Allogeneic stem cell transplantation for Fanconi Anaemia. Severe Aplastic Anaemia Working Party of the EBMT and EUFAR. European Group for Blood and Marrow Transplantation.

Abstract:

:Fanconi anaemia is a hereditary disorder characterised by chromosomal breaks increased by cross-linking agents. Bone marrow transplantation is the treatment of choice when a HLA identical sibling donor has been identified. The use of low-dose cyclophosphamide with thoraco-abdominal irradiation for the conditioning regimen of FA patients has lead to a dramatic improvement of survival, with a long-term survival of 75% at our institution. However, if most patients are completely cured of their haematological disease, there is concern about an increased frequency of secondary tumours, mostly head and neck squamous cell carcinomas of poor prognosis. Results of BMT using alternative donors (HLA mismatched related and unrelated donors) have also improved during the last decade. A better selection of the donor via high-resolution techniques for class-II HLA matching, and more recently the use of T cell depleted grafts are probably the main explanations. Despite a short follow-up and the small number of patients analysed, transplants using HLA matched family cord blood give some promising results. On the other hand, first results with unrelated cord blood remind that this approach is clearly an experimental one that has to be evaluated through international registries and prospective studies. New approaches including autologous stem cell transplantations and gene therapy are currently explored.

journal_name

Bone Marrow Transplant

authors

Guardiola P,Socié G,Pasquini R,Dokal I,Ortega JJ,van Weel-Sipman M,Marsh J,Locatelli F,Souillet G,Cahn JY,Ljungman P,Miniero R,Shaw J,Vermylen C,Archimbaud E,Bekassy AN,Krivan G,Di Bartolomeo P,Bacigalupo A,Gluckman

subject

Has Abstract

pub_date

1998-04-01 00:00:00

pages

S24-7

eissn

0268-3369

issn

1476-5365

journal_volume

21 Suppl 2

pub_type

杂志文章,评审
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    abstract::Adjuvant transfusion of donor lymphocytes (aDLT) may reduce the risk of relapse after allogeneic stem cell transplantation in high-risk AML. We performed a retrospective analysis on the safety and efficacy of aDLT in a cohort of 46 patients. To be eligible for aDLT, patients had to be in CR for at least 120 days from ...

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    pub_type: 杂志文章

    doi:10.1038/bmt.2015.234

    authors: Jedlickova Z,Schmid C,Koenecke C,Hertenstein B,Baurmann H,Schwerdtfeger R,Tischer J,Kolb HJ,Schleuning M

    更新日期:2016-05-01 00:00:00

  • Challenges in the use of allogeneic hematopoietic SCT for ectodermal dysplasia with immune deficiency.

    abstract::Genetic mutations of proteins regulating nuclear factor of kappa-light polypeptide gene enhancer in B lymphocyte (NF-kappaB) activation result in heritable diseases of development and immunity. Hypomorphic, X-linked mutations in the IKBKG gene (NF-kappaB essential modulator (NEMO) protein), and hypermorphic, autosomal...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,多中心研究

    doi:10.1038/bmt.2008.308

    authors: Fish JD,Duerst RE,Gelfand EW,Orange JS,Bunin N

    更新日期:2009-02-01 00:00:00

  • Granulomatous pneumonitis following bone marrow transplantation.

    abstract::We describe the rare occurrence of a granulomatous pneumonitis seen in a patient following allogeneic bone marrow transplantation. Interestingly sarcoidosis was diagnosed in the marrow donor less than a year after donating his bone marrow. ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1703192

    authors: Sundar KM,Carveth HJ,Gosselin MV,Beatty PG,Colby TV,Hoidal JR

    更新日期:2001-09-01 00:00:00

  • Evidence of alloreactive T lymphocytes in fetal liver: implications for fetal hematopoietic stem cell transplantation.

    abstract::The use of hematopoietic stem cells for in utero transplantation to create permanent hematochimerism represents a new concept in fetal therapy, although this approach has provided heterogeneous results. In this paper we have undertaken molecular, phenotypic and functional studies aimed at identifying the presence of f...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1702108

    authors: Renda MC,Fecarotta E,Dieli F,Markling L,Westgren M,Damiani G,Jakil C,Picciotto F,Maggio A

    更新日期:2000-01-01 00:00:00

  • The role of bone marrow transplantation following nuclear accidents.

