genetic modification to engineer long-term efficacy (Laskowski et al. 2022; Liu et al. 2020). Other subsets, like γδT cells, appear to have a more prolonged antileukemia effect (Handgretinger and Schilbach 2018; Sebestyen et al. 2020) and are also helpful in controlling CMV reactivation (Scheper et al. 2013; de Witte et al. 2018). However, also, donor γδT cells can lose activity over time, and sustainable activity requires, outside the context of an HCT, most likely further modifications (Sebestyen et al. 2020; Li et al. 2023). NKT cells, like regulatory T cells, have been mainly reported to influence GVHD. While an increase in NKT cells in the graft associates with a reduced GVHD incidence (Malard et al. 2016), depletion of regulatory T cells in donor lymphocyte infusions (DLI) improves GVL effects, although it augments the risk of GVHD (Maury et al. 2010). Thus, lymphocyte infusions as part of the graft at the time of transplantation, or delayed as DLI, have multiple effector cells that need to be considered in terms of different alloreactive effects (for review see also (Schmid et al. 2021)).
At-Home HCT
Authors: Fernández-Avilés Francesc 
Published: April 2024

AbstractThe main indications for autoHCT are lymphoid malignancies (90%) with plasma cell disorders (MM and others) comprising 55% of all autoHCT (Passweg et al. 2021). Toxicity and mortality associated with autoHCT have been reduced significantly with the use of mobilized peripheral blood HSC, the extended use of cryotherapy associated with MEL, and the improvements in prophylactic antibiotic and antiemetic regimens. Besides this, outpatient parenteral antimicrobial treatment has been proven feasible and safe, thanks to modern CVC and infusion devices. All these advances have led to the development of outpatient autoHCT programs, and several studies have demonstrated their feasibility and safety (González et al. 2021).
Haploidentical HCT
Authors: Bacigalupo Andrea , Lankester Arjan , Ciceri Fabio , Bertaina Alice 
Published: April 2024

AbstractWe will discuss two platforms of haploidentical HSCT(haplo-HSCT): ex vivo T cell depletion and unmanipulated in vivo T-cell depletion. The former has evolved from positive selection of CD34+ cells to selection of CD34+ cells associated with alpha/beta T cell and CD19 B cell depletion. We will outline the outcome of these procedures in children and adults. More recently selective add back of Treg Tcon has also been developed and will be discussed. The second platform is unmanipulated haplo-HSCT: PTCy and ATG have been used alone or in combination to optimize prevention of GvHD. We will discuss the outcome in patients with hematologic malignancies as well as in patients with non-malignant disorders, such as aplastic anemia, hemoglobinopathies, and immune deficiencies.
Acute Myeloid Leukaemia in Children
Authors: Gibson Brenda E. S. , Sauer Martin G. , Ramanathan Subramaniam , Amrolia Persis J. 
Published: April 2024

AbstractThe outcome for children with acute myeloid leukaemia (AML) continues to improve although not at the pace of acute lymphoblastic leukaemia (ALL). The current 3-year EFS and OS are 65–70% and 75–80%, respectively. The improved survival is attributed to better supportive care, refinement in risk stratification (particularly directing patients to allogeneic HCT in CR1), and improved salvage in relapsed/refractory AML (R/R AML). The enhanced understanding of the genomic landscape of paediatric AML has lead to the strategic use of novel treatments. The future of AML treatment lies in optimising the therapeutic potential of new treatments, including CAR T-cells, in combination with conventional chemotherapy and allogeneic HCT.
Myelodysplastic/Myeloproliferative Neoplasms
Authors: Onida Francesco , Chalandon Yves 
Published: April 2024

