The hemophilias and their clinical management: reports in the 55th ASH annual meeting / 白血病·淋巴瘤

Outcomes for patients with hemophilia have improved dramatically over the past 50 years.With the increased availability of safe clotting factor concentrates,the primary focus in clinical management is now the prevention of long-term complications,most notably the debilitating hemophilic arthropathy that is associated with severe disease.Definitive evidence of improved clinical results from primary prophylaxis started in young patients-with severe hemophilia A and a minimal bleeding history is presented.Furthermore,recent studies showing benefits for initiating prophylaxis in older adolescents and adults with established joint disease are examined.lnhibitors to factor Ⅷ are the most problematic complication of factor replacement therapy.Patient-specific and treatment-related factors that contribute to the risk of inhibitor formation are discussed and controversies and clinical evidence related to approaches for tolerance induction are reviewed.Immune tolerance induction is the proven method for eradication of inhibitors.This article reviews latest research presented at the 55th ASH annual meeting on the hemophilias and their clinical management.

Reports on research progress of mantle cell lymphoma in the 55th ASH annual meeting / 白血病·淋巴瘤

Mantle cell lymphoma (MCL),as a kind of highly aggressive non-Hodgkin's lymphoma,is characterized by malignant B cell infiltration of lymph node,gastrointestinal tract and bone marrow.Patients suffering from MCL are prone to drug resistance or progression of disease.The 55th ASH annual meeting about MCL explored all aspects.In preclinical research aspects:new mutations including ATM,POT1 and tumor microenvironment further improved the pathogenesis mechanism of MCL.In prognostic indicators,minimal residual disease detected by molecular techniques and whole genome methylation analysis provided new tools for monitoring MCL prognosis and curative effect to guide further risk stratification and individual treatment.Exploration of high effective and low toxic side effective treatment strategies for elderly MCL patients has become a major focus of the meeting.Chemoimmunotherapy including Lenalidomide,bendamustine,rituximab and autologous stem cell transplantation provide more options for the treatment of elderly MCL patients.Novel drugs such as ibrutinib,ABT-199 and MLN8237 have shown encouraging results in preclinical trials which provide new hope for MCL patients.

Molecular cytogenetic pathogenesis of multiple myeloma: reports in the 55th ASH annual meeting / 白血病·淋巴瘤

The effect of molecular cytogenetics in pathogenesis of multiple myeloma (MM) is fundamental.The risk stratification system that based on molecular cytogenetics of MM is widely applicable in the prognosis of MM.The aberration of molecular cytogenetics is the most important marker of MM.At the same time,high-risk MM is associated with intra-clonal tumor heterogeneity and clonal evolution.The research of intra-clonal tumor heterogeneity and clonal evolution which based on the aberration of molecular cytogenetics will provide some additional information of the biology of MM and contribute to the optimum treatment for MM patients.

Research progress of monoclonal antibody in the treatment of adult acute lymphoblastic leukemia:reports from the 55th ASH annual meeting / 白血病·淋巴瘤

Humanized monoclonal antibody and CARs have became the focus of treatment in adult acute lymphoblastic leukemia (ALL) at the 55th ASH annual meeting.Treating with chemotherapy plus monoclonal antibody will get a develop effect in relapse/refractory adult ALL.More immune-pharmaceutics will be used in clinical trials.

Development of chronic myeloid leukemia in the 55th ASH annual meeting / 白血病·淋巴瘤

In patients with chronic myeloid leukemia (CML) in chronic phase who have achieved complete molecular remission on imatinib therapy,whether and when to discontinue the drug become a hot topic.The famous clinical trials of discontinue tyrosine kinase inhibitor (TKI) from France and Australia have demonstrated that long-term monotherapy with TKI is not curative in the majority of patients with CML.This has focused attention on strategies to eradicate residual disease in CML patients,which should result in cure and long-term treatment-free remission and leukemia-free survival.This review provides a brife summary of the 55th ASH annual meeting,focusing on the potential combination therapeutics in the era of TKI.

Report on recent discontinuation of tyrosine kinase inhibitors and alternative approaches to eradicating the chronic myeloid leukemia stem cells in the 55th ASH annual meeting / 白血病·淋巴瘤

Tyrosine kinase inhibitor (TKI) may significantly improve the treatment outcome in chronic myeloid leukemia (CML).It is the most frequent question about whether the patients with durable complete molecular response (CMR) can safely discontinue TKI treatment without relapse.This has focused attention on the strategies to eradicate residual CML cells,especially the CML stem cells,which should result in long term leukemia-free survival and permanent cure.Here,the progress on discontinuation of TKI therapy and alternative approaches to eradicating CML stem cells in the 55th ASH annual meeting is reviewed.

