Umbilical cord blood stem cell transplantation from unrelated HLA-matched donor in an infant with severe congenital neutropenia.

Abstract:

:We report here a 6-month-old boy with severe congenital neutropenia (SCN) successfully treated by cord blood stem cell transplantation (CBSCT) from an unrelated donor. He had recurrent life-threatening respiratory infection due to severe neutropenia that was refractory to recombinant human granulocyte colony-stimulating factor (rhG-CSF). Because he had no HLA-matched sibling and no time to wait for unrelated donor, he received HLA-matched unrelated CBSCT as determined by DNA typing. A total of 6.4 x 10(7) CB nucleated cells/kg was infused after conditioning with busulfan/horse antihuman thymocyte serum/cyclophosphamide. No GVHD developed under the treatment with cyclosporin A and methyl prednisolone. The neutrophil count reached 0.5 x 10(9)/l on day 14, reticulocyte 1% on day 13 and platelet count over 50 x 10(9)/l on day 31. We conclude that unrelated CBSCT can be an indication for some cases of SCN, who have recurrent life-threatening infections and are refractory to rhG-CSF, and have no HLA-matched sibling.

journal_name

Bone Marrow Transplant

authors

Mino E,Kobayashi R,Yoshida M,Suzuki Y,Yamada M,Kobayashi K

doi

10.1038/sj.bmt.1704439

subject

Has Abstract

pub_date

2004-05-01 00:00:00

pages

969-71

issue

9

eissn

0268-3369

issn

1476-5365

pii

1704439

journal_volume

33

pub_type

杂志文章
  • Etiology, clinical features and outcomes of pre-engraftment and post-engraftment bloodstream infection in hematopoietic SCT recipients.

    abstract::We conducted an observational study to assess the etiology, clinical features and outcomes of bloodstream infection (BSI) in 172 hematopoietic SCT (HCST) recipients. One hundred episodes of BSI in the pre-engraftment period (early onset) were compared with 89 episodes in the post-engraftment phase (late onset). More p...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2014.37

    authors: Gudiol C,Garcia-Vidal C,Arnan M,Sánchez-Ortega I,Patiño B,Duarte R,Carratalà J

    更新日期:2014-06-01 00:00:00

  • GI complications in pediatric patients post-BMT.

    abstract::This retrospective study comprehensively examined hepatic and gastrointestinal complications post-bone marrow transplant (BMT) in a heterogeneous group of 132 pediatric patients that underwent 142 transplants. Hyperbilirubinemia occurred in 28% of this population with clinically evident jaundice in 16%. Acute graft-ve...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1705004

    authors: Barker CC,Anderson RA,Sauve RS,Butzner JD

    更新日期:2005-07-01 00:00:00

  • Dapsone for Pneumocystis carinii prophylaxis in children undergoing bone marrow transplantation.

    abstract::Children who undergo bone marrow transplantation (BMT) are at risk for Pneumocystis carinii pneumonia (PCP). Prophylaxis using trimethoprim/sulfamethoxazole (TMP/SMX) is highly effective but the incidence of adverse drug reactions is significant. We retrospectively reviewed 33 pediatric BMT (25 allogeneic and eight au...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审

    doi:10.1038/sj.bmt.1700978

    authors: Maltezou HC,Petropoulos D,Choroszy M,Gardner M,Mantzouranis EC,Rolston KV,Chan KW

    更新日期:1997-11-01 00:00:00

  • High probability of long-term survival in 2-year survivors of autologous hematopoietic cell transplantation for AML in first or second CR.

    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

    authors: Majhail NS,Bajorunaite R,Lazarus HM,Wang Z,Klein JP,Zhang MJ,Rizzo JD

    更新日期:2011-03-01 00:00:00

  • Pharmacodynamics of tandem high-dose melphalan with peripheral blood stem cell transplantation in children with neuroblastoma and medulloblastoma.

    abstract::Repeated high-dose (HD) chemotherapy with peripheral blood stem cell (PBSC) transplantation is a new modality aimed at increasing both the dose and its intensity in the treatment of chemosensitive tumours. The aim of this study was to evaluate the tolerance, pharmacokinetics (PK) and pharmacodynamics (PD) of HD single...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:10.1038/sj.bmt.1702806

    authors: Vassal G,Tranchand B,Valteau-Couanet D,Mahé C,Couanet D,Schoeppfer C,Grill J,Kalifa C,Hill C,Ardiet C,Hartmann O

    更新日期:2001-03-01 00:00:00

  • Enteral budesonide in treatment for mild and moderate gastrointestinal chronic GVHD.

