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PURPOSE: Infant and young childhood medulloblastoma (iMB) is usually treated without craniospinal irradiation (CSI) to avoid neurocognitive late effects. Unfortunately, many children relapse. The purpose of this study was to assess salvage strategies and prognostic features of patients with iMB who relapse after CSI-sparing therapy. METHODS: We assembled a large international cohort of 380 patients with relapsed iMB, age younger than 6 years, and initially treated without CSI. Univariable and multivariable Cox models of postrelapse survival (PRS) were conducted for those treated with curative intent using propensity score analyses to account for confounding factors. RESULTS: The 3-year PRS, for 294 patients treated with curative intent, was 52.4% (95% CI, 46.4 to 58.3) with a median time to relapse from diagnosis of 11 months. Molecular subgrouping was available for 150 patients treated with curative intent, and 3-year PRS for sonic hedgehog (SHH), group 4, and group 3 were 60%, 84%, and 18% (P = .0187), respectively. In multivariable analysis, localized relapse (P = .0073), SHH molecular subgroup (P = .0103), CSI use after relapse (P = .0161), and age ≥ 36 months at initial diagnosis (P = .0494) were associated with improved survival. Most patients (73%) received salvage CSI, and although salvage chemotherapy was not significant in multivariable analysis, its use might be beneficial for a subset of children receiving salvage CSI < 35 Gy (P = .007). CONCLUSION: A substantial proportion of patients with relapsed iMB are salvaged after initial CSI-sparing approaches. Patients with SHH subgroup, localized relapse, older age at initial diagnosis, and those receiving salvage CSI show improved PRS. Future prospective studies should investigate optimal CSI doses and the role of salvage chemotherapy in this population.
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Neoplasias Encefálicas , Neoplasias Cerebelosas , Irradiación Craneoespinal , Meduloblastoma , Niño , Humanos , Lactante , Preescolar , Meduloblastoma/radioterapia , Estudios de Cohortes , Estudios Prospectivos , Irradiación Craneoespinal/efectos adversos , Proteínas Hedgehog , Recurrencia Local de Neoplasia , Neoplasias Encefálicas/terapia , Enfermedad Crónica , Neoplasias Cerebelosas/radioterapiaRESUMEN
PURPOSE: Intracranial growing teratoma syndrome (iGTS) is a rare phenomenon of paradoxical growth of a germ cell tumor (GCT) during treatment despite normalization of tumor markers. We sought to evaluate the frequency, clinical characteristics and outcome of iGTS in Western countries. METHODS: Pediatric patients from 22 North American and Australian institutions diagnosed with iGTS between 2000 and 2017 were retrospectively evaluated. RESULTS: From a total of 777 cases of central nervous system (CNS) GCT, 39 cases of iGTS were identified for an overall frequency of 5%. Pineal region was a more frequent location for iGTS as compared to cases of GCT without iGTS (p < 0.00001). In patients with an initial tissue diagnosis of GCT, immature teratoma was present in 50%. Serum AFP or ßhCG was detectable in 87% of patients (median values 66 ng/mL and 44 IU/L, respectively). iGTS occurred at a median of 2 months (range 0.5-32) from diagnosis, in the majority of patients. All patients underwent surgical resection, leading to gross total resection in 79%. Following surgery, all patients resumed adjuvant therapy or post treatment follow-up for GCT. At a median follow-up of 5.3 years (range 0.2-11.8), 37 (95%) of patients are alive, including 5 with stable residual mass. CONCLUSION: iGTS occurs in 5% of patients with GCT in Western countries. Tumors of the pineal region and GCT containing immature teratoma appear to be associated with a higher risk of developing iGTS. Complete surgical resection is the mainstay of treatment. Overall survival of patients developing iGTS remains favorable.
