Abstract:
:Acute myeloid leukemia (AML) is a complex and heterogeneous disease with distinct age-associated genomic and epigenomic alterations. A large number of somatic karyotypic and molecular alterations have been identified in AML to date; however, very few predict outcome or identify potential therapeutic targets. Here we describe the current state of known molecular and genetic alterations in pediatric AML. Further, as recent advances in sequencing technologies have revolutionized our ability to interrogate cancer genome, transcriptome, and epigenome, we will also review the emerging genomic data identified by next-generation sequencing and discuss their potential impact as tools for therapeutic interventions in the near future. In coming years, a wealth of data from large-scale discovery phase projects such as the Children's Oncology Group/ National Cancer Institute (COG/NCI) TARGET AML initiative will be available to researchers to discover new biomarkers for risk and target identification in pediatric AML.
journal_name
Semin Hematoljournal_title
Seminars in hematologyauthors
Schuback HL,Arceci RJ,Meshinchi Sdoi
10.1053/j.seminhematol.2013.09.003subject
Has Abstractpub_date
2013-10-01 00:00:00pages
325-32issue
4eissn
0037-1963issn
1532-8686pii
S0037-1963(13)00122-4journal_volume
50pub_type
杂志文章,评审abstract::A new high-purity recombinant factor VIII preparation has been developed for the treatment of hemophilia A. Structurally, this factor VIII preparation, B-domain deleted recombinant factor VIII (BDDrFVIII), differs from other recombinant and plasma-derived factor VIII preparations in that most of the B-domain has been ...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1016/s0037-1963(01)90103-9
更新日期:2001-04-01 00:00:00
abstract::Both thalidomide and intermittent high-dose dexamethasone are agents with established activity against multiple myeloma. We summarized our experience with thalidomide alone, and then in combination with dexamethasone, for groups of patients with myeloma resistant or relapsing despite standard treatments. Criteria of r...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1053/j.seminhematol.2003.09.006
更新日期:2003-10-01 00:00:00
abstract::Virtually all human cells are endowed with the capacity to commit suicide using an evolutionarily conserved mechanism that involves activation of caspase-family cell death proteases. Caspase activation culminates in a cell death process known as "apoptosis." The activation of these intracellular proteases is carefully...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1016/s0037-1963(00)90055-6
更新日期:2000-10-01 00:00:00
abstract::The purpose of this study was to assess the efficacy and side effect profile of the repeated use of anti-D for the treatment of pediatric immune thrombocytopenia (ITP) in a large pediatric hematology center. We performed a retrospective analysis of patient records for children (aged 4 months-18 years) treated for ITP ...
journal_title:Seminars in hematology
pub_type: 杂志文章
doi:10.1053/j.seminhematol.2016.04.019
更新日期:2016-04-01 00:00:00
abstract::High-dose chemoradiotherapy with allogeneic bone marrow or peripheral blood stem cell transplantation (SCT) is a potentially curative treatment for advanced or poor-prognosis hematological malignancies. This procedure was initially considered as a means to deliver supralethal doses of chemotherapy and radiation for th...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1053/shem.2002.30909
更新日期:2002-01-01 00:00:00
abstract::The use of combination chemotherapy has been successful in treating advanced or widespread Hodgkin's disease (HD). For patients with relapsed disease, the long-term prognosis is much poorer, despite the good rate of reinduction into a second or further remission using conventional treatments. The majority of such pati...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:
更新日期:1999-07-01 00:00:00
abstract::Smoldering myeloma (SMM) is a precursor state of multiple myeloma. It is defined by an M-protein concentration ≥3 g/dL and/or ≥10% clonal bone marrow plasma cells, in the absence of end-organ damage. Based on clinical observations, the natural history of SMM varies greatly, from stable, monoclonal gammopathy of undete...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1053/j.seminhematol.2010.11.009
更新日期:2011-01-01 00:00:00
abstract::The standard treatment of multiple myeloma is systemic chemotherapy. Despite 30 years of drug development in myeloma, there are no new drug regimens significantly superior to melphalan and prednisone. In addition, phase II studies of new drugs in myeloma have been disappointing, with low response rates and no prolonga...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:
更新日期:1998-07-01 00:00:00
