Abstract:
:Fanconi anaemia is a hereditary disorder characterised by chromosomal breaks increased by cross-linking agents. Bone marrow transplantation is the treatment of choice when a HLA identical sibling donor has been identified. The use of low-dose cyclophosphamide with thoraco-abdominal irradiation for the conditioning regimen of FA patients has lead to a dramatic improvement of survival, with a long-term survival of 75% at our institution. However, if most patients are completely cured of their haematological disease, there is concern about an increased frequency of secondary tumours, mostly head and neck squamous cell carcinomas of poor prognosis. Results of BMT using alternative donors (HLA mismatched related and unrelated donors) have also improved during the last decade. A better selection of the donor via high-resolution techniques for class-II HLA matching, and more recently the use of T cell depleted grafts are probably the main explanations. Despite a short follow-up and the small number of patients analysed, transplants using HLA matched family cord blood give some promising results. On the other hand, first results with unrelated cord blood remind that this approach is clearly an experimental one that has to be evaluated through international registries and prospective studies. New approaches including autologous stem cell transplantations and gene therapy are currently explored.
journal_name
Bone Marrow Transplantjournal_title
Bone marrow transplantationauthors
Guardiola P,Socié G,Pasquini R,Dokal I,Ortega JJ,van Weel-Sipman M,Marsh J,Locatelli F,Souillet G,Cahn JY,Ljungman P,Miniero R,Shaw J,Vermylen C,Archimbaud E,Bekassy AN,Krivan G,Di Bartolomeo P,Bacigalupo A,Gluckmansubject
Has Abstractpub_date
1998-04-01 00:00:00pages
S24-7eissn
0268-3369issn
1476-5365journal_volume
21 Suppl 2pub_type
杂志文章,评审abstract::Adjuvant transfusion of donor lymphocytes (aDLT) may reduce the risk of relapse after allogeneic stem cell transplantation in high-risk AML. We performed a retrospective analysis on the safety and efficacy of aDLT in a cohort of 46 patients. To be eligible for aDLT, patients had to be in CR for at least 120 days from ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2015.234
更新日期:2016-05-01 00:00:00
abstract::Genetic mutations of proteins regulating nuclear factor of kappa-light polypeptide gene enhancer in B lymphocyte (NF-kappaB) activation result in heritable diseases of development and immunity. Hypomorphic, X-linked mutations in the IKBKG gene (NF-kappaB essential modulator (NEMO) protein), and hypermorphic, autosomal...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,多中心研究
doi:10.1038/bmt.2008.308
更新日期:2009-02-01 00:00:00
abstract::We describe the rare occurrence of a granulomatous pneumonitis seen in a patient following allogeneic bone marrow transplantation. Interestingly sarcoidosis was diagnosed in the marrow donor less than a year after donating his bone marrow. ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1703192
更新日期:2001-09-01 00:00:00
abstract::The use of hematopoietic stem cells for in utero transplantation to create permanent hematochimerism represents a new concept in fetal therapy, although this approach has provided heterogeneous results. In this paper we have undertaken molecular, phenotypic and functional studies aimed at identifying the presence of f...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702108
更新日期:2000-01-01 00:00:00
abstract::Hematopoietic stem cells are extremely sensitive to damage by ionizing radiation. At high doses of radiation, toxicity to the bone marrow may be operationally irreversible. Insufficient functional hematopoietic stem cells remain or irradiated individuals cannot survive sufficiently long enough for functional stem cell...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1987-06-01 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::We have tested the immunologic status and hematologic parameters of mice 2 months (short-term survivors) or 18 months (long-term survivors) after lethal total body irradiation and syngeneic bone marrow transplantation (SBMT), and of normal mice of corresponding age. Long-term SBMT survivors showed significantly lowere...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1990-11-01 00:00:00
abstract::In an effort to reduce the relapse rate after unpurged autologous bone marrow transplantation (ABMT) in patients with acute myeloid leukemia (AML) in first complete remission (CR1), the standard conditioning regimens (cyclophosphamide/busulphan and cyclophosphamide/TBI) were intensified by adding idarubicin. Seventeen...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/sj.bmt.1701279
