RESUMEN
BACKGROUND: Outcomes for children with high-risk renal (HRR) and INI-1-deficient (INI-) tumors are unacceptably poor. Concerns about excessive toxicity-as many are infants and/or undergo nephrectomy-have resulted in decreased chemotherapy dosing and omission of the nephrotoxic drug ifosfamide in collaborative group studies. As cause of death for children with these cancers remains overwhelmingly more from progressive disease rather than treatment toxicity, we examined the tolerability of an intensive ifosfamide-containing regimen. PROCEDURE: Retrospective review of children with HRR/INI- tumors treated at a single institution with vincristine, doxorubicin, cyclophosphamide alternating with ifosfamide, carboplatin, etoposide (VDC-ICE) from 2006-2016. The primary outcome was regimen tolerability, including kidney injury and grade 3-5 nonhematologic toxicities. RESULTS: Fourteen patients with a median age of 1.7 years (range: 0.1-10.5) treated with VDC-ICE were identified. Diagnosis included malignant rhabdoid tumor (n = 9) (primary renal [n = 2]); diffuse anaplastic Wilms tumor (n = 3); clear cell sarcoma of the kidney (n = 1); and anaplastic chordoma (n = 1). All children with primary renal tumors (43%) underwent complete (n = 5) or partial nephrectomy (n = 1) before chemotherapy. Nine (64%) completed all intended cycles of chemotherapy; n = 5 (36%) did not due to disease progression. Unplanned hospitalizations occurred in 13 (93%) patients, most commonly for febrile neutropenia. No patient experienced severe organ toxicity, diminished renal function, treatment discontinuation due to toxicities, or treatment-related death. CONCLUSIONS: In children with HRR/INI- tumors, VDC-ICE chemotherapy was well-tolerated without excessive toxicities, even amongst young patients with solitary kidneys. Concerns about toxicity should not preclude an intensive ifosfamide-containing regimen from use in future trials in this population.
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Ifosfamida , Neoplasias , Niño , Lactante , Humanos , Preescolar , Protocolos de Quimioterapia Combinada Antineoplásica/efectos adversos , Neoplasias/tratamiento farmacológico , Ciclofosfamida , Etopósido , Carboplatino , Vincristina , Riñón/fisiologíaRESUMEN
PURPOSE: We sought to characterize clinical outcomes for adult and pediatric patients with primary CNS tumors harboring DICER1 mutations or loss of DICER1. METHODS: We conducted a retrospective cohort study of 98 patients who were treated between 1995 and 2020 for primary CNS tumors containing DICER1 mutations or loss of DICER1 on chromosome 14q, identified by targeted next generation sequencing. Kaplan-Meier plots and log rank tests were used to analyze survival. Cox proportional-hazards model was used for univariate and multivariable analyses for all-cause mortality (ACM). RESULTS: Within our cohort, the most common malignancies were grade 3/4 glioma (61%), grade 1/2 glioma (17%), and CNS sarcoma (6%). Sarcoma and non-glioma histologies, and tumors with biallelic DICER1 mutations or deletions were common in the pediatric population. Mutations occurred throughout DICER1, including missense mutations in the DexD/H-box helicase, DUF283, RNaseIIIa, and RNaseIIIb domains. For patients with grade 3/4 glioma, MGMT methylation (Hazard ratio [HR] 0.35, 95% Confidence Interval [CI] 0.16-0.73, p = 0.005), IDH1 R132 mutation (HR 0.11, 95% CI 0.03-0.41, p = 0.001), and missense mutation in the DexD/H-box helicase domain (HR 0.06, 95% CI 0.01-0.38, p = 0.003) were independently associated with longer time to ACM on multivariable analyses. CONCLUSION: DICER1 mutations or loss of DICER1 occur in diverse primary CNS tumors, including previously unrecognized grade 3/4 gliomas as the most common histology. While prior studies have described RNaseIIIb hotspot mutations, we document novel mutations in additional DICER1 functional domains. Within the grade 3/4 glioma cohort, missense mutation in the DexD/H-box helicase domain was associated with prolonged survival.
