Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease.

Abstract:

:We developed a risk score to predict event-free survival (EFS) after allogeneic hematopoietic cell transplantation for sickle cell disease. The study population (n = 1425) was randomly split into training (n = 1070) and validation (n = 355) cohorts. Risk factors were identified and validated via Cox regression models. Two risk factors of 9 evaluated were predictive for EFS: age at transplantation and donor type. On the basis of the training cohort, patients age 12 years or younger with an HLA-matched sibling donor were at the lowest risk with a 3-year EFS of 92% (score, 0). Patients age 13 years or older with an HLA-matched sibling donor or age 12 years or younger with an HLA-matched unrelated donor were at intermediate risk (3-year EFS, 87%; score, 1). All other groups, including patients of any age with a haploidentical relative or HLA-mismatched unrelated donor and patients age 13 years or older with an HLA-matched unrelated donor were high risk (3-year EFS, 57%; score, 2 or 3). These findings were confirmed in the validation cohort. This simple risk score may guide patients with sickle cell disease and hematologists who are considering allogeneic transplantation as a curative treatment relative to other available contemporary treatments.

journal_name

Blood

journal_title

Blood

authors

Brazauskas R,Scigliuolo GM,Wang HL,Cappelli B,Ruggeri A,Fitzhugh CD,Hankins JS,Kanter J,Meerpohl JJ,Panepinto JA,Rondelli D,Shenoy S,Walters MC,Wagner JE,Tisdale JF,Gluckman E,Eapen M

doi

10.1182/blood.2020005687

subject

Has Abstract

pub_date

2020-07-30 00:00:00

pages

623-626

issue

5

eissn

0006-4971

issn

1528-0020

pii

460738

journal_volume

136

pub_type

杂志文章

相关文献

BLOOD文献大全
  • IGF-1 suppresses Bim expression in multiple myeloma via epigenetic and posttranslational mechanisms.

    abstract::Insulin-like growth factor-1 (IGF-1) is an important growth and survival factor in multiple myeloma (MM). Here, we demonstrate that IGF-1 induces significant down-regulation of the proapoptotic BH3-only protein Bim in MM cells. Reduced Bim levels by RNA interference (RNAi) protected cells from drug-induced cell death....

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2009-07-232801

    authors: De Bruyne E,Bos TJ,Schuit F,Van Valckenborgh E,Menu E,Thorrez L,Atadja P,Jernberg-Wiklund H,Vanderkerken K

    更新日期:2010-03-25 00:00:00

  • Epstein-Barr virus latent membrane protein-1 oncogene deletions: correlations with malignancy in Epstein-Barr virus--associated lymphoproliferative disorders and malignant lymphomas.

    abstract::LMP-1, an Epstein-Barr viral (EBV) latency protein, is considered a viral oncogene because of its ability to transform rodent fibroblasts in vivo and render them tumorigenic in nude mice. In human B cells, EBV LMP-1 induces DNA synthesis and abrogates apoptosis. LMP-1 is expressed in EBV-transformed lymphoblastoid cel...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Kingma DW,Weiss WB,Jaffe ES,Kumar S,Frekko K,Raffeld M

    更新日期:1996-07-01 00:00:00

  • The HRI-regulated transcription factor ATF4 activates BCL11A transcription to silence fetal hemoglobin expression.

    abstract::Reactivation of fetal hemoglobin remains a critical goal in the treatment of patients with sickle cell disease and β-thalassemia. Previously, we discovered that silencing of the fetal γ-globin gene requires the erythroid-specific eIF2α kinase heme-regulated inhibitor (HRI), suggesting that HRI might present a pharmaco...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood.2020005301

    authors: Huang P,Peslak SA,Lan X,Khandros E,Yano JA,Sharma M,Keller CA,Giardine B,Qin K,Abdulmalik O,Hardison RC,Shi J,Blobel GA

    更新日期:2020-06-11 00:00:00

  • Autologous and MHC class I-negative allogeneic tumor cells secreting IL-12 together cure disseminated A20 lymphoma.

