Abstract:
:Bone marrow transplantation was performed on an 8-month-old boy who was diagnosed as having fucosidosis following the diagnosis of the disease in his older brother. Although he was asymptomatic and his development was normal, abnomalities were found on an MRI scan prior to transplant. In the absence of a suitable related donor, an unrelated volunteer donor was used. Conditioning for the transplant consisted of busulphan and cyclophosphamide. Graft-versus-host disease prophylaxis consisted of in vitro T cell-depletion of the bone marrow and in vivo administration of cyclosporin. The post-transplant period was complicated by moderately severe graft-versus-host disease. Engraftment was documented by the presence of donor levels of alpha-fucosidase, donor blood group and tissue type (difference in the DQ antigen), and chromosomal polymorphism pattern of donor origin. Eighteen months after transplant, there is evidence of mild neurodevelopmental delay. By contrast, his elder sibling showed far greater developmental delay at the same age. The patient's MRI scan shows improvement. We believe this to be the first case of human fucosidosis treated by bone marrow transplantation.
journal_name
Bone Marrow Transplantjournal_title
Bone marrow transplantationauthors
Vellodi A,Cragg H,Winchester B,Young E,Young J,Downie CJ,Hoare RD,Stocks R,Banerjee GKsubject
Has Abstractpub_date
1995-01-01 00:00:00pages
153-8issue
1eissn
0268-3369issn
1476-5365journal_volume
15pub_type
杂志文章abstract::Six patients with high risk haematological malignancies received peripheral blood progenitor cells (PBPC) from unrelated donors. Four patients received PBPC as primary treatment and 2 following graft failure. Five donors were HLA-A, -B and -DR identical and one had a one antigen mismatch. PBPC were mobilised by treatm...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:
更新日期:1996-03-01 00:00:00
abstract::Multiple sclerosis (MS) is considered an inflammatory autoimmune disorder. Approved immunotherapies are only moderately effective in reducing disease exacerbations and brain inflammation in a subset of patients. Autologous hematopoietic stem cell transplantation (HSCT) has emerged in recent years as the first opportun...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:10.1038/sj.bmt.1704096
更新日期:2003-08-01 00:00:00
abstract::Changes in body composition related to metabolic syndrome are frequent among survivors of haematopoietic stem cell transplantation (HSCT), but insights into predisposing factors are incomplete, and it is unknown to what degree these changes persist at long term. We cross-sectionally investigated body composition by du...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-020-01038-3
更新日期:2020-09-02 00:00:00
abstract::Reduced-intensity conditioning (RIC) hematopoietic SCT (HSCT) is a potentially curative therapeutic option for patients with advanced follicular lymphoma (FL), but disease relapse remains the most common cause of failure. Radioimmunoconjugates administered before RIC allo-HSCT may enhance cytoreduction and allow more ...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/bmt.2010.339
更新日期:2011-12-01 00:00:00
abstract::We report the results of a retrospective analysis in 27 pediatric patients who received low-dose MTX as the second-line treatment for steroid-refractory or -dependent acute and chronic GVHD. Between July 2000 and May 2006, 10 patients with aGVHD and 17 with cGVHD were treated with MTX at a dose of 3-10 mg/m(2) weekly....