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Neoplasias del Sistema Nervioso Central , Glioma , Sarcoma , Adulto , Neoplasias del Sistema Nervioso Central/genética , Niño , ARN Helicasas DEAD-box/genética , Glioma/patología , Humanos , Mutación , Pronóstico , Estudios Retrospectivos , Ribonucleasa III/genética , Sarcoma/patologíaRESUMEN
Central nervous system (CNS) metastases are rare, but devastating complications of pediatric solid tumors. Radiotherapy alone or postresection serves as an important treatment; however, data on the use of whole-brain radiotherapy (WBRT) versus focal radiotherapy, including stereotactic radiosurgery or stereotactic radiotherapy, for these indications are limited. We report a single institution experience of 26 pediatric patients treated with radiotherapy for solid tumor CNS metastases without leptomeningeal disease. Focal radiotherapy (n = 10) was well tolerated and survival outcomes did not differ between patients treated with WBRT (n = 16) versus focal radiation, suggesting that focal radiotherapy may be considered for patients with limited CNS metastases.
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Neoplasias Encefálicas , Neoplasias Meníngeas , Neoplasias Primarias Secundarias , Radiocirugia , Neoplasias Encefálicas/patología , Sistema Nervioso Central/patología , Niño , Irradiación Craneana/efectos adversos , Humanos , Neoplasias Primarias Secundarias/etiología , Radiocirugia/efectos adversosRESUMEN
The survival of patients with acute lymphoblastic leukemia (ALL) has improved significantly with the use of intensive multimodality treatment regimens including chemotherapy, high-dose chemotherapy and stem cell rescue, and radiation therapy when indicated. This report summarizes the treatment strategies, especially radiation therapy in the Children's Oncology Group for children with ALL. Currently, radiation therapy is only indicated for children with high-risk CNS involvement at diagnosis or relapse, testicular relapse and as part of the conditioning regimen for hematopoietic stem cell transplantation. Future research strategies regarding the indications for and dosages of radiation therapy and novel radiation techniques are discussed.
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Trasplante de Células Madre Hematopoyéticas/métodos , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Irradiación Corporal Total/métodos , Niño , Terapia Combinada , Humanos , Leucemia-Linfoma Linfoblástico de Células Precursoras/patología , Pronóstico , Tasa de SupervivenciaRESUMEN
The clinical outcomes for infants with malignant tumors are often worse than older children due to a combination of more biologically aggressive disease in some cases, and increased toxicity-or deintensification of therapies due to concern for toxicity-in others. Especially in infants and very young children, finding the appropriate balance between maximizing treatment efficacy while minimizing toxicity-in particular late side effects-is crucial. We review here the management of malignant tumors in infants and very young children, focusing on central nervous system (CNS) malignancies and rhabdomyosarcoma.
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Neoplasias del Sistema Nervioso Central/radioterapia , Rabdomiosarcoma/radioterapia , Neoplasias del Sistema Nervioso Central/psicología , Niño , Humanos , Recién Nacido , Resultado del TratamientoRESUMEN
Radiation therapy (RT) is often used as a palliative treatment for children with recurrent malignant disease to ameliorate or prevent symptoms. However, no guidelines exist regarding the clinical indications or dose fractionation for palliative RT. The goal of this report is to provide guidelines for the use of palliative RT in children with cancer. In this guideline, appropriate indications for palliative RT, recommended dose-fractionation schedules, relevant toxicities, and avenues for future research are explored. RT is an effective palliative treatment for bone, brain, liver, lung, abdominopelvic and head-and-neck metastases, spinal cord compression, superior vena cava syndrome, and bleeding. Single-fraction regimens (8 Gy in one fraction) for children with short life expectancy are recommended for simple, uncomplicated bone metastases and can be considered for some patients with lung or liver metastases. A short, hypofractionated regimen (20 Gy in five fractions) may be used for other indications to minimize overall burden of therapy. There are little data supporting use of more prolonged fractionation regimens, though they may be considered for patients with very good performance status. Future research should focus on response and outcomes data collection, and to rigorously evaluate the role of stereotactic body RT in well-designed, prospective studies.
