Conditioning regimens in allo-SCT for thalassemia major.

Abstract:

:Allogeneic hematopoietic SCT remains the only treatment that can correct the hematological manifestations in patients with thalassemia major. Improving the clinical outcomes of high-risk, heavily transfused patients with liver fibrosis and inadequate iron chelation remains a challenge. Because of the relatively high probability of graft rejection and regimen-related toxicity in many patients receiving SCT for advanced thalassemia major, further development of new treatment regimens is warranted. This review addresses the reported clinical studies in patients with advanced thalassemia major and we have summarized our suggested conditioning approach to improve the outcome after SCT.

journal_name

Bone Marrow Transplant

authors

Mathews V,Savani BN

doi

10.1038/bmt.2013.216

subject

Has Abstract

pub_date

2014-05-01 00:00:00

pages

607-10

issue

5

eissn

0268-3369

issn

1476-5365

pii

bmt2013216

journal_volume

49

pub_type

杂志文章,评审
  • Complete heart block in association with graft-versus-host disease.

    abstract::An infant who received haploidentical BM for severe combined immunodeficiency (SCID) developed acute, reversible complete heart block in association with an exacerbation of GVHD. Respiratory distress and myocardial dysfunction were also seen with this and previous GVHD exacerbations. The patient had not received chemo...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701038

    authors: Gilman AL,Kooy NW,Atkins DL,Ballas Z,Rumelhart S,Holida M,Lee N,Goldman F

    更新日期:1998-01-01 00:00:00

  • Allogeneic stem cell transplantation in second rather than first complete remission in selected patients with good-risk acute myeloid leukemia.

    abstract::Through two consecutive trials, a policy that considered allogeneic stem cell transplantation (SCT) from a sibling donor in second rather than first complete remission (CR) in selected younger patients with acute myeloid leukemia (AML) with t(8;21)/inv(16) (core binding factor (CBF) group) or a normal karyotype (NN gr...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:10.1038/sj.bmt.1704884

    authors: de Labarthe A,Pautas C,Thomas X,de Botton S,Bordessoule D,Tilly H,de Revel T,Bastard C,Preudhomme C,Michallet M,Fenaux P,Bastie JN,Socié G,Cordonnier C,Dombret H,Acute Leukemia French Association.

    更新日期:2005-04-01 00:00:00

  • Hepatic veno-occlusive disease with severe capillary leakage after peripheral stem cell transplantation: treatment with recombinant plasminogen activator and C1-esterase inhibitor concentrate.

    abstract::Severe veno-occlusive disease (VOD), characterised by elevated serum bilirubin levels, is a known complication in the first 3 weeks after peripheral blood stem cell transplantation (PBSCT). Severe VOD is associated with capillary leakage and multiple organ dysfunction and leads to high mortality. We report a 17-year-o...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701211

    authors: Heying R,Nürnberger W,Spiekerkötter U,Göbel U

    更新日期:1998-05-01 00:00:00

  • Lymphocyte expansion after unrelated cord blood allogeneic stem cell transplantation in adults.

    abstract::Limited information is available regarding the incidence and features of lymphocyte expansions after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Large granular lymphocytes (LGL) expansions have been reported after bone marrow or peripheral blood, but not after unrelated cord blood (UCB) allo-HSCT, ...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:10.1038/bmt.2016.364

    authors: Le Bris Y,Guillaume T,Ménard A,Illiaquer M,Martin J,Malard S,Duquesne A,Peterlin P,Debord C,Robillard N,Eveillard M,Wuillème S,Delaunay J,Mohty M,Garnier A,Moreau P,Béné MC,Chevallier P

    更新日期:2017-06-01 00:00:00

  • 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 prot...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1704580

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

    更新日期:2004-08-01 00:00:00

  • Effects of incubation temperature and time after thawing on viability assessment of peripheral hematopoietic progenitor cells cryopreserved for transplantation.

    abstract::Three widely used viability assessments were compared: (1) membrane integrity of nucleated cells using trypan blue (TB) exclusion and a fluorometric membrane integrity assay (SYTO 13 and propidium iodide), (2) enumeration of viable CD34+ cells, and (3) clonogenic assay (granulocyte-macrophage colony-forming units, CFU...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1704247

    authors: Yang H,Acker JP,Cabuhat M,McGann LE

    更新日期:2003-11-01 00:00:00

  • Herpes simplex infection of the jejunum occurring in the early post-transplantation period.

