1,459 results match your criteria Clinical Hematology[Journal]


Regulatory T cells and CD20 B cells in pediatric very severe aplastic anemia: possible clinical markers for evaluating the therapeutic efficacy and prognosis.

Hematology 2018 Jul 11:1-5. Epub 2018 Jul 11.

a Department of Clinical Laboratory , The Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University , Guangzhou , People's Republic of China.

Objectives: To investigate the immune status of children with very severe aplastic anemia (VSAA), and evaluate the frequencies of CD20 B cells and Regulatory T cells (Tregs) as potential markers for evaluating the therapeutic efficacy and prognosis.

Methods: We systematically analyzed CD20 B cells and Tregs using Flow Cytometry in 36 children with VSAA (14 newly diagnosed cases and 22 cases in remission after therapy with HDIVIG + r-ATG + CSA).

Results: In newly diagnosed VSAA patients, the percentage of CD20 B cells was higher than that in healthy children (P < . Read More

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July 2018
1 Read

Coexisting driver mutations in MPN: clinical and molecular characteristics of a series of 11 patients.

Hematology 2018 Jul 11:1-8. Epub 2018 Jul 11.

d Department of Hematology , University Hospitals Leuven , Leuven , Belgium.

Objectives: CML, PV, ET and PMF are so called classical MPN with distinct clinical phenotypes. The discovery of the BCR-ABL1 translocation and mutations in driver genes JAK2, MPL and CALR has provided novel insights in their pathogenesis. While these mutations are thought to be mutually exclusive, rare cases of MPN with coexisting driver mutations have been reported. Read More

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Serum chitotriosidase: a circulating biomarker in polycythemia vera.

Hematology 2018 Jul 11:1-10. Epub 2018 Jul 11.

b Division of Hematology, Department of Internal Medicine , University Hospital Center Zagreb , Zagreb , Croatia.

Objectives: Serum chitotriosidase activity (CHIT1) is a biomarker of macrophage activation with an important role in inflammation-induced tissue remodeling and fibrosis. Macrophages have been described to play a crucial role in regulating pathological erythropoiesis in polycythemia vera (PV). The aim of this study was to evaluate CHIT1 in patients diagnosed with Philadelphia-negative myeloproliferative neoplasms (MPNs). Read More

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July 2018
1 Read

Epstein-Barr virus-associated hemophagocytic lymphohistiocytosis in adults and adolescents-a life-threatening disease: analysis of 133 cases from a single center.

Hematology 2018 Jun 29:1-7. Epub 2018 Jun 29.

a Department of Hematology , Beijing Friendship Hospital, Capital Medical University , Beijing , People's Republic of China.

Objectives: Epstein-Barr virus-associated hemophagocytic lymphohistiocytosis is the most common type of infection-associated HLH. Previous studies were focused on pediatric EBV-HLH patients, therefore there lack of adult data.

Method: We performed a retrospective analysis of 133 EBV-HLH patients (≥14 years old) in Beijing Friendship Hospital from March 2009 to April 2016 to evaluate the clinical manifestation and the effects and prognosis of existing regimens of EBV-HLH in adult and adolescents. Read More

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June 2018
1 Read

Hyperferritinemia: causes and significance in a general hospital.

Hematology 2018 Jun 19:1-6. Epub 2018 Jun 19.

c Center for Clinical Epidemiology , St. Luke's International Hospital , Tokyo , Japan.

Objective: To elucidate conditions which cause elevation of the serum ferritin, extent of the elevation in each condition, and clinical relevance of hyperferritinemia in general practice.

Methods: We retrospectively studied medical records of all patients who had at least one serum ferritin measurement above 500 μg L. Patients who had a marked elevation of the serum ferritin over 10,000 μg L were studied separately. Read More

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Acute myeloid leukemia carrying ETV6 mutations: biologic and clinical features.

Hematology 2018 Jun 12:1-5. Epub 2018 Jun 12.

a Department of Hematology and Oncology, Zhongda Hospital, Medical School , Southeast University , Nanjing , People's Republic of China.

Objectives: E26 transformation-specific variant 6 gene (ETV6) is one of the most consistently rearranged genes in acute leukaemia. It encodes a principal hematopoietic transcription factor.

Methods: We performed a systematic review focusing on the mechanisms responsible for etv6 acquisition, and its effect on the development of AML. Read More

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Synchronous dual hematological malignancies: new or underreported entity?

Hematology 2018 Jun 7:1-4. Epub 2018 Jun 7.

a Simcoe Muskoka Regional Cancer Program , Royal Victoria Regional Health Centre , Barrie , ON , Canada.

