Abstract:
:Hematopoietic stem cell transplantation (HCT) has been increasingly used for patients with inherited metabolic disorders (IMD). Immune mediated cytopenias (IMCs) after HCT, manifesting as hemolytic anemia, thrombocytopenia, and/or neutropenia, are recognized as a significant complication in this patient population, yet our understanding of the incidence, risk factors, and pathophysiology is currently limited. Review of the published literature demonstrates a higher incidence in younger patients who undergo HCT for a nonmalignant disease indication. However, a few reports suggest that the incidence is even higher among those with IMD (incidence ranging from 10 to 56%). This review summarizes the literature, provides an approach to better understanding of the possible etiology of IMCs, and proposes a diagnostic and management plan for patients with IMD who develop single or multi-lineage cytopenias after HCT.
journal_name
Bone Marrow Transplantjournal_title
Bone marrow transplantationauthors
Gupta AO,Jan Boelens J,Ebens CL,Kurtzberg J,Lund TC,Smith AR,Wagner JE,Wynn R,Blazar BR,Orchard PJdoi
10.1038/s41409-020-01179-5subject
Has Abstractpub_date
2021-01-13 00:00:00eissn
0268-3369issn
1476-5365pii
10.1038/s41409-020-01179-5pub_type
杂志文章,评审abstract::Twenty three allogeneic bone marrow transplant (BMT) patients with female donors and 23 female autologous transplant patients were assessed for clonality status after transplant to determine the nature of haemopoietic reconstitution. The X chromosome probes PGK, HPRT and M27 beta were used to assess clonality by analy...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1995-01-01 00:00:00
abstract::Non-compliance has received significant attention in medicine, yet few studies have examined its correlates in autologous hematopoietic SCT (AHSCT) patients. This study examined predictors of non-compliance in a sample of 151 AHSCT patients treated in an outpatient setting. Before AHSCT, participants completed a valid...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/bmt.2011.129
更新日期:2012-04-01 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::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::Hematopoietic stem cell transplantation (HSCT) is becoming an increasingly recognized indication for treatment of autoimmune diseases and severe immune-mediated disorders. However, multicenter registry data have demonstrated higher than anticipated early toxicity, approximately 10% for autoimmune diseases in general, ...
journal_title:Bone marrow transplantation
pub_type: 社论,评审
doi:10.1038/sj.bmt.1704671
更新日期:2004-11-01 00:00:00
abstract::Children with high risk or relapsed acute lymphoblastic leukaemia (ALL) can benefit from allogeneic haematopoietic stem cell transplantation (SCT). To reduce transplantation-associated complications, the BFM study group, the IBFM study group and the PD-WP-EBMT initiated a prospective cooperative multicentre trail for ...
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.1704835
更新日期:2005-03-01 00:00:00
abstract::Younger children are considered to be more vulnerable to late effects (LE), which prompted us to study LE in patients after haematopoietic stem cell transplantation (HSCT) for a haematological malignancy before the age of 3. In this multicentre EBMT study, cumulative incidence (CI) and severity of endocrine LE, centra...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,多中心研究
doi:10.1038/bmt.2016.139
更新日期:2016-11-01 00:00:00
abstract::We analyzed late cardiovascular outcomes of 661 patients who survived at least 2 years from hematopoietic cell transplantation for childhood hematologic malignancy between 1995 and 2008. Center for International Blood and Marrow Transplant Research data was supplemented with surveys focused on cardiotoxicity and poten...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-018-0155-z
更新日期:2018-10-01 00:00:00
abstract::One hundred and one donors who had received filgrastim (rhG-CSF) for the purpose of donating either granulocytes or peripheral blood stem cells (PBSC) for their relatives more than 3 years ago were contacted. All donors had received daily rhG-CSF at a median dose of 16 microg/kg/day (range 3-16) for a median of 6 days...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702072
更新日期:2000-01-01 00:00:00
abstract::Children receiving a bone marrow transplant (BMT) are at risk for neuropsychological late effects because of potentially neurotoxic chemotherapy and total body irradiation. The goal of this study was to prospectively and longitudinally assess the intellectual and adaptive functioning of children receiving a BMT. This ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1700699
更新日期:1997-03-01 00:00:00
abstract::The purpose of this trial was to study feasibility and tolerance of a dose-intensive multicyclic alternating induction chemotherapy with repeated stem cell support in a series of 43 metastatic breast cancer patients. Anthracycline-naive patients (n = 21) received cyclophosphamide 2.5 g/m(2) plus doxorubicin 80 mg/m(2)...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/sj.bmt.1703125
更新日期:2001-08-01 00:00:00
abstract::The administration of G-CSF post transplant has been shown to accelerate the time to neutrophil engraftment. However, this does not necessarily translate into a meaningful clinical benefit to the patient. This randomized study was designed to determine the role of G-CSF following transplantation in patients with breas...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章,多中心研究,随机对照试验
doi:10.1038/sj.bmt.1703539
更新日期:2002-05-01 00:00:00
abstract::Clinical trials have shown that a strategy for haploidentical transplantation based on the infusion of high numbers of T-cell-depleted hematopoietic progenitor cells and no post-transplant immunosuppression controls graft rejection and GvHD in patients with acute leukemia. Overall, event-free survival compares favorab...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2015.88
更新日期:2015-06-01 00:00:00
