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Background/Objectives: Patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) often receive medical nutrition therapy (MNT) during intensive remission-induction treatment. Since little is known about changes in nutritional status, specifically body composition, in this patient population, these changes and their associations with physical and clinical outcomes were assessed. Subjects/Methods: In this multicenter prospective observational study, newly diagnosed AML/MDS patients who received intensive remission-induction chemotherapy, routine dietary counseling by a dietician and MNT immediately upon inadequate nutritional intake, were included. At treatment initiation and discharge, nutritional status, including Patient-Generated Subjective Global Assessment (PG-SGA)-scores and body composition, physical outcomes and fatigue were assessed. Associations of nutritional status/body composition with physical outcomes, fatigue, fever duration, number of complications, time to neutrophil engraftment and hospital length of stay (LOS) (collected from medical records) were examined using multiple regression analysis. Results: In >91% of the 126 AML/MDS patients included, nutritional intake was adequate, with 61% receiving MNT. Nevertheless, body weight decreased significantly (p < 0.001) and mainly consisted of a loss of muscle/fat-free mass (FFM) (p < 0.001), while fat mass (FM) remained unchanged (p-value range = 0.71–0.77). Body weight and waist circumference showed significant negative associations with fever duration and/or number of complications. Significant positive associations were found between mid-upper arm muscle circumference (MUAMC) and physical functioning and between PG-SGA-scores and fatigue. Body weight and MUAMC were also negatively associated with LOS. Conclusion: Despite MNT in AML/MDS patients undergoing intensive chemotherapy, muscle/FFM decreased while FM remained unchanged. Maintenance of nutritional status was associated with improved physical and clinical outcomes. [ABSTRACT FROM AUTHOR]

Introduction: Myelodysplastic syndromes (MDS) are characterized by bone marrow failure, peripheral blood cytopenias and a high risk of progression to acute myeloid leukemia (AML), which is associated with a poor prognosis and low survival rates. This study combined surveys with patient chart reviews to document real-world clinical practice and burden of MDS, including perspectives of physicians, patients and caregivers and underlying discrepancies. Methods: Physicians in major European countries and the US provided information on 1445 patients, stratified into lower- (LR) and higher-risk (HR) MDS. Patients had the opportunity to complete questionnaires describing the impact of MDS. Caregivers had the option to report on the burden of caring for a patient with MDS. Results: While supportive treatment was common, mainly with erythropoietins (52%), anti-AML agents were more frequently used in HR than in LR patients (70% vs 20%), while HR patients generally received more transfusions (48% vs 36%). Symptoms with the largest discordance between patient vs physician reporting were excessive bruising (30% vs 14%), GI side effects (19% vs 6%) and feeling tired or fatigued (68% vs 56%). A bigger impact of fatigue was reported on the European Organization for the Research and Treatment of Cancer Quality of Life questionnaire (EORTC QLQ-C30) for HR vs LR patients (43.2 vs 36.5 on a scale from 0 to 100). There was discordance between caregivers vs physicians on reporting of weekly caregiver hours (45.4 vs 29.2) with a Zarit Burden Interview score (ZBI, score 0–88) of 25.4. Conclusions: Patients reported a higher frequency than their physicians of top symptoms, with MDS-related disruptions in daily life for both patients and caregivers. There is a need for new therapeutic strategies, along with shared understanding and decision making among patients, caregivers and physicians, to optimize disease management and improve quality of life in people living with MDS. [ABSTRACT FROM AUTHOR]

Simple Summary: Patients with myeloid malignancies (blood cancers such as leukemia and myelodysplastic syndromes) that have returned or worsened despite chemotherapy or a stem cell transplant face limited treatment choices. Immunotherapy holds promise in deploying the body's own immune system to target and destroy cancer cells that are otherwise evading immune detection. This review article summarizes the most up to date information on immunotherapy options for patients with myeloid malignancies as well as discusses the challenges faced in this evolving field. Immunotherapy has revolutionized the treatment of myeloid oncologic diseases, particularly for patients resistant to chemotherapy or ineligible for allogeneic stem cell transplantation due to age or fitness constraints. As our understanding of the immunopathogenesis of myeloid malignancies expands, so too do the treatment options available to patients. Immunotherapy in myeloid malignancies, however, faces numerous challenges due to the dynamic nature of the disease, immune dysregulation, and the development of immune evasion mechanisms. This review outlines the progress made in the field of immunotherapy for myeloid malignancies, addresses its challenges, and provides insights into future directions in the field. [ABSTRACT FROM AUTHOR]

Simple Summary: Several recent studies have demonstrated encouraging results in treating patients with myelodysplastic syndromes/neoplasms (MDSs). This review focuses on the latest conceptual and therapeutic advances in the management of adults with MDS, addressing diagnostic principles, classification updates, and prognostic stratification systems, as well as improvements in clinical approaches that provide significant benefits through novel treatments, which may, in turn, represent a valuable basis for developing future clinical trials. Myelodysplastic syndromes/neoplasms (MDSs) encompass a range of hematopoietic malignancies, commonly affecting elderly individuals. Molecular alterations in the hematopoietic stem cell compartment drive disease pathogenesis. Recent advancements in genomic profiling have provided valuable insights into the biological underpinnings of MDSs and have expanded therapeutic options, particularly for specific molecularly defined subgroups. This review highlights the diagnostic principles, classification updates, prognostic stratification systems, and novel treatments, which could inform future clinical trials and enhance the management of adult MDS patients, particularly for specific molecularly defined subgroups. [ABSTRACT FROM AUTHOR]

Introduction: Chronic Myelomonocytic Leukemia (CMML) and Myelodysplastic Syndromes (MDS) are increasingly represented in the general population. We propose a screening strategy based on algorithms calculated from quantitative and analytical data from the XN analyser. Materials and Methods: We tested the performance of previously published MDS and CMML scores on an evaluation cohort of 749 individual eligible patients over 50 years of age. These patients were classified into 3 groups as follows: 713 patients without MDS or CMML, 18 patients with MDS, and finally 18 patients with CMML. In a second step, a routine cohort of 37 828 samples was studied to evaluate the impact of this approach. Results: The concordance rate between cytology and the two scores is 92.1%. The sensitivity and specificity of the CMML score are 100% and 96.2%, respectively. For the MDS score, they are 83.3% and 89.6% respectively. The ratio of platelets measured by fluorescence on board (PLT‐F) as reflex tests generated is 1.5% after 6 months. The additional smear ratio for suspected MDS is calculated at 0.6%. Conclusion: We propose a flowchart using embedded artificial intelligence to help the cytologist in an optimized smear review and thus improve guidance to the clinician and the patients in the diagnosis process. This strategy permits a more comprehensive approach to MDS and CMML detection fitting with the new definition of CMML according to the recommendations of the World Health Organization (WHO) published in 2022. [ABSTRACT FROM AUTHOR]

