Abstract:
:HLA-identical sibling donor transplantation remains the treatment of choice for Wiskott-Aldrich Syndrome (WAS). Since 1990, utilization of alternative donor sources has increased significantly. We report the hematopoietic cell transplantation (HCT) outcomes of 47 patients with WAS treated at a single center since 1990. Improved outcomes were observed after 2000 despite the increased number of alternative donors. Five-year OS improved from 62.5% (95% CI: 34.9% to 81.1%) to 90.8% (95% CI: 67.7% to 97.6%) for patients transplanted during 1990-2000 and 2001-2009, respectively. In multivariate analysis, transplant era significantly impacted OS (P=0.04), whereas age was only marginally significant (P=0.06, Cox proportional hazard analysis). No TRM occurred within the first 100 days among patients transplanted during 2001-2009 compared with 3/16 during 1990-2000, (P=0.03, Fisher's exact test). The extent of HLA mismatch did not significantly affect the incidence of acute GVHD, chronic GVHD or survival. Post-HCT autoimmune cytopenias were frequently diagnosed after 2001: 17/31 (55%) patients. Their occurrence was not associated with transplant donor type (P=0.53), acute GVHD (P=0.74), chronic GVHD (P=0.12), or post-transplant mixed chimerism (P=0.50).
journal_name
Bone Marrow Transplantjournal_title
Bone marrow transplantationauthors
Shin CR,Kim MO,Li D,Bleesing JJ,Harris R,Mehta P,Jodele S,Jordan MB,Marsh RA,Davies SM,Filipovich AHdoi
10.1038/bmt.2012.31subject
Has Abstractpub_date
2012-11-01 00:00:00pages
1428-35issue
11eissn
0268-3369issn
1476-5365pii
bmt201231journal_volume
47pub_type
杂志文章abstract::The role of various cell subpopulations involved in the graft-versus-leukemia (GVL) effect induced by allogeneic bone marrow transplantation (BMT) was investigated in (BALB/c x C57BL/6)F1 (F1) recipients, inoculated with murine B cell leukemia (BCL1), by using monoclonal murine anti-IL-2 receptor antibodies or rabbit ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1995-09-01 00:00:00
abstract::Myeloablative chemotherapy with autologous hematopoietic progenitor cell rescue has been evaluated in the treatment of children and young adults with brain tumors for whom conventional therapy is either too toxic (for example, radiotherapy in infants) or ineffective (for example, recurrent malignant tumors). With this...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
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更新日期:2008-01-01 00:00:00
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journal_title:Bone marrow transplantation
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journal_title:Bone marrow transplantation
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doi:10.1038/s41409-020-01095-8
更新日期:2020-10-31 00:00:00
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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.1703912
更新日期:2003-05-01 00:00:00
abstract::Patients undergoing bone marrow transplantation are profoundly immunosuppressed as a result of their intensive myeloablative chemotherapy and are at high risk for opportunistic fungal infections mainly caused by Candida spp and Aspergillus spp. Trichosporon beigelii (T beigelii) has emerged as a life-threatening oppor...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702231
更新日期:2000-04-01 00:00:00
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journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701297
更新日期:1998-07-01 00:00:00
abstract::Busulfan was added at the dose of 4 mg/kg to 200 mg/kg cyclophosphamide in 81 patients (3-53 years, median 24) with aplastic anemia to reduce graft rejection. Graft-versus-host disease (GVHD) prophylaxis comprised cyclosporine-methotrexate. The number of prior transfusions was 0-276 (median 26), and 48% had received p...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1038/sj.bmt.1704325
更新日期:2004-01-01 00:00:00
abstract::Fludarabine is a purine analog that has demonstrated significant activity in B-cell malignancies, including CLL. Fludarabine also possesses an immunosuppressive effect and is being used to prevent GVHD in hematopoietic stem cell transplantation. However, the molecular mechanism by which fludarabine inhibits immunoreac...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705901
更新日期:2008-02-01 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::This study was designed to test the efficacy and toxicity of combining high-dose cytarabine (3 g/m2 every 12 h x 8 doses day -7 to day -4, total dose 24 g/m2), methyl prednisolone (0.5 mg/kg every 4 h day -7 to day -1), and cyclophosphamide (CY) (60 mg/kg day -3 and day -2) with either total body irradiation (TBI) (90...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1990-05-01 00:00:00
abstract::The pharmacokinetics of tacrolimus following its administration as monotherapy or in combination with corticosteroids or methotrexate to 31 BMT patients are presented. All patients received i.v. tacrolimus initially and were subsequently switched to p.o. dosing. Patients received methotrexate by i.v. bolus on post-tra...