更新日期:1998-07-01 00:00:00
abstract::Many factors, including lower risk of GVHD, rapid availability of 4/6-6/6 matched cord blood (CB) units and incremental gains in the outcomes, have led to an increasing use of CB transplantation (CBT) to treat many patients who lack fully matched adult BM donors. A large electronically searchable worldwide inventory o...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:10.1038/bmt.2009.290
更新日期:2009-11-01 00:00:00
abstract::Hematopoietic cell transplantation (HCT) following nonmyeloablative conditioning (NMSCT) may be associated with a reduced risk of infection compared to standard allogeneic HCT. We retrospectively analyzed incidence and risk factors of infection in 62 patients undergoing NMSCT with low-dose TBI +/- fludarabine and post...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705255
更新日期:2006-02-01 00:00:00
abstract::The Japan Marrow Donor Program (JMDP), established in 1991, has continued to grow in its capacity to facilitate unrelated bone marrow (BMT) and peripheral blood stem cell transplantation (PBSCT) for the past 25 years in Japan. The current donor pool is 463,465 (as of 31 December 2016) and 20,237 transplants were perfo...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-017-0084-2
更新日期:2018-05-01 00:00:00
abstract::We have used a standardized 21-day expansion protocol to produce cytokine-induced killer (CIK) cells starting from very small amounts of nucleated cells (approximately 15 x 10(6) cells) isolated from cord blood. Mononuclear cells are stimulated with anti CD3 (OKT3) and IFNgamma and then expanded with IL-2. Moreover, w...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705503
更新日期:2006-11-01 00:00:00
abstract::Thrombotic microangiopathy (TMA) is a significant complication after hematopoietic stem-cell transplantation (HSCT); however, there is little information on it following reduced-intensity cord blood transplantation (RI-CBT). We reviewed the medical records of 123 adult patients who received RI-CBT at Toranomon Hospita...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705099
更新日期:2005-09-01 00:00:00
abstract::Optimal overnight (ON) storage of PBPC aphereses is becoming an increasingly important issue and different options for storing PBPC products exist. The survival of primitive progenitor cells is of major interest, as recent data suggest that these progenitors are not only important for long-term engraftment but also co...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702104
更新日期:2000-01-01 00:00:00
abstract::The growing number of BMTs has increased interest in safe and standardized in vitro bone marrow processing techniques. We describe our experience with a rapid automated method for the isolation of mononuclear cells (MNC) from large volumes of bone marrow using a Fenwal CS-3000 cell separator without employing density ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1992-12-01 00:00:00
abstract::We performed a survey of the European Cooperative Group for Blood and Marrow Transplantation to analyze the outcome of 625 acute promyelocytic leukemia (APL) patients transplanted with auto- or allogeneic-hematopoietic stem cell transplantation (autoHSCT, alloHSCT) after 1993, in first (CR1) or in second complete remi...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,多中心研究
doi:10.1038/sj.bmt.1705620
更新日期:2007-04-01 00:00:00
abstract::Limiting dilution analysis was used to quantify the frequency of cytotoxic T cell precursors (CTLp) against minor histocompatibility (H) antigens induced by HLA-identical BMT. The development of CTLp was monitored serially in ten patients developing either acute (n = 3), acute and chronic (n = 4) or no (n = 3) GVHD. I...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1993-05-01 00:00:00
abstract::We describe two patients who developed respiratory syncytial virus (RSV) pneumonia after BMT. One died of RSV pneumonia after three courses of steroid pulse therapy. Surprisingly, RSV antigen was identified in the bronchoalveolar lavage fluid (BALF) obtained post mortem. Steroid pulse therapy might have suppressed ant...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702559
更新日期:2000-09-01 00:00:00
abstract::NK cells can exert potent anti-leukemia activity after either autologous or allogeneic BMT. However, in autologous blood or marrow transplant patients, NK cell number and/or function could be reduced, and also may vary according to the sampling site. In order to evaluate the hypothesis that blood or marrow grafts from...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1996-03-01 00:00:00
