Abstract:
:Hurler syndrome (MPS1H) is a progressive inborn error of mucopolysaccharide metabolism leading to premature death. Allogeneic hematopoietic cell transplantation (HCT) can achieve stabilization and improve long-term survival. However, large studies have shown that preparative regimen-related toxicity (RRT) and graft failure rates have been relatively high. We transplanted five Hurler children with a fludarabine-based conditioning regimen, consisting of fludarabine/busulphan/ATG for matched family donor (MFD), with the addition of melphalan for mismatched family donor and matched unrelated donor (MUD) transplantations. Median age at HCT was 27 months (range 10-36). The source of stem cells was bone marrow in one MFD and CD34-selected PBSC in four patients. Median CD34+ cell dose was 25 x 10(6)/kg (range 11.5-54). No RRT > grade II was observed. All patients are surviving at a median of 32 months (range 14-41) and show sustained donor engraftment with 3/5 having full donor chimerism, and 2/5 mixed chimerism (> 85%). We conclude that this regimen is feasible and has low toxicity in Hurler children. In combination with high doses of CD34+ selected cells (> 10 x 10(6)/kg) and donor lymphocyte infusions, stable engraftment could be achieved in unrelated and mismatched related transplantations.
journal_name
Bone Marrow Transplantjournal_title
Bone marrow transplantationauthors
Grigull L,Beilken A,Schrappe M,Das A,Luecke T,Sander A,Stanulla M,Rehe K,Sauer M,Schmid H,Welte K,Lukacs Z,Gal A,Sykora KWdoi
10.1038/sj.bmt.1704786subject
Has Abstractpub_date
2005-02-01 00:00:00pages
265-9issue
3eissn
0268-3369issn
1476-5365pii
1704786journal_volume
35pub_type
临床试验,杂志文章abstract::High-dose carboplatin, etoposide and ifosfamide (CEI) is an active chemotherapy regimen (HDCT) in solid tumors and lymphomas. In patients with previous exposure to cisplatin, its nephrotoxicity is dose-limiting. To determine the implications of nephrotoxicity on hematological recovery and clinical outcome, we analyzed...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/sj.bmt.1700980
更新日期:1997-11-01 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::Auto-SCT and Allo-SCT are procedures conventionally associated with intensive transfusion support. This dependence has historically prevented SCT in individuals with religious or personal objections to transfusion. More recently, a growing body of literature supports the feasibility of 'bloodless transplants': SCT wit...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:10.1038/bmt.2008.5
更新日期:2008-05-01 00:00:00
abstract::With the increasing use and success of BMT, larger numbers of children survive transplantation. Still, cancer treatment in children causes damage to the endocrine glands, often inducing growth deficiency, pubertal delay and thyroid dysfunction. This paper will deal with some of the most common endocrine disorders rela...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:
更新日期:1998-04-01 00:00:00
abstract::Immune recovery was retrospectively analyzed in a cohort of 41 patients with acute leukemia, myelodysplastic syndrome and nonmalignant diseases, who received αβ T- and B-cell-depleted allografts from haploidentical family donors. Conditioning regimens consisted of fludarabine or clofarabine, thiotepa, melphalan and se...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2015.87
更新日期:2015-06-01 00:00:00
abstract::Total body irradiation (TBI) at bone marrow transplantation (BMT) is shown to cause salivary gland dysfunction in children. The aim of the investigation was to study the function of major salivary glands in long-term surviving children following treatment with TBI, using salivary gland scintigraphy (SGS). Thirteen pat...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702556
更新日期:2000-10-01 00:00:00
abstract::We describe the long-term outcomes of autologous hematopoietic cell transplantation (HCT) for 315 AML patients in first or second complete remission (CR). All patients were in continuous CR for ≥2 years after HCT. Patients were predominantly transplanted in CR1 (78%) and had good or intermediate cytogenetic risk disea...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,多中心研究
doi:10.1038/bmt.2010.115
更新日期:2011-03-01 00:00:00
abstract::Between December 1983 and November 1985 we treated 39 patients with chronic myeloid leukaemia by chemoradiotherapy and transplantation from HLA-identical sibling donors using bone marrow that had been depleted of T cells ex vivo with the rat monoclonal antibody Campath-1. Twenty-eight of the patients were in the chron...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1986-05-01 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::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::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::ABO incompatibility is not considered a contraindication for allogeneic haematopoietic stem cell transplantation (HSCT) despite its association with several immunohaematological complications. At present, there is no general agreement concerning the best methods to reduce these problems. To survey current practice rel...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,多中心研究
doi:10.1038/sj.bmt.1704579
更新日期:2004-08-01 00:00:00
abstract::Despite its potential to cause myocardial damage, high-dose CY in doses up to 200 mg/kg is an integral part of preparative regimens for BMT. Conventional tests, such as an electrocardiogram or echocardiogram, have lacked sensitivity in prediction of cardiotoxicity in this patient population. We prospectively compared ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1993-08-01 00:00:00
abstract::The medical staff caring for children with hematological or oncological diseases is often faced with delicate ethical and, at times, legal problems. Although many of these are common to other branches of pediatrics where patients are children lacking decision-making capacity, others, such as bone marrow transplantatio...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:
更新日期:1996-11-01 00:00:00
