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 are documented. Clinical manifestations and course are highly variable, ranging from self-healing solitary bone lesion to disseminated, multi-organ involvement with 20% fatality rate despite standard chemotherapy. HSCT has been applied in less than 50 cases, outside any trial, with good disease control but elevated early toxicity. The familial form of HLH (FHL) has been recognized as congenital immune deficiency, with mutations of PRF1, Munc13-4, syntaxin11 genes resulting in defective cellular cytotoxicity machinery. Chemo-immunotherapy allows temporary disease control, but HSCT holds as the only procedure with potential for cure. Rapid identification of genetic defects allows differential diagnosis from transient, virus-associated HLH, thus indicating early HSCT. The role of HSCT in childhood histiocytoses is thus very important. Better understanding of the pathogenesis, in particular of genetic and immune function defects, will help to tailor indications and, possibly less toxic, conditioning regimens, reducing treatment-related mortality, and thus disclosing the way to final cure.
journal_name
Bone Marrow Transplantjournal_title
Bone marrow transplantationauthors
Caselli D,Aricò M,EBMT Paediatric Working Party.doi
10.1038/bmt.2008.46subject
Has Abstractpub_date
2008-06-01 00:00:00pages
S8-S13eissn
0268-3369issn
1476-5365pii
bmt200846journal_volume
41 Suppl 2pub_type
杂志文章abstract::From 1983 to 1994 two types of trials were performed. Between 1983 and 1987 a modified VAPA protocol (post-remission therapy with intensive sequential blocks for 12-16 months) was given to 40 patients from two institutions. CR was attained in 75% and 5-year EFS was 35%. In 1988 a post-remission protocol based on inten...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1996-11-01 00:00:00
abstract::Busulfan is currently used as a main component in the conditioning regimen prior to allogeneic stem cell transplantation (SCT). Several studies have shown a correlation between exposure to busulfan and transplantation-related liver toxicity, such as venoocclusive disease (VOD) in patients undergoing SCT. Busulfan is m...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704143
更新日期:2003-08-01 00:00:00
abstract::We initiated a randomized study of amifostine (the organic thiophosphate formerly known as WR-2721) given to patients during myeloablative conditioning therapy for allogeneic bone marrow transplantation. Amifostine was given at a dose of 1000 mg/day of conditioning and was well tolerated if attention was given to seru...
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.1704521
更新日期:2004-07-01 00:00:00
abstract::Peripheral blood progenitor cell (PBPC) populations used for transplantation were analyzed for the presence of CD34+ cells, colony-forming cells (initial CFC), and long-term culture initiating cells (LTC-IC) cultured on irradiated stroma for 5 weeks. Thirty-eight leukapheresis products were studied from 11 patients wi...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1994-04-01 00:00:00
abstract::The introduction of hematopoietic growth factors (HGFs) offers new opportunities for autologous transplantation by facilitating and enriching collection of circulating progenitor cells from peripheral blood as a source of stem cell rescue. Substitution of peripheral blood progenitor cells (PBPC) from bone marrow in au...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1994-12-01 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::Several approaches have been developed to overcome historical barriers associated with poor outcomes in the setting of HLA-haploidentical allogeneic transplantation (HaploSCT). Here, we examine the outcome of patients with various hematological disorders undergoing HaploSCT with high-dose, post-transplantation cycloph...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,多中心研究
doi:10.1038/s41409-019-0475-7
更新日期:2019-10-01 00:00:00
abstract::We report a patient with CML who developed a reversible dilated cardiomyopathy with cardiac failure following 10 months of IFN therapy. Despite the previous cardiomyopathy, he tolerated subsequent allogeneic BMT without any adverse cardiac events. Reversible IFN-induced cardiomyopathy should not be considered a contra...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701188
更新日期:1998-04-01 00:00:00
abstract::Skin graft versus host disease (GVHD) can affect quality of life in hematopoietic stem cell transplant recipients. Therapeutic options for steroid-refractory GVHD are limited. We report the first prospective pilot study evaluating the topical vitamin D3 analog Calcipotriene (DOVONEX 0.005% cream) for acute skin GVHD i...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-020-01189-3
更新日期:2021-01-08 00:00:00
abstract::Bone marrow transplantation has been successfully employed in the treatment of Diamond-Blackfan anemia (DBA). Transplantation with bone marrow stem cells involves, however, considerable treatment-related toxicity, partially in the form of GVHD. Through the use of cord blood stem cells it may be possible to reduce the ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1700675
更新日期:1997-03-01 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 role of high-dose therapy and autologous stem cell transplantation (ASCT) in the treatment of patients with Ewing's sarcoma (EWS) remains uncertain. From November 1985 to September 1994, 13 patients aged 16-30 years (median 20.5) received high-dose melphalan (HDM) 140-200 mg/m2 +/- 500 cGy TBI followed by ASCT for...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1996-08-01 00:00:00
abstract::Data on long-term follow-up of donors for hematopoietic stem cell transplantation (HSCT) are limited. Donors of 612 adult allogeneic HSCT were studied, at a median of 81 (14-181) months post-HSC donation. Nine donors had severe health problems. Five donors died from aggressive malignancies or terminal illness, at a me...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705274
更新日期:2006-03-01 00:00:00
abstract::Acute GVHD (aGVHD) is an immunologic complication of allogeneic hematopoietic cell transplantation (HCT) that can range from mild to life-threatening. Models to predict patients at risk of poor outcomes have been developed using both clinical and laboratory data, and the time to test these models in clinical trials ha...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:10.1038/bmt.2015.261