    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:

    authors: Gale RP

    更新日期:1987-06-01 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

  • Return of immunohematopoietic impairment a long time after murine syngeneic bone marrow transplantation.

    abstract::We have tested the immunologic status and hematologic parameters of mice 2 months (short-term survivors) or 18 months (long-term survivors) after lethal total body irradiation and syngeneic bone marrow transplantation (SBMT), and of normal mice of corresponding age. Long-term SBMT survivors showed significantly lowere...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Skórski T,Kawalec M,Ratajczak M,Szczylik C,Kawiak J

    更新日期:1990-11-01 00:00:00

  • Idarubicin to intensify the conditioning regimens of autologous bone marrow transplantation for patients with acute myeloid leukemia in first complete remission.

    abstract::In an effort to reduce the relapse rate after unpurged autologous bone marrow transplantation (ABMT) in patients with acute myeloid leukemia (AML) in first complete remission (CR1), the standard conditioning regimens (cyclophosphamide/busulphan and cyclophosphamide/TBI) were intensified by adding idarubicin. Seventeen...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:10.1038/sj.bmt.1701279

    authors: Jerjis S,Roovers E,Muus P,Schaap N,de Witte T

    更新日期:1998-07-01 00:00:00

  • Umbilical cord blood transplantation for non-malignant diseases.

    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 o...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审

    doi:10.1038/bmt.2009.290

    authors: Prasad VK,Kurtzberg J

    更新日期:2009-11-01 00:00:00

  • Infections after allogeneic hematopoietic stem cell transplantation with a nonmyeloablative conditioning regimen.

    abstract::Hematopoietic cell transplantation (HCT) following nonmyeloablative conditioning (NMSCT) may be associated with a reduced risk of infection compared to standard allogeneic HCT. We retrospectively analyzed incidence and risk factors of infection in 62 patients undergoing NMSCT with low-dose TBI +/- fludarabine and post...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1705255

    authors: Frère P,Baron F,Bonnet C,Hafraoui K,Pereira M,Willems E,Fillet G,Beguin Y

    更新日期:2006-02-01 00:00:00

  • The Japan Marrow Donor Program, 25 years of experience in achieving 20,000 bone marrow transplantations: organization structure, activity, and financial basis.

    abstract::The Japan Marrow Donor Program (JMDP), established in 1991, has continued to grow in its capacity to facilitate unrelated bone marrow (BMT) and peripheral blood stem cell transplantation (PBSCT) for the past 25 years in Japan. The current donor pool is 463,465 (as of 31 December 2016) and 20,237 transplants were perfo...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/s41409-017-0084-2

    authors: Saito H,Ito M,Kato S,Kodera Y,Okamoto S,Taniguchi S,Takanashi M,Kanamori H,Masaoka T,Takaku F,JMDP.

    更新日期:2018-05-01 00:00:00

  • Rapid and massive expansion of cord blood-derived cytokine-induced killer cells: an innovative proposal for the treatment of leukemia relapse after cord blood transplantation.

    abstract::We have used a standardized 21-day expansion protocol to produce cytokine-induced killer (CIK) cells starting from very small amounts of nucleated cells (approximately 15 x 10(6) cells) isolated from cord blood. Mononuclear cells are stimulated with anti CD3 (OKT3) and IFNgamma and then expanded with IL-2. Moreover, w...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1705503

    authors: Introna M,Franceschetti M,Ciocca A,Borleri G,Conti E,Golay J,Rambaldi A

    更新日期:2006-11-01 00:00:00

  • Intestinal thrombotic microangiopathy following reduced-intensity umbilical cord blood transplantation.

    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

    authors: Narimatsu H,Kami M,Hara S,Matsumura T,Miyakoshi S,Kusumi E,Kakugawa Y,Kishi Y,Murashige N,Yuji K,Masuoka K,Yoneyama A,Wake A,Morinaga S,Kanda Y,Taniguchi S

    更新日期:2005-09-01 00:00:00

  • Evaluation of optimal survival of primitive progenitor cells (LTC-IC) from PBPC apheresis products after overnight storage.

    abstract::Optimal overnight (ON) storage of PBPC aphereses is becoming an increasingly important issue and different options for storing PBPC products exist. The survival of primitive progenitor cells is of major interest, as recent data suggest that these progenitors are not only important for long-term engraftment but also co...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1702104

    authors: Petzer AL,Gunsilius E,Zech N,Clausen J,Hoflehner E,Nussbaumer W,Gastl G

    更新日期:2000-01-01 00:00:00

  • Rapid and automated processing of bone marrow grafts without Ficoll density gradient for transplantation of cryopreserved autologous or ABO-incompatible allogeneic bone marrow.

    abstract::The growing number of BMTs has increased interest in safe and standardized in vitro bone marrow processing techniques. We describe our experience with a rapid automated method for the isolation of mononuclear cells (MNC) from large volumes of bone marrow using a Fenwal CS-3000 cell separator without employing density ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Schanz U,Gmür J

