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 of publicly banked CB units allows for quicker donor identification and selection. In this review, we examine the current status and cumulative experience of related and unrelated donor CBT for the treatment of non-malignant diseases, including hemoglobinopathies, BM failure syndromes, primary immunodeficiency diseases (PIDs) and inherited metabolic disorders (IMDs), and conclude that CBT offers a promising and effective therapy for these diseases. Future strategies to facilitate earlier diagnosis and to decrease transplant-related risks should further improve the short- and long-term outcomes. Every effort should be made to perform transplantation early in the course of disease before extensive damage to various tissues and organs ensues.
journal_name
Bone Marrow Transplantjournal_title
Bone marrow transplantationauthors
Prasad VK,Kurtzberg Jdoi
10.1038/bmt.2009.290subject
Has Abstractpub_date
2009-11-01 00:00:00pages
643-51issue
10eissn
0268-3369issn
1476-5365pii
bmt2009290journal_volume
44pub_type
杂志文章,评审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::Infectious complications are frequent events in patients undergoing high-dose cytotoxic chemotherapy with subsequent autologous peripheral blood stem cell transplantation (PBSCT). To evaluate whether a single subcutaneous injection of pegfilgrastim (6 mg) is as safe and effective as daily filgrastim (5 mug/kg/day), 60...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704927
更新日期:2005-05-01 00:00:00
abstract::Extracorporeal photopheresis (ECP) is beneficial in patients with T-cell-mediated disorders, including GvHD, but the underlying immunological mechanisms are incompletely understood. Myeloid-derived suppressor cells (MDSCs) are innate immune cells characterized by their capacity to suppress T-cell proliferation. We qua...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2013.236
更新日期:2014-04-01 00:00:00
abstract::Pretransplant influenza vaccination of the donor or allogeneic hematopoietic SCT (HSCT) candidate was evaluated in a randomized study. One hundred and twenty-two HSCT recipients and their donors were assigned to three randomization groups: no pretransplant vaccination (n=38), donor pretransplant vaccination (n=44) or ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,多中心研究,随机对照试验
doi:10.1038/bmt.2015.47
更新日期:2015-06-01 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::Twenty eight patients underwent bone marrow transplantation (BMT) for sickle cell anemia in Belgium. The patients were originating from Central Africa, were young and symptomatic. Engraftment occurred in all patients and was sustained in 25. In 3 patients, a bone marrow rejection was observed. One of these patients un...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1993-01-01 00:00:00
abstract::Preparative regimens without total body irradiation (TBI) have been reported for alternative donor hemopoietic stem cell transplants (HSCT). Between 7 September 1994 and 7 June 1999 48 patients with advanced hematologic malignancies were conditioned with thiotepa (THIO) 15 mg/kg, cyclophosphamide (CY) 150 mg/kg and an...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702719
更新日期:2000-12-01 00:00:00
abstract::The diagnosis of aplastic anemia in children requires exclusion of a variety of inherited or acquired BM failure syndromes with similar phenotypes. An efficient diagnostic plan is important because time from diagnosis to 'final' treatment is directly related to outcome regardless of the therapeutic option chosen. The ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:10.1038/bmt.2012.235
更新日期:2013-02-01 00:00:00
abstract::Data on 477 patients with hematologic malignancies undergoing allogeneic bone marrow transplantation from HLA-identical siblings were analyzed for correlation between donor-recipient ABO blood group incompatibility and the development of elevated bilirubin levels (over 17 mmol/l) after transplantation. The median bili...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1995-06-01 00:00:00
abstract::A 20-month-old boy with infantile leukemia was treated with total body irradiation, etoposide, cyclophosphamide and unrelated cord blood transplantation with a one-antigen mismatch. He relapsed on day 100 and achieved remission after ubenimex administration, and also developed chronic graft-versus-host disease of the ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1703679
更新日期:2002-10-01 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::Urinary trypsin inhibitor has attracted attention as an index of the systemic inflammatory response syndrome. In this study, the urine concentration of trypsin inhibitor was measured to compare the immunological insult of conventional chemotherapy and conditioning chemotherapy for bone marrow transplantation. We also ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702755
更新日期:2001-01-01 00:00:00
abstract::The optimal preparation for stem cell transplantation (SCT) in children with congenital immunodeficiencies is currently unknown. In all, 81 children with immunodeficiency underwent 82 SCTs using reduced-intensity conditioning (RIC). The incidence of significant GVHD was low; viral reactivation was prominent with an un...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704846
更新日期:2005-03-01 00:00:00
abstract::This retrospective study from the Italian Association of Pediatric Hematology Oncology-Bone Marrow Transplant Group (AIEOP-TMO) reports the results of consolidation with high-dose melphalan and autologous hematopoietic stem cell transplantation (auto-HSCT) in patients with acute myeloid leukemia (AML) in first complet...
