pinecone/core-2020-05-10-deduplication huggingface dataset:
The Bt-DUX: Development of a subjective measure of health-related quality of life in patients who underwent surgery for lower extremity malignant bone tumor

Background. To examine the practical applicability, internal consistency, and validity of the Bt-DUX, a disease-specific Health Related Quality of Life (HRQoL) instrument. The Bt-Dux was developed to examine patients' individual values of their life after a malignant bone tumor of the lower extremity at four domains (cosmetic, social, emotional, and functional). Procedure. Patients were eligible for this cross-sectional, multicenter study if they underwent surgery for a malignant tumor of the leg in a period ranging between 12 and 60 months before the recruitment. Assessments included: Bt-DUX, Toronto Extremity Salvage Score (TESS) Short Form (SF)-36, TNO-AZL Questionnaire for Adult's Quality of Life (TAAQOL), and TNO-AZL Children's Quality of Life Questionnaire (TACQOL). Results. Seventy-two patients (35 male, 37 female), mean age 17 (SD 4) years were included. Limb sparing surgery took place in 32 patients and ablative surgery in 40 patients. The Bt-DUX was completed in less than 5 min and easy to comprehend. The mean Bt-DUX score was 69.8 (SD 15.5), with Cronbach's alpha being 0.92. Domain-total correlations ranged between 0.84 and 0.88 (P<0.01). Correlations between Bt-DUX Total score and TESS, SF-36 Physical and Mental Component Summary scales and selected TACQOL and TAAQOL scores were statistically significant (P<0.05), except for the social scale of the TACQOL. The Bt-DUX was able to discriminate between patients with higher and lower TESS scores (P<0.05). Conclusion. The Bt-DUX was found to be a practical and valid instrument. Its added value compared with existing HRQoL measures needs to be further established
Pre-B-cell acute lymphoblastic leukemia with bulk extramedullary disease and chromosome 22 (EWSR1) rearrangement masquerading as Ewing sarcoma

We report a 2-year-old female with a subcutaneous tumor who was initially misdiagnosed as suffering from Ewing sarcoma with a positive EWSR1 rearrangement and EWS/FLI1 transcript. After finding lymphoblasts in peripheral blood, the diagnosis of acute lymphoblastic leukemia was established. This necessitated further analysis of the subcutaneous tumor. The tissue was positive for immature B-cell markers and an immunoglobulin heavy chain gene rearrangement, which confirmed the final diagnosis of common type acute lymphoblastic leukemia with bulk extramedullary disease. The patient was treated with chemotherapy and was in remission 30 months after the diagnosis
Area-based differentials in childhood cancer incidence in Australia, 1996-2006

Background International studies examining the association between the incidence of childhood cancer and characteristics of the area in which the patient lives have generally reported inconsistent patterns. Area-based differentials in childhood cancer throughout Australia have not been previously published at a national level.  Procedure Population-based information from the Australian Paediatric Cancer Registry was used to identify all children aged 0- to 14-years old diagnosed with invasive cancer or intracranial and intraspinal tumors of benign or uncertain behavior between 1996 and 2006. Age-standardized incidence rates per million children per year and the corresponding incidence rate ratios were calculated, categorized by remoteness of residence and an area-based index of socioeconomic disadvantage. Results were also stratified by the most common types of childhood cancer.  Results There was a significant, decreasing gradient in the incidence of childhood cancer as remoteness of residence increased. Children living in remote or very remote areas were 21% less likely to be diagnosed with cancer compared to children in major cities, mainly due to differences in the incidence of leukemias and lymphomas. This differential was no longer significant when only non-Indigenous children were considered. No clear relationship was found between incidence and socioeconomic status (SES) in contrast to similar earlier studies.  Conclusions The findings by remoteness of residence are consistent with the lower incidence rates of cancer that are typically associated with Indigenous Australians. There is also a suggestion that the etiological factors associated with childhood leukemia and SES may have altered over time.Full Tex
Impact of psychiatric diagnoses on hospital length of stay in children with sickle cell anemia

