Abstract:
:Outcomes after relapse of childhood B-acute lymphoblastic leukemia (B-ALL) are poor, and optimal therapy is unclear. Children's Oncology Group study AALL0433 evaluated a new platform for relapsed ALL. Between March 2007 and October 2013 AALL0433 enrolled 275 participants with late bone marrow or very early isolated central nervous system (iCNS) relapse of childhood B-ALL. Patients were randomized to receive standard versus intensive vincristine dosing; this randomization closed due to excess peripheral neuropathy in 2010. Patients with matched sibling donors received allogeneic hematopoietic cell transplantation (HCT) after the first three blocks of therapy. The prognostic value of minimal residual disease (MRD) was also evaluated in this study. The 3-year event free and overall survival (EFS/OS) for the 271 eligible patients were 63.6% +/- 3.0% and 72.3% +/- 2.8% respectively. MRD at the end of Induction-1 was highly predictive of outcome, with 3-year EFS/OS of 84.9 +/- 4.0% and 93.8 +/- 2.7% for patients with MRD <0.1%, vs. 53.7 +/- 7.8% and 60.6 +/- 7.8% for patients with MRD ≥0.1% (p<0.0001). Patients who received HCT vs. chemotherapy alone had an improved 3-year disease-free survival (77.5 +/- 6.2% vs. 66.9 +/- 4.5%, p=0.03) but not OS (81.5 +/- 5.8% for HCT vs. 85.8 +/- 3.4% for chemotherapy, p=0.46). Patients with early iCNS relapse fared poorly, with a 3-year EFS/OS of 41.4% +/- 9.2% and 51.7% +/- 9.3%, respectively. Infectious toxicities of the chemotherapy platform were significant. The AALL0433 chemotherapy platform is efficacious for late bone marrow relapse of B-ALL, but with significant toxicities. The MRD threshold of 0.1% at the end of Induction-1 was highly predictive of outcome. The optimal role for HCT for this patient population remains uncertain. This trial is registered at clinicaltrials.gov (NCT# 00381680).
journal_name
Haematologicajournal_title
Haematologicaauthors
Lew G,Chen Y,Lu X,Rheingold SR,Whitlock JA,Devidas M,Hastings CA,Winick NJ,Carroll WL,Wood BL,Borowitz MJ,Pulsipher MA,Hunger SPdoi
10.3324/haematol.2019.237230subject
Has Abstractpub_date
2021-01-01 00:00:00pages
46-55issue
1eissn
0390-6078issn
1592-8721journal_volume
106pub_type
杂志文章相关文献
HAEMATOLOGICA文献大全abstract::Germline RUNX1 mutations lead to thrombocytopenia and platelet dysfunction in familial platelet disorder with predisposition to acute myelogenous leukemia (AML). Multiple aspects of platelet function are impaired in these patients, associated with altered expression of genes regulated by RUNX1 We aimed to identify RUN...
journal_title:Haematologica
pub_type: 杂志文章
doi:10.3324/haematol.2018.188904
更新日期:2019-06-01 00:00:00
abstract::In ribosomopathies, the Diamond-Blackfan anemia (DBA) or 5q- syndrome, ribosomal protein (RP) genes are affected by mutation or deletion, resulting in bone marrow erythroid hypoplasia. Unbalanced production of ribosomal subunits leading to a limited ribosome cellular content, regulates translation at the expense of th...
journal_title:Haematologica
pub_type: 杂志文章
doi:10.3324/haematol.2019.239970
更新日期:2020-04-23 00:00:00
abstract::Fludarabine (FLU) is a new antimetabolite chemotherapeutic agent with promising activity in lymphoproliferative disorders and, in particular, in low-grade non-Hodgkin's lymphoma (LG-NHL). Recently, a few reports have described interesting results using FLU in polychemotherapy regimens. In order to evaluate FLU in comb...
journal_title:Haematologica
pub_type: 临床试验,杂志文章
doi:
更新日期:1996-03-01 00:00:00
abstract::A prospective neurological and electroencephalographic (EEG) study was performed in sixteen leukemia patients receiving busulfan (BU) and cyclophosphamide before autologous bone marrow transplantation. All patients were given anticonvulsant prophylaxis with a combination of phenobarbital (PB) and clonazepam (CLZ). Neu...
journal_title:Haematologica
pub_type: 临床试验,杂志文章
doi:
更新日期:1995-11-01 00:00:00
abstract::Suspicion of myelodysplastic syndromes (MDS) is one of the commonest reasons for bone marrow aspirate in elderly patients presenting with persistent peripheral blood (PB) cytopenia of unclear etiology. A PB assay that accurately rules out MDS would have major benefits. The diagnostic accuracy of the intra-individual r...
journal_title:Haematologica
pub_type: 杂志文章
doi:10.3324/haematol.2018.202275
更新日期:2019-12-01 00:00:00
abstract::It is well known that thalassemic patients exhibit an increased frequency of thrombotic events. Most individuals with resistance to activated protein C (APCR) are the result of a point mutation replacing Arg 506 with Gln in the factor V aminoacidic sequence (factor V Leiden). Recently APCR has been shown to account fo...
