Abstract：
:Hydroxyurea (HU) is the first widely used treatment to have an impact on the severity of disease in adult patients with sickle cell anemia, but limited data are available for younger patients or those with variant genotypes. We reviewed 324 months of experience with HU in 16 patients from 5.3 to 18.4 years of age treated for 6 to 50 months. The major toxicity was reversible neutropenia. Linear growth continued unchanged, and all patients gained weight. Hematologic results were similar to those reported in adults with increases in mean corpuscular volume (MCV) and total and fetal hemoglobin (HbF). We noted that the maximal hematologic effects occurred at less than the maximum dose. Clinically, patients experienced an 80% reduction in episodes of acute chest syndrome and a reduced need for blood transfusion, as well as a 30% decrease in the number of hospitalizations for painful events during HU therapy compared with an equivalent number of months before HU. These highly statistically significant results confirmed the value of HU in ameliorating the severe clinical course of pediatric patients. Similar effects were observed in three patients with sickle beta degrees-thalassemia, sickle beta+-thalassemia, and S-O Arab. Recurrent acute splenic sequestration and progressive symptomatic osteonecrosis were observed during HU. Thus, HU may not prevent the development of complications once organ damage is present. The challenge remains to determine when and to which pediatric patients with sickle cell disease HU should be offered.
journal_name
Semin Hematoljournal_title
Seminars in hematologyauthors
Rogers ZRsubject
Has Abstractpub_date
1997-07-01 00:00:00pages
42-7issue
3 Suppl 3eissn
0037-1963issn
1532-8686journal_volume
34pub_type
临床试验,杂志文章abstract：:In this review, we discuss disease-causing alterations of RUNT-related transcription factor 1 (RUNX1), a master regulator of hematopoietic differentiation. Familial platelet disorder with predisposition to myeloid leukemia (FPDMM) typically presents with (1) mild to moderate thrombocytopenia with normal-sized platelet...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：10.1053/j.seminhematol.2017.04.006
更新日期：2017-04-01 00:00:00
abstract：:Natural killer cells are an essential component of the innate immune system and play a crucial role in immunity against malignancies, without, at difference with T cells, requiring antigen priming or inducing graft-versus-host-disease. Hence, Natural Killer cells can provide a valuable source of allogeneic "off-the-sh...
journal_title：Seminars in hematology
pub_type： 杂志文章
doi：10.1053/j.seminhematol.2020.10.004
更新日期：2020-10-01 00:00:00
abstract：:Myelofibrosis with myeloid metaplasia, also known as idiopathic myelofibrosis (IF) or agnogenic myeloid metaplasia, is one of the characteristic manifestations of polycythemia vera (PV) in the spent phase, and has a particularly adverse prognosis. IF may also present de novo. To date, treatment strategies for both spe...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：10.1053/shem.2003.50032
更新日期：2003-01-01 00:00:00
abstract：:An immune hemolytic anemia occurs in a few patients in whom the concentration of antibody on the red cell is below the level for detection by the usual antiglobulin test. Clinically, these patients are identical to patients with warm type Coombs-positive hemolytic anemia, except for the quantity of antibody on the cel...
journal_title：Seminars in hematology
pub_type： 杂志文章
doi：
更新日期：1976-10-01 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：: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：:While the prevalence of iron deficiency has remained relatively constant, there has been continuing refinement in its laboratory recognition, especially with the recent introduction of serum ferritin and FEP measurements. It is helpful to classify iron deficiency into three stages. Storage iron depletion is identified...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：
更新日期：1982-01-01 00:00:00
abstract：:The value of prophylaxis against venous thromboembolism (VTE) is increasingly accepted in most surgical specialties, although the potential reduction in fatal pulmonary embolism has recently been questioned. The burden of VTE in hospital patients nevertheless remains high, partly attributable to underuse of thrombopro...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：10.1053/shem.2001.25188
更新日期：2001-04-01 00:00:00
abstract：:From a computational perspective, Bayesian methods can be viewed as a natural extension of familiar confidence intervals and significance tests, which sheds light on their meaning. This viewpoint shows that no special software is required to compute Bayesian results, leaving the distinctions between conventional and B...