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Recurrencia Local de Neoplasia/prevención & control , Neoplasias/radioterapia , Cuidados Paliativos/métodos , Radioterapia/métodos , Niño , Humanos , Neoplasias/patología , Pronóstico , Tasa de SupervivenciaRESUMEN
BACKGROUND: Nonmalignant vascular anomalies (VA) comprise a heterogeneous spectrum of conditions characterized by aberrant growth or development of blood and/or lymphatic vessels and can cause significant morbidity. Little is known about outcomes after radiotherapy in pediatric and young adult patients with nonmalignant VA. METHODS: Thirty patients who were diagnosed with nonmalignant VA and treated with radiotherapy prior to 2017 and before the age of 30 were identified. Clinical and treatment characteristics and outcomes were recorded. RESULTS: Median age at first radiotherapy was 15 years (range 0.02-27). Median follow-up from completion of first radiotherapy was 9.8 years (range 0.02-67.4). Lymphatic malformations (33%), kaposiform hemangioendothelioma (17%), and venous malformations (17%) were the most common diagnoses. The most common indication for first radiotherapy was progression despite standard therapy and/or urgent palliation for symptoms (57%). After first radiotherapy, 14 patients (47%) had a complete response or partial response, defined as decrease in size of treated lesion or symptomatic improvement. After first radiotherapy, 27 (90%) required additional treatment for progression or recurrence. Long-term complications included telangiectasias, fibrosis, xerophthalmia, radiation pneumonitis, ovarian failure, and central hypothyroidism. No patient developed secondary malignancies. At last follow-up, three patients (10%) were without evidence of disease, 26 (87%) with disease, and one died of complications (3.3%). CONCLUSIONS: A small group of pediatric and young adult patients with nonmalignant, high-risk VA experienced clinical benefit from radiotherapy with expected toxicity; however, most experienced progression. Prospective studies are needed to characterize indications for radiotherapy in VA refractory to medical therapy, including targeted inhibitors.
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Radioterapia , Adolescente , Adulto , Niño , Preescolar , Hemangioendotelioma , Humanos , Lactante , Recién Nacido , Síndrome de Kasabach-Merritt , Anomalías Linfáticas , Estudios Retrospectivos , Sarcoma de Kaposi , Malformaciones Vasculares , Adulto JovenRESUMEN
BACKGROUND: Ependymoma is the third most common malignant CNS tumor in children. Despite multimodal therapy, prognosis of relapsed ependymoma remains poor. Approaches to therapy for relapsed ependymoma are varied. We present a single-institution retrospective review of the outcomes after first relapse of intracranial ependymoma in children. PROCEDURE: We performed a retrospective, IRB-approved chart review of patients with recurrent intracranial ependymoma treated at Dana-Farber/Boston Children's Cancer and Blood Disorders Center from 1990 to 2019. RESULTS: Thirty-four patients with relapsed intracranial ependymoma were identified. At initial diagnosis, 11 patients had supratentorial disease, 22 with posterior fossa disease and one with metastatic disease. Median time-to-first relapse was 14.9 months from initial diagnosis (range 1.4-52.5). Seven patients had metastatic disease at first relapse. Gross total resection (GTR) was associated with improved 5-year progression-free survival (PFS) relative to subtotal resection (STR) and no surgery (p = .005). Localized disease at relapse was associated with improved 5-year overall survival (OS) when compared to metastatic disease (p = .02). Irradiation at first relapse seemed to delay progression but was not associated with statistically prolonged PFS or OS. Tumor location, histology, and chromosomal 1q status did not impact outcome at first relapse, although available molecular data were limited making definitive conclusions difficult. Median time-to-second relapse was 10 months (range 0.7-124). Five-year PFS and OS after first relapse were 19.9% and 45.1%, respectively. Median PFS and OS were 10.0 and 52.5 months after first relapse, respectively. CONCLUSIONS: Relapsed intracranial ependymoma has a poor prognosis despite multimodal therapy. Novel therapeutic strategies are desperately needed for this disease.