    abstract::Budesonide (BUD) is a steroid with a low bioavailability, which has been used for the treatment of oral manifestations of chronic GVHD (cGVHD). We retrospectively evaluated the efficacy of BUD in the treatment of gastrointestinal cGVHD. Thirteen patients (median age 47 years) receiving BUD for the treatment of cGVHD a...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2008.209

    authors: Andree H,Hilgendorf I,Leithaeuser M,Junghanss C,Holzhueter S,Loddenkemper C,Steiner B,Freund M,Wolff D

    更新日期:2008-10-01 00:00:00

  • Combined foscarnet -ganciclovir treatment for cytomegalovirus infections after allogeneic hemopoietic stem cell transplantation (Hsct).

    abstract::Thirty two allogeneic bone marrow transplant (BMT) recipients, aged 16-55 (median 35), with CMV antigenemia (= > 5 positive cells) developing at a median interval from BMT of 49 days, were given combined treatment with foscarnet and ganciclovir for 15 days. Maintenance was given with foscarnet and ganciclovir on alter...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Bacigalupo A,Bregante S,Tedone E,Isaza A,Van Lint MT,Moro F,Trespi G,Occhini D,Gualandi F,Lamparelli T,Marmont AM

    更新日期:1996-11-01 00:00:00

  • In vitro and in vivo depletion of T cells.

    abstract::Transplant related mortality and relapse after bmt have a negative influence on the outcome of patients transplanted for acute leukaemia in first remission. Transplant related mortality includes graft-versus-host disease, infections and graft failure. To prevent gvhd and associated infections without increased graft r...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Arnold R,Bunjes D,Wiesneth M,Hertenstein B,Theobald M,Heimpel H,Hale G,Waldmann H

    更新日期:1993-01-01 00:00:00

  • Multiple cycles of dose-intensive chemotherapy with repeated stem cell support as induction treatment in metastatic breast cancer: a feasibility study.

    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

    authors: García-Rayo S,Pérez-Calvo J,Martín-Algarra S,Martínez-Monge R,Fernández-Hidalgo O,Subirá L,Martínez-Aguillo M,Rebollo J,Azinovic I,Brugarolas A

    更新日期:2001-08-01 00:00:00

  • Pulmonary toxicity associated with high dose chemotherapy in the treatment of solid tumors with autologous marrow transplant: an analysis of four chemotherapy regimens.

    abstract::We retrospectively reviewed the pulmonary toxicity of six high dose chemotherapy protocols using four chemotherapy regimens in the treatment of solid tumors. All protocols used either high dose cyclophosphamide or ifosfamide in combination with one to three additional chemotherapeutic agents. In each protocol autologo...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Seiden MV,Elias A,Ayash L,Hunt M,Eder JP,Schnipper LE,Frei E 3rd,Antman KH

    更新日期:1992-07-01 00:00:00

  • Admission of bone marrow transplant recipients to the intensive care unit: outcome, survival and prognostic factors.

    abstract::The role of ICU support in BMT patients is controversial. In an era of constrained resources, the use of prognostic factors predicting outcome may be helpful in identifying patients who are most likely (or unlikely) to benefit from this intervention. We attempted to define the survival of patients admitted to ICU foll...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701158

    authors: Jackson SR,Tweeddale MG,Barnett MJ,Spinelli JJ,Sutherland HJ,Reece DE,Klingemann HG,Nantel SH,Fung HC,Toze CL,Phillips GL,Shepherd JD

    更新日期:1998-04-01 00:00:00

  • Single dose of filgrastim (rhG-CSF) increases the number of hematopoietic progenitors in the peripheral blood of adult volunteers.

    abstract::Hematopoietic progenitor cell levels were monitored in the peripheral blood of ten healthy adults receiving a single dose of recombinant human granulocyte colony-stimulating factor (rhG-CSF). The objective was to determine the time and number of progenitor cells released into the peripheral blood, induced by a single ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Schwinger W,Mache C,Urban C,Beaufort F,Töglhofer W

    更新日期:1993-06-01 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

  • 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

  • Serious acute or chronic graft-versus-host disease after hematopoietic cell transplantation: a comparison of myeloablative and nonmyeloablative conditioning regimens.

    abstract::We previously reported a 25% incidence of serious graft-versus-host disease (GVHD) (that is, acute or chronic GVHD that caused death, lengthy hospitalization or disability, or resulted in recurrent major infections) among 171 hematopoietic cell transplantation (HCT) recipients after nonmyeloablative (NMA) regimen. Her...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1705987

    authors: Sala-Torra O,Martin PJ,Storer B,Traina F,Sorror ML,Storb R,Sandmaier BM,Flowers ME

    更新日期:2008-05-01 00:00:00

  • 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 ...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:10.1038/sj.bmt.1705485

