bluuebunny/crossref_metadata_2025_split huggingface dataset:
Differences in activities of daily living performance between long‐term pediatric sarcoma survivors and a matched comparison group on standardized testing
Authors: Parks Rebecca , Rasch Elizabeth K. , Mansky Patrick J. , Oakley Frances 
Published: July 2009

AbstractBackgroundIn a cross‐sectional study examining late effects of pediatric sarcoma therapy, long‐term survivors were evaluated on their activities of daily living (ADL) performance.ProcedureThirty‐two persons with Ewing sarcoma family of tumors, rhabdomyosarcoma, and non‐rhabdomysarcoma‐soft tissue sarcoma enrolled an average of 17 years after treatment. Participants were evaluated using the Assessment of Motor and Process Skills (AMPS) 1, a standardized observational evaluation of ADL task performance. Means and 95% confidence intervals for ADL motor and ADL process ability measures were calculated for four groups: (1) sarcoma survivors, (2) “well” adults matched for age and gender, (3) “well” adults matched for gender that were 10 years older, and (4) “well” adults matched for gender that were 20 years older.ResultsADL motor ability was significantly lower for sarcoma survivors than for the age‐ and gender‐matched comparison group (P < 0.05). There was no significant difference between ADL motor ability of sarcoma survivors and the comparison group 10 years older, but sarcoma survivors had significantly better ADL motor ability (P < 0.05) than the oldest comparison group (20 years older). Sarcoma survivors had significantly worse ADL process ability than the age‐matched group (P < 0.05). There was no difference in ADL process ability between the sarcoma survivors and comparison groups that were 10 and 20 years older.ConclusionsThis first report of a clinical evaluation of ADL limitation in pediatric sarcoma survivors treated with intensive multimodal cancer therapy suggests that influences on performance of daily life activities are more common than previously reported. Pediatr Blood Cancer 2009;53:622–628. Published 2009 Wiley‐Liss, Inc.
Prevalence of abnormal bone density of pediatric patients prior to blood or marrow transplant
Authors: Klopfenstein Kathryn J. , Clayton Julie , Rosselet Robin , Kerlin Bryce , Termuhlen Amanda , Gross Thomas 
Published: July 2009

AbstractOsteoporosis and osteopenia are long‐term side effects of bone marrow transplant (BMT). The purpose of this study was to determine the prevalence of bone mineral density (BMD) abnormalities in pediatric patients prior to BMT. Forty‐four pediatric patients were evaluated with DEXA scans. The average Z‐score was −0.37. Thirty‐six percent had abnormal BMD. Sixty‐seven percent of ALL patients had abnormal BMD. Patients with non‐malignant diseases were significantly more likely to have abnormal BMD. Patients with ALL had more defects than solid tumor patients. Females had more defects than males. These results demonstrate BMD defects are common in children prior to BMT, especially in patients with ALL. Pediatr Blood Cancer 2009;53:675–677. © 2009 Wiley‐Liss, Inc.
Clinicopathological characteristics of ganglioneuroma and ganglioneuroblastoma: A report from the CCG and COG
Authors: Okamatsu Chizuko , London Wendy B. , Naranjo Arlene , Hogarty Michael D. , Gastier‐Foster Julie M. , Look A. Thomas , LaQuaglia Michael , Maris John M. , Cohn Susan L. , Matthay Katherine K. , Seeger Robert C. , Saji Tsutomu , Shimada Hiroyuki 
Published: July 2009

