Abstract:
:Infantile osteopetrosis is a lethal disorder resulting from a severe defect in the ability of osteoclasts to resorb bone. The only therapy shown to be capable of providing lasting benefit is allogeneic hematopoietic stem cell transplantation (HCT). We report the outcome of 10 patients with infantile malignant osteopetrosis treated with HCT from an HLA A, B, DRB1 matched (n=6) or A or B locus mismatched (n=4) family member or unrelated donor at the University of Minnesota between 1978 and 1997. Eight of 10 patients achieved primary engraftment; secondary graft failure was seen in two patients. Five of 10 patients survive; three with full or partial donor chimerism and two with autologous hematological recovery. Transient or partial donor chimerism can be sufficient to correct the hematological manifestations of osteopetrosis. We recommend early referral for consideration of HCT with a related or unrelated donor as neurosensory manifestations of osteopetrosis are generally not reversible. Donor engraftment may be easier to achieve early in the course of the disease.
journal_name
Bone Marrow Transplantjournal_title
Bone marrow transplantationauthors
Eapen M,Davies SM,Ramsay NK,Orchard PJdoi
10.1038/sj.bmt.1701474subject
Has Abstractpub_date
1998-11-01 00:00:00pages
941-6issue
10eissn
0268-3369issn
1476-5365journal_volume
22pub_type
杂志文章abstract::Haploidentical hematopoietic stem cell transplantation using T-cell-depleted grafts is a valid option for pediatric patients with hematological malignancies in need of an allogeneic transplantation and lacking an HLA-identical donor. Seventy-five transplantations were performed in 70 patients. Thirty-eight patients ha...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2016.101
更新日期:2016-09-01 00:00:00
abstract::Autologous BMT (auto-BMT) has been conducted for 17 high-risk common ALL antigen (CALLA)-positive non-T cell type ALL patients. Ex vivo purging of leukemia cells from infused BM cells was performed using complement and three kinds of mouse monoclonal antibodies reactive to CALLA-positive leukemia cells: NL-1 (IgG2a), ...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:
更新日期:1993-04-01 00:00:00
abstract::Unusual cytomegalovirus (CMV)-related complications were seen after autologous stem cell transplantation (SCT) in a 50-year-old patient with diffuse large B cell lymphoma. One month after SCT, the patient developed life-threatening upper gastrointestinal tract (GIT) bleeding with several episodes of hemorrhagic shock....
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1703519
更新日期:2002-04-01 00:00:00
abstract::We investigated the use of 'prophylactic' donor lymphocyte infusions (DLI) containing 1 x 107 CD3+ cells, given at 30, 60 and 90 days post-allogeneic blood and marrow transplantation (BMT), following conditioning with fludarabine 30 mg/m(2)/4 days and melphalan 70 mg/m(2)/2 days. GVHD prophylaxis consisted of cyclospo...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702726
更新日期:2001-01-01 00:00:00
abstract::A 41-year-old male with IgG kappa multiple myeloma is described. He developed a free gamma heavy chain without an accompanying light chain following high-dose chemotherapy and autologous peripheral blood stem cell transplantation. The free gamma heavy chain was detected in serum and urine specimens 2 months after tran...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702850
更新日期:2001-03-01 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::Complex factors determine how physicians select between alternative therapies. We conducted a survey of 352 leukemia experts to determine consensus regarding optimal treatment for leukemia and to identify factors correlated with therapy decisions. The study evaluated both general treatment policies and recommendations...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1992-01-01 00:00:00
abstract::Patients who develop veno-occlusive disease (VOD) of the liver may have low plasma levels of the natural anticoagulants protein C and antithrombin III, but large vessel thromboses are not commonly reported in these patients. We reviewed the records of 1847 consecutive patients for evidence of portal vein thrombosis. E...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1703368
更新日期:2002-02-01 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 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::We retrospectively studied a series of 23 patients (median age 50 years, range 29-59 years) with multiple myeloma (MM), treated in first relapse by a sequential autologous-allogeneic tandem approach. Tandem transplantation (TT) consisted in high dose melphalan (HDT) and auto-SCT followed by an (allo-SCT) preceded by t...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2010.90
更新日期:2011-02-01 00:00:00
abstract::Despite immunosuppressive therapy using cyclosporine (CsA) and prednisolone and methotrexate (MTX), the incidence for aGVHD grade II to IV after transplantation from HLA matched unrelated donors (MUD) is 78%, the incidence for grade III and IV 36%. Since GVHD contributes to morbidity and mortality after MUD-BMT, a mor...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702346
更新日期:2000-05-01 00:00:00
abstract::Liver dysfunction is a common problem in BMT recipients and it is important to determine the etiology in order to institute appropriate therapy. The purpose of this study was to evaluate the possible causes of liver dysfunction during the first post-transplant year in BMT recipients and to identify a possible relation...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702453
更新日期:2000-07-01 00:00:00
abstract::Treatment with allogeneic hematopoietic stem cell transplantation (HSCT) is associated with short and long-term toxicities that can result in alterations in sexual functioning. The aims of this prospective evaluation were to determine: (1) associations between HSCT and increased sexual dysfunction 1 year after treatme...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2015.342
