Abstract:
:MHC Class II deficiency is a rare primary immunodeficiency disease characterized by absent HLA Class II expression resulting in CD4 lymphopenia, lack of Ag-specific responses and recurrent infection. Without successful allogeneic SCT, most children succumb to infection within the first decade of life. To date, alternative donor transplants for this disorder have been inferior to SCT for other forms of combined immunodeficiency disease due to an increased incidence of graft rejection, GVHD and death from infections generally acquired before haematopoietic cell transplantation. This study details the transplant outcome of 16 affected children consecutively transplanted at four centers since 1990, 8 of whom required mechanical ventilation pretransplant. Stem cells were derived from an HLA-mismatched family member (n=10), an HLA-matched unrelated adult donor (n=4), or an unrelated cord blood donor (n=2). Graft failure occurred in five children, all of whom underwent a second SCT. Six patients developed acute GVHD although no patient developed chronic GVHD after primary transplantation. CD4 T-cell reconstitution remained below the normal range for age, suggesting defective thymopoiesis after allo-SCT. Nonetheless, 69% of children survive without GVHD at a median follow-up of 5.7 years, indicating improved outcomes compared with previous studies.
journal_name
Bone Marrow Transplantjournal_title
Bone marrow transplantationauthors
Small TN,Qasim W,Friedrich W,Chiesa R,Bleesing JJ,Scurlock A,Veys P,Sparber-Sauer Mdoi
10.1038/bmt.2012.140subject
Has Abstractpub_date
2013-02-01 00:00:00pages
226-32issue
2eissn
0268-3369issn
1476-5365pii
bmt2012140journal_volume
48pub_type
杂志文章abstract::Allogeneic bone marrow transplantation (BMT) from an HLA-identical sibling donor is effective therapy for patients with bone marrow failure states and those with hematologic malignancies. However, only a minority of them will have an HLA-identical sibling donor; unrelated donors, matched or partially mismatched, have ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1700874
更新日期:1997-08-01 00:00:00
abstract::Childhood histiocytoses comprise two main diseases, Langerhans cell histiocytosis (LCH) and hemophagocytic lymphohistiocytosis (HLH). LCH is a rare disorder with obscure pathogenesis. Data on clonality suggested neoplastic origin, yet were not convincing. Dysregulation of cytokines and of DC trafficking and cross-talk...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2008.46
更新日期:2008-06-01 00:00:00
abstract::Cord blood transplantation, which has lower risk of graft-versus-host disease than bone marrow transplantation, might have higher risk of infections. A system to quantify four herpesviruses, CMV, human herpesvirus 6 (HHV6), EBV, varicella-zoster virus using the real-time PCR assay was established and applied for prosp...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702710
更新日期:2000-12-01 00:00:00
abstract::Bone marrow transplant-associated thrombotic microangiopathy (BMT-TM) ranges in severity from a self-limiting to a fatal disorder. There is no specific therapy for this condition to date. We have previously described a simple clinical grading system (grade 0-4) for BMT-TM; patients with grade 3-4 BMT-TM do poorly. A p...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1996-01-01 00:00:00
abstract::CAMPATH-1G is an IgG2b rat antihuman (CDw52) monoclonal antibody (MoAb) which is currently being used for T cell depletion in the setting of allogeneic bone marrow transplantation (BMT). In addition it elicits substantial lymphoid depletion, an effect which is being explored for remission induction in patients with ly...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1995-12-01 00:00:00
abstract::Data from human clinical trials and animal experiments have suggested that T lymphocytes in donor marrow help to facilitate engraftment after allogeneic bone marrow transplantation, possibly through a suppressive effect on the immunity of the recipient. In previous studies marrows from HLA-identical donors were treate...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:
更新日期:1988-09-01 00:00:00
abstract::Allogeneic blood stem cell transplantation is associated with multiple complications. We report a case of pneumoperitoneum without peritonitis associated with colonic pneumatosis in a patient who had undergone an allogeneic peripheral blood cell transplant ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701255
更新日期:1998-06-01 00:00:00
abstract::HLA-C matching is an important determinant of outcome after myeloablative unrelated donor (URD) hematopoietic stem cell transplantation. However, its importance in non-myeloablative stem cell transplantation (NST) is not known. We report a retrospective analysis of 111 patients who underwent URD NST, of whom 78 were 1...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,多中心研究
doi:10.1038/sj.bmt.1705315
更新日期:2006-05-01 00:00:00
abstract::The medical staff caring for children with hematological or oncological diseases is often faced with delicate ethical and, at times, legal problems. Although many of these are common to other branches of pediatrics where patients are children lacking decision-making capacity, others, such as bone marrow transplantatio...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:
更新日期:1996-11-01 00:00:00
abstract::We report the unusual complication of vaginal stenosis occurring after allogeneic bone marrow transplantation (BMT) for leukaemia. This was in all likelihood a manifestation of chronic graft-versus-host disease (cGVHD), although the patient has no other stigmata of this and suffered little acute graft-versus-host dise...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701603
更新日期:1999-03-01 00:00:00
abstract::Acquired factor VII (FVII) deficiency in the absence of vitamin K deficiency, oral anticoagulant therapy, synthetic liver dysfunction, or DIC is rare, with only a handful of cases thus far reported. In the period from 1990 to 1996 we identified eight patients with acquired FVII deficiency, all of whom presented with p...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1703381
更新日期:2002-03-01 00:00:00
abstract::The purpose of this study was to determine the nature and prevalence of the psychological symptomatology in parents of children undergoing bone marrow transplantation (BMT) and to investigate the manner in which certain psychosocial factors are related to parental distress associated with the informed consent process....