journal_title:Haematologica
pub_type: 杂志文章
doi:
更新日期:1997-11-01 00:00:00
abstract::Alemtuzumab serum levels and clinical response after subcutaneous administration (10 mg 3 times/week for six weeks) have been explored in 29 chronic lymphocytic leukemia patients receiving the monoclonal antibody as consolidation. Serum concentrations after each administration gradually increased during the first week...
journal_title:Haematologica
pub_type: 临床试验,杂志文章
doi:10.3324/haematol.2010.033159
更新日期:2011-06-01 00:00:00
abstract::A case of Di Guglielmo's syndrome passed through the three stages of chronic erythromyelosis, erythroleukemia and acute myeloid leukemia (AML). According to the FAB classification the subsequent stages of this syndrome were refractory anemia (RA), RA with excess of blasts (RAEB), AML-M6, AML-M2 and undifferentiated AM...
journal_title:Haematologica
pub_type: 杂志文章
doi:
更新日期:1997-07-01 00:00:00
abstract:PURPOSE:Various experiences show no substantial differences between children and adults in the treatment of Hodgkin's disease. In consideration of some peculiar characteristics of these cases which might influence the therapeutical choice, particularly regarding long-term survival and therapeutical complications, we re...
journal_title:Haematologica
pub_type: 杂志文章
doi:
更新日期:1996-05-01 00:00:00
abstract::To our knowledge, there is little information about functional thrombin activatable fibrinolysis inhibitor (TAFI) levels and the risk of acute coronary artery disease (CAD). We investigated the risk of acute CAD related to plasma levels of functional TAFI. We found that functional TAFI levels in plasma (above 126%), i...
journal_title:Haematologica
pub_type: 信件
doi:
更新日期:2004-07-01 00:00:00
abstract::Hemophagocytic lymphohistiocytosis is a life-threatening systemic hyperinflammatory disorder with primary and secondary forms. Primary hemophagocytic lymphohistiocytosis is associated with inherited defects in various genes that affect the immunological cytolytic pathway. Secondary hemophagocytic lymphohistiocytosis i...
journal_title:Haematologica
pub_type: 杂志文章
doi:10.3324/haematol.2018.209551
更新日期:2019-10-01 00:00:00
abstract::This review deals with the chronic lymphoproliferative disease of large granular lymphocytes endowed with T and natural killer cell characteristics. The disease is sufficiently characterized to allow its distinction from other lymphoproliferative disorders of the T cell type. The heterogeneous clinical course and labo...
journal_title:Haematologica
pub_type: 杂志文章,评审
doi:
更新日期:1989-01-01 00:00:00
abstract::To date, few mutations associated with a dominant quantitative deficiency of von Willebrand factor (VWF) and a high penetrance have been reported. This phenotype was confirmed in seven unrelated families of several patients diagnosed with von Willebrand's disease out of 70 who requested genetic studies of the VWF gene...
journal_title:Haematologica
pub_type: 杂志文章
doi:
更新日期:2006-08-01 00:00:00
abstract::Next generation sequencing technologies have provided insights into the molecular heterogeneity of various myeloid neoplasms, revealing previously unknown somatic genetic events. In our cohort of 1444 cases analyzed by next generation sequencing, somatic mutations in the gene BRCA1-BRCA2-containing complex 3 (BRCC3) w...
journal_title:Haematologica
pub_type: 杂志文章
doi:10.3324/haematol.2014.111989
更新日期:2015-08-01 00:00:00
abstract::Our preliminary results suggest the existence of quantitative and qualitative differences in immune cells and type1 and type2 cytokines between granulocyte colony-stimulating factor (G-CSF) primed bone marrow (G-BM) and G-CSF-mobilized peripheral blood grafts (G-PB). Our findings suggest that lower T-cell hyporesponsi...
journal_title:Haematologica
pub_type: 信件
doi:
更新日期:2005-05-01 00:00:00
abstract:BACKGROUND AND OBJECTIVES:The efficacy of antithymocyte globulin (ATG) in the treatment of graft-versus-host disease (GvHD) is controversial. In the present study we report on the use of low dose ATG (thymoglobuline, Sangstat) and steroids in 28 patients with moderate to severe acute GvHD. DESIGN AND METHODS:Fifteen p...
journal_title:Haematologica
pub_type: 临床试验,杂志文章,随机对照试验
doi:
更新日期:2002-09-01 00:00:00
abstract::Hematopoietic stem cell (HSC) aging was originally thought to be essentially an HSC-autonomous process, which is the focus of another review in the same issue of Haematologica However, studies on the microenvironment that maintains and regulates HSC (HSC niche) over the past 20 years have suggested that microenvironme...
journal_title:Haematologica
pub_type: 杂志文章,评审
doi:10.3324/haematol.2018.211334
更新日期:2020-01-01 00:00:00
abstract::Mechanistic target of rapamycin (mTOR) complex 1 is a central integrator of nutrient and growth factor inputs that controls cell growth in eukaryotes. The second generation of mTOR kinase inhibitors (TORKi), directly targeting the mTOR catalytic site, are more effective than rapamycin and its analogs in cancer treatme...
