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Pediatric Oncology

Pediatric Oncology Organiations
Specialist Journals
Recent Research Publications
Childhood Cancers (menu)

Pediatric Oncology Organiations (17 links)

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    American Society of Pediatric Hematology/Oncology ASPHO USA
    Association of Pediatric Hematology / Oncology NursesAPHON International
    Australian and New Zealand Children's Haematology/Oncology GroupANZCHOG Australia
    Children’s Cancer and Leukaemia GroupCCLG UK
    Children’s Oncology GroupCOG USA
    Danish Paediatric Haematology and Oncology GroupDAPHO   Denmark
    European Society of Paediatric Oncology SIOP-E Europe
    International Society of Paediatric OncologySIOP International
    Japanese Society Of Pediatric OncologyJSPO Japan
    Malaysian Society of Paediatric Haematology and OncologyMASPHO Malaysia
    Nordic Society of Pediatric Haematology and OncologyNOPHO Nordic Countries
    Pediatric Oncology Group of OntarioPOGO Canada
    Philippine Society of Pediatric OncologyPSPO Philippines
    Society for Paediatric Oncology and HaematologyGPOH Germany
    South African Children's Study Cancer GroupSACCSG South Africa
    Swedish Paediatric Society - Division of Oncology and Hematology   Sweden
    Swiss Paediatric Oncology Group Switzerland

    Specialist Journals (3 links)

    See also: Oncology Journals

    Recent Research Publications

    Brown CE, Alizadeh D, Starr R, et al.
    Regression of Glioblastoma after Chimeric Antigen Receptor T-Cell Therapy.
    N Engl J Med. 2016; 375(26):2561-9 [PubMed] Related Publications
    A patient with recurrent multifocal glioblastoma received chimeric antigen receptor (CAR)-engineered T cells targeting the tumor-associated antigen interleukin-13 receptor alpha 2 (IL13Rα2). Multiple infusions of CAR T cells were administered over 220 days through two intracranial delivery routes - infusions into the resected tumor cavity followed by infusions into the ventricular system. Intracranial infusions of IL13Rα2-targeted CAR T cells were not associated with any toxic effects of grade 3 or higher. After CAR T-cell treatment, regression of all intracranial and spinal tumors was observed, along with corresponding increases in levels of cytokines and immune cells in the cerebrospinal fluid. This clinical response continued for 7.5 months after the initiation of CAR T-cell therapy. (Funded by Gateway for Cancer Research and others; ClinicalTrials.gov number, NCT02208362 .).

    Ramirez MD, Mertens AC, Esiashvili N, et al.
    Yield of Urinalysis Screening in Pediatric Cancer Survivors.
    Pediatr Blood Cancer. 2016; 63(5):893-900 [PubMed] Article available free on PMC after 01/05/2017 Related Publications
    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 Version 3.0 of the COG Long-Term Follow-Up Guidelines and identify possible risk factors for abnormal screening in a survivor population.
    PROCEDURE: A database of pediatric cancer survivors evaluated between January 2008 and March 2012 at Children's Healthcare of Atlanta was queried for survivors at risk for genitourinary late effects. The frequency of abnormal urinalyses (protein ≥1+ and/or presence of glucose and/or ≥5 red blood cells per high power field) was estimated. Risk factors associated with abnormal screening were identified.
    RESULTS: Chart review identified 773 survivors (57% male; 67% Caucasian; 60% leukemia/lymphoma survivors; mean age at diagnosis, 5.7 years [range: birth to 17.7 years]; time from diagnosis to initial screening, 7.6 years [range: 2.3 to 21.5 years]) who underwent urinalysis. Abnormal results were found in 78 (5.3%) of 1,484 total urinalyses. Multivariable analysis revealed higher dose ifosfamide (odds ratio [OR] = 6.8, 95% confidence interval [CI] 2.9-16.0) and total body irradiation (TBI, OR = 3.0, 95% CI 1.0-8.4) as significant risk factors for abnormal initial urinalysis screening.
    CONCLUSIONS: Pediatric cancer survivors exposed to higher dose ifosfamide or TBI may be at higher risk of abnormal findings on urinalysis screening. Targeted screening of these higher risk patients should be considered.

    Stulac S, Mark Munyaneza RB, Chai J, et al.
    Initiating Childhood Cancer Treatment in Rural Rwanda: A Partnership-Based Approach.
    Pediatr Blood Cancer. 2016; 63(5):813-7 [PubMed] Related Publications
    BACKGROUND: More than 85% of pediatric cancer cases and 95% of deaths occur in resource-poor countries that use less than 5% of the world's health resources. In the developed world, approximately 81% of children with cancer can be cured. Models applicable in the most resource-poor settings are needed to address global inequities in pediatric cancer treatment.
    PROCEDURE: Between 2006 and 2011, a cohort of children received cancer therapy using a new approach in rural Rwanda. Children were managed by a team of a Rwandan generalist doctor, Rwandan nurse case manager, Rwanda-based US-trained pediatrician, and US-based pediatric oncologist. Biopsies and staging studies were obtained in-country. Pathologic diagnoses were made at US or European laboratories. Rwanda-based clinicians and the pediatric oncologist jointly generated treatment plans by telephone and email.
    RESULTS: Treatment was provided to 24 patients. Diagnoses included lymphomas (n = 10), sarcomas (n = 9), leukemias (n = 2), and other malignancies (n = 3). Standard chemotherapy regimens included CHOP, ABVD, VA, COP/COMP, and actino-VAC. Thirteen patients were in remission at the completion of data collection. Two succumbed to treatment complications and nine had progressive disease. There were no patients who abandoned treatment. The mean overall survival was 31 months and mean disease-free survival was 18 months.
    CONCLUSIONS: These data suggest that chemotherapy can be administered with curative intent to a subset of cancer patients in this setting. This approach provides a platform for pediatric cancer care models, relying on local physicians collaborating with remote specialist consultants to deliver subspecialty care in resource-poor settings.

    Dupuis LL, Lu X, Mitchell HR, et al.
    Anxiety, pain, and nausea during the treatment of standard-risk childhood acute lymphoblastic leukemia: A prospective, longitudinal study from the Children's Oncology Group.
    Cancer. 2016; 122(7):1116-25 [PubMed] Article available free on PMC after 01/04/2017 Related Publications
    BACKGROUND: This prospective study describes the procedure-related anxiety, treatment-related anxiety, pain, and nausea experienced by children with standard-risk acute lymphoblastic leukemia (ALL) during the first year of treatment.
    METHODS: This study was undertaken at 31 Children's Oncology Group (COG) sites. Eligible children who were 2 to 9.99 years old were enrolled in a COG trial for patients with newly diagnosed standard-risk ALL from 2005 to 2009. Parents completed a demographic survey at the baseline and the Pediatric Quality of Life Inventory 3.0 Cancer Module (proxy version) and the General Functioning Scale of the Family Assessment Device 1, 6, and 12 months after the diagnosis. The association between patient-related (age, sex, ethnicity, and treatment), parent-related (marital status and education), and family-related factors (functioning, income, and size) and symptom scores was evaluated.
    RESULTS: The mean scores for procedure-related anxiety, treatment-related anxiety, and pain improved during the first year of treatment (P < .0389). The mean nausea score was poorer 6 months after the diagnosis in comparison with the other assessments (P = .0085). A younger age at diagnosis was associated with significantly worse procedure-related anxiety (P = .004). An older age (P = .0002) and assignment to the intensified consolidation study arm (P = .02) were associated with significantly worse nausea.
    CONCLUSIONS: Children with ALL experienced decreasing treatment-related anxiety, procedure-related anxiety, and pain during the first year of treatment. In comparison with scores at 1 and 12 months, nausea was worse 6 months after the diagnosis. Minimization of procedure-related anxiety in younger children and improved nausea control in older children and those receiving more intensified treatment should be prioritized.