更新日期:2016-06-01 00:00:00
abstract::Omenn syndrome is a variant of SCID, inherited as an autosomal recessive disorder, and characterized by severe eczematoid dermatitis, eosinophilia, elevated serum IgE and a distinctive histology in enlarged lymph nodes. The etiology of Omenn syndrome is unknown, however, unlike other forms of SCID; patients with Omenn...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1995-09-01 00:00:00
abstract::In order to detect chimerism after allogeneic hematopoietic SCT (HSCT), several methods have been developed. In this study we describe the use of a set of insertion/deletion (Indel) polymorphic loci to determine the level of donor cell engraftment. We analyzed 50 DNA samples from patients who had undergone HSCT, and a...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2014.173
更新日期:2014-11-01 00:00:00
abstract::The aim of this study was to define factor(s) influencing fetal erythropoiesis following bone marrow transplantation. Thirty-one transplanted patients (14 males, 17 females) were studied. The underlying diseases were chronic myelogenous leukaemia (CML, 18 patients), acute myeloblastic leukaemia (AML, 7 patients) and a...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1994-11-01 00:00:00
abstract::The efficacy and safety of oral valganciclovir was compared to ganciclovir i.v. in pre-emptive treatment of cytomegalovirus (CMV) in T-cell-depleted allogeneic stem cell transplant (allo-SCT) recipients. A therapeutic guideline was developed to allow the safe application of valganciclovir in allo-SCT recipients requir...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705311
更新日期:2006-04-01 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::The purpose of this study was to evaluate pre-transplant T-cell status in autologous hematopoietic progenitor-cell transplantation (HPCT) recipients. Between 1999 and 2002 we prospectively enrolled 85 autologous HPCT recipients with solid tumors (N = 50) or hematological malignancies (n = 35). Patient diagnoses includ...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/sj.bmt.1705073
更新日期:2005-09-01 00:00:00
abstract::The effects of in vitro radiation and exposure to 4-hydroperoxycyclophosphamide (4-HC) on the production of G- and GM-CSF by different components of the human hematopoietic microenvironment are described. The marrow microenvironment is composed of fibroblasts, endothelial cells, macrophages, and adipocytes. To study t...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1992-12-01 00:00:00
abstract::We evaluated the efficacy and safety of the conditioning regimen that consisted of TBI and melphalan (L-PAM), followed by hematopoietic SCT (HSCT) in 23 children with advanced hematological malignancies. The median age at HSCT was 9 (range, 2-15) years. The underlying diseases were ALL in 16 patients (5 in CR2, 3 in C...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2010.253
更新日期:2011-08-01 00:00:00
abstract::This retrospective analysis was done to determine the response rate and survival of women with metastatic breast cancer with bone marrow involvement treated with high-dose cyclophosphamide and thiotepa and peripheral progenitor cell rescue. Eligibility criteria included histologically-documented metastatic breast canc...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1994-04-01 00:00:00
abstract::Severe veno-occlusive (VOD) disease is a major cause of morbidity and mortality in allogeneic bone marrow transplant recipients, and new approaches in managing patients who develop serious VOD is needed. In this report, we describe a patient who underwent transjugular intrahepatic portosystemic stent-shunt (TIPS) for ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1996-08-01 00:00:00
abstract::Overexpression of microRNA-99a (miR-99a) have been associated with adverse prognosis in acute myeloid leukemia (AML). Nevertheless, whether it also predicts poor outcome in post-allogeneic hematopoietic stem cell transplantation (allo-HSCT) AML patients remains unclear. To further elucidate the prognostic value of miR...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-018-0146-0
更新日期:2018-09-01 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::A questionnaire comprised of a series of clinical vignettes illustrating potential difficulties in diagnosis, grading and treatment of chronic graft-versus-host disease (GVHD) was completed by 65 bone marrow transplant physicians from 51 transplant centers worldwide. The concordance index (CI) for responses to each qu...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1989-05-01 00:00:00
abstract::Hematopoietic SCT (HSCT) and high-dose chemotherapy are being explored as therapy for various human refractory immune-mediated conditions, including inflammatory bowel diseases (IBD). Nevertheless, the exact immunological mechanisms by which the BM cells (BMCs) or immunosuppression provide remission from these disease...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2010.6
更新日期:2010-10-01 00:00:00
abstract::The incidence and severity of GVHD following bone marrow transplantation increases with recipient age. The role of recipient age on the development of GVHD was analyzed in a semi-allogeneic (C57BL/6-->(C57BL/6 x DBA/2)F1) murine GVHD system. Young adult (2 months) and old (12-14 months) recipient mice were lethally ir...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1700719
更新日期:1997-04-01 00:00:00
abstract::We report the experience of 14 centers which have used the combination of cytosine arabinoside (ara-C; 24-36 g/m2) and total body irradiation (TBI) to prepare 213 patients with acute lymphoblastic leukemia (ALL) for allogeneic BMT. The overall 3-year disease-free survival (DFS) probability is 38% (95% CI: 31-45%); 75 ...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章,多中心研究
doi:
更新日期:1993-01-01 00:00:00