Abstract:
:The diagnosis of aplastic anemia in children requires exclusion of a variety of inherited or acquired BM failure syndromes with similar phenotypes. An efficient diagnostic plan is important because time from diagnosis to 'final' treatment is directly related to outcome regardless of the therapeutic option chosen. The gold standard of therapy remains hematopoietic SCT with a graft of BM cells for those children with matched sibling donors. Conversely for children without a sibling donor the high response and markedly improved overall survival rates of combined immunosuppressive therapy have proven robust, especially when horse derived anti-thymocyte globuline plus ciclosporine A are used. Incomplete response, relapse and progression to myelodysplasia/leukemia however have emerged as significant long-term issues. Improvements in outcome of alternative donor transplantation and the use of established and novel immunosuppressive agents provide multiple alternatives for treating refractory or relapsed patients. Regardless of the type of therapeutic approach, patients require centralized treatment in a center of excellence, ongoing monitoring for recurrence of disease and/or therapy-related immediate side effects and long-term effects.
journal_name
Bone Marrow Transplantjournal_title
Bone marrow transplantationauthors
Korthof ET,Békássy AN,Hussein AAdoi
10.1038/bmt.2012.235subject
Has Abstractpub_date
2013-02-01 00:00:00pages
191-5issue
2eissn
0268-3369issn
1476-5365pii
bmt2012235journal_volume
48pub_type
杂志文章,评审abstract::Allogeneic hematopoietic cell transplantation (allo-HCT) for patients with AML is increasingly able to impact the historically poor outcomes in this disease. Nonetheless, even with transplant, the rates of post-HCT relapse are unacceptably high, and remain a great challenge in the treatment of patients with AML. Maint...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:10.1038/bmt.2016.160
更新日期:2016-11-01 00:00:00
abstract::Cyclosporine A (CsA) therapy based on 2-h concentrations (C2) after oral administration has demonstrated low acute rejection rates after solid organ transplantation. We analysed the correlation between C2 and trough (C0) levels of oral CsA therapy in samples obtained twice in consecutive weeks from 58 patients during ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705788
更新日期:2007-10-01 00:00:00
abstract::We previously reported a 25% incidence of serious graft-versus-host disease (GVHD) (that is, acute or chronic GVHD that caused death, lengthy hospitalization or disability, or resulted in recurrent major infections) among 171 hematopoietic cell transplantation (HCT) recipients after nonmyeloablative (NMA) regimen. Her...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705987
更新日期:2008-05-01 00:00:00
abstract::Graft-versus-host disease (GVHD) is a major complication of allogeneic hematopoietic stem cell transplantation. Pretransplant conditioning regimes cause release of proinflammatory cytokines that stimulate alloreactive donor T cells to attack recipient tissues. IL-10 has been shown to directly downregulate CD4+ T cells...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705218
更新日期:2006-01-01 00:00:00
abstract::Thirty-four patients diagnosed with breast cancer were included in a prospective study evaluating the bone marrow (BM) CD34+/CD71- cell content, as a predictive parameter of the CD34+ cell mobilization after rG-CSF administration. Analysis of the concentration of medullary CD34+/CD71- cells before priming schedules wa...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701227
更新日期:1998-05-01 00:00:00
abstract::We studied rates of granulocyte and platelets recovery in 359 consecutive subjects receiving blood cell infusions in the context of autotransplants for plasma cell myeloma (N = 216) and lymphomas (N = 143). Blood cells were mobilised with filgrastim given for 4-5 days and collected after a median of 2 (range, 1-2) aph...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-017-0047-7
更新日期:2018-04-01 00:00:00
abstract::The p19 dimer of interleukin 23 (IL-23) has been reported to have a major role in the pathogenesis of many experimental and clinical autoimmune diseases and may also have a prominent role in transplantation. We reasoned that deficiency of p19 in the allogeneic donor transplant might reduce the inflammation caused by a...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2009.215
更新日期:2010-04-01 00:00:00
