A single-center experience using alemtuzumab, fludarabine, melphalan, and thiotepa as conditioning for transplantation in pediatric patients with chronic granulomatous disease.

Abstract:

:Chronic granulomatous disease (CGD) is an immune deficiency characterized by defective neutrophil function and increased risk of life-threatening infections. Allogeneic hematopoietic cell transplantation is curative for CGD, and conditioning regimen impacts transplant-related outcomes. We report a single-center prospective study (NCT01821781) of four patients with CGD transplanted using a reduced-intensity conditioning regimen (RIC) containing alemtuzumab, fludarabine, melphalan, and thiotepa. Patients had early immune reconstitution with low incidence of infections. Disease-free survival was 75% at a median of five years after transplant. This RIC regimen presents an alternative approach for transplant of patients with CGD who may not tolerate busulfan-based conditioning.

journal_name

Pediatr Blood Cancer

journal_title

Pediatric blood & cancer

authors

Bhatt ST,Schulz G,Hente M,Slater A,Murray L,Shenoy S,Bednarski JJ

doi

10.1002/pbc.28030

subject

Has Abstract

pub_date

2020-01-01 00:00:00

pages

e28030

issue

1

eissn

1545-5009

issn

1545-5017

journal_volume

67

pub_type

临床试验,杂志文章
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    journal_title:Pediatric blood & cancer

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    更新日期:2018-09-01 00:00:00

  • Toxicity profile of delayed high dose sodium thiosulfate in children treated with carboplatin in conjunction with blood-brain-barrier disruption.

    abstract:PURPOSE:To assess the safety of delayed high dose intravenous (i.v.) sodium thiosulfate (STS) in a case series of 12 children with malignant brain tumors who were treated with intraarterial (i.a.) carboplatin in conjunction with blood-brain-barrier disruption (BBBD). METHODS:Twelve children ages 17 months-12 years und...

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    doi:10.1002/pbc.20529

    authors: Neuwelt EA,Gilmer-Knight K,Lacy C,Nicholson HS,Kraemer DF,Doolittle ND,Hornig GW,Muldoon LL

    更新日期:2006-08-01 00:00:00

  • Follicular variant papillary thyroid carcinoma in a pediatric population.

    abstract:BACKGROUND:Follicular variant of papillary thyroid carcinoma (FVPTC) has been shown to be an intermediate entity between papillary (PTC) and follicular/Hurtle cell (FTC) thyroid carcinoma in adults. However, the tumor characteristics and prognosis of FVPTCs has not been studied in the pediatric population and is the fo...

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    pub_type: 杂志文章

    doi:10.1002/pbc.25623

    authors: Lerner J,Goldfarb M

    更新日期:2015-11-01 00:00:00

  • Detection of cell-free RNA in children with neuroblastoma and comparison with that of whole blood cell RNA.

    abstract:BACKGROUND:Since there is no validated assay to monitor disease in children with neuroblastoma (NB), we tested whether NB specific cell-free RNA could be detected in their plasma samples. Moreover, with the aim of reducing patients' discomfort, we compared this assay to a recently standardized procedure that uses a lar...

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    doi:10.1002/pbc.22498

    authors: Corrias MV,Pistorio A,Cangemi G,Tripodi G,Carlini B,Scaruffi P,Fardin P,Garaventa A,Pistoia V,Haupt R

    更新日期:2010-07-01 00:00:00

  • Hepatoblastoma in a child with neurofibromatosis type I.

    abstract::A major hallmark of NF1 is the development of benign tumors, including peripheral neurofibromas, plexiform neurofibromas, gliomas of the optic tract, other low grade gliomas, and pheochromocytomas. Hepatoblastoma have not been previously reported in patients with neurofibromatosis type 1. We present a case of a 9-mont...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.20663

    authors: Uçar C,Calişkan U,Toy H,Günel E

    更新日期:2007-09-01 00:00:00

  • Center-Based Quality Initiative Targets Youth Preparedness for Medical Independence: HEMO-Milestones Tool in a Comprehensive Hemophilia Clinic Setting.

    abstract:BACKGROUND:Patient transition readiness self-assessment tools and investigation into patient and parent perceptions of the transition process from pediatric to adult care models have informed recognition of gaps in care, particularly for those with chronic disease. Implementation of a longitudinal transition process wi...

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    pub_type: 杂志文章

    doi:10.1002/pbc.25807

    authors: Croteau SE,Padula M,Quint K,D'Angelo L,Neufeld EJ

    更新日期:2016-03-01 00:00:00

  • Functional imaging of RAS pathway targeting in malignant peripheral nerve sheath tumor cells and xenografts.

