Abstract:
BACKGROUND:Few data exist regarding the clinical outcome of patients with Ewing sarcoma (EWS) treated with pencil beam scanning proton therapy (PT). We report the outcome of children, adolescents and young adults (AYA) treated with PT at the Paul Scherrer Institute. MATERIALS:Thirty-eight patients (median age, 9.9 years) received a median dose of 54.9 Gy(RBE) (where RBE is relative biologic effectiveness). Size of the tumor ranged from 1.7 to 24 cm. Most common primary site was axial/pelvic (n = 27; 71%). Four patients (11%) presented with metastases at diagnosis. Twenty (53%) patients had chemo-PT only. Median follow-up was 49.6 months (range, 9.2-131.7). RESULTS:The 5-year actuarial rate of local control (LC), distant metastasis-free survival (DMFS), and overall survival (OS) were 81.5%, 76.4%, and 83.0%, respectively. All local recurrences occurred in field and in patients with nonextremity primaries. Six patients died, all of tumor progression. Age < 10 years was a favorable factor of borderline significance for LC (P = 0.05) and OS (P = 0.05), but was significant for DMFS (P = 0.003). Tumor volume <200 ml was a significant prognostic factors for DMFS (P = 0.03), but not for OS (P = 0.07). Metastasis at diagnosis was a strong predictor of local failure (P = 0.003). Only two grade 3 late toxicities were observed. The 5-year actuarial rate of grade 3 toxicity-free survival was 90.9%. CONCLUSIONS:These preliminary data suggest that the outcomes of children and AYA with EWS are good and PT was well tolerated with few late adverse events. The local and distant tumor control for older patients with large pre-PT tumor volumes remains problematic.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Weber DC,Murray FR,Correia D,Bolsi A,Frei-Welte M,Pica A,Lomax AJ,Schneider R,Bachtiary Bdoi
10.1002/pbc.26688subject
Has Abstractpub_date
2017-12-01 00:00:00issue
12eissn
1545-5009issn
1545-5017journal_volume
64pub_type
杂志文章abstract::Ovarian tumors have a low incidence in childhood, accounting for 1% of malignancies within the ages of 0-17 years. Small cell carcinoma of the ovary is a rare histology and historically has a poor prognosis. We report a case of an 11-year-old female diagnosed with small cell carcinoma of the ovary and hypercalcemia (S...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22184
更新日期:2009-12-15 00:00:00
abstract::Acute lymphoblastic leukemia (ALL) is the most common type of pediatric neoplasia. Highly heterogeneous, ALL includes several genetic subtypes with varying clinical outcome. Although, some features are well established as prognostic predictors, the details of the molecular mechanisms underlying different phenotypes ar...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.24167
更新日期:2012-10-01 00:00:00
abstract:BACKGROUND:Clear cell sarcoma of the kidney (CCSK) is known for its propensity to metastasize to bone, but it also spreads to other sites including the brain. This study was undertaken to describe the treatment and outcomes of patients with recurrent CCSK involving the brain. METHODS:A retrospective records review was...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21131
更新日期:2008-02-01 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:BACKGROUND:To determine the maximum-tolerated duration and dose-limiting toxicity of a daily schedule of orally administered cyclophosphamide and topotecan in pediatric patients with recurrent or refractory malignant solid tumors. METHODS:Patients received oral cyclophosphamide (50 mg/m2/dose) in the morning followed ...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.10456
更新日期:2004-01-01 00:00:00
abstract:BACKGROUND:Low expression of HLA class II antigens has been associated with more aggressive disease in several human malignancies including adult adrenocortical tumors (ACT), but their clinical relevance in pediatric ACT needs to be investigated. PROCEDURE:This study analyzed the expression profile of three class II h...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25118
更新日期:2014-11-01 00:00:00
abstract:BACKGROUND:Methylenetetrahydrofolate reductase (MTHFR) C677T and A1298C polymorphisms have been implicated in childhood acute lymphoblastic leukemia (ALL) risk, but previously published studies were inconsistent and recent meta-analyses were not adequate. PROCEDURES:In a meta-analysis of 21 publications with 4,706 cas...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,meta分析,评审
doi:10.1002/pbc.23137
更新日期:2012-04-01 00:00:00
