Fludarabine, low-dose busulfan and antithymocyte globulin as conditioning for Fanconi anemia patients receiving bone marrow transplantation from HLA-compatible related donors.

Abstract:

:Allogeneic hematopoietic stem cell transplantation (SCT) from unaffected donors remains the only modality for the correction of hematological abnormalities in Fanconi anemia (FA) patients. We performed four HLA-matched related donor SCT using a novel irradiation and cyclophosphamide-free conditioning regimen. The protocol included fludarabine 150 mg/m(2), busulfan 4 mg/kg, and antithymocyte globulin 90 mg/kg. Graft-versus-host disease (GVHD) prophylaxis was cyclosporin A, MTX, and daclizumab. The engraftment and occurrence of full stable donor hemopoiesis was rapid in all cases with minimal short-term toxic complications. There were no infections or febrile episodes during the inpatient phase. Three patients developed acute GVHD grade I-II involving gut and skin and one patient progressed to extensive chronic GVHD. The preparative conditioning regimen is safe and associated with low organ toxicity and effective immunosupression for the stable engraftment in FA patients undergoing SCT with matched related donors.

journal_name

Bone Marrow Transplant

authors

Maschan AA,Trakhtman PE,Balashov DN,Shipicina IP,Skorobogatova EV,Skvortsova YV,Dyshlevaja ZM,Samochatova EV,Rumiantsev AG

doi

10.1038/sj.bmt.1704580

subject

Has Abstract

pub_date

2004-08-01 00:00:00

pages

305-7

issue

4

eissn

0268-3369

issn

1476-5365

pii

1704580

journal_volume

34

pub_type

杂志文章
  • Upfront stem cell transplantation for newly diagnosed multiple myeloma with del(17p) and t(4;14): a study from the CMWP-EBMT.

    abstract::We analyzed newly diagnosed multiple myeloma patients with del(17p) and/or t(4;14) undergoing either upfront single autologous (auto), tandem autologous (auto-auto) or tandem autologous/reduced-intensity allogeneic (auto-allo) stem cell transplantation. 623 patients underwent either auto (n = 446), auto-auto (n = 105)...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/s41409-020-01007-w

    authors: Gagelmann N,Eikema DJ,de Wreede LC,Rambaldi A,Iacobelli S,Koster L,Caillot D,Blaise D,Remémyi P,Bulabois CE,Passweg J,Leleu X,Zver S,Kobbe G,Ljungman P,Chevallier P,Ringhoffer M,Martin M,Salmenniemi U,Poiré X,Lenh

    更新日期:2021-01-01 00:00:00

  • Successful treatment of parvovirus B19 infection and red cell aplasia occurring after an allogeneic bone marrow transplant.

    abstract::Chronic parvovirus B19 infection in the immunocompromised host may cause severe anaemia secondary to failure of erythropoiesis. This has been previously documented in patients with the Acquired Immune Deficiency Syndrome (AIDS), congenital immunodeficiencies and in children with acute lymphoblastic leukaemia during ma...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Corbett TJ,Saw H,Popat U,MacMahon E,Cohen BJ,Knowles WA,Beard S,Prentice HG

    更新日期:1995-11-01 00:00:00

  • Low CD4 lymphocyte count in a patient with P. carinii pneumonia after autologous bone marrow transplantation.

    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:

    authors: Castagnola E,Dini G,Lanino E,Tasso L,Dallorso S,Garaventa A,Rossi GA,Giacchino R

    更新日期:1995-06-01 00:00:00

  • Long-term outcome of childhood aplastic anemia patients who underwent allogeneic hematopoietic SCT from an HLA-matched sibling donor in Japan.

    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

    authors: Kikuchi A,Yabe H,Kato K,Koh K,Inagaki J,Sasahara Y,Suzuki R,Yoshida N,Kudo K,Kobayashi R,Tabuchi K,Kawa K,Kojima S

    更新日期:2013-05-01 00:00:00

  • Human herpesvirus 6 is an important pathogen in infectious lung disease after allogeneic bone marrow transplantation.

    abstract::Two hundred and ten bronchoalveolar lavage (BAL) samples were obtained from 50 patients 10 days before and on defined days after allogeneic bone marrow transplantation (BMT). The samples were examined for human cytomegalovirus (HCMV) and human herpesvirus-6 (HHV-6) by polymerase chain reaction (PCR). Fifteen patients ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1702569

    authors: Buchbinder S,Elmaagacli AH,Schaefer UW,Roggendorf M

    更新日期:2000-09-01 00:00:00

  • Evaluation of chimerism after bone marrow transplantation with single locus minisatellite DNA probes.

