Hematopoetic cell transplant

CLINICAL ISSUE: Multiple myeloma (MM) is a malignancy of hematopoetic system and is associated with destruction of bone, suppressed bone marrow function and renal failure. It is characterized by strong proliferation of malignant plasma cells. STANDARD TREATMENT: Classic therapies contained an alkylating agent and a glucocorticoid. In the 1990s, treatments were supplemented with transplantation of peripheral blood stem cells. TREATMENT INNOVATIONS: During the 2000s, new therapies emerged, combining an immunomodulator (thalidomide, lenalidomide or pomalidomide), a proteasome inhibitor (bortezomib, carfilzomib or ixazomib), and a monoclonal antibody against CD38...

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Graft-versus-host disease is a common complication following allogeneic hematopoetic stem cell transplantation that can affect multiple organ systems, including the eyes. Ocular GVHD (oGVHD) is characterized by a T cell-mediated immune response that leads to immune cell infiltration and inflammation of ocular structures, including the lacrimal glands, eyelids, cornea and conjunctiva. oGVHD has a significant negative impact on visual function and quality of life and successful management requires a multi-disciplinary approach with frequent monitoring...

OBJECTIVE: To describe fertility characteristics, outcomes of oocyte cryopreservation cycles, and safety of ovarian stimulation in patients with GATA binding protein 2 (GATA2) deficiency, dedicator of cytokinesis 8 (DOCK8) deficiency, and sickle cell disease (SCD) preparing for hematopoetic stem cell transplantation (HSCT). DESIGN: Retrospective case series. SETTING: The National Institutes of Health. PATIENTS: Female patients with GATA2 deficiency, DOCK8 deficiency, and SCD aged between 13 and 38 years...

High-dose chemotherapy followed by autologous stem cell transplantation is a major component in the treatment of patients with multiple myeloma. As a prerequisite, the successful collection of a sufficient number of viable peripheral blood hematopoietic CD34+ cells is critical. A common standard protocol for mobilization is currently not defined and critically discussed especially in German-speaking Europe. In times of the Covid-19 pandemic, safe and effective strategies have to be chosen to minimize hospitalization times and severe courses...

Objective: Although inhibition of complement system at different steps is a promising therapy modality in PNH (paroxysmal paroxysmal nocturnal hemoglobinuria) patients, allogeneic hematopoetic stem cell transplantation (HCT) is still the only curative therapy especially for patients with intractable hemolysis or bone marrow failure. The aim of this study is to evaluate the outcomes of allogeneic HCT in PNH patients with or without aplastic anemia ( PNH-AA). Material and Methods: 35 PNH / PNH-AA patients who were treated with allegeneic HCT in ten transplantation centers in Turkey were retrospectively analyzed...

Background: Wiskott Aldrich syndrome (WAS) is characterized by bleeding manifestations, recurrent infections, eczema, autoimmunity, and malignancy. Over the last decade, improved awareness and better in-house diagnostic facilities at several centers in India has resulted in increased recognition of WAS. This study reports collated data across major primary immunodeficiency diseases (PID) centers in India that are involved in care of children with WAS and highlights the varied clinical presentations, genetic profile, and outcomes of patients in India...

RATIONALE: Steroid refractory graft-vs-host disease (sr-GvHD) represents a challenging complication after allogeneic hematopoietic cell transplantation (allo-HCT). Intestinal microbiota (IM) diversity and dysbiosis were identified as influencing factors for the development of acute GvHD. Fecal microbiota transfer (FMT) is hypothesized to restore IM dysbiosis, but there is limited knowledge about the significance of FMT in the treatment of sr-GvHD. OBJECTIVES: We studied the effects of FMT on sr-GvHD in allo-HCT patients from two German tertiary clinical centers (n = 11 patients; period: March 2017 until July 2019)...

OBJECTIVE: The selection of graft-vs. -host disease (GvHD) prophylaxis is vital for the success of hematopoetic stem cell transplantation (HSCT), and calcineurin inhibitors (CNI) have been used for decades as the backbone of GvHD prophylaxis. The aim of this study is to analyze the results of switching cyclosporine (CSA) to tacrolimus because of acute GvHD, engraftment syndrome (ES), persistent low level of CSA, or various CSA-associated adverse events in the first 100 days of pediatric HSCT...

Contemporary studies of systemic sclerosis (SSc) consistently demonstrate that interstitial lung (ILD) is a leading cause of disease-related death. This review summarizes morbidity and mortality outcomes in SSc-ILD patients from high-quality observational and interventional studies over the last 50 years. The data presented suggest a trend for improved morbidity and mortality outcomes among present day SSc-ILD patients. Specifically, SSc-ILD patients appear to be living longer from the time of the initial diagnosis...

