Clinical outcomes in children with sickle cell disease living in England: a neonatal cohort in East London.

Abstract:

BACKGROUND AND OBJECTIVES:We investigated outcomes in a UK neonatal cohort as a benchmark for care of children with sickle cell disease (SCD). DESIGN AND METHODS:Two-hundred and fifty-two children (180 with hemoglobin [Hb] SS, 64 with HbSC, and 8 with HbS/beta thalassemia), identified during 1983-2005 by universal birth screening in East London, were followed in a hospital and community-based program which included penicillin V prophylaxis from 3 months of age, 23-valent pneumococcal polysaccharide vaccine from 1993, conjugate pneumococcal vaccine from 2002 and transcranial Doppler screening from 1991. RESULTS:At the end of 2005, there were 2158 patient years of observation. The median age of the patients was 7.8 (interquartile range 3.3-13.0) years, and 2.8% of those enrolled had been lost to follow-up. The estimated survival of children with HbSS at 16 years was 99.0% (95% confidence interval, CI, 93.2 to 99.9%) and pneumococcal sepsis rate was 0.3 (95% CI 0.1-0.8) episodes per 100 patient-years. The risk of overt stroke was 4.3% (95%CI 1.5 to 11.4%) and could be further reduced by transcranial Doppler screening from infancy and transfusing all children with high-risk scans. No deaths, strokes or episodes of pneumococcal sepsis were observed in children with HbSC or HbS/beta thalassemia. The mortality rates from HbSS were significantly lower than those in other reported cohorts. INTERPRETATION AND CONCLUSIONS:Mortality in childhood SCD can virtually be eliminated in a well-resourced health service setting linking community-based care with a specialized, hospital-based center. SCD continues to cause substantial morbidity from acute complications and chronic organ damage. We recommend setting up of clinical networks to optimize the management of SCD.

journal_name

Haematologica

journal_title

Haematologica

authors

Telfer P,Coen P,Chakravorty S,Wilkey O,Evans J,Newell H,Smalling B,Amos R,Stephens A,Rogers D,Kirkham F

doi

10.3324/haematol.10937

subject

Has Abstract

pub_date

2007-07-01 00:00:00

pages

905-12

issue

7

eissn

0390-6078

issn

1592-8721

journal_volume

92

pub_type

杂志文章
  • High-dose dexamethasone regulates interleukin-18 and interleukin-18 binding protein in idiopathic thrombocytopenic purpura.

    abstract::To evaluate the effects of high-dose dexamethasone (HD-DXM) on the balance of interleukin-18 (IL-18) and its endogenous antagonist IL-18 binding protein (IL-18BP) in ITP patients, IL-18, IL-18BP as well as IFN-gamma, IL-4 plasma levels and platelet counts were determined in 17 ITP patients receiving DXM 40 mg/day for ...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2009.007708

    authors: Shan NN,Zhu XJ,Wang Q,Wang CY,Qin P,Peng J,Hou M

    更新日期:2009-11-01 00:00:00

  • Fibrin(ogen) in human disease: both friend and foe.

    abstract::Fibrinogen is an abundant protein synthesized in the liver, present in human blood plasma at concentrations ranging from 1.5-4 g/L in healthy individuals with a normal half-life of 3-5 days. With fibrin, produced by thrombin-mediated cleavage, fibrinogen plays important roles in many physiological processes. Indeed, t...

    journal_title:Haematologica

    pub_type: 杂志文章,评审

    doi:10.3324/haematol.2019.236901

    authors: Vilar R,Fish RJ,Casini A,Neerman-Arbez M

    更新日期:2020-01-31 00:00:00

  • Management of multiple myeloma and related-disorders: guidelines from the Italian Society of Hematology (SIE), Italian Society of Experimental Hematology (SIES) and Italian Group for Bone Marrow Transplantation (GITMO).

    abstract:OBJECTIVES:Perceiving the need for rigorous recommendations to facilitate decisions concerning the management of patients with multiple myeloma (MM), the Italian Society of Hematology (SIE) and the two affiliate societies (SIES and GITMO) commissioned a project to develop guidelines for the therapy of MM using evidence...

    journal_title:Haematologica

    pub_type: 杂志文章,实务指引,评审

    doi:

    authors: Barosi G,Boccadoro M,Cavo M,Corradini P,Marchetti M,Massaia M,Merlini G,Tosi P,Tura S,Italian Society of Hematology.,Italian Society of Experimental Hematology.,Italian Group for Bone Marrow Transplantation.