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Neoplasias de Células Germinales y Embrionarias/epidemiología , Teratoma/epidemiología , Adolescente , Adulto , Niño , Preescolar , Femenino , Humanos , Lactante , Masculino , Neoplasias de Células Germinales y Embrionarias/complicaciones , Pinealoma/complicaciones , Pinealoma/epidemiología , Estudios Retrospectivos , Teratoma/complicaciones , Resultado del Tratamiento , Adulto JovenRESUMEN
PURPOSE: Atypical teratoid/rhabdoid tumor (AT/RT) is an aggressive, early-childhood brain tumor without standard effective treatment. To our knowledge, we conducted the first AT/RT-specific cooperative group trial, ACNS0333, to examine the efficacy and safety of intensive postoperative chemotherapy and focal radiation to treat AT/RT. PATIENTS AND METHODS: Patients from birth to 22 years of age with AT/RT were eligible. After surgery, they received 2 courses of multiagent chemotherapy, followed by 3 courses of high-dose chemotherapy with peripheral blood stem cell rescue and involved-field radiation therapy. Timing of radiation was based on patient age and disease location and extent. Central testing of tumor and blood for SMARCB1 status was mandated. Tumor molecular subclassification was performed retrospectively. The primary analysis was event-free survival (EFS) for patients < 36 months of age compared with a cooperative groups' historical cohort. Although accrual was based on the therapeutic question, potential prognostic factors, including age, tumor location, M stage, surgical resection, order of therapy, germline status, and molecular subtype, were explored. RESULTS: Of 65 evaluable patients, 54 were < 36 months of age. ACNS0333 therapy significantly reduced the risk of EFS events in patients < 36 months of age compared with the historical cohort (P < .0005; hazard rate, 0.43; 95% CI, 0.28 to 0.66). Four-year EFS and overall survival for the entire cohort were 37% (95% CI, 25% to 49%) and 43% (95% CI, 31% to 55%), respectively. Timing of radiation did not affect survival, and 91% of relapses occurred by 2 years from enrollment. Treatment-related deaths occurred in 4 patients. CONCLUSION: The ACNS0333 regimen dramatically improved survival compared with historical therapies for patients with AT/RT. Clinical characteristics and molecular subgrouping suggest prognostic differences. ACNS0333 results lay a foundation on which to build future studies and incorporate testing of new therapeutic agents.
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Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Radioterapia Conformacional/métodos , Tumor Rabdoide/terapia , Teratoma/terapia , Adolescente , Protocolos de Quimioterapia Combinada Antineoplásica/efectos adversos , Niño , Preescolar , Terapia Combinada , Femenino , Humanos , Lactante , Recién Nacido , Masculino , Pronóstico , Tumor Rabdoide/genética , Tumor Rabdoide/mortalidad , Proteína SMARCB1/genética , Teratoma/genética , Teratoma/mortalidad , Adulto JovenRESUMEN
PROBLEM: Compassion has been described as a central construct or essential feature of quality healthcare and is as important to patients' and families' overall healthcare experience as the health interventions and treatments they receive. However, there is little shared understanding of what constitutes compassion, how it is delivered within a pediatric setting, and pediatric patients' and families perspectives and preferences for receiving it. ELIGIBILITY CRITERIA: Studies that (1) described the nature of the existing literature on compassion in pediatric healthcare; (2) summarized key concepts in the existing evidence base that pertain to compassion in pediatric healthcare; and 3) identified factors that are associated with compassion in pediatric healthcare were eligible for inclusion in this review. SAMPLE: Twenty-nine papers were included in the review. RESULTS: Findings revealed several factors are associated with compassion in pediatric healthcare, including continuity of care, communication, and coordination of care. Most notably, identified studies treated compassion in a subsidiary fashion, and this review revealed no studies that provided a patient-informed evidence-based definition of compassion in the pediatric healthcare setting. CONCLUSION: Future research is required to generate a comprehensive and accurate understanding of the terms 'compassion' and 'compassionate care' when used in the context of pediatric healthcare. IMPLICATIONS: This research will inform the therapeutic processes and ultimately enable the development of strategies to improve the delivery of compassionate healthcare to pediatric patients.