abstract::Fifteen patients with stage II-III multiple myeloma were intended to undergo two sequential cycles of myeloablative chemoradiotherapy with autologous bone marrow or blood stem cell transplantation after response to primary induction chemotherapy. BM grafts were used in 13 of the 15 first transplants whereas PBSC were ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1995-03-01 00:00:00
abstract::Patients undergoing bone marrow transplantation (BMT) have decreased levels of salivary Ig over long periods of time. However, shortly after transplantation, a transient rise of Ig concentration in their saliva gland is detected. In order to trace the origin of this Ig, seven BM donors were immunized with tetanus toxo...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1994-12-01 00:00:00
abstract::Two patients who underwent autologous bone marrow transplantation for recurrent non-Hodgkin's lymphoma relapsed at 46 and 28 days after the transplant. Both patients had an HLA-identical sibling and were treated with high-dose chemotherapy and allogeneic marrow transplantation. One patient is now 24 months after the a...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1989-01-01 00:00:00
abstract::Allogeneic hematopoietic cell transplantation (alloHCT) may be the only curative option for some older adults with hematologic malignancies, and its associated risks of significant morbidity and mortality warrant a clear, informed decision-making process. As older adults have not been transplanted routinely until rece...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:10.1038/bmt.2015.241
更新日期:2016-05-01 00:00:00
abstract::We retrospectively studied a series of 23 patients (median age 50 years, range 29-59 years) with multiple myeloma (MM), treated in first relapse by a sequential autologous-allogeneic tandem approach. Tandem transplantation (TT) consisted in high dose melphalan (HDT) and auto-SCT followed by an (allo-SCT) preceded by t...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2010.90
更新日期:2011-02-01 00:00:00
abstract::This retrospective analysis compared two regimens of fludarabine combined with i.v. BU 6.4 mg/kg (FB2) or BU 12.8 mg/kg (FB4) for allografting of AML in first CR. A total of 437 patients (median age: 50 years) were administered FB2 (n = 225, 51%) or FB4 (n = 212, 49%). Median follow-up time was 28 months. Use of FB2 r...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,多中心研究
doi:10.1038/bmt.2014.133
更新日期:2014-09-01 00:00:00
abstract::Data regarding the optimal approach for second allogeneic hematopoietic stem cell transplantation (HSCT) after graft failure (GF) in acquired severe aplastic anemia (SAA) are still limited and heterogeneous. We examined 24 patients who underwent second HLA-matched sibling donor (MSD) peripheral blood HSCT for GF. The ...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/s41409-018-0154-0
更新日期:2018-10-01 00:00:00
abstract::Successful stem cell mobilization and collection is possible in B-CLL after a favorable response to preceding chemotherapy, and also after treatment with the new nucleoside analogues fludarabine and cladribine. A poor response, on the other hand, seemed to predict a mobilization failure. CD34+ cell selection resulted ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1700730
更新日期:1997-04-01 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::Progressive multifocal leukoencephalopathy (PML) is a rare subacute demyelinating disorder of the central nervous system (CNS) caused by the DNA JC human polyomavirus. In immunocompromised hosts, PML is caused by reactivation of a latent infection rather than de novo primary exposure. PML in the setting of hematopoiet...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:10.1038/sj.bmt.1705548
更新日期:2007-01-01 00:00:00
abstract::Acute graft-versus-host disease (aGVHD) is a major cause of morbidity mortality in critically ill hematopoietic stem cell transplantation recipients. We assessed aGVHD trajectories in 191 allogeneic-HSCT recipients (age 42 (27-46)) admitted to our ICU between 2005 and 2015. aGVHD affected 130 (68%) patients (including...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-020-0857-x
更新日期:2020-10-01 00:00:00
abstract::Allogeneic SCT remains the only potential cure for patients with thalassemia. However, most BMT candidates lack a suitable family donor and require an unrelated donor (UD). We evaluated whether BMT using UDs in high-risk adult thalassemia patients can offer a probability of cure comparable to that reported employing a...
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.1705173
更新日期:2005-12-01 00:00:00
abstract::Candida kruzei-related systemic infections are increasing in frequency, particularly in patients receiving prophylaxis with antifungal triazoles. A Caucasian male with newly diagnosed acute myeloid leukaemia (AML M1) developed severe and persistent fever associated with a micropustular eruption scattered over the trun...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701539
更新日期:1999-01-01 00:00:00
abstract::We characterized CD34+ cells purified from bone marrow (BM), mobilized peripheral blood (PB) and cord blood (CB) and we tried to establish correlations between the cell cycle kinetics of the CD34+CD38- and CD34+CD38+ subpopulations, their sensitivity to SCF and IL-3 and their expression of receptors for these two CSFs...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702145
更新日期:2000-02-01 00:00:00
abstract::Allogeneic bone marrow transplantation (BMT) from an HLA-identical sibling donor is effective therapy for patients with bone marrow failure states and those with hematologic malignancies. However, only a minority of them will have an HLA-identical sibling donor; unrelated donors, matched or partially mismatched, have ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1700874
更新日期:1997-08-01 00:00:00
abstract::It remains unclear how specific innovations in allogeneic hematopoietic cell transplantation (HCT) attained over the past decades have contributed to improvement in transplantation outcomes. To address this question, we conducted a registry-based study of adults with acute myeloid leukemia in first or second complete ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/s41409-018-0301-7
更新日期:2019-04-01 00:00:00