Abstract:
:Four human fetuses were treated by transplantation of human fetal liver stem cells. Two of them had severe immunodeficiency disease and the two other ones had thalassemia major. Three of these in utero transplants were followed by engraftment. The three patients are now born: the first one is now very healthy thanks to the reconstitution of cell-mediated immunity associated with this transplant, and he lives normally at home; the two other ones, who have been more recently treated, have a significant improvement of their condition and they also live normally at home. This procedure, for the first time used in humans, has therefore demonstrated its feasibility and its efficacy: during early fetal development, foreign cells engraft readily and may result in cure or significant correction of a large variety of inherited diseases.
journal_name
Bone Marrow Transplantjournal_title
Bone marrow transplantationauthors
Touraine JL,Raudrant D,Rebaud A,Roncarolo MG,Laplace S,Gebuhrer L,Betuel H,Frappaz D,Freycon F,Zabot MTsubject
Has Abstract,Author List Incompletepub_date
1992-01-01 00:00:00pages
121-6eissn
0268-3369issn
1476-5365journal_volume
9 Suppl 1pub_type
杂志文章abstract::Ovarian failure after allogeneic stem cell transplant (allo-SCT) is an important risk factor for development of osteoporosis. We investigated the effects of various antiresorptive treatments in long-term surviving females with ovarian failure after allo-SCT. A total of 60 women with osteoporosis or osteopenia were div...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,随机对照试验
doi:10.1038/sj.bmt.1705196
更新日期:2006-01-01 00:00:00
abstract::The graft status of 14 patients, 13 of whom had received an allogeneic bone marrow transplant (BMT) for severe combined immunodeficiency disease (SCID) and one patient with SCID in whom intrauterine maternal engraftment was suspected, was examined using bone marrow or peripheral blood DNA and a combination of locus-sp...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1990-03-01 00:00:00
abstract::We evaluated the 100-day mortality rates associated with busulfan-based myeloablative conditioning regimens based on data from 1812 chronic myelogenous leukemia patients who underwent allogeneic blood or marrow transplantation (allotx). In all, 47 patients received intravenous (i.v.) busulfan and cyclophosphamide (i.v...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,多中心研究
doi:10.1038/sj.bmt.1704461
更新日期:2004-06-01 00:00:00
abstract::A nonmyeloablative conditioning regimen consisting of fludarabine (FLU) and 2 Gy TBI has been used extensively and with substantial engraftment success without promoting excessive nonrelapse mortality in medically infirm patients requiring hematopoietic cell transplantation. In this paper, we studied this same low-tox...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1038/bmt.2010.154
更新日期:2011-04-01 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
abstract::Propylene Glycol-Free melphalan HCL for Injection (PGF-Mel) is a new formulation that incorporates Captisol, a specially modified cyclodextrin, to improve melphalan stability. In this phase IIa, open-label, randomized, cross-over design bioequivalence study, the pharmacokinetics of PGF-Mel were compared with the marke...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,多中心研究,随机对照试验
doi:10.1038/bmt.2014.120
更新日期:2014-08-01 00:00:00
abstract::At our institution, the cytomegalovirus (CMV) prophylaxis protocol for allogeneic bone marrow transplant (BMT) recipients who are CMV-seropositive or receive marrow from a CMV-seropositive donor consists of a surveillance bronchoscopy approximately 35 days posttransplant. Patients with a positive surveillance bronchos...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/sj.bmt.1701525
更新日期:1999-01-01 00:00:00
abstract::The potent immunostimulatory cytokine interleukin-2 (IL-2) has been extensively investigated for its potential to induce anti-tumor immunity in a number of tumor models. Only recently the complex interplay of mutually suppressive or supportive cytokines of the IL-2-induced network of cytokines has been better characte...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/sj.bmt.1702431
更新日期:2000-07-01 00:00:00
abstract::To evaluate the efficacy and toxicity of two different etoposide (VP-16) dosages (30 or 45 mg/kg) in combination with busulfan/cyclophosphamide as conditioning therapy followed by stem cell transplantation in acute myeloid leukemia (AML), 90 patients with AML received either 30 mg/kg (n = 60) or 45 mg/kg (n = 30) etop...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702598
更新日期:2000-10-01 00:00:00
abstract::The Janus-activated kinase 1 (JAK1) and JAK2 inhibitor ruxolitinib is effective in decreasing symptomatic splenomegaly and myelofibrosis (MF)-related symptoms. However, allogeneic hematopoietic cell transplantation (HCT) remains the only curative option. We evaluated the impact of ruxolitinib on the outcome after HCT....
