bluuebunny/crossref_metadata_2025_split huggingface dataset:
Levels of mRNA of iLBP family genes and retinoic acid receptors in embryonal tumors in children.
Authors: Popovyan Olga P. , Kit Oleg I. , Potemkin Dmitrii S. , Timoshkina Natalya N. , Kuznetsov Sergei A. 
Published: May 2020

e22517 Background: Neuro- and nephroblastoma are the most common pediatric solid tumors. Their varying clinical behavior determines challenging prognosis and choice of treatment tactics. Along with many clinical parameters, the genetic characteristics of tumor cells appear promising. Methods: The study included patients diagnosed with neuroblastoma (n = 10) and nephroblastoma (n = 10) (median age from 2 months to 8 years; 10 boys and 10 girls). Total RNA was extracted from paired tumor/normal samples using the miRNeasy Mini Kit (Qiagen, USA) and cDNA libraries obtained with the Reverta-L kit. Expression of the CRABP1, CRABP2, FABP4, FABP5, RAR, RXRA, RXRB, and PPARD genes was evaluated by RT-PCR on the CFX96 amplifier (Bio-Rad, USA). GAPDH and B2M were reference loci. Relative expression (Exp) was calculated by the 2-ΔCt method. Results: The qPCR-RT showed a significant increase in the transcriptional activity in both tumors, compared to normal tissues, for the FABP4, FABP5, and RXRA loci (p < 0.01). Interestingly, we observed multidirectional changes in mRNA levels in neuroblastomas and nephroblastomas for the CRABP1, RAR, and PPARD loci. Unlike solid tumors of adult patients, the expression activity of the CRABP1 gene either did not differ from the conditional norm (in nephroblastoma), or significantly dropped to 70% (in neuroblastoma). In contrast to adult solid tumors, the CRABP1 gene expression activity in children was normal (in nephroblastoma) or decreased significantly up to 70% (in neuroblastoma). Conclusions: The pilot study revealed differentiating aberrant expression of genes encoding the family of retinoic acid transporters and receptors in pediatric neuroblastomas and nephroblastomas. This may be essential for choosing the optimal treatment volume and establishing a prognosis and resistance to chemotherapy in patients with these tumors.
Image-guided core needle biopsy for relapsed and refractory neuroblastoma: A focus on sample adequacy for genetic sequencing.
Authors: Samoyedny Andrew , States Lisa , Alai Emma , Srinivasan Abhay , Maris John M. , Mosse Yael P. , Cahill Anne Marie 
Published: May 2020

e22521 Background: Relapsed and refractory neuroblastoma (NBL) is a prime target for novel therapies targeting specific mutations. The genomic landscape of NBL can change between time of initial diagnosis and relapse, necessitating subsequent biopsy and next generation sequencing (NGS) to detect mutations within the relapsed neoplasm. Tissue of insufficient quality and quantity leads to specimen failure during NGS. Thus the goal of this study is to review core needle biopsy for relapsed and refractory NBL in a pediatric interventional radiology department and explore factors that enhance biopsy adequacy. Methods: Retrospective review of clinical records and images for 66 patients (36M, 30F) with median age 8.7 years (range 0.9 – 49.3 y) who underwent 95 biopsies (55 bone, 40 soft tissue) over a 12-year period. Results: Biopsy yield for neuroblastic tissue from 123I mIBG-avid masses was 89.7% overall, 84.9% in bone, and 91.6% in soft tissue. 87/95 masses were mIBG-avid; 2 non-avid masses were NBL-positive (but also 18F-FDG avid). 48/59 samples sent for NGS were adequate for analysis (81.4%); bone and soft tissue biopsies did not differ in adequacy (77.1% vs 87.5%, p = 0.32). Tumor cell percent (TC%) for adequate samples was 49.9% vs 11.1% for NGS failures (p = 0.0003). Number of needle passes performed in bone and soft tissue lesions was positively correlated with NGS adequacy (Bone: r2= 0.68, Soft: r2= 0.33). In bone and soft tissue, adequate biopsies tended towards higher mean needle passes than inadequate ones (Bone: 5.1 vs 3.4, p = 0.0582) (Soft: 10.5 vs 4.7, p = 0.0428). Total needle volume passed into a soft tissue mass (calculated as inner needle cylindrical volume * # of passes) was higher in the “adequate” vs “inadequate” group (0.20 cm3 vs 0.05 cm3, p = 0.0004). Lesion mIBG avidity was quantified with a ratio of lesion:liver SUV; this mIBG SUV ratio was positively correlated with TC% (R2 = 0.55, N = 14). Using a linear regression line, a TC% of 50% (the mean value for samples adequate for NGS) corresponded to an SUV ratio of 2.89. The rate of minor complications (requiring only nominal therapy) was 7/95 (7.4%). The rate of major complications (requiring longer hospital stay) was 1/95 (1.5%). Conclusions: Image-guided core needle biopsy for relapsed and refractory NBL has a moderately high yield for procuring tumor samples adequate for NGS with a low rate of major complications. Adequate bone and soft tissue biopsies averaged >5 and >10 needle passes, respectively. An approximate lesion:liver mIBG SUV ratio of >3 may maximize specimen quality for NGS.
“Being healthy and living life as if I never had cancer”: The meaning of “living well” from adolescents with cancer.
Authors: Schreiner Katherine , Grossoehme Daniel H. , Baker Justin N. , Needle Jennifer , Friebert Sarah , Lyon Maureen E 
Published: May 2020