journal_title：Seminars in hematology
pub_type： 杂志文章
doi：10.1053/j.seminhematol.2008.04.004
更新日期：2008-07-01 00:00:00
abstract：:The risk of postoperative venous thromboembolism (VTE) is dependent on the type of surgical procedure and specific characteristics of the patient. Patients with intrinsic factors placing them at higher risk of VTE remain at high risk, even if the procedural risk is low. The identification of factors such as age, obesi...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：10.1016/s0037-1963(00)90094-5
更新日期：2000-07-01 00:00:00
abstract：:Chronic lymphocytic leukemia (CLL) is a clonal proliferation of mature-appearing, but maturationally immature B cells; T cells are rarely involved. Most data suggest that CLL cells are frozen at an early step in maturation; this can be overcome by several techniques such as in vitro incubation with tumor promotors. Al...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：
更新日期：1987-10-01 00:00:00
abstract：:Imatinib (Gleevec) (formerly STI571) competitively targets the adenosine 5-triphosphate (ATP) binding site of the kinase domain of ABL and was recently approved for the treatment of chronic myeloid leukemia (CML). Point mutations occurring in the kinase domain of BCR-ABL have been identified as a cause of imatinib res...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：10.1053/shem.2003.50046
更新日期：2003-04-01 00:00:00
abstract：:Our advanced knowledge of coagulation has led to the synthesis of novel procoagulant substances, such as recombinant activated factor VII (rFVIIa; NovoSeven, Novo Nordisk, Bagsvaerd, Denmark). Similarly, in-depth understanding of the interaction between anticoagulant proteins and their natural inhibitors has led to th...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：10.1053/j.seminhematol.2005.11.023
更新日期：2006-01-01 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：:Idiopathic thrombocytopenic purpura (ITP) is an illness of primary acquired thrombocytopenia occurring in the absence of marrow failure. Splenectomy was first used as a treatment for ITP in 1913. However, with the realization that opsonin (critical for the optimal killing of invasive micro-organisms by white blood cel...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：10.1016/s0037-1963(00)90114-8
更新日期：2000-01-01 00:00:00
abstract：:Thrombopoietin (TPO) regulates thrombopoiesis through activation of TPO receptors on the megakaryocyte cell surface, resulting in increased platelet production. The TPO receptor agonists are novel treatments for patients with chronic ITP aimed at increasing platelet production through interactions with the TPO recepto...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：10.1053/j.seminhematol.2013.03.005
更新日期：2013-01-01 00:00:00
abstract：:Stem cell transplantation is a recognized treatment for many hematologic malignancies. Human leukocyte antigen (HLA) testing is a key assessment in the evaluation of potential recipients and selection of the appropriate stem cell donor. Over the past several years, technological advances have moved HLA testing from a ...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：10.1016/s0037-1963(01)90052-6
更新日期：2001-04-01 00:00:00
abstract：:Thrombus forms on catheters and other indwelling synthetic materials. Systemic heparin infusion decreases, but does not eliminate thrombus formation. The day is approaching when systemic heparinization may no longer be indicated to suppress catheter thrombogenesis. Passivation of catheters by heparin immobilization is...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：
更新日期：1991-10-01 00:00:00
abstract：:Copper is an essential trace element that is required for a number of enzymes which are necessary for normal metabolic function. Metabolic balance studies have demonstrated that daily copper losses are approximately 1.3 mg/day. In order to remain in copper balance, the average adult male must consume a diet which cont...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：
更新日期：1983-04-01 00:00:00
abstract：:The results of the Y-chromosome in situ hybridization experiments, the MRA assessment, and the long-term production of CFU-GM in vitro indicate that our protocol to sort low density WGA+, 15/1.1-, Rh123 dull cells enriches about 200-fold for PHSC. Assays for spleen colony formation (CFU-S) and radioprotection (30-day ...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：
更新日期：1991-04-01 00:00:00
abstract：:The farnesyltransferase inhibitors (FTIs) have been shown in early clinical trials to elicit antitumor actions in a broad range of solid and hematologic malignancies. The mechanism of FTI action involves blockade of farnesyltransferase, an enzyme implicated in multiple cell-signaling pathways involved in proliferation...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：10.1053/shem.2002.35984
更新日期：2002-07-01 00:00:00
abstract：:The cyclophosphamide, vincristine, prednisone, bleomycin, doxorubicin, procarbazine (COP-BLAM) programs of combination chemotherapy were administered to patients with advanced diffuse large cell lymphoma. The original COP-BLAM programs were designed to deliver intense multidrug therapy maximizing tumor kill. COP-BLAM ...
