Abstract:
:Gene therapy targeting hematopoietic cells has arrived at a new stage of potency. While the potential for curing inherited disorders of the immune system has been demonstrated in clinical trials, we were also confronted with the first serious adverse events related to random insertion of foreign DNA into cellular chromosomes. As it is likely that the manifestation of severe side effects results from a multifactorial process, it will be of crucial importance to define the significance of the individual risk factors involved. The future of the field will depend on our ability to define risk classifications of clinical approaches, to continuously improve transgene technologies, and to introduce new concepts for targeted selection of transgenic cells. Interestingly, correction of genetic disorders by homologous gene repair in defined stem cell clones is on the horizon, but far from being available for clinical use.
journal_name
Acta Haematoljournal_title
Acta haematologicaauthors
Baum C,von Kalle Cdoi
10.1159/000072459subject
Has Abstractpub_date
2003-01-01 00:00:00pages
107-9issue
2-3eissn
0001-5792issn
1421-9662pii
72459journal_volume
110pub_type
杂志文章,评审abstract::Chromosomal aberrations in a case of atypic myeloproliferative syndrome are reported. The analysis was carried out on short-term cultures of sternal bone marrow with the ASG and fluorescence method. 90% of the metaphases studied showed pseudoliploidy: one of the chromosomes 12 was missing and one submedian marker chro...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000208052
更新日期:1975-01-01 00:00:00
abstract::Many bovine leukemic lymphocytes produce virus particles when kept in survival cultures in Eagle's Minimum Essential Medium supplemented with 20 percent of inactivated fetal calf serum. Virus particles equilibrate at a density of 1.16 g/ml in sucrose gradients and at a density of 1.12 g/ml in metrizamide gradients. Si...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000208076
更新日期:1975-01-01 00:00:00
abstract::A 29-year-old man with acute myelomonocytic leukemia had an initial leukocyte count of 192 X 10(9) cells/l and 84% blasts. During the initial treatment with hydroxyurea, 1 g/m2 orally tid, he developed pulmonary leukostasis which responded rapidly to whole-lung radiation with 1.5 Gy in one fraction. Pulmonary leukosta...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000205987
更新日期:1987-01-01 00:00:00
abstract::All-trans-retinoic acid (ATRA) combined with anthracyclines is currently the standard treatment for acute promyelocytic leukemia (APL). In elderly patients the presence of comorbidities, such as cardiomyopathy or different organ failures, often represents an absolute contraindication to standard chemotherapy. In this ...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000097880
更新日期:2007-01-01 00:00:00
abstract::Phagocytic activity of leukaemic blasts in 20 adults and 16 children suffering from acute leukaemia was studied in vitro by the use of ferrioxidsaccharate. Most frequently phagocytosing blasts were encountered in myelomonocytic leukaemias. The morphological character of positive blasts resembled often that of monocyto...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000208150
更新日期:1975-01-01 00:00:00
abstract::The short-term effect of a domestically produced equine antithymocyte globulin (ATG) was analyzed in 6 patients with acquired severe aplastic anemia (AA). All patients received 5 doses of ATG every other day in a 60-min intravenous infusion. Five peripheral blood immunoregulatory mononuclear cell (MNC) subsets, define...
journal_title:Acta haematologica
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1159/000205557
更新日期:1989-01-01 00:00:00
abstract::Treating patients with hemophilia and inhibitors is often problematic. The presence of inhibitors negatively impacts the effectiveness of treatment to achieve hemostasis especially in patients with hemophilia B, owing mainly to allergic reactions to factor IX (FIX) concentrates and the low success rate of immune toler...
journal_title:Acta haematologica
pub_type:
doi:10.1159/000508722
更新日期:2020-07-22 00:00:00
abstract::Iron deficiency is the main cause of anemia in both sexes, with women being more commonly affected. Iron therapy is currently considered an effective and safe remedy to replenish the iron storages. Iron can be administrated both orally and intravenously. In particular, intravenous (IV) iron therapy is widely used when...