abstract:BACKGROUND:Premature newborns are vulnerable to iron imbalance, although the iron homeostasis during the perinatal period remains unclear. To clarify the iron metabolism of premature infants, we measured serum prohepcidin concentrations of preterm infants, and analyzed the association with iron parameters. METHODS:Sev...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22773
更新日期:2011-02-01 00:00:00
abstract::Langerhans cell histiocytosis is known to mimic many other conditions. We present two patients where anti-tubercular therapy was instituted when clinical and radiological features suggested tuberculosis. The correct diagnosis of histiocytosis was reached only on further work-up including immunohistochemistry following...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21930
更新日期:2009-07-01 00:00:00
abstract::A male patient diagnosed with severe congenital protein C (PC) deficiency during the neonatal period was treated with long-term warfarin but frequently developed purpura fulminans and bleeding. At four years of age, edoxaban was initiated (direct oral anticoagulant [DOAC]). His d-dimer and fibrin/fibrinogen degradatio...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27686
更新日期:2019-06-01 00:00:00
abstract:BACKGROUND:High birth weight increases the risk of childhood acute lymphoid leukemia (ALL) through unknown mechanisms. Whether this risk is specific to ALL subtypes is unknown, and low case numbers have prevented investigation of the rarer leukemias. Here we address these associations using a large population-based dat...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23209
更新日期:2012-01-01 00:00:00
abstract:BACKGROUND:The efficacy of combination chemotherapy with methotrexate (MTX) and asparaginase is not well known in relapsed and refractory acute leukemia after contemporary therapy. PROCEDURE:A retrospective study of pediatric patients with relapsed or refractory acute myeloid leukemia (AML) who received MTX and aspara...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24470
更新日期:2013-07-01 00:00:00
abstract:BACKGROUND:The outcome for patients with Ewing sarcoma family of tumors (ESFTs) of bone with metastases at diagnosis remains poor despite new approaches to treatment. We evaluated whether a dose-intensity chemotherapy regimen improved survival for patients with ESFTs of bone with metastases at diagnosis. METHODS:We en...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.21233
更新日期:2007-12-01 00:00:00
abstract:BACKGROUND:Glucocorticoids and asparaginase, used to treat acute lymphoblastic leukemia (ALL), can cause hypertriglyceridemia. We compared triglyceride levels, risk factors, and associated toxicities in two ALL trials at St. Jude Children's Research Hospital with identical glucocorticoid regimens, but different asparag...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28040
更新日期:2020-01-01 00:00:00
abstract::Leydig cell tumors account for 3% of testicular tumors and have never been reported after treatment for Ewing's sarcoma. We report the unusual occurrence of a patient who developed a Leydig cell tumor of the testis 18 years after successful treatment for Ewing's sarcoma. Additional monitoring for second malignancies m...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20825
更新日期:2007-12-01 00:00:00
abstract::Aplastic anemia (AA) is an immune-mediated disease. Although most patients are responsive to immunosuppressive therapy (IST) with a combination of anti-thymocyte globulin (ATG) and cyclosporine (CsA), some patients relapse or are refractory to IST. Sirolimus (rapamysin) inhibits the serine-threonine kinase mammalian t...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22865
更新日期:2011-07-01 00:00:00
abstract::The molecular detection of minimal residual disease (MRD) is standard of care in acute lymphoblastic leukemia to personalize the stratification of patients to appropriate intensity chemotherapy regimens. High-throughput sequencing (HTS) techniques are driving changes to MRD methodologies. Our study demonstrates HTS ca...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27787
更新日期:2019-08-01 00:00:00
abstract:BACKGROUND:Adolescents with acute lymphoblastic leukaemia (ALL) have languished in the shadow of success of the outcome of therapy in childhood ALL. Their treatment has always been incorporated into either paediatric or adult clinical trials depending on the mode of referral and hence there is a need to address an age ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20749
更新日期:2007-03-01 00:00:00
abstract::Scrotal ulcers are a rare manifestation in patients with acute promyelocytic leukemia. Fournier's gangrene (FG) is even rarer. We describe three adolescents and young adults who developed scrotal ulcerations during induction with all-trans-retinoic acid. One patient developed FG. These lesions are predominantly seen i...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21549