    Tulstrup M, Larsen HB, Castor A, et al.
    Parents' and Adolescents' Preferences for Intensified or Reduced Treatment in Randomized Lymphoblastic Leukemia Trials.
    Pediatr Blood Cancer. 2016; 63(5):865-71 [PubMed] Related Publications
    BACKGROUND: When offered participation in clinical trials, families of children with cancer face a delicate balance between cure and toxicity. Since parents and children may perceive this balance differently, this paper explores whether adolescent patients have different enrollment patterns compared to younger children in trials with different toxicity profiles.
    PROCEDURE: Age-dependent participation rates in three consecutive, randomized childhood leukemia trials conducted by the Nordic Society of Paediatric Haematology and Oncology were evaluated. The ALL2000 dexamethasone/vincristine (Dx/VCR) trial tested treatment intensifications to improve cure, and the back-to-back ALL2008 6-mercaptopurine (6MP) and ALL2008 PEG-asparaginase (ASP) trials tested treatment intensifications (6MP) and toxicity reduction without compromising survival (ASP). Patient randomization and toxicity data were prospectively registered by the treating physicians.
    RESULTS: Parents of young children favored treatment intensifications (Dx/VCR: 12% refusal; 6MP: 14%; ASP: 21%), whereas parents of adolescents favored treatment reductions (Dx/VCR: 52% refusal; 6MP: 30%; ASP: 8%). Adolescents were more likely to refuse intensification trials than young children (adjusted ORs 6.3; P < 0.01 [Dx/VCR] and 2.1; P = 0.04 [6MP]). Adolescents were less likely to refuse the ASP trial, with varying effect size depending on the length of the preceding consolidation treatment (adjusted OR for median consolidation length 0.15; P = 0.01). Younger children participated more frequently in only 6MP than in only ASP (14% vs. 5%), and adolescents vice versa (2% vs. 17%; P = 0.001).
    CONCLUSIONS: Parents' and adolescents' divergent inclinations toward intensified or reduced therapy emphasize the necessity of actively involving adolescents in the informed consent process, which should also address motives for trial participation.

    Tuckuviene R, Ranta S, Albertsen BK, et al.
    Prospective study of thromboembolism in 1038 children with acute lymphoblastic leukemia: a Nordic Society of Pediatric Hematology and Oncology (NOPHO) study.
    J Thromb Haemost. 2016; 14(3):485-94 [PubMed] Related Publications
    UNLABELLED: ESSENTIALS: Children with acute lymphoblastic leukemia (ALL) are at risk of thromboembolism (TE). This is a prospective evaluation of the incidence, risk factors and outcomes of TE in 1038 children with ALL. TE occurred in 6.1% of children, with the highest incidence (20.5%) among those aged 15-17 years. A TE-associated case fatality of 6.4% indicates that TE is a severe complication of ALL treatment.

    Yen HJ, Chang WH, Liu HC, et al.
    Outcomes Following Discontinuation of E. coli l-Asparaginase Upon Severe Allergic Reactions in Children With Acute Lymphoblastic Leukemia.
    Pediatr Blood Cancer. 2016; 63(4):665-70 [PubMed] Related Publications
    BACKGROUND: Discontinuation of E. coli l-asparaginase in patients with acute lymphoblastic leukemia (ALL) is unavoidable upon severe allergic reaction. We sought to examine outcomes following E. coli l-asparaginase discontinuation due to severe allergic reactions.
    PROCEDURE: We evaluated the outcome of children enrolled in Taiwan Pediatric Oncology Group-2002-ALL protocol between 2002 and 2012, who had E. coli l-asparaginase discontinued due to severe allergic reactions, and compared the outcomes of those who continued with Erwinia l-asparaginase (Erwinase) with those who did not.
    RESULTS: Among 700 patients enrolled in this study, 33 patients had E. coli l-asparaginase treatment discontinued due to severe allergic reactions. Five-year overall survival did not differ significantly among the 648 patients without discontinuation (81 ± 1.6%, mean ± SE), compared to 17 patients with allergic reactions and treated with Erwinase (88 ± 7.8%) and 16 patients with allergic reactions but not treated with Erwinase (87 ± 8.6%). Among 16 patients who did not receive Erwinase, all 10 who received ≥50% of the scheduled doses of E. coli l-asparaginase before discontinuation survived without events.
    CONCLUSIONS: Erwinase treatment may not be needed for some ALL patients with severe allergy to E. coli l-asparaginase if ≥50% of prescribed doses were received and/or therapy is augmented with other agents.

    Casanova-García C, Lerma Lara S, Pérez Ruiz M, et al.
    Non-pharmacological treatment for neuropathic pain in children with cancer.
    Med Hypotheses. 2015; 85(6):791-7 [PubMed] Related Publications
    Neuropathic pain (NP) associated with childhood cancer is currently a difficult problem to control. It is treated with drugs that not only fail to provide the expected improvements, but which also have side effects. Therefore, the main aim of this pilot study is to assess whether non-pharmacological treatments, Graded Motor Imagery (GMI) and Neural Mobilization (NM), have a positive effect on this pain, thus improving the associated comorbid factors and, consequently, the quality of life of the children. In an n = 6, the results after 4 weeks of treatment show a 10-point improvement in the pain threshold and a 3.1-point improvement in the perception of pain.

    Agrawal C, Guthrie L, Sturm MS, et al.
    Comparison of Thyroid Nodule Prevalence by Ultrasound in Childhood Cancer Survivors With and Without Thyroid Radiation Exposure.
    J Pediatr Hematol Oncol. 2016; 38(1):43-8 [PubMed] Related Publications
    PURPOSE: Children receiving radiotherapy of the head, neck, or chest as treatment of primary malignancies are at increased risk for secondary thyroid malignancy. We hypothesized that current standards (yearly thyroid physical examination) lead to a substantial number of missed thyroid nodules. Our objectives were: (1) use thyroid ultrasound to assess thyroid nodules in childhood cancer survivors; and (2) compare prevalence of thyroid nodules in thyroid radiation-exposed patients as compared with cancer survivors without radiation exposure.
    METHODS: We recruited 60 patients with thyroid radiation and chemotherapy exposure (median age at cancer diagnosis 10.8 y) and 59 patients with chemotherapy exposure alone (median age at diagnosis 4.3 y) from our long-term survivorship clinics. Each patient had a thyroid physical examination and thyroid ultrasound performed.
    RESULTS: Thirty-three patients (27.7%) had nodules >0.3 cm of which 2 were palpated (6.1%). We found 22 radiated patients (36.7%) with nodules versus 11 nonradiated patients (18.6%) (P=0.03). Eleven patients were biopsied and 1 diagnosis of secondary papillary thyroid carcinoma was confirmed.
    CONCLUSION: Our study supports further examination of incorporating thyroid ultrasounds into long-term survivorship follow-up guidelines in radiation-exposed patients for the detection of thyroid nodules and secondary malignancies.