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

    更新日期:2006-10-01 00:00:00

  • Reduced-intensity allogeneic stem cell transplantation for patients aged 50 years or older with B-cell ALL in remission: a retrospective study by the Adult ALL Working Group of the Japan Society for Hematopoietic Cell Transplantation.

    abstract::We retrospectively assessed the outcome and pretransplantation predictors of the outcome in 118 patients aged ≥ 50 years who received fludarabine-containing reduced-intensity allo-SCT (RIST) for B-cell ALL in the first or second CR. Eighty patients received transplants from unrelated donors. Seventy-eight patients wer...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2013.140

    authors: Kanamori H,Mizuta S,Kako S,Kato H,Nishiwaki S,Imai K,Shigematsu A,Nakamae H,Tanaka M,Ikegame K,Yujiri T,Fukuda T,Minagawa K,Eto T,Nagamura-Inoue T,Morishima Y,Suzuki R,Sakamaki H,Tanaka J

    更新日期:2013-11-01 00:00:00

  • Allogeneic hematopoietic stem cell transplantation for poor-risk CLL: dissecting immune-modulating strategies for disease eradication and treatment of relapse.

    abstract::To elucidate factors contributing to the effectiveness of allogeneic hematopoietic stem cell transplantation (alloHCT) in high-risk CLL, immune interventions, GvHD and clinical outcome of 77 consecutive patients allografted for CLL were analyzed. Immune modulation (immunosuppression tapering, rituximab-augmented donor...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2015.150

    authors: Hahn M,Böttcher S,Dietrich S,Hegenbart U,Rieger M,Stadtherr P,Bondong A,Schulz R,Ritgen M,Schmitt T,Tran TH,Görner M,Herth I,Luft T,Schönland S,Witzens-Harig M,Zenz T,Kneba M,Ho AD,Dreger P

    更新日期:2015-10-01 00:00:00

  • Disseminated fungal infection and early onset microangiopathy after allogeneic bone marrow transplantation.

    abstract::We report a patient who developed early onset microangiopathic hemolytic anemia (MAHA) and thrombocytopenia after allogeneic bone marrow transplantation (BMT). The clinical features and laboratory findings were not consistent with cyclosporin toxicity, graft-versus-host disease or cytomegalovirus infection as causativ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Grigg A,Clouston D

    更新日期:1995-05-01 00:00:00

  • Intermediate-dose busulfan and cyclophosphamide as a conditioning regimen for bone marrow transplantation in a case of Fanconi anemia in myelodysplastic transformation.

    abstract::We report an 11-year old female with myelodysplastic (refractory anemia with excess of blasts) presentation of Fanconi anemia. After failure of initial chemotherapy with low doses of 6-mercaptopurine and prednisolone she underwent allogeneic bone marrow transplantation (BMT) from her HLA-matched sibling. Busulfan 8 mg...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1700662

    authors: Maschan AA,Kryzanovskii OI,Yourlova MI,Skorobogatova EV,Pashanov ED,Potapova YE,Timonova LA,Bogatcheva NY,Samochatova EV,Roumjantzev AG

    更新日期:1997-02-01 00:00:00

  • Turkish Transplant Registry: a comparative analysis of national activity with the EBMT European Activity Survey.

    abstract::SCT is a curative approach using chemo-, radio- and immunotherapy for malignant and non-malignant hematological disorders. The European Group for Blood and Marrow Transplantation (EBMT) has been collecting yearly data on a survey basis since 1990. The variables within the survey are limited to detailed indications, nu...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审

    doi:10.1038/bmt.2008.144

    authors: Arat M,Arpaci F,Ertem M,Gürman G,Turkish Transplant Registry.

    更新日期:2008-08-01 00:00:00

  • Serum levels of IL-7 in bone marrow transplant recipients: relationship to clinical characteristics and lymphocyte count.

    abstract::IL-7 is produced by stromal cells and is the major lympho- and thymopoietic cytokine. IL-7 induces proliferation and differentiation of immature thymocytes, and protects thymocytes from apoptosis by induction of bcl-2 expression. The regulation of IL-7 production is poorly characterized, although down-regulation by tr...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701655

    authors: Bolotin E,Annett G,Parkman R,Weinberg K

    更新日期:1999-04-01 00:00:00

  • Patient education in allogeneic hematopoietic cell transplant: what patients wish they had known about quality of life.

    abstract::Quality of life (QOL) is increasingly recognized as an important clinical outcome of hematopoietic cell transplantation (HCT), but patient education is often overlooked. The aim of the current qualitative study was to examine education regarding post-HCT QOL from the patient's perspective. Allogeneic HCT recipients pa...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2013.158

    authors: Jim HS,Quinn GP,Gwede CK,Cases MG,Barata A,Cessna J,Christie J,Gonzalez L,Koskan A,Pidala J

