Abstract:
:Approaches to determine whether one transplant-related therapy is better than another include: (1) using experimental data, such as those from randomized controlled trials (RCTs); (2) using observational data, such as those from observational databases (ODBs) and (3) using conclusions from the structured quantification of expert opinion based on a consideration of evidence from RCTs, ODBs and other sources. Large RCTs are widely and appropriately regarded as the gold standard of clinical investigation. However, data from large RCTs are rarely available for transplant-related therapy questions. We discuss some of the limitations of RCTs in the transplant setting often including small size and short follow-up. These limitations are only partly solved by meta-analyses of RCTs. Data from high-quality ODBs are not only often useful in this setting but also have limitations. Biases may be difficult or impossible to identify and/or adjust for. However, ODBs have large numbers of diverse subjects receiving diverse therapies and analyses that often give answers more useful to clinicians than RCTs. Side-by-side comparisons suggest analyses from high-quality ODBs often give similar conclusions to meta-analyses of high-quality RCTs. Meta-analyses combining data from RCTs and ODBs are sometimes appropriate. Quantitation of expert opinion, when of high quality, is also useful: experts rarely disagree under precisely defined circumstances and their consensus conclusions are often concordant with results of high-quality RCTs and ODBs. We suggest increased use of ODBs and expert opinion as reliable and effective ways to determine relative efficacies of new therapies in transplant settings.
journal_name
Bone Marrow Transplantjournal_title
Bone marrow transplantationauthors
Gale RP,Eapen M,Logan B,Zhang MJ,Lazarus HMdoi
10.1038/bmt.2008.447subject
Has Abstractpub_date
2009-03-01 00:00:00pages
435-46issue
6eissn
0268-3369issn
1476-5365pii
bmt2008447journal_volume
43pub_type
杂志文章abstract::Graft rejection has been a problem after bone marrow transplantation for patients with severe aplastic anemia (SAA). Ten children with SAA were conditioned for bone marrow transplantation from HLA-identical siblings, using cyclophosphamide (CY, 50 mg/kg) plus antithymocyte globulin (ATG, 15 mg/kg) for 4 successive day...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1700795
更新日期:1997-06-01 00:00:00
abstract::Intensive high-dose chemotherapy with peripheral blood progenitor cell (PBPC) transplantation is a common strategy for aggressive non-Hodgkin's lymphomas (NHL). A retrospective cost-effectiveness analysis of CD34+ cell dose was carried out. Between 1994 and 1998, 28 patients were included. Efficacy was measured by the...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702378
更新日期:2000-05-01 00:00:00
abstract::We performed quantitative PCR-based serial chimerism testing of whole blood (WB) and CD3+ cells and retrospectively correlated the results of chimerism tests and the risk of graft loss in children undergoing transplant for non-malignant disorders. Twenty-four children were included in this study. All patients initiall...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2008.89
更新日期:2008-07-01 00:00:00
abstract::Nine patients with advanced Hodgkin's lymphoma (HL) who had undergone allogeneic stem cell transplantation (allo-SCT) received donor leukocyte infusions (DLIs) for treatment of persistent or progressive disease (PD). A total of 15 DLIs were performed, with four patients receiving more than one DLI. In four patients, p...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704621
更新日期:2004-09-01 00:00:00
abstract::Reverse transcriptase polymerase chain reaction (RT-PCR) was applied to evaluate the frequency of tumour cells in PBPC products from 15 high risk Ewing tumour (ET) patients who were treated according to EICESS 92 with high-dose chemotherapy (HDC) and stem cell rescue. Initial tumour cell contamination of the bone marr...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701924
更新日期:1999-08-01 00:00:00
abstract::Conditioning regimens preceding hematopoietic SCT (HSCT) usually consist of high-dose chemotherapy. Chemotherapy and radiation therapy are associated with increased formation of free radicals and depletion of critical plasma and tissue antioxidants. Oxidative stress and antioxidant depletion have been described during...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2011.145
更新日期:2012-05-01 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::Among the drugs used in conditioning regimens for stem cell transplantation, high-dose melphalan (HDM) plays an important role for both its strong myeloablative effect and for its favourable dose-response ratio. Here we report five cases of high frequency atrial fibrillation (AF) developing after HDM. Duration of the ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701217
更新日期:1998-05-01 00:00:00
abstract::Information regarding the chimeric status of hematopoietic stem cell transplantation (HSCT) recipients is of great significance when comparing different conditioning and prophylactic therapies. In recent years, short tandem repeats/variable number tandem repeats (STRs/VNTRs) have emerged as the best tool for chimerism...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,随机对照试验
doi:10.1038/sj.bmt.1705626
更新日期:2007-05-01 00:00:00
abstract::Lupus inhibitors have been reported in a number of pathologic states in which there is a disruption of normal immunoregulation. We report here the development of new lupus inhibitors following bone marrow transplantation. Retrospective analysis of 1292 patients undergoing transplantation at the University of Minnesota...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1995-02-01 00:00:00
abstract::The majority of adult patients have sexual concerns after post-haematopoietic cell transplantation. Even so, health-care professionals (HCP) do not routinely discuss these problems. We, therefore, surveyed all the members of the European Society for Blood and Marrow Transplantation to evaluate the barriers and facilit...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-017-0027-y
更新日期:2018-03-01 00:00:00