e22532 Background: Extensive resources are devoted to discovering novel cancer treatments. Meanwhile, patient priorities and experiences must also be addressed. Specifically, among adolescents with cancer, patients’ definitions of “living well” may elucidate treatment preferences, guide care teams and families, and influence future behavioral interventions. The objective was to develop an empirical definition of “living well” for adolescents with cancer to enhance shared decision-making. Methods: Video recordings were analyzed from the Next Steps: Respecting Choices interviews with n = 30 adolescents ages 14-21 years with cancer and their families, a subsample of N = 126 adolescent/family dyads participating in a randomized clinical trial, FAmily CEntered (FACE) Advance Care Planning. Interviews were transcribed, verified and anonymized. A phenomenological analytic method was used to identify psychological meaning in participants’ statements. Results: Adolescents with cancer conceptualize living well as meaning maintaining physical, mental and emotional health, while engaging in purposeful, typical, adolescent-appropriate activities with people important to the adolescents. This has four components: living mindfully; living an identity as a healthy adolescent; spending time with friends and family; and living a purposeful life without depression. Living well means most often enjoying these activities while spending time with family and friends. An exemplary quotation is, “...being able to have a life where everything is going good, and being able to live healthy...gets rid of some of your worries not only for yourself but for your friends and family...less worries means a less stressful life...” Conclusions: By determining what “living well” means to adolescents with cancer, care teams and families can more easily understand patients’ priorities. A firm grasp on patients’ definitions of “living well” could relieve significant burden from families and increase families’ willingness to honor adolescent treatment preferences. Findings may guide future psychosocial interventions. Clinical trial information: NCT01670461.
Implementation of psychosocial support project in a pediatric oncology camping program.
Authors: Mozzilli Simone Lehwess , Salvetti Marina , Manhaes Maria Fernanda , Szylit Regina , Freitas Mariana , Garcia Fernanda Rafaela 
Published: May 2020

e22534 Background: The oncological treatment can generate psychosocial distress for patients and their families. The Beaba Camping aims to educate and empower patients and caregivers through Technology Information and Gamification. In August 2019, in the third edition of the project, a pilot of Psychosocial Support Program was implemented to provide an integrative care to 71 children and adolescents under cancer treatment and 68 caregivers. Methods: Five psychologists, specialized in paediatric psycho oncology and palliative care, were selected to structure the Psychosocial Support Program and conduct interventions during three days of the camping. Based on evidences and experiences, the psychologists divided the interventions in: 1. Individually - children and adolescents. 2. Group - caregivers: 2.1. Belonging: identification and recognition of peers. Identification of psychological distress and similar fears; 2.2. Psychological Group Intervention: creation of groups to facilitate the intervention and bonding; 2.3. Feeling of the Day: expression of which emotion was the most prevalent each day of camping; 2.4. A Word of Support: exchange supportive statements among the peers. Results: 20 children/adolescents and 19 caregivers were attended individually, with demands variating from: Anxiety and sadness; Fear of future and prognosis; Social isolation and adaptation. 68 adults participated in groups that brought discussions involving: Biggest fears and difficult moments of the treatment; Relationship with their children and adolescents; Coping strategies and spirituality. Conclusions: The Psychosocial Support is an important and essential part of the cancer treatment. The Camping with Psychosocial Support Program may enable the demystification of the role of the psychologist, the link between professionals and patients and their caregivers, the binding among peers and the education about how the psychosocial support can be helpful to everyone involved in the treatment. For future improvements, more psychologists will participate in order to attend a larger number of participants and to perform more group interventions, including with children and adolescents.
Risk factors associated to infectious complications in children with febrile neutropenia induced by chemotherapy: A cohort study.
Authors: Díaz Ramírez Karla , Ortiz Morales Daniel , Zapata Tarres Marta 
Published: May 2020