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Neoplasias Encefálicas , Ependimoma , Neoplasias Encefálicas/terapia , Preescolar , Enfermedad Crónica , Ependimoma/terapia , Humanos , Lactante , Recurrencia Local de Neoplasia/terapia , Estudios RetrospectivosRESUMEN
PURPOSE: To develop postoperative surveillance protocols that yield efficient detection rates of tumor recurrence or progression using fewer imaging studies and less cost. METHOD: This is a retrospective cohort study of all pediatric craniopharyngioma patients who have been diagnosed and treated at Boston Children's Hospital (BCH) between 1990 and 2017. All statistical analyses were performed using Stata. RESULTS: Eighty patients (43 males and 37 females) fulfilled the inclusion criteria. The mean age at time of diagnosis was 8.6 ± 4.4 years. The mean follow-up period was 10.9 ± 6.5 years. Overall 30/80 (37.5%) patients experienced tumor recurrence/progression. The median latency to recurrence/progression was 12.75 months (range 3 to 108 months), with 76.6% of the recurrences/progressions taking place within the first 2 years postoperatively. Given the lack of any clinical symptoms/signs associated with the vast majority of the recurrent/progressed cases, we propose postoperative MR imaging surveillance protocols that are substantially less intensive than the current practice. Therefore, we recommend the following postoperative MR imaging surveillance protocols, stratified by management strategies; 0, 9, 15, 36, 48, and 60 months for patients who underwent GTR, 0, 3, 6,12, 18, and 24 months for patients who underwent STR alone and 0, 3, 12, 72, 96, and 120 months for patients who underwent STR followed by subsequent XRT. CONCLUSION: The proposed postoperative MR imaging surveillance protocols would provide a potential 50% decrement of healthcare costs. It may also minify the psychological burden of frequent MR scanning for these patients and their families.
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Craneofaringioma , Neoplasias Hipofisarias , Niño , Craneofaringioma/diagnóstico por imagen , Craneofaringioma/cirugía , Femenino , Estudios de Seguimiento , Humanos , Imagen por Resonancia Magnética , Masculino , Recurrencia Local de Neoplasia/diagnóstico por imagen , Neoplasias Hipofisarias/diagnóstico por imagen , Neoplasias Hipofisarias/cirugía , Estudios RetrospectivosRESUMEN
PURPOSE: To identify the independent risk factors for developing morbid hypothalamic obesity, to propose a predictive scoring system for morbid hypothalamic obesity, and to propose an algorithm for management in order to minimize the risk of developing morbid hypothalamic obesity in patients with pediatric craniopharyngioma. METHODS: A retrospective analysis of all pediatric craniopharyngioma patients diagnosed and treated at Boston Children's Hospital (BCH) between 1985 and 2017. Analysis of the data was conducted using IBM SPSS Statistics. RESULTS: We identified 105 patients, 90 (47 males and 43 females) fulfilled the inclusion criteria. The median age of patients at time of diagnosis was 8.4 years. The median follow-up was 10.6 years. Morbid hypothalamic obesity was evident in 28 (31.1%) patients at the last follow-up visit. Age of patients at time of diagnosis > 10 years (P = 0.023), preoperative body mass index (BMI) > 95th percentile (P = 0.006), and preoperative papilledema (P < 0.001) were the independent risk factors for developing morbid hypothalamic obesity. CONCLUSION: We developed a unique predictive scoring system in order to differentiate between patients with and without high risk for developing morbid hypothalamic obesity.