abstract::Since approximately 30% of leukemia patients relapse after allogeneic BMT using total body irradiation (TBI)-based preparative regimens, treatment intensity may be suboptimal. The killing of leukemia cells is proportional to the radiation absorbed dose. We studied the feasibility and toxicity of escalating the doses o...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,多中心研究
doi:10.1038/sj.bmt.1702230
更新日期:2000-04-01 00:00:00
abstract::Prognostic factors for survival following allogeneic BMT for AML include age, disease status and cytogenetic risk classification. Lactate dehydrogenase (LDH) levels have not been studied as a potential risk factor. We reviewed our experience with BMT for AML and included LDH at the time of admission in an analysis of ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705811
更新日期:2007-10-01 00:00:00
abstract::The aim of this study was to develop a comprehensive system for predicting non-relapse mortality after allogeneic hematopoietic cell transplantation (HCT) during first complete remission (CR) of acute myeloid leukemia (AML). After dividing 2344 eligible patients randomly into a training set and a validation set, we fi...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-020-01032-9
更新日期:2020-08-14 00:00:00
abstract::Reverse transcriptase polymerase chain reaction (RT-PCR) was applied to evaluate the frequency of tumour cells in PBPC products from 15 high risk Ewing tumour (ET) patients who were treated according to EICESS 92 with high-dose chemotherapy (HDC) and stem cell rescue. Initial tumour cell contamination of the bone marr...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701924
更新日期:1999-08-01 00:00:00
abstract::Considerable evidence in preclinical models as well as in human transplantation now suggests that donor-derived natural killer (NK) cells can contribute to alloimmune recognition of recipient residual tumour cells. This makes the NK cell population an attractive target for in vitro or in vivo manipulations, in order t...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704711
更新日期:2005-01-01 00:00:00
abstract::We describe seven patients who developed symptoms including severe headache, circumoral paresthesia, and facial flushing during high-dose carmustine (BCNU) infusion as part of the preparative regimen for autologous peripheral blood stem cell (PBSC) transplantation for metastatic breast cancer. Five patients responded ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1700743
更新日期:1997-04-01 00:00:00
abstract::Autologous hematopoietic stem cell transplantation (HSCT) is currently being evaluated as a therapy for patients with progressive multiple sclerosis (MS) at risk of debilitating neurological impairment. While preliminary results from a few studies have been reported, little is known about toxicities or outcome of HSCT...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1703324
更新日期:2002-01-01 00:00:00
abstract::Hurler syndrome (MPS1H) is a progressive inborn error of mucopolysaccharide metabolism leading to premature death. Allogeneic hematopoietic cell transplantation (HCT) can achieve stabilization and improve long-term survival. However, large studies have shown that preparative regimen-related toxicity (RRT) and graft fa...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/sj.bmt.1704786
更新日期:2005-02-01 00:00:00
abstract::Our growing physiological understanding of hematopoietic progenitor cells has led to the clinical use of circulating progenitor cells, including stem cells, for either reconstitution of hematopoietic function, up to the transduction of functional genes into a self-renewing cell system. In the following, an attempt has...
journal_title:Bone marrow transplantation
pub_type: 历史文章,杂志文章
doi:
更新日期:1996-05-01 00:00:00
abstract::Salivary gland dysfunction is a common sequela of hematopoietic progenitor cell transplantation (HPCT). The investigation of major salivary gland dysfunction with sodium pertechnetate scintigraphy is a non-invasive method that provides images of the parotid and submandibular glands. In this prospective trial, 20 HPCT ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705351
更新日期:2006-05-01 00:00:00
abstract::In a 34-year-old patient toxoplasma retinitis/encephalitis developed 9 months after bone marrow transplantation. The BMT was complicated with a serious GVHD. Although she initially responded well to antibiotic therapy she died 2 years after BMT due to progressive infection. ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701128
更新日期:1998-03-01 00:00:00