abstract::Traditionally, acute myeloid leukemia (AML) has been diagnosed and classified based on the morphologic and cytochemical criteria of the French-American-British (FAB) classification system. However, more recent studies have demonstrated that the cytogenetic and molecular genetic abnormalities consistently associated wi...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:
更新日期:1999-10-01 00:00:00
abstract::Primary immune thrombocytopenic purpura (ITP) remains a diagnosis of exclusion both from nonimmune causes of thrombocytopenia and immune thrombocytopenia that develops in the context of other disorders (secondary immune thrombocytopenia). The pathobiology, natural history, and response to therapy of the diverse causes...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1053/j.seminhematol.2008.12.005
更新日期:2009-01-01 00:00:00
abstract::Though peripheral T-cell lymphoma (PTCL) is an area of significant unmet therapeutic need, a number of new treatment options are available for patients, especially those with relapsed or refractory disease. A plethora of drugs are now in development for PTCL, but drugs that truly target novel disease biology are notic...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1053/j.seminhematol.2010.01.014
更新日期:2010-04-01 00:00:00
abstract::Even though the overall outcome after allogeneic transplant has improved significantly in the last decades, late infectious diseases are still the most important causes of late morbidity and mortality. Here, impaired immune reconstitution and therapy of chronic graft-versus-host disease (GVHD) represent the major risk...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1053/j.seminhematol.2011.10.009
更新日期:2012-01-01 00:00:00
abstract::This article summarizes clinical results of the Intergroupe Francophone du Myélome (IFM) trials: high-dose therapy (HDT) supported with autologous stem cells improves survival, melphalan 200 mg/m(2) is the best preparative regimen, unpurged peripheral blood stem cells (PBSC) are the recommended source of stem cells to...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1016/s0037-1963(01)90014-9
更新日期:2001-07-01 00:00:00
abstract::Trauma is rapidly replacing stroke and cardiovascular disease as a leading cause of death in Western countries such as the United States, and almost a third (30%) of trauma deaths are due to blood loss. Although the new intervention strategies that have been developed and adopted by emergency care staff have reduced t...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1053/j.seminhematol.2003.11.009
更新日期:2004-01-01 00:00:00
abstract::Continuous daily treatment with all-trans-retinoic acid (ATRA) in patients with acute promyelocytic leukemia has been associated with a marked decline in the plasma drug concentration at the time of relapse. Recent pharmacologic studies have attempted to determine the reasons for the progressive reduction of plasma co...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:
更新日期:1994-10-01 00:00:00
abstract::The introduction of chimeric antigen receptor (CAR) T-cell therapy in acute lymphoblastic leukemia (ALL) has dramatically altered the landscape of treatment options available to children and adults with ALL. With complete remission induction rates exceeding 70% in most trials and FDA approval of one CD19 CAR T-cell co...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1053/j.seminhematol.2020.07.002
更新日期:2020-07-01 00:00:00
abstract::The activation, function, and development of peripheral T lymphocytes are dependent on the ability to signal properly through the surface T-cell antigen receptor (TCR)-CD3 complex. Transmission of such signals requires the activation of specific cytoplasmic protein tyrosine kinases (PTK) associated with the TCR. Recen...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:
更新日期:1998-10-01 00:00:00
abstract::Chronic lymphocytic leukemia (CLL) comprises a substantial proportion of leukemias in adults in the western hemisphere. Male gender, increasing age, ethnicity (high in Caucasians, lowest in Asians), and family history are risk factors. Although no specific extrinsic etiologic factors have been established, farming and...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1053/j.seminhematol.2004.05.002
更新日期:2004-07-01 00:00:00
abstract::Gene expression profiling of cancer began as a research tool but is rapidly moving towards clinical application. The diagnostic category of diffuse large B-cell lymphoma (DLBCL) can now be viewed as an amalgam of several different diseases that have distinct gene expression profiles, oncogenic mechanisms, and clinical...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1016/s0037-1963(03)00194-x
更新日期:2003-10-01 00:00:00
abstract::A multicenter clinical trial of the thrombin inhibitor argatroban (Novastan; Texas Biotechnology, Houston, TX; Smith-Kline Beecham Pharmaceuticals, Philadelphia, PA) was recently conducted in patients with heparin-induced thrombocytopenia (HIT) and HIT that had progressed to thrombosis (HITTS). In patients defined by ...