Background  Patients with sickle cell anemia (SCA) experience a broad range of psychiatric disorders, placing them at risk for more complicated and longer hospitalizations for vaso‐occlusive crises (VOC). The current study examined the frequency of psychiatric disorders in SCA patients (ages birth to 20 years) admitted for VOC in a nationally representative sample and the association between psychiatric disorders and hospital length of stay (LOS).    Procedure  Patients with a primary diagnosis of SCA with crisis identified through the nationally representative Kids' Inpatient Database (KID) 2006 from the Agency of Healthcare Research and Quality were included for analysis. Patients with psychiatric disorders listed as secondary diagnoses were categorized under specific psychiatric disorders (mood disorder, anxiety disorder, disruptive behavior disorder, substance use disorder). Non‐parametric and regression analyses were utilized for nationally weighted data, to determine the effect of psychiatric disorders on LOS, while controlling for significant covariates.    Results  For 21,255 hospital discharges for children with SCA with crisis in 2006, the mean LOS was 4.51 days. Approximately 6% of the patients discharged had a psychiatric disorder. After adjusting for significant covariates, mood disorders, anxiety disorders, and any psychiatric disorder were each associated with significantly longer LOS ( P  < 0.01). Substance and disruptive behavior disorders were not associated with LOS.    Conclusions  Pediatric patients diagnosed with a psychiatric disorder, specifically mood or anxiety disorders, have longer LOS for VOC. These findings suggest that future interventions aimed at managing VOC may need to consider adjunctive psychiatric assessment and intervention. Pediatr Blood Cancer 2012; 58: 239–243. © 2011 Wiley Periodicals, Inc
Good prognosis of localized osteosarcoma in young patients treated with limb-salvage surgery and chemotherapy

BACKGROUND: The objective of this report was to estimate long-term outcome and prognostic factors in children and adolescents with osteosarcoma. A large group of osteosarcoma patients were analyzed at our national oncology center. PROCEDURE: To evaluate the efficacy of surgery and multiagent chemotherapy for treating osteosarcoma, we reviewed 122 cases (65 male, 57 female, mean age 13.8 +/- 3.6 years) treated at the Second Department of Pediatrics in Budapest between 1988 and 2006. Demographic parameters, tumor-related and treatment-related variables, response, overall survival (OS) and event-free survival (EFS) were analyzed. RESULTS: The 5-year OS was 68% and 5-year EFS was 62%. OS of patients without metastasis was 79%, while OS with early metastasis was 17%. Survival of patients with amputation (n = 30) was not significantly different from patients with limb-salvage surgery (n = 82), but all patients without radical surgery died. Gender and histological classification had no prognostic significance. Patients with localized tumors in extremities had increased survival compared to patients with axial skeleton tumors (P = 0.013). Poor histological response to neoadjuvant chemotherapy (rate of survivor tumor cells >10%) was associated with decreased survival (P = 0.018). Patients under 14 years had better EFS than patients over 14 years (P = 0.008). CONCLUSIONS: Our results demonstrate that younger patients with localized osteosarcoma of the extremities who receive limb-salvage surgery and chemotherapy have an excellent survival. Pediatr Blood Cancer 2011; 57: 415-422. (c) 2011 Wiley-Liss, Inc
Obesity and metabolic syndrome in adolescent survivors of standard risk childhood acute lymphoblastic leukemia in Saudi Arabia

This study estimated prevalence of unhealthy weight status and metabolic syndrome (MS) amongst Saudi survivors of standard risk ALL. Procedure. We recruited 56 survivors, mean age 13.4 years (SD 4.1), a mean of 9.1 years (SD 4.1) postdiagnosis. The BMI for age was used to deﬁne weight status relative to national (Saudi) and international (Cole et al., Cole-IOTF, WHO, and CDC) reference data. We measured body composition by dualenergy X-ray absorptiometry (DXA), waist circumference, blood pressure, lipid proﬁle (HDL-C, Triglycerides), fasting glucose and insulin. Results. According to international deﬁnitions based on BMI for age, around half of the sample had unhealthy weight status. All of the approaches based on BMI for age underestimated overfatness, present in 27/51 (53%) of the sample according to DXA. Prevalence of MS was 7.1% (3/42 of those over 9-years old) and 5.4% (3/56) by applying the International Diabetes Federation (IDF) deﬁnition and National Cholesterol Education Program Third Adult Treatment panel Guidelines (NCEP III), respectively. However, MS by the NCEP III deﬁnition was present in 19% of the overweight and obese survivors and 7.1% of the sample had at least two of the components of MS. Conclusion. Unhealthy body weight and overfatness may be common amongst adolescent Saudi survivors of standard risk ALL, though overweight and obesity may be no more common than in the general Saudi adolescent population. Deﬁning weight status using BMI underestimates overfatness. Ideally, body composition and cardiometabolic risk factors should be monitored at late effects clinics. Pediatr Blood Cancer 2012;59: 133–137. 2011 Wiley Periodicals, Inc
The limited screening value of insulin-like growth factor-i as a marker for alterations in body composition in very long-term adult survivors of childhood cancer