Background: Patients with a single hematological malignancy may be unexpectedly diagnosed with a clonally unrelated synchronous dual hematological malignancy (SDHM). The presence of a secondary hematological malignancy may be overlooked and only identified in situations presenting with discordant clinical or laboratory findings. Clinical management of these patients can be challenging, in part due to the relatively unknown etiopathology of SDHM and the impact of therapy on the secondary malignancy. Read More

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Predictive factors of daily opioid use and quality of life in adults with sickle cell disease.

Hematology 2018 May 31:1-8. Epub 2018 May 31.

a Medical Sciences Institute, BloodCenter of Wisconsin , Milwaukee , WI , USA.

Objectives: In adults with sickle cell disease (SCD), pain often necessitates opioid use. Few studies have examined the relationship between opioid use and health-related quality of life (HRQOL) in adults with SCD. We tested the hypothesis that higher doses of opioids are associated with worse HRQOL. Read More

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May 2018
1 Read

Screening of prognostic risk microRNAs for acute myeloid leukemia.

Hematology 2018 May 19:1-9. Epub 2018 May 19.

a Department of Hematology , The Second Affiliated Hospital of Harbin Medical University , Harbin , People's Republic of China.

Objectives: This study aimed to investigate the risk miRNAs (microRNAs) for AML (acute myeloid leukemia) prognosis and related regulatory mechanisms.

Methods: MiRNA and gene expression data, as well as clinical data of 176 patients were first downloaded from TCGA. Then miRNAs and genes significantly affecting the survival time based on KM survival curve were identified using Log Rank test. Read More

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May 2018
3 Reads

The clinical characteristics and therapy response of patients with acquired pure red cell aplasia.

Hematology 2018 May 12:1-7. Epub 2018 May 12.

a The Department of Hematology , General Hospital of Tianjin Medical University , Tianjin , People's Republic of China.

Objective: To summarize the clinical characteristics of acquired pure red cell aplasia (PRCA) patients diagnosed in our hospital in the last 10 years.

Method: The clinical features, immune state and treatment response of acquired PRCA patients diagnosed in our hospital from January 2007 to January 2017 were retrospectively analyzed.

Results: The results showed that thymoma (13. Read More

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May 2018
1 Read

Sequence typing of human adenoviruses isolated from Polish patients subjected to allogeneic hematopoietic stem cell transplantation - a single center experience.

Hematology 2018 Mar 28:1-6. Epub 2018 Mar 28.

a Department of Medical Microbiology , Medical University of Warsaw , Warsaw , Poland.

Purpose: Human adenoviruses (HAdV) from species A, B and C are commonly recognized as pathogens causing severe morbidity and mortality in hematopoietic stem cell transplant (HSCT) recipients. The purpose of the present study was to determine HAdV types responsible for viremia in HSCT recipients at a large tertiary hospital in Poland.

Methods: Analysis of partial nucleotide sequences of HAdV hexon gene was used to type 40 clinical isolates of HAdV obtained from 40 HSCT recipients. Read More

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March 2018
2 Reads

Health-related quality of life before and after hematopoietic stem cell transplant: evidence from a survey in Suzhou, China.

Hematology 2018 Mar 27:1-7. Epub 2018 Mar 27.

a Department of Nursing , The First Affiliated Hospital of Soochow University , Suzhou , Jiangsu , People's Republic of China.

Objectives: The aim of our longitudinal study was to explore changes in HRQOL over a 6-month period and to identify factors associated with the HRQOL of HSCT recipients.

Method: Our study comprised 191 HSCT patients; their data were collected before transplantation and at 30, 90, and 180 days posttransplantation. The Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) questionnaire was used to assess HRQOL. Read More

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March 2018
5 Reads

Comparative assessment of prophylactic transfusions of platelet concentrates obtained by the PRP or buffy-coat methods, in patients undergoing allogeneic hematopoietic stem cell transplantation.

Hematology 2018 Mar 27:1-7. Epub 2018 Mar 27.

a Servicio de Hematología y Oncología Médica, Hospital Universitario Morales Meseguer, Centro Regional de Hemodonación , Universidad de Murcia, CIBERER, IMIB-Arrixaca , Murcia , Spain.