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Actitud del Personal de Salud , Empatía , Calidad de la Atención de Salud , Niño , Comunicación , Personal de Salud , Humanos , Investigación CualitativaRESUMEN
PURPOSE: Nodular desmoplastic medulloblastoma (ND) and medulloblastoma with extensive nodularity (MBEN) have been associated with a more favorable outcome in younger children. However, treatment-related neurotoxicity remains a significant concern in this vulnerable group of patients. PATIENTS AND METHODS: ACNS1221 was a prospective single-arm trial of conventional chemotherapy for nonmetastatic ND and MBEN based on a modified HIT SKK 2000 regimen excluding intraventricular methotrexate, aiming to achieve similar outcome (2-year progression-free survival [PFS] ≥ 90%) with reduced treatment-related neurotoxicity. Secondary objectives included feasibility of timely central pathology review and evaluation of tumor molecular profile. RESULTS: Twenty-five eligible patients (15 males and 10 females; median age, 18.7 months) were enrolled. Eighteen patients had ND and 7 had MBEN histology. Three patients had residual disease at baseline. The study closed early because of a higher than expected relapse rate. Twelve patients experienced relapse-local (n= 6), distant (n = 3), and combined (n = 3)-at a median of 9.8 months from diagnosis (range, 8.9-13.7 months), and 2 patients died of disease. Two-year PFS and overall survival rates were 52% (95% CI, 32.4% to 71.6%) and 92% (95% CI, 80.8% to 100.0%) respectively. Patients older than 12 months of age (P = .036) and ND histology (P = .005) were associated with worse PFS. No patients with MBEN histology experienced relapse. All tumor samples clustered within the sonic hedgehog (SHH) group. Methylation analysis delineated 2 subgroups, SHH-I and SHH-II, which were associated with 2-year PFS rates of 30.0% (95% CI, 1.6% to 58.4%) and 66.7% (95% CI, 44.0% to 89.4%), respectively (P = .099). CONCLUSION: The proposed modified regimen of conventional systemic chemotherapy without serial intraventricular methotrexate injection failed to achieve the targeted 2-year PFS of 90%. With this cohort, we prospectively confirmed the existence of two SHH subgroups and observed a trend toward worse outcome for SHH-I patients.
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Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Neoplasias Cerebelosas/tratamiento farmacológico , Neoplasias Cerebelosas/patología , Meduloblastoma/tratamiento farmacológico , Meduloblastoma/patología , Neoplasias Cerebelosas/mortalidad , Preescolar , Femenino , Humanos , Lactante , Masculino , Meduloblastoma/mortalidad , Supervivencia sin ProgresiónRESUMEN
Despite the availability of tools to assess psychosocial screening in pediatric oncology, little is known about the feasibility and acceptability of systematic screening. We aimed to assess the feasibility of implementing a tool, or set of tools, capable of screening for psychosocial distress in pediatric cancer patients across the cancer continuum (on treatment, off treatment). Psychometric criteria were also evaluated. Patients 8-18 years were recruited from a pediatric oncology program. Patients completed self-report measures of the Distress Thermometer (DT) and Pediatric Quality of Life Inventory (PedsQL). One parent of each patient completed three screening tools: DT (proxy-report); PedsQL (proxy-report), and the Psychosocial Assessment Tool adapted for the Canadian context (PATrev), as well as a measure of patient psychological functioning (Behavioral Assessment System for Children-2), and an assessment of screening tool acceptability. Recruitment rates and acceptability informed feasibility of implementation. Ninety-five patients (58 men) with a mean age of 11.47 participated in the study (on treatment, n = 43; off treatment, n = 52). Recruitment rates were on treatment: 56.6% and off treatment: 47.3%. Mean acceptability scores of tools ranged from 3.41 to 4.97 out of 7. Screening tools were comparable with respect to their psychometric properties. The DT took the least amount of time to complete, while the PATrev offered the most robust data with respect to psychometrics. Feasibility of screening for psychosocial distress with our tool was moderate and may be enhanced when administered by a known health-care provider. Future research exploring how to further enhance feasibility of implementation for pediatric cancer patients is warranted.