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2013.173
更新日期:2014-02-01 00:00:00
abstract::Chimerism studies after allogeneic BMT are performed to determine the donor and/or recipient origin of peripheral blood and marrow lymphoid and hematopoietic cells. These studies have been performed mostly in leukemias and aplastic anemia. We report DNA-based chimerism studies in three patients transplanted for advanc...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1995-12-01 00:00:00
abstract::We report long-term outcomes of 329 childhood severe aplastic anemia (SAA) patients who underwent hematopoietic SCT (HSCT) from an HLA-matched sibling donor in the Japanese Hematopoietic Cell Transplantation Registry. OS and EFS at 10 years were as high as 89.7+/-1.7% and 85.5+/-2.0%, respectively. Five cases of late ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2012.205
更新日期:2013-05-01 00:00:00
abstract::We report two cases of secondary myelodysplastic syndrome (SMDS) which followed successful treatment of a primary malignancy with high-dose chemotherapy supported by reinfusion of autologous stem cells. The SMDS was diagnosed 24 months and 40 months, respectively, following autografting. Both patients lived for 7 mont...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1994-02-01 00:00:00
abstract::Very long delay to engraftment (or non-engraftment) was observed in eight patients presenting common immunological features, i.e. excess of blood and bone marrow lymphocytes coexpressing CD8 and CD57 (HNK1) antigens. They were therefore treated by CD8 immunotherapy. Such treatment induced a complete clearance of CD8+,...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1990-01-01 00:00:00
abstract::We report the case of a 4-year-old female with high-risk ALL in first CR who received a BMT from an 11-month-old matched sibling treated with G-CSF in order to obtain an adequate number of mononuclear cells in a limited volume of bone marrow. The absence of toxicity, efficacy of the procedure and quality of the post-t...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1996-03-01 00:00:00
abstract::To control disease before allogeneic hematopoietic cell transplantation (HCT) for relapsed/refractory AML, we used clofarabine cytoreduction. Seventeen patients received clofarabine 30-40 mg/m(2) i.v. daily for 5 days with plans to initiate conditioning during the nadir, 14 days later. Bone marrow biopsy 12 days afte...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2010.32
更新日期:2010-12-01 00:00:00
abstract::Through two consecutive trials, a policy that considered allogeneic stem cell transplantation (SCT) from a sibling donor in second rather than first complete remission (CR) in selected younger patients with acute myeloid leukemia (AML) with t(8;21)/inv(16) (core binding factor (CBF) group) or a normal karyotype (NN gr...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/sj.bmt.1704884
更新日期:2005-04-01 00:00:00
abstract::Bone marrow transplantation is known to be associated with considerable morbidity and mortality. The aim of this study was to determine the influence of nutritional status and development of sick euthyroid syndrome as prognostic factors for outcome after BMT. In 100 patients who underwent transplantation the following...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701502
更新日期:1998-12-01 00:00:00
abstract::Transplant related mortality and relapse after bmt have a negative influence on the outcome of patients transplanted for acute leukaemia in first remission. Transplant related mortality includes graft-versus-host disease, infections and graft failure. To prevent gvhd and associated infections without increased graft r...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1993-01-01 00:00:00
abstract::Bloodstream infection (BSI) is a significant complication following allogeneic hematopoietic stem cell transplantation (allo-SCT). Corticosteroids mask inflammatory responses, delaying the initiation of antibiotics. We reviewed medical records of 69 allo-SCT patients who had been on >0.5 mg/kg prednisolone to investig...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704830