journal_title:Haematologica
pub_type: 杂志文章
doi:10.3324/haematol.2016.159160
更新日期:2017-04-01 00:00:00
abstract::Myeloproliferative neoplasms are a varied group of disorders that can have prolonged chronic phases, but eventually accelerate and can transform into a secondary acute myeloid leukemia that is ultimately fatal. Triapine is a novel inhibitor of the M2 subunit of ribonucleotide reductase. Sequential inhibition of ribonu...
journal_title:Haematologica
pub_type: 杂志文章
doi:10.3324/haematol.2013.097246
更新日期:2014-04-01 00:00:00
abstract::There is emerging consensus that a pro-inflammatory condition contributes to the vaso-occlusive complications of sickle cell disease (SCD). We evaluated the potential value of inflammatory mediators as early markers of severity of painful vaso-occlusive crises (VOC) in SCD. We assayed the plasma levels of cytokines, s...
journal_title:Haematologica
pub_type: 信件
doi:
更新日期:2004-07-01 00:00:00
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
更新日期:2008-09-01 00:00:00
abstract:BACKGROUND AND OBJECTIVE:The expression of CD36 antigen has not been conclusively associated with human B-lymphocytes although CD36 was recently detected in a human B-cell angiotropic lymphoma where it might be involved in lymphoblast-endothelial cell adhesion. We investigated the expression of CD36 in B-cell chronic l...
journal_title:Haematologica
pub_type: 临床试验,杂志文章
doi:
更新日期:1999-05-01 00:00:00
abstract::Krüppel-like factor 4 is a transcription factor with anti-proliferative effects in differentiated cells, but with the ability to reprogram adult cells into cell-cycling pluripotent cells. In cancer, Krüppel-like factor 4 acts as either an anti-oncogene or an oncogene. We analyzed Krüppel-like factor 4 gene expression ...
journal_title:Haematologica
pub_type: 杂志文章
doi:10.3324/haematol.2012.066944
更新日期:2013-09-01 00:00:00
abstract::Daily intravenous arsenic trioxide administered with all-trans retinoid acid, the standard-of-care for acute promyelocytic leukemia, is costly and challenging to administer. ORH-2014 is a novel, oral arsenic trioxide formulation, consisting of micron-size drug particles with rapid dissolution and high bioavailability....
journal_title:Haematologica
pub_type: 杂志文章
doi:10.3324/haematol.2019.229583
更新日期:2020-06-01 00:00:00
abstract:BACKGROUND AND OBJECTIVE:Telomerase is the enzyme that stabilizes and elongates the telomeric ends of chromosomes. It is expressed in germline and malignant cells and absent in most human somatic cells. The selective expression of telomerase has thus been proposed to be a basis for the immortality of germline and malig...
journal_title:Haematologica
pub_type: 杂志文章
doi:
更新日期:1997-05-01 00:00:00
abstract:BACKGROUND:The risk for donors of allogeneic hematopoietic stem cells transplants is generally considered negligible. Scattered reports of severe complications and a recent controversy on hematopoietic malignancies after granulocyte colony-stimulating factor administration have challenged this opinion. DESIGN AND METH...
journal_title:Haematologica
pub_type: 杂志文章
doi:10.3324/haematol.13668
更新日期:2009-01-01 00:00:00
abstract::Patients with advanced myeloma experience a high symptom burden particularly near the end of life, making timely hospice use crucial. Little is known about the quality and determinants of end-of-life care for this population, including whether potential increases in hospice use are also accompanied by "late" enrollmen...
journal_title:Haematologica
pub_type: 杂志文章
doi:10.3324/haematol.2018.187609
更新日期:2018-08-01 00:00:00
abstract::Extramedullary (EM) disease in patients with acute myeloid leukemia (AML) is a known phenomenon. Since the prevalence of EM AML has so far only been clinically determined on examination, we performed a prospective study in patients with AML. The aim of the study was to determine the prevalence of metabolically active ...
journal_title:Haematologica
pub_type: 杂志文章
doi:10.3324/haematol.2019.223032
更新日期:2020-06-01 00:00:00
abstract:BACKGROUND:The purpose of our cooperative trial was to investigate whether epirubicin (EPI) at 90 mg/m2 in a CHOP-like combination (called CEOP) could increase complete response (CR) and survival rates in non-Hodgkin lymphoma (NHL) patients while maintaining a tolerable degree of toxicity. METHODS:Between September 19...
journal_title:Haematologica
pub_type: 临床试验,杂志文章,多中心研究
doi:
更新日期:1995-07-01 00:00:00
abstract::Platelet-type von Willebrand disease (PT-VWD) is a rare autosomal dominant bleeding disorder which is due to a mutation in the gene encoding for platelet glycoprotein Ibalpha (GPIbalpha) resulting in enhanced affinity for von Willebrand factor (VWF). PT-VWD is often mistakenly diagnosed as type 2B VWD for the similari...
journal_title:Haematologica
pub_type: 杂志文章
doi:10.3324/haematol.2009.015990
更新日期:2010-06-01 00:00:00