    更新日期:2004-06-01 00:00:00

  • Comparison of genetic and clinical aspects in patients with acute myeloid leukemia and myelodysplastic syndromes all with more than 50% of bone marrow erythropoietic cells.

    abstract:BACKGROUND:The World Health Organization separates acute erythroid leukemia (erythropoiesis in ≥50% of nucleated bone marrow cells; ≥20% myeloblasts of non-erythroid cells) from other entities with increased erythropoiesis - acute myeloid leukemia with myelodysplasia-related changes (≥20% myeloblasts of all nucleated c...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2011.043687

    authors: Bacher U,Haferlach C,Alpermann T,Kern W,Schnittger S,Haferlach T

    更新日期:2011-09-01 00:00:00

  • Differentiation of monoclonal gammopathy of undetermined significance and multiple myeloma using flow cytometric characteristics of plasma cells.

    abstract:BACKGROUND AND OBJECTIVES:The differential diagnosis between multiple myeloma (MM) and monoclonal gammopathy of undetermined significance (MGUS) may be uncertain in some cases; this problem is reflected by discrepancies between different classification systems with an accordance in only 2/3 of cases. We studied whether...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:

    authors: Sezer O,Heider U,Zavrski I,Possinger K

    更新日期:2001-08-01 00:00:00

  • Monitoring BCR-ABL transcript levels by real-time quantitative polymerase chain reaction: a linear regression equation to convert from BCR-ABL/B2M ratio to estimated BCR-ABL/ABL ratio.

    abstract::In order to overcome the problem of different control genes for BCR-ABL normalization, we used a linear regression equation to compare our results previously obtained using B2M as the control gene with those calculated using the ABL gene and validated the slope as a factor to convert from B2M to ABL results. ...

    journal_title:Haematologica

    pub_type: 信件

    doi:10.3324/haematol.10632

    authors: Iacobucci I,Galletti L,Amabile M,Soverini S,Baccarani M,Martinelli G

    更新日期:2007-03-01 00:00:00

  • Activity of everolimus (RAD001) in relapsed and/or refractory multiple myeloma: a phase I study.

    abstract::The mammalian target of rapamycin plays an important role in multiple myeloma. The allosteric mammalian target of rapamycin inhibitor everolimus has long been approved for immunosuppression and has shown activity in certain cancers. This investigator-initiated phase I trial explored the use of everolimus in relapsed a...

    journal_title:Haematologica

    pub_type: 杂志文章,多中心研究

    doi:10.3324/haematol.2014.116269

    authors: Günther A,Baumann P,Burger R,Kellner C,Klapper W,Schmidmaier R,Gramatzki M

    更新日期:2015-04-01 00:00:00

  • Placental/umbilical cord blood transplantation.

    abstract::In this article we summarize the clinical outcome of unrelated placental/umbilical cord blood (CB) transplantation, discuss the biological characteristics of CB hematopoietic progenitor/stem cells (HPC) and balance the relative advantages and disadvantages of this therapy as compared with transplantation of other HPC ...

    journal_title:Haematologica

    pub_type: 杂志文章,评审

    doi:

    authors: Sirchia G,Rebulla P

    更新日期:1999-08-01 00:00:00

  • Multivariate analysis of Hodgkin's disease prognosis. Fitness and use of a directly predictive equation.

    abstract::The main purpose of this work is to give methodologic details and further verification regarding the predictive system recently elaborated for Hodgkin's disease (HD) patients, by which, under standard accuracy and conventional treatment, patient survival can be directly estimated (within a given confidence interval) w...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:

    authors: Gobbi PG,Gobbi PG,Mazza P,Zinzani PL

    更新日期:1989-01-01 00:00:00

  • Efficient generation and characterization of tumor cell subclones with different adhesion pathways involved in cell lysis.

    abstract:BACKGROUND:Specific tumor cell recognition is required for optimal tumor directed therapy. Lymphokine activated killer (LAK) cells are able to recognize tumor targets specifically because LAK cells can distinguish between normal and tumor cells. This study was aimed at analyzing receptor molecules on tumor cells and th...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:

    authors: Lefterova P,Negrin R,Neubauer A,Huhn D,Blume K,Schmidt-Wolf I

    更新日期:1993-11-01 00:00:00

  • Generation and administration of HA-1-specific T-cell lines for the treatment of patients with relapsed leukemia after allogeneic stem cell transplantation: a pilot study.

    abstract::Since HA-1-specific T cells have been shown to make a significant contribution to the clinical responses in patients with relapsed leukemia, we investigated the feasibility of adoptive transfer of in vitro induced HA-1-specific CD8 positive T cells to patients with relapsed leukemia after allogeneic stem cell transpla...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2011.053371

    authors: Meij P,Jedema I,van der Hoorn MA,Bongaerts R,Cox L,Wafelman AR,Marijt EW,Willemze R,Falkenburg JH

