Abstract:
DISEASE OVERVIEW:Primary myelofibrosis (PMF) is a myeloproliferative neoplasm (MPN) characterized by stem cell-derived clonal myeloproliferation that is often but not always accompanied by JAK2, CALR, or MPL mutations; additional disease features include bone marrow stromal reaction including reticulin fibrosis, abnormal cytokine expression, anemia, hepatosplenomegaly, extramedullary hematopoiesis (EMH), constitutional symptoms, cachexia, leukemic progression, and shortened survival. DIAGNOSIS:Diagnosis of PMF is based on bone marrow morphology. Presence of JAK2, CALR, or MPL mutation, expected in ∼ 90% of the patients, is supportive but not essential for diagnosis. The revised 2016 World Health Organization (WHO) classification system distinguishes "prefibrotic" from "overtly fibrotic" PMF; the former might mimic ET in its presentation and it is prognostically relevant to distinguish the two. RISK STRATIFICATION:Two new prognostic systems for PMF have recently been introduced: GIPSS (genetically inspired prognostic scoring system) and MIPSS70+ version 2.0 (mutation- and karyotype-enhanced international prognostic scoring system). GIPSS is based exclusively on mutations and karyotype. MIPSS70+ version 2.0 utilizes both genetic and clinical risk factors. GIPSS features four and MIPSS70+ version 2.0 five risk categories. MIPSS70+ version 2.0 requires an online score calculator (http://www.mipss70score.it) while GIPPS offers a lower complexity prognostic tool. RISK-ADAPTED THERAPY:Observation alone is advised for MIPSS70+ version 2.0 "low" and "very low" risk disease (estimated 10-year survival 56%-92%); allogeneic stem cell transplant is the preferred treatment of choice for "very high" and "high" risk disease (estimated 10-year survival 0-13%); treatment-requiring patients with intermediate-risk disease (estimated 10-year survival 30%) are best served by participating in clinical trials. All other treatment approaches, including the use of JAK2 inhibitors, are mostly palliative and should not be used in the absence of clear treatment indications. Conventional treatment for anemia includes androgens, prednisone, thalidomide and danazol, for symptomatic splenomegaly hydroxyurea and ruxolitinib and for constitutional symptoms ruxolitinib. Splenectomy is considered for drug-refractory splenomegaly and involved field radiotherapy for nonhepatosplenic EMH and extremity bone pain.
journal_name
Am J Hematoljournal_title
American journal of hematologyauthors
Tefferi Adoi
10.1002/ajh.25230subject
Has Abstractpub_date
2018-12-01 00:00:00pages
1551-1560issue
12eissn
0361-8609issn
1096-8652journal_volume
93pub_type
杂志文章abstract::Familial and metachronous aggregations of malignant lymphoma are well-documented, but the molecular basis of a predisposition for development of lymphoma is as yet unclear. Malignant lymphomas have been described as part of the spectrum of neoplasias in Li-Fraumeni syndrome (LFS), which is associated with constitution...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/(sici)1096-8652(199911)62:3<144::aid-ajh3>
更新日期:1999-11-01 00:00:00
abstract::This paper describes, for the first time, the utilization of nebulized morphine in the management of severe chest pain in two young adult African-American patients who suffered from generalized acute sickle cell painful episodes. While hospitalized, both patients developed new sharp chest wall pain, and were treated w...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.20064
更新日期:2004-06-01 00:00:00
abstract::Platelet-rich plasma were treated with increasing concentrations of vitamin E (alpha-tocopherol). Washed platelets were exposed to oxidized low density lipoprotein (LDL) and examined by aggregometry and electron microscopy. The treatment of washed platelets by oxidized LDL induced morphological signs of activation lik...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/(sici)1096-8652(199903)60:3<242::aid-ajh15
更新日期:1999-03-01 00:00:00
abstract::Chuvash polycythemia results from a homozygous 598C>T mutation in exon 3 of the von Hippel-Lindau (VHL) gene. This disrupts the normoxia pathway for degrading hypoxia inducible factor (HIF)-1alpha and HIF-2alpha causing altered expression of HIF-1 and HIF-2 inducible genes. As hypoxia induces expression of pro-inflamm...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.21327
更新日期:2009-02-01 00:00:00
abstract::Retroviral-mediated gene transfer into hematopoietic stem cells provides the only means of stable transduction of these cells and their progeny for use with a variety of potentially therapeutic genes. Expression of the Moloney amphotropic retroviral receptor-pit-2 or GLVR-2-is critical to the recognition and entry of ...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/(sici)1096-8652(199908)61:4<243::aid-ajh4>
更新日期:1999-08-01 00:00:00
abstract::The human spleen normally retains about one-third of the body's platelets in an exchangeable pool which can be released into the circulation by alpha-adrenergic stimulation. Some previous investigators concluded that the splenic platelet population was enriched in a subpopulation of large, young, dense platelets (mega...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.2830340302
更新日期:1990-07-01 00:00:00
abstract::The purpose of the study was to assess consensus and interobserver agreement among an international panel of six hematopathologists regarding characterization and reproducibility of bone marrow (BM) histologic features used to diagnose early stage myeloproliferative neoplasms, in particular differentiation of so-calle...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.24837
更新日期:2017-10-01 00:00:00
abstract::In patients with light-chain myeloma or primary AL-amyloidosis, 24-hr light-chain excretion in the urine is considered an essential marker of the tumor mass. However, 24-hr urine collection and analysis may be cumbersome and prone to inaccuracy. Recently, a sensitive immunonephelometric assay for immunoglobulin free l...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.20007
更新日期:2004-04-01 00:00:00
abstract::In HIV-positive patients with non-Hodgkin lymphoma (NHL), no benefit of adding rituximab to chemotherapy was seen in a randomized controlled trial (RCT). We performed a meta-analysis of prospective studies to ascertain outcomes in HIV-positive NHL patients treated with chemotherapy (chemo) versus rituximab and chemo (...
