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Abstract : The immunoglobulin light-chain amyloidosis is a multisystemic disease which manifests by damage to the vital organs by light chain-derived amyloid fibril. Traditionally, the treatment has been directed to the underlying plasma cell clone with or without high dose chemotherapy followed by autologous stem cell transplantation using melphalan based conditioning. Now with the approval of highly tolerable anti-CD38 monoclonal antibody daratumumab based anti-plasma cell therapy in 2021, high rates of hematologic complete responses are possible even in patients who are otherwise deemed not a candidate for autologous stem cell transplantation. However, despite the progress, there remains a limitation in the strategies to improve symptoms particularly in patients with advanced cardiac involvement, those with nephrotic syndrome and autonomic dysfunction due to underlying systemic AL amyloidosis. The symptoms can be an ordeal for the patients and their caregivers and effective strategies are urgently needed to address them. The supportive care is aimed to counteract the symptoms of the disease and the effects of the treatment on involved organs’ function and preserve patients’ quality of life. Here we discuss multidisciplinary approach in a system-based fashion to address the symptom management in this dreadful disease. In addition to achieving excellent anti-plasma cell disease control, using treatment directed to remove amyloid from the vital organs can theoretically hasten recovery of the involved organs thereby improving symptoms at a faster pace. Ongoing phase III clinical trials of CAEL-101 and Birtamimab will address this question.

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Abstract : The incidence of hematologic malignancy increases with age; thus, the number of older patients who require intensive chemotherapy is expected to increase with the aging population. In Korea, 61.8%, 59.3%, 47.0%, and 46.7% of newly diagnosed cases of multiple myeloma, myelodysplastic syndrome, myeloproliferative disorder, and non-Hodgkin lymphoma, respectively, occurred in patients aged ＞65 years in 2018. Health status among older patients, defined by frailty, age-related syndrome of physiological decline and increased vulnerability, is associated with adverse health outcomes. Health status is highly heterogeneous among older patients, and treatment outcomes vary according to frailty and physiologic age rather than chronologic age. Comprehensive geriatric assessment (CGA) is a multidimensional and multidisciplinary diagnostic and treatment process that identifies multiple domains, including functional status, cognition, comorbidities, medications, socioeconomic status, and nutritional status, to develop a coordinated plan to improve treatment-related outcomes and quality of life. Frailty can be assessed with CGA findings, and CGA is considered the “gold standard of care” for frail, older patients. Through CGA, unidentified problems can be assessed, and pre-emptive and non-oncologic interventions can be delivered. CGA is an objective and reliable tool for predicting further treatment-related complications and identifying patients for whom intensive chemotherapy with curative intent is appropriate. CGA should be considered a routine practice before starting treatment planning in older patients diagnosed with hematologic malignancies who require intensive chemotherapy. Further study is needed to allocate individualized treatment plans or multidisciplinary geriatric interventions according to CGA results.

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Abstract : Platelet transfusion refractoriness (PTR), in which platelet counts do not increase after transfusion, occurs in many patients receiving platelet transfusions. PTR is a clinical condition that can harm patients. The causes of PTR can be divided into two types: immune and non-immune. Most cases of PTR are non-immune. Among immune causes, the most common is human leukocyte antigen (HLA) class I molecules. PTR caused by anti-HLA antibodies is usually managed by transfusing HLA-matched platelets. Therefore, it is important, especially for hemato-oncologists who frequently perform transfusion, to accurately diagnose whether the cause of platelet transfusion failure is alloimmune or non-immunological when determining the treatment direction for the patient. In this review, we discuss the definitions, causes, countermeasures, and prevention methods of PTR.

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Abstract : Inherited platelet disorders (IPDs) can cause mucocutaneous bleeding due to impaired primary hemostatic function of platelets, thrombocytopenia, or both. Recent advances in molecular technology can help identify many genes related to platelet biology, control the overall steps of megakaryopoiesis, and cause IPD. In this article, currently available laboratory tools for diagnosing IPDs with the characteristic laboratory features of each IPD are reviewed, and a general diagnostic approach for the evaluation of IPD patients is presented.