Simple Summary: Allogeneic stem cell transplantation (allo-SCT) represents the only potentially curative treatment for high-risk acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), but up to 50% of patients relapse after allo-SCT. The salvage therapy after disease recurrence is not standardized, and the outcome remains unfavorable. Therefore, there is a growing interest in determining the most effective approach to manage the post-transplant phase with the goal of promptly detecting disease recurrence or preventing it. In this context, we conducted a retrospective study to assess the overall survival (OS) of patients with relapsed AML or MDS after allo-SCT with the aim of acquiring useful information for identifying the best prospective therapeutic strategy. The OS was evaluated according to the type of therapy, whether it included donor lymphocyte infusion (DLI), the timing of administration, and whether it occurred during an overt hematological relapse or in a preemptive setting. Background: Disease relapse after allogeneic stem cell transplantation (allo-SCT) is the main challenge for curing acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). We investigated the overall survival (OS) after allo-SCT relapse according to different therapeutic approaches. Methods: We analyzed 134 patients who relapsed after allo-SCT performed between 2015 and 2021 at Saint-Antoine University Hospital, Paris and Spedali Civili di Brescia, Brescia. Of these, 103 (77%) were treated, comprising 69/103 (67%) who received therapy in overt relapse and 34/103 (33%) who were treated in a pre-emptive manner when molecular/cytogenetics recurrence or mixed chimerism occurred. The treatment was donor lymphocyte infusion (DLI)-based for 40/103 (39%) patients. Results: The 1-, 2-, and 5-year OS of patients treated with DLI (n = 40) was 67%, 34%, and 34%, respectively, for those treated preventively (n = 20) and 43%, 20%, and 20%, respectively, for those treated in overt relapse (n = 20) (p < 0.01). The 1-, 2-, and 5-year OS of patients treated without DLI (n = 63) was 54%, 40%, and 26%, respectively, for those treated preventively (n = 14) and 17%, 5%, and 0%, respectively, for those treated in overt relapse (n = 49) (p < 0.01). Conclusions: Relapse treatment with a pre-emptive strategy was associated with improved outcomes, particularly when DLI was employed. [ABSTRACT FROM AUTHOR]

PURPOSE: BMT CTN 1102 was a phase III trial comparing reduced-intensity allogeneic hematopoietic cell transplantation (RIC alloHCT) to standard of care for persons with intermediate- or high-risk myelodysplastic syndrome (MDS). We report results of a cost-effectiveness analysis conducted alongside the clinical trial. METHODS: Three hundred eighty-four patients received HCT (n = 260) or standard of care (n = 124) according to availability of a human leukocyte antigen–matched donor. Cost-effectiveness was calculated from US commercial and Medicare perspectives over a 20-year time horizon. Health care utilization and costs were estimated using propensity score–matched cohorts of HCT recipients in the OptumLabs Data Warehouse (age 50-64 years) and Medicare (age 65 years and older). EuroQol 5 Dimension (EQ-5D) surveys of trial participants were used to derive health state utilities. RESULTS: Extrapolated 20-year overall survival for those age 50-64 years was 29% for HCT (n = 105) versus 13% for usual care (n = 44) and 31% for HCT (n = 155) versus 12% for non-HCT (n = 80) for those age 65 years and older. HCT was more effective (+2.36 quality-adjusted life-years [QALYs] for age 50-64 years and +2.92 QALYs for age 65 years and older) and more costly (+$452,242 in US dollars (USD) for age 50-64 years and +$233,214 USD for age 65 years and older) than usual care, with incremental cost-effectiveness ratios of $191,487 (USD)/QALY and $79,834 (USD)/QALY, respectively. For persons age 50-64 years, there was a 29% chance that HCT was cost-effective using a willingness-to-pay (WTP) threshold of $150K (USD)/QALY and 51% at a $200K (USD)/QALY. For persons age 65 years and older, the probability was 100% at a WTP >$150K (USD)/QALY. CONCLUSION: Among patients age 65 years and older with high-risk MDS, RIC HCT is a high-value strategy. For those age 50-64 years, HCT is a lower-value strategy but has similar cost-effectiveness to other therapies commonly used in oncology. For patients with high-risk myelodysplastic syndrome, bone marrow transplant is a cost-effective therapy option. [ABSTRACT FROM AUTHOR]

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Simple Summary: In our systematic review, we analyzed and summarized observational studies revealing a potential association between myelodysplastic syndromes (MDS) and hepcidin. The extensive studies available in this area enabled us to draw the conclusion that hepcidin has a potential importance in the pathophysiology of MDS and the prediction of poor MDS patient outcomes. A summary of the mechanisms leading to iron overload in MDS and the potential causes of elevated serum hepcidin levels are shown in a graphical abstract created with BioRender. It is important to note that this systematic review is based on a relatively small number of MDS patients and control participants. Hence, additional research is crucial for the further exploration of these findings. Iron overload emerges as a serious complication in myelodysplastic syndromes (MDS), particularly associated with frequent transfusions during the course of the disease. The discovery and description of hepcidin's mechanisms of action have contributed to a deeper understanding of iron metabolism. The existing literature reports a potential role of hepcidin in MDS, yet these data are fragmented and presented in an unstructured, somewhat chaotic manner. Hence, to address the existing data, we performed a systematic review of observational studies examining hepcidin levels in MDS. An extensive review of three bibliographic databases (Pubmed, Web of Science, and Scopus) enabled us to identify 12 observational studies. These studies focused primarily on adult patients with low-risk MDS who underwent transfusions and chelation therapy. An in-depth analysis of these manuscripts led to four main conclusions: (1) although high serum hepcidin levels are associated with MDS, most studies generally have not found a significant difference in these levels between patients and healthy individuals; (2) serum hepcidin levels are specific to MDS type; (3) serum hepcidin levels in MDS are strongly associated with transfusions and the genetic status of patients; and (4) high-risk MDS is associated with high serum hepcidin levels. While we have furnished a comprehensive summary of the significance of hepcidin in MDS, there are still gaps that future research should address. This pertains primarily to the capacity of hepcidin in predicting adverse outcomes for MDS patients and evaluating the efficacy of chelation therapy or the need for transfusion. [ABSTRACT FROM AUTHOR]

Simple Summary: Complex karyotype (CK), defined as ≥3 unrelated chromosomal abnormalities, is associated with a poor prognosis in both acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). Despite their high genetic complexity, complex chromosomal abnormalities in AML and MDS may share dysregulated gene expression signatures, leading to poorer outcomes compared to those of standard chemotherapy. We aimed to investigate a subset of genes and their signatures to predict CK in AML and MDS with excess blast (MDS-EB) using gene expression data. The CK signature (CKS) was established and validated, and its prognostic impact on overall survival (OS) was evaluated in comparison with previously reported risk stratification models using gene expression. A 10-gene CKS demonstrated high predictive accuracy for CK and was associated with shorter OS with comparable performance to previously established risk stratification models. Complex karyotype (CK) is associated with a poor prognosis in both acute myeloid leukemia (AML) and myelodysplastic syndrome with excess blasts (MDS-EB). Transcriptomic analyses have improved our understanding of the disease and risk stratification of myeloid neoplasms; however, CK-specific gene expression signatures have been rarely investigated. In this study, we developed and validated a CK-specific gene expression signature. Differential gene expression analysis between the CK and non-CK groups using data from 348 patients with AML and MDS-EB from four cohorts revealed enrichment of the downregulated genes localized on chromosome 5q or 7q, suggesting that haploinsufficiency due to the deletion of these chromosomes possibly underlies CK pathogenesis. We built a robust transcriptional model for CK prediction using LASSO regression for gene subset selection and validated it using the leave-one-out cross-validation method for fitting the logistic regression model. We established a 10-gene CK signature (CKS) predictive of CK with high predictive accuracy (accuracy 94.22%; AUC 0.977). CKS was significantly associated with shorter overall survival in three independent cohorts, and was comparable to that of previously established risk stratification models for AML. Furthermore, we explored of therapeutic targets among the genes comprising CKS and identified the dysregulated expression of superoxide dismutase 1 (SOD1) gene, which is potentially amenable to SOD1 inhibitors. [ABSTRACT FROM AUTHOR]