abstract::Hormonal assessment including luteotropic hormone (LH), follicle stimulating hormone (FSH), testosterone (T), oestradiol and sex hormone binding globulin was performed 1-6 years after allogeneic, syngeneic and autologous bone marrow transplantation in nine patients with severe aplastic anaemia (SAA) and 14 patients wi...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1991-01-01 00:00:00
abstract::A potential link between arsenic (ATO)-based therapy and delayed hematopoietic recovery after autologous hematopoietic SCT (HSCT) for acute promyelocytic leukemia (APL) has previously been reported. We retrospectively reviewed the clinical histories of 58 patients undergoing autologous HSCT for APL at 21 institutions ...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1038/bmt.2014.201
更新日期:2015-01-01 00:00:00
abstract::Limited information is available regarding the incidence and features of lymphocyte expansions after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Large granular lymphocytes (LGL) expansions have been reported after bone marrow or peripheral blood, but not after unrelated cord blood (UCB) allo-HSCT, ...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/bmt.2016.364
更新日期:2017-06-01 00:00:00
abstract::Allogeneic bone marrow transplantation (BMT) is an effective therapy for a variety of malignancies and blood disorders, but rarely serves as a frontline treatment because of numerous, potential complications. Important and frequent complications relate to the profound immunosuppression that inevitably occurs during th...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1703185
更新日期:2001-09-01 00:00:00
abstract::Guillain-Barré syndrome is a rare neurologic complication after allogeneic BMT. In the non-transplant setting, Guillain-Barré syndrome has typically been associated with antecedent acute infections and numerous reports have suggested an association between Campylobacter jejuni infection and the subsequent development ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1994-03-01 00:00:00
abstract::We report a successful pregnancy in a woman who at the age of 28 years received total body irradiation (TBI; 7.5 Gy) and high-dose chemotherapy prior to autografting of purged bone marrow for acute lymphoblastic leukemia. Four years after transplantation she delivered a healthy girl. Only five previous cases of succes...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1993-12-01 00:00:00
abstract::AlloSCT is a potentially curative procedure for haematological malignancies and marrow failure syndromes. However, unlike leukaemia and lymphoproliferative disorders, AlloSCT has yet to find its place in the clinical management of patients with multiple myeloma. AlloSCT in multiple myeloma is associated with a high pr...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:10.1038/bmt.2008.397
更新日期:2009-01-01 00:00:00
abstract::Attenuated live virus vaccinations are generally recommended 24 months following hematopoietic cell transplantation (HCT) in patients not receiving immunosuppressive therapy. To date, there are no data regarding the safety of live-attenuated herpes zoster or measles-mumps-rubella (MMR) vaccinations in multiple myeloma...
journal_title:Bone marrow transplantation
pub_type: 信件
doi:10.1038/s41409-018-0112-x
更新日期:2018-07-01 00:00:00
abstract::The origin of cells in almost all allogeneic donor-recipient pairs can be determined through the use of highly polymorphic minisatellite DNA probes. Single-locus probes were cloned from hypervariable fragments in a human DNA fingerprint detected with a multi-locus probe. While each probe is highly polymorphic and locu...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1993-09-01 00:00:00
abstract::Reduced-intensity conditioning (RIC) extends hematopoietic stem cell transplants (HSCT) to elderly or debilitated patients who are not candidates for HSCT. The incidence and outcomes of cardiac complications have been reported following myeloablative HSCT. We assessed the incidence and outcomes of cardiac complication...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2009.97
更新日期:2010-01-01 00:00:00
abstract::CY in combination with BU is a widely used conditioning regimen for haematopoietic SCT (HSCT). The aim of this study was to evaluate the pharmacokinetics (PK) of CY and its major metabolite 4-hydroxyCY (HCY) in patients with thalassemia undergoing HSCT. A total of 55 patients received BU (16 mg/kg) followed by CY (160...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2011.254
更新日期:2012-09-01 00:00:00
abstract::An amendment to this paper has been published and can be accessed via a link at the top of the paper. ...