journal_title:Acta haematologica
pub_type:
doi:10.1159/000503768
更新日期:2020-01-01 00:00:00
abstract::The clinical pattern of haemolysis associated with Donath-Landsteiner antibodies has undergone a change over the years. In the current study 13 patients developed the acute form of the disease whilst only 1 presented with the classical picture of chronic paroxysmal cold haemoglobinuria. The acute illness typically occ...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000206992
更新日期:1982-01-01 00:00:00
abstract::The effect of total-body cold exposure on plasma concentrations of von Willebrand factor (vWF), endothelin-1 (ET) and thrombomodulin (TM), all of which are considered to be generated from the endothelium, was studied in systemic lupus erythematosus (SLE) patients with and without Raynaud's phenomenon. The plasma level...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000204684
更新日期:1992-01-01 00:00:00
abstract::A 59-year-old man with beta-thalassaemia major is unusually well. He has no beta-chains in his haemoglobin but is heterozygous for the genes responsible for alphaA and for alphaG Philadelphia. In addition he is also heterozygous for the genes responsible for gammaF and a new gamma-chain, gamma75(E19) Ile-Thr, named ga...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000208204
更新日期:1975-01-01 00:00:00
abstract::We studied neutrophil chemotaxis and surface membrane glycoproteins in 12 patients suffering from myeloid disorders with abnormal karyotype using in vitro techniques in all 12. Chromosome studies were also carried out virtually simultaneously. We chose to study only patients showing a deficit of chemotaxis (p less tha...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000205543
更新日期:1989-01-01 00:00:00
abstract::A 13-year-old Saudi girl presented with severe clinical features of systemic lupus erythematosus of 3 months' duration. In addition to laboratory evidence of the disease, the patient was found to have pancytopenia and myelofibrosis. While pancytopenia was considerably improved by steroids, myelofibrosis was not revers...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000205641
更新日期:1988-01-01 00:00:00
abstract::Twenty-four infants and children suffering from glucose-6-phosphate dehydrogenase (G6PD) deficiency during hemolytic crisis were included in this study. Their ages ranged between 3 and 36 months with a median of 10 months. 22 were males and 2 were females. Fourteen out of them received a single bolus dose of desferrio...
journal_title:Acta haematologica
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1159/000205320
更新日期:1989-01-01 00:00:00
abstract::Thalidomide acts on the microenvironment of myelodysplastic syndromes (MDS) by influencing cytokine networks, and growing evidence supports thalidomide's usefulness in the management of haematological malignancies, such as MDS. The European Collaboration Group on Myelofibrosis with Myeloid Metaplasia reviewed patients...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000087042
更新日期:2005-01-01 00:00:00
abstract::A 67-year-old woman with agnogenic myeloid metaplasia, who underwent splenectomy 1 year after the diagnosis, experienced spontaneous hematologic remission 6 years after splenectomy. Ten months before her anemia improved, peripheral leukocytes no longer showed a shift to the left and peripheral erythroblasts had disapp...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000205163
更新日期:1990-01-01 00:00:00
abstract::Pyrimidine 5'-nucleotidase (P5'N) partial deficiency has been described in several hematological disorders and also in the beta-thalassemic trait. To check if the P5'N deficiency in thalassemia was acquired we used thalassemic red cells (from either homo- or heterozygous subjects), whose P5'N activity was lower than i...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000205286
更新日期:1989-01-01 00:00:00
abstract::A 12-year-old male of Chinese ancestry had life-long hemolytic anemia attributed to alpha-thalassemia. Restriction endonuclease mapping of his DNA revealed that in reality, he had three alpha-globin loci, but he was homozygous for pyruvate kinase deficiency. The new pyruvate kinase variant carried by this patient was ...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000206830
更新日期:1983-01-01 00:00:00
abstract::The detection of cellular antigens in bone marrow sections depends on the method of embedding, the nature of antigen and antibody, antigen retrieval techniques and the sensitivity of the immunohistochemical method. This study evaluated a fluorescyl-tyramide-enhanced immunostaining method on methyl-methacrylate-embedde...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000097458