journal_title:Seminars in hematology
pub_type: 临床试验,杂志文章,多中心研究
doi:
更新日期:1999-01-01 00:00:00
abstract::Current therapeutic strategies for autoimmune diseases primarily rely on immunosuppression, but global immune suppression results in an increased risk for severe infection and malignancy. In contrast, immuomodulation is another therapeutic approach employing intrinsic or environmental regulators that exert modulatory ...
journal_title:Seminars in hematology
pub_type: 杂志文章
doi:10.1053/j.seminhematol.2016.04.009
更新日期:2016-04-01 00:00:00
abstract::Because optimal management of iron chelation therapy in patients with sickle cell disease and transfusional iron overload requires accurate determination of the magnitude of iron excess, a variety of techniques for evaluating iron overload are under development, including measurement of serum ferritin iron levels, x-r...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1016/s0037-1963(01)90059-9
更新日期:2001-01-01 00:00:00
abstract::Various pathways lead to the development of venous thrombosis. Risk factors are common and can be genetic or acquired. Since the identification of factor V Leiden and prothrombin 20210 G-->A, the field of genetic epidemiology has developed rapidly and many new genetic variants have been described in the past decade. H...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1053/j.seminhematol.2007.01.007
更新日期:2007-04-01 00:00:00
abstract::Hemangiomas and lymphangiomas are two main types of angiomatous disease that occur most commonly in infancy and childhood. Most hemangiomas resolve spontaneously, but some endanger vital structures such as the lung, as in pulmonary hemangiomatosis, a rare and universally fatal disease. Occasionally, hemangiomatous les...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:
更新日期:1990-07-01 00:00:00
abstract::Purine analogues have been shown to be active in a variety of B- and T-cell malignancies. Among them, pentostatin is also a tight binding inhibitor of adenosine deaminase (ADA), a key enzyme of purine metabolism. ADA is present in all human tissues, with the highest levels in the lymphoid system. Early clinical trials...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1053/j.seminhematol.2005.12.005
更新日期:2006-04-01 00:00:00
abstract::The prolonged and severe myelosuppression associated with high-dose chemotherapy and autologous bone marrow transplantation is a major causative factor in patient morbidity and mortality resulting from infection and other causes. The recent molecular cloning, in vitro expression, and formulation of recombinant human c...
journal_title:Seminars in hematology
pub_type: 杂志文章
doi:
更新日期:1989-04-01 00:00:00
abstract::The successful clinical development of thalidomide, bortezomib, and lenalidomide not only transformed the therapeutic management of multiple myeloma (MM) but also catalyzed a renewed interest in the development of additional classes of novel agents for this disease. This review focuses on a series of new therapeutics ...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1053/j.seminhematol.2009.02.003
更新日期:2009-04-01 00:00:00
abstract::Cytogenetic data suggest that acute myeloid leukemia (AML) develops through a process of branching evolution, especially during relapse and progression. Recent genomic data from AML cases using digital sequencing, temporal comparisons, xenograft cloning, and single-cell analysis indicate that most, if not all, AML cas...
journal_title:Seminars in hematology
pub_type: 杂志文章
doi:10.1053/j.seminhematol.2014.08.004
更新日期:2014-10-01 00:00:00
abstract::Advances in recombinant DNA manufacturing technology have now made possible the production of a highly purified and active recombinant factor IX (rFIX) product. Recombinant factor IX was developed by (1) stable insertion of the genes for both factor IX and PACE-SOL (a truncated, soluble serine protease needed to enhan...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:
更新日期:1998-04-01 00:00:00
abstract::The stem cell origination of the clonal process in chronic myeloproliferative disorders (CMPDs) is well established. In chronic myelogenous leukemia (CML), the primary genetic process has been characterized. However, current information regarding the mechanisms of phenotypic diversity among the CMPD and the downstream...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:
更新日期:1999-01-01 00:00:00