    更新日期:1992-12-01 00:00:00

  • Hematopoietic stem cell transplantation for adults with acute promyelocytic leukemia in the ATRA era: a survey of the European Cooperative Group for Blood and Marrow Transplantation.

    abstract::We performed a survey of the European Cooperative Group for Blood and Marrow Transplantation to analyze the outcome of 625 acute promyelocytic leukemia (APL) patients transplanted with auto- or allogeneic-hematopoietic stem cell transplantation (autoHSCT, alloHSCT) after 1993, in first (CR1) or in second complete remi...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,多中心研究

    doi:10.1038/sj.bmt.1705620

    authors: Sanz MA,Labopin M,Gorin NC,de la Rubia J,Arcese W,Meloni G,Bacigalupo A,Alessandrino P,Carreras E,Iriondo A,Novitzky N,Jacobs P,Bandini G,Lo-Coco F,Frassoni F,Rocha V,Acute Leukemia Working Party (ALWP) of European Cooper

    更新日期:2007-04-01 00:00:00

  • High frequencies of cytotoxic T cell precursors against minor histocompatibility antigens after HLA-identical BMT: absence of correlation with GVHD.

    abstract::Limiting dilution analysis was used to quantify the frequency of cytotoxic T cell precursors (CTLp) against minor histocompatibility (H) antigens induced by HLA-identical BMT. The development of CTLp was monitored serially in ten patients developing either acute (n = 3), acute and chronic (n = 4) or no (n = 3) GVHD. I...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: de Bueger M,Bakker A,Bontkes H,van Rood JJ,Goulmy E

    更新日期:1993-05-01 00:00:00

  • Donor lymphocyte infusion for treatment of life-threatening respiratory syncytial virus infection following bone marrow transplantation.

    abstract::We describe two patients who developed respiratory syncytial virus (RSV) pneumonia after BMT. One died of RSV pneumonia after three courses of steroid pulse therapy. Surprisingly, RSV antigen was identified in the bronchoalveolar lavage fluid (BALF) obtained post mortem. Steroid pulse therapy might have suppressed ant...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1702559

    authors: Kishi Y,Kami M,Oki Y,Kazuyama Y,Kawabata M,Miyakoshi S,Morinaga S,Suzuki R,Mori S,Muto Y

    更新日期:2000-09-01 00:00:00

  • Phenotypic and functional characterization of peripheral blood and bone marrow natural killer cells prior to autologous transplantation.

    abstract::NK cells can exert potent anti-leukemia activity after either autologous or allogeneic BMT. However, in autologous blood or marrow transplant patients, NK cell number and/or function could be reduced, and also may vary according to the sampling site. In order to evaluate the hypothesis that blood or marrow grafts from...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Arbour S,Toupin S,Bélanger R,Gyger M,Hallé JP,Perreault C,Roy DC

    更新日期:1996-03-01 00:00:00

  • A dose escalation study of total body irradiation followed by high-dose etoposide and allogeneic blood stem cell transplantation for the treatment of advanced hematologic malignancies.

    abstract::Since approximately 30% of leukemia patients relapse after allogeneic BMT using total body irradiation (TBI)-based preparative regimens, treatment intensity may be suboptimal. The killing of leukemia cells is proportional to the radiation absorbed dose. We studied the feasibility and toxicity of escalating the doses o...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,多中心研究

    doi:10.1038/sj.bmt.1702230

    authors: Sobecks RM,Daugherty CK,Hallahan DE,Laport GF,Wagner ND,Larson RA

    更新日期:2000-04-01 00:00:00

  • Elevated lactate dehydrogenase is an adverse predictor of outcome in HLA-matched sibling bone marrow transplant for acute myelogenous leukemia.

    abstract::Prognostic factors for survival following allogeneic BMT for AML include age, disease status and cytogenetic risk classification. Lactate dehydrogenase (LDH) levels have not been studied as a potential risk factor. We reviewed our experience with BMT for AML and included LDH at the time of admission in an analysis of ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1705811

    authors: Kalaycio M,Rybicki L,Pohlman B,Dean R,Sweetenham J,Andresen S,Sobecks R,Sekeres MA,Advani A,Brown S,Bolwell B

    更新日期:2007-10-01 00:00:00

  • Predicting non-relapse mortality following allogeneic hematopoietic cell transplantation during first remission of acute myeloid leukemia.