Simple Summary: Current research is exploring the evolving landscape of Myelodysplastic Syndrome (MDS) treatment, a challenging condition often progressing to acute myeloid leukemia. For low-risk MDS, the focus is on personalized care through precise risk assessment and tailored interventions, utilizing means like erythropoiesis-stimulating agents, lenalidomide, and luspatercept. High-risk MDS treatments are shifting towards upfront doublet or triplet therapies and minimal residual disease (MRD) monitoring. A holistic approach integrates treatments like stem cell transplants and post-transplant maintenance, guided by individual patient circumstances. Precision medicine, driven by Next Generation Sequencing (NGS), aids in early diagnosis, prognosis, and customized interventions, allowing for investigations through clinical trials within more homogeneous patient cohorts characterized by similar molecular profiles. Based on these premises, the future of MDS treatment irrefutably moves towards personalized care, leveraging advanced technologies and molecular insights to enhance patient outcomes in the realm of hematological malignancies. This perspective delves into the evolving landscape of Myelodysplastic Syndrome (MDS) treatment. MDS presents a significant clinical challenge, often progressing to acute myeloid leukemia. For low-risk MDS, the emphasis is on personalized care through comprehensive risk assessment, clinical monitoring, and tailored interventions, including promising agents like erythropoiesis-stimulating agents, lenalidomide, and luspatercept, with the anticipation of an expanding therapeutic arsenal and early intervention for improved outcomes. In contrast, high-risk MDS treatment is evolving towards upfront doublet or triplet therapies with a focus on minimal residual disease (MRD) monitoring. A holistic approach integrates various modalities, including stem cell transplant and post-transplant maintenance, all guided by individual patient circumstances. Risk-adapted strategies are crucial for enhancing patient outcomes. Precision medicine for MDS treatment is budding, largely driven by Next Generation Sequencing (NGS). NGS aids in early diagnosis, prognostication, and the targeting of specific mutations, with molecular data increasingly informing treatment responses and allowing for tailored interventions. Clinical trials within homogeneous patient groups with similar molecular profiles are becoming more common, enhancing treatment precision. In conclusion, the future of MDS treatment is moving towards personalized medicine, leveraging advanced technologies like NGS and molecular insights to improve outcomes in the realm of hematological malignancies. [ABSTRACT FROM AUTHOR]

Simple Summary: Myelodysplastic neoplasms (MDS) are a group of hematologic malignancies with an increased risk of transformation to acute myeloid leukemia. Non-coding RNAs are RNA molecules of variable size that do not translate into proteins but regulate gene expression during multiple cellular processes. These RNAs have been found deregulated in several cancers, including MDS. In this review, we aim to summarize research findings on the biological role of different non-coding RNAs in MDS development and progression, with emphasis on molecules that have exhibited prognostic or predictive value and could, hence, guide decision-making in clinical practice. Myelodysplastic syndromes or neoplasms (MDS) are a heterogeneous group of myeloid clonal disorders characterized by peripheral blood cytopenias, blood and marrow cell dysplasia, and increased risk of evolution to acute myeloid leukemia (AML). Non-coding RNAs, especially microRNAs and long non-coding RNAs, serve as regulators of normal and malignant hematopoiesis and have been implicated in carcinogenesis. This review presents a comprehensive summary of the biology and role of non-coding RNAs, including the less studied circRNA, siRNA, piRNA, and snoRNA as potential prognostic and/or predictive biomarkers or therapeutic targets in MDS. [ABSTRACT FROM AUTHOR]

Background Older patients with myelodysplastic syndromes (MDS), particularly those with no or one cytopenia and no transfusion dependence, typically have an indolent course. Approximately, half of these receive the recommended diagnostic evaluation (DE) for MDS. We explored factors determining DE in these patients and its impact on subsequent treatment and outcomes. Patients and Methods We used 2011-2014 Medicare data to identify patients ≥66 years of age diagnosed with MDS. We used Classification and Regression Tree (CART) analysis to identify combinations of factors associated with DE and its impact on subsequent treatment. Variables examined included demographics, comorbidities, nursing home status, and investigative procedures performed. We conducted a logistic regression analysis to identify correlates associated with receipt of DE and treatment. Results Of 16 851 patients with MDS, 51% underwent DE. patients with MDS with no cytopenia (n = 3908) had the lowest uptake of DE (34.7%). Compared to patients with no cytopenia, those with any cytopenia had nearly 3 times higher odds of receiving DE [adjusted odds ratio (AOR), 2.81: 95% CI, 2.60-3.04] and the odds were higher for men than for women [AOR, 1.39: 95%CI, 1.30-1.48] and for Non-Hispanic Whites [vs. everyone else (AOR, 1.17: 95% CI, 1.06-1.29)]. The CART showed DE as the principal discriminating node, followed by the presence of any cytopenia for receiving MDS treatment. The lowest percentage of treatment was observed in patients without DE, at 14.6%. Conclusion In this select older patients with MDS, we identified disparities in accurate diagnosis by demographic and clinical factors. Receipt of DE influenced subsequent treatment but not survival. [ABSTRACT FROM AUTHOR]

Context • Myelodysplastic syndrome (MDS) is a group of highly heterogeneous, malignant clonal diseases derived from hematopoietic stem cells. PD-1 monoclonal antibodies can have a synergistic effect with hypomethylating agents (HMAs), especially for patients with drug resistance to demethylation drugs. TCM in the treatment of MDS can improve hematological indexes, and for some patients, control the proliferation of primitive cells and delay or even block the transformation to leukemia. Objective • The study intended to examine the therapeutic effects of programmed cell death-1 (PD-1) inhibitors and azacitidine combined with the Yisuifang Thick Decoction in the treatment of MDS with older, high-risk patients. Design • The research team performed five prospective case studies. Setting • The study took place at the East Hospital affiliated with Beijing University of Chinese Medicine in Beijing, China. Participants • Participants were five older, high-risk MDS patients at the hospital who received PD-1 and azacitidine combined with Yisuifang Thick Decoction between April 2020 and June 2021. Outcome Measures • The research team measured: (1) treatment duration, (2) curative effects, (3) myelosuppression, (4) immune-related adverse reactions, (5) ending outcomes, and (6) progression-free survival (PFS). Results • The male to female ratio for the five participants was 3:2, and the median age was 69 years, with a range from 62 to 79 years. Four participants had refractory HR-MDS and one had primary MDS. The median treatment duration was 3 months, with a range from 2 to 4 months, and the median progression-free survival (PFS) was 5 months, with a range from 3 to 14 months. All participants achieved a partial response (PR) or a complete remission with incomplete count recovery (CRi) and showed improvement in serological indexes. Conclusions • Older, high-risk MDS patients generally have poor physical conditions, often accompanied by a poor karyotype prognosis and a poor prognosis for survival. Therefore, the combination of PD-1, azacytidine, and Yisuifang Thick Decoction may be an effective way to treat HR-MDS. [ABSTRACT FROM AUTHOR]