abstract::Transplantation of growth factor-mobilized peripheral blood progenitor cells (PBPC) is widely used in the treatment of several neoplastic diseases. While in PBPC harvests the presence of several accessory immune and tumor cells has been documented, that of stromal cells has not been reported. In the present study, we ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1700890
更新日期:1997-08-01 00:00:00
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::We retrospectively analyzed red blood cell (RBC) support and alloimmunization rate in 218 consecutive patients - 128 from the Pediatric Department and 90 from the adult Hematology Department - undergoing hematopoietic stem cell transplantation (HSCT) between 1994 and 2000. In the pre-HSCT period, the pediatric patient...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704114
更新日期:2003-07-01 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 an 11-year old female with myelodysplastic (refractory anemia with excess of blasts) presentation of Fanconi anemia. After failure of initial chemotherapy with low doses of 6-mercaptopurine and prednisolone she underwent allogeneic bone marrow transplantation (BMT) from her HLA-matched sibling. Busulfan 8 mg...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1700662
更新日期:1997-02-01 00:00:00
abstract::Plerixafor augments PBSC collection, but the optimal approach for incorporating it into mobilization is uncertain. Forty-nine consecutive patients mobilized with G-CSF alone were analyzed, and a day 4 peripheral blood CD34(+) cell count of 0.015/ml was found to predict for a day 5 apheresis yield of 2 × 10(6) CD34(+) ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2012.74
更新日期:2012-12-01 00:00:00
abstract::Hematopoietic cell transplantation (HCT) following nonmyeloablative conditioning (NMSCT) may be associated with a reduced risk of infection compared to standard allogeneic HCT. We retrospectively analyzed incidence and risk factors of infection in 62 patients undergoing NMSCT with low-dose TBI +/- fludarabine and post...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705255
更新日期:2006-02-01 00:00:00
abstract::A phase I/II trial evaluated early administration and dose escalation of interleukin (IL)-2 with granulocyte macrophage colony stimulating factor (GM-CSF) post-transplant. Following melphalan (200 mg/m(2)) and an autologous transplant, IL-2 was initiated (day 0) and continued for 4 weeks. GM-CSF (250 mcg/m(2)/day) beg...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705665
更新日期:2007-06-01 00:00:00
abstract::Rapid, specific and sensitive methods are essential for early detection of CMV infection in patients after marrow transplantation. Thus, in a prospective study, PCR from whole blood, plasma-PCR, pp65-antigenemia and virus culture were used and compared in 20 consecutive marrow transplant recipients for early diagnosis...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1996-05-01 00:00:00
abstract::Repeated high-dose (HD) chemotherapy with peripheral blood stem cell (PBSC) transplantation is a new modality aimed at increasing both the dose and its intensity in the treatment of chemosensitive tumours. The aim of this study was to evaluate the tolerance, pharmacokinetics (PK) and pharmacodynamics (PD) of HD single...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/sj.bmt.1702806
更新日期:2001-03-01 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::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::Inadequate stem cell mobilization is seen in approximately 25% of patients undergoing autotransplantation for hematologic malignancies. Remobilization strategies include chemotherapy/cytokine combinations or high-dose cytokines alone or in combination. From 1/1997 to 7/2002, we remobilized 86 patients who failed an in...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704486
更新日期:2004-05-01 00:00:00
abstract::We retrospectively reviewed the pulmonary toxicity of six high dose chemotherapy protocols using four chemotherapy regimens in the treatment of solid tumors. All protocols used either high dose cyclophosphamide or ifosfamide in combination with one to three additional chemotherapeutic agents. In each protocol autologo...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1992-07-01 00:00:00
abstract::We report a case of unexplained graft failure in a 33-year-old man who received an allogeneic bone marrow graft that contained a donor-recipient mismatch involving the highly immunogenic NA1 neutrophil-specific alloantigen system. Laboratory and clinical data suggest that an alloimmune process related to the neutrophi...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1993-03-01 00:00:00
abstract::AlloSCT is a potentially curative procedure for haematological malignancies and marrow failure syndromes. However, unlike leukaemia and lymphoproliferative disorders, AlloSCT has yet to find its place in the clinical management of patients with multiple myeloma. AlloSCT in multiple myeloma is associated with a high pr...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:10.1038/bmt.2008.397
更新日期:2009-01-01 00:00:00
abstract::Patients with poor-risk leukemia have a high relapse rate despite allogeneic transplant. We report on the phase-2 trial of an intensified allogeneic transplant regimen whose aim was tolerable toxicity and durable remission. Study patients (n=30) had unfavorable first remission cytogenetics, progression from myelodyspl...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2010.295
更新日期:2011-09-01 00:00:00
abstract::Forty-six children with juvenile myelomonocytic leukemia (JMML) diagnosed between 1978 and 1993 in 12 centers were retrospectively studied. There is no evidence that any conventional treatment influences the long-term evolution of JMML. Among 28 patients treated without bone marrow transplantation (BMT), 26 died (medi...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,多中心研究
doi:
更新日期:1996-12-01 00:00:00
abstract::A total of 11 high-risk Korean acute myeloid leukemia (AML) patients received stem cell transplantation from human leukocyte antigen (HLA) haploidentical donors. Specifically, for eight patients with 2-3 mismatched antigens and bidirectional vectors, we used a newly designed conditioning regimen that consists of total...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704900
更新日期:2005-05-01 00:00:00