Progress of targeted therapy in diffuse large B-cell lymphoma: reports in the 55th ASH annual meeting / 白血病·淋巴瘤

With the further study on heterogeneity and molecular mechanism of diffuse large B-cell lymphoma (DLBCL),many novel targeted drugs have been developed,which also showed clinical benefit in early-stage studies.Therapeutic targets for DLBCL mainly include the surface antigens of B lymphocyte,B cell receptor signaling and the cellular microenvironment.Several new clinical trials about targeted therapy for DLBCL had been reported in the 55th ASH annual meeting,and the results were encouraging.The advances in this field will be summarized in this paper based on the new reports in the 55th ASH annual meeting.

Next generation of sequencing techniques guide the new progress of malignant blood diseases: reports in the 55th ASH annual meeting / 白血病·淋巴瘤

The biggest highlight in the 55th ASH annual meeting is a series of revolutionary progress in the field of diagnosis,pathogenesis,prediction,prognosis and minimal residue disease (MRD) of malignant blood diseases guiding with next generation of sequencing (NGS) techniques.This article will share the major contents of 7 papers using NGS techniques in the ASH annual meeting with domestic readers here.

T-cell therapy for viral infections after hematopoietic stem cell transplantation in the 55th ASH annual meeting / 白血病·淋巴瘤

Viral infections remain a major cause of morbidity in patients with immunodeficiency,such as recipients of hematopoietic stem cell transplantation (HSCT).Adoptive transfer of donor-derived virusspecific cytotoxic T lymphocytes is a strategy to restore virus-specific immunity to prevent or treat viral diseases and has been tested in the clinical setting for more than 20 years.The 55th ASH annual meeting reported lots of basic and clinical experience about CTL and summarized lots of trails to evaluate it,which will give us some new thought about the antivirus therapy after HSCT.

Advances in drug resistance mechanisms of multiple myeloma: reports from the 55th ASH annual meeting / 白血病·淋巴瘤

Multiple myloma represents the second most common blood cancer.Although new drugs emerge rapidly,multiple myeloma is still incurable.Drug resistance is one of the main causes of myeloma recurrence and relapse.In this paper,advances about the drug resistance mechanisms reported in the 55th ASH annual meeting are summarized.

Report on the research progress of Hodgkin lymphoma in the 55th ASH annual meeting / 白血病·淋巴瘤

The role of consolidating radiation therapy (RT) is very controversial in Hodgkin lymphoma (HL),especially in adolescents and young adults (AYA) HL.The key problem is how to achieve better therapeutic effect but bear less toxicities in chemotherapy and radiotherapy,which will be hopefully solved by clinical trials adopting interim positron emission tomography (iPET) scanning to guide therapy for HL.The new technologies including whole genome amplification (WGA),high-throughput gene sequencing (NGS) assay and genome-wide association study (GWAS) further illustrate abnormal signaling pathways in HL,such as NF-κB,JAK/STAT and PI3K pathway,which may provide new therapeutic targets for the disease.

A new era in the treatment of chronic lymphocytic leukemia and a part of lymphoma: reports in the 55th ASH annual meeting / 白血病·淋巴瘤

In this year,ASH annual meeting reported several new targeting therapeutic drugs for chronic lymphocytic leukemia (CLL) and a part of lymphoma.Those drugs focus on some key targets in the signal transductional pathway of B cells.Although these targeting drugs individually did not improve the complete remission rate of CLL,but several clinical trial results showed that the combination therapy of those drugs with other drugs significantly improved the efficacy of CLL,and prolonged the progression-free survival of CLL patients.Therefore,experts believe that combination therapy and sequential therapy will start a new era in the treatment of CLL.This paper will share only the latest advances in the treatment of CLL and parts of lymphoma with domestic readers.

New progress of allogeneic transplantation in myelodysplastic syndromes in the 55th ASH annual meeting / 白血病·淋巴瘤

New progress of myelodysplastic syndomes (MDS) in the 55th ASH annual meeting on allogeneic transplantation were reviewed.Even though the IPSS was developed mainly to determine the prognostic risk in newly diagnosed MDS patients,its predictive value concerning posttransplantation outcome has bcen confirmed in several studies.According to a decision model published a decade ago,patients with intermediate-2 or high-risk MDS by IPSS criteria should be considered for hematopoietic cell transplantation (HCT) at the time of diagnosis if an HLA-matched donor is available at this time.Given the age of most MDS patients,therapeutic interventions are per se often limited to rather nonintensive treatment approaches.This article reviews the current evidence for allogeneic HCT as a therapeutic option in the context of disease-specific characteristics and current available alternative treatments.