Simple Summary: Myelodysplastic/myeloproliferative (MDS/MPN) neoplasms are blood disorders characterized by abnormal cell growth and development. These disorders encompass various subtypes, including chronic myelomonocytic leukemia, atypical chronic myeloid leukemia, and others. They are caused by genetic changes in different cell components and have distinct clinical features. Current treatment options mostly involve drugs that control the disease, including hypomethylating agents, ruxolitinib, lenalidomide, and venetoclax, but do not offer a cure. However, allogeneic bone marrow transplantation has the potential to cure these disorders. Several factors, such as overall health, spleen enlargement, and genetic alterations, can influence the outcome of transplantation. Future research is crucial to improving treatment approaches and patient outcomes for MDS/MPN neoplasms. This review provides an overview of the diagnosis, biology, and current and upcoming treatments, including bone marrow transplantation, for these complex blood disorders. This review will shed light on the complexities of MDS/MPN neoplasms and will inform future research for improved therapeutic strategies and patient care in the future. Myelodysplastic/myeloproliferative neoplasms (MDS/MPN) are hematological disorders characterized by both proliferative and dysplastic features. According to the 2022 International Consensus Classification (ICC), MDS/MPN consists of clonal monocytosis of undetermined significance (CMUS), chronic myelomonocytic leukemia (CMML), atypical chronic myeloid leukemia (aCML), MDS/MPN with SF3B1 mutation (MDS/MPN-T-SF3B1), MDS/MPN with ring sideroblasts and thrombocytosis not otherwise specified (MDS/MPN-RS-T-NOS), and MDS/MPN-NOS. These disorders exhibit a diverse range of genetic alterations involving various transcription factors (e.g., RUNX1), signaling molecules (e.g., NRAS, JAK2), splicing factors (e.g., SF3B, SRSF2), and epigenetic regulators (e.g., TET2, ASXL1, DNMT3A), as well as specific cytogenetic abnormalities (e.g., 8 trisomies, 7 deletions/monosomies). Clinical studies exploring therapeutic options for higher-risk MDS/MPN overlap syndromes mostly involve hypomethylating agents, but other treatments such as lenalidomide and targeted agents such as JAK inhibitors and inhibitors targeting PARP, histone deacetylases, and the Ras pathway are under investigation. While these treatment modalities can provide partial disease control, allogeneic bone marrow transplantation (allo-BMT) is the only potentially curative option for patients. Important prognostic factors correlating with outcomes after allo-BMT include comorbidities, splenomegaly, karyotype alterations, and the bone marrow blasts percentage at the time of transplantation. Future research is imperative to optimizing therapeutic strategies and enhancing patient outcomes in MDS/MPN neoplasms. In this review, we summarize MDS/MPN diagnostic criteria, biology, and current and future treatment options, including bone marrow transplantation. [ABSTRACT FROM AUTHOR]

Simple Summary: Myelodysplastic syndromes are a heterogenous group of bone-marrow cancers that prelude a possible evolution to acute myeloid leukemia. Most treatments aim at reinstalling bone marrow function and delay evolution to acute leukemia. The disease has a negative impact on health-related quality of life and amelioration is an independent treatment goal. In order to capture the impact of current treatments outside clinical trials we aimed to monitor quality of life during the course of treatment. Another key element in our study was to prospectively assess disease perception and the impact on quality of life. Not reaching our inclusion goal, we cannot make any conclusions or assumptions on our first objective. We can, however, demonstrate a clear significant relation between disease perception and health related quality of life in this patient population that may lead to a positive impact for future patients. Patients with myelodysplastic syndromes suffer from an impaired quality of life that is only partially explained by physical symptoms. In an observational study, we aimed to investigate the impact of current MDS treatments and the influence of disease perception on quality of life. Serial measurement of health-related quality of life was performed by 'the QUALMS', a validated MDS-specific patient reported outcome tool. Disease perception was evaluated by means of the Brief Illness Perception Questionnaire (B-IPQ). We prospectively collected data on 75 patients that started on a new treatment and could not demonstrate a significant change in QUALMS score or B-IPQ score during treatment. Six out of eight items evaluated in the B-IPQ correlated significantly with QUALMS score. In this small sample, no significant difference in QUALMS score was found between lower vs. higher risk MDS patients or other studied variables, e.g., targeted hemoglobin showed no correlation with QUALMS score. In daily practice attention must be paid to initial formation of disease perception as it correlates independently with health-related quality of life and does not change during treatment (clinicaltrials.gov identifier: NCT04053933). [ABSTRACT FROM AUTHOR]

Myelodysplastic syndromes (MDS) can present as a cytopenia—often as unexplained anemia. Because MDS can progress to acute myelogenous leukemia, primary care providers should be aware of the signs and symptoms, which are associated with the corresponding cytopenia that patients experience and may include fatigue, infection, easy bruising, and bleeding. Treatment options center on managing related cytopenias. The only cure, stem cell transplant, is not readily available. [ABSTRACT FROM AUTHOR]

The article offers information on myelodysplastic syndrome which are gradually being recognized as disorders related to immune disorders. Topics include examines based on this view of MDS as an inflammatory-based disorders, researchers have tested immunotherapy as a potential therapy for MDS with some success.

Simple Summary: Relapse as the most common reason for treatment failure after allogeneic hematopoietic stem-cell transplantation (allo-SCT) in myeloid neoplasms usually occurs during the first year post-transplant, but several patients experience late relapse. This retrospective study aimed to characterize early and late relapses regarding clinical and cytogenetic parameters. Analyzing 91 consecutive patients, we demonstrated an improved overall survival for late compared with early relapsed patients and carved out cytogenetics and disease risk stratification at diagnosis as well as pretransplant strategy as major determinants for the timepoint of relapse. The impact of cytogenetics was highlighted by comparative karyotype analyses demonstrating a higher frequency of clonal evolution in early relapses. Improving knowledge about factors predicting early relapse may enable a selection of patients who may benefit from strategies to prevent or delay relapse, and identifying patients being at risk for late relapse may trigger prolonged surveillance strategies, including bone marrow biopsies and measurable residual disease (MRD) assessment. An improved understanding of relapse kinetics is required to optimize detection and treatment strategies for the post-transplant relapse of myeloid neoplasms. Therefore, we retrospectively analyzed data from 91 patients allografted for MDS (n = 54), AML-MRC (n = 29) and chronic myelomonocytic leukemia (CMML, n = 8), who relapsed after transplant. Patients with early (<12 months, n = 56) and late relapse (>12 months, n = 35) were compared regarding patient-, disease- and transplant-related factors, including karyotype analyses at diagnosis and relapse. After a median follow-up of 17.4 months after relapse, late relapses showed improved outcomes compared with early relapses (2-yr OS 67% vs. 32%, p = 0.0048). Comparing frequency of distinct patient-, disease- and transplant-related factors among early and late relapses, complex karyotype (p = 0.0004) and unfavorable disease risk at diagnosis (p = 0.0008) as well as clonal evolution at relapse (p = 0.03) were more common in early than in late relapses. Furthermore, patients receiving transplant without prior cytoreduction or in complete remission were more frequently present in the group of late relapses. These data suggest that cytogenetics rather than disease burden at diagnosis and transplant-related factors determine the timepoint of post-transplant relapse and that upfront transplantation may be favored in order to delay relapse. [ABSTRACT FROM AUTHOR]