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    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.28639

    authors: Butler E,Schwettmann B,Geboers S,Hao G,Kim J,Nham K,Sun X,Laetsch TW,Xu L,Williams NS,Skapek SX

    更新日期:2020-12-01 00:00:00

  • Clinical management of pediatric aggressive fibromatosis involving the mandible.

    abstract:BACKGROUND:Pediatric aggressive fibromatosis (AF) is a rare, benign tumor with locally infiltrative growth. Therefore, how to prevent reoccurrence while maintaining the mandible contour and continuity as much as possible is very important when the mandible is involved. PROCEDURE:We selected 10 pediatric patients with ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.24145

    authors: Zhou Y,Zhang Z,Fu H,Qiu W,Wang L,He Y

    更新日期:2012-10-01 00:00:00

  • Sex ratio among childhood cancers by single year of age.

    abstract:BACKGROUND:The male excess in childhood cancer incidence is well-established; however, the underlying biologic mechanisms remain unknown. Examining the association between male sex and childhood cancer by single year of age and tumor type may highlight important periods of risk such as variation in growth and hormonal ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.27620

    authors: Williams LA,Richardson M,Marcotte EL,Poynter JN,Spector LG

    更新日期:2019-06-01 00:00:00

  • Chronic lymphocytic leukemia in a child: a challenging diagnosis in pediatric oncology practice.

    abstract::Chronic lymphocytic leukemia/lymphoma (CLL) is an extremely rare disease during childhood. We report a 16-year-old female who presented with lymphadenopathies and she was diagnosed as T cell lymphoblastic lymphoma. Her chemotherapy response was minimal and clinical findings were unusual. Therefore, her biopsy specimen...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.24865

    authors: Demir HA,Bayhan T,Üner A,Kurtulan O,Karakuş E,Emir S,Özyörük D,Ceylaner S

    更新日期:2014-05-01 00:00:00

  • Comparison of clinical features and outcomes in patients with extraskeletal versus skeletal localized Ewing sarcoma: A report from the Children's Oncology Group.

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    pub_type: 杂志文章

    doi:10.1002/pbc.26096

    authors: Cash T,McIlvaine E,Krailo MD,Lessnick SL,Lawlor ER,Laack N,Sorger J,Marina N,Grier HE,Granowetter L,Womer RB,DuBois SG

    更新日期:2016-10-01 00:00:00

  • Household material hardship in families of children post-chemotherapy.

    abstract::Poverty is an important patient-reported outcome of therapy and a potential predictor of outcome disparities in pediatric cancer. We previously identified that nearly 30% of pediatric cancer families experience household material hardship (HMH), a concrete measure of poverty including food, energy, or housing insecuri...

    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章

    doi:10.1002/pbc.26743

    authors: Bilodeau M,Ma C,Al-Sayegh H,Wolfe J,Bona K

    更新日期:2018-01-01 00:00:00

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    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,多中心研究

    doi:10.1002/pbc.26087

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    更新日期:2016-10-01 00:00:00

  • Persistent jaundice in an infant with homozygous beta thalassemia due to co-inherited Crigler-Najjar syndrome.

    abstract::Clinically apparent jaundice is unusual in patients with beta-thalassemia major. Co-inheritance of Gilbert syndrome has been reported to cause hyperbilirubinemia in these subjects. Crigler-Najjar syndrome is another rare disorder of bilirubin metabolism caused by mutation in the gene coding the enzyme UGT1A1. We repor...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.22313

    authors: Aggarwal V,Seth A,Sharma S,Aneja S,Sammarco P,Fabiano C

    更新日期:2010-04-01 00:00:00

  • Oxandrolone for the treatment of bone marrow failure in Fanconi anemia.

    abstract:BACKGROUND:A majority of Fanconi anemia (FA) patients will experience bone marrow failure (BMF) and androgen therapy (most often oxymetholone) may be utilized as a treatment to improve BMF-related cytopenias. However, oxymetholone is associated with toxicities making identification of other agents of interest. In this ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.24617

    authors: Rose SR,Kim MO,Korbee L,Wilson KA,Ris MD,Eyal O,Sherafat-Kazemzadeh R,Bollepalli S,Harris R,Jeng MR,Williams DA,Smith FO

    更新日期:2014-01-01 00:00:00

  • CNS-directed therapy in young children with T-lineage acute lymphoblastic leukemia: High-dose methotrexate versus cranial irradiation.

    abstract:BACKGROUND:Prophylactic use of cranial radiation therapy (CRT) in young children with acute lymphoblastic leukemia (ALL) is associated with significant long-term morbidity. Therefore, current treatment protocols for pediatric B-precursor ALL have abandoned prophylactic CRT in favor of intrathecal chemotherapy, combined...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.10392

    authors: Nathan PC,Maze R,Spiegler B,Greenberg ML,Weitzman S,Hitzler JK

    更新日期:2004-01-01 00:00:00

  • Neuroblastoma with symptomatic epidural compression in the infant: the AIEOP experience.

    abstract:BACKGROUND:Symptoms of epidural compression (SEC) in children with neuroblastoma (particularly infants) may be misinterpreted, leading to delay in diagnosis. PATIENTS AND METHODS:Clinical, imaging and follow-up data of 34 infants with neuroblastoma and SEC diagnosed between 2000 and 2011 at Italian AIEOP centers were ...