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705922
更新日期:2008-03-01 00:00:00
abstract::Two patients with high-risk acute myeloid leukemia (AML) whose bone marrow aspirates showed more than 25% blasts between 2 and 4 weeks after the first induction chemotherapy immediately received modified conditioning therapy with intravenous busulfan at 50% of the usual dose and fludarabine, before hematologic recover...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702819
更新日期:2001-03-01 00:00:00
abstract::The application of myeloablative Allo-SCT is limited by its associated morbidity and mortality. Reduced-intensity conditioning regimens attempt to diminish these, but are associated with a higher risk of disease relapse. Given the evidence of activity of clofarabine and cytarabine in myelodysplastic syndrome/acute mye...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/bmt.2008.423
更新日期:2009-07-01 00:00:00
abstract::Up to 10% of germ cell tumor patients require salvage high-dose chemotherapy with stem cell support, achieving cure rates in the range of 10-60%. Stem cell mobilization may be difficult in these patients because of multiple lines of treatment known to seriously hamper stem cell recovery. Plerixafor significantly enhan...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1038/bmt.2010.264
更新日期:2011-08-01 00:00:00
abstract::A recent shortage of melphalan has prompted the use of alternatives to BEAM (BCNU, Etoposide, Cytarabine, Melphalan) conditioning for autologous stem cell transplantion (ASCT). The BEAC (BCNU, Etoposide, Cytarabine, Cyclophosphamide) regimen has been employed as a conditioning regimen in lymphoma patients. However, th...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-018-0196-3
更新日期:2018-12-01 00:00:00
abstract::A monoclonal antibody of the IgM class, reacting with the CD9 (p24) antigen is described. The antibody (FMC27) is cytotoxic against cells of the common type of acute lymphoblastic leukaemia (c-ALL), giving killing at higher dilutions than an IgG antibody (FMC8) against the same antigen. FMC27 and FMC8 recognise differ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1987-02-01 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::Four patients with acute myeloid leukaemia relapsed within 6 months of allogeneic BMT. Three patients were treated with cytosine arabinoside and amsacrine while the fourth received no chemotherapy. All patients received infusions of leucocytes obtained by repeated leukapheresis from the original bone marrow donor. Thr...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1993-02-01 00:00:00
abstract::Threshold levels of CMV-specific T-cell populations presumably affording protection from active CMV infection in allo-SCT recipients have been proposed, but lack extensive validation. We quantified CMV pp65 and immediate-early 1-specific IFN-γ CD8(+) and CD4(+) T cell responses at days +30, +60 and +90 after transplan...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2010.330
更新日期:2011-11-01 00:00:00
abstract::Nine patients with haematological malignancy relapsed 3-32 months after receiving cyclophosphamide 120 mg/kg, 12-14 Gy fractionated total body irradiation and an HLA-identical sibling bone marrow transplant. They were reconditioned with melphalan 180-220 mg/m2 and retransplanted using the same donor and the same cyclo...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1986-12-01 00:00:00
abstract::BU and CY (BU/CY; 200 mg/kg) before HLA-matched sibling allo-SCT in children with sickle cell disease (SCD) is associated with ~85% EFS but is limited by the acute and late effects of BU/CY myeloablative conditioning. Alternatives include reduced toxicity but more immunosuppressive conditioning. We investigated in a p...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/bmt.2014.84
更新日期:2014-07-01 00:00:00
abstract::High-dose melphalan (HDM) has been adopted as standard therapy in the treatment of multiple myeloma. This treatment is associated with non-selective cytotoxicity, causing oral mucositis as the major non-hematological side-effect. Amifostine is a cytoprotector which prevents toxicity induced by anticancer therapy. We p...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/sj.bmt.1703757
更新日期:2002-12-01 00:00:00
abstract::A 42-year-old male underwent an HLA-matched sibling PBSC transplant for hypereosinophilic syndrome (HES) diagnosed in August 1995. Prior to transplant he experienced progressive cardiac and pulmonary dysfunction with red cell and platelet transfusion dependence despite therapy with hydroxyurea, steroids and interferon...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701783
更新日期:1999-05-01 00:00:00
abstract::The aim of the present trial was to investigate the feasibility of high-dose therapy followed by autologous peripheral blood stem cell transplantation (PBSCT) as a component of front-line treatment in patients with disseminated intermediate- and high-grade non-Hodgkin's lymphoma (NHL) aged 61-65 years. From October 19...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701272