Our case focuses on a 60-year-old male patient, a farmer, with a previous diagnosis of myelodysplastic syndrome, under regular surveillance at the haemato-oncology department. The patient was referred to the emergency room with an inflammatory ulcerated plaque in the dorsum of his left hand, with 2 weeks evolution. There were two moderately swollen, very painful ulcerated plaques on the dorsal aspect of the left hand, with grayish borders and irregular margins. A skin biopsy showed panniculitis and a dense neutrophilic dermal infiltrate. Attending to his underlying disease, clinical hallmarks of the lesion, and skin biopsy, the diagnosis of a neutrophilic dermatosis of the dorsal hands was made. The patient was treated with oral prednisolone 0.5 mg/kg/day and colchicine 1mg/day with complete regression after 1 month. Neutrophilic dermatosis is a heterogeneous group of skin diseases characterized by dense infiltration of neutrophils in the affected tissue. Neutrophilic dermatosis of dorsal hands is one of them and is considered a localized variant of Sweet syndrome. The etiology is unknown, but about half of the cases are associated with hematological diseases. [ABSTRACT FROM AUTHOR]

Background/purpose: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the curative therapy for acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS), but advanced age with multiple comorbidities limits the eligibility for allo-HSCT. We conducted a retrospective study to investigate the comorbidities assessments and prognostic factors that predict outcomes for these patients.Methods: Clinical data of patients older than 50 years who had received diagnoses of AML or MDS and underwent allo-HSCT were obtained. Information on patient characteristics, including age, gender, allogeneic transplant type, conditioning regimens, Charlson comorbidity index (CCI), and presence of acute graft-versus-host disease (GVHD) or chronic GVHD, were collected and analyzed.Results: Two hundred fifty-five elderly patients with a median age at allo-HSCT of 57 years were included. The significant prognostic factors associated with worse overall survival (OS) were CCI ≥3 (hazard ratio: 1.88) and grade III-IV acute GVHD (3.18). Similar findings were noted in the non-relapse mortality analysis. To investigate the effects of chronic GVHD on patient outcomes, OS analysis was performed for those with survival >100 days after transplantation. The results revealed CCI ≥3 (1.88) and grade III-IV acute GVHD (2.73) remained poor prognostic factors for OS, whereas mild chronic GVHD (0.43) was associated with better OS.Conclusion: This cohort study suggests that CCI ≥3 predicts poor outcomes, primarily due to a higher NRM risk. Careful management of GVHD after transplantation could improve outcomes in elderly patients with AML or MDS after allo-HSCT. [ABSTRACT FROM AUTHOR]

There is increasing recognition of the role of inheritance in myeloid malignancies. Differentiating germline from somatic variants in a hematologic malignancy is challenging but important. Oncology nurses need to be knowledgeable about the germline risk associated with myeloid malignancies; the inherited risk is well established and has implications for affected individuals as well as family members who may be at risk for malignancy themselves or who are being evaluated to serve as a related donor for allogeneic hematopoietic stem cell transplantation. [ABSTRACT FROM AUTHOR]

No two diagnoses of myelodysplastic syndrome are genuinely alike, owing to differing and dynamic mutational topography and epigenetic aberrancy. Consequently, no two patients with myelodysplastic syndrome are identical and disease-specific and patient-specific factors are considered in formulating the optimal treatment, which includes few that are disease modifying. Age itself should not be an absolute contraindication to therapy, including intensive therapy such as allogeneic hematopoietic stem cell transplantation, which is the only curative therapy. However, age associates with an increased prevalence of frailty and comorbidities that must be considered and may preclude a path to cure. Palliative therapies are the mainstay for many patients with myelodysplastic syndrome, which is a disease of older adults with the majority of patients diagnosed at age ≥ 75 years. The older patient requires heightened attention to end organ function/reserve and drug–drug interactions as well as insurance, income, cost, and socioeconomic and psychosocial issues that influence management. Many prior studies have included relatively younger populations or have not specifically performed high-quality subgroup analyses of older patients. In this review, we discuss the available standard-of-care therapies for myelodysplastic syndrome as they specifically relate to the older population and assess the emerging therapeutics that may further the pursuit for personalized treatment and improve both the outcomes and quality of life of the older patient with myelodysplastic syndrome. [ABSTRACT FROM AUTHOR]

According to the cancer stem cell (CSC) hypothesis, CSCs are the only cancer cells that can give rise to and sustain all cells that constitute a cancer as they possess inherent or acquired self-renewal potential, and their elimination is required and potentially sufficient to achieve a cure. Whilst establishing CSC identity remains challenging in most cancers, studies of low-intermediate risk myelodysplastic syndromes (MDS), other chronic myeloid malignancies and clonal haematopoiesis of indeterminant potential (CHIP) strongly support that the primary target cell usually resides in the rare haematopoietic stem cell (HSC) compartment. This probably reflects the unique self-renewal potential of HSCs in normal human haematopoiesis, combined with the somatic initiating genomic driver lesion not conferring extensive self-renewal potential to downstream progenitor cells. Mutational 'fate mapping' further supports that HSCs are the only disease-propagating cells in low-intermediate risk MDS, but that MDS-propagating potential might be extended to progenitors upon disease progression. The clinical importance of MDS stem cells has been highlighted through the demonstration of selective persistence of MDS stem cells in patients at complete remission in response to therapy. This implies that MDS stem cells might possess unique resistance mechanisms responsible for relapses following otherwise efficient treatments. Specific surveillance of MDS stem cells should be considered to assess the efficiency of therapies and as an early indicator of emerging relapses in patients in clinical remission. Moreover, further molecular characterization of purified MDS stem cells should facilitate identification and validation of improved and more stem cell-specific therapies for MDS. [ABSTRACT FROM AUTHOR]

• This ESMO Clinical Practice Guideline provides key recommendations for managing myelodysplastic syndromes. • It covers diagnosis, classification, staging and risk assessment of myelodysplastic syndromes. • Treatment recommendations for lower- and higher-risk myelodysplastic syndromes are also provided. • All recommendations were compiled by a multidisciplinary group of experts. • Recommendations are based on available scientific data and the authors' expert opinions. [ABSTRACT FROM AUTHOR]