Allogeneic hematopoetic stem cell transplantation yields improved long-term survival for patients with high-risk malignant and non-malignant hematologic disease. However, it is associated with high morbidity and mortality. A proportion of patients need intensive care due to infectious, immunological and/or toxic complications. The utility of intensive care unit (ICU) treatments as mechanical ventilation and renal replacement therapy for these patients is uncertain since mortality is high. We describe the most frequent complications and the treatment options concerning the ICU in recipients of allogeneic hematopoetic stem cells...

Infectious complications are the major cause of morbidity and mortality after solid organ and stem cell transplantation. To better understand host and environmental factors associated with an increased risk of infection as well as the effect of infections on function and survival of transplanted organs, we established the DZIF Transplant Cohort, a multicentre prospective cohort study within the organizational structure of the German Center for Infection Research. At time of transplantation, heart-, kidney-, lung-, liver-, pancreas- and hematopoetic stem cell- transplanted patients are enrolled into the study...

Acute myeloid leukemia (AML) is a kind of malignant hematological disease with high mortality. Patients 5-year survival rate is less than 25% and that of elderly patients is lower than 10%. Although the standardized chemotherapy or hematopoetic stem cell transplantation can significantly improve the therapeutic efficacy for AML, but disease recurrence is still a difficult problem in most patients. Chemotherapy combined with immunotherapy has been regarded as the most promising treatment for AML in recent years, but immunotherapy is prone to immune escape, which has become an important factor affecting the therapeutic efficacy...

BACKGROUND: Bone marrow stem cell clonal dysfunction by somatic mutation is suspected to affect post-infarction myocardial regeneration after coronary bypass surgery (CABG). METHODS: Transcriptome and variant expression analysis was studied in the phase 3 PERFECT trial post myocardial infarction CABG and CD133+ bone marrow derived hematopoetic stem cells showing difference in left ventricular ejection fraction (∆LVEF) myocardial regeneration Responders (n=14; ∆LVEF +16% day 180/0) and Non-responders (n=9; ∆LVEF -1...

BACKGROUND: Cytomegalovirus (CMV) infection is one of the most common opportunistic infections following organ transplantation, despite administration of CMV prophylaxis. CMV-specific T-cell immunity (TCI) has been associated with reduced rates of CMV infection. We describe for the first time clinical experience using the CMV T-Cell Immunity Panel (CMV-TCIP), a commercially available assay which measures CMV-specific CD4+ and CD8+ T-cell responses, to predict clinically significant CMV events...

Background: Hematopoetic stem cell transplantation is considered as a standard treatment for cancer patients to stay hopeful toward treatment outcome. However, these patients experience many complications which might affect different aspects of their life. The aim of this study was to investigate the lived experience of patients after hematopoetic stem cell transplantation and introduce supportive care strategies. Materials and Methods: In this study, Van Manen's Hermeneutic phenomenological approach was used...

Importance: Patients with immunosuppression have a higher incidence of cutaneous squamous cell carcinoma (cSCC) and often present with more aggressive, multifocal disease. Objectives: To determine the risks for mortality in patients with cSCC and immunosuppression compared with nonimmunosuppression and to compare the difference in mortality risk based on the cause of immunocompromise. Design, Setting, and Participants: This retrospective cohort study of patients with cSCC of the head and neck recruited participants from a tertiary cancer care center...

Objective: To evaluate the association between insulin-dose adjusted A1C (IDAA1c) and microvascular complications (MC) and hypoglycemia in a representative Brazilian population of Type 1 diabetes mellitus (T1DM) patients. Research Design and Methods: This was a cross-sectional study based on a previous study, "Microvascular Complications in Type 1 Diabetes: a comparative analysis of patients treated with autologous nonmyeloablative hematopoietic stem-cell transplantation (AHST) and conventional medical therapy (CT)"...

After autologous hematopoietic cell transplantation (HCT) in the first complete remission (CR1), patients with acute myeloid leukemia (AML) may relapse and undergo allogeneic HCT in the second complete remission (CR2). The aim of this study was to analyze the outcome of allogeneic HCT performed in CR2 comparing patients with prior consolidation by autologous HCT versus patients with chemotherapy consolidation. Included were 2619 adults with allogeneic HCT in CR2 from 2000 to 2017 with (n = 417) or without (n = 2202) prior autologous HCT...

The proportion of patients with acute myeloid leukemia (AML) cured is increased by administering high-dose cytarabine (HiDAC). It remains uncertain whether to administer HiDAC as induction or consolidation, and whether ≥1 cycle of HiDAC is required. Our retrospective study of 416 adult AML patients, excluding good risk cytogenetics, compared a single cycle of HiDAC-based therapy followed by 2 cycles of standard-dose cytarabine (SDAC) (HiDAC induction cohort) with SDAC-based chemotherapy followed by 2 cycles of HiDAC-based chemotherapy (HiDAC consolidation cohort)...