    abstract::Cytokine gene-modified tumor cells have increased immunogenicity and retain the antigenic repertoire of a particular neoplasia. However, practical concerns have led to an increased interest in allogeneic gene-transduced bystander cells as a broader source of cytokines for autologous tumor cell-based vaccines. Here, we...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2002-03-0991

    authors: Curti A,Parenza M,Colombo MP

    更新日期:2003-01-15 00:00:00

  • Transmaternal cell flow leads to antigen-experienced cord blood.

    abstract::Umbilical cord blood (UCB) is used for HSCT. It is known that UCB can comprise Ag-specific T cells. Here we question whether solely transmaternal cell flow may immunize UCB. Twenty-three female UCB samples were collected from healthy mothers and analyzed for minor histocompatibility Ag HY-specific responses. Forty-two...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2012-02-410571

    authors: Dierselhuis MP,Blokland EC,Pool J,Schrama E,Scherjon SA,Goulmy E

    更新日期:2012-07-19 00:00:00

  • Epithin/PRSS14 proteolytically regulates angiopoietin receptor Tie2 during transendothelial migration.

    abstract::Epithin/PRSS14, a type II transmembrane serine protease, is involved in normal epithelial development and tumor progression. Here we report, as an interacting substrate of epithin, a receptor tyrosine kinase Tie2 that is well known for important roles in the vessel stability. Epithin interacts with and degrades the Ti...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2010-03-275289

    authors: Kim C,Lee HS,Lee D,Lee SD,Cho EG,Yang SJ,Kim SB,Park D,Kim MG

    更新日期:2011-01-27 00:00:00

  • The principal results of the International Immune Tolerance Study: a randomized dose comparison.

    abstract::The International Immune Tolerance Study was a multicenter, prospective, randomized comparison of high-dose (HD; 200 IU/kg/d) and low-dose (LD; 50 IU/kg 3 times/week) factor VIII regimens in 115 "good-risk," severe high-titer inhibitor hemophilia A subjects. Sixty-six of 115 subjects reached the defined study end poin...

    journal_title:Blood

    pub_type: 杂志文章,多中心研究,随机对照试验

    doi:10.1182/blood-2011-08-369132

    authors: Hay CR,DiMichele DM,International Immune Tolerance Study.

    更新日期:2012-02-09 00:00:00

  • Membrane cholesterol regulates LFA-1 function and lipid raft heterogeneity.

    abstract::Many surface receptors and signaling molecules are thought to associate with unique membrane microdomains termed lipid rafts. We examined the involvement of lipid rafts in the activation of leukocyte function-associated antigen-1 (LFA-1). Depletion or sequestration of cholesterol with methyl-beta-cyclodextrin (MCD) or...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2002-10-3195

    authors: Marwali MR,Rey-Ladino J,Dreolini L,Shaw D,Takei F

    更新日期:2003-07-01 00:00:00

  • The presence of novel amino acids in the cytoplasmic domain of stem cell factor results in hematopoietic defects in Steel(17H) mice.

    abstract::Stem cell factor (SCF) is expressed as an integral membrane growth factor that may be differentially processed to produce predominantly soluble (S) (SCF(248)) or membrane-associated (MA) (SCF(220)) protein. A critical role for membrane presentation of SCF in the hematopoietic microenvironment (HM) has been suggested f...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Kapur R,Cooper R,Xiao X,Weiss MJ,Donovan P,Williams DA

    更新日期:1999-09-15 00:00:00

  • Fibrinogen Bern I: substitution gamma 337 Asn-->Lys is responsible for defective fibrin monomer polymerization.

    abstract::An inherited fibrinogen variant, fibrinogen Bern I, was isolated from plasma of an asymptomatic woman. Routine coagulation studies showed prolonged thrombin and reptilase clotting times. Fibrinogen concentration was diminished when determined by a functional assay, but was normal by the heat precipitation method. The ...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Steinmann C,Reber P,Jungo M,Lämmle B,Heinemann G,Wermuth B,Furlan M

