Stem cell transplantation in children with infantile osteopetrosis is associated with a high incidence of VOD, which could be prevented with defibrotide.

Abstract:

:Malignant infantile osteopetrosis (MIOP) is a rare hereditary disorder of osteoclast function, which can be reversed by hematopoietic stem cell transplantation (SCT). We observed a high incidence of hepatic veno-occlusive disease (VOD) in transplanted patients and explored the prevention of this complication by using defibrotide (DF) as a prophylaxis. Twenty children with MIOP were consecutively transplanted in our center between 1996 and 2005. Eleven of these patients were transplanted between 1996 and 2001 and experienced an overall incidence of VOD of 63.6% (7/11). VOD was severe in three patients and one patient succumbed to VOD-related multi-organ failure. Owing to this very high incidence of VOD, DF prophylaxis was initiated in nine patients consecutively transplanted between 2001 and 2005. In this group, only one patient (11.1%) was diagnosed with moderate VOD. We report here a very high risk in patients with MIOP to develop VOD after transplantation. Prophylactic DF was implemented in our current transplant protocol and reduced the VOD rate significantly in this high-risk population.

journal_name

Bone Marrow Transplant

authors

Corbacioglu S,Hönig M,Lahr G,Stöhr S,Berry G,Friedrich W,Schulz AS

doi

10.1038/sj.bmt.1705485

subject

Has Abstract

pub_date

2006-10-01 00:00:00

pages

547-53

issue

8

eissn

0268-3369

issn

1476-5365

pii

1705485

journal_volume

38

pub_type

临床试验,杂志文章
  • Two cases of progressive multifocal leukoencephalopathy after allogeneic hematopoietic cell transplantation and a review of the literature.

    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

    authors: Kharfan-Dabaja MA,Ayala E,Greene J,Rojiani A,Murtagh FR,Anasetti C

    更新日期:2007-01-01 00:00:00

  • Respiratory syncytial virus infection in adult BMT recipients: effective therapy with short duration nebulised ribavirin.

    abstract::Respiratory syncytial virus (RSV), a common cause of both upper and lower respiratory tract infection (LRTI) in infants and children, is rarely described as an infective agent in adults. It has been reported in bone marrow transplant (BMT) recipients and patients with malignancy immunosuppressed by chemotherapy. Such ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701091

    authors: McColl MD,Corser RB,Bremner J,Chopra R

    更新日期:1998-02-01 00:00:00

  • Syngeneic peripheral blood stem cell transplantation with brief immunosuppression for severe aplastic anemia.

    abstract::We report the cases of two severe aplastic anemia (SAA) patients who were successfully treated with syngeneic peripheral blood stem cell transplantation (PBSCT) using immunosuppression without high-dose chemotherapy or irradiation for conditioning. A 21-year-old woman with SAA of 6 years duration had been transfused h...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1702150

    authors: Hwang WL,Yang Y,Chen GR,Tsai CS,Jour JH

    更新日期:2000-02-01 00:00:00

  • Analysis of remobilization success in patients undergoing autologous stem cell transplants who fail an initial mobilization: risk factors, cytokine use and cost.

    abstract::Inadequate stem cell mobilization is seen in approximately 25% of patients undergoing autotransplantation for hematologic malignancies. Remobilization strategies include chemotherapy/cytokine combinations or high-dose cytokines alone or in combination. From 1/1997 to 7/2002, we remobilized 86 patients who failed an in...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1704486

    authors: Boeve S,Strupeck J,Creech S,Stiff PJ

    更新日期:2004-05-01 00:00:00

  • SCT for severe autoimmune diseases: consensus guidelines of the European Society for Blood and Marrow Transplantation for immune monitoring and biobanking.

    abstract::Over the past 15 years, SCT has emerged as a promising treatment option for patients with severe autoimmune diseases (ADs). Mechanistic studies recently provided the proof-of-concept that restoration of immunological tolerance can be achieved by haematopoietic SCT in chronic autoimmunity through eradication of the pat...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审

    doi:10.1038/bmt.2014.251

    authors: Alexander T,Bondanza A,Muraro PA,Greco R,Saccardi R,Daikeler T,Kazmi M,Hawkey C,Simoes BP,Leblanc K,Fibbe WE,Moore J,Snarski E,Martin T,Hiepe F,Velardi A,Toubert A,Snowden JA,Farge D

    更新日期:2015-02-01 00:00:00

  • Delayed or delayed sequential bone marrow transplantation: relevance for acute graft-versus-host disease prevention after major H2 incompatible transplantation.

