Abstract:
:Serial liver biopsies were obtained in 20 patients undergoing bone marrow transplantation (BMT) for mucopolysaccharidosis (MPS). The 13 patients with MPS I, one with MPS II, four with MPS III, and two with MPS VI underwent liver biopsy prior to and from 1 to 37 months after BMT. The amount of accumulated glycosaminoglycan (GAG) was assessed by semiquantitation of Kupffer cell staining with colloidal iron and by counting the number of hepatocellular GAG-containing lysosomes in electron micrographs. Eleven of 13 patients with MPS I achieved engraftment, and 10 of the 11 cleared the Kupffer cells and hepatocytes of GAG by 3 to 19 months post-BMT. Two patients with autologous recovery demonstrated persistent hepatocyte inclusions. The three patients with MPS II and MPS VI engrafted and showed clearance of hepatocyte and Kupffer cell GAG by 7 months after BMT. All four patients with MPS III engrafted. Although the Kupffer cells in these patients were cleared of GAG by 12 months after BMT, hepatocellular inclusions persisted in all four. For MPS I, II and VI, donor engraftment was associated with resolution of lysosomal storage material in donor-derived Kupffer cells and untransplanted hepatocytes, indicative of transcellular metabolic correction. Failure of hepatocyte clearance in one case of MPS I and all patients with MPS III suggested a diminished capacity of the graft-derived enzyme to enter the hepatocyte lysosomes in these patients.
journal_name
Bone Marrow Transplantjournal_title
Bone marrow transplantationauthors
Resnick JM,Krivit W,Snover DC,Kersey JH,Ramsay NK,Blazar BR,Whitley CBsubject
Has Abstractpub_date
1992-09-01 00:00:00pages
273-80issue
3eissn
0268-3369issn
1476-5365journal_volume
10pub_type
杂志文章abstract::A 36-year-old man underwent an allogeneic BMT for acute lymphoblastic leukemia. Eighteen days later the patient developed sudden onset of painful, gross hematuria due to adenovirus type 11 infection that did not respond to conservative therapy. There was an increase in serum creatinine levels and delayed recovery of t...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1994-11-01 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::Twenty-five patients with hematologic malignancies (n = 21) or aplastic anemia (n = 4) undergoing an allogeneic BMT from an HLA-identical sibling developed cytomegalovirus (CMV) antigenemia at a mean interval from BMT of 41 days (range 16-141 days). All patients were treated at the time of antigenemia in the absence o...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:
更新日期:1994-06-01 00:00:00
abstract::The efficacy of posttransplant cyclophosphamide (PTCy) and antithymocyte globulin (ATG) in controlling GVHD has been previously reported. We aim to study the safety and efficacy of the use of dual T-cell depletion with ATG and PTCy for peripheral blood reduced intensity conditioning regimen allo-HSCT in 270 patients w...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-020-0813-9
更新日期:2020-09-01 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::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::Immunological effects of IL-21 on T, B and natural killer (NK) cells have been reported, but the role of IL-21 in GVHD remains obscure. Here, we demonstrate that morbidity and mortality of GVHD was significantly reduced after BMT with splenocytes from IL-21R(-/-) mice compared with those from wild type mice. To furthe...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2009.223
更新日期:2010-04-01 00:00:00
abstract::The International Bone Marrow Transplant Registry (IBMTR), established in 1972, collects data on recipients of allogeneic bone marrow transplants from over 320 institutions, worldwide. Its database includes information for about 40% of all allogeneic transplants performed between 1964 and 1995. ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1996-05-01 00:00:00
abstract::Basic fibroblast growth factor (bFGF) inhibits radiation-induced apoptosis, and radioprotects haematopoietic, cartilage growth plate, pulmonary and gastrointestinal tissues. Conversely, chronic overexpression of bFGF may promote fibrosis. We measured the endogenous circulating bFGF in blood of patients undergoing cond...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701778
更新日期:1999-06-01 00:00:00
abstract::The First International Symposium on Photopheresis in Hematopoietic Stem Cell Transplantation was held in Vienna, Austria with an educational grant from Therakos Inc. from 25 May to 27 May 2005. Three general issues were addressed: (1) pathophysiology of graft-versus-host disease (GvHD), (2) induction of immune tolera...