Objectives: Whole blood-derived platelet concentrates can be obtained by the platelet-rich plasma (PRP-PCs) or the buffy-coat (BC-PCs) method. Few studies have shown that BC-PCs display lower in vitro platelet activation, but scarce information exists regarding transfusion efficacy. We have performed a retrospective study assessing platelet transfusion in patients undergoing allogeneic hematopoietic cell transplantation (AHCT) in our clinic, before and after the implementation of BC-PCs. Read More

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March 2018
2 Reads

Calreticulin mutation analysis in non-mutated Janus kinase 2 essential thrombocythemia patients in Chiang Mai University: analysis of three methods and clinical correlations.

Hematology 2018 Mar 9:1-7. Epub 2018 Mar 9.

d Department of Pathology, Faculty of Medicine , Chiang-Mai University , Chiang Mai , Thailand.

Objectives: The primary objective was to determine the prevalence of calreticulin (CALR) mutation in patients with non-JAK2V617F mutated essential thrombocythemia (ET). The secondary objectives were to evaluate the accuracy of CALR mutation analysis by high-resolution melting (HRM) analysis and real-time polymerase chain reaction (PCR) compared with DNA sequencing and to compare clinical characteristics of CALR mutated and JAK2V617F mutated ET.

Methods: This was a prospective cohort study involving ET patients registered at Chiang Mai University in the period September 2015-September 2017 who were aged more than 2 years, and did not harbor JAK2V617F mutation. Read More

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March 2018
1 Read

The prognostic impact of long noncoding RNA HOTAIR in leukemia and lymphoma: a meta-analysis.

Hematology 2018 Mar 7:1-8. Epub 2018 Mar 7.

a Department of Hematology , The First Affiliated Hospital of Xiamen University , Xiamen , People's Republic of China.

Objectives: Multiple studies have shown that the expression level of Homeobox (HOX) transcript antisense RNA (HOTAIR) has a correlation with the diagnosis and prognosis of acute leukemia (AL) and lymphoma. The prognostic significance of HOTAIR on AL and lymphoma has been controversial. Our study thus was performed to further reveal its prognostic value in leukemia and lymphoma. Read More

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March 2018
1 Read

Preliminary investigation about the expression of tubulin in platelets from patients with iron deficiency anemia and thrombocytosis.

Hematology 2018 Mar 2:1-9. Epub 2018 Mar 2.

a Division of Hematology , The Second Affiliated Hospital of Shantou University Medical College , Shantou , Guangdong Province , People's Republic of China.

Objective: In order to inquire into the pathogenesis of increased platelet counts in peripheral blood of patients with iron deficiency anemia (IDA), the phenomenon of thrombocytosis was confirmed, and then the expression of tubulin within platelets from IDA patients was investigated.

Methods: Peripheral blood samples were collected from 79 patients with IDA and were divided into 2 groups, group of IDA with normal platelet counts (34 cases), and group of IDA with increased platelet counts (thrombocytosis) (45 cases). Additionally, 45 peripheral blood samples from healthy volunteers were enrolled as a group of healthy controls. Read More

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March 2018
3 Reads

Renal involvement in paroxysmal nocturnal hemoglobinuria: an update on clinical features, pathophysiology and treatment.

Hematology 2018 Feb 28:1-9. Epub 2018 Feb 28.

e Department of Transfusion Service and Clinical Hemostasis , 'Saint Savvas' Oncology Hospital , Athens , Greece.

Objectives: The present review summarizes the available knowledge regarding acute and chronic kidney dysfunction in patients with paroxysmal nocturnal hemoglobinuria (PNH) focusing on its clinical features, pathophysiology and treatment.

Methods: A thorough PubMed search was performed using as main keywords: 'paroxysmal nocturnal hemoglobinuria', 'acute kidney injury', 'chronic kidney disease' and 'eculizumab'.

Results: PNH's etiopathogenesis is based on acquired mutations that lead to the reduction or absence of CD55 and CD59 complement regulators, which are responsible for some of the disease's major clinical features, like intravascular hemolysis, cytopenias and thrombosis. Read More

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February 2018
2 Reads

Clinical outcomes of myeloid/lymphoid neoplasms with fibroblast growth factor receptor-1 (FGFR1) rearrangement.

Hematology 2018 Feb 28:1-8. Epub 2018 Feb 28.

a Division of Hematology, Department of Medicine , Jichi Medical University , Tochigi , Japan.

Objective: Myeloid/lymphoid neoplasms with fibroblast growth factor receptor-1 (FGFR1) rearrangement are hematopoietic stem cell disorders with a poor prognosis, but no established standard therapy.