    abstract::The origin of cells in almost all allogeneic donor-recipient pairs can be determined through the use of highly polymorphic minisatellite DNA probes. Single-locus probes were cloned from hypervariable fragments in a human DNA fingerprint detected with a multi-locus probe. While each probe is highly polymorphic and locu...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Harano H,Maruta A,Matsuzaki M,Kanamori H,Fukawa H,Noguchi T,Miyashita H,Ogawa K,Motomura S,Kodama F

    更新日期:1993-09-01 00:00:00

  • Effect of slow lymphocyte recovery and type of graft-versus-host disease prophylaxis on relapse after allogeneic bone marrow transplantation for acute myelogenous leukemia.

    abstract::Allogeneic BMT is potentially curative for patients with acute myelogenous leukemia (AML) in first remission. However, many patients relapse after transplantation. Various immunotherapeutic options have been attempted with variable success in preventing relapse. Early identification of patients at high risk for relaps...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1703262

    authors: Kumar S,Chen MG,Gastineau DA,Gertz MA,Inwards DJ,Lacy MQ,Tefferi A,Litzow MR

    更新日期:2001-11-01 00:00:00

  • Second EBMT Workshop on reduced intensity allogeneic hemopoietic stem cell transplants (RI-HSCT).

    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

    authors: Bacigalupo A

    更新日期:2002-02-01 00:00:00

  • High-dose therapy with autologous stem cell transplantation in patients with peripheral T cell lymphomas.

    abstract::Peripheral T cell lymphomas (PTCL) have a poorer prognosis after conventional treatment than do high-grade B cell lymphomas. The place for high-dose therapy (HDT) with autologous stem cell support in these patients is still not clear. Forty patients, 10 women and 30 men, median age 41.5 years (range 16-61) with PTCL w...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,多中心研究

    doi:10.1038/sj.bmt.1702867

    authors: Blystad AK,Enblad G,Kvaløy S,Berglund A,Delabie J,Holte H,Carlson K,Kvalheim G,Bengtsson M,Hagberg H

    更新日期:2001-04-01 00:00:00

  • Therapy decisions in leukemia.

    abstract::Complex factors determine how physicians select between alternative therapies. We conducted a survey of 352 leukemia experts to determine consensus regarding optimal treatment for leukemia and to identify factors correlated with therapy decisions. The study evaluated both general treatment policies and recommendations...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Atkinson KA,Horowitz MM,Sobocinski KA,Rimm AA,Bortin MM,Gale RP

    更新日期:1992-01-01 00:00:00

  • Single and double autotransplants for relapsing/refractory Hodgkin's disease: results of two consecutive trials.

    abstract::To evaluate a strategy of one cycle of dose-intensive chemotherapy for patients with Hodgkin's disease in sensitive relapse and two cycles for those with refractory disease, 122 patients received dose-intensive chemotherapy followed by autotransplant in two consecutive studies. Patients with refractory disease were of...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:10.1038/sj.bmt.1700682

    authors: Ahmed T,Lake DE,Beer M,Feldman EJ,Preti RA,Seiter K,Helson L,Mittelman A,Kancherla R,Ascensao J,Akhtar T,Cook P,Goldberg R,Coleman M

    更新日期:1997-03-01 00:00:00

  • GM-CSF-mobilized peripheral blood CD34+ cells differ from steady-state bone marrow CD34+ cells in adhesion molecule expression.

    abstract::To determine the effect of growth factor mobilization on the expression of adhesion molecules, we compared CD34+ progenitor cell (PC) populations from steady-state bone marrow (BM) with granulocyte-macrophage colony-stimulating factor (GM-CSF)-mobilized apheresis products (peripheral blood stem cell (PSC)) using flow ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1700814

    authors: Watanabe T,Dave B,Heimann DG,Lethaby E,Kessinger A,Talmadge JE

    更新日期:1997-06-01 00:00:00

  • Consolidation with a busulfan-containing regimen followed by stem cell transplantation in infants with poor prognosis stage 4 neuroblastoma.

    abstract::Although infants with stage 4 neuroblastoma (NB) usually have a good prognosis, metastatic relapses after 1 year of age and amplification of the N-myc oncogene are established poor prognostic factors. In order to improve the survival of patients with such high-risk factors, we performed consolidation with a busulfan (...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1702376

    authors: Valteau-Couanet D,Benhamou E,Vassal G,Stambouli F,Lapierre V,Couanet D,Lumbroso J,Hartmann O

    更新日期:2000-05-01 00:00:00

  • Umbilical cord blood banking in The Netherlands.