更新日期:2005-03-01 00:00:00
abstract::Allogeneic transplantation may be curative in a proportion of patients with multiple myeloma (MM), but relapse is a major cause of treatment failure. We sought to improve complete remission (CR) rates by the use of alpha-interferon (alpha-IFN) in patients not in CR when evaluated 4 months post-transplant. We report fi...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701403
更新日期:1998-10-01 00:00:00
abstract::We performed a retrospective analysis of the incidence, risk factors, and clinical outcome of hepatic veno-occlusive disease (VOD) in 50 children prepared for bone marrow transplantation with busulfan (16 mg/kg) and cyclophosphamide (200 mg/kg). The overall incidence of VOD was 28% (14/50). The incidence of VOD among ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1991-06-01 00:00:00
abstract::The immunostimulatory activity of viridans streptococcal strains isolated from neutropenic patients with severe sepsis (n=9) or uncomplicated bacteraemia (n=10) was compared. Peripheral blood mononuclear cells from healthy individuals were stimulated with heat-killed bacteria or culture supernatants, and cytokine prod...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704302
更新日期:2004-01-01 00:00:00
abstract::The effect of hematopoietic growth factors on neutrophil recovery after allogeneic transplantation is well-recognized. Recent laboratory studies demonstrated that these cytokines may also modify T-cell and dendritic cell function, but whether the effect is strong enough to alter the risk of GVHD is unclear. We perform...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,meta分析
doi:10.1038/sj.bmt.1704228
更新日期:2003-10-01 00:00:00
abstract::We describe the case of a HBsAg+, HBeAg+ carrier, treated with lamivudine, who experienced exacerbation of hepatitis after BMT from an anti-HBs+, anti-HBc+, anti-HBe+ donor. The serological profile of the donor and the timing of exacerbation suggested that the adoptive immunity transfer played a major pathogenetic rol...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704471
更新日期:2004-05-01 00:00:00
abstract::Abnormalities in liver function tests are common in hematopoietic SCT (HSCT) recipients. We retrospectively investigated the role of liver biopsy in determining the cause of elevated liver enzymes and its impact on the management of patients in the post-HSCT setting. A total of 24 consecutive liver biopsies were obtai...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2008.193
更新日期:2008-10-01 00:00:00
abstract::This was an open-label, single-center, phase II study of 20 patients with multiple myeloma who were either proven poor mobilizers (n=10; group A) or predicted poor mobilizers (n=10; group B) and were planned for autologous hematopoietic SCT. The aim was to assess the safety and efficacy of plerixafor for stem cell mob...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2009.130
更新日期:2010-01-01 00:00:00
abstract::We examined whether pegylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF) is capable of improving thrombocytopenia and promoting thrombopoietic reconstitution following lethal irradiation and bone marrow transplantation (BMT) in mice. Immediately after receiving 10 Gy whole body irradia...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1996-12-01 00:00:00
abstract::Patients undergoing bone marrow transplantation are profoundly immunosuppressed as a result of their intensive myeloablative chemotherapy and are at high risk for opportunistic fungal infections mainly caused by Candida spp and Aspergillus spp. Trichosporon beigelii (T beigelii) has emerged as a life-threatening oppor...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702231
更新日期:2000-04-01 00:00:00
abstract::Forty-five adult HLA-matched sibling BMT recipients were randomized to receive inactivated poliovirus vaccine (IPV) at 6, 8 and 14 months (early group, n = 23) or at 18, 20 and 26 months after BMT (late group, n = 22). Ninety-five percent of the early group patients had protective antibody titres of > or = 4 to poliov...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1038/sj.bmt.1700959
更新日期:1997-10-01 00:00:00