    abstract::Reactivation of infections with herpes viruses is a frequent and major cause of morbidity after bone marrow transplantation. In this case report we stress that HSV infections of the colon and small intestine should be considered in the differential diagnosis of diarrhea and intestinal bleeding in the early post-transp...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1700995

    authors: Kingreen D,Nitsche A,Beyer J,Siegert W

    更新日期:1997-12-01 00:00:00

  • Growth and endocrine function in children with acute myeloid leukaemia after bone marrow transplantation using busulfan/cyclophosphamide.

    abstract::Longitudinal studies of growth and endocrine function of children with AML transplanted with BUCY are limited. We report a cohort of 23 children with AML transplanted (15 autologous and eight allogeneic) following a single chemotherapy protocol and surviving at least 2 years after BMT. Busulfan was given as a single d...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1702384

    authors: Afify Z,Shaw PJ,Clavano-Harding A,Cowell CT

    更新日期:2000-05-01 00:00:00

  • Phase I trial of histone deacetylase inhibitor panobinostat in addition to glucocorticoids for primary therapy of acute graft-versus-host disease.

    abstract::Glucocorticoids for primary therapy of acute GVHD have limited responses. A phase I/II trial tested 4 weeks of deacetylase inhibitor panobinostat started within 48 h of glucocorticoids (1 mg/kg/day prednisone or equivalent) as primary treatment for patients with either classic acute GVHD (n = 16) or acute GVHD overlap...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/s41409-018-0163-z

    authors: Perez L,Fernandez H,Horna P,Riches M,Locke F,Field T,Powers J,Sahakian E,Villagra A,Mishra A,Betts B,Kharfan-Dabaja M,Beato F,Ochoa-Bayona L,Pidala J,Anasetti C

    更新日期:2018-11-01 00:00:00

  • Long-term follow-up of metastatic renal cancer patients undergoing reduced-intensity allografting.

    abstract::SCT from an HLA-compatible sibling donor is an adoptive immunotherapy for cytokine-refractory, metastatic clear-cell renal cell cancer (RCC). However, the recent introduction of targeted therapy compounds has reduced the interest in this therapeutic strategy. We have reanalyzed our series with the aim to assess long-t...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:10.1038/bmt.2009.9

    authors: Bregni M,Bernardi M,Servida P,Pescarollo A,Crocchiolo R,Treppiedi E,Corradini P,Ciceri F,Peccatori J

    更新日期:2009-08-01 00:00:00

  • The role of FDG-PET imaging and involved field radiotherapy in relapsed or refractory diffuse large B-cell lymphoma.

    abstract::We examined the role of fluorodeoxyglucose-positron emission tomography (FDG-PET) and the addition of involved field radiotherapy (IFRT) as potential modifiers of salvage therapy. From January 2000 to June 2007, 83 patients with chemosensitive relapsed or primary refractory diffuse large B-cell lymphoma (DLBCL) underw...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2008.408

    authors: Hoppe BS,Moskowitz CH,Zhang Z,Maragulia JC,Rice RD,Reiner AS,Hamlin PA,Zelenetz AD,Yahalom J

    更新日期:2009-06-01 00:00:00

  • Lack of utility of chimerism studies obtained 2-3 months after myeloablative hematopoietic cell transplantation for ALL.

    abstract::Lineage-specific chimerism studies are commonly obtained at several time points after nonmyeloablative hematopoietic cell transplantation to assess the tempo and degree of engraftment, and to monitor graft rejection. For patients who receive myeloablative transplants, the value of frequent chimerism analyses using sen...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2008.155

    authors: Doney K,Loken M,Bryant E,Smith A,Appelbaum F

    更新日期:2008-08-01 00:00:00

  • High-dose cyclophosphamide, etoposide and carboplatin with autologous bone marrow support for metastatic breast cancer: long-term results.

    abstract::We report long-term results of high-dose cyclophosphamide, etoposide and carboplatin with ABMT in 20 patients with metastatic breast cancer. Median age of the group was 41 years, ECOG performance status = 0 in 18 patients and 1 in two patients. Twelve patients had received adjuvant chemotherapy. Predominant sites of m...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701173

    authors: Baker WJ,Vukelja SJ,Burrell LM,Lee N,Perry JJ

    更新日期:1998-04-01 00:00:00

  • Successful cord blood stem cell transplantation for congenital erythropoietic porphyria (Gunther's disease).