    更新日期:1993-10-01 00:00:00

  • Disturbed sialic acid recognition on endothelial cells and platelets in complement attack causes atypical hemolytic uremic syndrome.

    abstract::Uncontrolled activation of the complement system against endothelial and blood cells is central to the pathogenesis of atypical hemolytic uremic syndrome (aHUS). aHUS patients frequently carry mutations in the inhibitory complement regulator factor H (FH). Mutations cluster in domains 19 and 20 (FH19-20), which are cr...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2015-11-680009

    authors: Hyvärinen S,Meri S,Jokiranta TS

    更新日期:2016-06-02 00:00:00

  • G6PD isoenzyme analysis of myeloid and lymphoid cells in human multilineage colonies.

    abstract::Some multilineage hemopoietic colonies contain, in addition to myeloid cells, T lymphocytes. These proliferate extensively in liquid suspension culture under the influence of a T cell growth factor provided by phytohemagglutinin-T cell-conditioned medium (PHA-TCM). The clonal origin of these myeloid and lymphoid compo...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Lim B,Jamal N,Tritchler D,Messner HA

    更新日期:1984-06-01 00:00:00

  • Activation of human platelets by the rabbit anticardiolipin antibodies.

    abstract::Affinity purified anticardiolipin antibodies (ACLA) raised in rabbits showed cross-reactivities with various negatively charged phospholipids as shown by both the solid phase enzyme-linked immunosorbent assay (ELISA) and inhibition studies. In ELISA, ACLA showed strong cross-reactivity to both sphingomyelin (SM) and p...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Lin YL,Wang CT

    更新日期:1992-12-15 00:00:00

  • Demonstration and mode of action of an inhibitor for activated Hageman factor (factor XIIa) of the intrinsic blood coagulation pathway from Schistosoma mansoni.

    abstract::An anticoagulant activity from adult Schistosoma mansoni whole worm homogenate is described. The inhibitor appears to be specific for the contact activation step of the intrinsic pathway. Experiments with both human and mouse plasmas have defined the specificity of the inhibitor as follows: (1) It lengthens the partia...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Tsang VC,Damian RT

    更新日期:1977-04-01 00:00:00

  • Sustained signaling through the B-cell receptor induces Mcl-1 and promotes survival of chronic lymphocytic leukemia B cells.

    abstract::The clinical course of chronic lymphocytic leukemia (CLL) differs significantly between patients with mutated (M-CLL) and unmutated (U-CLL) immunoglobulin (Ig) variable heavy-chain (V(H)) genes, implying a role for B-cell receptor (BCR) signaling in the pathogenesis of this disease. We have now investigated activation...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2004-07-2669

    authors: Petlickovski A,Laurenti L,Li X,Marietti S,Chiusolo P,Sica S,Leone G,Efremov DG

    更新日期:2005-06-15 00:00:00

  • Retinoic acid inhibits CD40 + interleukin-4-mediated IgE production in vitro.

    abstract::To elucidate the role of retinoic acid (RA) in anti-CD40 + interleukin-4 (IL-4)-mediated B-cell activation, the effect of 10(-12) to 10(-6) mol/L RA was studied in anti-CD40 (1 microgram/mL) + IL-4 (5 ng/mL)-mediated proliferation and Ig synthesis by human peripheral blood mononuclear cells (PBMC) and B cells in healt...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Worm M,Krah JM,Manz RA,Henz BM

    更新日期:1998-09-01 00:00:00

  • Beyond hairy cell: the activity of cladribine in other hematologic malignancies.

    abstract::Before the contemporary development of rationally designed antineoplastic therapies, cladribine was identified as a lymphocyte-specific agent. Its profound impact on the natural history of hairy cell leukemia, with responses approaching 100% and a median duration of response of nearly a decade after only a single 7-da...

    journal_title:Blood

    pub_type: 杂志文章,评审

    doi:10.1182/blood-2010-02-246140

    authors: Sigal DS,Miller HJ,Schram ED,Saven A

    更新日期:2010-10-21 00:00:00

  • JMML and RALD (Ras-associated autoimmune leukoproliferative disorder): common genetic etiology yet clinically distinct entities.

