Abstract:
:Sickle cell disease (SCD) is an inherited disorder secondary to a point mutation at the sixth position of the beta chain of human hemoglobin resulting in the replacement of valine for glutamic acid. This recessive genetic abnormality precipitates the polymerization of the deoxygenated form of hemoglobin S inducing a major distortion of red blood cells (S-RBC), which decreases S-RBC deformability leading to chronic hemolysis and vaso-occlusion. These processes can result in severe complications including chronic pain, end-organ dysfunction, stroke, and early mortality. The only proven curative therapy for patients with SCD is myeloablative conditioning and allogeneic stem cell transplantation from HLA-matched sibling donors. In this review, we discuss the most recent advances in allogeneic stem cell transplantation in patients with SCD including more novel approaches such as reduced toxicity conditioning and the use of alternative allogeneic donors, including matched unrelated donors (MUDs), unrelated cord blood donors (UCBT), and familial haploidentical (FHI) donors. The results to date are very encouraging regarding allogeneic stem cell transplantation for patients with SCD including high survival rates and enabling a greater number of patients suffering from this chronic and debilitating condition to receive curative allogeneic stem cell therapies. However, we still have several areas to investigate and barriers to overcome to successfully cure the majority of patients with severe SCD through allogeneic stem cell therapies.
journal_name
Eur J Haematoljournal_title
European journal of haematologyauthors
Talano JA,Cairo MSdoi
10.1111/ejh.12447subject
Has Abstractpub_date
2015-05-01 00:00:00pages
391-9issue
5eissn
0902-4441issn
1600-0609journal_volume
94pub_type
杂志文章,评审abstract::In this study, Escherichia coli LPS dose-dependently (100-500 microg/ml) and time-dependently (10-60 min) inhibited platelet aggregation in human and rabbit platelets stimulated by agonists. LPS also dose-dependently inhibited the intracellular Ca2+ mobilization in human platelets stimulated by collagen. In addition, ...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/j.1600-0609.1999.tb01909.x
更新日期:1999-05-01 00:00:00
abstract::A patient who represented acute hemolytic crisis was studied. Analysis of the erythrocyte membrane proteins by SDS-PAGE revealed a deficiency of band 4.2. In the family, the sister of the patient who had been clinically normal was also shown to be deficient in band 4.2. Binding studies showed that the propositus' memb...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/j.1600-0609.1993.tb00164.x
更新日期:1993-05-01 00:00:00
abstract::Forty-one patients with codon 17, A-T mutation of beta-thalassemia, which is commonly found in Thailand, were studied to determine whether it is possible to predict phenotypic severity from genetic factors. The clinical phenotype of homozygotes for codon 17, A-T and compound heterozygotes for codon 17, A-T and beta+-t...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1034/j.1600-0609.2001.00305.x
更新日期:2001-02-01 00:00:00
abstract:OBJECTIVE:Interferon-alpha (IFNalpha) was the first effective pharmacologic treatment of hairy cell leukemia (HCL). Since 1990 purine analogs replaced IFNalpha because of higher rates of complete remission and an invariable disease recurrence after cessation of IFNalpha. However, there are only limited data about long-...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/j.1600-0609.2008.01190.x
更新日期:2009-03-01 00:00:00
abstract:BACKGROUND:Constitutive activation of STAT5 (by phosphorylation) has been identified in a number of malignancies, including acute myeloid leukemia (AML). OBJECTIVES:We investigated whether the level of phosphorylated STAT5 (pSTAT5) expression correlates with clinical outcome in AML. METHODS:Adult patients with newly ...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/j.1600-0609.2012.01825.x
更新日期:2012-10-01 00:00:00
abstract::Upshaw-Schulman syndrome (USS) is due to severe congenital deficiency of von Willebrand factor (VWF)-cleaving protease ADAMTS13 (a disintegrin and metalloprotease with thrombospondin type 1 domains, nr 13) activity resulting in the presence of unusually large forms of VWF in the circulation, causing intravascular plat...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/ejh.12197
更新日期:2014-02-01 00:00:00
abstract:PURPOSE:To determine the safety, dose-limiting toxicity and maximum tolerated dose (MTD) of the novel hydroxamate histone deacetylase inhibitor belinostat (PXD101) in patients with advanced hematological neoplasms. PATIENTS AND METHODS:Sequential dose-escalating cohorts of three to six patients with hematological mali...