journal_title：Seminars in hematology
pub_type： 杂志文章
doi：
更新日期：1988-04-01 00:00:00
abstract：:Two membrane proteins express the antigens that comprise the Kell blood group system. A single antigen, Kx, is carried on XK, a 440-amino acid protein that spans the membrane 10 times, and more than 20 antigens reside on Kell, a 93-kd, type II glycoprotein. XK and Kell are linked, close to the membrane surface, by a s...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：10.1016/s0037-1963(00)90036-2
更新日期：2000-04-01 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
abstract：:Acute promyelocytic leukemia (APL) is characterized by reciprocal chromosomal translocations that always Involve the retinoic acid receptor-alpha (RARalpha) gene on chromosome 17. RARalpha variably fuses to the PML, PLZF, NPM, NuMA, and STAT 5b genes (X genes), leading to the generation of X-RARalpha and RARalpha-X fu...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：10.1016/s0037-1963(01)90006-x
更新日期：2001-01-01 00:00:00
abstract：:The immunomodulatory drug (IMiD) lenalidomide is a more potent immunomodulator than thalidomide with respect to its effects on cytokine modulation and increased T-cell proliferation. Of all the IMiDs, clinical trial data are most mature for lenalidomide. In phase I studies, dose-limiting toxicities of lenalidomide wer...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：10.1053/j.seminhematol.2005.10.004
更新日期：2005-10-01 00:00:00
abstract：:It is now well accepted that a subgroup of patients with myelodysplastic syndromes (MDS) can recover from pancytopenia following immunosuppressive treatment (IST). For many years immunosuppression with antilymphocyte antibodies has been a standard treatment approach for patients with severe aplastic anemia (SAA). The ...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：10.1053/j.seminhematol.2012.07.004
更新日期：2012-10-01 00:00:00
abstract：:Antibody-mediated rejection (AMR) is a major risk factor for graft loss following kidney transplantation. Traditional anti-humoral therapies provide suboptimal therapy and they do not deplete plasma cells, which are the source of antibody production. Proteasome inhibitors (PI) have been shown to deplete both transform...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：10.1053/j.seminhematol.2012.04.008
更新日期：2012-07-01 00:00:00
abstract：:Hodgkin Lymphoma (HL) is a lymphoproliferative disorder of B cells that commonly has a favorable prognosis when treated with either combination chemotherapy and radiation therapy, or chemotherapy alone. However, the prognosis for patients who relapse, or have evidence for refractory disease, is poor and new treatments...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：10.1053/j.seminhematol.2016.05.011
更新日期：2016-07-01 00:00:00
abstract：:Anemia is frequently diagnosed in patients with cancer and its treatment is an important clinical problem. The deficiency in red blood cells (RBCs) can be a debilitating problem, and anemia correlates with poor performance status, deteriorates quality of life, and may negatively influence the prognosis of cancer patie...
journal_title：Seminars in hematology
pub_type： 杂志文章,评审
doi：10.1053/j.seminhematol.2007.04.004
更新日期：2007-07-01 00:00:00