Methods: We experienced a patient with T-lymphoblastic lymphoma (LBL) associated with FGFR1 rearrangement who underwent cord blood transplantation, but died of pulmonary complication. We collected the clinical data of patients with FGFR1 rearrangement from the medical literature and analyzed 45 patients, including our patient. Read More

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February 2018
9 Reads

Mometasone furoate inhibits growth of acute leukemia cells in childhood by regulating PI3K signaling pathway.

Hematology 2018 Feb 8:1-8. Epub 2018 Feb 8.

a Department of Pediatrics , No. Four Hospital of Jinan , Jinan , Shandong 250000 , People's Republic of China.

Objectives: Acute lymphoblastic leukemia (ALL) is the most common cancer before the age of 15 years, seriously endangering the health of children. The main treatment for Childhood ALL was pharmacotherapy. But these drugs have many side effects and some of them could develop drug resistance quickly. Read More

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February 2018
5 Reads

Low expression of Mda-7/IL-24 and high expression of C-myb in tumour tissues are predictors of poor prognosis for Burkitt lymphoma patients.

Hematology 2018 Feb 8:1-8. Epub 2018 Feb 8.

b Research Center, The Fourth Hospital of Hebei Medical University , Shijiazhuang , People's Republic of China.

Objectives: Burkitt lymphoma is one of the most common types of haematopoietic malignancy in children and adolescents. Mda-7/IL-24 had been identified as a differentiation inducer of Burkitt lymphoma cells. Previous studies have revealed that knockdown of C-myb can also lead to the terminal differentiation of Burkitt lymphoma cells. Read More

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February 2018
4 Reads

Danazol increases T regulatory cells in patients with aplastic anemia.

Hematology 2018 Feb 8:1-5. Epub 2018 Feb 8.

b Department of Internal Medicine , Post Graduate Institute of Medical Education and Research (PGIMER) , Chandigarh , India.

Objectives: Danazol is an attenuated androgen and is used in the treatment of aplastic anemia (AA) in resource constraint settings. We chose to study the role of CD4+ CD25 CD127 FoxP3+ T regulatory cells (T-regs) in the pathophysiology of AA and their response to treatment with Danazol alone or in combination with immunosuppressive treatment (IST).

Methods: T-regs' percentages of 25 acquired idiopathic AA patients and 25 healthy controls who completed study protocol were analyzed by performing multicolor flowcytometry on peripheral blood samples. Read More

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February 2018
4 Reads

Dyspareunia is associated with chronic pain in premenopausal women with sickle cell disease.

Hematology 2018 Jan 29:1-6. Epub 2018 Jan 29.

a Medical Sciences Institute, Blood Center of Wisconsin , Milwaukee , WI , USA.

Objectives: Pain is common in women with sickle cell disease (SCD), but the prevalence of dyspareunia in this unique patient population is unknown. In this study, we sought to determine whether chronic pain is associated with an increased prevalence of dyspareunia in premenopausal women with SCD.

Methods: A cross-sectional study of premenopausal women with SCD was systematically assessed for symptoms of dyspareunia and chronic pain using a standard questionnaire. Read More

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January 2018
3 Reads

Adjuvant radiotherapy in patients with diffuse large B-cell lymphoma in advanced stage (III/IV) improves the outcome in the rituximab era.

Hematology 2018 Jan 17:1-5. Epub 2018 Jan 17.

b Department of Hematology , Oncology Hospital National Medical Center, IMSS , Mexico D.F. , Mexico.

Objectives: To assess the efficacy and toxicity of adjuvant radiotherapy (RT) in patients with diffuse large B-cell lymphoma (DLBCL) and nodal bulky disease, on complete response, after six cycles of RCHOP (rituximab, cyclophosphamide, doxorubicin, vincristine and prednisone), we began an open-label clinical trial in a large cohort with longer follow-up to evaluate the outcome measured from progression-free survival (PFS) and overall survival (OS).

Patients And Methods: Between 2006 and 2010, 258 consecutive patients with DLBCL and nodal bulky disease (tumor mass >10 cm) were randomly assigned to receive either RT (involved field, 30 Gy) (127 patients) or no (control group) (131 patients).

Results: The actuarial curves at 5 years of PFS were 87% (95% confidence interval (CI): 72-97%) in the RT group, which was significantly different from the control group value of 45% (95% CI: 34-60%) (p < 0. Read More

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January 2018
2 Reads

Thrombopoietin and its receptor expression in pediatric patients with chronic immune thrombocytopenia.