journal_title:European journal of haematology
pub_type: 杂志文章,多中心研究
doi:10.1111/j.1600-0609.2008.01102.x
更新日期:2008-09-01 00:00:00
abstract:INTRODUCTION:Lower risk myelodysplastic syndromes (MDSs) are characterised by increased apoptosis of haematopoietic cells in the bone marrow (BM). The mechanism driving this excessive apoptosis involves multiple immune molecules, including inflammatory cytokines such as interferon-γ (IFN-γ), tumour necrosis factor-α (T...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/ejh.12074
更新日期:2013-05-01 00:00:00
abstract::A 59-yr-old man developed fevers, shortness of breath, persistent cough and weight loss, shortly after initiation of therapy with interferon-alpha 2a and cytosine arabinoside for treatment of chronic myelogenous leukemia. Radiologic pulmonary infiltrates and lung tissue biopsy were consistent with bronchiolitis oblite...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1034/j.1600-0609.2001.00503.x
更新日期:2001-11-01 00:00:00
abstract::Fludarabine is frequently used for treatment of B-cell chronic lymphocytic leukemia and in conditioning regimes for hematopoietic cell transplantations. The total body clearance of the principal metabolite 2-fluoro-ara-A (2F-Ara-A) correlates with the creatinine clearance. We report data on total dialysate concentrati...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/j.1600-0609.2005.00439.x
更新日期:2005-06-01 00:00:00
abstract::We report here the first case with Diamond-Blackfan anemia (DBA) who responded to rituximab. The patient is an 8-yr-old Japanese girl with refractory DBA accompanied by complex congenital heart disease. She received two doses of rituximab, 375 mg/m(2)/wk. She became transfusion independent 6 months after the treatment...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/j.1600-0609.2004.00394.x
更新日期:2005-05-01 00:00:00
abstract::Tumor necrosis factor (TNF)/cachectin can produce both beneficial and harmful manifestations. Mechanisms may operate to counteract potentially harmful effects such as shock and cachexia. The TNF binding protein (TNF-BP), which is found at increased levels in serum and urine of patients with chronic renal failure, may ...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/j.1600-0609.1989.tb00111.x
更新日期:1989-03-01 00:00:00
abstract:OBJECTIVE:We report an extension study of patients with essential thrombocythaemia (ET) in the Hungarian Myeloproliferative Neoplasm (HUMYPRON) Registry, which demonstrated that over 6 years anagrelide significantly decreased the number of patients experiencing minor arterial and minor venous thrombotic events (TEs) vs...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/ejh.13459
更新日期:2020-10-01 00:00:00
abstract::Most cytostatic drugs exert their effect on cells in active cell cycle. To improve the effect of cytostatic drugs we have tried, prior to treatment in vitro, to recruit tumor cells from G0 with growth factors. Leukemic cells from the bone marrows of 26 patients with AML and CML in blast crisis were incubated with G-CS...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/j.1600-0609.1996.tb00285.x
更新日期:1996-01-01 00:00:00
abstract:OBJECTIVES:Internal tandem duplications (ITDs) of the fms-like tyrosine kinase 3 ( FLT3) gene occur in 13-35% of patients with acute myeloid leukemia (AML). FLT3-ITD is associated with poor clinical outcome and is an indication for allogeneic stem cell transplantation (allo-SCT). METHODS:To investigate FLT3-ITD length...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/j.1600-0609.2012.01785.x
更新日期:2012-07-01 00:00:00
abstract::Signal transduction via receptors for N-formylmethionyl peptide chemoattractants (FPR) on human neutrophils is a highly regulated process. It involves direct interaction of receptors with heterotrimeric G-proteins and may be under the control of cytoskeletal elements. Evidence exists suggesting that the cytoskeleton a...
journal_title:European journal of haematology
pub_type: 杂志文章,评审
doi:10.1111/j.1600-0609.1993.tb01610.x
更新日期:1993-11-01 00:00:00
abstract::An automated reticulocyte counter using flowcytometric analysis, the R-3000 (Sysmex Inc. Kobe, Japan), has recently been modified to determine reticulated platelets (RPs) and large platelets (LPs). We measured frequencies of RPs, LPs in total platelet count and serum thrombopoietin concentration comprehensively in non...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/j.1600-0609.1999.tb01131.x
更新日期:1999-11-01 00:00:00
abstract::Acute myelogenous leukemia with t(8;21)(q22;q22) developed in a 42-yr-old woman having thrombocytopenia with absent radii syndrome (TARS). Standard induction and postremission therapies were safely administered. With each successive chemotherapy, the onset of platelet recovery was not delayed, but peak platelet counts...