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Craneofaringioma , Obesidad Mórbida , Neoplasias Hipofisarias , Índice de Masa Corporal , Niño , Craneofaringioma/complicaciones , Craneofaringioma/cirugía , Femenino , Estudios de Seguimiento , Humanos , Masculino , Obesidad Mórbida/complicaciones , Neoplasias Hipofisarias/complicaciones , Neoplasias Hipofisarias/cirugía , Estudios RetrospectivosRESUMEN
Radiation-induced acute oral mucositis is associated with inflammation and pain. In other realms of pain research, nociceptors are known to be activated by inflammatory cytokines; for example, tumor necrosis factor alpha (TNF-α) can activate transient receptor potential ion channels on sensory neurons. But there is an unclear relationship between inflammatory cytokines and molecular mediators of pain in radiation-induced mucositis (RIM) and radiation-associated pain (RAP). In this prospective, analytical, experimental pilot study, a common drug (pentoxifylline [PTX]) was used with the goal of inhibiting TNF-α signaling in mice that underwent lingual irradiation to induce severe acute oral RIM/RAP. Body weight and glossitis scores were recorded daily. Eye wiping behaviors were assayed as a surrogate measure of oral discomfort (which is possible due to cross-sensitization of the mandibular and ophthalmic branches of the trigeminal nerve). Quantitative real-time reverse transcription polymerase chain reaction was performed on irradiated tongue tissue to measure changes in expression of TNF-α, its receptor, nuclear factor kappa-light-chain-enhancer of activated B cells, transient receptor potential vanilloid type 1 (TRPV1), and transient receptor potential vanilloid type 4 (TRPV4). Responsiveness of afferent sensory trigeminal neurons to TNF-α, a TRPV1 agonist (capsaicin), and a partial TRPV4 agonist (histamine) was measured via calcium imaging. Although PTX treatment did not reduce glossitis severity or mitigate weight loss in mice with RIM/RAP, it did inhibit the upregulation of TNF-α's receptor that normally accompanies RIM, and it also reduced neuronal responsiveness to each of the aforementioned chemical stimuli. These results provide provisional evidence that inhibition of TNF-α signaling with PTX treatment may serve as a useful tool for reducing pain in head and neck cancer patients.
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Dolor/veterinaria , Pentoxifilina/uso terapéutico , Radioterapia/efectos adversos , Estomatitis/complicaciones , Animales , Capsaicina/farmacología , Histamina/farmacología , Ratones , Dolor/prevención & control , Proyectos Piloto , Estudios Prospectivos , Protectores contra Radiación/uso terapéutico , Fármacos del Sistema Sensorial/farmacología , Transducción de Señal/efectos de los fármacos , Estomatitis/tratamiento farmacológico , Estomatitis/etiología , Factor de Necrosis Tumoral alfa/análisis , Factor de Necrosis Tumoral alfa/metabolismoRESUMEN
We estimate that there will be 13·7 million new cases of childhood cancer globally between 2020 and 2050. At current levels of health system performance (including access and referral), 6·1 million (44·9%) of these children will be undiagnosed. Between 2020 and 2050, 11·1 million children will die from cancer if no additional investments are made to improve access to health-care services or childhood cancer treatment. Of this total, 9·3 million children (84·1%) will be in low-income and lower-middle-income countries. This burden could be vastly reduced with new funding to scale up cost-effective interventions. Simultaneous comprehensive scale-up of interventions could avert 6·2 million deaths in children with cancer in this period, more than half (56·1%) of the total number of deaths otherwise projected. Taking excess mortality risk into consideration, this reduction in the number of deaths is projected to produce a gain of 318 million life-years. In addition, the global lifetime productivity gains of US$2580 billion in 2020-50 would be four times greater than the cumulative treatment costs of $594 billion, producing a net benefit of $1986 billion on the global investment: a net return of $3 for every $1 invested. In sum, the burden of childhood cancer, which has been grossly underestimated in the past, can be effectively diminished to realise massive health and economic benefits and to avert millions of needless deaths.
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Países en Desarrollo , Costos de la Atención en Salud , Accesibilidad a los Servicios de Salud/organización & administración , Neoplasias/epidemiología , Neoplasias/terapia , Niño , Costo de Enfermedad , HumanosRESUMEN
OBJECTIVES: Palliative radiation therapy (pRT) is often used to improve quality of life for pediatric patients. Though palliative doses are generally lower than those for cure, pRT may still introduce undesirable effects. The decision to pursue additional therapy for a child may be challenging and depends on parents' knowledge and expectations. The goal of this study was to explore parental perceptions of pRT. METHODS: Twenty-eight children referred for pRT were enrolled in our prospective study. Parents were counseled regarding the indication and expected outcomes. They then completed a series of questionnaires to assess their understanding of pRT, side effects that their child experienced, and how the outcomes compared to their expectations. RESULTS: The majority of parents listed pain relief and addressing new disease as the main indication for pRT. When asked about expectations, the majority chose improvement in quality of life and prolongation of their child's life. Interestingly, 32% of parents expected pRT to cure their child's disease. Most patients undergoing pRT did not experience any adverse symptoms. The outcomes of pRT in the majority of cases exceeded parental expectations. CONCLUSION: Improved quality of life with pRT sometimes blurs the distinction between palliation and cure. We found that most parents understand the aim to improve quality of life, although a proportion of parents perceived pRT as a cure to their child's disease. Despite this, the majority of parents reported that the outcome of the pRT course exceeded their expectations. We postulate that parents derive comfort from pursuing active treatment.