AbstractBackgroundThe International Neuroblastoma Pathology Classification (INPC) was the first to clearly define prognostic subgroups in ganglioneuroma (GN) and ganglioneuroblastoma (GNB).ProcedureHistopathology and tumor resectability of 552 GN/GNB cases from the Children's Cancer Group (CCG) and Children's Oncology Group (COG) neuroblastoma studies were reviewed. The results were analyzed along with clinical information and biological data of the cases.ResultsAccording to the INPC, 300 tumors were classified into the Favorable Histology (FH) group and 252 were into the Unfavorable Histology (UH) group. Tumors in the FH group included 43 ganglioneuroma‐maturing (GN‐M), 198 ganglioneuroblastoma‐intermixed (GNB‐I), and 59 ganglioneuroblastoma‐nodular, favorable subset (GNB‐N‐FS), and were often (91%) resected completely by single or multiple surgical procedures. Patients with the FH tumors had an excellent prognosis with no tumor‐related deaths. The UH group included ganglioneuroblastoma‐nodular, unfavorable subset (GNB‐N‐US) tumors. Patients with the UH tumors had a high incidence (53%) of distant metastasis at the time of diagnosis, and their prognosis significantly depended on clinical stage (5‐year EFS: 80.1% for non‐stage 4 patients; 16.7% for stage 4 patients): Complete primary tumor resection was not beneficial to those GNB‐N‐US patients, regardless of whether metastasis was present or not. MYCN amplification was detected in four tumors in the FH group and six tumors in the UH group. The majority (160/191, 84%) of GN‐M and GNB‐I tumors had a diploid pattern determined by flow cytometry.ConclusionsStringent application of the INPC along with clinical staging was critical for prognostic evaluation of the patients with this group of tumors. Pediatr Blood Cancer 2009;53:563–569. © 2009 Wiley‐Liss, Inc.
Premature carotid artery disease in pediatric cancer survivors treated with neck irradiation
Authors: Meeske Kathleen A. , Siegel Stuart E. , Gilsanz Vicente , Bernstein Leslie , Nelson Mary B. , Sposto Richard , Weaver Fred A. , Lavey Robert S. , Mack M.P.H. Wendy J. , Nelson Marvin D. 
Published: July 2009

AbstractBackgroundWhile carotid artery disease and strokes have been documented in adult cancer patients treated with neck irradiation, little information is available on pediatric patients. The purpose of this study is to determine if carotid disease is more prevalent among pediatric cancer survivors treated with neck irradiation than among healthy controls.ProcedureThirty pediatric cancer survivors who received neck irradiation (2,000–6,660 cGy) and 30 healthy subjects underwent bilateral carotid ultrasounds. Study outcome measures were common carotid intima‐media thickness (IMT) and plaque (present or absent). Multivariate methods were used to compare cases and controls and to identify risk factors related to carotid disease in childhood cancer survivors.ResultsIMT was greater for cancer survivors than controls (0.46 mm (SD 0.12) vs. 0.41 mm (SD 0.06), P < 0.001). Plaque was present in 18% of irradiated vessels and 2% of non‐irradiated vessels (P < 0.01). Among cancer survivors, IMT was positively associated with female gender (P < 0.05), non‐white ethnicity (P < 0.01), positive family history of stroke/heart attack (P < 0.05), BMI (P < 0.001), total cholesterol (P < 0.01), cancer relapse (P < 0.001), and years off treatment (P < 0.0001). Plaque was positively associated with relapse (P < 0.05) and C‐reactive protein (P < 0.01). There was no significant relationship between radiation dose at levels ≥2,000 cGy and IMT or plaque.ConclusionsCarotid artery disease was more prevalent among cancer survivors treated with neck irradiation than among controls. Due to the high risk of stroke associated with advanced carotid disease, larger prospective studies are needed to better define disease risk in these long‐term survivors. Pediatr Blood Cancer 2009;53:615–621. © 2009 Wiley‐Liss, Inc.
Pediatric‐onset mastocytosis: A long term clinical follow‐up and correlation with bone marrow histopathology
Authors: Uzzaman Ashraf , Maric Irina , Noel Pierre , Kettelhut Brett V. , Metcalfe Dean D. , Carter Melody C. 
Published: July 2009

AbstractBackgroundPediatric onset mastocytosis usually presents as urticaria pigmentosa; and less often as diffuse cutaneous mastocytosis. While the literature indicates that disease often resolves, there has been a move to more aggressive therapy for mastocytosis early in life. We addressed the long term prognosis of pediatric‐onset disease by examining 17 children with mastocytosis which we had reported on in 1989 [1].ProcedureWe successfully contacted 15 of these patients and data was collected regarding their clinical status. Original bone marrow specimens were re‐stained, re‐examined, and correlated with disease outcome using consensus criteria. Three of five patients with persistent disease underwent repeat bone marrow biopsies.ResultsThere was complete regression of disease as defined by cutaneous findings and symptoms (clinical disease severity) in 10 of 15 patients (67%). Three patients had major (20%) and two had partial regression of disease (13%). Repeat marrow examinations on three patients with persistent disease documented systemic mastocytosis based on marrow findings in one patient who had partial regression of disease and was the only patient with initial morphologic evidence of systemic disease. Of the remaining two patients, one demonstrated partial regression and the other major regression of disease; and neither had evidence of systemic mastocytosis.ConclusionThis study demonstrates that initial bone marrow biopsies were prognostic in that those without evidence of systemic disease experienced disease regression; and that the long term prognosis for children managed symptomatically with mastocytosis is highly encouraging. Pediatr Blood Cancer 2009;53:629–634. © 2009 Wiley‐Liss, Inc.
Cost of childhood acute lymphoblastic leukemia care in Shanghai, China
Authors: Liu Yin , Chen Jing , Tang JingYan , Ni Shengxing , Xue Huiliang , Pan Ci 
Published: July 2009