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Neoplasias/psicología , Calidad de Vida , Estrés Psicológico/diagnóstico , Estrés Psicológico/psicología , Adolescente , Niño , Estudios de Factibilidad , Femenino , Humanos , Masculino , Neoplasias/epidemiología , Estrés Psicológico/epidemiologíaRESUMEN
Pediatric glioblastoma (pGBM) is a lethal cancer with no effective therapies. To understand the mechanisms of tumor evolution in this cancer, we performed whole-genome sequencing with linked reads on longitudinally resected pGBM samples. Our analyses showed that all diagnostic and recurrent samples were collections of genetically diverse subclones. Clonal composition rapidly evolved at recurrence, with less than 8% of nonsynonymous single-nucleotide variants being shared in diagnostic-recurrent pairs. To track the origins of the mutational events observed in pGBM, we generated whole-genome datasets for two patients and their parents. These trios showed that genetic variants could be (i) somatic, (ii) inherited from a healthy parent, or (iii) de novo in the germlines of pGBM patients. Analysis of variant allele frequencies supported a model of tumor growth involving slow-cycling cancer stem cells that give rise to fast-proliferating progenitor-like cells and to nondividing cells. Interestingly, radiation and antimitotic chemotherapeutics did not increase overall tumor burden upon recurrence. These findings support an important role for slow-cycling stem cell populations in contributing to recurrences, because slow-cycling cell populations are expected to be less prone to genotoxic stress induced by these treatments and therefore would accumulate few mutations. Our results highlight the need for new targeted treatments that account for the complex functional hierarchies and genomic heterogeneity of pGBM. SIGNIFICANCE: This work challenges several assumptions regarding the genetic organization of pediatric GBM and highlights mutagenic programs that start during early prenatal development.Graphical Abstract: http://cancerres.aacrjournals.org/content/canres/79/9/2111/F1.large.jpg.
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Biomarcadores de Tumor/genética , Neoplasias Encefálicas/genética , Glioblastoma/genética , Mutación , Recurrencia Local de Neoplasia/genética , Células Madre Neoplásicas/metabolismo , Animales , Neoplasias Encefálicas/patología , Niño , Perfilación de la Expresión Génica , Glioblastoma/patología , Humanos , Estudios Longitudinales , Ratones , Recurrencia Local de Neoplasia/patología , Células Madre Neoplásicas/patología , Células Tumorales Cultivadas , Secuenciación Completa del Genoma , Ensayos Antitumor por Modelo de XenoinjertoRESUMEN
BACKGROUND: It is questionable whether enrollment on clinical trials offers any survival advantage at the population level over standard-of-care treatment. The objectives of this study were to describe the impact of trial enrollment on event-free survival and overall survival in pediatric acute myeloid leukemia (AML) using the Cancer in Young People in Canada (CYP-C) database. METHODS: Children were included if they had had AML newly diagnosed between ages birth and 14 years from 2001 to 2012. CYP-C is a national pediatric cancer population-based database that includes all cases of pediatric cancer diagnosed and treated at 1 of the 17 tertiary pediatric oncology centers in Canada. Univariate and Cox proportional hazards models were used to evaluate the impact of initial trial enrollment on survival. RESULTS: In total, 397 eligible children with AML were included in the analysis, of whom 94 (23.7%) were enrolled on a clinical trial at initial diagnosis. The most common reason for non-enrollment was that no trial was available. The event-free survival rate at 5 years was 57.8% ± 5.2% for those enrolled versus 54.8% ± 2.9% for those not enrolled (P = .75). The overall survival rate at 5 years was 70.1% ± 4.9% for those enrolled versus 66.3% ± 2.8% for those not enrolled (P = .58). Enrollment on a trial was not associated with improved event-free or overall survival in multiple regression analyses. CONCLUSIONS: Enrollment on a clinical trial was not associated with improved survival for children with AML in a population-based cohort. Rationale for trial enrollment should not include the likelihood of benefit compared with non-enrollment.