AbstractThe myelodysplastic syndrome-myeloproliferative neoplasms (MDS/MPNs) are a heterogeneous group of hematologic malignancies characterized by dysplastic and myeloproliferative clinical, laboratory, and morphological overlapping features, both in marrow and in blood. MDS/MPNs include chronic myelomonocytic leukemia (CMML), MDS/MPN with neutrophilia, MDS/MPN with SF3B1 mutation (in its absence with ringed sideroblasts) and thrombocytosis (MDS/MPN-SF3B1-T), and MDS/MPN not otherwise specified (MDS/MPN-NOS). Prognosis of MDS/MPN is highly variable, being dismal in the majority of patients with CMML, MDS/MPN with neutrophilia and MDS/MPN-NOS. In the absence of disease-modifying treatment options, allo-HCT represents the only curative option for eligible patients. With regard to allo-HCT indication in CMML patient, a number of prognostic systems have been developed over the years. As far as pre-transplant phase, in high-risk patients with MDS/MPN and low blast count (<10%), upfront transplantation is the most frequently preferred strategy. In patients with high blast count, pre-transplant treatment with HMAs, or combination of HMAs with other new agents in clinical trials may be considered. In case of rising leukocytosis and/or organ infiltration, hydroxyurea is the drug of choice. In MDS/MPN patients, the choice of conditioning regimen depends on many different conditions, the major ones being comorbidities, patient age, disease phase at transplant, type of donor, and HSC source. In general, myeloablative regimens may be advisable in young patients without comorbidities aiming to reduce the relapse risk, while reduced-intensity regimens are preferred for patients with older age or comorbidities. As disease recurrence represents the major cause of transplant failure in MDS/MPN, there is a growing interest toward possible post-transplant treatment strategies, both as preemptive and as prophylactic modalities.
Acquired Bone Marrow Failure: Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
Authors: de Latour Régis Peffault , Risitano Antonio , Kulasekararaj Austin , Dufour Carlo 
Published: April 2024

AbstractSevere aplastic anemia (SAA) is an autoimmune disorder due to the attack of autoreactive cytotoxic T lymphocytes to the hematopoietic component of the bone marrow. SAA might be associated with PNH, resulting from the clonal expansion of hematopoietic stem cells that have somatic mutations in the X-linked gene PIG-A. This review is summarizing the management of SAA from the diagnosis, where inherited disorders need to be excluded, to the overall treatment algorithm of this disease, including immunosuppression and bone marrow transplantation.
Hemoglobinopathies (Sickle Cell Disease and Thalassemia)
Authors: Cappelli Barbara , Gluckman Eliane , Corbacioglu Selim , de la Fuente Josu , Abboud Miguel R. 
Published: April 2024

AbstractHematopoietic cell transplantation (HCT) using an HLA-matched sibling donor is a well-established curative therapy for pediatric patients with sickle cell disease (SCD) and transfusion-dependent thalassemias (TDT). In order to expand the donor pool, new approaches such as related haploidentical donor HCT have been used with encouraging results. These approaches aim for a higher overall survival, an effective reduction of acute and chronic GvHD and a reduced toxicity. Due to these alternative approaches and adult patients being increasingly transplanted, the number of HCT has dramatically increased in the last decade. Furthermore, different gene therapy and gene editing strategies are being developed in clinical trials, showing promising results.
Multiple Myeloma
Authors: Mohty Mohamad , Bruno Benedetto , Gagelmann Nico , Mateos Maria-Victoria 
Published: April 2024

AbstractThe concept of high-dose therapy (HDT) followed by autologous hematopoietic cell transplantation (AHCT) remains the standard for treating newly diagnosed multiple myeloma in young and in select, fit, elderly patients. The introduction of ImiDs and proteasome inhibitors administered before and/or after HDT/AHCT gave way to the groundbreaking achievement of stringent complete response (sCR), immunophenotypic CR, and molecular CR, in addition to significantly increased CR and CR plus very good partial response rate (VGPR; Table 81.1). In randomized studies, age of participants is limited to 65 years to avoid selection bias and limit toxicities and withdrawal from studies. However, this does not mean that AAHCT is not feasible in older patients. A study whereby the median age of patients was 72 years old concluded that elderly multiple myeloma patients should not be excluded from transplantation displaying good results with melphalan 140 mg/m2. Currently, in many centers, fit patients up to age 70, and even 75 years old, receive AHCT.
Indolent Lymphoma
Authors: Serroukh Yasmina , Montoto Silvia 
Published: April 2024