What is known and objective: Many refractory/relapsed haematological malignancies, in non‐remission state, still have poor prognosis even after allogeneic haematopoietic stem cell transplantation. Recently, decitabine or umbilical cord blood transplantation (UCBT) seemed to be effective in these patients. However, few studies have added decitabine to myeloablative conditioning regimens for UCBT in patients with haematological malignancies not in remission. Therefore, the objective was to evaluate the clinical outcomes of patients with refractory/relapsed acute myeloid leukaemia (AML) or myelodysplastic syndrome (MDS) using decitabine as part of a myeloablative conditioning regimen prior to salvaged unrelated UCBT at our centre. Methods: We enrolled 20 consecutive patients with refractory/relapsed AML/MDS between 2013 and 2018. All patients were in non‐remission state before transplantation. All transplants were performed with decitabine as part of the myeloablative conditioning regimen, which was decitabine + fludarabine/busulfan/cyclophosphamide. Results and discussion: All patients achieved neutrophil and platelet engraftment. Incidence of grade III/IV acute graft‐vs‐host disease (GVHD) was 20.0%, which was also decreased compared to non‐decitabine group (P =.025). The median follow‐up time after UCBT was 29 months (range 14‐64 months). The 2‐year probability of GVHD‐free relapse‐free survival (GRFS) was higher in the decitabine group. Univariate showed that the decitabine group was associated with a higher GRFS than the non‐decitabine group. The estimated probability of overall survival and relapse was 55% and 20.0%, respectively. What is new and conclusions: Our results suggest that addition of decitabine as part of the myeloablative conditioning regimen prior to UCBT for refractory/relapsed AML/MDS in patients who are not in remission is safe and might be an effective treatment option. [ABSTRACT FROM AUTHOR]

The prognosis of patients with hypoplastic myelodysplastic syndrome (hMDS) after receiving allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains unclear. This study aimed to evaluate the outcomes of patients with hMDS after allo-HSCT. Between September 2013 and October 2019, a total of 20 consecutive patients with hMDS and 1 patient with clonal cytopenia of undermined significance (CCUS) who underwent allo-HSCT, which included procedures with 9 matched sibling donors, 2 matched unrelated donors, 4 mismatched unrelated donors and 6 haploidentical donors, were enrolled in this study. The median time for myeloid engraftment was 11 days (range 9-17 days), and that for platelet engraftment was 10 days (range 7-17 days). The cumulative incidence (CI) of myeloid and platelet recovery was 95.2 ± 6.0% and 90.5 ± 7.3%, respectively. The CI rates were 40.0 ± 11.3% for grades II-III acute graft-versus-host disease (GVHD), 36.8 ± 11.5% for chronic GVHD and 23.8 ± 9.6% for nonrelapse mortality. No patients experienced relapse. Sixteen surviving patients were followed up for a median of 1113 days (range 110-2305 days), and the overall survival and relapse-free survival rates were both 72.7 ± 10.6%. This limited retrospective analysis suggests that patients with hMDS had a favorable survival after allo-HSCT. [ABSTRACT FROM AUTHOR]

Clinical trial participation among patients with myelodysplastic syndromes is low even for those treated at large academic centers. Efforts to enhance clinical trial enrollment are warranted and include changes to ensure the generalizability of results and to advance patient safety and outcomes. [ABSTRACT FROM AUTHOR]

See article on page 2192–2198, in this issue [ABSTRACT FROM AUTHOR]

Background: Extracorporeal photopheresis (ECP) is an immunomodulatory cellular therapy which has been shown to induce a tolerogenic state in patients with acute and chronic graft-vs-host disease. ECOG-ACRIN explored the activity of ECP as a part of a reduced intensity conditioning regimen in two multicenter trials in patients with MDS (E1902) and lymphomas (E1402). While both studies closed before completing accrual, we report results in 23 patients (17 MDS and 6 lymphoma).Study Design and Methods: Patients received 2 days of ECP followed by pentostatin 4 mg/m2 /day for two consecutive days, followed by 600 cGy of total body irradiation prior to stem cell infusion. Immunosuppression for aGVHD was infusional cyclosporine A or tacrolimus and methotrexate on day +1, +3, with mycophenolate mofetil starting on day 100 for chronic GVHD prophylaxis.Results: All patients engrafted, with median time to neutrophil and platelet engraftment of 15-18 days and 10-18 days respectively. Grade 3 or 4 aGVHD occurred in 13% and chronic extensive GVHD in 30%.Conclusions: These studies demonstrate that ECP/pentostatin/TBI is well tolerated and associated with adequate engraftment of neutrophils and platelets in patients with lymphomas and MDS. [ABSTRACT FROM AUTHOR]

Allogeneic stem cell transplantation (allo-SCT) represents the most beneficial treatment for patients with active relapsed/refractory (R/R) hematologic malignancies. Recently, sequential regimens combining debulking chemotherapy followed by reduced-intensity conditioning (RIC) have shown encouraging results for these patients. In this retrospective study, we report the extended results of a sequential regimen of clofarabine, cytosine arabinoside, and RIC in 131 adults with active R/R myeloid disease at transplant. Conditioning consisted of clofarabine (30 mg/m2/day) and cytosine arabinoside (1 g/m2/day) for 5 days, followed, after a rest of 3 days, by an RIC combining cyclophosphamide (60 mg/kg) for 1 day, iv busulfan (3.2 mg/kg/day) for 2 days, and anti-thymocyte globulin (2.5 mg/kg/day) for 2 days. Between 2007 and 2016, 131 patients (males n = 75, median age: 52.6 years) were identified from the SFGM-TC registry. There were 111 acute myeloid leukemia (AML) patients and 20 cases with myelodysplastic or myeloproliferative syndrome. Status at transplant was known for all but 4 patients and was primary refractory (n = 81) and 1st or 2nd relapse (n = 46). All patients received allo-SCT from a matched donor (sibling n = 64, unrelated n = 67). Engraftment was observed in 105/122 (86%) evaluable cases and 63% of the patients achieved complete remission (CR) after transplant. The 1-year overall survival, disease-free survival, relapse incidence, non-relapse mortality, and graft-versus-host disease-free/relapse-free survival were 39.2%, 28.1%, 41.0%, 30.8%, and 22.2%, respectively. This study confirms that this sequential clofarabine-based regimen provides a high CR rate in this critical population, although relapse remains a matter of concern. [ABSTRACT FROM AUTHOR]