journal_title:European journal of haematology
pub_type: 杂志文章,评审
doi:10.1034/j.1600-0609.2003.00054.x
更新日期:2003-04-01 00:00:00
abstract::We describe a case of Philadelphia-positive chronic myeloid leukaemia occurring simultaneously with B-cell chronic lymphocytic leukaemia in a 69-yr-old male. Gene probe analysis of DNA from both peripheral blood and bone marrow provided evidence for the independent evolution of 2 clones in this case, with a predominan...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/j.1600-0609.1988.tb00818.x
更新日期:1988-02-01 00:00:00
abstract::Reduced-intensity conditioning regimens have demonstrated lower toxicity but increased relapse rates in the context of allogeneic hematopoietic stem cell transplantation (aSCT) for patients with acute myelogenous leukemia (AML). The FLAMSA- reduced-intensity conditioning (RIC) regimen, combining a cytoreductive and a ...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/ejh.12615
更新日期:2016-05-01 00:00:00
abstract::The prevalence of obesity is increasing and progressively influencing physician-patient interactions. While there is a sizable amount of data demonstrating that obesity is a state of low-grade inflammation, to our knowledge, there is no single review summarizing its effects on hematologic parameters and thrombotic ris...
journal_title:European journal of haematology
pub_type: 杂志文章,评审
doi:10.1111/ejh.13560
更新日期:2020-12-03 00:00:00
abstract::We evaluated the serum/plasma levels of cytokines [interleukin (IL)-6, vascular endothelial growth factor (VEGF), transforming growth factor (TGF)-beta2] and markers of coagulation, fibrinolysis, endothelial and platelet activation during the first 4 wk of treatment with the thalidomide analogue Actimid (CC-4047) in 1...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/j.1600-0609.2004.00393.x
更新日期:2005-04-01 00:00:00
abstract:INTRODUCTION:Symptomology of AL amyloidosis can be vague, with a broad range of manifestations and potential etiologies. We sought to determine whether time from initial patient-reported symptom onset to diagnosis was associated with survival. METHODS:The Boston University Amyloidosis Patient Database was queried for ...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/ejh.13472
更新日期:2020-10-01 00:00:00
abstract::Paraneoplastic neurologic syndromes may develop in Hodgkin's disease (HD). We describe three young female patients with neurological disorders, not explained by other causes, preceding diagnosis or relapse of HD. The lack of response of the paraneoplastic syndrome to successful treatment of HD among our three patients...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1034/j.1600-0609.2001.00240.x
更新日期:2001-01-01 00:00:00
abstract:INTRODUCTION/OBJECTIVES:Acute myeloid leukemia (AML) accounts for 90% of all cases of acute leukemia in adults. In Brazil, the mortality from myeloid leukemia is 1.74/100 000 men and 1.37/100 000 women. Our aim was to review and update guidelines of the Brazilian Society of Bone Marrow Transplantation on indications of...
journal_title:European journal of haematology
pub_type: 杂志文章,实务指引
doi:10.1111/ejh.12808
更新日期:2017-02-01 00:00:00
abstract:OBJECTIVES:As new, effective therapies emerge for acute lymphoblastic leukaemia (ALL), the results of clinical trials need to relate to standard of care. METHODS:We used the population-based Swedish ALL Registry to evaluate characteristics, treatment and long-term outcome in 933 patients with diagnosis between 1997 an...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/ejh.13247
更新日期:2019-08-01 00:00:00
abstract::10 patients with CLL and 2 with CML were treated with gradually increasing doses of 1 alpha(OH)D3, up to 4 micrograms daily during 6 wk. 3 patients with preleukemia and 1 with myelofibrosis were treated with 2 micrograms daily of 1 alpha(OH)D3 for a prolonged period up to 17 wk. The treatment with 1 alpha (OH)D3 did n...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/j.1600-0609.1987.tb00160.x
更新日期:1987-07-01 00:00:00
abstract::Immunoglobulin gene rearrangements in B-cell lymphoma subtypes may not always be detected by PCR if only one primer set is applied. We therefore analysed a range of low and high grade B-cell lymphoma subtypes for monoclonality using PCR, to determine appropriate primer selection strategies for routine diagnostic use. ...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/j.1600-0609.1997.tb00966.x
更新日期:1997-09-01 00:00:00
abstract::In Western countries, the age distribution of Hodgkin lymphoma (HL) follows a characteristic bimodal curve showing an early and a late peak at approximately 35 and 70 yr, respectively. Furthermore, the presence of latent Epstein-Barr virus (EBV) genome in the Hodgkin Reed-Sternberg cells, the tumour cell population of...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/ejh.12463
更新日期:2015-08-01 00:00:00
abstract::Human red blood cells (RBC) are heterogeneous with respect to their size; the physiological significance of this heterogeneity has not yet been fully elucidated. To further investigate this problem, some characteristics of human RBC fractionated according to their mean corpuscular volume (MCV) by counterflow centrifug...
journal_title:European journal of haematology
pub_type: 杂志文章
doi:10.1111/j.1600-0609.1992.tb00570.x
更新日期:1992-02-01 00:00:00