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Toma de Decisiones , Neoplasias/radioterapia , Cuidados Paliativos , Padres/psicología , Calidad de Vida , Radioterapia/métodos , Adolescente , Niño , Femenino , Estudios de Seguimiento , Humanos , Masculino , Neoplasias/patología , Estudios Prospectivos , Encuestas y Cuestionarios , Resultado del TratamientoRESUMEN
The COVID-19 pandemic is one of the most serious global challenges to delivering affordable and equitable treatment to children with cancer we have witnessed in the last few decades. This Special Report aims to summarize general principles for continuing multidisciplinary care during the SARS-CoV-2 (COVID-19) pandemic. With contributions from the leadership of the International Society for Pediatric Oncology (SIOP), Children's Oncology Group (COG), St Jude Global program, and Childhood Cancer International, we have sought to provide a framework for healthcare teams caring for children with cancer during the pandemic. We anticipate the burden will fall particularly heavily on children, their families, and cancer services in low- and middle-income countries. Therefore, we have brought together the relevant clinical leads from SIOP Europe, COG, and SIOP-PODC (Pediatric Oncology in Developing Countries) to focus on the six most curable cancers that are part of the WHO Global Initiative in Childhood Cancer. We provide some practical advice for adapting diagnostic and treatment protocols for children with cancer during the pandemic, the measures taken to contain it (e.g., extreme social distancing), and how to prepare for the anticipated recovery period.
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Infecciones por Coronavirus/epidemiología , Manejo de la Enfermedad , Neoplasias/terapia , Neumonía Viral/epidemiología , Betacoronavirus , COVID-19 , Niño , Consenso , Humanos , Oncología Médica , Neoplasias/complicaciones , Neoplasias/diagnóstico , Pandemias , Pediatría , SARS-CoV-2 , Sociedades MédicasRESUMEN
To examine the educational background, clinical practice, and preferences regarding continuing medical education (CME) among radiation oncologists who attended the 2019 meeting of the Pediatric Radiation Oncology Society (PROS), a survey consisting of 20 questions was distributed asking for demographic and educational background, clinical practice, and preferences regarding pediatric radiation oncology CME. Of 188 participants, 130 (69.2%) returned the questionnaire. More than 80% reported access to CT simulation, three-dimensional radiotherapy, and general anesthesia while <30% had access to intraoperative radiotherapy, proton, and heavy particle therapy. After residency, 12.1% did further training in pediatric radiation oncology. When asked about further training in pediatrics after residency, 88.8% answered that there should be a formal training program beyond residency in order to treat children. More than 75% acquired knowledge in pediatric radiation oncology through journals, books, live meetings, and tumor boards. The results of this survey may help Pediatric Radiation Oncology Society (PROS) in creating guidelines and recommendations for improvement in pediatric radiation oncology training and practice support as well as the development of CME activities most likely to benefit practitioners.
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Actitud del Personal de Salud , Educación Médica Continua/normas , Neoplasias/radioterapia , Pediatría/educación , Oncólogos de Radiación/educación , Oncología por Radiación/educación , Niño , Femenino , Humanos , Masculino , Encuestas y CuestionariosRESUMEN
BACKGROUND: Desmoplastic small round cell tumor (DSRCT) is a rare aggressive sarcoma that affects children and young adults, and portends poor outcomes despite intensive multimodal treatment approaches. We report toxicity, response, and outcomes of patients with DSRCT treated with the addition of vincristine, irinotecan, and temozolomide (VIT) to interval-compressed chemotherapy as per Children's Oncology Group ARST08P1. METHODS: All newly diagnosed pediatric patients with DSRCT treated at Dana-Farber Cancer Institute and Boston Children's Hospital between 2014 and 2019 as per ARST08P1, Arm P2 with replacement of VAC cycles with VIT, were identified. Medical records were reviewed for clinical and disease characteristics, and treatment response and outcomes. RESULTS: Six patients were treated as per the above regimen. Median age at diagnosis was 15.1 years (range 3.2-16.4) and five patients were male. Five patients had abdominal primary tumors, of which one had exclusively intraabdominal and four had extraabdominal metastases. Two initial cycles of VIT were well tolerated with nausea, vomiting, diarrhea, and constipation as the most common adverse events. Overall response rate defined as partial or complete response after two initial cycles of VIT was 50%. For local control, all patients had surgical resection followed by radiotherapy, and two patients received hyperthermic intraperitoneal chemotherapy at the time of surgery. Of the four patients who have completed therapy to date, three remain disease-free with median follow-up time of 46.7 months. CONCLUSIONS: The addition of VIT to interval-compressed chemotherapy is tolerable and active in DSRCT, with activity warranting additional investigation.