    更新日期:2012-08-01 00:00:00

  • Impact on survival through consolidation radiotherapy for diffuse large B-cell lymphoma: a comprehensive meta-analysis.

    abstract::Rituximab has improved response rates and overall survival in B-cell lymphoma (DLBCL). Radiotherapy is an effective treatment modality for lymphomas, but there is uncertainty on its use as consolidation after chemo-immunotherapy mainly in advanced stages. We evaluated its efficacy with a comprehensive meta-analysis an...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2020.249680

    authors: Berger MD,Trelle S,Büchi AE,Jegerlehner S,Ionescu C,Lamy de la Chapelle T,Novak U

    更新日期:2020-06-18 00:00:00

  • Increased platelet and leukocyte activation as contributing mechanisms for thrombosis in essential thrombocythemia and correlation with the JAK2 mutational status.

    abstract:BACKGROUND AND OBJECTIVES:The mechanisms accounting for the increased risk of thrombosis in patients with essential thrombocythemia (ET) are not well known. The aim of the present study was to ascertain the role of platelet and leukocyte activation in the thrombosis of ET. DESIGN AND METHODS:The activation status of p...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:

    authors: Arellano-Rodrigo E,Alvarez-Larrán A,Reverter JC,Villamor N,Colomer D,Cervantes F

    更新日期:2006-02-01 00:00:00

  • Normal thrombin generation in neonates in spite of prolonged conventional coagulation tests.

    abstract::Conventional coagulation tests might be inadequate to explore mechanisms regulating thrombin generation in neonates, because they do not allow full activation of the reduced levels of protein C. Therefore, they do not reflect the action of pro- and anti-coagulants as does the endogenous thrombin potential assessed in ...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.12566

    authors: Tripodi A,Ramenghi LA,Chantarangkul V,De Carli A,Clerici M,Groppo M,Mosca F,Mannucci PM

    更新日期:2008-08-01 00:00:00

  • Cytogenetics and long-term survival of patients with refractory or relapsed and refractory multiple myeloma treated with pomalidomide and low-dose dexamethasone.

    abstract::Patients with refractory or relapsed and refractory multiple myeloma who no longer receive benefit from novel agents have limited treatment options and short expected survival. del(17p) and t(4;14) are correlated with shortened survival. The phase 3 MM-003 trial demonstrated significant progression-free and overall su...

    journal_title:Haematologica

    pub_type: 杂志文章,多中心研究,随机对照试验

    doi:10.3324/haematol.2014.117077

    authors: Dimopoulos MA,Weisel KC,Song KW,Delforge M,Karlin L,Goldschmidt H,Moreau P,Banos A,Oriol A,Garderet L,Cavo M,Ivanova V,Alegre A,Martinez-Lopez J,Chen C,Spencer A,Knop S,Bahlis NJ,Renner C,Yu X,Hong K,Sternas L,

    更新日期:2015-10-01 00:00:00

  • Bone marrow endothelial cell-derived interleukin-4 contributes to thrombocytopenia in acute myeloid leukemia.

    abstract::Normal hematopoiesis can be disrupted by the leukemic bone marrow microenvironment, which leads to cytopenia-associated symptoms including anemia, hemorrhage and infection. Thrombocytopenia is a major and sometimes fatal complication in patients with acute leukemia. However, the mechanisms underlying defective thrombo...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2018.214593

    authors: Gao A,Gong Y,Zhu C,Yang W,Li Q,Zhao M,Ma S,Li J,Hao S,Cheng H,Cheng T

    更新日期:2019-10-01 00:00:00

  • Development of a coordinated allo T cell and auto B cell response against autosomal PTK2B after allogeneic hematopoietic stem cell transplantation.

    abstract::It is well known that allo-reactive T cells play a crucial role in graft-versus-leukemia and graft-versus-host disease after allogeneic hematopoietic stem cell transplantation (alloSCT). Allo-reactive CD4(+) T cells can mediate direct cytolysis, but may also stimulate production of IgG antibodies as helper cells. Immu...

    journal_title:Haematologica

    pub_type: 临床试验,杂志文章

    doi:10.3324/haematol.2013.086652

    authors: Kremer AN,van der Griendt JC,van der Meijden ED,Honders MW,Ayoglu B,Schwenk JM,Nilsson P,Falkenburg JH,Griffioen M

    更新日期:2014-02-01 00:00:00

  • A sequential approach with imatinib, chemotherapy and transplant for adult Ph+ acute lymphoblastic leukemia: final results of the GIMEMA LAL 0904 study.