    abstract::Cord blood banks are being developed in the United States and Europe. In The Netherlands, the EuroCord Nederland Foundation (ECN) has been established to coordinate the development of a national cord blood bank for unrelated transplants. The aim of ECN is to collect at least 5000 transplants for patients who lack an H...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Jacobs HC,Falkenburg JH

    更新日期:1998-07-01 00:00:00

  • Standardized parenteral alanyl-glutamine dipeptide supplementation is not beneficial in autologous transplant patients: a randomized, double-blind, placebo controlled study.

    abstract::We conducted a controlled, double-blind study of parenteral glutamine supplementation in an unselected group of consecutive autologous transplant patients. Patients received 30 g of alanyl-glutamine dipeptide (Dipeptiven; Fresenius-Kabi, Bad Homburg, Germany) or glutamine-free amino acid solution i.v. from day +1 to d...

    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.1703759

    authors: Pytlík R,Benes P,Patorková M,Chocenská E,Gregora E,Procházka B,Kozák T

    更新日期:2002-12-01 00:00:00

  • Transient engraftment of T cell-depleted allogeneic bone marrow in mice improves survival rate following lethal irradiation.

    abstract::C3H/HeJ mice were exposed to 8 or 9 Gy total body irradiation prior to transplantation of 1-15 x 10(6) H-2 incompatible T cell-depleted bone marrow cells from C57BL/6 donors. Survival was greatly enhanced compared to irradiated controls, even at the lowest cell doses. Analysis of spleen cells 1-7 weeks post-transplant...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Lapidot T,Singer TS,Reisner Y

    更新日期:1988-03-01 00:00:00

  • Risk factors and prognosis of hepatic acute GvHD after allogeneic hematopoietic cell transplantation.

    abstract::Hepatic acute GvHD (aGvHD) is associated with high mortality owing to poor response to immunosuppressive therapy. The pathogenesis of hepatic aGvHD differs from that of other lesions, and specific risk factors related to pre-transplant liver conditions should be determined. We conducted a cohort study by using a Japan...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章,多中心研究

    doi:10.1038/bmt.2015.205

    authors: Arai Y,Kanda J,Nakasone H,Kondo T,Uchida N,Fukuda T,Ohashi K,Kaida K,Iwato K,Eto T,Kanda Y,Nakamae H,Nagamura-Inoue T,Morishima Y,Hirokawa M,Atsuta Y,Murata M,GVHD working group of the Japan Society for Hematopoietic Ce

    更新日期:2016-01-01 00:00:00

  • Pathology of the liver in mucopolysaccharidosis: light and electron microscopic assessment before and after bone marrow transplantation.

    abstract::Serial liver biopsies were obtained in 20 patients undergoing bone marrow transplantation (BMT) for mucopolysaccharidosis (MPS). The 13 patients with MPS I, one with MPS II, four with MPS III, and two with MPS VI underwent liver biopsy prior to and from 1 to 37 months after BMT. The amount of accumulated glycosaminogl...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Resnick JM,Krivit W,Snover DC,Kersey JH,Ramsay NK,Blazar BR,Whitley CB

    更新日期:1992-09-01 00:00:00

  • Practices for cytomegalovirus diagnosis, prophylaxis and treatment in allogeneic bone marrow transplant recipients: a report from the Working Party for Infectious Diseases of the EBMT.

    abstract::During the past few years major progress has been made in the diagnosis and therapy of CMV infection after allogeneic BMT. The aim of this survey was to investigate the use of diagnostic techniques, use of prophylaxis and the therapeutic strategies among members of the EBMT. Seventy centers from 20 countries responded...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Ljungman P,De Bock R,Cordonnier C,Einsele H,Engelhard D,Grundy J,Locasciulli A,Reusser P,Ribaud P

    更新日期:1993-10-01 00:00:00

  • Alefacept treatment for refractory chronic extensive GVHD.

    abstract::Alefacept (Amevive) is an immunosuppressive dimeric fusion protein that is used for psoriasis control. We recently showed its effect in acute steroid-resistant/dependent GVHD. In this study, we describe the effect of alefacept treatment on chronic extensive GVHD (cGVHD). Twelve patients were included in this study; of...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2008.324

    authors: Shapira MY,Abdul-Hai A,Resnick IB,Bitan M,Tsirigotis P,Aker M,Gesundheit B,Slavin S,Or R

    更新日期:2009-02-01 00:00:00

  • Are there roles for observational database studies and structured quantification of expert opinion to answer therapy controversies in transplants?