Objective: To evaluate the clinical risk factors contributing to the development of extended spectrum beta-lactamase (ESBL)- producing asymptomatic bacteriuria in myelodysplastic children performing clean intermittent catheterization (CIC). Materials and Methods: The clinical risk factors for ESBL-producing bacteriuria were retrospectively investigated in 60 myelodysplastic children who had asymptomatic bacteriuria and were performing CIC. A total of 60 children were included in this study, 30 children (17 females, 13 males) with ESBL-positive bacteriuria in urine culture were identified as the study group and 30 age- and gender-matched ESBL-negative children (16 females, 14 males) served as controls. All children had neurogenic bladder due to myelodysplasia and had been used anticholinergics. The two groups were compared in terms of age, gender, presence of constipation and motor deficit, antibiotic prophylaxis, number of hospital admission, ultrasound findings, and presence of renal scarring in dimercapto succinic acid scintigraphy and urodynamic findings. Results: The mean age of the children was 77±50 months in study and 78±69 months in control groups. There was no statistically significant difference in terms of maximum bladder capacity, leak point pressure, constipation status and scarring. In study and control groups, 83% and 46% of children were on antimicrobial prophylaxis, respectively (p=0.007). Conclusion: ESBL-producing bacteriuria was found to be associated with long-term antibiotic prophylaxis. Thus, it was concluded that the use of antibiotics for asymptomatic bacteriuria should be kept to a minimum. [ABSTRACT FROM AUTHOR]

The association between myelodysplastic syndrome (MDS) and Behçet syndrome (BS) is recognized for over 25 years. High frequency of trisomy 8 and intestinal ulcers are striking features of this association. There are no recommendations for how these patients should be treated. A systematic literature review was performed in PubMed using the keyword combination "(((((intestinal) OR gastrointestinal) OR ulcer) OR Behcet*)) AND ((myelodysplastic syndrome) OR MDS)" in March 2019. Our aim was to gain insight regarding clinical responses to individual treatment modalities. A recent case was also presented and included in the analysis. Data from 41 articles reporting on a total of 53 patients carried adequate information to assess treatment responses. Glucocorticoids provided benefit in 23 of 43 patients. Azacitidine, decitabine, thalidomide, and cyclosporine contributed to a clinical improvement in 4/6, 2/3, 3/4, and 5/8 patients respectively. Hematopoietic stem cell transplantation was successful in 9 of 13 patients. With the use of TNF inhibitors, azathioprine, and mesalamine derivatives, clinical improvement was observed in 3/11, 0/4, and 6/18 patients respectively. Patients with MDS and BS-like features who are resistant to glucocorticoids have so far benefited more from treatment approaches directed at MDS, rather than the immunosuppressive agents used for BS. [ABSTRACT FROM AUTHOR]

Background: Iron chelation therapy (ICT) in patients with lower-risk myelodysplastic syndromes (MDS) has not been evaluated in randomized studies.Objective: To evaluate event-free survival (EFS) and safety of ICT in iron-overloaded patients with low- or intermediate-1-risk MDS.Design: Multicenter, randomized, double-blind, placebo-controlled trial (TELESTO). (ClinicalTrials.gov: NCT00940602).Setting: 60 centers in 16 countries.Participants: 225 patients with serum ferritin levels greater than 2247 pmol/L; prior receipt of 15 to 75 packed red blood cell units; and no severe cardiac, liver, or renal abnormalities.Intervention: Deferasirox dispersible tablets (10 to 40 mg/kg per day) (n = 149) or matching placebo (n = 76).Measurements: The primary end point was EFS, defined as time from date of randomization to first documented nonfatal event (related to cardiac or liver dysfunction and transformation to acute myeloid leukemia) or death, whichever occurred first.Results: Median time on treatment was 1.6 years (interquartile range [IQR], 0.5 to 3.1 years) in the deferasirox group and 1.0 year (IQR, 0.6 to 2.0 years) in the placebo group. Median EFS was prolonged by approximately 1 year with deferasirox versus placebo (3.9 years [95% CI, 3.2 to 4.3 years] vs. 3.0 years [CI, 2.2 to 3.7 years], respectively; hazard ratio, 0.64 [CI, 0.42 to 0.96]). Adverse events occurred in 97.3% of deferasirox recipients and 90.8% of placebo recipients. Exposure-adjusted incidence rates of adverse events (≥15 events per 100 patient treatment-years) in deferasirox versus placebo recipients, respectively, were 24.7 versus 23.9 for diarrhea, 21.8 versus 18.7 for pyrexia, 16.7 versus 22.7 for upper respiratory tract infection, and 15.9 versus 0.9 for increased serum creatinine concentration.Limitations: The protocol was amended from a phase 3 to a phase 2 study, with a reduced target sample size from 630 to 210 participants. There was differential follow-up between treatment groups.Conclusion: The findings support ICT in iron-overloaded patients with low- to intermediate-1-risk MDS, with longer EFS compared with placebo and a clinically manageable safety profile. Therefore, ICT may be considered in these patients.Primary Funding Source: Novartis Pharma AG. [ABSTRACT FROM AUTHOR]

Background: Patients with congenital haptoglobin deficiency can develop anti-haptoglobin antibodies after exposure to blood products, and they can suffer from life-threatening anaphylactic transfusion reactions. Here, we present a case of a 57-year-old Chinese male with myelodysplastic syndrome who manifested an anaphylactic transfusion reaction during the transfusion of platelets. The only abnormality detected during his reaction laboratory workup was an undetectable haptoglobin level in the absence of evidence of hemolysis.Study Design and Methods: Surface plasmon resonance (SPR) was explored as a method to be able to detect the presence of anti-haptoglobin antibodies in serum. First, haptoglobin was immobilized to the surface of an SPR sensor chip. The patient's serum sample was injected, and the binding response was monitored in real time. Serum samples from five healthy volunteers were used as negative controls. Binding specificity was assessed in competition experiments using soluble haptoglobin. Anti-IgG, -IgA, -IgM, -IgD and -IgE antibodies were used to identify the antibody isotype.Results: An IgG anti-haptoglobin antibody was detected in the patient's serum with SPR.Conclusion: SPR provided a rapid, readily available method for the detection of an IgG anti-haptoglobin antibody in an anhaptoglobinemic individual. This confirmed the underlying etiology of the anaphylactic nonhemolytic transfusion reaction and justified the necessity of stringently washed cellular products for all future transfusions and strong caution for future use of plasma-containing products. [ABSTRACT FROM AUTHOR]

Introduction: Allogeneic hematopoietic cell transplantation (allo-HCT) is a standard of care for patients with acute myeloid leukemia (AML) and patients with intermediate and high-risk myelodysplastic syndrome (MDS). Despite many years of experience there is still no standard for conditioning regimen. The aim of this study is to analyse the efficacy and safety of a conditioning treatment with cladribine in combination with total body irradiation (TBI). Material and methods: A group of 40 adult patients referred for allo-HCT due to AML and MDS are to be enrolled in the study. The inclusion criteria are: informed consent, chemo-sensitivity for cladribine treatment regimens (when used in induction therapy), age 18-60, and performance status 0-2 according to World Health Organization. The conditioning regimen consists of cladribine and TBI at a total dose of 12 Gy in three fractions given over three consecutive days. The goal of the study is to assess the tolerability and efficacy of the regimen. Results: Our results may stimulate further investigation in this field i.e. phase III trials to compare this regimen to others. Conslusion: A myeloablative conditioning regimen consisting of total body irradiation in combination with cladribine may contribute to improved outcomes after allo-HCT for AML and MDS patients. [ABSTRACT FROM AUTHOR]

The article focuses on the use of the recombinant fusion protein luspatercept to treat patients with lower-risk myelodysplastic syndromes. It discusses the method and design of the randomized, double-blind, placebo-controlled trial conducted in eleven countries and notes that 229 patients with lower-risk myelodysplastic syndromes with ring sideroblasts were enrolled. Adverse events and transfusion independence are mentioned.