    Braam KI, van Dijk-Lokkart EM, Kaspers GJ, et al.
    Cardiorespiratory fitness and physical activity in children with cancer.
    Support Care Cancer. 2016; 24(5):2259-68 [PubMed] Article available free on PMC after 01/04/2017 Related Publications
    PURPOSE: This study assessed cardiorespiratory fitness (CRF), physical activity (PA), and sedentary behavior (SB), as well as factors associated with these outcomes in children during or shortly after cancer treatment.
    METHODS: Cross-sectionally, CRF data, obtained by the cardiopulmonary exercise test, and PA and SB data, obtained by an accelerometer, were assessed in children with cancer (8-18 years old). Linear regression models were used to determine associations between CRF, PA, or SB and patient characteristics.
    RESULTS: Among 60 children with cancer, mean age 12.6 years, 35 boys, 28 % were during cancer treatment. CRF, reported as the z score of VO2peak, showed that 32 children had a VO2peak z score which was -2 below the predicted value. CRF was significantly associated with PA and SB: each additional activity count per minute resulted in 0.05 ml/kg/min VO2peak increase and each additional minute sedentary reduced VO2peak by 0.06 ml/kg/min. Multiple linear regression models of PA and SB showed that decreased activity was significantly associated with higher age, being fatigued, being during childhood cancer treatment (p < 0.001), or having a higher percentage of fat mass. The multiple linear regression model showed that lower CRF was significantly associated with increased fatigue, being during cancer treatment, having a higher percentage of fat mass, and lower belief of own athletic competence (p < 0.001).
    CONCLUSION: This study revealed that children during or shortly after cancer treatment have low CRF scores. The most inactive children had a higher fat mass, were fatigued, older, and during childhood cancer treatment. Unexpectedly, treatment-related factors showed no significant association with activity behavior.

    Oskarsson T, Söderhäll S, Arvidson J, et al.
    Relapsed childhood acute lymphoblastic leukemia in the Nordic countries: prognostic factors, treatment and outcome.
    Haematologica. 2016; 101(1):68-76 [PubMed] Article available free on PMC after 01/04/2017 Related Publications
    Relapse is the main reason for treatment failure in childhood acute lymphoblastic leukemia. Despite improvements in the up-front therapy, survival after relapse is still relatively poor, especially for high-risk relapses. The aims of this study were to assess outcomes following acute lymphoblastic leukemia relapse after common initial Nordic Society of Paediatric Haematology and Oncology protocol treatment; to validate currently used risk stratifications, and identify additional prognostic factors for overall survival. Altogether, 516 of 2735 patients (18.9%) relapsed between 1992 and 2011 and were included in the study. There were no statistically significant differences in outcome between the up-front protocols or between the relapse protocols used, but an improvement over time was observed. The 5-year overall survival for patients relapsing in the period 2002-2011 was 57.5±3.4%, but 44.7±3.2% (P<0.001) if relapse occurred in the period 1992-2001. Factors independently predicting mortality after relapse included short duration of first remission, bone marrow involvement, age ten years or over, unfavorable cytogenetics, and Down syndrome. T-cell immunophenotype was not an independent prognostic factor unless in combination with hyperleukocytosis at diagnosis. The outcome for early combined pre-B relapses was unexpectedly poor (5-year overall survival 38.0±10.6%), which supports the notion that these patients need further risk adjustment. Although survival outcomes have improved over time, the development of novel approaches is urgently needed to increase survival in relapsed childhood acute lymphoblastic leukemia.

    Conklin HM, Ogg RJ, Ashford JM, et al.
    Computerized Cognitive Training for Amelioration of Cognitive Late Effects Among Childhood Cancer Survivors: A Randomized Controlled Trial.
    J Clin Oncol. 2015; 33(33):3894-902 [PubMed] Article available free on PMC after 01/04/2017 Related Publications
    PURPOSE: Children receiving CNS-directed therapy for cancer are at risk for cognitive problems, with few available empirically supported interventions. Cognitive problems indicate neurodevelopmental disruption that may be modifiable with intervention. This study evaluated short-term efficacy of a computerized cognitive training program and neural correlates of cognitive change.
    PATIENT AND METHODS: A total of 68 survivors of childhood acute lymphoblastic leukemia (ALL) or brain tumor (BT) with identified cognitive deficits were randomly assigned to computerized cognitive intervention (male, n = 18; female, n = 16; ALL, n = 23; BT, n = 11; mean age ± standard deviation, 12.21 ± 2.47 years) or waitlist (male, n = 18; female, n = 16; ALL, n = 24; BT, n = 10; median age ± standard deviation, 11.82 ± 2.42 years). Intervention participants were asked to complete 25 training sessions at home with weekly, telephone-based coaching. Cognitive assessments and functional magnetic resonance imaging scans (intervention group) were completed pre- and postintervention, with immediate change in spatial span backward as the primary outcome.
    RESULTS: Survivors completing the intervention (n = 30; 88%) demonstrated greater improvement than controls on measures of working memory (mean ± SEM; eg, Wechsler Intelligence Scale for Children [fourth edition; WISC-IV] spatial span backward, 3.13 ± 0.58 v 0.75 ± 0.43; P = .002; effect size [ES], 0.84), attention (eg, WISC-IV spatial span forward, 3.30 ± 0.71 v 1.25 ± 0.39; P = .01; ES, 0.65), and processing speed (eg, Conners' Continuous Performance Test hit reaction time, -2.10 ± 1.47 v 2.54 ± 1.25; P = .02; ES, .61) and showed greater reductions in reported executive dysfunction (eg, Conners' Parent Rating Scale III, -6.73 ± 1.51 v 0.41 ± 1.53; P = .002; ES, 0.84). Functional magnetic resonance imaging revealed significant pre- to post-training reduction in activation of left lateral prefrontal and bilateral medial frontal areas.
    CONCLUSION: Study findings show computerized cognitive training is feasible and efficacious for childhood cancer survivors, with evidence for training-related neuroplasticity.