    abstract::During this study, BalB/C mice were used as recipients and C57 bl/6 mice as donors. Recipients were given 800 cGys of total body irradiation (TBI) on day 0. Transplantation was carried out as follows: group (1): TBI on day 0; group (2): TBI on day 0 and transplantation on day +1; group (3): TBI on day 0 and transplant...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1704877

    authors: Mabed M,Maroof S,Zalta K,El-Awadee M

    更新日期:2005-04-01 00:00:00

  • Improved outcome of treatment-resistant high-risk Langerhans cell histiocytosis after allogeneic stem cell transplantation with reduced-intensity conditioning.

    abstract::Children with multisystem Langerhans cell histiocytosis (LCH) and risk organ involvement who fail to respond to conventional chemotherapy have an extremely poor prognosis. Myeloablative stem cell transplantation (SCT) as a possible salvage approach for these patients has been associated with a high risk of transplant-...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1705015

    authors: Steiner M,Matthes-Martin S,Attarbaschi A,Minkov M,Grois N,Unger E,Holter W,Vormoor J,Wawer A,Ouachee M,Woessmann W,Gadner H

    更新日期:2005-08-01 00:00:00

  • Elevations of tumor necrosis factor receptor 1 at day 7 and acute graft-versus-host disease after allogeneic hematopoietic cell transplantation with nonmyeloablative conditioning.

    abstract::Acute GVHD has remained a significant cause of nonrelapse mortality after allogeneic hematopoietic cell transplantation (HCT) with nonmyeloablative conditioning. The role of TNF-alpha in the biology of acute GVHD after nonmyeloablative conditioning has not been studied thus far. Here, we measured TNF receptor 1 (TNFR1...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2009.360

    authors: Willems E,Humblet-Baron S,Dengis O,Seidel L,Beguin Y,Baron F

    更新日期:2010-09-01 00:00:00

  • Rapid and sustained hematopoietic reconstitution by peripheral blood stem cell infusion alone following high-dose chemotherapy.

    abstract::We utilized mobilized peripheral blood stem cells (PBSC) as sole support for hematologic reconstitution following high-dose chemotherapy in 52 patients with advanced solid tumors and lymphoma. PBSC were collected by large scale leukapheresis after mobilization with chemotherapy. Each apheresis product was analysed for...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Schwartzberg L,Birch R,Blanco R,Wittlin F,Muscato J,Tauer K,Hazelton B,West W

    更新日期:1993-05-01 00:00:00

  • Low T-cell chimerism is not followed by graft rejection after nonmyeloablative stem cell transplantation (NMSCT) with CD34-selected PBSC.

    abstract::We investigate the feasibility of CD34-selected peripheral blood stem cell (PBSC) transplantation followed by pre-emptive CD8-depleted donor lymphocyte infusions (DLI) after a minimal conditioning regimen. Six patients with advanced hematological malignancies ineligible for a conventional myeloablative transplant (n=5...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:10.1038/sj.bmt.1704220

    authors: Baron F,Baudoux E,Frère P,Tourqui S,Schaaf-Lafontaine N,Herens C,DePrijck B,Fillet G,Beguin Y

    更新日期:2003-10-01 00:00:00

  • High-dose chemotherapy with BEAC regimen and autologous bone marrow transplantation for intermediate grade and immunoblastic lymphoma: durable complete remissions, but a high rate of regimen-related toxicity.

    abstract::Forty-eight adult patients with recurrent or refractory intermediate grade or immunoblastic lymphoma received high-dose carmustine (BCNU), etoposide, Ara C and cyclophosphamide (BEAC), followed by autologous bone marrow transplantation (BMT). Median follow-up is 906 days (range 613-2067 days). The complete remission r...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: van Besien K,Tabocoff J,Rodriguez M,Andersson B,Mehra R,Przepiorka D,Dimopoulos M,Giralt S,Suki S,Khouri I

    更新日期:1995-04-01 00:00:00

  • Reduced-intensity rituximab-BEAM-CAMPATH allogeneic haematopoietic stem cell transplantation for follicular lymphoma is feasible and induces durable molecular remissions.

    abstract::The indolent non-Hodgkin's lymphomas are theoretically curable through allogeneic haematopoietic stem cell transplantation (allo-HSCT). The applicability of standard conditioning allo-HSCT is, however, restricted by its toxicity. Recently, reduced-intensity conditioning regimens have demonstrated efficacy with signifi...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1703898

    authors: Ho AY,Devereux S,Mufti GJ,Pagliuca A

    更新日期:2003-04-01 00:00:00

  • A comparison of serological, cellular and DNA-RFLP methods for HLA matching in the selection of related bone marrow donors.

    abstract::Serological, cellular and DNA-RFLP (restriction fragment length polymorphism) methods of determining HLA compatibility between 10 leukaemic patients and potential related bone marrow donors were systematically compared. DR beta/DQ alpha/DQ beta/DNA-RFLP typing of these families gave results in agreement with those obt...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Howell WM,Evans PR,Spellerberg MB,Wilson PJ,Smith JL