AbstractBackgroundAcute lymphoblastic leukemia (ALL) is the most common and curable malignant pediatric disease in children. In low‐ and middle‐income countries, however, economic factors prevent many patients from receiving complete treatment, even as government and insurance entities lack complete data on the costs for ALL therapies. Here, we analyzed the overall costs for pediatric ALL therapies and their constitutive elements.ProcedureAll cost information was drawn from our hospital database. We analyzed the costs of 45 newly diagnosed children with ALL from May 2005 to June 2006; 20 were classified into low‐risk group, 25 into medium‐risk group, and all had completed the ALL‐2005 protocol treatment. Forty cases were B‐lineage, four were T‐lineage, and one was double‐lymphoid lineage.ResultsAverage total clinic expense per person was US $3,694.58 ± 1,376.85. Average total in‐hospital expense per patient was US $7,299.48 ± 6,526.39, with a large individual difference. Medicine and laboratory test costs represented 40.5% and 21.5%, respectively. Patients stayed at home or attended clinic for more than 90% of the therapy period. In‐hospital and clinic constituted 66.4% and 33.6% of costs, respectively. Costs among risk groups and with/without severe complications differed significantly (P < 0.05). Thirty‐month event‐free survivals were 94.14% and 82.81% for the low‐ and medium‐risk groups, respectively.ConclusionRisk group and severe complications were main factors affecting total medical costs. Average overall costs for childhood ALL in this study were less than US $11,000, with reasonable clinical results. Pediatr Blood Cancer 2009;53:557–562. © 2009 Wiley‐Liss, Inc.
MDM2 SNP309 genotype influences survival of metastatic but not of localized neuroblastoma
Authors: Perfumo Chiara , Parodi Stefano , Mazzocco Katia , Defferrari Raffaella , Inga Alberto , Scarrà Giovanna Bianchi , Ghiorzo Paola , Haupt Riccardo , Tonini Gian Paolo , Fronza Gilberto 
Published: July 2009

AbstractBackgroundMDM2 is a major negative regulator of p53 function and is directly regulated by MYCN in neuroblastoma (NB) cells. MDM2 SNP309, a T‐to‐G substitution in the MDM2 promoter associated with higher gene expression compared to wild‐type, may attenuate the p53 pathway in NB, in which p53 mutations are rare. We investigated its impact on NB development and survival in relation with major clinical and biological characteristics.ProcedureA consecutive cohort of 497 NB children, diagnosed in Italy between 1995 and 2005, and a healthy control population of 471 adults were genotyped for MDM2 SNP309. NB patients were followed up until June 30, 2008.ResultsPatients and controls showed similar distribution of MDM2 SNP309 genotypes. In patients, the polymorphism was not associated with any characteristic at diagnosis. In localized stages no effect of the polymorphism on survival was evident. In stage 4 patients overall survival (OS), event free survival (EFS) and survival after relapse (SAR) were significantly poorer for TG/GG than for TT patients (P = 0.008; P = 0.013; P = 0.046, respectively). In this group, such an effect was more evident in patients with MYCN amplification (OS: P < 0.001; EFS: P = 0.028; SAR: P < 0.001).ConclusionsWhile MDM2 SNP309 status does not affect the risk of developing NB nor disease outcome for localized cancer cases, it significantly correlates with survival in stage 4 NB patients, particularly in the presence of MYCN amplification. The impact of small molecule inhibitors of MDM2 activity in the management of such patients could be usefully considered. Pediatr Blood Cancer 2009;53:576–583. © 2009 Wiley‐Liss, Inc.
Phase II evaluation of intravenous vinorelbine (Navelbine) in recurrent or refractory pediatric malignancies: A Children's Oncology Group study
Authors: Kuttesch John F. , Krailo Mark D. , Madden Timothy , Johansen Mary , Bleyer Archie 
Published: July 2009