Hematology 2018 Aug 9;23(7):433-438. Epub 2018 Jan 9.

a Department of Hematology and Oncology , Shanghai Children's Hospital, Shanghai Jiao Tong University , Shanghai , People's Republic of China.

Objectives: Chronic immune thrombocytopenia (cITP) is common in children. However, the pathogenesis has not been fully elucidated. This study aimed to determine whether thrombopoietin (TPO) and its receptor c-mannosylation of the TPO receptor (c-Mpl) have an impact on childhood cITP. Read More

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August 2018
5 Reads

The potential association of CMV-specific CD8+ T lymphocyte reconstitution with the risk of CMV reactivation and persistency in post allogeneic stem cell transplant patients.

Hematology 2018 Jan 9:1-7. Epub 2018 Jan 9.

d Clinical and Chemical Pathology, Egyptian Anti Doping Lab , International Medical Center , Cairo , Egypt.

Objectives: development of cytomegalovirus (CMV)-specific CD8+ T cell response is crucial in preventing symptomatic CMV infection specially, in stem cell transplant (SCT) patients. The aim of this study was to evaluate CMV-specific CD8+ T cell reconstitution in allogeneic SCT recipients and to study the possible association between CMV-specific CD8+ T cell recovery with protection from CMV reactivation and persistency.

Methods: Human leuKocyte antigen (HLA)-tetramers were used for CMV-specific CD8+ cell quantitation by Flow cytometry in twenty post-allogeneic SCT patients. Read More

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January 2018
3 Reads

Is there a difference in phenotype between males and females with non-transfusion-dependent thalassemia? A cross-sectional evaluation.

Hematology 2018 Jan 5:1-4. Epub 2018 Jan 5.

c UOSD Malattie Rare del Globulo Rosso , AORN A. Cardarelli , Naples , Italy.

Objectives: Non-transfusion-dependent thalassemia includes a variety of phenotypes and genotypes that rarely require regular transfusions. However, these patients can experience a wide range of complications. The objective of this retrospective study was to verify whether there is a significant difference in non-transfusion-dependent thalassemia-related complications and treatment among males and females. Read More

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January 2018
6 Reads

Eltrombopag in the management of aplastic anaemia: real-world experience in a non-trial setting.

Hematology 2018 Aug 5;23(7):399-404. Epub 2018 Jan 5.

a Department of Medicine , Queen Mary Hospital , Hong Kong.

Objective: The thrombopoietin mimetic eltrombopag has been used in clinical trials for the frontline and salvage treatment of aplastic anaemia (AA). Eltrombopag was investigated in AA patients on a non-trial all-comer basis.

Methods: Consecutive newly diagnosed and relapsed/refractory AA patients were treated with eltrombopag. Read More

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August 2018
4 Reads

The clinical significance of FLT3 ITD mutation on the prognosis of adult acute promyelocytic leukemia.

Hematology 2018 Aug 18;23(7):379-384. Epub 2017 Dec 18.

a Department of Laboratory Diagnosis , Shidong Hospital of Yangpu Distric , Shanghai , People's Republic of China.

Background And Aims: To explore the relationship between FLT3 (encoding Fms related tyrosine kinase 3) internal tandem duplication (ITD) mutations with the prognosis of acute promyelocytic leukemia. The PubMed database, the Cochrane Library, conference proceedings, the EMBASE databases, and references of published trials and review articles were searched. Two reviewers independently assessed the quality of the trials and extracted the data. Read More

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August 2018
7 Reads

Understanding of the significance and health implications of asplenia in a cohort of patients with haemaglobinopathy: possible benefits of a spleen registry.

Hematology 2017 Dec 13:1-5. Epub 2017 Dec 13.

a Faculty of Medicine, Nursing and Health Sciences , Monash University , Clayton , Australia.

Objectives: Asplenia and hyposplenism carry a significant risk of ongoing morbidity and mortality which can be reduced by education, vaccination and antibiotic use. We aimed to assess education and other methods of prevention in a cohort of patients with haemoglobinopathy in a tertiary referral centre, which also had access to a post-splenectomy registry created to reduce post-splenectomy infection risk.

Methods: A standardized questionnaire was used on patients who attended the service for regular therapy. Read More

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December 2017
3 Reads

Inferior vena cava filter use and patient safety: legacy or science?

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):686-692

Thromboembolism Program, Sunnybrook Health Sciences Centre, Toronto, ON, Canada.