    更新日期:1989-01-01 00:00:00

  • Diffuse alveolar hemorrhage following autologous bone marrow infusion.

    abstract::Autologous BMT performed in a 57-year-old woman with relapsed large cell lymphoma was complicated by two consecutive episodes of diffuse alveolar hemorrhage (DAH). The second episode occurred immediately after infusion of autologous BM. DAH is an increasingly recognized complication of autologous BMT and carries a hig...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Corso S,Vukelja SJ,Wiener D,Baker WJ

    更新日期:1993-09-01 00:00:00

  • Quality of life and behavioral adjustment after pediatric bone marrow transplantation.

    abstract::The purpose of this study was two-fold: to describe the quality of life and behavioral adjustment of survivors of pediatric bone marrow transplantation (BMT) prior to and 6 months post-BMT; and, to identify correlates of survivors' quality of life and behavioral adjustment. Participants were 26 children and adolescent...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1702527

    authors: Barrera M,Boyd-Pringle LA,Sumbler K,Saunders F

    更新日期:2000-08-01 00:00:00

  • Growth hormone treatment impact on growth rate and final height of patients who received HSCT with TBI or/and cranial irradiation in childhood: a report from the French Leukaemia Long-Term Follow-Up Study (LEA).

    abstract::The literature contains a substantial amount of information about factors that adversely influence the linear growth in up to 85% of patients undergoing haematopoietic SCT (HSCT) with TBI and/or cranial irradiation (CI) for acute leukaemia (AL). By contrast, only a few studies have evaluated the impact of growth hormo...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2011.139

    authors: Isfan F,Kanold J,Merlin E,Contet A,Sirvent N,Rochette E,Poiree M,Terral D,Carla-Malpuech H,Reynaud R,Pereira B,Chastagner P,Simeoni MC,Auquier P,Michel G,Deméocq F

    更新日期:2012-05-01 00:00:00

  • Immunologic recovery after autologous blood stem cell transplantation in patients with AL-amyloidosis.

    abstract::We prospectively studied absolute lymphocyte (ALC) and monocyte counts (AMC), lymphocyte subsets and proliferative in vitro responses to mitogen and antigen in 12 patients with AL-amyloidosis (AL) undergoing autologous blood stem cell transplantation (SCT) with high-dose i.v. melphalan. Myeloid and lymphoid recovery (...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1703298

    authors: Akpek G,Lenz G,Lee SM,Sanchorawala V,Wright DG,Colarusso T,Waraska K,Lerner A,Vosburgh E,Skinner M,Comenzo RL

    更新日期:2001-12-01 00:00:00

  • Feasibility and timing of unrelated donor identification for patients with ALL.

    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:

    authors: Davies SM,Ramsay NK,Weisdorf DJ

    更新日期:1996-05-01 00:00:00

  • A case of EBV-associated lymphoproliferative disease following high-dose therapy and CD34-purified autologous peripheral blood progenitor cell transplantation.

    abstract::A fatal case of EBV-associated lymphoproliferative disorder arising after a CD34-selected autologous peripheral blood stem cell transplant is reported in a patient with multiple myeloma in first plateau phase. It is suggested that this is likely to be a consequence of the accessory cell depletion associated with the C...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701335

    authors: Peniket AJ,Perry AR,Williams CD,MacMillan A,Watts MJ,Isaacson PG,Goldstone AH,Linch DC

    更新日期:1998-08-01 00:00:00

  • Underlying coronary artery disease and successful bone marrow transplantation: a case report.

    abstract::The cardiovascular risk of concurrent coronary artery disease in patients undergoing bone marrow transplantation (BMT) is not well known. We present a case of a 32-year-old male Hodgkin's disease patient with prior chemotherapy and radiotherapy, who underwent autologous BMT. His underlying single vessel coronary arter...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Schechter D,Drakos P,Nagler A

    更新日期:1994-05-01 00:00:00

  • Successful treatment of veno-occlusive disease with recombinant tissue plasminogen activator in a patient requiring peritoneal dialysis.

    abstract::There have been encouraging reports of the use of recombinant tissue plasminogen activator (tPA) in established veno-occlusive disease (VOD). Haemodialysis has been considered a contraindication to this therapy in view of the potential haemostatic complications. We report a case of a woman who developed moderately sev...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Simpson DR,Browett PJ,Doak PB,Palmer SJ

    更新日期:1994-10-01 00:00:00

  • Early herpes zoster infection in adult patients with Hodgkin's disease undergoing autologous bone marrow transplant.

