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 Cancerjournal_title
Pediatric blood & cancerauthors
Bhatt ST,Schulz G,Hente M,Slater A,Murray L,Shenoy S,Bednarski JJdoi
10.1002/pbc.28030subject
Has Abstractpub_date
2020-01-01 00:00:00pages
e28030issue
1eissn
1545-5009issn
1545-5017journal_volume
67pub_type
临床试验,杂志文章abstract:BACKGROUND:Acute lymphoblastic leukemia (ALL) is the most common cancer in children. Because of major improvements in treatment protocols, the survival rate now exceeds 80%. However, ALL treatments can cause long-term neurocognitive sequelae, which negatively impact academic achievement and quality of life. Therefore, ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.27259
更新日期:2018-09-01 00:00:00
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...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20529
更新日期:2006-08-01 00:00:00
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...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25623
更新日期:2015-11-01 00:00:00
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...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22498
更新日期:2010-07-01 00:00:00
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
更新日期:2007-09-01 00:00:00
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...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25807
更新日期:2016-03-01 00:00:00
abstract:BACKGROUND:Malignant peripheral nerve sheath tumor (MPNST) is an aggressive form of soft-tissue sarcoma (STS) in children. Despite intensive therapy, relatively few children with metastatic and unresectable disease survive beyond three years. RAS pathway activation is common in MPNST, suggesting MEK pathway inhibition ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28639
更新日期:2020-12-01 00:00:00
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
更新日期:2012-10-01 00:00:00
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
更新日期:2019-06-01 00:00:00
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
更新日期:2014-05-01 00:00:00
abstract:BACKGROUND:The prognostic significance of having extraskeletal (EES) versus skeletal Ewing sarcoma (ES) in the setting of modern chemotherapy protocols is unknown. The purpose of this study was to compare the clinical characteristics, biologic features, and outcomes for patients with EES and skeletal ES. METHODS:Patie...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26096
更新日期:2016-10-01 00:00:00
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
更新日期:2018-01-01 00:00:00
abstract:BACKGROUND:Robatumumab (19D12; MK-7454 otherwise known as SCH717454) is a fully human antibody that binds to and inhibits insulin-like growth factor receptor-1 (IGF-1R). This multiinstitutional study (P04720) determined the safety and clinical efficacy of robatumumab in three separate patient groups with resectable ost...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.26087
更新日期:2016-10-01 00:00:00
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
更新日期:2010-04-01 00:00:00
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
更新日期:2014-01-01 00:00:00
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
更新日期:2004-01-01 00:00:00
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
更新日期:2014-08-01 00:00:00
abstract:BACKGROUND:The aim of this study is to evaluate the relationship between the latency to diagnosis (LD) and the time to completion of chemotherapy (TCC) with clinical outcomes in children with osteosarcoma. METHODS:We performed a retrospective analysis of all patients who received treatment for osteosarcoma in two tert...
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
更新日期:2020-04-01 00:00:00
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
更新日期:2008-03-01 00:00:00
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
更新日期:2012-02-01 00:00:00
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
更新日期:2006-10-15 00:00:00
abstract:BACKGROUND/OBJECTIVES:β-Thalassemia intermedia (β-TI) accounts for up to one-fourth of β-thalassemia patients. Evaluating and improving quality of life (QOL) should be a goal in β-TI follow-up and management strategies. Patients' perceptions of their illness and its treatment may impact their QOL. This study aimed to e...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.27735
更新日期:2019-07-01 00:00:00
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
更新日期:2020-11-01 00:00:00
abstract:INTRODUCTION:To describe the quality of life (QOL) of pediatric brain tumor survivors (PBTSs) prospectively and to identify potential medical, personal and family contextual factors associated with QOL. METHODS:Ninety-one PBTSs (8-16 years) who were off treatment and attending a regular classroom participated. Self- a...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26481
更新日期:2017-09-01 00:00:00
abstract:BACKGROUND:Pulmonary hypertension (PHT) may be the leading cause of death in β-thalassemia patients; however, its pathophysiologic mechanisms are still unclear. Recent studies indicate that asymmetric dimethylarginine (ADMA) plays a role in the initiation and progression of a variety of diseases, especially the cardiov...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25076
更新日期:2014-09-01 00:00:00
abstract:BACKGROUND:The objective was to investigate factors associated with patient-related timing (PRT) to seek healthcare and health service-related timing (HSRT) to diagnose cancer and provide treatment to children without social security in Mexico. PROCEDURE:A cross-sectional survey was conducted in 13 Ministry of Health ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28331
更新日期:2020-09-01 00:00:00
abstract:BACKGROUND:Right hemicolectomy (RHC) in patients with neuroendocrine tumors (NET) of the appendix has been advocated for tumors more than 2 cm, or with mesoappendix/vessel invasion, or with raised mitotic activity. No study demonstrated prolonged survival with RHC over appendectomy alone. The aim of this study was to c...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.25823
更新日期:2016-04-01 00:00:00
abstract:BACKGROUND:Long-term adverse effects of Imatinib in children with chronic myeloid leukemia (CML) are uncertain. The aim was to study the effect of imatinib on growth in children with CML. PROCEDURE:Children ≤13 years of age at diagnosis were enrolled retrospectively, from 2004 to 2011, from a single center in India. P...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23389
更新日期:2012-09-01 00:00:00
abstract::Aciclovir prophylaxis was previously given to all immunocompromised patients treated by our unit, following contact with varicella zoster. In 2003, we changed practice according to National Guidelines, giving prophylaxis only to patients without serum varicella zoster immunoglobulin G antibody (VZ IgG) at diagnosis of...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21521
更新日期:2008-10-01 00:00:00
abstract:INTRODUCTION:Our objectives were to design and validate methods to identify relapse and hematopoietic stem cell transplantation (HSCT) in children with acute lymphoblastic leukemia (ALL) using administrative data representing hospitalizations at US pediatric institutions. METHODS:We developed daily billing and ICD-9 c...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28315
更新日期:2020-05-11 00:00:00