    abstract::The aim of this study was to develop a comprehensive system for predicting non-relapse mortality after allogeneic hematopoietic cell transplantation (HCT) during first complete remission (CR) of acute myeloid leukemia (AML). After dividing 2344 eligible patients randomly into a training set and a validation set, we fi...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/s41409-020-01032-9

    authors: Yanada M,Konuma T,Mizuno S,Saburi M,Shinohara A,Tanaka M,Marumo A,Sawa M,Uchida N,Ozawa Y,Onizuka M,Yoshioka S,Nakamae H,Kondo T,Kimura T,Kanda J,Fukuda T,Atsuta Y,Nakasone H,Yano S

    更新日期:2020-08-14 00:00:00

  • Low incidence of molecular evidence for tumour in PBPC harvests from patients with high risk Ewing tumours.

    abstract::Reverse transcriptase polymerase chain reaction (RT-PCR) was applied to evaluate the frequency of tumour cells in PBPC products from 15 high risk Ewing tumour (ET) patients who were treated according to EICESS 92 with high-dose chemotherapy (HDC) and stem cell rescue. Initial tumour cell contamination of the bone marr...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701924

    authors: Fischmeister G,Zoubek A,Jugovic D,Witt V,Ladenstein R,Fritsch G,Höcker P,Gadner H,Kovar H

    更新日期:1999-08-01 00:00:00

  • rhG-CSF does not affect the phenotype of adult donor peripheral blood NK cells.

    abstract::Considerable evidence in preclinical models as well as in human transplantation now suggests that donor-derived natural killer (NK) cells can contribute to alloimmune recognition of recipient residual tumour cells. This makes the NK cell population an attractive target for in vitro or in vivo manipulations, in order t...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1704711

    authors: Lassailly F,Sielleur I,Blaise D,Chabannon C

    更新日期:2005-01-01 00:00:00

  • Headache, circumoral paresthesia, and facial flushing associated with high-dose carmustine infusion.

    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

    authors: Woo MH,Ippoliti C,Bruton J,Mehra R,Champlin R,Przepiorka D

    更新日期:1997-04-01 00:00:00

  • Engraftment syndrome: a common cause for rash and fever following autologous hematopoietic stem cell transplantation for multiple sclerosis.

    abstract::Autologous hematopoietic stem cell transplantation (HSCT) is currently being evaluated as a therapy for patients with progressive multiple sclerosis (MS) at risk of debilitating neurological impairment. While preliminary results from a few studies have been reported, little is known about toxicities or outcome of HSCT...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1703324

    authors: Oyama Y,Cohen B,Traynor A,Brush M,Rodriguez J,Burt RK

    更新日期:2002-01-01 00:00:00

  • Transplantation of allogeneic CD34-selected stem cells after fludarabine-based conditioning regimen for children with mucopolysaccharidosis 1H (M. Hurler).

    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 fa...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:10.1038/sj.bmt.1704786

    authors: 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 KW

    更新日期:2005-02-01 00:00:00

  • The evolution of clinical peripheral blood stem cell transplantation.

    abstract::Our growing physiological understanding of hematopoietic progenitor cells has led to the clinical use of circulating progenitor cells, including stem cells, for either reconstitution of hematopoietic function, up to the transduction of functional genes into a self-renewing cell system. In the following, an attempt has...

    journal_title:Bone marrow transplantation

    pub_type: 历史文章,杂志文章

    doi:

    authors: Körbling M,Fliedner TM

    更新日期:1996-05-01 00:00:00

  • Major salivary gland damage in allogeneic hematopoietic progenitor cell transplantation assessed by scintigraphic methods.

    abstract::Salivary gland dysfunction is a common sequela of hematopoietic progenitor cell transplantation (HPCT). The investigation of major salivary gland dysfunction with sodium pertechnetate scintigraphy is a non-invasive method that provides images of the parotid and submandibular glands. In this prospective trial, 20 HPCT ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1705351

    authors: Coracin FL,Pizzigatti Correa ME,Camargo EE,Peterson DE,de Oliveira Santos A,Vigorito AC,Borba Oliveira G,de Brito Eid KA,Zulli R,De Souza CA

    更新日期:2006-05-01 00:00:00

  • Toxoplasma retinitis/encephalitis 9 months after allogeneic bone marrow transplantation.

    abstract::In a 34-year-old patient toxoplasma retinitis/encephalitis developed 9 months after bone marrow transplantation. The BMT was complicated with a serious GVHD. Although she initially responded well to antibiotic therapy she died 2 years after BMT due to progressive infection. ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701128

    authors: Brinkman K,Debast S,Sauerwein R,Ooyman F,Hiel J,Raemaekers J

    更新日期:1998-03-01 00:00:00