e22535 Background: Children cancer is the second cause of mortality, most of deaths are related to infectious complications due to immunosuppression provoked by chemotherapy. Febrile neutropenia is one of the most common issues in oncological patients and it’s considered an emergency. Studies have tried risk predicting models implementation, although they still need to be validated, updated and reproducible in different scenarios. The aim of this study was the early identification of conditioning factors to severe infectious complications in patients with febrile neutropenia that would allow taking decisions for diagnosis and treatment. Methods: We present a prospective cohort study that estimated the risk associated to clinical and paraclinical factors and the development of infectious complications in pediatric patients with febrile neutropenia induced by chemotherapy on their admission of an emergency room (ER). The clinical features analyzed were: age, type of cancer, chemotherapy regime, time between chemotherapy administration and ER admission, use of colony-stimulating factors, presence of central venous catheter, highest temperature registered, tachycardia, polypnea, hypoxemia and hypotension. The paraclinical features included were leucocyte, neutrophil and monocyte count, and C-reactive protein levels. Results: From 186 febrile neutropenia events, 101 cases reported infectious complications (54%). Clinical infection was the most frequent issue (38%). On the multivariate analysis we found that the features independently associated to infectious complication were tachycardia (OR1.07, CI95% 1.70-6.99) and highest temperature level reported in the ER (OR1.07, CI95% 1.00-1.13). Conclusions: Febrile neutropenia is the most common complication in pediatric oncological patients and it is related to life-threatening infections and high mortality rate. Risk prediction during the initial assessment in the ER is important to stratify patients and offer target therapy to decrease complications and prolonged hospitalization.
Functional and oncologic outcomes after excision of proximal femur primary bone tumors using a cost-effective indigenous prosthesis.
Authors: Gulia Ashish , Harsha Sri , Gupta Srinath , Puri Ajay 
Published: May 2020

e23500 Background: Proximal femur endoprosthetic replacement(PFER) has become aprimary treatment option for aggressive benign and malignant tumors of proximal femur allowing early return to weight bearing and improvement in functional status. We present our experience in these cases with respect to oncological outcomes, functional outcomes and implant survival. Methods: All cases were retrieved from our prospectively maintained surgical database. 98 patients(35 males and 63 females) with a mean age of 29 years(10– 69 years)were operated between August 2006 and September 2017. Median followup was 49 months(1 - 160 months).The diagnosis included osteosarcoma(18), Ewing’s sarcoma(38), chondrosarcoma(32), and GCT(8) synovial sarcoma (2) .They were evaluated by Musculoskeletal Tumor Society score, recurrence free survival, patient survival, and implant survival. Results: Mean resection length is 18.5cm (8 – 35cm). 8 patients underwent revision surgery,7 for mechanical (3 for aseptic loosening, 4 for dislocations and for implant failures), 1 for non mechanicalcause (infection) and 2 patients underwent amputation for local recurrence. One patient had dislocation recently for whom revision is adbvised. At last follow-up, out of 98 cases 6 were lost to follow up, 32 patients had died(28 due to disease and 4 due to other causes) and 60 are alive. There were 3 only local recurrences(3 alive-1 external hemipelvectomy, 1 excision, 1 definitive chemo radiation), 9 had both local and distant recurrence (1 alive – hip disarticulation for local, definitive cheomoradiation for distant recurrence), and 26 had distant recurrences(6 alive - 3 pulmonary metastetectomy,1 nodal metastasis, 1 lung metastasis and 1 bone metastasis treated with definitive cheomoradiation) . The 5year OS, LRFS,DRFS were 68.3,85.7,61.5% respectively. The mean Musculoskeletal Tumor Society score was 26(22 – 28). The implant survival was 87.4% at 5 years. Conclusions: The use of a PFER in the treatment of primary tumours of bone is oncologically safe and gives consistent and predictable results with low rates of complication. We found that low cost indigenous prosthesis (one fourth the cost of imported prosthesis) fare equivalent to existing imported prosthesis. This can immensely helpful in offering limb salvage in resource constraint settings.
The outcome of vincristine, dactinomycin D, ifosfamide and doxorubicin (VAIA) as first-line therapy for adult-patients with metastatic ewing sarcoma; a single-center experience.
Authors: Atallah Jean Paul , Elshenawy Mahmoud Abdelsatar , Badran Ahmed ali , Alata Maaz Kamal , Gad Ahmed , Alharbi Abdulrahaman H. , Alquaydheb Hisham N. , Alshamsan Bader I 
Published: May 2020