    de Vathaire F, Haddy N, Allodji RS, et al.
    Thyroid Radiation Dose and Other Risk Factors of Thyroid Carcinoma Following Childhood Cancer.
    J Clin Endocrinol Metab. 2015; 100(11):4282-90 [PubMed] Related Publications
    CONTEXT: Thyroid carcinoma is a frequent complication of childhood cancer radiotherapy. The dose response to thyroid radiation dose is now well established, but the potential modifier effect of other factors requires additional investigation.
    OBJECTIVE: This study aimed to investigate the role of potential modifiers of the dose response.
    DESIGN: We followed a cohort of 4338 5-year survivors of solid childhood cancer treated before 1986 over an average of 27 years. The dose received by the thyroid gland and some other anatomical sites during radiotherapy was estimated after reconstruction of the actual conditions in which irradiation was delivered.
    RESULTS: Fifty-five patients developed thyroid carcinoma. The risk of thyroid carcinoma increased with a radiation dose to the thyroid of up to two tenths of Gy, then leveled off for higher doses. When taking into account the thyroid radiation dose, a surgical or radiological splenectomy (>20 Gy to the spleen) increased thyroid cancer risk (relative risk [RR] = 2.3; 95% confidence interval [CI], 1.3-4.0), high radiation doses (>5 Gy) to pituitary gland lowered this risk (RR = 0.2; 95% CI, 0.1-0.6). Patients who received nitrosourea chemotherapy had a 6.6-fold (95% CI, 2.5-15.7) higher risk than those who did not. The excess RR per Gy of radiation to the thyroid was 4.7 (95% CI, 1.7-22.6). It was 7.6 (95% CI, 1.6-33.3) if body mass index at time of interview was equal or higher than 25 kg/m(2), and 4.1 (95% CI, 0.9-17.7) if not (P for interaction = .1).
    CONCLUSION: Predicting thyroid cancer risk following childhood cancer radiation therapy probably requires the assessment of more than just the radiation dose to the thyroid. Chemotherapy, splenectomy, radiation dose to pituitary gland, and obesity also play a role.

    Ruble K, Scarvalone S, Gallicchio L, et al.
    Group Physical Activity Intervention for Childhood Cancer Survivors: A Pilot Study.
    J Phys Act Health. 2016; 13(3):352-9 [PubMed] Related Publications
    BACKGROUND: Inadequate physical activity (PA) in childhood cancer survivors may lead to compromised health outcomes. The purpose of this pilot study was to evaluate the feasibility and effect of a PA intervention in childhood cancer survivors ages 8-12 who report < 1 hour of moderate-to-vigorous physical (MVPA) per day.
    METHODS: Twenty survivors were randomized to a 6-month group PA intervention or to a control group. A pre/post measure of MVPA was completed by all participants, and a pre/post measure of self-efficacy was completed by the intervention group. Analysis included measures of feasibility, change in percentage of awake time spent in MVPA, self-efficacy scores, and correlations in MVPA and self-efficacy.
    RESULTS: All feasibility parameters were confirmed. Increases in percent of awake time spent in MVPA were seen in 67% of the intervention group and 14% of the control group. A medium effect size (r = 0.55) was calculated for the correlation between change in MVPA and change in total self-efficacy scores; the largest effect size (r = 0.62) was found for the subscale for adequacy.
    CONCLUSIONS: Increases in MVPA can be seen in childhood cancer survivors who participate in a group intervention that includes support of self-efficacy.

    Schündeln MM, Hauffa PK, Bauer JJ, et al.
    Pediatric Survivors of Retinoblastoma Are at Risk for Altered Bone Metabolism After Chemotherapy Treatment Early in Life.
    Pediatr Hematol Oncol. 2015; 32(7):455-66 [PubMed] Related Publications
    Survivors of childhood cancer frequently suffer from endocrine late effects, which are, at least partly, attributed to toxic effects of chemotherapy. Treatment of retinoblastoma typically involves chemotherapy at a very young age. The authors conducted a cross-sectional study to assess bone health in a pediatric cohort of 33 survivors of retinoblastoma (mean age: 4.4 years) who had undergone chemotherapy treatment at an especially young age (mean age: 0.76 years). Of these patients, 14 had unilateral and 19 bilateral retinoblastoma. Polychemotherapy consisted of treatment with cyclophosphamide, etoposide, vincristine, and carboplatin. Ten patients had undergone external beam radiotherapy. Clinical and biochemical parameters of growth, pubertal development, and bone health were obtained. A vitamin D deficiency was found in 51.7% of the patients, and 13.7% of patients displayed severe vitamin D deficiency. Secondary hyperparathyroidism and altered readings for bone formation or resorption markers were present in 15%. Nine percent reported bone pain or experienced fractures of the long bones after primary diagnosis. No difference between children with bilateral and unilateral disease or irradiated versus nonirradiated children was observed. The parameters of thyroid function, growth, and pubertal development were within age-appropriate norms in almost all children. In conclusion, altered parameters of bone health can be present in survivors of retinoblastoma at a young age and warrant regular follow-up in these children. The endocrine hypothalamic-pituitary axes, however, were not impaired at this early age in this group of survivors of retinoblastoma.

    Mitchell HR, Lu X, Myers RM, et al.
    Prospective, longitudinal assessment of quality of life in children from diagnosis to 3 months off treatment for standard risk acute lymphoblastic leukemia: Results of Children's Oncology Group study AALL0331.
    Int J Cancer. 2016; 138(2):332-9 [PubMed] Article available free on PMC after 01/04/2017 Related Publications
    Standard risk acute lymphoblastic leukemia (SR-ALL) has high cure rates, but requires 2-3 years of therapy. We aimed to (i) prospectively evaluate health-related quality of life (HRQOL) during and after SR-ALL therapy, and (ii) identify associated predictors. Parents of 160 SR-ALL patients enrolled on Children's Oncology Group (COG) therapeutic trial AALL0331 at 31 sites completed the Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scales (physical, emotional and social functioning) and Family Assessment Device-General Functioning (FAD-GF) at 1, 6 and 12 months after diagnosis, and 3 months post-therapy. Mean PedsQL scores in physical, emotional and social functioning were impaired 1 month after diagnosis but steadily improved. Three months post-therapy, impaired physical and social functioning was observed in 27.8 and 25.8% of patients, respectively. In repeated-measures analysis, problematic family functioning predicted emotional (OR = 1.85, 95% CI 1.03-3.34) and social (OR = 1.99, 95% CI 1.21-3.27) impairment. Larger household size was associated with social impairment (OR = 1.21, 95% CI 1.02-1.45). Adverse neurological event(s) during therapy predicted post-therapy physical (OR = 5.17, 95% CI 1.61-16.63) and social (OR = 8.17, 95% CI 1.19-56.16) impairment. HRQOL 1 month after diagnosis was not predictive of HRQOL 3 months after therapy completion. In conclusion, children with SR-ALL experience considerable impairment in HRQOL at the end of induction, but rapidly improve. However, many still experience physical and social impairment 3 months post-therapy, suggesting a role for continued family and physical functioning support. Longer follow-up is needed to determine if post-therapy deficits change over time.