更新日期:2016-02-01 00:00:00
abstract::Among 145 consecutive patients undergoing bone marrow transplantation (BMT) for leukemia or aplastic anemia. 30 (21%) were found positive for hepatitis B surface antigen (HBsAg) in serum either before or after BMT. Their serologic profile and clinical outcome are described. Nine out of 30 patients were HBsAg positive ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1990-07-01 00:00:00
abstract::In this study, cytogenetic and molecular analyses were employed to assess the response to therapy in 29 chronic myeloid leukemia patients undergoing high dose chemotherapy followed by autologous stem cell infusion. Of these, 11 had previously achieved hematologic remission and cytogenetic improvement after alpha-2b in...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1990-10-01 00:00:00
abstract::The present study analyses the influence of high-dose chemotherapy (HD) and autologous stem cell transplantation on natural and vaccine-induced specific immunity in breast cancer patients. Peripheral blood was collected from five breast cancer patients at serial time points in connection with treatment and in a follow...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1703521
更新日期:2002-04-01 00:00:00
abstract::As a consequence of the significantly larger inoculum of lymphoid cells present in peripheral blood stem cell (PBSC) harvests compared to bone marrow (BM), it is possible that autoPBSCT recipients may have an earlier and*or enhanced response to vaccines. Until data to confirm this become available, the European Blood ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1703239
更新日期:2001-10-01 00:00:00
abstract::Thrombotic microangiopathy (TMA) is a significant complication after hematopoietic stem-cell transplantation (HSCT); however, there is little information on it following reduced-intensity cord blood transplantation (RI-CBT). We reviewed the medical records of 123 adult patients who received RI-CBT at Toranomon Hospita...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1705099
更新日期:2005-09-01 00:00:00
abstract::Reduced sexual function may have negative implications on health related quality of life among lymphoma survivors. A national cross-sectional study among long-term lymphoma survivors after high-dose therapy with autologous stem-cell transplantation auto-SCT treated during 1987-2008 was conducted in 2012-2014. The curr...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-019-0745-4
更新日期:2020-05-01 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::A total of 81 adults with acute myeloid leukemia (AML) (47% favorable karyotypes) were autografted in first remission after melphalan-total body irradiation, having received 0 (n=7), 1 (n=19), 2 (n=51), or 3 (n=4) consolidation chemotherapy cycles before harvest. The cumulative 5-year incidences of relapse and transpl...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704119
更新日期:2003-07-01 00:00:00
abstract::We describe seven patients who developed symptoms including severe headache, circumoral paresthesia, and facial flushing during high-dose carmustine (BCNU) infusion as part of the preparative regimen for autologous peripheral blood stem cell (PBSC) transplantation for metastatic breast cancer. Five patients responded ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1700743
更新日期:1997-04-01 00:00:00
abstract::IL-7 is produced by stromal cells and is the major lympho- and thymopoietic cytokine. IL-7 induces proliferation and differentiation of immature thymocytes, and protects thymocytes from apoptosis by induction of bcl-2 expression. The regulation of IL-7 production is poorly characterized, although down-regulation by tr...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701655
更新日期:1999-04-01 00:00:00
abstract::Bone marrow (BM) angiogenesis is increased in multiple myeloma and is an important prognostic factor. However, prior studies were mainly done on patients receiving conventional chemotherapy and there is limited data on its prognostic value in patients undergoing high-dose therapy (HDT) as initial therapy. We studied B...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704555
更新日期:2004-08-01 00:00:00
abstract::The protection of hematopoietic stem and progenitor cells and their environment is required for recovery from radiation-induced (RI) myelosuppression. To achieve this goal, we propose a new gene therapy strategy based on local and short-term synthesis and expression of Sonic hedgehog morphogene (Shh) at the niche leve...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2013.162
更新日期:2014-02-01 00:00:00
abstract::Ovarian failure after allogeneic stem cell transplant (allo-SCT) is an important risk factor for development of osteoporosis. We investigated the effects of various antiresorptive treatments in long-term surviving females with ovarian failure after allo-SCT. A total of 60 women with osteoporosis or osteopenia were div...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,随机对照试验
doi:10.1038/sj.bmt.1705196
更新日期:2006-01-01 00:00:00
abstract::Sixty-nine adolescents and adults 15-51 years of age with untreated acute lymphoblastic leukaemia (ALL, 54 patients) or lymphoblastic lymphoma (LL, 15 patients) were referred for intensive antileukaemic therapy. Patients were treated according to one of two protocols. Both included induction and consolidation with vin...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:
更新日期:1994-11-01 00:00:00
abstract::Graft failure (GF) remains a major complication of cord blood transplantation (CBT). Although the presence of pretransplant, donor-specific anti-HLA antibodies (DSA) was reported to be associated with an increased risk of GF after CBT, data are still limited. Thus, we conducted a retrospective analysis of recipients o...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-019-0712-0
更新日期:2020-04-01 00:00:00
abstract::A potential link between arsenic (ATO)-based therapy and delayed hematopoietic recovery after autologous hematopoietic SCT (HSCT) for acute promyelocytic leukemia (APL) has previously been reported. We retrospectively reviewed the clinical histories of 58 patients undergoing autologous HSCT for APL at 21 institutions ...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1038/bmt.2014.201
更新日期:2015-01-01 00:00:00