e23501 Background: Ewing sarcoma family of tumors (ESFT) is a rare malignancy among adults. Most studies from western countries have reported improvement in outcomes following multi-agent chemotherapy. We report our experience in the management of this disease among Arab ethnicity. The aim of this study is to assess the outcome of VAIA combination as a first-line treatment in Adult-patients with metastatic Ewing sarcoma. Methods: Patients who were newly diagnosed as metastatic Ewing sarcoma between 03/1997 and 11/2016 at King Faisal Specialist Hospital and Research Center, who received VAIA as first-line therapy were eligible. The patient's characteristics were summarized using Medians with interquartile ranges (IQR) and frequencies for continuous and categorical variables, respectively. Variables including age, sex, primary tumor size, site (skeletal vs extraskeletal) and extent of metastasis in correlation with progression and overall survival were analyzed using the Kaplan–Meier method and Cox proportional hazards regression. Results: Thirty-nine patients were identified. Male (26, 66.7%), Female (13, 33.3%). Skeletal (27, 69.2%), Extraskeletal (12, 30.8%). The median longest diameter of the primary tumor 9.75 (IQR 8-15). The most common metastatic sites were Lungs (22, 56.4%) & Bone (10, 25.6%), however, the least common sites were Bone Marrow (3, 7.7%) and liver (2, 5.1%). The median number of VAIA cycles was 10 cycles (IQR 5-14). Objective response rate (ORR) was noticed in 20 patients (51.2%) (Complete Remission (7, 17.9%), Partial Remission (13, 33.3%). One patient had stable disease and 12 (30.8%) patients had progressive disease. The assessment was not feasible in 3 (7.7%) patients. With a median follow up duration of 18 months (1-44).20 patients died (62.5%). The median PFS and OS was 10,18 months respectively with 3,5 years overall survival rate of;35.7%,26.9% respectively. Univariate analysis correlation among different variables were insignificant. Conclusions: VAIA chemotherapy combination showed poor outcomes among our patients in comparison to literature further improvement is needed in this aggressive malignancy in our region.
The treatment results in patients with Ewing Sarcoma: The Polish Sarcoma Group Experience.
Authors: Lugowska Iwona , Jagodzinska-Mucha Paulina , Raciborska Anna , Bilska Katarzyna , Kosela-Paterczyk Hanna , Kozak Katarzyna , Dawidowska Anna , Poleszczuk Jan , Rutkowski Piotr 
Published: May 2020

e23503 Background: Ewing Sarcoma is a rare type of aggressive tumor that occurs in bones or in the soft tissue around the bones. The multidisciplinary approach chemotherapy (CHT), radiotherapy (RTH) and surgery (S) is the standard of care. The aim of our study was to analyze prognostic factors and treatment results in patients with Ewing Sarcoma. Methods: 504 patient with Ewing sarcoma treated between 1998 and 2019, F:M 1:1.34, with metastases at presentation was 195 patients (39%), axial localization was in 277 patients (55%). All patient were treated with multimodal treatment based on chemotherapy, radiotherapy and surgery. Kaplan-Meier estimator, log rank test and multivariate Cox model were used for statistical analysis. Results: Five year overall survival (5y-OS) was 54% (CI:49-60%), prognostic factors were: gender (5y-OS for females and males equal to 63% and 48%, respectively), age (5y-OS stratified patients between < 10y; 10-15y; 15-25 and 25+ was 65%, 70%, 41% and 55%;p < 0.001), metastases (5y-OS in group with M1 was 31% and in M0 - 70%; p < 0.001), type of local treatment (5y-OS in group CHT/S/RTH was 61%, CHT/S 59%, only RTH 50% and palliative treatment without local treatment equals 12%; p < 0.001). Multivariate Cox analysis revealed death hazard dependence on gender (male vs. female; HR = 1.80; p < 0.01), metastatic status (M1 vs. M0; HR = 2.91; p < 0.001), bone/bone marrow involvement (present vs. absent; HR = 2.10; p < 0.01) and chemotherapy regime (other vs. VIDE/VAI; HR = 0.69; p = 0.049). Conclusions: The treatment results in Ewing sarcoma are significantly better in children than in adults, which can be related to the more intensive chemotherapy regiments applied. The treatment should be provided in referral centers and based on up-to-dated guideline.
Updated 5-year local control (LC), metastasis-free survival (MFS), and overall survival (OS) data from a phase I study of nilotinib plus radiation (RT) in high-risk chordoma.
Authors: Cote Gregory Michael , DeLaney Thomas F. , Miao Ruoyu , Schwab Joseph H. , Raskin Kevin , Lozano Calderón Santiago , Mullen John Thomas , Haynes Alex Bernard , Hornicek Francis J. , Chen Yen-Lin , Choy Edwin 
Published: May 2020