更新日期:2007-01-01 00:00:00
abstract:BACKGROUND:Not all patients with diffuse large B-cell lymphoma (DLBCL) are candidates for aggressive regimens. (90)Y ibritumomab tiuxetan ((90)Y-IT), an anti-CD20 radionuclide-conjugated antibody, has demonstrated clinical efficacy in DLBCL with a favorable toxicity profile. METHODS:This phase II trial investigated th...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000368291
更新日期:2015-01-01 00:00:00
abstract::A patient with regional enteritis had received iron dextran for treatment of iron deficiency. Subsequently he developed a large (3.1 g/100 ml) IgG-K serum spike which had precipitin activity against dextran sulfate but not a variety of other antigens. There has been no evidence of multiple myeloma and the spike gradua...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000207755
更新日期:1978-01-01 00:00:00
abstract::A case of isolated lambda-light chain proteinemia and proteinuria is reported. During a 42-month follow-up, no sign of myelomatosis or amyloidosis developed and Bence Jones proteinuria remained nearly of the same magnitude. This case of apparently idiopathic Bence Jones gammopathy, the first lambda-type so far reporte...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000207409
更新日期:1980-01-01 00:00:00
abstract:BACKGROUND/AIMS:The aim of this work was to investigate the efficacy and predictive factors of CLAG treatment in refractory or relapsed (R/R) acute myeloid leukemia (AML) patients. METHODS:Sixty-seven R/R AML patients were enrolled in this prospective cohort study and treated by a CLAG regimen: 5 mg/m2/day cladribine ...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000493250
更新日期:2019-01-01 00:00:00
abstract::A case of Philadelphia-positive (Ph) acute lymphoblastic leukaemia (ALL) in a 40-year-old male is presented. At diagnosis, 80% of bone marrow cells were Ph. Remission with normal blood counts was achieved but the marrow became hypercellular, indicating conversion to chronic granulocytic leukaemia (GCL). The Ph clone p...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000204497
更新日期:1993-01-01 00:00:00
abstract:OBJECTIVE:Adherence to tyrosine kinase inhibitor treatment is a significant factor in the achievement of a good clinical response in chronic myeloid leukemia (CML). The aim of this retrospective study is to investigate 1- and 2-year medication adherence to imatinib treatment, linking adherence rates with the clinical o...
journal_title:Acta haematologica
pub_type: 评论,杂志文章
doi:10.1159/000444626
更新日期:2016-01-01 00:00:00
abstract::It has been suggested that CD44 mediates adhesive interactions between hematopoietic progenitor cells and the stromal microenvironment. Ligands of CD44 include several extracellular matrix components, such as hyaluronic acid and fibronectin. Antibodies against CD44 have been shown to induce homotypic T cell aggregatio...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000203886
更新日期:1996-01-01 00:00:00
abstract::Increased levels of hemoglobin A(2) (HbA(2)) are present in most beta-thalassemia carriers. The mechanism of this effect is not understood, although the increase may result from transcriptional and posttranscriptional changes. In the present study, we quantitate delta-globin mRNA levels in peripheral-blood-enriched re...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000040959
更新日期:1999-01-01 00:00:00
abstract::We have demonstrated the log normality of the distribution of sheep rosette-forming cells and mouse rosette-forming cells' values obtained with lymphocytes isolated from the peripheral blood of 135 healthy human beings and 57 patients suffering from chronic lymphocytic leukemia and well differentiated lymphocytic lymp...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000207783
更新日期:1978-01-01 00:00:00
abstract::In a comparison of 47 patients with Philadelphia-chromosome (Ph)-positive chronic myeloid leukemia (CML) in the Nagasaki University School of Medicine and 64 patients with the same disease in the Roswell Park Memorial Institute, the correlation between the modal number of chromosomes and the therapeutic response and/o...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000204713
更新日期:1992-01-01 00:00:00
abstract::In a dose titration study we tested the efficacy and tolerance of recombinant human erythropoietin (rhEPO) in 10 patients with myelodysplasia (MDS) and 2 patients with idiopathic myelofibrosis. Patients with a haemoglobin level < 100 g/l were treated as out-patients for 12 weeks with daily doses ranging from 30 U/kg b...
journal_title:Acta haematologica
pub_type: 杂志文章
doi:10.1159/000204378
更新日期:1993-01-01 00:00:00