journal_title:Bone marrow transplantation
pub_type: 已发布勘误
doi:10.1038/s41409-019-0767-y
更新日期:2020-02-01 00:00:00
abstract::The purpose of this study was to evaluate the effect of partial renal shielding used in conjunction with total body irradiation (TBI) on the incidence of bone marrow transplantation nephropathy (BMT Np) seen as a late sequelae after transplantation. Of 402 patients who have undergone bone marrow transplantation (BMT) ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701022
更新日期:1997-12-01 00:00:00
abstract::We investigated the nature of hemostatic alterations occurring after bone marrow transplantation. In 45 patients, we evaluated the coagulation parameters, naturally occurring anticoagulants and thrombomodulin at days +15 and +22 after conditioning therapy. It was observed that endothelial cell damage is a central path...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1996-08-01 00:00:00
abstract::A second allograft was offered to 58 relapsed AML patients after conditioning with fludarabine 90-150 mg/m(2) and thiotepa 15 mg/kg, in most cases with active disease. Median age was 53 years (range 23-69), median time to relapse after the first allo-SCT was 326 (47-2189) days and median follow-up was 6.7 years. GVHD ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2012.267
更新日期:2013-07-01 00:00:00
abstract::Allogeneic hematopoietic SCT (allo-HCT) from matched sibling donor (MSD) is recommended for younger patients with intermediate cytogenetic risk AML in first CR (CR1), whereas the role of alternative donor transplants in these patients is unknown. We retrospectively analyzed 605 patients with intermediate-risk AML, who...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2012.84
更新日期:2013-01-01 00:00:00
abstract::Approaches to determine whether one transplant-related therapy is better than another include: (1) using experimental data, such as those from randomized controlled trials (RCTs); (2) using observational data, such as those from observational databases (ODBs) and (3) using conclusions from the structured quantificatio...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2008.447
更新日期:2009-03-01 00:00:00
abstract::We designed a phase I clinical trial of escalating doses of topotecan with CY and carboplatin in combination with autologous hematopoietic SCT (AHSCT) for the treatment of relapsed or persistent platinum sensitive ovarian or primary peritoneal carcinoma. After stem cell collection, 16 patients received topotecan at 1....
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2009.181
更新日期:2010-03-01 00:00:00
abstract::A 55-year-old man with acute myeloid leukemia in second relapse presented 4 months after haploidentical CD34+-selected hematopoietic stem cell transplantation (HSCT) with symmetric, progressive neurological deficits of the lower extremities. Although there was no molecular evidence for drug resistance in the cerebral-...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704311
更新日期:2004-01-01 00:00:00
abstract::We analyzed late cardiovascular outcomes of 661 patients who survived at least 2 years from hematopoietic cell transplantation for childhood hematologic malignancy between 1995 and 2008. Center for International Blood and Marrow Transplant Research data was supplemented with surveys focused on cardiotoxicity and poten...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-018-0155-z
更新日期:2018-10-01 00:00:00
abstract::Peripheral T cell lymphomas (PTCL) have a poorer prognosis after conventional treatment than do high-grade B cell lymphomas. The place for high-dose therapy (HDT) with autologous stem cell support in these patients is still not clear. Forty patients, 10 women and 30 men, median age 41.5 years (range 16-61) with PTCL w...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,多中心研究
doi:10.1038/sj.bmt.1702867
更新日期:2001-04-01 00:00:00
abstract::Sickle cell anemia (SCA) remains associated with high risks of morbidity and early death. Allogeneic hematopoietic SCT (HSCT) is the only curative treatment for SCA. We report our experience with transplantation in a group of patients with the non-Black African variant and the Black African variant of SCA. This study ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2014.167
更新日期:2014-11-01 00:00:00
abstract::In this retrospective study, we evaluated the predictability of PBSC dose for hematopoietic engraftment comparing that calculated by ideal body weight (IBW) vs another calculated by actual body weight (ABW) for each patient. Sixty-three consecutive patients treated similarly using one transplant protocol were analyzed...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701731
更新日期:1999-05-01 00:00:00