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.1701054
更新日期:1998-01-01 00:00:00
abstract::The remission state of 13 Philadelphia positive chronic myeloid leukemia patients was studied after bone marrow transplantation (BMT) by cytogenetic and Southern blot analysis of the breakpoint cluster region (BCR) gene. Eight of 13 patients showed neither clinical nor genetic evidence of residual disease. In two pati...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1989-07-01 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::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::The interaction between CXCL12 and its receptor CXCR4 plays a crucial role in the homing and mobilization of haematopoietic progenitors. We investigated the putative association between a CXCL12 gene polymorphism, the G --> A transition at position 801 in the 3'-untranslated region (3'UTR), and the yield of CD34(+) pr...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2009.30
更新日期:2009-09-01 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::The indolent non-Hodgkin's lymphomas are theoretically curable through allogeneic haematopoietic stem cell transplantation (allo-HSCT). The applicability of standard conditioning allo-HSCT is, however, restricted by its toxicity. Recently, reduced-intensity conditioning regimens have demonstrated efficacy with signifi...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1703898
更新日期:2003-04-01 00:00:00
abstract::In a series of 74 patients with hematological malignancies undergoing allogeneic bone marrow or peri- pheral blood stem cell transplants from an HLA-identical sibling donor, four developed diffuse alveolar hemorrhage (DAH) between days 0 and 23 post transplant. Diagnosis was made by the radiographic finding of diffuse...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701995
更新日期:1999-10-01 00:00:00
abstract::We investigated the proliferation and therapeutic utility of anti-CD3 activated splenocytes infused into mice following BMT. Using congenic mouse strains we demonstrated that splenocytes activated briefly ex vivo with anti-CD3 plus IL-2 (T-activated killer cells or T-AK) and infused intravenously following BMT had a g...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1994-10-01 00:00:00
abstract::Fourteen children with high risk leukaemia received allogeneic bone marrow transplants from HLA-identical MLC-compatible sibling donors. All bone marrows were T cell depleted and a T cell addback was prepared from the donor's peripheral blood so that the mean total number of CD3+ cells given was 2.6 (1.0-4.1) x 10(5)/...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1991-11-01 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::Acute hemorrhagic cystitis due to adenovirus infections may be more severe or protracted in immunocompromised patients. This patient with chronic graft-versus-host disease following allogeneic marrow transplantation for acute myelogenous leukemia developed painful hematuria due to adenovirus infection that failed to r...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1991-03-01 00:00:00
abstract::The role of autologous bone marrow transplantation in the treatment of malignant disease is currently being evaluated. The peripheral blood represents an alternative source of haemopoietic progenitors and the use of circulating rather than bone marrow stem cells permits autografting in patients with infiltrated bone m...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:
更新日期:1986-12-01 00:00:00
abstract::A double-blind, placebo-controlled study was conducted to evaluate the effect of orally administered pilocarpine on unstimulated whole-saliva flow and composition in 28 patients with chronic graft-versus-host disease (cGVHD). Thirteen patients were treated with pilocarpine of 20 mg/day orally for 7 days, and 15 patien...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705621
更新日期:2007-04-01 00:00:00
abstract::Hepatic venocclusive disease (VOD) is a common toxicity associated with myeloablative chemotherapy or chemoradiotherapy used to prepare patients for stem cell transplantation. A sizable proportion of patients who develop VOD die. It is clear that injury to endothelial cells and hepatocytes in zone 3 of the liver acinu...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
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更新日期:2001-06-01 00:00:00
abstract::In the United States the increasing number of Food and Drug Administration (FDA)-approved, innovative, and potentially effective commercial cancer therapies pose a significant financial burden on public and private payers. Chimeric antigen receptor (CAR) T cells are prototypical of this challenge. In 2017 and 2018, ti...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
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更新日期:2020-09-01 00:00:00
abstract::This retrospective study was conducted to determine whether the total number of nucleated cells (TNC)/kg collected at marrow harvest was associated with outcome in 151 patients with acute myelogenous leukemia (AML) who received an autologous purged (n = 67) or non-purged (n = 84) marrow transplant. At the time of tran...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1995-06-01 00:00:00
abstract::A retrospective comparison was carried out on adult patients receiving HLA-identical allogeneic haemopoietic stem cell transplants from siblings in Australia in 1996, comparing bone marrow with G-CSF-mobilised peripheral blood stem cells. A total of 131 transplant recipients from nine centres were included in this stu...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,多中心研究
doi:10.1038/sj.bmt.1703088
更新日期:2001-07-01 00:00:00
abstract::A cohort of 138 children with 144 hematopoietic stem cell transplantation (HSCT) performed in 1997-2006 were analyzed to evaluate risk factors and mortality predictors of hepatic veno-occlusive disease (VOD). Nineteen patients (13.2%) developed VOD (nine boys, median age 3.5 years) at 1-21 days after HSCT (median 13 d...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
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更新日期:2007-11-01 00:00:00