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Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Adolescente , Niño , Preescolar , Tumor Desmoplásico de Células Pequeñas Redondas , Femenino , Estudios de Seguimiento , Humanos , Irinotecán/administración & dosificación , Masculino , Pronóstico , Estudios Retrospectivos , Temozolomida/administración & dosificación , Factores de Tiempo , Vincristina/administración & dosificaciónRESUMEN
This report by the Radiation Oncology Discipline of Children's Oncology Group (COG) describes the practice patterns of pediatric image-guided radiotherapy (IGRT) based on a member survey and provides practice recommendations accordingly. The survey comprised of 11 vignettes asking clinicians about their recommended treatment modalities, IGRT preferences, and frequency of in-room verification. Technical questions asked physicists about imaging protocols, dose reduction, setup correction, and adaptive therapy. In this report, the COG Radiation Oncology Discipline provides an IGRT modality/frequency decision tree and the expert guidelines for the practice of ionizing image guidance in pediatric radiotherapy patients.
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Neoplasias/radioterapia , Guías de Práctica Clínica como Asunto/normas , Pautas de la Práctica en Medicina/normas , Oncología por Radiación/normas , Planificación de la Radioterapia Asistida por Computador/métodos , Radioterapia Guiada por Imagen/métodos , Niño , Humanos , Neoplasias/patología , Dosificación RadioterapéuticaRESUMEN
PURPOSE: To demonstrate the paradigm shift in management strategies of pediatric craniopharyngioma at our institution over the past six decades. METHODS: Retrospective analysis of all pediatric patients with craniopharyngioma treated at Boston Children's Hospital between 1960 and 2017. RESULTS: One hundred seventy-eight patients with craniopharyngioma were treated between 1960 and 2017; 135 (70 males and 65 females) fulfilled the inclusion criteria. Forty-five patients were treated in the old era (1960-1984) and 90 patients were treated in the new era (1985-2017). Gross total resection (GTR) was achieved in 4% and 43% of patients in old and new eras respectively. Sub-total resection (STR) and radiotherapy (XRT) were performed in 27% and 28% of patients in old and new eras respectively. STR without XRT was performed in 20% and 29% of patients in old and new era respectively. Cyst drainage and adjuvant radiotherapy were performed in 49% of patients in the old era while no patients in the new era underwent such conservative management. Aggressive surgical resection was associated with a higher risk of worsening visual outcomes (20% vs 16%), panhypopituitarism and diabetes insipidus (86% vs 53%), psycho-social impairment (42% vs 26%), and new-onset obesity (33% vs 22%). The mortality rate was higher in the old era in comparison with that of the new one (9% vs 2%). CONCLUSION: There was a paradigm shift in management strategies of pediatric craniopharyngioma over the past six decades which in turn affected the long-term outcomes and quality of life of patients.