There has been a dramatic increase in vena cava filter (VCF) use over the past 20 years in the absence of evidence that filters provide a net patient benefit or are required in most cases. This increase is largely attributable to the availability of retrievable filters and expanded indications, particularly as primary prophylaxis in patients thought to be at high risk of pulmonary embolism. Substantial variability in VCF use, unrelated to patient clinical factors, has been shown between hospitals, from region to region, and among various countries. Read More

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December 2017
6 Reads

Assessing thrombocytopenia in the intensive care unit: the past, present, and future.

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):660-666

Division of Critical Care, Department of Internal Medicine, University of Manitoba, Winnipeg, MB, Canada; and.

Thrombocytopenia is common among patients admitted to the intensive care unit (ICU). Multiple pathophysiological mechanisms may contribute, including thrombin-mediated platelet activation, dilution, hemophagocytosis, extracellular histones, ADAMTS13 deficiency, and complement activation. From the clinical perspective, the development of thrombocytopenia in the ICU usually indicates serious organ system derangement and physiologic decompensation rather than a primary hematologic disorder. Read More

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December 2017
2 Reads

Plasma exchange in thrombotic microangiopathies (TMAs) other than thrombotic thrombocytopenic purpura (TTP).

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):632-638

Therapeutic Apheresis Treatment Unit, Division of Transfusion Medicine, Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, MN.

Thrombotic microangiopathies (TMAs) are a diverse group of disorders that are characterized by common clinical and laboratory features. The most commonly thought-of TMA is thrombotic thrombocytopenic purpura (TTP). Because of the marked improvement in patient mortality associated with the use of therapeutic plasma exchange (TPE) in TTP, this therapy has been applied to all of the TMAs. Read More

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December 2017
5 Reads

Developing T-cell therapies for lymphoma without receptor engineering.

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):622-631

Center for Cancer and Immunology Research, Children's National Health System, Washington, DC; and.

T-cell therapy has emerged from the bench for the treatment of patients with lymphoma. Responses to T-cell therapeutics are regulated by multiple factors, including the patient's immune system status and disease stage. Outside of engineering of chimeric antigen receptors and artificial T-cell receptors, T-cell therapy can be mediated by ex vivo expansion of antigen-specific T cells targeting viral and/or nonviral tumor-associated antigens. Read More

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December 2017
3 Reads

Harnessing the power of the immune system in non-Hodgkin lymphoma: immunomodulators, checkpoint inhibitors, and beyond.

Authors:
Stephen M Ansell

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):618-621

Mayo Clinic, Rochester, MN.

Non-Hodgkin lymphoma is a malignancy of B lymphocytes that typically infiltrate sites of disease, including the lymph nodes, spleen, and bone marrow. Beyond the presence of malignant cells, many immune cells are also present within the tumor microenvironment. Although these immune cells have the potential to regulate the growth of malignant B cells, intratumoral immune cells are unable to eradicate lymphoma cells and most patients with lymphoma have clinical evidence of disease progression. Read More

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December 2017
4 Reads

Novel alternate hemostatic agents for patients with inhibitors: beyond bypass therapy.

Authors:
Margaret V Ragni

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):605-609

Department of Medicine, Division Hematology/Oncology, University of Pittsburgh, and Hemophilia Center of Western Pennsylvania, Pittsburgh, PA.

Inhibitor formation is among the most severe complications of hemophilia treatment. With a cumulative incidence of ∼30% in those with severe hemophilia A and ∼3% in those with severe hemophilia B, inhibitors are caused by a T-cell response directed against infused coagulation factor; these inhibitors neutralize factor VIII or IX activity and disrupt normal hemostasis. Inhibitor patients become unresponsive to standard factor treatment and, as an alternative, use bypass treatment (eg, recombinant factor VIIa or factor VIII inhibitor bypass activity). Read More

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December 2017
3 Reads

Using pharmacokinetics to individualize hemophilia therapy.

Authors:
Alfonso Iorio

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):595-604

Department of Health Research, Methods, Evidence, and Impact, and.

Prevention and treatment of bleeding in hemophilia requires that plasma clotting factor activity of the replaced factor exceeds a defined target level. Most clinical decisions in hemophilia are based on implicit or explicit application of pharmacokinetic measures. The large interindividual variability in pharmacokinetics of factor concentrates suggests that relying on the average pharmacokinetic characteristics of factor concentrates would not allow optimizing the treatment of individual patients; for example, adjusting the frequency of infusions and targeting a specific clotting factor activity level on a case-by-case basis. Read More

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December 2017
16 Reads

Hemophilia gene therapy comes of age.

Authors:
Lindsey A George

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):587-594

Division of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA; and.