Background: Using the Recipient and Donor Epidemiology Study-III (REDS-III) recipient and donor databases, we performed a retrospective analysis of platelet use in 12 US hospitals that were participants in REDS-III.Study Design and Methods: Data were electronically extracted from participating transfusion service and blood center computer systems and from medical records of the 12 REDS-III hospitals. All platelet transfusions from 2013 to 2016 given to patients aged 18 years and older were included in the analysis.Results: There were 28,843 inpatients and 2987 outpatients who were transfused with 163,719 platelet products (103,371 apheresis, 60,348 whole blood derived); 93.5% of platelets were leukoreduced and 72.5% were irradiated. Forty-six percent were transfused to patients with an International Classification of Diseases, 9th/10th Revision (ICD-9/10) diagnosis of leukemia, myelodysplastic syndrome (MDS), or lymphoma. The general ward and the intensive care unit (ICU) were the most common issue locations. Only 54% of platelet transfusions were ABO identical; and 60.6% of platelet transfusions given to Rh-negative patients were Rh positive. The most common pretransfusion platelet count range for inpatients was 20,000 to 50,000/μL, for outpatients it was 10,000 to 20,000/μL. Among ICU patients, 35% of platelet transfusion episodes had a platelet count of greater than 50,000/μL; this was only 8% for general ward and 2% for outpatients. The median posttransfusion increment, not corrected for platelet dose and/or patient size, ranged from 12,000 to 20,000/μL for inpatients, and from 17,000 to 27,000/μL for outpatients.Conclusions: These data from one of the largest reviews of platelet transfusion practice to date provide guidance for where to focus future clinical research studies and platelet blood management programs. [ABSTRACT FROM AUTHOR]

Objectives: To investigate the relationship between gene mutations and response to Compound Qinghuang Powder (复方青黄散, CQHP) in patients with myelodysplastic syndrome (MDS). Methods: Forty-three MDS patients were genotyped by ultra-deep targeted sequencing and the clinical data of patients were collected and the relationship between them was analyzed. Results: Up to 41.86% of patients harbored genet mutations, in most cases with more than one mutation. The most common mutations were in SF3B1, U2AF1, ASXL1, and DNMT3A. After treatment with CQHP, about 88.00% of patients no longer required blood transfusion, or needed half of prior transfusions. Conclusions: CQHP is an effective treatment for patients with MDS, especially those with gene mutations in SF3B1, DNMT3A, U2AF1, and/or ASXL1. [ABSTRACT FROM AUTHOR]

Epstein-Barr virus (EBV) reactivation is a life-threatening complication after allogeneic haematopoietic stem cell transplantation (allo-HSCT). In this study, we investigated the characteristics of EBV reactivation in 186 consecutive myelodysplastic (MDS) patients who underwent allo-HSCT in our centre. In 35 patients (18.8%) who experienced EBV reactivation after allo-HSCT, the median onset was 53 days (range 4-381 days). The cumulative incidence of EBV reactivation at the first, sixth, and twelfth month after allo-HSCT was 10.7%, 15.1%, and 17.9%, respectively. Twenty-five patients (71.4%) received pre-emptive rituximab therapy, and no patients developed post-transplant lymphoproliferative disorders. Stem cell source was proven to be a risk factor correlated with EBV reactivation. The cumulative incidence of relapse in the EBV-positive group was 11.4%, 25.2%, and 31.0% at the first, second, and third year after transplantation, respectively, being significantly higher than the corresponding 6.8%, 10.2%, and 10.2%, in the EBV-negative group (P = 0.014). Prognostic analysis showed that EBV reactivation was an independent risk factor for relapse-free survival (RFS). Patients in the EBV-positive group showed obviously shorter RFS than those in the EBV-negative group, with 3-year RFS of 62% and 85%, respectively (P = 0.017). [ABSTRACT FROM AUTHOR]

Introduction: Noninvasive ventilation (NIV) is commonly used as first-line therapy for immunocompromised patients with acute respiratory failure. However, it may not be appropriate for all patients, as failure of NIV and delayed endotracheal intubation (ETI) may increase mortality. We report our center's experience and outcomes for patients with active hematologic malignancy (HM) treated with NIV. Methods: We conducted a retrospective study of consecutive patients with HM who were admitted to the intensive care unit (ICU) of Mount Sinai Hospital for acute respiratory failure between January 1, 2010, and May 31, 2015, and were initially treated with NIV. We compared the characteristics of patients who were successfully treated with NIV and avoided intubation and those who failed NIV. Results: Seventy-nine patients (mean age 56 ± 14 years, mean Acute Physiology and Chronic Health Evaluation II score 27 ± 5) with HM were treated with NIV for acute respiratory failure. The etiology of respiratory failure was multifactorial in 31 (39%) patients, with features of pneumonia in 61 (77%) patients, severe sepsis or septic shock in 33 (42%) patients, and pulmonary edema in 24 (30%) patients. The majority of patients were admitted with acute leukemia (n = 60, 76%), 8 (10%) with lymphoma, and 11 (14%) with chronic leukemia, multiple myeloma, or myelodysplastic syndrome. Of the 79 patients treated with NIV, 44 (56%) failed NIV and required ETI, 7 (9%) had a do-not-intubate (DNI) order and died, and 28 (35%) avoided ETI. Compared with patients who avoided ETI, those who failed NIV or had a DNI order and died were more likely to have acute leukemia (84% vs 61%; P = .02) and at baseline had higher PaCO2 (39 vs 30; P = .038), higher fraction of inspired oxygen (FIO2) requirements (0.6 vs 0.4; P = .002), and more vasopressor use (31% vs 11%; P = .059). The ICU mortality was 42%; 3-month mortality was 57% overall and was significantly lower in the NIV success patients compared with the NIV failure group (21% vs 74%; P < .001). Conclusion: Two-thirds of patients with HM and respiratory failure failed NIV and required ETI, and had high subsequent mortality. Patients who failed NIV had higher PaCO2, higher FIO2, and a trend toward more vasopressor use. [ABSTRACT FROM AUTHOR]

A hemodialysis patient with higher-risk myelodysplastic syndrome treated with standard-dose azacitidine. Azacitidine for injection (AZA) (Vidaza, Celgene Corp., Summit, NJ), represents a well-established and registered agent in Myelodysplastic Syndromes (MDS) and in acute myeloid leukemia (AML) [[1]]. In hemodialysis patients there are only a few case reports of administration of AZA and to our knowledge it has never been investigated to which extent AZA is removed by hemodialysis [[14]]. [Extracted from the article]

Andrew M. Brunner, M.D., of Massachusetts General Hospital, shared his insights on key practice‐changing research presented at ASH 2020. [ABSTRACT FROM AUTHOR]