    abstract::Congenital erythropoietic porphyria (Gunther's disease, GD) is a rare autosomal recessive disease. It results from the deficiency of uroporphyrinogen III synthase, the fourth enzyme on the metabolic pathway of heme synthesis. GD leads to severe scarring of the face and hands as a result of photosensitivity and fragili...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Zix-Kieffer I,Langer B,Eyer D,Acar G,Racadot E,Schlaeder G,Oberlin F,Lutz P

    更新日期:1996-07-01 00:00:00

  • Use of cytosine arabinoside and total body irradiation as conditioning for allogeneic marrow transplantation in patients with acute lymphoblastic leukemia: a multicenter survey.

    abstract::We report the experience of 14 centers which have used the combination of cytosine arabinoside (ara-C; 24-36 g/m2) and total body irradiation (TBI) to prepare 213 patients with acute lymphoblastic leukemia (ALL) for allogeneic BMT. The overall 3-year disease-free survival (DFS) probability is 38% (95% CI: 31-45%); 75 ...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章,多中心研究

    doi:

    authors: Weyman C,Graham-Pole J,Emerson S,August C,Champlin R,Coccia P,Fay J,Harris R,Koch P,Johnson L

    更新日期:1993-01-01 00:00:00

  • Systemic candidiasis with candida vasculitis due to Candida kruzei in a patient with acute myeloid leukaemia.

    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

    authors: Gregory RK,Powles RL,Treleaven JG,Smith ML,Mortimer PS,Wotherspoon A,Riley U

    更新日期:1999-01-01 00:00:00

  • Secondary myelodysplastic syndrome following bone marrow transplantation: report of two cases.

    abstract::We report two cases of secondary myelodysplastic syndrome (SMDS) which followed successful treatment of a primary malignancy with high-dose chemotherapy supported by reinfusion of autologous stem cells. The SMDS was diagnosed 24 months and 40 months, respectively, following autografting. Both patients lived for 7 mont...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Chan T,Juneja S,Wolf M,Januszewicz E,Cooper I

    更新日期:1994-02-01 00:00:00

  • Blood and marrow transplantation during the emerging COVID-19 pandemic: the Seattle approach.

    abstract::On January 20, 2020, the first patient with coronavirus disease 2019 (COVID-19) in the United States of America was diagnosed in Washington state, which subsequently experienced rapidly increasing numbers of COVID-19 cases, hospitalizations, and deaths. This placed the Seattle Blood and Marrow Transplant Program at Fr...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审

    doi:10.1038/s41409-020-01068-x

    authors: Ueda Oshima M,Sandmaier BM,Petersdorf E,Flowers ME,Hill GR,Lee SJ,Appelbaum FR,Carpenter PA,Baker KS,Connelly-Smith L,McCool A,Elgar S,Pergam SA,Liu C,Stewart FM,Mielcarek M

    更新日期:2020-09-26 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

  • Treatment of acute graft-versus-host disease with PUVA (psoralen and ultraviolet irradiation): results of a pilot study.

    abstract::Acute graft-versus-host disease (aGVHD) is a frequent and major complication after allogeneic stem cell transplantation. For many years psoralen and ultraviolet (UV)-A light have been used in the treatment of chronic cutaneous graft-versus-host disease, but few patients have received PUVA therapy for aGVHD. We assesse...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701537

    authors: Wiesmann A,Weller A,Lischka G,Klingebiel T,Kanz L,Einsele H

    更新日期:1999-01-01 00:00:00

  • HLA-C mismatch is associated with inferior survival after unrelated donor non-myeloablative hematopoietic stem cell transplantation.

    abstract::HLA-C matching is an important determinant of outcome after myeloablative unrelated donor (URD) hematopoietic stem cell transplantation. However, its importance in non-myeloablative stem cell transplantation (NST) is not known. We report a retrospective analysis of 111 patients who underwent URD NST, of whom 78 were 1...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,多中心研究

    doi:10.1038/sj.bmt.1705315

    authors: Ho VT,Kim HT,Liney D,Milford E,Gribben J,Cutler C,Lee SJ,Antin JH,Soiffer RJ,Alyea EP