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Craneofaringioma , Diabetes Insípida , Neoplasias Hipofisarias , Niño , Craneofaringioma/cirugía , Femenino , Humanos , Masculino , Neoplasias Hipofisarias/cirugía , Calidad de Vida , Estudios Retrospectivos , Resultado del TratamientoRESUMEN
BACKGROUND: Heterotopic ossification (HO) is a well-recognized complication of proximal femoral resection (PFR) surgery in children with cerebral palsy (CP). Although single-dose radiation prophylaxis (SDRP) has been shown to be effective at lowering the rates of HO following adult total hip arthroplasty; there has been limited study examining the efficacy of SDRP for HO prevention in children with CP undergoing PFR. The purpose of this study was to assess the efficacy of SDRP in children with CP undergoing PFR. METHODS: This retrospective case control series identified all patients from one tertiary children's hospital undergoing PFHR. Patients were dichotomized into (1) SDRP and (2) non-SDRP groups. In SDRP, radiation was delivered preoperatively at a dose of 7.5 Gy utilizing a 6 MV photon beam. The incidence of HO in the SDRP cohort was compared to historic data using binomial testing. The size of HO lesions was compared using Wilcoxon signed-rank test. McCarthy, Brooker, and Anatomic Classifications of HO were compared using repeated measures logistic regression. RESULTS: Twenty-three patients (mean age, 15.5) and 35 hips (17 SDRP, 18 Non-SDRP) were included in the analysis. There were 17 females and 6 males in the cohort with the majority classified as GMFCS V, 21/23 (91%). HO was seen in 6 of the SDRP cohort (6/17, 35%) and 15 of the non-SDRP cohort (15/18, 83%) (P=0.015). The average size of HO at maturity was 282.7 mm in the SDRP cohort compared with 1221.5 mm in the Non-SDRP cohort (P=0.026). Radiation treatment was associated with a 938.9 mm decrease in HO size at maturity (P= 0.026). Multivariate repeated measures logistic regression analysis found that non-SDRP hips had 13 times higher odds of developing HO (P=0.015). There were no significant differences in infection rates between the 2 cohorts and there were no radiation-associated complications. CONCLUSIONS: Short-term follow up demonstrates that SDRP is a safe and efficacious intervention in decreasing the incidence and size of HO in children with CP undergoing PFR. LEVELS OF EVIDENCE: Level III-Case control cohort study.
Asunto(s)
Parálisis Cerebral/complicaciones , Cabeza Femoral/cirugía , Osificación Heterotópica/prevención & control , Osificación Heterotópica/radioterapia , Radioterapia/métodos , Terapia Recuperativa/métodos , Adolescente , Estudios de Casos y Controles , Niño , Preescolar , Femenino , Articulación de la Cadera/cirugía , Humanos , Incidencia , Lactante , Masculino , Análisis Multivariante , Osificación Heterotópica/epidemiología , Osificación Heterotópica/etiología , Estudios Retrospectivos , Factores de RiesgoRESUMEN
BACKGROUND: Few reports of palliative radiotherapy (RT) for pedialltric malignancies have been published. We described clinical indications, outcomes, and toxicities for children who received palliative RT. PROCEDURE: Pediatric patients (age ≤18 years) treated with palliative RT for incurable cancer from January 1 2008 to February 26, 2014 were included. Diagnosis, details of RT, treatment response, toxicity, and survival were retrospectively reviewed. RESULTS: Forty-six patients received 76 RT courses. Fifteen patients (33%) had ≥2 courses. Median age at palliative RT was 10.3 years; 54% were male. The most common diagnoses were neuroblastoma (20%) and diffuse intrinsic pontine glioma (17%). The most common indications for RT were oligometastatic disease in asymptomatic patients (39%) and pain (25%). The most common treatment sites were brain (32%) and bone (29%). Median RT dose was 30 Gy. Median number of RT fractions was 12. Sixty-five treatment courses (86%) were delivered with fraction sizes ≥2.5 Gy. Twenty-seven treatment courses (36%) were given under general anesthesia. Median follow-up was 3.9 months. Grade 1-2 RT-related toxicity occurred in 21% of treatment courses and 4-8% up to 12 months after RT. Two patients had Grade 3 toxicity during RT (esophagitis). Of symptomatic patients, 91%, 73%, 58%, and 43% had improved or stable symptoms during RT and 0-3, 3-6, and 6-12 months afterwards, respectively. Median survival after palliative RT was 4.2 months. Four of 21 surviving patients (19%) had hospice care at last follow-up. CONCLUSIONS: Palliative RT was well tolerated in children with incurable malignancies, with most cases associated with acceptable toxicity, and improved or stable symptoms.