AbstractBackgroundA Phase II trial was developed to determine the efficacy and toxicity of intravenous vinorelbine, a semi‐synthetic vinca alkaloid, in children, adolescent, and young adults with recurrent or refractory solid malignancies.ProceduresFifty patients were enrolled among three strata: soft tissue sarcomas [rhabdomyosarcoma (RMS), non‐rhabdomyosarcoma, primitive neuroepithelial tumor] (20 patients); brain tumors [astrocytoma (4 patients), medulloblastoma (2 patients), other (16 patients)] (22 patients); neuroblastoma (8 patients). Vinorelbine was given weekly for 6 consecutive weeks during an 8‐week interval. The response rate and toxicity profile was assessed.ResultsAmong the first 35 patients treated at 33.75 mg/m2/dose, 25 experienced grades 3–4 neutropenia (75%). The dose was decreased to 30 mg/m2/dose in the remaining 15 patients. The median age was 10 years (range, 1–25). Four responses (one complete, three partial) occurred within the soft tissue sarcoma strata (all with RMS) and two occurred in the brain tumor group (medulloblastoma and astrocytoma). The most common toxicities were hematological and neurological.ConclusionVinorelbine at dose of 30 mg/m2 can be safely administered to children with recurrent or refractory solid malignancies. The study design identified vinorelbine to be active in the sarcoma category, with a response rate of 36% (4/11) among RMS patients. Pediatr Blood Cancer 2009;53:590–593. © 2009 Wiley‐Liss, Inc.
Growth patterns in children with sickle cell anemia during puberty
Authors: Rhodes Melissa , Akohoue Sylvie A. , Shankar Sadhna M. , Fleming Irma , Qi An Angel , Yu Chung , Acra Sari , Buchowski Maciej S. 
Published: July 2009

AbstractBackgroundPrevious studies of children with homozygous sickle cell anemia (SCA) show impaired growth and maturation. The correlation of this suboptimal growth with metabolic and hematological factors during puberty is poorly understood.ProcedureWe studied a group of pre‐adolescent children with SCA (19 males, 14 females) and healthy controls (16 males, 15 females) matched for race, sex, body size, and pubertal development. Height, weight, body mass index (BMI), and body composition changes were longitudinally assessed over a 2‐year period and compared between the groups and with Z scores based on US growth charts. These changes were correlated with hemoglobin (Hgb) concentration and with energy expenditure (EE) measured using indirect whole‐room calorimetry.ResultsChildren with SCA progressed through puberty slower than control children. While, after 2 years, pubertal males with SCA were shorter, their annual increases in weight were not different from controls. The mean fat free mass (FFM) increments were significantly less in males and females with SCA than in control children. In males with SCA, growth in height declined over time and was significantly slower than in matched controls (P < 0.05).ConclusionGrowth delays were present during puberty in children with SCA. Decreased growth velocity in children with SCA was independently associated with decreased Hgb concentration and increased total EE. Pediatr Blood Cancer 2009;53:635–641. © 2009 Wiley‐Liss, Inc.
Preoperative chemotherapy for patients with Wilms tumor in Malawi is feasible and efficacious
Authors: Israëls Trijn , Molyneux Elizabeth M. , Caron Huib N. , Jamali Monica , Banda Kondwani , Bras Hans , Kamiza Steve , Borgstein Eric , de Kraker Jan 
Published: July 2009

AbstractBackgroundWilms tumor has a survival rate of 85–90% in well resourced countries but in low income countries survival is lower. Malawi is a country with very limited resources. We studied the feasibility, toxicity and efficacy of preoperative chemotherapy for Wilms tumor in Malawian children.MethodsAll patients diagnosed with a Wilms tumor, admitted in Blantyre, Malawi, from 2006 to 2008, were included. These patients received SIOP‐based preoperative chemotherapy followed by surgery and risk‐stratified post‐operative chemotherapy. Social support and counseling were provided to prevent abandonment of treatment.ResultsTwenty patients were included. Mean tumor volume at diagnosis was 2,500 ml and eight patients (40%) had metastases. Ninety‐five percent of patients presented with hypertension, 80% with microscopic hematuria and 60% with a raised platelet count. Preoperative chemotherapy resulted in >50% tumor reduction in 55% of patients with localized disease and 75% of patients with metastatic disease. During preoperative chemotherapy, 11 of 18 patients experienced ≥ grade 3 anaemia, 7 patients experienced ≥ grade 3 neutropenia. In 12 patients the tumor was resected. Reasons of treatment failure were: abandonment of treatment (N = 3), death during anaesthesia induction (N = 1), inoperability (N = 5, due to metastatic disease in N = 4) and relapse (N = 2). One patient died of malaria 2 months after completion of chemotherapy. Eight patients (40%) are alive with a median follow up of 8 months (range 0–1.5 years).ConclusionPreoperative chemotherapy for Wilms tumor is feasible, tolerated and efficacious in Malawi. Continued efforts are needed to encourage early presentation and to prevent abandonment. Pediatr Blood Cancer 2009;53:584–589. © 2009 Wiley‐Liss, Inc.
Fanconi anemia and biallelic BRCA2 mutation diagnosed in a young child with an embryonal CNS tumor
Authors: DeWire Mariko D. , Ellison David W. , Patay Zoltan , McKinnon Peter J. , Sanders Robert P. , Gajjar Amar 
Published: July 2009