更新日期:1998-06-01 00:00:00
abstract::This study assessed the incidence of long-term oral complications in 88 survivors of allogeneic haematopoietic cell transplantation (HCT). Patients examined were between 6 months and 6 years post-HCT and aged from 19 to 65 years. Subjects were investigated for both the subjective and objective features of long-term ad...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2011.63
更新日期:2012-02-01 00:00:00
abstract::Children with multisystem Langerhans cell histiocytosis (LCH) and risk organ involvement who fail to respond to conventional chemotherapy have an extremely poor prognosis. Myeloablative stem cell transplantation (SCT) as a possible salvage approach for these patients has been associated with a high risk of transplant-...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705015
更新日期:2005-08-01 00:00:00
abstract::We report on 24 patients with leukemia (19 pts), congenital disorders (4 pts) or severe aplastic anemia (1 pt) who received bone marrow transplantation from unrelated volunteer donors in 8 Italian Institutions. All the donor/recipient pairs were serologically HLA-A,B,DR matched; MLR was non reactive in 21 out of 24 ca...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1993-01-01 00:00:00
abstract::Two women suffering from severe aplastic anemia were treated by bone marrow transplantation (BMT). They became pregnant 17 and 40 months after the procedure. During the first 20 weeks of pregnancy, one of them continued to receive cyclosporin A (CSA) to prevent graft-versus-host disease. Her CSA serum levels during th...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1987-10-01 00:00:00
abstract::This prospective pilot study aimed to evaluate the predictive value of (18)F-FDG PET/CT for early diagnosis of acute gastrointestinal GVHD (GI-GVHD). In all, 42 consecutive patients who received allo-SCT were included. (18)F-FDG PET/CT was systematically performed at a median of 28 (range, 24-38) days after allo-SCT. ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2013.144
更新日期:2014-01-01 00:00:00
abstract::We conducted an open label, randomised clinical trial to compare amphotericin B colloidal dispersion (ABCD, Amphocil) 2 mg/kg/day intravenously with fluconazole 200 mg/day orally, for the prevention of fungal disease in neutropenic patients with haematological malignancies. In the event of unresolved fever after 4 day...
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.1702243
更新日期:2000-04-01 00:00:00
abstract::Cytomegalovirus (CMV) represents a major cause of morbidity after allogeneic stem cell transplantation (allo-SCT). Using interferon-gamma-enzyme-linked immunospot (ELISPOT) assay and HLA-peptide tetramers, we analysed 54 patients who received a reduced-intensity conditioning regimen, including fludarabine, busulphan a...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704442
更新日期:2004-04-01 00:00:00
abstract::Thirty patients with relapsed pediatric solid tumors received high-dose carboplatin and etoposide with autologous marrow support in a dose-escalation trial. These patients had received extensive prior treatment, which included both cisplatin and etoposide in 25 cases. Six patient cohorts received carboplatin in doses ...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:
更新日期:1992-11-01 00:00:00
abstract::HLA-haploidentical hematopoietic SCT (HSCT) provides an opportunity for almost all patients who lack HLA-matched sibling donors. The donor availability can be increased by including the collateral related donors (CRDs). We compared clinical outcomes of patients with hematological malignancies, who underwent haploident...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2013.223
更新日期:2014-04-01 00:00:00
abstract::We report a 5-year-old boy with juvenile myelomonocytic leukemia (JMML) which relapsed after an allogeneic bone marrow transplant who was successfully treated with interferon-alpha (IFN-alpha). One year after starting the therapy, he remains clinically well and in complete remission while continuing treatment with IFN...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702584
更新日期:2000-09-01 00:00:00
abstract::To investigate the possibility that a hypercoagulable state develops during autologous bone marrow transplantation (BMT), we measured levels of circulating natural anticoagulants and fibrinolytic proteins before and weekly during the hospital course of 18 patients undergoing autologous BMT for Hodgkin's and non-Hodgki...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1991-12-01 00:00:00
abstract::Post-transplant lymphoproliferative disorders (PTLD) are morphologically/clinically heterogeneous. The main goal of this study was to define the histogenesis of PTLD (B-cell phenotype, EBV-related) in seven pediatric patients after allogeneic T-cell-depleted bone marrow transplantation. Immunohistochemical stains usin...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704395
更新日期:2004-02-01 00:00:00