    abstract::In the GIMEMA LAL 0904 protocol, adult Philadelphia positive acute lymphoblastic leukemia patients were treated with chemotherapy for induction and consolidation, followed by maintenance with imatinib. The protocol was subsequently amended and imatinib was incorporated in the induction and post-remission phase togethe...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2016.144535

    authors: Chiaretti S,Vitale A,Vignetti M,Piciocchi A,Fazi P,Elia L,Falini B,Ronco F,Ferrara F,De Fabritiis P,Luppi M,La Nasa G,Tedeschi A,Califano C,Fanin R,Dore F,Mandelli F,Meloni G,Foà R

    更新日期:2016-12-01 00:00:00

  • Peripheral blood or bone marrow cells in reduced-intensity or myeloablative conditioning allogeneic HLA identical sibling donor transplantation for multiple myeloma.

    abstract:BACKGROUND AND OBJECTIVES:Peripheral blood stem cells (PBSC) following reduced intensity conditioning (RIC) are being increasingly used for allogeneic transplantation in multiple myeloma. The purpose of this study was to compare outcome of patients transplanted with either PBSC or bone marrow (BM) following RIC or myel...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.11353

    authors: Gahrton G,Iacobelli S,Bandini G,Björkstrand B,Corradini P,Crawley C,Hegenbart U,Morgan G,Kröger N,Schattenberg A,Schönland SO,Verdonck LF,Volin L,de Witte T,Niederwieser D,Myeloma Subcommittee of the EBMT.

    更新日期:2007-11-01 00:00:00

  • Fibrinogen survival and fibrinopeptide A in acute leukemia.

    abstract::BACKGROUND. Hypofibrinogenemia and increased fibrin(ogen) degradation products in acute leukemia have been attributed to intravascular thrombin generation triggered by leukemic cells. However, the strict relationship between fibrinogen catabolism and turnover of fibrinopeptide A (FPA), which is a sensitive and specifi...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:

    authors: Castaman G,Galloni E,Dri AV,Rodeghiero F

    更新日期:1993-11-01 00:00:00

  • Submicroscopic deletions at 7q region are associated with recurrent chromosome abnormalities in acute leukemia.

    abstract:BACKGROUND AND OBJECTIVES:Loss of heterozygosity (LOH) on the long arm of chromosome 7 (7q) has been frequently reported in several types of human cancer including hematologic malignancies. Moreover, monosomy of chromosome 7 and 7q deletions have been associated in acute myeloid leukemia (AML) with aggressive disease a...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:

    authors: Basiricò R,Pirrotta R,Fabbiano F,Mirto S,Cascio L,Pagano M,Cammarata G,Magrin S,Santoro A

    更新日期:2003-04-01 00:00:00

  • Chromosomal alterations detected by comparative genomic hybridization in subgroups of gene expression-defined Burkitt's lymphoma.

    abstract:BACKGROUND:Burkitt's lymphoma is an aggressive B-cell lymphoma characterized by typical morphological, immunophenotypic and molecular features. Gene expression profiling provided a molecular signature of Burkitt's lymphoma, but also demonstrated that a subset of aggressive B-cell lymphomas not fulfilling the current Wo...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.13071

    authors: Salaverria I,Zettl A,Beà S,Hartmann EM,Dave SS,Wright GW,Boerma EJ,Kluin PM,Ott G,Chan WC,Weisenburger DD,Lopez-Guillermo A,Gascoyne RD,Delabie J,Rimsza LM,Braziel RM,Jaffe ES,Staudt LM,Müller-Hermelink HK,Campo E,

    更新日期:2008-09-01 00:00:00

  • Cell type specific novel lncRNAs and circRNAs in the BLUEPRINT haematopoietic transcriptomes atlas.

    abstract::Transcriptional profiling of hematopoietic cell subpopulations has helped characterize the developmental stages of the hematopoietic system and the molecular bases of malignant and non-malignant blood diseases for the past three decades. Previously, only the genes targeted by expression microarrays could be profiled g...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2019.238147

    authors: Grassi L,Izuogu OG,Jorge NA,Seyres D,Bustamante M,Burden F,Farrow S,Farahi N,Martin FJ,Frankish A,Mudge JM,Kostadima M,Petersen R,Lambourne JJ,Rowlston S,Martin-Rendon E,Clarke L,Downes K,Estivill X,Flicek P,Marte

    更新日期:2020-07-23 00:00:00

  • Allogeneic hematopoietic stem cell transplantation in myelofibrosis: the 20-year experience of the Gruppo Italiano Trapianto di Midollo Osseo (GITMO).

    abstract:BACKGROUND:Allogeneic stem cell transplantation is a potentially curative treatment for myelofibrosis, although its use is limited by a high rate of transplant-related mortality. In this study, we evaluated the outcome of patients with myelofibrosis who underwent allogeneic stem cell transplantation, and the impact of ...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.12828

    authors: Patriarca F,Bacigalupo A,Sperotto A,Isola M,Soldano F,Bruno B,van Lint MT,Iori AP,Santarone S,Porretto F,Pioltelli P,Visani G,Iacopino P,Fanin R,Bosi A,GITMO.