AbstractMedulloblastoma, the most common pediatric malignant brain tumor often arises sporadically; however, in a subgroup of patients, there exist familial conditions such as Fanconi anemia with biallelic BRCA2 mutation that predispose patients to developing medulloblastoma. Biallelic inactivation of BRCA2 in Fanconi anemia has been previously described in only 11 patients with medulloblastoma in the literature to date. Here we report two siblings diagnosed with central nervous system embryonal tumors at an early age in association with biallelic BRCA2 inactivation, including the first reported case of a spinal cord primitive neuroectodermal tumor (PNET) in a BRCA2/FANCD1 kindred. Pediatr Blood Cancer 2009;53:1140–1142. © 2009 Wiley‐Liss, Inc.
Antifungal prophylaxis with micafungin in patients treated for childhood cancer
Authors: Kusuki Shigenori , Hashii Yoshiko , Yoshida Hisao , Takizawa Sachiko , Sato Emiko , Tokimasa Sadao , Ohta Hideaki , Ozono Keiichi 
Published: July 2009

AbstractBackgroundInvasive fungal infections (IFIs) remain a major cause of infectious morality in neutropenic patients receiving chemotherapy or hematopoietic stem cell transplantation (HSCT). Micafungin exhibits broad antifungal activity against both Aspergillus and Candida species. We performed a retrospective study to determine the efficacy and safety of prophylactic micafungin against IFI in pediatric neutropenic patients during chemotherapy or HSCT.ProcedureForty patients were given micafungin (3 mg/kg/day) intravenously for neutropenia: 131 patient‐cycles (39 patients) after chemotherapy and 15 patient‐cycles (14 patients) after HSCT. Median duration of neutropenia and micafungin prophylaxis was 13 and 23 days after chemotherapy and HSCT, respectively.ResultsTreatment success rate, defined as absence of proven, probable, possible, or suspected IFIs, was 93.9% (121/131) and 80.0% (12/15) for chemotherapy and HSCT, respectively. Proven or probable IFI was documented in only one patient after HSCT. No adverse events were observed that could be related to micafungin prophylaxis.ConclusionsThese results suggest that prophylactic micafungin is well tolerated and may prevent IFIs in pediatric patients with neutropenia receiving chemotherapy or HSCT. Pediatr Blood Cancer 2009;53:605–609. © 2009 Wiley‐Liss, Inc.
PARP1 expression in pediatric central nervous system tumors
Authors: Barton Valerie N. , Donson Andrew M. , Kleinschmidt‐DeMasters B.K. , Gore Lia , Liu Arthur K. , Foreman Nicholas K. 
Published: July 2009

AbstractBackgroundDespite advances in therapy, outcome in many high‐grade pediatric central nervous system (CNS) tumors remains poor. The focus of neuro‐oncology research has thus turned towards identifying novel therapeutic targets. Poly(ADP‐ribose) polymerase‐1 (PARP1) is a DNA repair protein that has been studied in a variety of malignancies and may interfere with therapy‐induced DNA damage, however expression in pediatric CNS tumors is unknown.ProcedureWe evaluated PARP1 mRNA expression in 81 pediatric CNS tumors using microarray technology. Protein expression was examined by Western blot.ResultsPARP1 mRNA is highly expressed in high‐grade tumors (P < 0.0001). PARP1 mRNA expression was greater in high‐grade glioma than pilocytic astrocytoma (P = 3.5 × 10−5) and in large cell medulloblastoma over classic medulloblastoma (P = 0.0053). PARP1 protein was also prominent in high‐grade tumors (P = 0.022).ConclusionThese findings indicate that PARP1 is expressed in high‐grade pediatric CNS tumors, implicating PARP1 inhibition as a potential therapeutic target. Pediatr Blood Cancer 2009; 53:1227–1230. © 2009 Wiley‐Liss, Inc.