Background: The clinical relevance of low IGF-I levels, caused by cranial radiotherapy, in adult childhood cancer survivors has not been studied extensively. We evaluated whether IGF-I is a useful marker for altered body composition and growth hormone deficiency (GHD) in this group. Procedure: We analyzed retrospective data from 610 adult childhood cancer survivors, retrieved from the late effects clinic. Median age at diagnosis was 6 years (interquartile range 3-11) and follow-up time was 18 years (13-24). We assessed IGF-I standard deviation scores (SDS), anthropometrical measures, growth hormone stimulation tests in patients with clinical signs of GHD, and measures of body composition (assessed by dual X-ray absorptiometry, Lunar Prodigy). Results: In 58 cranially irradiated acute leukemia survivors (25 Gy (24-25)) and 56 locally irradiated brain tumor survivors (42 Gy (35-54)) we found significantly lower IGF-I SDS (P<0.001), lower height SDS (P<0.001), higher body mass index (P=0.01), higher waist-hip ratio (WHR; P=0.001), higher total fat percentage SDS (P<0.001), and lower lean body mass SDS (P<0.001), as compared to 452 not cranially irradiated survivors. IGF-I showed a weak inverse correlation with BMI (r=-0.12, P=0.04), WHR (r=-0.15, P=0.01), total fat percentage (r=-0.14, P=0.02), and a positive correlation with lean body mass (r=0.15, P=0.01). In patients with low IGF-I levels, IGF-I did not significantly differ between subjects with and without GHD as determined by GH-stimulation testing (P=0.39). Conclusion: This study shows that IGF-I has limited value as a marker for alterations in body composition in adult childhood cancer survivors. Pediatr Blood Cancer 2012;59:711-716
Scaling up cancer care for children without medical insurance in developing countries: The case of Mexico

Background: In 2006, the Mexican government launched the Fund for Protection Against Catastrophic Expenditures (FPGC) to support financially healthcare of high cost illnesses. This study aimed at answering the question whether FPGC improved coverage for cancer care and to measure survival of FPGC affiliated children with cancer. Procedure A retrospective cohort study (2006–2009) was conducted in 47 public hospitals. Information of children and adolescents with cancer was analyzed. The coverage was estimated in accordance with expected number of incident cases and those registered at FPGC. The survival was analyzed by using Kaplan–Meier survival curves and Cox proportional hazards regression modeling. Results: The study included 3,821 patients. From 2006 to 2009, coverage of new cancer cases increased from 3.3% to 55.3%. Principal diagnoses were acute lymphoblastic leukemia (ALL, 46.4%), central nervous system (CNS) tumors (8.2%), and acute myeloid leukemia (AML, 7.4%). The survival rates at 36 months were ALL (50%), AML (30.5%), Hodgkin lymphoma (74.5%), Non-Hodgkin lymphoma (40.1%), CNS tumors (32.8%), renal tumors (58.4%), bone tumors (33.4%), retinoblastoma (59.2%), and other solid tumors (52.6%). The 3-year overall survival rates varied among the regions; children between the east and south-southeast had the higher risks (hazard ratio 3.0; 95% CI: 2.3–3.9) and 2.4; 95% CI: 2.0–2.8) of death from disease when compared with those from the central region. Conclusion: FPGC has increased coverage of cancer cases. Survival rates were different throughout the country. It is necessary to evaluate the effectiveness of this policy to increase access and identify opportunities to reduce the differences in survival
Different fever definitions and the rate of fever and neutropenia diagnosed in children with cancer: A retrospective two-center cohort study