    Naumann FL, Hunt M, Hunt M, et al.
    Assessment of Fundamental Movement Skills in Childhood Cancer Patients.
    Pediatr Blood Cancer. 2015; 62(12):2211-5 [PubMed] Related Publications
    BACKGROUND: The improved treatment protocols and subsequent improved survival rates among childhood cancer patients have shifted the focus toward the long-term consequences arising from cancer treatment. Children who have completed cancer treatment are at a greater risk of delayed development, diminished functioning, disability, compromised fundamental movement skill (FMS) attainment, and long-term chronic health conditions. The aim of the study was to compare FMS of childhood cancer patients with an aged matched healthy reference group.
    METHODS: Pediatric cancer patients aged 5-8 years (n = 26; median age 6.91 years), who completed cancer treatment (<5 years) at the Sydney Children's Hospital, were assessed performing seven key FMS: sprint, side gallop, vertical jump, catch, over-arm throw, kick, and leap. Results were compared to the reference group (n = 430; 6.56 years).
    RESULTS: Childhood cancer patients scored significantly lower on three out of seven FMS tests when compared to the reference group. These results equated to a significantly lower overall score for FMS.
    CONCLUSIONS: This study highlighted the significant deficits in FMS within pediatric patients having completed cancer treatment. In order to reduce the occurrence of significant FMS deficits in this population, FMS interventions may be warranted to assist in recovery from childhood cancer, prevent late effects, and improve the quality of life in survivors of childhood cancer.

    Asdahl PH, Winther JF, Bonnesen TG, et al.
    The Adult Life After Childhood Cancer in Scandinavia (ALiCCS) Study: Design and Characteristics.
    Pediatr Blood Cancer. 2015; 62(12):2204-10 [PubMed] Related Publications
    BACKGROUND: During the last five decades, survival of childhood cancer has increased from 25% to 80%. At the same time, however, it has become evident that survivors experience a broad range of therapy-related late adverse health effects. The aim of the Adult Life after Childhood Cancer in Scandinavia (ALiCCS) study is to investigate long-term health consequences of past and current therapies in order to improve follow-up care of survivors and to reduce treatment-related morbidity of future patients.
    PROCEDURE: Childhood cancer survivors were identified through the five Nordic cancer registries and a comparison cohort was established through random selection of cancer-free individuals from the civil registration systems. A unique personal identification number was used to link between different health registries. Abstraction of treatment information for a subset of survivors allows investigation of the association between the various components of cancer therapy and late occurring comorbidity.
    RESULTS: The childhood cancer survivor cohort comprises 33,160 1-year survivors and the comparison cohort comprises 212,892 cancer free individuals from the general population. In the childhood cancer survivor cohort, all types of childhood cancer are represented including leukemia (21%), lymphoma (14%), central nervous system tumors (24%), sarcomas (5%), retinoblastoma (3%), and neuroblastoma (4%). Among the survivors, 22% have been followed beyond the age of 40 years.
    CONCLUSION: The ALiCCS study constitutes a new large resource for research on late effects of childhood cancers that include all types of childhood malignancies and has followed a large proportion of the survivors well into late adulthood.

    Tesi B, Chiang SC, El-Ghoneimy D, et al.
    Spectrum of Atypical Clinical Presentations in Patients with Biallelic PRF1 Missense Mutations.
    Pediatr Blood Cancer. 2015; 62(12):2094-100 [PubMed] Related Publications
    BACKGROUND: Perforin, encoded by PRF1, is a pore-forming protein crucial for lymphocyte cytotoxicity. Biallelic PRF1 nonsense mutations invariably result in early-onset hemophagocytic lymphohistiocytosis (HLH), termed familial HLH type 2 (FHL2). In contrast, biallelic PRF1 missense mutations may give rise to later-onset disease and more variable manifestations.
    PROCEDURE: We retrospectively searched our database for patients from families with siblings carrying biallelic PRF1 missense mutations where at least one sibling did not develop HLH, and for patients with biallelic PRF1 missense mutations and an atypical presentation of disease. We reviewed their clinical, genetic, and immunological characteristics.
    RESULTS: In all, we identified 10 such patients, including three sibling pairs with discordant manifestations. Interestingly, in two families, siblings of late-onset HLH patients developed Hodgkin lymphoma but no HLH. In a third family, one sibling presented with recurrent HLH episodes, whereas the other remains healthy. Of note, the affected sibling also suffered from systemic lupus erythematosus. Additional unrelated patients with biallelic PRF1 missense mutations were affected by neurological disease without classical signs of HLH, gastrointestinal inflammation as initial presentation of disease, as well as a hematological malignancy. Compared to early-onset FHL2 patients, the patients with an atypical presentation displayed a partial recovery of NK cell cytotoxicity upon IL-2 stimulation in vitro.
    CONCLUSIONS: Our findings substantiate and expand the spectrum of clinical presentations of perforin deficiency, linking PRF1 missense mutations to lymphoma susceptibility and highlighting clinical variability within families. PRF1 mutations should, therefore, be considered as a cause of several diseases disparate to HLH.

    Pritchard-Jones K, Bergeron C, de Camargo B, et al.
    Omission of doxorubicin from the treatment of stage II-III, intermediate-risk Wilms' tumour (SIOP WT 2001): an open-label, non-inferiority, randomised controlled trial.
    Lancet. 2015; 386(9999):1156-64 [PubMed] Related Publications
    BACKGROUND: Before this study started, the standard postoperative chemotherapy regimen for stage II-III Wilms' tumour pretreated with chemotherapy was to include doxorubicin. However, avoidance of doxorubicin-related cardiotoxicity effects is important to improve long-term outcomes for childhood cancers that have excellent prognosis. We aimed to assess whether doxorubicin can be omitted safely from chemotherapy for stage II-III, histological intermediate-risk Wilms' tumour when a newly defined high-risk blastemal subtype was excluded from randomisation.
    METHODS: For this international, multicentre, open-label, non-inferiority, phase 3, randomised SIOP WT 2001 trial, we recruited children aged 6 months to 18 years at the time of diagnosis of a primary renal tumour from 251 hospitals in 26 countries who had received 4 weeks of preoperative chemotherapy with vincristine and actinomycin D. Children with stage II-III intermediate-risk Wilms' tumours assessed after delayed nephrectomy were randomly assigned (1:1) by a minimisation technique to receive vincristine 1·5 mg/m(2) at weeks 1-8, 11, 12, 14, 15, 17, 18, 20, 21, 23, 24, 26, and 27, plus actinomycin D 45 μg/kg every 3 weeks from week 2, either with five doses of doxorubicin 50 mg/m(2) given every 6 weeks from week 2 (standard treatment) or without doxorubicin (experimental treatment). The primary endpoint was non-inferiority of event-free survival at 2 years, analysed by intention to treat and a margin of 10%. Assessment of safety and adverse events included systematic monitoring of hepatic toxicity and cardiotoxicity. This trial is registered with EudraCT, number 2007-004591-39, and is closed to new participants.
    FINDINGS: Between Nov 1, 2001, and Dec 16, 2009, we recruited 583 patients, 341 with stage II and 242 with stage III tumours, and randomly assigned 291 children to treatment including doxorubicin, and 292 children to treatment excluding doxorubicin. Median follow-up was 60·8 months (IQR 40·8-79·8). 2 year event-free survival was 92·6% (95% CI 89·6-95·7) for treatment including doxorubicin and 88·2% (84·5-92·1) for treatment excluding doxorubicin, a difference of 4·4% (95% CI 0·4-9·3) that did not exceed the predefined 10% margin. 5 year overall survival was 96·5% (94·3-98·8) for treatment including doxorubicin and 95·8% (93·3-98·4) for treatment excluding doxorubicin. Four children died from a treatment-related toxic effect; one (<1%) of 291 receiving treatment including doxorubicin died of sepsis, three (1%) of 292 receiving treatment excluding doxorubicin died of varicella, metabolic seizure, and sepsis during treatment for relapse. 17 patients (3%) had hepatic veno-occlusive disease. Cardiotoxic effects were reported in 15 (5%) of 291 children receiving treatment including doxorubicin. 12 children receiving treatment including doxorubicin, and ten children receiving treatment excluding doxorubicin, died, with the remaining deaths from tumour recurrence.
    INTERPRETATION: Doxorubicin does not need to be included in treatment of stage II-III intermediate risk Wilms' tumour when the histological response to preoperative chemotherapy is incorporated into the risk stratification.
    FUNDING: See Acknowledgments for funders.