journal_title:Bone marrow transplantation
pub_type:
doi:10.1038/sj.bmt.1705440
更新日期:2006-08-01 00:00:00
abstract::IL-7 is produced by stromal cells and is the major lympho- and thymopoietic cytokine. IL-7 induces proliferation and differentiation of immature thymocytes, and protects thymocytes from apoptosis by induction of bcl-2 expression. The regulation of IL-7 production is poorly characterized, although down-regulation by tr...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701655
更新日期:1999-04-01 00:00:00
abstract::Myasthenia gravis (MG) is a rare complication of allogeneic bone marrow transplantation (BMT). We present the 11th case in the medical literature, a 23-year-old female 100 months post-allogeneic bone marrow transplantation for acute myelogenous leukemia (AML). After discontinuation of immunosuppression for chronic gra...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1700759
更新日期:1997-05-01 00:00:00
abstract::We investigated the impact of the most commonly used preparative regimens on the outcome of 395 patients with diffuse large cell lymphoma (DLCL), consecutively reported to the registry of the Spanish GEL/TAMO. Among them, 139 (35%) were autografted in 1st CR, 86 (22%) in 2nd/3rd CR, 124 (31%) had chemosensitive diseas...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,多中心研究
doi:10.1038/sj.bmt.1702795
更新日期:2001-02-01 00:00:00
abstract::In an attempt to improve the dismal prognosis of adults with recurrent medulloblastoma, six patients were treated with aggressive salvage therapy including high-dose chemotherapy (HDCT) and autologous stem cell transplantation (ASCT). At relapse, all patients underwent surgical debulking followed by HDCT/ASCT and then...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1703725
更新日期:2002-11-01 00:00:00
abstract::The effect of prophylactic intravenous administration of a cytomegalovirus (CMV) hyperimmune globulin with a high titer of neutralizing antibodies plus oral acyclovir was studied in 93 consecutive bone marrow transplant recipients. In spite of receiving blood products unscreened for CMV only six patients developed CMV...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1988-11-01 00:00:00
abstract::Invasive fungal infections (IFIs) are major complications after allogeneic hematopoietic SCT (HSCT). PCR-based assays able to detect fungal DNA have been reported to precede clinical diagnosis of IFI. We performed a prospective study to evaluate a PCR-based pre-emptive approach. Ninety-nine patients undergoing reduced...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,随机对照试验
doi:10.1038/bmt.2010.38
更新日期:2010-12-01 00:00:00
abstract::Bone marrow transplantation is the first line of treatment for all patients with aplastic anaemia who have an identical twin. Age is not an important consideration in these circumstances. For patients with an HLA identical sibling donor bone marrow transplantation should be offered as a matter of urgency to those pati...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章,评审
doi:
更新日期:1989-12-01 00:00:00
abstract::The purpose of the study was to evaluate the effect of lisofylline (LSF) on engraftment, regimen-related toxicities (RRT), and mortality in patients undergoing allogeneic bone marrow transplantation (BMT). We performed a multicenter, randomized placebo-controlled trial in 60 patients with hematologic malignancies rece...
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.1702114
更新日期:2000-02-01 00:00:00
abstract::The influence of the CFU-GM content of donor marrow on the outcome of allogeneic marrow transplantation (BMT) has been debated. We now report 38 patients (25 acute leukemias, 10 chronic myeloid leukemias, two myeloma; 24 in first CR/CP and 14 in more advanced phases of their disease) undergoing unmanipulated HLA-ident...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1995-02-01 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::A case of pneumatosis intestinalis with perforation is reported in a patient after bone marrow allograft for chronic myeloid leukemia. Risk factors included the transplant, prolonged immunosuppression and neutropenia, graft-versus-host disease, extended use of corticosteroids, infection and lower gastrointestinal endo...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1994-08-01 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::Seventeen patients with Philadelphia (Ph) chromosome-positive chronic myeloid leukemia (CML) were treated with the ICE regimen plus G-CSF with the aim of mobilizing and collecting Ph-negative peripheral stem cells (PSC) in the setting of an autotransplant program. Fifteen patients had CML in first chronic phase (CP), ...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:
更新日期:1996-11-01 00:00:00
abstract::The protection of hematopoietic stem and progenitor cells and their environment is required for recovery from radiation-induced (RI) myelosuppression. To achieve this goal, we propose a new gene therapy strategy based on local and short-term synthesis and expression of Sonic hedgehog morphogene (Shh) at the niche leve...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2013.162
更新日期:2014-02-01 00:00:00
abstract::Recommendations on indications for allogeneic haematopoietic SCT have been presented, but transplantation techniques remain poorly standardized. Pre-transplant risk factors are well defined, and reported outcomes vary markedly among patients with similar risk characteristics. It would be of importance to know the impa...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2012.45
更新日期:2012-11-01 00:00:00
abstract::Daily administration of granulocyte colony-stimulating Factor (G-CSF) results in progenitor cell mobilization with maximum blood levels achieved after 4-7 days. In this study the short-term effects of glycosylated G-CSF at a dose of 5 microg/kg s.c. were determined so as to allow optimization of the timing of progenit...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701099
更新日期:1998-02-01 00:00:00
abstract::Twenty-two patients with locally advanced or metastatic soft tissue sarcomas received high dose chemotherapy with autologous bone marrow graft. Eleven patients receiving melphalan also received fractionated total body irradiation. Six patients (four in CR and two in PR) were intensified after first line therapy. Thirt...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1992-11-01 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::During the reproductive period, mothers and offspring exchange hematopoietic cells and develop a form of immunological tolerance bidirectionally. To examine whether previous experience of such communication has any remote effect when maternal hematopoietic cells are later transplanted to the children, we retrospective...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1703146
更新日期:2001-08-01 00:00:00
abstract::Seventy-nine patients with AML in CR1 received allo-SCT between May 2006 and May 2011, and the prognostic impact of FMS-like tyrosine kinase 3/internal tandem duplication (FLT3/ITD) mutation was evaluated in the context of other clinical prognostic factors. Patients with FLT3/ITD + AML had significantly inferior DFS (...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2012.88
更新日期:2012-12-01 00:00:00