    abstract::The incidence of varicella-zoster-virus infection/reactivation in adult patients with Hodgkin's disease undergoing autologous bone marrow transplantation (BMT) at the University of Minnesota Hospital and Clinic was determined. Seven of 28 evaluable patients (25%) developed varicella-zoster infections in the first 150 ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Christiansen NP,Haake RJ,Hurd DD

    更新日期:1991-06-01 00:00:00

  • Tumor load in patients with follicular lymphoma post stem cell transplantation may correlate with clinical course.

    abstract::The purpose of this study was to evaluate if the tumor load, as determined by a real-time quantitative PCR (RQ-PCR) assay, correlated with the clinical course of follicular lymphoma patients after stem cell transplantation (SCT). Cryopreserved bone marrow and/or peripheral blood samples obtained at different time inte...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1704130

    authors: Chang CC,Bredeson C,Juckett M,Logan B,Keever-Taylor CA

    更新日期:2003-08-01 00:00:00

  • ABO-incompatible bone marrow transplantation: a GITMO survey of current practice in Italy and comparison with the literature.

    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

    authors: Raimondi R,Soli M,Lamparelli T,Bacigalupo A,Arcese W,Belloni M,Rodeghiero F,Gruppo Italiano Trapianto di Midolla Osseo.

    更新日期:2004-08-01 00:00:00

  • Cerebral toxoplasmosis - a late complication of allogeneic haematopoietic stem cell transplantation.

    abstract::Toxoplasma gondii infection reactivation predominantly occurs among patients after allogeneic haematopoietic stem cell transplantation. Mostly, reactivation occurs during first 3 months after transplant, especially when risk factors are present. We report a case of late cerebral toxoplasmosis reactivation, which was p...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1702075

    authors: Zver S,Cernelc P,Mlakar U,Pretnar J

    更新日期:1999-12-01 00:00:00

  • Pilot study of combined cryosupernatant and protein A immunoadsorption exchange in the treatment of grade 3-4 bone marrow transplant-associated thrombotic microangiopathy.

    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:

    authors: Zeigler ZR,Shadduck RK,Nath R,Andrews DF 3rd

    更新日期:1996-01-01 00:00:00

  • Double reinforcement with fludarabine/high-dose cytarabine enhances the impact of autologous stem cell transplantation in acute myeloid leukemia patients.

    abstract::Reinforced chemotherapy based on a double high-dose consolidation regimen could be a different way to enhance in vivo purging prior to autologous stem cell transplantation (auto-SCT) in acute myeloid leukemia (AML). We investigated the impact on outcome of auto-SCT after two different strategies of early intensificati...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:10.1038/sj.bmt.1703003

    authors: Visani G,Lemoli RM,Isidori A,Piccaluga PP,Martinelli G,Malagola M,Gugliotta L,Bonini A,Bonifazi F,Motta MR,Rizzi S,Castellani S,Tura S

    更新日期:2001-04-01 00:00:00

  • Histoincompatibilities in ABDR-matched unrelated donor recipient combinations.

    abstract::To get an insight into the degree of major histocompatibility mismatches in donor/recipient (D/R) combinations who were 'ABDR-matched' by serology for class I and by oligotyping for DR1-14 (low resolution typing), we performed additional HLA testing using a combination of molecular, biochemical and cellular techniques...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Rufer N,Tiercy JM,Breur-Vriesendorp B,Gauchat-Feiss D,Shi X,Slavcev A,Lardy N,Speiser D,Gratwohl A,Chapuis B

    更新日期:1995-11-01 00:00:00

  • Targeting mycophenolate mofetil for graft-versus-host disease prophylaxis after allogeneic blood stem cell transplantation.

    abstract::As low trough levels of mycophenolic acid (MPA) have been measured in recipients of allo-SCTs, we performed a pilot study targeting mycophenolate mofetil (MMF) doses according to the MPA area under the concentration (AUC) levels. Twenty-nine patients were transplanted from matched sibling (n=7) and unrelated donors (n...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2008.85

    authors: Haentzschel I,Freiberg-Richter J,Platzbecker U,Kiani A,Schetelig J,Illmer T,Ehninger G,Schleyer E,Bornhäuser M

    更新日期:2008-07-01 00:00:00

  • Successful treatment of severe hemorrhagic cystitis after hemopoietic cell transplantation by selective embolization of the vesical arteries.

    abstract::Hemorrhagic cystitis (HC) is a common and sometimes life-threatening complication of hemopoietic cell transplantation (HCT) occurring in 7-52% of transplant recipients. In this setting it is usually either related to the use of cyclophosphamide or to a viral infection (BK, JC viruses and adenovirus type 11). Treatment...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1703954

    authors: Giné E,Rovira M,Real I,Burrel M,Montaña J,Carreras E,Montserrat E

    更新日期:2003-05-01 00:00:00