    Ekman S, Harmenberg J, Frödin JE, et al.
    A novel oral insulin-like growth factor-1 receptor pathway modulator and its implications for patients with non-small cell lung carcinoma: A phase I clinical trial.
    Acta Oncol. 2016; 55(2):140-8 [PubMed] Related Publications
    BACKGROUND: A phase Ia/b dose-escalation study was performed to characterize the safety, efficacy and pharmacokinetic properties of the oral small molecule insulin-like growth factor-1-receptor pathway modulator AXL1717 in patients with advanced solid tumors.
    MATERIAL AND METHODS: This was a prospective, single-armed, open label, dose-finding phase Ia/b study with the aim of single day dosing (phase Ia) to define the starting dose for multi-day dosing (phase Ib), and phase Ib to define and confirm recommended phase II dose (RP2D) and if possible maximum tolerated dose (MTD) for repeated dosing.
    RESULTS AND CONCLUSION: Phase Ia enrolled 16 patients and dose escalations up to 2900 mg BID were successfully performed without any dose limiting toxicity (DLT). A total of 39 patients were treated in phase Ib. AXL1717 was well tolerated with neutropenia as the only dose-related, reversible, DLT. RP2D dose was found to be 390 mg BID for four weeks. Some patients, mainly with NSCLC, demonstrated signs of clinical benefit, including four partial tumor responses (one according to RECIST and three according to PET). The 15 patients with NSCLC with treatment duration longer than two weeks with single agent AXL1717 in third or fourth line of therapy showed a median progression-free survival of 31 weeks and overall survival of 60 weeks. Down-regulation of IGF-1R on granulocytes and increases of free serum levels of IGF-1 were seen in patients treated with AXL1717. AXL1717 had an acceptable safety profile and demonstrated promising efficacy in this heavily pretreated patient cohort, especially in patients with NSCLC. RP2D was concluded to be 390 mg BID for four weeks. Trial number is NCT01062620.

    Ramjaun A, AlDuhaiby E, Ahmed S, et al.
    Echocardiographic Detection of Cardiac Dysfunction in Childhood Cancer Survivors: How Long Is Screening Required?
    Pediatr Blood Cancer. 2015; 62(12):2197-203 [PubMed] Article available free on PMC after 01/04/2017 Related Publications
    BACKGROUND: Childhood cancer survivors treated with anthracycline chemotherapy are at an increased risk of long-term cardiac toxicity, and guidelines recommend that exposed survivors undergo echocardiography every 1-5 years. However, it is unclear whether survivors should undergo echocardiographic screening indefinitely, or if a period of echocardiographic stability indicates that screening is no longer necessary. The objective of this study was to evaluate the outcomes of echocardiographic screening to aid in the refinement of existing guidelines.
    METHODS: We retrospectively analyzed the results of echocardiographic screening in a cohort of adult survivors of childhood cancer treated with anthracyclines and/or cardiac radiation therapy. Interval regression analysis was performed to identify predictors of single-episode or sustained abnormal echocardiograms.
    RESULTS: The cohort constituted 333 survivors, with median follow-up time of 15.8 years post-treatment (range: 5.0-47.9), and median age at treatment of 8 years (range: 1.5-18). Forty-nine survivors had an abnormal echocardiogram (14.7%), and 29 (8.7%) had reproducible abnormal findings. An ongoing continual increase in the incidence of sustained echocardiographic abnormality was seen among patients treated with >250 mg/m(2) doxorubicin at age <5 years, reaching 43% by 20 years of therapy. In contrast, no sustained abnormal echocardiographic findings arose after 10 years of therapy in survivors treated with <250 mg/m(2) at age ≥5 years.
    CONCLUSIONS: Single-episode echocardiographic abnormalities are often not reproduced in subsequent evaluations. The duration of echocardiographic screening for childhood cancer survivors should be reassessed for patients who received lower doses of anthracycline after age 5.

    Borowitz MJ, Wood BL, Devidas M, et al.
    Prognostic significance of minimal residual disease in high risk B-ALL: a report from Children's Oncology Group study AALL0232.
    Blood. 2015; 126(8):964-71 [PubMed] Article available free on PMC after 01/04/2017 Related Publications
    Minimal residual disease (MRD) is highly prognostic in pediatric B-precursor acute lymphoblastic leukemia (B-ALL). In Children's Oncology Group high-risk B-ALL study AALL0232, we investigated MRD in subjects randomized in a 2 × 2 factorial design to receive either high-dose methotrexate (HD-MTX) or Capizzi methotrexate (C-MTX) during interim maintenance (IM) or prednisone or dexamethasone during induction. Subjects with end-induction MRD ≥0.1% or those with morphologic slow early response were nonrandomly assigned to receive a second IM and delayed intensification phase. MRD was measured by 6-color flow cytometry in 1 of 2 reference labs, with excellent agreement between the two. Subjects with end-induction MRD <0.01% had a 5-year event-free survival (EFS) of 87% ± 1% vs 74% ± 4% for those with MRD 0.01% to 0.1%; increasing MRD amounts was associated with progressively worse outcome. Subjects converting from MRD positive to negative by end consolidation had a relatively favorable 79% ± 5% 5-year disease-free survival vs 39% ± 7% for those with MRD ≥0.01%. Although HD-MTX was superior to C-MTX, MRD retained prognostic significance in both groups (86% ± 2% vs 58% ± 4% for MRD-negative vs positive C-MTX subjects; 88% ± 2% vs 68% ± 4% for HD-MTX subjects). Intensified therapy given to subjects with MRD >0.1% did not improve either 5-year EFS or overall survival (OS). However, these subjects showed an early relapse rate similar to that seen in MRD-negative ones, with EFS/OS curves for patients with 0.1% to 1% MRD crossing those with 0.01% to 0.1% MRD at 3 and 4 years, thus suggesting that the intensified therapy altered the disease course of MRD-positive subjects. Additional interventions targeted at the MRD-positive group may further improve outcome. This trial was registered at www.clinicaltrials.gov as #NCT00075725.