    abstract::Ras-associated autoimmune leukoproliferative disorder (RALD) is a chronic, nonmalignant condition that presents with persistent monocytosis and is often associated with leukocytosis, lymphoproliferation, and autoimmune phenomena. RALD has clinical and laboratory features that overlap with those of juvenile myelomonocy...

    journal_title:Blood

    pub_type: 杂志文章,评审

    doi:10.1182/blood-2014-11-567917

    authors: Calvo KR,Price S,Braylan RC,Oliveira JB,Lenardo M,Fleisher TA,Rao VK

    更新日期:2015-04-30 00:00:00

  • Comparison of activated protein C/protein S-mediated inactivation of human factor VIII and factor V.

    abstract::The proteolytic cleavage and subsequent inactivation of recombinant human factor VIII (rhFVIII) and human factor VIIIa (rhFVIIIa) by recombinant human activated protein C (rAPC) was analyzed in the presence and absence of human protein S and human factor V (FV). Membrane-bound rhFVIIIa spontaneously looses most of its...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Lu D,Kalafatis M,Mann KG,Long GL

    更新日期:1996-06-01 00:00:00

  • DNMT3A and TET2 dominate clonal hematopoiesis and demonstrate benign phenotypes and different genetic predispositions.

    abstract::Age-associated clonal hematopoiesis caused by acquired mutations in myeloid cancer-associated genes is highly prevalent in the normal population. Its etiology, biological impact on hematopoiesis, and oncogenic risk is poorly defined at this time. To gain insight into this phenomenon, we analyzed a cohort of 2530 relat...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2017-04-777029

    authors: Buscarlet M,Provost S,Zada YF,Barhdadi A,Bourgoin V,Lépine G,Mollica L,Szuber N,Dubé MP,Busque L

    更新日期:2017-08-10 00:00:00

  • How I manage ibrutinib-refractory chronic lymphocytic leukemia.

    abstract::The introduction of the Bruton tyrosine kinase (BTK) inhibitor ibrutinib has dramatically changed the management of chronic lymphocytic leukemia (CLL). Although responses have been durable in the majority of patients, relapses do occur, especially in the high-risk patient population. Most relapses occur as the result ...

    journal_title:Blood

    pub_type: 杂志文章,评审

    doi:10.1182/blood-2016-09-693598

    authors: Woyach JA

    更新日期:2017-03-09 00:00:00

  • Notch1 and TGFbeta1 cooperatively regulate Foxp3 expression and the maintenance of peripheral regulatory T cells.

    abstract::Notch and its ligands have been implicated in the regulation and differentiation of various CD4(+) T-helper cells. Regulatory T cells (T(regs)), which express the transcription factor Foxp3, suppress aberrant immune responses that are typically associated with autoimmunity or excessive inflammation. Previous studies h...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2008-03-144980

    authors: Samon JB,Champhekar A,Minter LM,Telfer JC,Miele L,Fauq A,Das P,Golde TE,Osborne BA

    更新日期:2008-09-01 00:00:00

  • K-Ras is essential for normal fetal liver erythropoiesis.

    abstract::In vitro studies suggest that Ras activation is necessary for erythroid cell development. However, genetic inactivation of the Ras isoforms H-Ras, N-Ras, and K-Ras in mice reportedly did not affect adult or fetal erythropoiesis, though K-Ras(-/-) embryos were anemic. Given these discrepancies, we performed a more deta...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2004-05-2021

    authors: Khalaf WF,White H,Wenning MJ,Orazi A,Kapur R,Ingram DA

    更新日期:2005-05-01 00:00:00

  • Whole genome scanning as a cytogenetic tool in hematologic malignancies.

    abstract::Over the years, methods of cytogenetic analysis evolved and became part of routine laboratory testing, providing valuable diagnostic and prognostic information in hematologic disorders. Karyotypic aberrations contribute to the understanding of the molecular pathogenesis of disease and thereby to rational application o...