journal_title:American journal of hematology
pub_type: 杂志文章,meta分析
doi:10.1002/ajh.22275
更新日期:2012-03-01 00:00:00
abstract::We report the case of a 64-year-old woman with plasma cytoid lymphoma of the ileum and gastric wall. Serum protein electrophoresis and immunoelectrophoresis showed a triclonal IgG kappa, IgA kappa, and IgM kappa gammopathy. Biopsy of the tumor revealed three different cell types, which were stained with antibodies spe...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.2830420213
更新日期:1993-02-01 00:00:00
abstract::Intravenous (IV) granulocyte colony stimulating factor (G-CSF) might be safer and more convenient than subcutaneous (SC) administration to hospitalized hemato-oncological patients receiving chemotherapy. To compare IV vs. SC G-CSF administration, we conducted a randomized, open-label trial. We included inpatients rece...
journal_title:American journal of hematology
pub_type: 杂志文章,随机对照试验
doi:10.1002/ajh.23622
更新日期:2014-03-01 00:00:00
abstract::We performed a laparoscopic splenectomy (LS) in 60 patients (age 9-83, 45 females) with idiopathic thrombocytopenic purpura (ITP) who did not achieve sustained remission on steroid therapy. Using a modified procedure, the mean duration of LS was 78 min (range 25-240 min) and surgery was associated with only 5% major a...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/(sici)1096-8652(200001)63:1<7::aid-ajh2>3.
更新日期:2000-01-01 00:00:00
abstract::Clinical management of lymphoplasmacytic lymphoma (LPL)/Waldenström macroglobulinemia (WM) has changed considerably over recent years, reflected in the use of new therapeutic agents (purine analogs, monoclonal antibodies, thalidomide- and bortezomib-based therapies). No population-based studies and few randomized tria...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.23351
更新日期:2013-01-01 00:00:00
abstract::Hemoglobin Lyon-Bron was found in two members of a family of German ascent presenting with a moderate normocytic anemia. In this alpha 2 globin variant, the N-terminal valine of the chain was replaced by an alanine. Electrospray mass spectrometry of the alpha chain showed that, as normally, the initiator methionine wa...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.10051
更新日期:2002-03-01 00:00:00
abstract::Cyclooxygenases (COX) are key enzymes in the conversion of arachidonic acid to prostaglandins. Several studies have shown a relation between angiogenesis and COX-2 expression. Elevated expression of cyclooxygenase-2 (COX-2), however, has not been reported in multiple myeloma (MM) in the literature. The aim of this stu...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.20460
更新日期:2005-11-01 00:00:00
abstract::Post-transfusion purpura (PTP) with severe thrombocytopenia occurred eight days after transfusion in a 28-year-old woman and responded to treatment with prednisone and plasma exchange. In contrast to nearly all previously studied cases of PTP, the patient's platelets were PlA1-positive and anti-PlA1 antibody could not...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.2830210110
更新日期:1986-01-01 00:00:00
abstract::We analyzed the genetic defects of 21 unrelated patients with venous thrombosis in whom hereditary protein C deficiency was diagnosed. Eleven mutations were detected in 18 families, while no mutation was detectable in the other three families. Among these mutations, a common genetic mutation of protein C (PROC) gene r...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.20043
更新日期:2004-05-01 00:00:00
abstract::Bacterial and protozoal infections can cause thrombocytopenia and may mimic idiopathic thrombocytopenic purpura (ITP). Brucella species and Toxoplasma are among the infectious agents with protean clinical manifestations which may induce immune thrombocytopenia. In rare cases, thrombocytopenia can be severe and may res...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.10382
更新日期:2003-09-01 00:00:00
abstract::Seventeen adults received the antifibrinolytic drug tranexamic acid during cardiac surgery utilizing extracorporeal circulation (ECC). In 8 patients, drug administration began prior to skin incision (pre-ECC); infusions commenced after ECC and protamine administration in another 9 patients (post-ECC). Compared with th...