Concurrent with the development of recombinant factor replacement products, the characterization of the and genes over 3 decades ago allowed for the development of recombinant factor products and made the hemophilias a target disease for gene transfer. The progress of hemophilia gene therapy has been announced in 3 American Society of Hematology scientific plenary sessions, including the first "cure" in a large animal model of hemophilia B in 1998, first in human sustained vector-derived factor IX activity in 2011, and our clinical trial results reporting sustained vector-derived factor IX activity well into the mild or normal range in 2016. This progression to clinically meaningful success combined with numerous ongoing recombinant adeno-associated virus (rAAV)-mediated hemophilia gene transfer clinical trials suggest that the goal of gene therapy to alter the paradigm of hemophilia care may soon be realized. Read More

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December 2017
3 Reads

Secondary CNS relapse in diffuse large B-cell lymphoma: defining high-risk patients and optimization of prophylaxis strategies.

Authors:
Kerry J Savage

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):578-586

Department of Medical Oncology, British Columbia Cancer Agency, Vancouver, BC, Canada.

Despite improvement in survival in diffuse large B-cell lymphoma (DLBCL) with the introduction of rituximab, central nervous system (CNS) relapse continues to represent a clinical challenge. A number of studies have evaluated clinical risk factors in an attempt to identify high-risk patients to direct CNS staging investigations and consider prophylaxis strategies. The CNS International Prognostic Index is a robust and reproducible risk model that can identity patients at high risk of CNS relapse, but its specificity remains limited. Read More

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December 2017
5 Reads

Biology of CNS lymphoma and the potential of novel agents.

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):556-564

Division of Hematology/Oncology, University of California, San Francisco, San Francisco, CA.

Primary and secondary CNS lymphomas are aggressive brain tumors that pose an immense challenge to define in terms of molecular pathogenesis, as well as to effectively treat. During the past 10 years improvements in survival have been achieved with the implementation of anti-CD20 immunotherapy and optimization of dose-intensive consolidation strategies. The applications of whole-exome sequencing, comparative genomic hybridization, transcriptional profiling, and examination of the tumor microenvironment, particularly in the context of clinical investigation, provide insights that create a roadmap for the development and implementation of novel targeted agents for this disease. Read More

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December 2017
3 Reads

Pain-measurement tools in sickle cell disease: where are we now?

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):534-541

Section of Pediatric Hematology/Oncology, Medical College of Wisconsin, Milwaukee, WI; and.

Pain is a complex multidimensional experience and the most common morbidity in patients with sickle cell disease (SCD). Tools to assess pain can be of use not only to guide pain treatment but also to provide insight into underlying pain neurobiology. Mechanisms of pain in SCD are multifactorial and are not completely elucidated. Read More

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December 2017
7 Reads

Optimizing the care model for an uncomplicated acute pain episode in sickle cell disease.

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):525-533

Departments of Haematology and Paediatric Haematology, Royal London Hospital, Bart's Health National Health Service Trust, London, United Kingdom.

The pathophysiology, clinical presentation, and natural history of acute pain in sickle cell disease are unique and require a disease-centered approach that also applies general principles of acute and chronic pain management. The majority of acute pain episodes are managed at home without the need to access health care. The long-term consequences of poorly treated acute pain include chronic pain, adverse effects of chronic opioid usage, psychological maladjustment, poor quality of life, and excessive health care utilization. Read More

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December 2017
2 Reads

Emerging options in multiple myeloma: targeted, immune, and epigenetic therapies.

Authors:
Shaji Kumar

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):518-524

Division of Hematology and Internal Medicine, Mayo Clinic, Rochester, MN.

Considerable progress has been made in the treatment of multiple myeloma in the past decade with median survival for the disease improving significantly. This has come through a combination of better understanding of the disease biology and coordinated research into new treatment approaches including better supportive care. However, patients eventually become refractory to available treatments and succumb to the disease, highlighting the need to develop new treatment approaches. Read More

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December 2017
2 Reads

Management of multiple myeloma in the relapsed/refractory patient.

Authors:
Pieter Sonneveld

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):508-517

Department of Hematology, Erasmus Medical Center Cancer Institute, Rotterdam, The Netherlands.

The approach to the patient with relapsed or relapsed/refractory multiple myeloma requires a careful evaluation of the results of previous treatments, the toxicities associated with it, and an assessment of prognostic factors. The majority of patients will have received prior therapy with drug combinations, including a proteasome inhibitor and an immune-modulatory agent. It is the physician's task to choose the right moment for the start of therapy and decide with the patient which goals need to be achieved. Read More

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December 2017
8 Reads

Management of multiple myeloma in the newly diagnosed patient.