    更新日期:2014-02-01 00:00:00

  • Liver disease during the first post-transplant year in bone marrow transplantation recipients: retrospective study.

    abstract::Liver dysfunction is a common problem in BMT recipients and it is important to determine the etiology in order to institute appropriate therapy. The purpose of this study was to evaluate the possible causes of liver dysfunction during the first post-transplant year in BMT recipients and to identify a possible relation...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1702453

    authors: Kim BK,Chung KW,Sun HS,Suh JG,Min WS,Kang CS,Sim SI,Shin WS,Kim CC

    更新日期:2000-07-01 00:00:00

  • Outcome of 67 patients with solid tumors relapsed after high-dose chemotherapy and peripheral blood stem cell transplantation.

    abstract::We retrospectively analyzed the outcome of 67 patients with breast (n=24), ovarian (n=11) or testicular cancer (n=32) treated for relapse after high-dose chemotherapy (HDC) and peripheral blood stem cell (PBSC) transplantation. Treatment, survival and toxicity were analyzed. Patients with breast, ovarian or testicular...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1704047

    authors: Bojko P,Akca A,Seeber S

    更新日期:2003-06-01 00:00:00

  • The role of high-dose chemotherapy with autologous bone marrow transplantation in the treatment of breast cancer.

    abstract::Autologous bone marrow reinfusion rapidly repopulates severely damaged bone marrow thus shortening the period of myelosuppression following high-dose chemotherapy programs. This strategy has been successfully employed in several hematologic malignancies such as acute leukemia, Hodgkin's disease, non-Hodgkin's lymphoma...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审

    doi:

    authors: Hortobagyi GN

    更新日期:1988-11-01 00:00:00

  • Bone marrow graft as a source of allogeneic hematopoietic stem cells in patients undergoing a reduced intensity conditioning regimen.

    abstract::In an attempt to reduce the incidence of chronic GVHD (cGVHD) after reduced-intensity conditioning (RIC), we used BM instead of PBSC and added melphalan 100 mg/m(2) to the classical association of fludarabine, 30 mg/m(2)/day for 3 days and TBI, 200 cGy (FLUIM regimen). Between 2000 and 2012, 51 patients received BM wi...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2014.193

    authors: Gomez E,Duléry R,Langlois C,Coiteux V,Terriou L,Magro L,Gauthier J,de Berranger E,Duhamel A,Yakoub-Agha I

    更新日期:2014-12-01 00:00:00

  • Efficiency of allogeneic hematopoietic SCT from HLA fully-matched non-sibling relatives: a new prospect of exploiting extended family search.

    abstract::The best donors for hematopoietic SCT (HSCT) are fully-matched siblings. In patients without fully-matched siblings, HLA registries or cord blood banks are alternative strategies with some restrictions. Owing to the high rate of consanguineous marriage in our country, between 2006 and 2013, extended family searches we...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2014.307

    authors: Hamidieh AA,Dehaghi MO,Paragomi P,Navaei S,Jalali A,Eslami GG,Behfar M,Ghavamzadeh A

    更新日期:2015-04-01 00:00:00

  • Clinical use of streptolysin-O to facilitate antisense oligodeoxyribonucleotide delivery for purging autografts in chronic myeloid leukaemia.

    abstract::Antisense oligodeoxyribonucleotides (ODN) targeted against the breakpoint in BCR-ABL mRNA will specifically decrease BCR-ABL mRNA, provided cells are first permeabilised with streptolysin-O (SL-O). We used 18-mer chimeric methylphosphonodiester: phosphodiester linked (4-9-4) ODN complementary to 9 bases either side of...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:10.1038/sj.bmt.1701801

    authors: Clark RE,Grzybowski J,Broughton CM,Pender NT,Spiller DG,Brammer CG,Giles RV,Tidd DM

    更新日期:1999-06-01 00:00:00

  • Salvage autologous or allogeneic transplantation for multiple myeloma refractory to or relapsing after a first-line autograft?

    abstract::Forty-two patients allografted for multiple myeloma after not having attained at least a partial remission (n = 19) or after having experienced disease progression (n = 23) following one autograft were compared with 42 pair-matched controls who underwent salvage autotransplantation under identical conditions. Autograf...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:10.1038/sj.bmt.1701208

    authors: Mehta J,Tricot G,Jagannath S,Ayers D,Singhal S,Siegel D,Desikan K,Munshi N,Fassas A,Mattox S,Vesole D,Crowley J,Barlogie B

    更新日期:1998-05-01 00:00:00