AbstractIndolent lymphomas (iNHL) form an heterogenous group accounings for 1/3 of all malignant lymphomas with Follicular lymphoma (FL) being the most common subtype. iNHL are characterized by repeated relapses. Among available treatment lines, autologous (auto) and allogeneic (allo) HCT are the only curative options for relapsed disease. Nevertheless, the roles of both forms of HCT are evolving and are currently being challenged as T-cell engaging therapies emerge. The indications for auto-HCT and allo-HCT in 2023 are reviewed in this chapter.
Chronic Lymphocytic Leukemia
Authors: Schetelig Johannes , Dreger Peter 
Published: April 2024

AbstractCLL is a rare indication for HCT/Cellular Therapy since it usually follows an indolent course. Allogeneic HCT is considered as standard of care in eligible high-risk patients who have failed at least two classes of modern pathway inhibitor-based therapy, and in select patients with CLL transformed in to an aggressive B-cell lymphoma (Richter transformation). Except for Richter transformation, there is no role for autologous HCT in CLL. In the absence of a labeled indication, CAR T-cells should not be used outside of clinical trials.
Large B-Cell Lymphoma
Authors: Bento Leyre , Glass Bertram , Schmitz Norbert 
Published: April 2024

AbstractThe most recent WHO classification of tumors of hematopoietic and lymphoid tissues (Alaggio et al. 2022) classifies large B cell lymphomas (LBCL) into diffuse large B cell lymphoma (DLBCL) NOS with distinct morphological and molecular (germinal center B-cell, activated B-cell) subtypes, high-grade B-cell lymphomas (with MYC and BCL2 rearrangements or NOS subtype), and other less frequent subtypes.
Other B- and T-Aggressive Lymphomas and Lymphomas Associated with HIV
Authors: Hübel Kai , Montoto Silvia , Güven Mustafa , Duarte Rafael F. 
Published: April 2024

AbstractBL accounts for around 2% of all adult NHL with a higher incidence in patients with immunodeficiency and in patients who have HIV infection. There is an endemic pediatric subtype in equatorial Africa, which is strongly associated with EBV. The clinical course of BL usually is highly aggressive with a Ki67 expression of nearly 100% requiring prompt institution of therapy.
Inborn Errors of Immunity
Authors: Albert Michael H. , Lankester Arjan , Gennery Andrew , Neven Bénédicte 
Published: April 2024

AbstractInborn errors of immunity (IEI) comprise diseases arising from genetic defects that lead to abnormalities in immune cell development or function with a wide spectrum in severity and clinical manifestations. The number of transplants for IEI has increased significantly over the last years, elicited by better insight in the pathogenesis of the IEI (and thus the “curability” of these diseases with allo-HCT), better outcomes even with unrelated and haploidentical donors, and more differentiated approaches to HCT including reduced toxicity conditioning regimens.
Inborn Errors of Metabolism and Osteopetrosis
Authors: Wynn Robert , Schulz Ansgar 
Published: April 2024

AbstractInborn errors of metabolism (IEM) comprise a large group of inherited disease, some of which are disorders of lysosomal, peroxisomal, or mitochondrial function, and only some can be improved following HCT. The mechanism of action varies between the different metabolic disorders. In the lysosomal disorders, healthy donor cells deliver the enzyme (secretion) to residual enzyme-deficient host cells. This is a changing area of medicine, in which autologous stem cell gene therapy is changing BMT practice, and this is likely to accelerate in the immediate future.Osteopetrosis is a disorder of bone remodelling. The defect usually lies in the osteoclast, which is involved in bone metabolism, and is a specialized tissue macrophage. HCT restores competent tissue osteoclasts and therefore corrects the disease.