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):498-507

Clínica Universidad de Navarra, Centro de Investigación Médica Aplicada, Navarra, Spain.

Multiple myeloma is the second most frequent hematological disease. The introduction of melphalan as high-dose therapy followed by autologous hematopoietic cell transplantation (HDT/ASCT) for young patients and the availability of novel agents for young and elderly patients with multiple myeloma have dramatically changed the perspective of treatment. However, further research is necessary if we want definitively to cure the disease. Read More

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December 2017
4 Reads

What are the current treatment approaches for patients with polycythemia vera and essential thrombocythemia?

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):480-488

Center of Research and Innovation of Myeloproliferative Neoplasms (CRIMM), Careggi University Hospital/University of Florence, Florence, Italy.

Polycythemia vera (PV) and essential thrombocythemia (ET) are chronic myeloproliferative neoplasms that are characterized by thrombohemorrhagic complications, symptom burden, and impaired survival mainly due to thrombosis, progression to myelofibrosis, and transformation to acute leukemia. In this manuscript, we will review the most recent changes in diagnostic criteria, the improvements in risk stratification, and the "state of the art" in the daily management of these disorders. The role of conventional therapies and novel agents, interferon α and the JAK2 inhibitor ruxolitinib, is critically discussed based on the results of a few basic randomized clinical studies. Read More

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December 2017
23 Reads

Myeloproliferative neoplasms: from origins to outcomes.

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):470-479

Department of Haematology, Cambridge Institute for Medical Research and Wellcome Trust/MRC Stem Cell Institute, University of Cambridge, United Kingdom; and.

Substantial progress has been made in our understanding of the pathogenetic basis of myeloproliferative neoplasms. The discovery of mutations in over a decade ago heralded a new age for patient care as a consequence of improved diagnosis and the development of therapeutic JAK inhibitors. The more recent identification of mutations in calreticulin brought with it a sense of completeness, with most patients with myeloproliferative neoplasm now having a biological basis for their excessive myeloproliferation. Read More

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December 2017
4 Reads

Incorporating novel approaches in the management of MDS beyond conventional hypomethylating agents.

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):460-469

Department of Medicine, Section of Hematology/Oncology, The University of Chicago, Chicago, IL; and The University of Chicago Comprehensive Cancer Center, Chicago, IL.

In the last decade, the treatment of higher-risk myelodysplastic syndromes (MDS) has revolved around the azanucleosides, azacitidine and decitabine, which at lower doses are postulated to work predominantly via their effects on inhibition of DNA methyltransferases and consequent DNA hypomethylation. For patients who relapse after, or do not respond to, hypomethylating agent therapy, the outcome is dismal, and new agents and approaches that have the potential to alter the natural history of these diseases are desperately needed. Allogeneic stem cell transplant is the only known potentially curative approach in MDS, but its applicability has been limited by the advanced age of patients and attendant comorbidities. Read More

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December 2017
3 Reads

Current treatment algorithm for the management of lower-risk MDS.

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):453-459

Department of Hematology, Oncology and Palliative Care, Marien Hospital Düsseldorf, Düsseldorf, Germany.

Lower risk myelodysplastic syndromes (MDS), defined as MDS with a Revised International Prognostic Scoring System score ≤3.5 points, will remain a challenging entity in 2018. Supportive care continues to be the linchpin of treatment, although the options to reduce transfusion needs are broadening. Read More

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December 2017
2 Reads

Uncoding the genetic heterogeneity of myelodysplastic syndrome.

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):447-452

Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA.

Myelodysplastic syndrome (MDS) is a clinically heterogeneous disease characterized by functional impairment of hematopoiesis and abnormal bone marrow morphology. The type and severity of hematopoietic dysfunction in MDS are highly variable, and the kinetics of disease progression are difficult to predict. Genomic studies have shown that MDS is typically driven by a multistep somatic genetic process affecting a core set of genes. Read More

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December 2017
3 Reads

Chronic organ failure in adult sickle cell disease.

Hematology Am Soc Hematol Educ Program 2017 12;2017(1):435-439

UCSF Benioff Children's Hospital Oakland, Oakland, CA.

Sickle cell disease is now a chronic adult illness characterized by progressive multiorgan failure, particularly involving the brain and kidney. The etiology is multifactorial; it includes hemolysis and nitric oxide deficiency. As patients age, most experience neurologic insult. Read More

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December 2017
6 Reads