    Brown BJ, Adeleye AO, Ibeh JN
    A Prospective Study on the Causes of Delayed Diagnosis of Childhood Cancer in Ibadan, Nigeria.
    Pediatr Hematol Oncol. 2015; 32(6):365-73 [PubMed] Related Publications
    Outcome in cancer may be improved by early diagnosis and prompt treatment. The objectives of this study were to determine the prediagnostic intervals (lag time) in childhood cancer and the factors that influence them at the University College Hospital (UCH), Ibadan. The study was prospective and observational and involved children diagnosed with cancer from July 2012 to June 2014 at UCH, Ibadan, Nigeria. A history of the illness was obtained and physical examination performed on each patient. Information obtained and analyzed included sociodemographic data, cancer diagnosis and stage, time intervals between onset of symptoms and diagnosis, and the reasons for delayed diagnosis. A total of 91 children were studied, comprising 46 males and 45 females. Their ages ranged from 1 month to 15.0 years, with a median of 4.0 years. Median parent lag time was 2.0 weeks, median health system or physician lag time 8.0 weeks, and median overall lag time 15.5 weeks. Overall lag time had a negative correlation with age of child at diagnosis, a positive correlation with the number of health facilities visited before diagnosis, and was shorter in mothers younger than 40 years of age. Lag time was significantly different among the diagnostic tumor categories, with Burkitt lymphoma having short times and retinoblastoma with long times. Delayed diagnosis of childhood cancer is a significant problem in Ibadan. Education of parents and health workers on early presentation and accurate diagnosis are recommended.

    Brown CE, Badie B, Barish ME, et al.
    Bioactivity and Safety of IL13Rα2-Redirected Chimeric Antigen Receptor CD8+ T Cells in Patients with Recurrent Glioblastoma.
    Clin Cancer Res. 2015; 21(18):4062-72 [PubMed] Article available free on PMC after 01/04/2017 Related Publications
    PURPOSE: A first-in-human pilot safety and feasibility trial evaluating chimeric antigen receptor (CAR)-engineered, autologous primary human CD8(+) cytotoxic T lymphocytes (CTL) targeting IL13Rα2 for the treatment of recurrent glioblastoma (GBM).
    EXPERIMENTAL DESIGN: Three patients with recurrent GBM were treated with IL13(E13Y)-zetakine CD8(+) CTL targeting IL13Rα2. Patients received up to 12 local infusions at a maximum dose of 10(8) CAR-engineered T cells via a catheter/reservoir system.
    RESULTS: We demonstrate the feasibility of manufacturing sufficient numbers of autologous CTL clones expressing an IL13(E13Y)-zetakine CAR for redirected HLA-independent IL13Rα2-specific effector function for a cohort of patients diagnosed with GBM. Intracranial delivery of the IL13-zetakine(+) CTL clones into the resection cavity of 3 patients with recurrent disease was well-tolerated, with manageable temporary brain inflammation. Following infusion of IL13-zetakine(+) CTLs, evidence for transient anti-glioma responses was observed in 2 of the patients. Analysis of tumor tissue from 1 patient before and after T-cell therapy suggested reduced overall IL13Rα2 expression within the tumor following treatment. MRI analysis of another patient indicated an increase in tumor necrotic volume at the site of IL13-zetakine(+) T-cell administration.
    CONCLUSIONS: These findings provide promising first-in-human clinical experience for intracranial administration of IL13Rα2-specific CAR T cells for the treatment of GBM, establishing a foundation on which future refinements of adoptive CAR T-cell therapies can be applied.

    Loh ML, Tasian SK, Rabin KR, et al.
    A phase 1 dosing study of ruxolitinib in children with relapsed or refractory solid tumors, leukemias, or myeloproliferative neoplasms: A Children's Oncology Group phase 1 consortium study (ADVL1011).
    Pediatr Blood Cancer. 2015; 62(10):1717-24 [PubMed] Article available free on PMC after 01/04/2017 Related Publications
    BACKGROUND: Ruxolitinib, an orally bioavailable JAK1/JAK2 inhibitor, may treat cancers with CRLF2 and/or JAK pathway mutations.
    PROCEDURE: A phase 1 trial of ruxolitinib was performed to determine the maximum tolerated or recommended phase 2 dose, dose-limiting toxicities (DLTs), pharmacokinetics (PK), and pharmacodynamics (PD) in children with recurrent/refractory solid tumors (STs). Ruxolitinib was administered twice daily (BID) in 28-day cycles at five dose levels (15, 21, 29, 39, and 50 mg/m(2)/dose). PK and PD studies were performed during cycle 1. Toxicity, preliminary efficacy, and PK/PD were also assessed in children with relapsed/refractory hematologic malignancies (HMs).
    RESULTS: Forty-nine patients were enrolled, 28 with STs (dose escalation cohort) and 21 with HMs. Ruxolitinib was well-tolerated with one DLT per cohort of six patients at dose levels (DLs) 2-5. One patient with an ST had grade 5 multi-organ failure at DL2. One patient each at DL3 and DL4 had a grade 4 neutropenia, and one patient at DL5 had a grade 4 creatinine phosphokinase elevation. No objective responses were observed in patients with STs. One patient with polycythemia vera achieved a partial response and received 18 cycles of ruxolitinib. The PK of ruxolitinib were similar to that in adults. Partial inhibition of phosphorylated JAK2, STAT5, and S6 was observed in in vitro plasma inhibitory activity PD assay.
    CONCLUSION: Ruxolitinib was well tolerated in children with refractory cancer. The recommended phase 2 dose for continuous BID oral administration is 50 mg/m(2)/dose. Subsequent evaluation of ruxolitinib in combination with cytotoxic chemotherapy in children, adolescents, and young adults with JAK-mutant leukemias is planned.