更新日期:2008-08-01 00:00:00
abstract:BACKGROUND:Radiation therapy (RT)-induced effects in children treated for low grade glioma (LGG) can result in worsening of neurologic symptoms and clinical and radiographic deterioration. Treatment for radiation-induced tumor enlargement is based on symptom control and usually involves steroids. PROCEDURE:We conducte...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25277
更新日期:2015-02-01 00:00:00
abstract:BACKGROUND:Solid pseudopapillary pancreatic tumors (SPPT) are an extremely rare entity in pediatric patients. Even if the role of radical surgical resection as primary treatment is well established, data about follow-up after pancreatic resection in children are scant. METHODS:A retrospective review of data from the I...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27519
更新日期:2019-03-01 00:00:00
abstract:BACKGROUND:Nephrogenic rests (NRs) are abnormally persistent foci of embryonal cells, thought to be the precursor lesion of Wilms tumors (WTs). To date, their presence has not been systematically examined in WTs treated with preoperative chemotherapy. METHODS:A systematic analysis of the data on NRs in WTs treated wit...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.26547
更新日期:2017-11-01 00:00:00
abstract::Family financial hardship has emerged as a burden of pediatric cancer treatment with negative implications for family well-being. As part of an extensive project to create evidence-based standards for the psychosocial care of children with cancer, we performed a literature review of pediatric cancer-associated financi...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.25714
更新日期:2015-12-01 00:00:00
abstract:BACKGROUND:Conflicting data exist regarding whether low bone mineral density (BMD) is associated with Fanconi anemia (FA). The current study identified the frequency of low BMD in FA, expecting low BMD even in childhood and before HCT. PROCEDURE:Thirty-seven FA patients (18 prior HCT, 19 no prior HCT), participating i...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22956
更新日期:2011-12-01 00:00:00
abstract:BACKGROUND:Alveolar soft-part sarcoma (ASPS), a rare vascular sarcoma with a clinically indolent course, frequently presents with metastases. Vascular endothelial growth factor (VEGF) is a promising therapeutic target. In a phase-II trial of the VEGF receptor inhibitor cediranib for adults with ASPS, the partial respon...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.27987
更新日期:2019-12-01 00:00:00
abstract:BACKGROUND:The median age of patients with Ewing sarcoma (EwS) at diagnosis is around 14-15 years. Older age is associated with a worse outcome. The correlation of age at diagnosis on sites of disease has not been fully described. OBJECTIVE:The goal of this study was to evaluate the differences in sites of primary tum...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27251
更新日期:2018-09-01 00:00:00
abstract:BACKGROUND AND AIM:We reviewed the results and pattern of failure of the consensus HB/HCC 1996 treatment protocol for pediatric hepatoblastoma (HB) in Hong Kong. The role of SIOPEL and Children's Hepatic tumors International Collaboration (CHIC) risk stratification was evaluated. METHODS:Patients enrolled on the proto...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27482
更新日期:2019-01-01 00:00:00
abstract::Sickle cell disease (SCD) is an inherited red blood cell disorder that occurs worldwide and results in significant morbidity and mortality. In addition to affected children who are born in the US, an increasing number of children with SCD are migrating to the US with their families or through international adoption. C...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27000
更新日期:2018-06-01 00:00:00
abstract::Dry eye disease is a well-known late complication of radiation therapy and is often difficult to treat. We evaluated the usefulness of cyclosporine 0.05% ophthalmic emulsion for the treatment of radiation-associated dry eye in children. Eleven children received cyclosporine 0.05% emulsion twice daily after failure of ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24461
更新日期:2013-07-01 00:00:00
abstract:BACKGROUND:The Children's Oncology Group (COG) publishes consensus guidelines with screening recommendations for early identification of treatment-related morbidities among childhood cancer survivors. We sought to estimate the yield of recommended yearly urinalysis screening for genitourinary complications as per Versi...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.25897
更新日期:2016-05-01 00:00:00