BACKGROUND: The definition of fever, and thus fever and neutropenia (FN), varies between different pediatric oncology centers. Higher temperature limit should reduce FN rates, but may increase rates of FN with complications by delaying therapy. This study determined if different fever definitions are associated with different FN rates. PROCEDURE: Two pediatric oncology centers had used three fever definitions in 2004-2011: ear temperature ≥38.5°C persisting ≥2 hours (low definition); axillary temperature ≥38.5°C ≥2 hours or ≥39.0°C once (middle); and ear temperature ≥39.0°C once (high). Clinical information was retrospectively extracted from charts. FN rates were compared using mixed Poisson regression. RESULTS: In 521 pediatric patients with cancer, 783 FN were recorded during 6,009 months cumulative chemotherapy exposure time (501 years; rate, 0.13/month [95% CI, 0.12-0.14]), 124 of them with bacteremia (16%; 0.021/month [0.017-0.025]). In univariate analysis, the high versus low fever definition was associated with a lower FN rate (0.10/month [0.08-0.11] vs. 0.15/month [0.13-0.16]; rate ratio, 0.66 [0.45-0.97]; P = 0.036), the middle definition was intermediate (0.13/month [0.11-0.15]). This difference was not confirmed in multivariate analysis (rate ratio, 0.94 [0.67-1.33]; P = 0.74). The high versus low definition was not associated with an increased rate of FN with bacteremia (multivariate rate ratio, 1.39 [0.53-3.62]; P = 0.50). CONCLUSION: A higher fever definition was not associated with a lower FN rate, nor with an increased rate of FN with bacteremia. These may be false negative findings due to methodological limitations. These questions, with their potential impact on health-related quality of life, and on costs, need to be assessed in prospective studies. Pediatr Blood Cancer © 2012 Wiley Periodicals, Inc
Treatment of high risk Sertoli-Leydig cell tumors of the ovary using a gonadotropin releasing hormone (GnRH) analog

Sertoli–Leydig cell tumors are rare ovarian neoplasms. We report
two unusual cases with bilateral SLCTs suggesting evidence of
genetic predisposition and at high risk of recurrence. To reduce this
risk, we exploited the use of GnRH analog to lower gondadotropin
and potentially directly inhibit the tumors through expressed GnRH
receptors. We used it as maintenance antitumor therapy for 2 years after completion of chemotherapy, to cover the period of risk for
recurrence. Both patients remain in complete remission at >2 years
after completing leuprorelin therapy. Of note, both patients carry
DICER1 mutations, frequently found in pleuropulmonary blastoma
syndrome.http://onlinelibrary.wiley.com/journal/10.1002/(ISSN)1545-5017am201
Emergency department visits in children with hemophilia

Background  The pediatric emergency department (ED) management of bleeding and other complications of hemophilia constitutes an increasingly important component of hemophilia therapy. This retrospective study examined the overall ED use by children with hemophilia in a single center, with a particular aim to investigate visits related to injury or bleeding, and those related to blood stream infection in patients with a central venous catheter (CVC).    Methods  Electronic medical records of patients with hemophilia presenting to Children's Hospital of Michigan ED were reviewed. Different categories of ED visits over a 5‐year period (January 2006–December 2010) were examined.    Results  There were 536 ED visits from 84 male patients (median age 4 years, range 0–21) with hemophilia over the 5‐year period. The reasons for ED visits were: injury or bleeding (61.2%); suspected CVC‐related infection (11.8%); causes unrelated to hemophilia (19.2%); and routine clotting factor infusion (7.8%). Eighteen visits from six patients were secondary to injury or bleeding in a patient not yet diagnosed with hemophilia. An intracranial hemorrhage was detected in five visits. Overall, 5.4% of all visits represented distinct episodes of bloodstream infection.    Conclusion  The pediatric ED is an indispensable component of the overall hemophilia care, because: (1) patients with potentially lethal problems such as ICH or CVC‐related infection may present to the ED for their initial management; (2) previously undiagnosed patients with hemophilia may also present to the ED for their first bleeding episodes, initiating the diagnostic investigations; (3) the ED provides after‐hours treatment service for many episodes of injury or bleeding, and also for clotting factor infusion. Pediatr Blood Cancer 2013; 60: 1188–1191. © 2012 Wiley Periodicals, Inc
Children's Oncology Group's 2013 blueprint for research: Nursing discipline

Integration of the nursing discipline within cooperative groups conducting pediatric oncology clinical trials provides unique opportunities to maximize nursing's contribution to clinical care, and to pursue research questions that extend beyond cure of disease to address important gaps in knowledge surrounding the illness experience. Key areas of importance to the advancement of the nursing discipline's scientific knowledge are understanding the effective delivery of patient/family education, and reducing illness‐related distress, both of which are integral to facilitating parental/child coping with the diagnosis and treatment of childhood cancer, and to promoting resilience and well‐being of pediatric oncology patients and their families. Pediatr Blood Cancer 2013; 60: 1031–1036. © 2012 Wiley Periodicals, Inc
Children's Oncology Group's 2013 blueprint for research: Non‐Hodgkin lymphoma