Novel therapy for Hodgkin lymphoma: reports in the 55th ASH annual meeting / 白血病·淋巴瘤

The 55th ASH annual meeting published a special report about Hodgkin lymphoma,which mainly contains management of early-stage Hodgkin lymphoma,novel targeted therapy and the optimal treatment of lymphocyte-predominant Hodgkin lymphoma.The therapy of Hodgkin lymphoma should be individualized,in order to improve both PFS and OS.More clinical trials should be designed on the target therapy and the treatment strategy of nodular lymphocyte-predominant Hodgkin lymphoma.

Diagnostic and therapeutic advances of high risk acute myeloid leukemia: reports from the 55th ASH annual meeting / 白血病·淋巴瘤

Acute myeloid leukemia (AML) is a group of heterogeneous diseases with diverse genetic abnormalities,variable responsiveness to therapy and prognosis.In recent years,a lot of information has become available regarding chromosome and gene mutations that occur in AML and their influence on prognosis.Improvements in the understanding of molecular biology of AML are critical for accurate diagnosis.risk stratification,monitoring of minimal residual disease and provides opportunities to develop targeted therapies and improve the clinical outcome.This article reviewed chromosome abnormalities and characteristic gene mutations,and discussed their clinical signiflcances and presented new drugs in clinical trials presented in the 55th ASH annual meeting.

New progress of myelodysplastic syndromes in the 55th ASH annual meeting / 白血病·淋巴瘤

New progress of myelodysplastic syndomes (MDS) in the 55th ASH annual meeting on 5-azacytidine and decitabine was reviewed.Except in older females,the survival time for a diagnosis of MDS in the United States does not seem to have substantially improved since the introduction of 5-azacytidine into the market in 2004.However,clinical and mechanism data suggest that judicious,mechanism-based application and optimization of these agents will have increasingly meaningful impact.Moreover,these drugs are exemplars of an inspiring alternative to the traditional cytotoxic paradigm and should pave the way for additional relatively nontoxic but broadly effective agents.

The hot topics of acute leukemia: reports in the 56th ASH annual meeting / 白血病·淋巴瘤

There are six main topics in acute leukemia at 56th (2014) American Society of Hematology annual meeting.The preleukemic cells were investigated in serial clinical trials.The application of High dose daunorbicin is the main topic in the area of acute myeloid leukemia (AML) chemotherapy.The application of tyrosine kinase inhibitors is the main topic in the area of target therapy of AML.The application of arsenic is the main topic in the area of acute promyelocytic leukemia (APL) treatment.AG221,an IDH2 inhibitor,and RG7388,MDM2 inhibitor,are promising target drugs for AML.CAR-T cellular immunotherapy is the hot topic in acute lymphoblastic leukemia (ALL).

Report on recent diagnosis and treatment of acute myeloid leukemia in the 54th ASH annul meeting / 白血病·淋巴瘤

Acute myeloid leukemia (AML) is the most commom hematologic malignancy in adults.There are so many highlights in the 54th ASH annual meeting in the field of mechanism,diagnosis and treatment of AML.Especially many studies have identified genes close related to AML development,clones related to relapse and molecular marker for prognosis using next-generation sequencing (NGS).Minimal residual disease (MRD) assay has been used for evaluation of therapy efficacy and posttherapy prognostication in AML treatment.The tyrosine kinase inhibitor targeting FLT3-ITD in this meeting is the best highlight in the treatment of refractory and relapsed AML.

Report on recent treatment of Hodgkin' s lymphoma in the 54th ASH annual meeting / 白血病·淋巴瘤

Early stage Hodgkin' s lymphoma (HL) is highly curable with abbreviated chemotherapy plus involved-filed radiotherapy or chemotherapy alone,but the role of radiation remains the subject of debate and disagreement.The management of recurrent or refractory HL remains challenging with limited effective treatments except high dose therapy with autologous stem cell transplantation.Emerging new safe and effective drugs such as brentuximab vedotin might promisingly improve the outcome of these patients in the future.The interim positron emission tomography (PET-i) scan has important prognostic value in patients with early and advanced stage HL,especially PET scan after 2 cycles.However,the PET-i guided treatment decisions are not currently recommended outside clinical trials.

Report on the application of high through-out technologies in lymphoma in the 54th ASH annual meeting / 白血病·淋巴瘤

Since the human gene code had been broken,the high through-out technologies based on genome-wide analysis have been well developed and extensively applied in cancers,which brought the innovation for the discovery of the pathogenesis and targeted drugs development of the cancer.After the successful application of these technologies in lymphomas,for example,gene expression profile in the diffuse large B cell lymphoma and next-generation sequencing technologies in hairy cell leukemia and Waldentr(o)m's macroglobulinemia,more and more researchers have tried to applied kinds of high through-out technologies in lymphomas,expecting to make improvements for the pathogenesis and prognosis of lymphomas.Many of the results had been reported in the 54th ASH annual meeting.