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Ensayos Clínicos como Asunto/estadística & datos numéricos , Leucemia Mieloide Aguda/mortalidad , Leucemia Mieloide Aguda/terapia , Selección de Paciente , Adolescente , Edad de Inicio , Niño , Preescolar , Supervivencia sin Enfermedad , Femenino , Humanos , Lactante , Recién Nacido , Masculino , Análisis de Supervivencia , Estados Unidos/epidemiologíaRESUMEN
PURPOSE: Prospective and longitudinal neuroimaging studies of posterior fossa tumors are scarce. Here we evaluate the early changes in white matter and intellectual outcome up to 3â¯years after diagnosis. PATIENTS AND METHODS: Twenty-two children with posterior fossa tumors and 24 similarly-aged healthy children participated. Patients included: (a) 12 individuals who received surgery, cranial-spinal radiation (CSR), and focal radiation to the tumor bed (CSR group) and (b) 10 individuals who received local therapy, either surgery only or surgery and focal radiation to the tumor bed (Local group). Diffusion tensor imaging (DTI) and intelligence measures were obtained an average of 3â¯months after diagnosis and then at 12, 24, and 36â¯months later. DTI tractography and voxel-wise approaches were employed. The Neurological Predictor Scale was used to summarize the type and amount of treatment for PF tumor patients. Linear mixed modelling was used to evaluate group differences at baseline and changes over time in DTI metrics for both the specific white matter tracts and voxel-wise, as well as for intelligence measures. RESULTS: Based on tractography, patients treated with CSR had significantly higher Axial and Mean diffusivity in the cortical-spinal tracts (CST) 3â¯month after diagnosis - particularly on the right side, pâ¯<â¯.003, compared to healthy children. Mean diffusivity in right CST decreased over time in this group of patients, pâ¯=â¯.001. No differences compared to controls were evident in specific tracts for the Local group, pâ¯>â¯.10. Voxel-wise analyses revealed multiple areas of white matter compromise in both patients groups. Notably, both patient groups had lower scores on intelligence measures compared to the Control group: The CSR group displayed lower performance 3â¯months following diagnosis, psâ¯<â¯0.001, and their performance remained stable over time psâ¯>â¯0.10, whereas the Local group displayed no differences at 3â¯months, ps>â¯0.10, but their performance declined over time, psâ¯<â¯0.01. At baseline, higher MD in right CST predicted lower Perceptual Reasoning scores across all participants, pâ¯=â¯.001. Furthermore, lower FA in left IFOF at baseline predicted decline in Processing Speed over time, pâ¯=â¯.001. In patients, more aggressive treatment protocols and presence of mutism were related to lower performance on intelligence measures at baseline, psâ¯<â¯0.04. CONCLUSIONS: Children treated with CSR displayed diffuse white matter compromise and poor intellectual outcome shortly after radiation treatment. There was evidence of subsequent growth of white matter structure, but stable intellectual insult. Conversely, in children treated with either surgery only or surgery and focal radiation to the tumor bed we observed less compromise of white matter early following treatment and no intellectual insult compared to healthy children. However, declines in intellectual function were evident for these children, though their performance remained within the average normative range. Overall, results suggest that early intervention is necessary to circumvent these deficits.