e23505 Background: Chordomas are malignant tumors arising from remnant notochordal tissue. Despite improved local control with preoperative/postoperative RT, progression-free survival and OS remain poor in patients (pts) with high-risk features. Chordoma has been identified to express and activate platelet-derived growth factor receptor signaling. We conducted a phase 1 trial to identify the maximum tolerated dose (MTD), safety, and feasibility of nilotinib with RT as either preoperative or definitive treatment for patients with high-risk chordoma. Enclosed is an updated report on LC, MFS, and OS. Methods: We recruited 23 pts with nonmetastatic chordoma to the phase I and dose expansion arms of the study. High-risk was defined as the presence of any of the following: local recurrence after surgery, previous intralesional resection, unplanned incomplete resection, unresectable/marginally resectable disease, or declining surgery due to excessive morbidity. Pts were treated with nilotinib and concurrent RT to 50.4 Gy relative biological effectiveness (RBE) followed by surgery and postoperative RT to a cumulative dose up to 70.2 Gy RBE or definitively up to 77.4 Gy RBE without surgery. On completion of RT, pts were eligible to continue nilotinib until disease progression. Results: The dose escalation arm of the study identified nilotinib 200 mg daily as the maximum tolerated dose (MTD). Eighteen evaluable pts were treated with nilotinib plus RT at the MTD. The objective best response rate was 6% (1 of 18 pts). The 4 and 5-year LC rates were 94.3% (95% CI 83.2-100) and 70.7% (95% CI 20.8-100), respectively. The MFS rate was 74.3 at 4 and 5 years (95% CI 51.8-96.7). The 4 and 5-year OS rates were 70.2% (95% CI 44.4-95.9) and 54.6% (95% CI 20.5-88.6). Conclusions: In updated data from a cohort of high-risk chordoma pts nilotinib 200 mg/d with RT +/- surgery, with long-term follow-up, local control and distant metastasis free survival remains favorable. Clinical trial information: NCT01407198 . [Table: see text]
Mesenchymal chondrosarcoma: An Australian multi-centre cohort study.
Authors: Strach Madeleine Cornelia , Grimison Peter S. , Boyle Richard , Stalley Paul , Connolly Elizabeth Anne , Bhadri Vivek 
Published: May 2020

e23509 Background: Mesenchymal chondrosarcoma (MCS) is a rare sarcoma that has a more aggressive course than conventional chondrosarcoma. Treatment is often multimodal with combinations of surgery, chemotherapy and radiotherapy. This study gives opportunity to evaluate prognostic factors and treatments in an Australian setting. Methods: We performed a retrospective study on patients with MCS registered on the ACCORD prospective sarcoma database from 6 Australian sarcoma centres since 2009. We collected data including baseline demographics, clinicopathological variables, treatment characteristics and survival status. We assessed outcomes of overall survival (OS), progression-free survival (PFS) and proportions of metastatic and localised patients receiving surgery, chemotherapy and/or radiotherapy. Results: We identified 20 patients diagnosed with MCS between Jan 2001 and Dec 2019. Median age was 35 years (range 20-69 years). Two (10%) had metastatic disease at diagnosis and 7 (35%) developed metastases on follow up. Median follow-up was 20 months (0.4 – 210 months). 17 (85%) patients underwent surgical resection (17 of primary, 8 of recurrence/metastasis), 10 (50%) received chemotherapy (95% anthracycline; 1 preoperative, 9 palliative), 12 (60%) received radiotherapy (7 to primary, 9 to recurrence/metastasis), and 13 (65%) received multimodality therapy. All six patients who received surgery alone remained alive at follow-up with no metastatic disease. Table shows survival estimates for the study population. There was no difference in median OS in patients who received all therapies compared to those who did not (8.4 v 7.5 years; p = 0.53). Median PFS was not prolonged in those who received all therapies compared to those who did not (37 vs 43 months, p = 0.4). Conclusions: This study demonstrates contemporary Australian treatment patterns of MCS. Understanding treatment patterns and outcomes helps promote standardisation of treatment and design of prospective trials for novel therapeutic strategies. Future analysis of prospective patients for patient and treatment factors influencing survival is planned. [Table: see text]