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, alterna...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2012.140
更新日期:2013-02-01 00:00:00
abstract:SUMMARY:High-dose busulfan is an important component of myeloablative regimens. Variable drug exposure may occur following oral administration. Therefore, the use of intravenous busulfan has been advocated. Previous work has suggested a cumulative dosage of 16 mg/kg for haematopoietic transplantation in children less t...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704209
更新日期:2003-10-01 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::One hundred patients with chronic myeloid leukemia (CML) submitted to bone marrow transplantation (BMT) were included in a cooperative cytogenetic study. Relapse (defined on the basis of hematological and cytogenetic findings) occurred in 24 (24%) patients at different intervals after BMT. In 18 of these patients (stu...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1988-09-01 00:00:00
abstract::Patients with relapsed ALL frequently have short duration second or later remissions, leaving only a brief window of time when it is possible to perform BMT. When no sibling donor is available, identifying and unrelated donor in a timely fashion can be difficult while autologous BMT (ABMT) can be performed more quickl...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1996-05-01 00:00:00
abstract::Nine individuals between 15 and 43 years of age with non-Hodgkin's lymphoma underwent allogeneic marrow transplantation following busulfan 16 mg/kg and cyclophosphamide 120 mg/kg. These individuals were not considered optimal candidates for autologous transplantation chiefly because of marrow involvement or resistance...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:
更新日期:1990-01-01 00:00:00
abstract::We should consider both the treatment effects and adverse effects of autologous hematopoietic stem cell transplantation (AHSCT) on multiple sclerosis (MS) and neuromyelitis optica spectrum disorders (NMOSD). Articles exploring the effect and safety of AHSCT in the treatment of MS and NMOSD and published before Decembe...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-020-0810-z
更新日期:2020-10-01 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::Hemorrhagic cystitis (HC) is a debilitating complication following allogenic hematopoietic cell transplantation (HCT). HLA disparity and T-cell depletion have been implicated as risk factors for HC. However, reports on the incidence and risk factors for HC in ex vivo T-cell depleted haploidentical HCT (haploHCT) in ch...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-019-0672-4
更新日期:2020-01-01 00:00:00
abstract::While bone marrow (BM) grafts were initially used for T-replete HLA-haploidentical related donors transplantation (Haplo-SCT) with post-transplantation cyclophosphamide (PT-Cy), the use of peripheral blood stem cell (PBSC) remains debated. We thus conducted a detailed analysis evaluating the incidence, risk factors, a...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,多中心研究
doi:10.1038/s41409-019-0500-x
更新日期:2019-11-01 00:00:00
abstract::On January 20, 2020, the first patient with coronavirus disease 2019 (COVID-19) in the United States of America was diagnosed in Washington state, which subsequently experienced rapidly increasing numbers of COVID-19 cases, hospitalizations, and deaths. This placed the Seattle Blood and Marrow Transplant Program at Fr...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:10.1038/s41409-020-01068-x
更新日期:2020-09-26 00:00:00
abstract::Acute graft-versus-host disease (aGVHD) is a frequent and major complication after allogeneic stem cell transplantation. For many years psoralen and ultraviolet (UV)-A light have been used in the treatment of chronic cutaneous graft-versus-host disease, but few patients have received PUVA therapy for aGVHD. We assesse...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701537
更新日期:1999-01-01 00:00:00
abstract::A second meeting on reduced intensity allogeneic stem cell transplants (RI-HSCT) was convened in Zurich in February 2001 and focused on transplant-related mortality (TRM) and graft-versus-host disease (GVHD). Retrospective and prospective studies from the EBMT, national groups and single institutions included over 900...
journal_title:Bone marrow transplantation
pub_type: 共识发展会议,杂志文章,评审
doi:10.1038/sj.bmt.1703355
更新日期:2002-02-01 00:00:00
abstract::In this retrospective study, we evaluated the predictability of PBSC dose for hematopoietic engraftment comparing that calculated by ideal body weight (IBW) vs another calculated by actual body weight (ABW) for each patient. Sixty-three consecutive patients treated similarly using one transplant protocol were analyzed...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701731
更新日期:1999-05-01 00:00:00
abstract::We present 60 patients with refractory (n=8) or relapsed (n=52) adult ALL who received allogeneic hematopoietic SCT (HSCT) with (n=41) or without (n=19) prior reinduction chemotherapy. In our center, omission of reinduction is recommended if a suitable donor is promptly available, tumor burden is moderate and disease ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2008.258
更新日期:2008-12-01 00:00:00
abstract::The best donors for hematopoietic SCT (HSCT) are fully-matched siblings. In patients without fully-matched siblings, HLA registries or cord blood banks are alternative strategies with some restrictions. Owing to the high rate of consanguineous marriage in our country, between 2006 and 2013, extended family searches we...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2014.307
更新日期:2015-04-01 00:00:00
abstract::Authors report a case of P. carinii pneumonia in a child with 5% CD4 lymphocytes (absolute number 5/microliters) after autologous bone marrow transplantation followed by pulmonary irradiation. Serial evaluation of CD4 lymphocyte count or percentage or, at least, detection of significant and persistent lymphopenia coul...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1995-06-01 00:00:00