    journal_title:Blood

    pub_type: 杂志文章,评审

    doi:10.1182/blood-2008-02-130435

    authors: Maciejewski JP,Mufti GJ

    更新日期:2008-08-15 00:00:00

  • Evidence for a pretranslational defect in hereditary and acquired myeloperoxidase deficiency.

    abstract::Myeloperoxidase (MPO) is a heme containing enzyme involved in the oxygen-dependent microbicidal activity of human polymorphonuclear leukocytes (PMN). Complete hereditary and acquired MPO deficiencies are defined as lack of peroxidase activity in PMN. Using this criterion, we studied a patient with complete hereditary ...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Tobler A,Selsted ME,Miller CW,Johnson KR,Novotny MJ,Rovera G,Koeffler HP

    更新日期:1989-05-15 00:00:00

  • Anti-CD33 monoclonal antibody and etoposide/cytosine arabinoside combinations for the ex vivo purification of bone marrow in acute nonlymphocytic leukemia.

    abstract::Pharmacologic and immunologic methods of ex-vivo bone marrow (BM) purging for acute nonlymphocytic leukemia (ANLL) were combined to augment the effect of either method alone. Etoposide (VP16; 20 to 30 micrograms/mL) with or without cytosine arabinoside (Ara C; 10 mg/mL) was used in tandem with the anti-CD33 monoclonal...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Stiff PJ,Schulz WC,Bishop M,Marks L

    更新日期:1991-01-15 00:00:00

  • CD62L expression identifies a unique subset of polyfunctional CD56dim NK cells.

    abstract::Human natural killer (NK) cells comprise 2 main subsets, CD56(bright) and CD56(dim) cells, that differ in function, phenotype, and tissue localization. To further dissect the heterogeneity of CD56(dim) cells, we have performed transcriptome analysis and functional ex vivo characterization of human NK-cell subsets acco...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2009-11-253286

    authors: Juelke K,Killig M,Luetke-Eversloh M,Parente E,Gruen J,Morandi B,Ferlazzo G,Thiel A,Schmitt-Knosalla I,Romagnani C

    更新日期:2010-08-26 00:00:00

  • Thrombopoietin upregulates the promoter conformation of p53 in a proliferation-independent manner coincident with a decreased expression of Bax: potential mechanisms for survival enhancing effects.

    abstract::Thrombopoietin (Tpo) has proliferative and maturational effects on immature and more committed cells, respectively. We previously reported a role for Tpo as a survival factor in the factor-dependent human cell line M07e by demonstrating that Tpo suppresses apoptosis in the absence of induced proliferation. Wild-type p...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Ritchie A,Gotoh A,Gaddy J,Braun SE,Broxmeyer HE

    更新日期:1997-12-01 00:00:00

  • Deletion of the zinc-binding motif of CD13/aminopeptidase N molecules results in loss of epitopes that mediate binding of inhibitory antibodies.

    abstract::The myeloid cell-surface glycoprotein CD13/aminopeptidase N (APN; EC 3.4.11.2) contains a pentapeptide (HExxH) in its extracellular domain that is characteristic of many zinc-dependent metalloproteinases. This region contains residues important for zinc binding and constitutes part of the catalytic domain of several m...

    journal_title:Blood

    pub_type: 杂志文章

    doi:

    authors: Ashmun RA,Shapiro LH,Look AT

    更新日期:1992-06-15 00:00:00

  • Heterogeneity in Fanconi anemia: evidence for 2 new genetic subtypes.

    abstract::Fanconi anemia (FA) is an autosomal recessive syndrome featuring diverse symptoms including progressive bone marrow failure and early occurrence of acute myeloid leukemia. Nine genetic subtypes have been described for FA (A, B, C, D1, D2, E, F, G, and L), all of which have been connected to distinct disease genes, exc...

    journal_title:Blood

    pub_type: 杂志文章

    doi:10.1182/blood-2003-08-2915

    authors: Levitus M,Rooimans MA,Steltenpool J,Cool NF,Oostra AB,Mathew CG,Hoatlin ME,Waisfisz Q,Arwert F,de Winter JP,Joenje H

    更新日期:2004-04-01 00:00:00