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Encéfalo/patología , Neoplasias Infratentoriales/patología , Neoplasias Infratentoriales/psicología , Inteligencia , Sustancia Blanca/patología , Adolescente , Encéfalo/diagnóstico por imagen , Niño , Imagen de Difusión Tensora , Femenino , Humanos , Neoplasias Infratentoriales/diagnóstico por imagen , Pruebas de Inteligencia , Estudios Longitudinales , Imagen por Resonancia Magnética , Masculino , Pronóstico , Estudios Prospectivos , Sustancia Blanca/diagnóstico por imagenRESUMEN
BACKGROUND: The purpose of this study was to examine the prevalence and predictors of social difficulties in adolescent survivors of central nervous system (CNS) tumors. METHODS: Six hundred sixty-five survivors of CNS tumors (53.8% male and 51.7% treated with cranial radiation therapy [CRT]), who had a current median age of 15.0 years (range, 2.0-17.0 years) and were a median of 12.1 years (range, 8.0-17.7 years) from their diagnosis, were compared with 1376 survivors of solid tumors (50.4% male), who had a median age of 15.0 years (range, 12.0-17.0 years) and were a median of 13.2 years (range, 8.3-17.9 years) from their diagnosis, and 726 siblings (52.2% male), who had a median age of 15.0 years (range, 12.0-17.0 years). Social adjustment was measured with parent-proxy responses to the Behavior Problems Index. Latent profile analysis defined social classes. Multinomial logistic regression, adjusted for age, sex, and age at diagnosis, identified predictors of class membership. Path analyses tested mediating effects of physical limitations, sensory loss, and cognitive impairment on social outcomes. RESULTS: Caregivers reported that survivors of CNS tumors were more likely to have 0 friends (15.3%) and to interact with friends less than once per week (41.0%) in comparison with survivors of solid tumors (2.9% and 13.6%, respectively) and siblings (2.3% and 8.7%, respectively). Latent profile analysis identified 3 social classes for survivors of CNS tumors: well-adjusted (53.4%), social deficits (16.2%), and poor peer relationships (30.4%). However, 2 classes were identified for survivors of solid tumors and siblings: well-adjusted (86.2% and 91.1%, respectively) and social deficits (13.8% and 8.9%, respectively). CRT predicted class membership for CNS survivors (odds ratio [OR] for poor peer relationships, 1.16/10 Gy; 95% confidence interval [CI], 1.08-1.25; OR for social deficits 1.14/10 Gy; 95% CI, 1.04-1.25; reference, well-adjusted). Cognitive impairment mediated the association between all social outcomes and CRT (P values < .001). CONCLUSION: Almost 50% of survivors of CNS tumors experience social difficulties; the pattern is unique in comparison with solid tumor and sibling groups. Cognitive impairment is associated with increased risk, and this highlights the need for multitargeted interventions.
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Conducta del Adolescente , Supervivientes de Cáncer/psicología , Neoplasias del Sistema Nervioso Central/epidemiología , Neoplasias del Sistema Nervioso Central/psicología , Ajuste Social , Adolescente , Conducta del Adolescente/psicología , Edad de Inicio , Supervivientes de Cáncer/estadística & datos numéricos , Estudios de Casos y Controles , Neoplasias del Sistema Nervioso Central/radioterapia , Niño , Irradiación Craneana/efectos adversos , Irradiación Craneana/estadística & datos numéricos , Femenino , Humanos , Neoplasias Renales/epidemiología , Neoplasias Renales/psicología , Masculino , Neuroblastoma/epidemiología , Neuroblastoma/psicología , Factores de Riesgo , Hermanos , Tumor de Wilms/epidemiología , Tumor de Wilms/psicologíaRESUMEN
PURPOSE: The aim of the study was to explore the relationship between repressive adaptive style and self-reports of social adjustment in survivors of pediatric cancer compared to their siblings. We hypothesized that there would be a greater proportion of repressors among survivors of pediatric cancer compared to siblings, and that repressive adaptive style would be significantly associated with more positive self-reports of social adjustment. METHODS: We utilized a cross-sectional approach. Seventy-seven families participated. Survivors of pediatric cancer (n = 77, 48% male; 8-18 years of age) and one sibling (n = 50, 48% male; 8-18 years of age) completed measures assessing repressive adaptive style and social adjustment. As well, one parent from each family completed a socio-demographic questionnaire. Questionnaire packages were mailed to eligible families who agreed to participate, and were mailed back to investigators in a pre-addressed, pre-stamped envelope. RESULTS: Chi-square analyses revealed there was no significant difference in the proportion of repressors among survivors and siblings. Social adjustment scores were subjected to a two (group: survivor, sibling) by two (repressor, nonrepressor) ANCOVA with gender and age as covariates. There was a significant main effect of repressive adaptive style (F = 5.69, p < .05, η2 = 0.05) with a modest effect. Survivors and siblings with a repressive style reported significantly higher social adjustment scores (M = 106.91, SD = 11.69) compared to nonrepressors (M = 99.57, SD = 13.45). CONCLUSIONS: Repressive adaptive style explains some of the variance in survivors and siblings' self-reports of social adjustment. Future research should aim to better understand the role of the repressive adaptive style in survivors and siblings of children with cancer.