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1990-12-01 00:00:00
abstract::With the purpose of contributing to a better knowledge of the phenotypic characteristics and functional activity of umbilical cord blood (UCB) lymphocytes, we have carried out extensive immunophenotyping of these cells, evaluated their immune response to polyclonal and allogeneic activation and then compared these res...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1998-07-01 00:00:00
abstract::Bone marrow (BM) histiocystosis in association with the administration of granulocyte-macrophage colony-stimulating factor (GM-CSF) is a rare phenomenon with few published cases. We report two cases of BM histiocystosis in patients with Hodgkin's disease treated with GM-CSF after autologous bone marrow transplantation...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:10.1038/sj.bmt.1701036
更新日期:1998-01-01 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::There have been encouraging reports of the use of recombinant tissue plasminogen activator (tPA) in established veno-occlusive disease (VOD). Haemodialysis has been considered a contraindication to this therapy in view of the potential haemostatic complications. We report a case of a woman who developed moderately sev...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1994-10-01 00:00:00
abstract::Safety of living donors is critical to the success of blood, tissue and organ transplantation. Structured and robust vigilance and surveillance systems exist as part of some national entities, but historically no global systems are in place to ensure conformity, harmonisation and the recognition of rare adverse events...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2013.104
更新日期:2013-11-01 00:00:00
abstract::Although intensive therapy with autologous bone marrow transplantation (ABMT) has improved the outcome of advanced neuroblastoma, nearly half the patients with this disease still relapse after a single ABMT. In our previous study, 10 of 22 patients relapsed within 16 months post-transplantation. Predictive risk-factor...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1996-06-01 00:00:00
abstract::SCT is a curative approach using chemo-, radio- and immunotherapy for malignant and non-malignant hematological disorders. The European Group for Blood and Marrow Transplantation (EBMT) has been collecting yearly data on a survey basis since 1990. The variables within the survey are limited to detailed indications, nu...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:10.1038/bmt.2008.144
更新日期:2008-08-01 00:00:00
abstract::One hundred and ninety-three patients with hematological malignancies and a follow-up > or =1 year, treated with stem cell transplantation (45 autologous, 99 allogeneic T cell-depleted matched, 49 allogeneic T cell-depleted mismatched) from July 1985 to May 1998, were considered evaluable for the development of catara...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1703400
更新日期:2002-03-01 00:00:00
abstract::Incubation of CML marrow in long-term culture (LTC) conditions may result in selection of normal (Ph-) LTC-initiating cells (LTC-IC) as early as 10 days, and in production of Ph- clonogenic cells and mature end cells within 5 weeks. This was the rationale for using marrow cells from 10-day-old LTC to autograft nine ch...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1700777
更新日期:1997-05-01 00:00:00
abstract::Recent reports have suggested that the total number of autologous or allogeneic hematopoietic stem cell (HSC) infused after high-dose chemotherapy might predict survival, post-transplant morbidity and rate of hematopoietic engraftment. However, HSC capable of long-term multilineage potential are still poorly defined, ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1998-07-01 00:00:00
abstract::Recombinant human (rHu) G-CSF has been widely used to treat neutropenia and mobilize PBPCs for their autologous and allogeneic transplantation. It shortens neutropenia and thus reduces the frequency of neutropenic fever. We compared the efficiency of glycosylated rHu and non-glycosylated Hu G-CSF in mobilizing hematop...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2009.150
更新日期:2010-02-01 00:00:00
abstract::HSV can cause oral lesions that exacerbate chemotherapy-related mucositis. Intravenous acyclovir is effective in preventing HSV reactivations, but expensive. Valacyclovir has good bioavailability and has not been studied for prophylaxis of HSV among PCT patients. We compared the efficacy and costs of valacyclovir in p...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/sj.bmt.1703354
更新日期:2002-02-01 00:00:00
abstract::The COVID-19 pandemic has serious implications also for patients with other diseases. Here, we describe the effects of the pandemic on unrelated hematopoietic stem cell donation and transplantation from the perspective of DKMS, a large international donor registry. Especially, we cover the development of PBSC and bone...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-020-01138-0
更新日期:2020-11-20 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::There exists limited information about the usefulness of hemopoietic stem cell transplantation (HSCT) for the treatment of patients with refractory Langerhans cell histiocytosis (LCH). We report here four Japanese pediatric patients with multisystem LCH disease who underwent HSCT between 1994 and 1997. Two of the four...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702001
更新日期:1999-10-01 00:00:00
abstract::This study was performed to investigate the therapeutic effect of umbilical cord serum eyedrops on dry eye associated with graft-versus-host disease (GVHD). Twenty-four eyes of 12 patients with severe dry eye syndrome associated with GVHD were treated with 20% umbilical cord serum eyedrops. Symptom scoring, corneal se...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/sj.bmt.1705566
更新日期:2007-02-01 00:00:00
abstract::A retrospective evaluation of 200 consecutive recipients of autologous peripheral blood stem cell transplantation (PBSCT) was conducted to ascertain the incidence and outcome of infection with Clostridium difficile. The diagnosis was confirmed in 14 patients with diarrhea (15 episodes) at a median of 33 days after ste...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701773
更新日期:1999-05-01 00:00:00
abstract::Allogeneic hematopoietic stem cell transplantation (allo-HSCT) has proven effective in adult T-cell leukemia/lymphoma (ATL) patients. To study the graft-versus-ATL (Gv-ATL) effects after allo-HSCT, we analyzed 21 ATL patients who had been treated at our hospital. Of these, 18 had acute-, 2 had lymphoma- and 1 had chro...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2008.39
更新日期:2008-06-01 00:00:00