Hematology. American Society of Hematology. Education Program最新文献

Five-year survival for childhood cancer now exceeds 85%. However, for many patients, treatment requires the use of intensive anthracycline-based chemotherapy and radiotherapy, both of which are associated with significant long-term cardiovascular toxicity. As such, late cardiovascular disease is now one of the leading causes of premature morbidity and mortality among childhood cancer survivors. Recent advances over the past decade have refined the cardiotoxic potential of various chemotherapeutics, and ongoing work seeks to determine the efficacy of various cardioprotective strategies in children receiving active cancer therapy. The development of risk prediction models offers an additional strategy to define risk for both newly treated and long-term survivors. Current screening strategies are primarily based on echocardiography, although there is active research investigating methods to further optimize screening through myocardial strain, cardiac magnetic resonance imaging, blood biomarkers, and genetics, along with the cost-effectiveness of different screening strategies. Active research is also underway investigating the efficacy of prevention strategies for childhood cancer survivors who have completed cancer therapy. This ranges from the use of medications to mitigate potential pathologic ventricular remodeling to reducing adverse and modifiable cardiovascular risk factors (eg, hypertension, dyslipidemia, insulin resistance, physical inactivity, tobacco exposure), many of which may be more common in cancer survivors vs the general population and are often underrecognized and undertreated in relatively young adult-aged survivors of childhood cancer.

Discussions regarding gonadal function and possible disease or treatment-related ovarian or testicular dysfunction, sexual dysfunction, and possible future infertility can be challenging in the sickle cell disease (SCD) pediatric care setting. A construct that stratifies topics into those that are time sensitive and those that require reproductive care expertise vs address gonadal health as a part of normal SCD care may be helpful. Pediatric health care discussions of gonadal function/dysfunction for patients with SCD can include (1) time-sensitive fertility consults preceding the start of gonadotoxic therapy and (2) targeted discussions at key time points during normally scheduled hematology clinic visits. The former conversations are best led by individuals with expertise in the risk for treatment-related infertility and fertility preservation. The latter discussions can be incorporated into targeted regularly scheduled visits with hematologists. These topics can be addressed as a part of planned education in pediatric care for adolescents and incorporated into transition plans as young adults transfer care to adult providers. Although the topics of puberty and gonadal health can be uncomfortable and many complex interdisciplinary and ethical issues arise in this process, these discussions can be aided by the collaterals and teaching handouts presented in this article.

Despite significant improvement in the treatment of multiple myeloma (MM), a cure remains elusive, and patients failing proteasome inhibitors, immunomodulatory drugs, and anti-CD38 monoclonal antibodies remain a challenge due to a lack of standard of care treatment and a dismal survival rate. The development of T-cell redirecting therapies, including bispecific T-cell engagers and chimeric antigen receptor (CAR) T cells, have transformed the outcome of triple-class exposed relapsed and refractory MM (RRMM). B-cell maturation antigen (BCMA) has proven to be an important target in MM, and BCMA-directed CAR T cells have shown unprecedented efficacy with a prolonged duration of response in a population with advanced RRMM, leading to the approval of 2 different BCMA CAR T-cell products. Still, and in contrast to prior experience in the field of CD19-directed CARs, no plateau has been seen in the survival curves, and relapses continue to occur. Therefore, further improvement is needed. Early use in the course of the disease as well as of next- generation CARs may further augment the efficacy of these therapies. In this review we address current state-of-the-art approved BCMA-directed CAR T-cell therapy in RRMM, as well as potential future developments focused on optimizing patient care and novel CAR designs.

This article reviews 3 products: pathogen-inactivated platelets, cold-stored platelets, and cryoplatelets. These are all coming to a transfusion service near you in the next few years. The article reviews the limitations of these new products and highlights the gaps in our understanding of their place in patient treatment.

Most people with von Willebrand disease (VWD) have a partial quantitative deficiency of plasma von Willebrand factor (VWF) or type 1 VWD. In contrast to type 2 and type 3 VWD, laboratory assays will not always establish the diagnosis in type 1 VWD. This is because plasma VWF levels in type 1 VWD, especially those with levels closer to 50 IU/dL, overlap with the general population. Assessment is further complicated by increased plasma VWF levels in response to physiologic stressors or aging. Diagnosis of those with type 1 VWD with plasma VWF levels 30 to 50 IU/dL (previously referred to as "low VWF") requires expert assessment of bleeding phenotype as well as an understanding of the limitations of both bleeding assessment tools (BATs) and laboratory testing. Using the available evidence and highlighting research gaps, we discuss common dilemmas facing providers relating to assessment of adolescents, transition from pediatrics to adult care, and older individuals with type 1 VWD.

Smoldering multiple myeloma (SMM) is an asymptomatic precursor condition to multiple myeloma (MM). The prevalence of SMM is 0.5% in persons over 40 years old; it is higher in men than women and increases with age. When SMM is diagnosed, a thorough diagnostic workup is necessary to exclude myeloma-defining events and stratify patients according to risk of progression to MM. While close monitoring for progression remains the best management for most patients with SMM, in this article, we discuss if treatment initiation before myeloma-defining events occur might be relevant in selected high-risk cases. Two randomized clinical trials have shown a clinical benefit of initiating treatment at the SMM stage, whereof 1 showed an overall survival benefit for those receiving treatment. We discuss various risk stratification models in SMM, important treatment trials, and ongoing trials. Finally, we present how to approach the clinical management of patients with SMM.

Novel therapies in multiple myeloma (MM) have increased the rates of conventional complete remission (CR) in patients. However, patients in CR can have highly heterogeneous outcomes. Novel and more sensitive methods of assessing residual disease burden after therapy will help prognosticate this group better and, ideally, allow individualized therapy adjustments based on response depth in the future. Here, we review novel bone marrow, peripheral blood, and imaging methods for assessing myeloma burden and discuss the opportunities and limitations of incorporating these in everyday clinical practice.