    Freycon F, Trombert-Paviot B, Casagranda L, et al.
    Age at Birth of First Child and Fecundity of Women Survivors of Childhood Acute Lymphoblastic Leukemia (1987-2007): A Study of the Childhood Cancer Registry of the Rhône-Alpes Region in France (ARCERRA).
    Pediatr Hematol Oncol. 2015; 32(4):273-83 [PubMed] Related Publications
    We studied the fecundity of 174 successive ALL (1987-2007) in females of the Childhood Cancer Registry of the Rhône-Alpes Region (ARCERRA) with a median age at follow-up of 25.6 years (18.0-37.4). We distinguished five treatment groups: Group Ia, chemotherapy only (n = 130); Ib, chemotherapy with cranial radiotherapy (n = 10); II, TBI conditioning allograft (n = 27); III, chemotherapy conditioning allograft (n = 4); IV, TBI conditioning autograft (n = 3). Twenty-three women had their first child at the mean age of 25.8 ±3.0 years, i.e., 2.0 ±2.9 years earlier than the general population of the Rhône-Alpes region (P = 0.003). The standardized fertility ratio (SFR), expressed as the number of actual births observed (O) to the number that would be expected in women of the same age in the general population (E) (SFR = O/E) was decreased for Group Ia (0.62; 95%CI, 0.52-0.74) and collapsed in Group II (0.17; 0.11-0.25). In univariate analysis, TBI (P = 0.013) and alkylating agents (P = 0.01) were negatively correlated with fecundity, but not with the age at diagnosis or the anthracyclines doses. In multivariate analysis including TBI and alkylating agents, we still found a negative correlation between TBI (P = 0.035), as well as alkylating agents (P = 0.028), and fecundity. More precisely, fecundity was negatively correlated with cumulative cyclophosphamide equivalent dose (P = 0.001), with a fecundity decreased for ≥1g/m(2), but without any dose effect; results not found in the Group Ia. Age at first child seems younger but the young median age of the cohort not allows concluding; fecundity is collapsed after fractionated total body irradiation and decreased after chemotherapy without any demonstrable cause. A delay of fertility is not excluded.

    Daniel CL, Armstrong GT, Keske RR, et al.
    Advancing Survivors' Knowledge (ASK) about skin cancer study: study protocol for a randomized controlled trial.
    Trials. 2015; 16:109 [PubMed] Article available free on PMC after 01/04/2017 Related Publications
    BACKGROUND: Advances in treatment have increased childhood cancer 5-year survival rates to greater than 80%. However, children previously treated with radiation are at significantly increased risk of developing subsequent neoplasms, the most common of which are skin cancers. The National Cancer Institute and Children's Oncology Group have issued recommendations for survivors treated with radiation to perform monthly skin self-examinations and receive a physician skin examination at least annually, as early detection has demonstrated markedly improved outcomes in the diagnosis and treatment of skin cancers. The goal of the present study is to increase rates of skin self-examinations and clinical skin examinations among adult survivors of childhood cancer treated with radiation.
    METHODS/DESIGN: This randomized controlled trial uses a 3-group comparative effectiveness design comparing: (1) Patient Activation and Education (PAE) including text messaging, print and web-based tutorials over 12 months; (2) PAE plus physician activation (PAE + MD) adding physician activation/educational materials about survivors' increased skin cancer risk and conducting full-body skin exams; and (3) PAE plus physician activation, plus teledermoscopy (PAE + MD + TD) adding participant receipt of a dermatoscope intended to empower them to photograph suspect moles or lesions for review by the study dermatologist.
    DISCUSSION: The current study addresses barriers to screening in this population by providing educational and motivational information for both survivors and physicians regarding the value of periodic skin examinations. It also utilizes innovative mobile health technology to encourage and motivate (that is activate) survivors to conduct skin self-examinations, request physician exams, and obtain treatment when worrisome lesions are found. Finally, as a comparative effectiveness trial, this study isolates the effects of adding specific components to the patient activation intervention to test the most effective intervention for enhancing skin examination vigilance among this high-risk group.
    TRIAL REGISTRATION: Clinicaltrials.gov: NCT02046811 ; Registration date: 22 January 2014.

    Tantawy AA, Barakat MM, Adly AA, et al.
    One-Year Prospective Study of Community Acquired Influenza and Parainfluenza Viral Infections in Hospitalized Egyptian Children with Malignancy: Single Center Experience.
    Pediatr Hematol Oncol. 2015; 32(5):304-14 [PubMed] Related Publications
    BACKGROUND: Respiratory viruses are widespread in the community and easily transmitted to immunocompromised patients.
    AIMS: Assess the prevalence of community-acquired respiratory viral infections among children with cancer presenting with clinical picture suggestive of lower respiratory tract infections (LRTIs), and evaluate its risk factors and prognosis.
    METHODS: Over a year, 90 hospitalized children with malignancy and LRTIs recruited, subjected to clinical assessment, investigated through hematology panel, blood culture, chest x-ray, CT chest and PCR for influenza A and B, parainfluenza (PIV) types 1 and 3 viruses, and respiratory syncytial virus (RSV), and prospectively followed up for the clinical outcome.
    RESULTS: Viral pathogens were identified in 34 patients (37.7%), with a seasonal peak from April to May. The most frequently detected virus was influenza virus [type A (16 cases; 47%), type B (4 cases; 12%)] followed by parainfluenza virus [PIV1 (9 cases; 26%), PIV3 (3 cases; 15%)], and none had RSV. Bacteria were identified in 26 patients, fungi in four, mixed infections [bacterial/viral and bacterial/fungal] in 13, and 36 cases had unidentified etiology. The majority of patients with influenza and parainfluenza infections had hematological malignancy, presented with fever, and had mild self-limited respiratory illness. Five patients with mixed viral and bacterial infection had severe symptoms necessitating ICU admission. Six patients died from infection-related sequelae; two had mixed PIV and Staphylococcal infections.
    CONCLUSIONS: Community acquired influenza and parainfluenza infections are common in pediatrics patients with malignancy, either as isolated or mixed viral/bacterial infections. Clinical suspicion is essential as hematological and radiological manifestations are nonspecific. Rapid diagnosis and management are mandatory to improve patients' outcome.

    Pole JD, Darmawikarta D, Gassas A, et al.
    Subsequent malignant neoplasms in pediatric cancer patients treated with and without hematopoietic SCT.
    Bone Marrow Transplant. 2015; 50(5):721-6 [PubMed] Related Publications
    Pediatric cancer patients are at increased risk of subsequent malignant neoplasms (SMNs). However, little is known about the contribution of hematopoietic SCT (HSCT) to the development of SMNs. The objective of this study was to compare the incidence of SMNs in a population cohort of childhood cancer survivors treated with and without HSCT. A cohort of 7986 children (age 0-14 years) diagnosed with cancer in the province of Ontario, Canada between 1985 and 2009 was identified in POGONIS (Pediatric Oncology Group of Ontario Networked Information System), a population-based active cancer registry, and linked to a clinical HSCT database. Among this cohort, 796 patients had an HSCT as part of their primary treatment. Of the 375 allogeneic HSCT patients, 14 (3.7%) developed a SMN at a median follow-up of 12.3 years (range: 2.0-22.9 years). Of the 421 autologous HSCT patients, 8 (1.9%) developed a SMN at a median of 4.5 years (range: 1.3-14.3 years). Of the 7190 patients who did not receive an HSCT, 160 (2.2%) developed a SMN at a median follow-up of 6.8 years (range: 0.0-24.9 years). The 15-year cumulative incidence of SMN was 3.1% among the allogeneic HSCT group, 2.5% among the autologous group and 2.3% in the non-HSCT group. The cumulative incidence curves for the allogeneic HSCT and non-transplant groups only diverged after ~15 years from primary diagnosis. Our findings further corroborate the observation that children who undergo allogeneic HSCT are at a significantly increased risk of developing SMN compared with pediatric cancer survivors treated without HSCT.

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