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Adaptación Psicológica , Supervivientes de Cáncer/psicología , Ajuste Social , Adolescente , Supervivientes de Cáncer/estadística & datos numéricos , Niño , Estudios Transversales , Femenino , Humanos , Masculino , Autoinforme , Hermanos/psicologíaRESUMEN
Medulloblastoma is the most common malignant brain tumor in children. Published survival rates for this tumor are â¼70%; however, there is limited published information on outcome after disease recurrence. This was an observational study which included all persons under the age of 18 years diagnosed with medulloblastoma from 1990 to 2009 inclusive in Canada. Data collected included date of diagnosis, age at diagnosis, sex, stage, pathology, treatment, recurrence, and current status. Survival rates were determined. In total, 550 cases were ascertained meeting the study criteria. The overall survival rate at 1 year was 83.6%±1.7%, at 3 years 77.2%±1.9%, and at 5 years 72.5%±20%. The progression-free survival rates were 78%±1.9%, 70%±2.1%, and 69±2.1% at 1, 3, and 5 years from initial diagnosis. In total, 173 (31.2%) were reported to have had tumor recurrence and 23 (11.4%) of them were alive at the time of survey with an overall survival rate at 1 year of 38.3%±4%, at 2 years of 16.9%±3.3%, and at 5 years of 12.4%±2.8%. Our data confirm that children with recurrent medulloblastoma have a poor prognosis, supporting the need for novel treatment approaches for this group.
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Neoplasias Cerebelosas/mortalidad , Meduloblastoma/mortalidad , Recurrencia Local de Neoplasia/mortalidad , Adolescente , Canadá/epidemiología , Niño , Preescolar , Femenino , Humanos , Lactante , MasculinoRESUMEN
OBJECTIVE: Clinical trials have failed to demonstrate a survival benefit of adjuvant chemotherapy in diffuse intrinsic pontine gliomas (DIPG). Radiation therapy (RT) is the only effective treatment thus far and reirradiation (rRT) has become an option at the time of progression. The aim of this study was to review the Canadian experience of DIPG rRT with a focus on the safety and possible efficacy of this approach. METHOD: We retrospectively reviewed the demographic, clinical, and RT data of patients with DIPG treated in Canada with rRT. RESULTS: Since January 2011, we identified 16 patients with progressive DIPG who received rRT. Median time from diagnosis to progression was 10.5 months (range, 4-37 months). rRT was given focally in 14 patients at a dose ranging from 21.6 to 36 Gy. rRT was well tolerated by all children but one. All but three patients showed neurological improvement. With a median follow-up from original diagnosis of 19.2 months, all patients died, with a median time from rRT to death of 6.48 months (range, 3.83-13.26 months). When compared to a historic cohort of 46 consecutive patients, the median time from progression to death was 92 days in the non-reirradiated patients versus 218 days in the reirradiated ones (P = 0.0001). CONCLUSION: In this limited experience, rRT was safe and feasible in patients with progressive DIPG, providing neurological improvement and a prolonged life span in most patients. Prospective Canadian rRT protocols are ongoing to further assess the benefit of this approach, including quality of life assessment.