    更新日期:2006-05-01 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

  • Late cardiovascular morbidity and mortality following pediatric allogeneic hematopoietic cell transplantation.

    abstract::We analyzed late cardiovascular outcomes of 661 patients who survived at least 2 years from hematopoietic cell transplantation for childhood hematologic malignancy between 1995 and 2008. Center for International Blood and Marrow Transplant Research data was supplemented with surveys focused on cardiotoxicity and poten...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/s41409-018-0155-z

    authors: Duncan CN,Brazauskas R,Huang J,Shaw BE,Majhail NS,Savani BN,Flowers MED,Battiwalla M,Beebe K,Dietz AC,Dvorak CC,Giller R,Jacobsohn DA,Kletzel M,Martin PL,Nemecek ER,Nuechterlein B,Talano JA,Pulsipher MA,Baker KS

    更新日期:2018-10-01 00:00:00

  • Varicella zoster infection after bone marrow transplantation: incidence, risk factors and complications.

    abstract::The cellular immunoincompetence which follows bone marrow transplantation (BMT) allows both primary and reactivation infection with herpes viruses. We report the overall incidence and timing of varicella zoster virus (VZV) infections after BMT, including the clinical course, complications and associated clinical risk ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Han CS,Miller W,Haake R,Weisdorf D

    更新日期:1994-03-01 00:00:00

  • Outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome.

    abstract::HLA-identical sibling donor transplantation remains the treatment of choice for Wiskott-Aldrich Syndrome (WAS). Since 1990, utilization of alternative donor sources has increased significantly. We report the hematopoietic cell transplantation (HCT) outcomes of 47 patients with WAS treated at a single center since 1990...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2012.31

    authors: Shin CR,Kim MO,Li D,Bleesing JJ,Harris R,Mehta P,Jodele S,Jordan MB,Marsh RA,Davies SM,Filipovich AH

    更新日期:2012-11-01 00:00:00

  • Effect of pilocarpine hydrochloride on unstimulated whole saliva flow rate and composition in patients with chronic graft-versus-host disease (cGVHD).

    abstract::A double-blind, placebo-controlled study was conducted to evaluate the effect of orally administered pilocarpine on unstimulated whole-saliva flow and composition in 28 patients with chronic graft-versus-host disease (cGVHD). Thirteen patients were treated with pilocarpine of 20 mg/day orally for 7 days, and 15 patien...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1705621

    authors: Agha-Hosseini F,Mirzaii-Dizgah I,Ghavamzadeh L,Ghavamzadeh A,Tohidast-Acrad Z

    更新日期:2007-04-01 00:00:00

  • Predicting the severity of graft-versus-host disease from interleukin-10 levels after bone marrow transplantation.

    abstract::Acute graft-versus-host disease (GVHD) is the most important complication of allogeneic bone marrow transplantation. We investigated the possibility of predicting severe acute GVHD using plasma interleukin-10 levels in 31 patients who underwent allogeneic bone marrow transplantation. In patients with acute GVHD, the i...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1702010

    authors: Takatsuka H,Takemoto Y,Okamoto T,Fujimori Y,Tamura S,Wada H,Okada M,Yamada S,Kanamaru A,Kakishita E

    更新日期:1999-11-01 00:00:00

  • Unusual recurrence of chronic myelogenous leukemia following bone marrow transplantation.

    abstract::We report a patient who developed a bone and adjacent soft tissue malignancy 22 months after bone marrow transplantation (BMT) for Philadelphia chromosome positive chronic myelogenous leukemia (CML). Concomitant bone marrow was cytologically and cytogenetically normal. Cytogenetic study of tumoral tissue was unsuccess...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Millot F,Facon T,Kerckaert JP,Fenaux P,Lai JL,Parent M,Bauters F,Jouet JP

    更新日期:1991-05-01 00:00:00

  • A risk-adapted approach to acute GVHD treatment: are we there yet?

    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

    authors: Holtan SG,MacMillan ML

    更新日期:2016-02-01 00:00:00

  • Cord blood stem cell transplantation for Diamond-Blackfan anemia.

    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

    authors: Vettenranta K,Saarinen UM

    更新日期:1997-03-01 00:00:00