Non‐Hodgkin lymphomas account for approximately 7% of cancers diagnosed in patients less than 20 years of age, with approximately 800 cases diagnosed annually at COG institutions. With current therapies, cure rates range from 70% to over 90%, even for children with disseminated disease. However, two major challenges need to be overcome: (i) to optimize upfront treatment to prevent relapse since prognosis for patients with relapsed disease remains poor and (ii) minimize long‐term side effects in survivors. Hence, the future initiatives for the treatment of pediatric NHL are to utilize novel targeted therapies to not only improve outcomes but to decrease bystander organ toxicities and late effects. Pediatr Blood Cancer 2013; 60: 979–984. © 2012 Wiley Periodicals, Inc
Children's Oncology Group's 2013 blueprint for research: Renal tumors

Renal malignancies are among the most prevalent pediatric cancers. The most common is favorable histology Wilms tumor (FHWT), which has 5‐year overall survival exceeding 90%. Other pediatric renal malignancies, including anaplastic Wilms tumor, clear cell sarcoma, malignant rhabdoid tumor, and renal cell carcinoma, have less favorable outcomes. Recent clinical trials have identified gain of chromosome 1q as a prognostic marker for FHWT. Upcoming studies will evaluate therapy adjustments based on this and other novel biomarkers. For high‐risk renal tumors, new treatment regimens will incorporate biological therapies. A research blueprint, viewed from the perspective of the Children's Oncology Group, is presented. Pediatr Blood Cancer 2013; 60: 994–1000. © 2012 Wiley Periodicals, Inc
Children's Oncology Group's 2013 blueprint for research: Stem cell transplantation

The role of SCT in pediatric oncology has continued to evolve with the introduction of new therapeutic agents and immunological insights into cancer. COG has focused its efforts on the study of hematopoietic stem cell transplantation in the treatment of pediatric malignancies in several major multi‐institutional Phase II and Phase III studies. These studies include addressing the impact of allogenicity in ALL (ASCT0431), and establishing autologous stem cell transplant as the standard of care in neuroblastoma. Reducing transplant‐associated toxicity was addressed in the ASCT0521 study, where the TNFα inhibitor etanercept was tested for the treatment of idiopathic pneumonia syndrome. Impact of cell dose was explored in the single versus tandem umbilical cord blood study CTN‐0501, in close collaboration with the BMT‐CTN. Pediatr Blood Cancer 2013; 60: 1044–1047. © 2012 Wiley Periodicals, Inc
Serum microRNA screening for DICER1-associated pleuropulmonary blastoma

CT chest scanning has been recommended to screen for pleuropulmonary blastoma (PPB) in babies and young children known to harbor germline DICER1 mutations. However, only a minority of these patients will develop PPB, and the use of CT scans is associated with risks such as secondary malignancy. Recently, we identified a panel of microRNAs that were highly abundant in the serum of a patient with a germline DICER1-mutated PPB, but present at normal levels in healthy relatives carrying the same germline mutation. Consequently, we advocate the addition of serum microRNA profiling to this programme of radiological surveillance, in order to establish its clinical utility as a PPB biomarker. If validated, this blood-based screening-tool may reduce our reliance on CT imaging.This is the accepted manuscript. The final version is available at http://onlinelibrary.wiley.com/doi/10.1002/pbc.25209/abstract
Biallelic somatic SMARCA4 mutations in small cell carcinoma of the ovary, hypercalcemic type (SCCOHT).

Small cell carcinoma of the ovary, hypercalcemic type (SCCOHT) is a rare, aggressive tumor that primarily affects young women. SCCOHT has recently been identified as a monogenic disorder caused by germline and/or somatic SMARCA4 mutations. We describe a 15-year-old Caucasian girl with a SCCOHT harboring a previously unreported somatic mutation in the SMARCA4 gene (c.1757delA;p.K586.fs) with loss of heterozygosity. No germline mutation was identified. Subsequent immunohistochemical staining confirmed loss of SMARCA4 protein. These molecular findings will aid with SCCOHT diagnosis through immunohistochemical staining for SMARCA4 and in the future may have implications for the management of this disease.This is the accepted manuscript. The final version is available at http://onlinelibrary.wiley.com/doi/10.1002/pbc.25279/abstract