    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 quantificatio...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2008.447

    authors: Gale RP,Eapen M,Logan B,Zhang MJ,Lazarus HM

    更新日期:2009-03-01 00:00:00

  • Delayed infusion of normal donor cells after MHC-matched bone marrow transplantation provides an antileukemia reaction without graft-versus-host disease.

    abstract::When allogeneic BMT is used for the treatment of leukemia, depletion of T cells from the donor BM to avoid GVHD may be accompanied by persistence of host cells and post-transplant relapse. In this report, a murine model of MHC-compatible BMT was used to show that delayed infusion of immunocompetent donor cells early a...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Johnson BD,Drobyski WR,Truitt RL

    更新日期:1993-04-01 00:00:00

  • Skin explant culture as a model for cutaneous graft-versus-host disease in humans.

    abstract::An in vitro skin explant model for graft-versus-host disease (GVHD) in humans has been used to study the role of effector T cells in the histological pathogenesis of GVHD. In 11 of 12 experiments clear GVHD changes of grades II-IV were induced in HLA-mismatched skin explants cultured with allogeneic T cells sensitized...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Dickinson AM,Sviland L,Carey P,Reid MM,Hamilton PJ,Pearson AJ,Proctor SJ

    更新日期:1988-07-01 00:00:00

  • Predictive value of flow cytometric analyses of blast cells in assessing the phenotype of the leukemia colony-forming cell (L-CFC) population in acute myeloid leukemia.

    abstract::Acute myeloid leukemia (AML) blast cells (BC) express antigens that are commonly found on their normal counterparts. The leukemia colony-forming cell (L-CFC) subpopulation, identified by its ability to form leukemia colonies in vitro, is thought to be the stem cell population that produces BC. To ascertain the associa...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Howell AL,Stukel TA,Bloomfield CD,Ball ED

    更新日期:1992-09-01 00:00:00

  • Pharmacokinetics of CsA during the switch from continuous intravenous infusion to oral administration after allogeneic hematopoietic stem cell transplantation.

    abstract::We investigated the serial changes in the blood CsA concentration during the switch from continuous intravenous infusion to twice-daily oral administration in allogeneic hematopoietic stem cell transplant recipients (n=12). The microemulsion form of CsA, Neoral, was started at twice the last dose in intravenous infusi...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:10.1038/bmt.2009.316

    authors: Kimura S,Oshima K,Okuda S,Sato K,Sato M,Terasako K,Nakasone H,Kako S,Yamazaki R,Tanaka Y,Tanihara A,Higuchi T,Nishida J,Kanda Y

    更新日期:2010-06-01 00:00:00

  • The role of BMT in childhood histiocytoses.

    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...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2008.46

    authors: Caselli D,Aricò M,EBMT Paediatric Working Party.

    更新日期:2008-06-01 00:00:00

  • Open-label, single arm, multicenter phase II study of VIDL induction chemotherapy followed by upfront autologous stem cell transplantation in patients with advanced stage extranodal NK/T-cell lymphoma.

    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

    authors: Song GY,Yoon DH,Suh C,Moon JH,Baek DW,Kim JS,Lee GW,Yi JH,Park Y,Jung KS,Kim SJ,Yang DH,Kim WS

    更新日期:2020-12-04 00:00:00

  • Monthly prospective analysis of hematopoietic chimerism after allogeneic hematopoietic cell transplantation.

    abstract:SUMMARY:Hematopoietic chimerism (HpC) was assayed monthly using a sensitive, polymerase chain reaction (PCR) -based method in consecutive patients. Between January 1998 and April 2002, 181 patients underwent non-T cell depleted allogeneic hematopoietic cell transplantation (HCT). A total of 163 patients were evaluable ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1704147

    authors: Lee KH,Lee JH,Choi SJ,Lee JH,Kim S,Seol M,Lee YS,Kim WK,Kwon MR,Choi SJ,Park CJ,Chi HS,Lee JS

    更新日期:2003-08-01 00:00:00

  • Graft-versus-host disease in children receiving HLA-identical allogeneic bone marrow transplants with a low adjusted T lymphocyte dose.

    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:

    authors: Potter MN,Pamphilon DH,Cornish JM,Oakhill A

    更新日期:1991-11-01 00:00:00

  • Reduced dose of posttransplant cyclophosphamide in HLA-haploidentical peripheral blood stem cell transplantation.

    abstract::Posttransplant cyclophosphamide (PTCy:100 mg/kg) has been increasingly used in allogeneic hematopoietic stem cell transplantation, however, few studies compared different doses of PTCy. We conducted two consecutive prospective multicenter phase II studies to evaluate the safety and efficacy of 80 mg/kg of PTCy in 137 ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/s41409-020-01065-0

    authors: Sugita J,Kamimura T,Ishikawa T,Ota S,Eto T,Kuroha T,Miyazaki Y,Kumagai H,Matsuo K,Akashi K,Taniguchi S,Harada M,Teshima T

    更新日期:2020-09-24 00:00:00