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.1703122
更新日期:2001-07-01 00:00:00
abstract::Adenovirus has been recognised as an important pathogen in BMT recipients, especially in patients with GVHD and those receiving T cell-depleted allografts. We report adenovirus infections from an ongoing surveillance study in four patients after a non-myeloablative transplant and their improved outcome following withd...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702508
更新日期:2000-08-01 00:00:00
abstract::Allogeneic SCT for older patients remains challenging at least in part due to graft-versus-host disease (GVHD) and higher non-relapse mortality (NRM). We conducted a prospective pilot study primarily for older patients undergoing matched unrelated donor (MUD) SCT using a reduced-intensity (RIC) melphalan-based conditi...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-018-0367-2
更新日期:2019-04-01 00:00:00
abstract::Introduction of genes conferring drug resistance into hematopoietic cells may allow for improved chemotherapy by protection of normally drug-sensitive cells from the toxic side-effects of antitumor agents. We recently reported that transplantation of murine marrow transgenic for drug-resistant dihydrofolate reductase ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701984
更新日期:1999-10-01 00:00:00
abstract::The clinical outcome of advanced-stage Extranodal NK/T cell lymphoma (ENKTL) patients using conventional chemotherapy is extremely poor. The aim of this study was to investigate the outcomes of advanced-stage ENKTL patients treated with non-anthracycline-based chemotherapy followed by upfront autologous stem cell tran...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-020-01160-2
更新日期:2020-12-04 00:00:00
abstract::Reactivation of infections with herpes viruses is a frequent and major cause of morbidity after bone marrow transplantation. In this case report we stress that HSV infections of the colon and small intestine should be considered in the differential diagnosis of diarrhea and intestinal bleeding in the early post-transp...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1700995
更新日期:1997-12-01 00:00:00
abstract::Over the past 15 years, SCT has emerged as a promising treatment option for patients with severe autoimmune diseases (ADs). Mechanistic studies recently provided the proof-of-concept that restoration of immunological tolerance can be achieved by haematopoietic SCT in chronic autoimmunity through eradication of the pat...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:10.1038/bmt.2014.251
更新日期:2015-02-01 00:00:00
abstract::In order to optimize peripheral blood stem cell (PBSC) collection for transplantation, absolute CD34 counts are necessary to determine the exact time-point for sufficient leukapheresis. In an effort to establish and to validate a rapid and reproducible assay for PBSC enumeration, different recommendations for selectio...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701397
更新日期:1998-10-01 00:00:00
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 osteopet...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701474
更新日期:1998-11-01 00:00:00
abstract::Patients with leukemia relapsing after allogeneic hematopoietic SCT have a dismal prognosis. A second SCT offers a further opportunity for cure, but has a high rate of treatment failure. To determine the utility of this option, we analyzed 59 consecutive patients relapsing after a myeloablative HLA-matched sibling T c...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/bmt.2013.39
更新日期:2013-09-01 00:00:00
abstract::Hematopoietic stem cells are extremely sensitive to damage by ionizing radiation. At high doses of radiation, toxicity to the bone marrow may be operationally irreversible. Insufficient functional hematopoietic stem cells remain or irradiated individuals cannot survive sufficiently long enough for functional stem cell...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1987-06-01 00:00:00
abstract::Forty-five adult HLA-matched sibling BMT recipients were randomized to receive inactivated poliovirus vaccine (IPV) at 6, 8 and 14 months (early group, n = 23) or at 18, 20 and 26 months after BMT (late group, n = 22). Ninety-five percent of the early group patients had protective antibody titres of > or = 4 to poliov...
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.1700959
更新日期:1997-10-01 00:00:00
abstract::Relapse rates in patients after autologous bone marrow transplantation (BMT) for acute myeloid leukemia (AML) continue to be high despite the use of aggressive conditioning regimens. Based on studies in the murine system a clinical protocol was developed that utilizes immunotherapy to obtain a graft-versus-leukemia (G...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:
更新日期:1994-09-01 00:00:00
abstract::The aim of the present study was to assess growth, final height, growth hormone (GH) secretion and growth factors after BMT including TBI in childhood. The median age of the 25 participants was 11.3 years at BMT, and a median of 7.5 years had elapsed since BMT. The median height standard deviation score (SDS) declined...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1996-07-01 00:00:00
abstract::For patients with ALL who relapse following allo-SCT, only a second SCT provides a realistic chance for long-term disease remission. We retrospectively analyzed the outcomes of 31 patients with relapsed ALL after a prior allo-SCT, who received a second SCT (SCT2) at our center. With a median follow-up of 3 years, 1- a...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2012.195
更新日期:2013-05-01 00:00:00
abstract::Sickle cell anemia (SCA) remains associated with high risks of morbidity and early death. Allogeneic hematopoietic SCT (HSCT) is the only curative treatment for SCA. We report our experience with transplantation in a group of patients with the non-Black African variant and the Black African variant of SCA. This study ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2014.167
更新日期:2014-11-01 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