    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章,多中心研究

    doi:10.1002/pbc.25028

    authors: De Bernardi B,Quaglietta L,Haupt R,Castellano A,Tirtei E,Luksch R,Mastrangelo S,Viscardi E,Indolfi P,Cellini M,Tamburini A,Erminio G,Gandolfo C,Sorrentino S,Vetrella S,Gigliotti AR

    更新日期:2014-08-01 00:00:00

  • Prognostic impact of diagnostic and treatment delays in children with osteosarcoma.

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    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章,多中心研究

    doi:10.1002/pbc.28180

    authors: Vasquez L,Silva J,Chavez S,Zapata A,Diaz R,Tarrillo F,Maza I,Sialer L,García J

    更新日期:2020-04-01 00:00:00

  • Presentation of acute promyelocytic leukemia as granulocytic sarcoma.

    abstract::Granulocytic sarcoma (GS) is a localized tumor composed of immature myeloid cells. This extramedullary tumor can present before, concurrent with or after the diagnosis of acute myeloid leukemia. GS is extremely uncommon in acute promyelocytic leukemia (APL). As a proportion of patients never develop systemic disease, ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.21190

    authors: Worch J,Ritter J,Frühwald MC

    更新日期:2008-03-01 00:00:00

  • Religious coping and the use of prayer in children with sickle cell disease.

    abstract:BACKGROUND:While adolescents and adults with sickle cell disease (SCD) have reported using religion to cope with SCD, there is no data examining religious coping in young children with SCD. The purpose of this qualitative study was to: (1) describe the types of religious coping used by children with SCD; (2) describe t...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.23038

    authors: Cotton S,Grossoehme D,McGrady ME

    更新日期:2012-02-01 00:00:00

  • Pharmacokinetics and pharmacodynamics of intravenous epoetin alfa in children with cancer.

    abstract:BACKGROUND:Epoetin alfa (EPO, PROCRIT) pharmacokinetics and pharmacodynamics were evaluated in children with malignant solid tumors receiving chemotherapy. PROCEDURE:Children initially received IV EPO 600 IU/kg (max dose 40,000 IU) or placebo once weekly for 16 weeks. Dose was increased to 900 IU/kg (max dose 60,000 I...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,随机对照试验

    doi:10.1002/pbc.20685

    authors: Freeman BB 3rd,Hinds P,Iacono LC,Razzouk BI,Burghen E,Stewart CF

    更新日期:2006-10-15 00:00:00

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    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章

    doi:10.1002/pbc.27735

    authors: Atwa ZT,Wahed WYA

    更新日期:2019-07-01 00:00:00

  • Childhood liver tumors in Argentina: Incidence trend and survival by treatment center. A report from the national pediatric cancer registry, ROHA network 2000-2015.

    abstract:BACKGROUND:Information on the epidemiology of pediatric liver tumors in Latin America is limited. PURPOSE:To describe the incidence of liver tumors in a pediatric registry in Argentina according to geographic region, national trends over 16 years, and survival related to stage, age, sex, and care center. METHODS:Newl...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.28583

    authors: Moreno F,Rose A,Chaplin MA,Cipolla MC,García Lombardi M,Nana M,Cervio G,Halac E,Viso M,Ayarzabal V,Bosaleh A,Liberto D,Sarabia E,Rizzi A,Morici M,Streitenberger P,de Dávila MTG

    更新日期:2020-11-01 00:00:00

  • Determinants of quality of life outcomes for survivors of pediatric brain tumors.

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    pub_type: 杂志文章

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    authors: Barrera M,Atenafu EG,Schulte F,Bartels U,Sung L,Janzen L,Chung J,Cataudella D,Hancock K,Saleh A,Strother D,McConnell D,Downie A,Hukin J,Zelcer S

    更新日期:2017-09-01 00:00:00

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    authors: de Lambert G,Lardy H,Martelli H,Orbach D,Gauthier F,Guérin F

    更新日期:2016-04-01 00:00:00

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    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.23389

    authors: Bansal D,Shava U,Varma N,Trehan A,Marwaha RK

    更新日期:2012-09-01 00:00:00

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    pub_type: 杂志文章

    doi:10.1002/pbc.21521

    authors: Manley S,Mallinson H,Caswell M,Keenan R,Pizer B

    更新日期:2008-10-01 00:00:00

  • Identifying relapses and stem cell transplants in pediatric acute lymphoblastic leukemia using administrative data: Capturing national outcomes irrespective of trial enrollment.

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    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.28315

    authors: Cahen VC,Li Y,Getz KD,Elgarten CW,DiNofia AM,Wilkes JJ,Winestone LE,Huang YV,Miller TP,Gramatges MM,Rabin KR,Fisher BT,Aplenc R,Seif AE

    更新日期:2020-05-11 00:00:00