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Neoplasias del Tronco Encefálico/radioterapia , Glioma/radioterapia , Calidad de Vida , Reirradiación , Adolescente , Neoplasias del Tronco Encefálico/patología , Canadá , Niño , Preescolar , Progresión de la Enfermedad , Femenino , Estudios de Seguimiento , Glioma/patología , Humanos , Masculino , Pronóstico , Estudios RetrospectivosRESUMEN
BACKGROUND: The objectives of this study were to describe the impact of trial enrollment at diagnosis on event-free and overall survival in paediatric acute lymphoblastic leukaemic (ALL) using a population-based approach. METHODS: We conducted a retrospective cohort study that included children newly diagnosed with ALL between 1 and 14 years of age. The data source was the Cancer in Young People in Canada (CYP-C) national paediatric cancer population-based database. We conducted univariate and multiple Cox proportional hazards models. RESULTS: There were 2569 children with ALL; 1408 (54.8%) were enrolled on a clinical trial at initial diagnosis. Event-free survival at 5 years was 89.8%±0.9 vs 84.1%±1.2. (P<0.0001) for those enrolled and not enrolled on a clinical trial, respectively. Overall survival at 5 years was higher for those enrolled (94.1%±0.7) vs not enrolled (90.5%±1.0; P=0.001). In a model that adjusted for demographic, leukaemic and socioeconomic factors, enrollment on trials was significantly associated with better event-free survival (hazard ratio (HR) 0.67, 95% confidence interval (CI) 0.47-0.95; P=0.023), but not overall survival (HR 0.69, 95% CI 0.44-1.08; P=0.102). CONCLUSIONS: Event-free survival was significantly better in children with ALL enrolled on a clinical trial. Future research should identify barriers to clinical trial enrollment for children with ALL.
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Selección de Paciente , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Adolescente , Canadá , Niño , Preescolar , Ensayos Clínicos como Asunto , Bases de Datos Factuales , Femenino , Humanos , Lactante , Masculino , Supervivencia sin Progresión , Proyectos de Investigación , Estudios Retrospectivos , Resultado del TratamientoRESUMEN
BACKGROUND: To determine if a group social skills intervention program improves social competence and quality of life (QOL) in pediatric brain tumor survivors (PBTS). METHODS: We conducted a randomized control trial in which PBTS (8-16 years old, off therapy for over 3 months) were allocated to receive social skills training (eg, cooperation, assertion, using social cognitive problem solving strategies, role playing, games, and arts and crafts) in 8 weekly 2-hour sessions, or an attention placebo control (games and arts and crafts only). Outcomes were self-reported, proxy-reported (caregiver), and teacher-reported using the Social Skills Rating System (SSRS), to measure social competence, and the Pediatric Quality of Life (PedsQL4.0, generic) to measure QOL at baseline, after intervention, and at 6 months follow-up. At baseline, SSRS were stratified into low and high scores and included as a covariate in the analysis. RESULTS: Compared to controls (n = 48), PBTS in the intervention group (n = 43) reported significantly better total and empathy SSRS scores, with improvements persisting at follow-up. The PBTS in the intervention group who had low scores at baseline reported the greatest improvements. Proxy and teacher reports showed no intervention effect. CONCLUSIONS: Participating in group social skills intervention can improve self-reported social competence that persisted to follow up. The PBTS should be given the opportunity to participate in social skills groups to improve social competence.
Asunto(s)
Terapia Conductista/métodos , Neoplasias Encefálicas/psicología , Cuidadores/psicología , Ajuste Social , Conducta Social , Habilidades Sociales , Sobrevivientes/psicología , Adolescente , Neoplasias Encefálicas/mortalidad , Niño , Práctica Clínica Basada en la Evidencia , Femenino , Humanos , Relaciones Interpersonales , Solución de Problemas , Calidad de Vida/psicologíaRESUMEN
This qualitative study employed hermeneutic phenomenology and narrative inquiry to examine the topic of sexuality and adolescents with cancer from the perspectives of survivors who had experienced cancer as adolescents. This investigation examined the potentially sensitive, disquieting, and often taboo issue of sexuality in the interest of optimizing wellness in young people, and, ultimately, in the health of adults. Understanding the adolescent body as a sensitive, sexual, and developing self can enrich our understanding of adolescent cancer and promote best health care and practices, examining ways that we might mitigate the long-term effects of arrested or delayed development of sexual identity. In this article, we discuss phase 1 of the study, which used hermeneutics as the method of inquiry. Findings included a general experience of adolescents having a sense of "losing themselves" while at the same time finding themselves in a new light. Other findings include the connection between sexuality, self, and identity; the unique "tribe" of adolescents with cancer; the necessity for sexuality to take a backseat to cancer; the changing mirror images from self and others; sexuality and fertility; and, ultimately, that sexuality is a relational experience.