journal_title:American journal of hematology
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1002/ajh.2830380208
更新日期:1991-10-01 00:00:00
abstract::We describe a 23-year-old retired military officer who was immunocompetent but diagnosed with hemophagocytic syndrome (HPS) by Plasmodium vivax infection. Initially, the patient was suspected to have toxic hepatitis related to heavy drinking. But abnormal hematologic findings required a further bone marrow examination...
journal_title:American journal of hematology
pub_type: 杂志文章,评审
doi:10.1002/ajh.10390
更新日期:2003-10-01 00:00:00
abstract::A 49-year-old Caucasian woman presented with features suggestive of thrombotic microangiopathy (TMA). She did not respond to treatment with repeated plasma exchange and corticosteroids. A bone marrow biopsy revealed presence of metastatic carcinoma. A limited autopsy revealed presence of breast cancer with rib metasta...
journal_title:American journal of hematology
pub_type: 杂志文章,评审
doi:10.1002/ajh.20783
更新日期:2007-04-01 00:00:00
abstract::Lentiviral modification combined with ex vivo erythroid differentiation was used to stably inhibit RhAG expression, a critical component of the Rh(rhesus) membrane complex defective in the Rh(null) syndrome. The cultured red cells generated recapitulate the major alterations of native Rh(null) cells regarding antigen ...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.23414
更新日期:2013-05-01 00:00:00
abstract::Quantitative changes of hemostatic molecular markers were studied in patients with nephrotic syndrome. The plasma levels of fibrinopeptide A (FPA), thrombin-antithrombin III complex (TAT), products of thrombin activation, and fragment F1 + 2 (F1 + 2), a product of prothrombin activation, were measured by enzyme immuno...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.2830440411
更新日期:1993-12-01 00:00:00
abstract::We molecularly characterized sixty-seven cases of Hb H disease by the polymerase chain reaction. The strategy depends on amplifying the alpha-thalassemia-1 (alpha-thal-1) gene by primers flanking the breakpoint and sequence differences of the 3' end of the alpha-globin gene and the nonhomologous elements I, II, and II...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.2830450405
更新日期:1994-04-01 00:00:00
abstract::Using an in vitro system to evaluate the simultaneous use of two drugs, we previously have confirmed the synergism of vincristine and prednisolone cytotoxicity against lymphoid cells. Experiments were now carried out to determine whether other steroid hormones can be substituted for prednisolone. Partial or complete p...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.2830050405
更新日期:1978-01-01 00:00:00
abstract::Eighteen patients with acute nonlymphocytic leukemia (ANLL), aged 17-73 years, were treated with high-dose cytosine arabinoside (HD-Ara-C) using 3 g/m2 IV q 12 hours X 12 doses. Seven patients were treated for relapse and four (57%) obtained a complete remission with a median duration of 19.5 weeks. In nine patients, ...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.2830160108
更新日期:1984-01-01 00:00:00
abstract::The acquired JAK2 V617F mutation is common in patients with myeloproliferative neoplasms. We previously showed that JAK2 V617F is also found in coronary patients, most of them affected by coronary atherosclerosis. Peripheral arterial disease (PAD) is another important manifestation of atherosclerosis. However, prevale...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.23874
更新日期:2015-01-01 00:00:00
abstract::A 1750 base pair cDNA to human terminal deoxynucleotidyl transferase (TdT) has been cloned. This cDNA detects a dominant 2200 base pair messenger RNA species in normal and leukemic cells synthesizing the enzyme. A quantitative dot blot assay was utilized to survey a number of clinical samples from patients with TdT po...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.2830250305
更新日期:1987-07-01 00:00:00
abstract::Acquired pure megakaryocytic aplasia is a rare disorder defined by severe thrombocytopenia with no other hematologic abnormalities and absent, or severely decreased marrow megakaryocytes. The etiology may be immune suppression of megakaryocyte development. Two patients are described who both responded rapidly to a com...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/(sici)1096-8652(199910)62:2<115::aid-ajh10
更新日期:1999-10-01 00:00:00
abstract::Several parameters may predict disease severity and overall survival in chronic lymphocytic leukemia (CLL). The purpose of our study of 190 CLL patients was to compare immunoglobulin heavy chain variable region (IgV(H)) mutation status, cytogenetic abnormalities, and leukemia cell CD38 and Zap-70 to older, traditional...
journal_title:American journal of hematology
pub_type: 杂志文章
doi:10.1002/ajh.20987
更新日期:2007-12-01 00:00:00