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Abstract : The term “monoclonal gammopathy of clinical significance” (MGCS) refers to any plasma cell or B-cell clonal disorder that does not meet the current criteria for malignant disorders but produces a monoclonal protein that directly or indirectly results in organ damage. The most commonly affected organs are the kidneys, nerves, and skin. This review summarizes the current classification of MGCS and its diagnostic and treatment approaches.

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Abstract : POEMS syndrome is an acronym for polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes. It is a rare paraneoplastic disorder related to plasma cell neoplasm. However, its pathophysiology has not yet been clearly elucidated. The production of pro-inflammatory cytokines is thought to play an important role in the pathogenesis of POEMS syndrome. Vascular endothelial growth factor level reflects disease activity, which is helpful for the diagnosis and evaluation of treatment response. Conventional agents such as corticosteroids and melphalan are effective and safe combination regimens. Autologous hematopoietic stem cell transplantation is another option for high-risk transplant-eligible patients. Radiotherapy is effective in patients with localized lesions. The anti-myeloma agents lenalidomide, thalidomide, and bortezomib have shown good treatment outcomes for POEMS syndrome; however, large-scale studies with long-term follow-up are required. Early identification and active treatment can improve the outcomes of POEMS syndrome patients.

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Abstract : FMS-like tyrosine kinase 3 (FLT3) mutations, the most frequently detected genetic aberrations in patients with acute myeloid leukemia (AML), are identified in approximately 30% of patients with newly diagnosed AML and are more common in patients with normal karyotypes. Since the discovery of FLT3 mutations in AML, clinical trials have been actively conducted in patients with FLT3 mutated AML, and FLT3 inhibitors have been introduced into clinical practice. The current standard treatment for patients with newly diagnosed FLT3-mutated AML is 7+3 induction chemotherapy combined with midostaurin. Additionally, gilteritinib is more effective than salvage chemotherapy for relapsed or refractory FLT3-mutated AML. Ongoing trials are expected to provide additional treatment options depending on the disease state and patient vulnerability. This review summarizes information on clinically available FLT3 inhibitors for the management of AML with FLT3 mutations.

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Abstract : Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a potentially life-threatening thrombotic microangiopathy caused by autoantibody-mediated severe ADAMTS13 deficiency. TTP should be suspected in patients with microangiopathic hemolytic anemia and thrombocytopenia without a definite cause. Early detection of iTTP and prompt treatment with plasma exchange and corticosteroids are essential. Rituximab administration should be considered for refractory or relapsed iTTP, and can be used as a first-line adjuvant or preemptive therapy. Treatment with caplacizumab, a novel anti-von Willebrand factor nanobody, resulted in a faster time to platelet count response, significant reduction in iTTP-related deaths, and reduced incidence of refractory iTTP. TTP survivors showed a higher rate of chronic morbidities, including cardiovascular disease and neurocognitive impairment, which can lead to a poor quality of life and higher mortality rate. Meticulous long-term follow-up of TTP survivors is crucial.

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Abstract : Venous thromboembolism (VTE) is a common complication among patients with cancer and is associated with delays in underlying cancer treatment and increases in morbidity and mortality. Acute and long-term treatments with low-molecular-weight-heparin (LMWH) have been recommended as a standard of care for patients with cancer with VTE for the past 20 years. Direct oral anticoagulants (DOACs) have recently emerged as a new therapeutic modality for cancer-associated VTE because of the convenience of oral administration and rapid onset of action. Our knowledge regarding DOACs for cancer-associated VTD has expanded in recent years. Thus, this study aimed to review recent major pivotal trials comparing DOACs with LMWH for managing cancer-associated VTE. Moreover, a recently updated understanding of DOACs in the treatment of cancer-associated VTE in specific challenging situations is presented.

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Abstract : Patients with inherited bone marrow failure syndrome (IBMFS) can develop peripheral blood cytopenia, which can ultimately progress to myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). Although some cases of IBMFS are diagnosed based on their typical presentation, variable disease penetrance and expressivity may result in diagnostic dilemmas. With recent advances in genomic evaluation including next-generation sequencing, many suspected cases of IBMFS with atypical presentations can be identified. Identification of the genetic causes of IBMFS has led to important advances in understanding DNA repair, telomere biology, ribosome biogenesis, and hematopoietic stem cell regulation. An overview of this syndromes is summarized in this paper.