    更新日期:2008-10-01 00:00:00

  • Effect of PEG-recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF) on growth and differentiation of the HEL cell line.

    abstract:BACKGROUND AND OBJECTIVE:Thrombopoietin has been established as the major regulator of megakaryocyte and platelet production. In this study we evaluated the effects of PEG-recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), a pegylated and truncated form of thrombopoietin, on the growth and dif...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:

    authors: Catani L,Gugliotta L,Motta M,Tazzari P,Baravelli S,Tura S

    更新日期:1998-05-01 00:00:00

  • Prevalence and patterns of symptomatic thromboembolism in oncohematology.

    abstract:BACKGROUND AND OBJECTIVE:Approximately 15% of patients with cancer will experience a thrombotic episode at some time. Some patients are at particularly high risk depending on the histology of the malignant disease. The aim of the study was to determine the actual prevalence of thrombotic episodes in oncohematologic pat...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:

    authors: Sgarabotto D,Prandoni P,Stefani PM,Scano F,Vianello F,Sartori R,Pietrogrande F,Caenazzo A,Girolami A

    更新日期:1998-05-01 00:00:00

  • Effect of the administration of n-3 polyunsaturated fatty acids on circulating levels of microparticles in patients with a previous myocardial infarction.

    abstract:BACKGROUND:Increased levels of microparticles exposing tissue factor circulate in the blood of patients with coronary heart disease, possibly disseminating their pro-thrombotic and pro-inflammatory potential. Because diets rich in n-3 (polyunsaturated) fatty acids have been associated with reduced incidence of coronary...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.11845

    authors: Del Turco S,Basta G,Lazzerini G,Evangelista M,Rainaldi G,Tanganelli P,Christensen JH,Schmidt EB,De Caterina R

    更新日期:2008-06-01 00:00:00

  • Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade: a report from the European Group for Blood and Marrow Transplantation (EBMT).

    abstract:BACKGROUND AND OBJECTIVES:The treatment of acquired aplastic anemia (AA) is based on allogeneic bone marrow transplantation (BMT) and immunosuppressive therapy. The aim of this study was to assess the outcome of children and adults with AA treated in the last decade, and to determine whether results have improved in tw...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.10075

    authors: Locasciulli A,Oneto R,Bacigalupo A,Socié G,Korthof E,Bekassy A,Schrezenmeier H,Passweg J,Führer M,Severe Aplastic Anemia Working Party of the European Blood and Marrow Transplant Group.

    更新日期:2007-01-01 00:00:00

  • Primary mediastinal B-cell lymphoma: hypermutation of the BCL6 gene targets motifs different from those in diffuse large B-cell and follicular lymphomas.

    abstract:BACKGROUND AND OBJECTIVES:Somatic hypermutation of the BCL6 gene and its expression in lymphoma represent specific markers for B-cell transit through the germinal center. Thus, analysis of BCL6 may aid in clarifying the relationship between primary mediastinal B-cell lymphoma (PMBL) and other non-thymic diffuse large c...

    journal_title:Haematologica

    pub_type: 杂志文章,meta分析

    doi:

    authors: Malpeli G,Barbi S,Moore PS,Scardoni M,Chilosi M,Scarpa A,Menestrina F

    更新日期:2004-09-01 00:00:00

  • Identification of residual leukemic cells by flow cytometry in childhood B-cell precursor acute lymphoblastic leukemia: verification of leukemic state by flow-sorting and molecular/cytogenetic methods.

    abstract::Reduction in minimal residual disease, measured by real-time quantitative PCR or flow cytometry, predicts prognosis in childhood B-cell precursor acute lymphoblastic leukemia. We explored whether cells reported as minimal residual disease by flow cytometry represent the malignant clone harboring clone-specific genomic...

    journal_title:Haematologica

    pub_type: 杂志文章

    doi:10.3324/haematol.2011.051383

    authors: Øbro NF,Ryder LP,Madsen HO,Andersen MK,Lausen B,Hasle H,Schmiegelow K,Marquart HV

    更新日期:2012-01-01 00:00:00