RESUMO
BACKGROUND: Technetium-99 (99m Tc) lymphoscintigraphy with blue dye injection is an accepted method for sentinel lymph node (SLN) mapping, but blue dye has known adverse effects, and injection of 99m Tc may increase time under anesthesia for pediatric patients. Indocyanine green (ICG) may serve as an adjunct to assist with visibility and identification of SLNs. We hypothesized that sensitivity of ICG was similar to blue dye in SLN biopsies. METHODS: Thirty patients (36 procedures with 96 total specimens) underwent preoperative intradermal injection of 99m Tc, followed by intradermal injection of isosulfan blue and ICG. Test characteristics of blue dye, ICG, and 99m Tc included sensitivity, specificity, positive predictive value (PPV), and negative predictive value (NPV). RESULTS: ICG had a sensitivity of 87% and PPV of 83% for detection of 99m Tc-hot lymph nodes; blue dye had a sensitivity of 44% and PPV of 97%. For detection of pathologically confirmed lymph nodes, ICG had a sensitivity of 84% and a positive predictive value (PPV) of 91%. 99m Tc had a sensitivity of 82% and a PPV of 94%. ICG had no significant difference in odds of being positive in pathology-confirmed lymph nodes compared to 99m Tc (odds ratio [OR], 0.818; 95% confidence interval [CI], 0.3-2.172; p = .823) and had higher odds than isosulfan blue (OR, 0.025, 95% CI, 0.001-0.148; p < .001). CONCLUSION: This study established the efficacy of ICG as an adjunct to SLNB in the pediatric and young adult population. ICG was safe, more efficacious than blue dye, and may obviate the need for lymphoscintigraphy in selected patients resulting in reduced time under anesthesia.
Assuntos
Verde de Indocianina , Linfonodo Sentinela , Humanos , Adulto Jovem , Criança , Linfonodo Sentinela/diagnóstico por imagem , Linfonodo Sentinela/cirurgia , Linfonodo Sentinela/patologia , Compostos Radiofarmacêuticos , Corantes , Biópsia de Linfonodo Sentinela/métodos , Linfonodos/diagnóstico por imagem , Linfonodos/cirurgia , Linfonodos/patologiaRESUMO
BACKGROUND: There are no consensus guidelines regarding the use of percutaneous needle biopsy for the diagnosis of soft tissue and bone tumors. The aim of this study was to understand the efficacy of image-guided percutaneous biopsy for pediatric patients with soft tissue and bony masses, the role of intraoperative image guidance, and diagnostic accuracy. PATIENTS AND METHODS: A retrospective institutional chart review was performed on patients who underwent percutaneous biopsy of soft tissue or bone tumors between 2007 and 2017. Data collected included preoperative imaging, type of biopsy, demographics, insurance status, number of samples taken, and pathologic results. RESULTS: One hundred forty-one children and young adults underwent 169 biopsies. Female patients received 48.2% of biopsies. The mean age was 14.3 ± 7.0 years. Core needle biopsies made up 89.4% of procedures, while 10.6% were fine needle aspirate. The mean number of samples per patient was 3.6 ± 2.5. All patients had imaging guidance, with computed tomography used in 44.7% of patients, 9.9% using fluoroscopy, 7.1% using ultrasound for guidance, and 53 (37.6%) patients had more than one modality. Diagnostic specimens were obtained in 97.9% of biopsies. The most common overall pathology was osteoid osteoma. The most common malignant tumors were osteosarcoma and Ewing's sarcoma. CONCLUSION: Image-guided percutaneous biopsy is a safe and effective method of obtaining accurate tissue samples in children and young adults with soft tissue or bone masses. LEVEL OF EVIDENCE: Level 4-Study of diagnostic test.
Assuntos
Neoplasias Ósseas , Padrão de Cuidado , Humanos , Criança , Feminino , Adulto Jovem , Adolescente , Adulto , Estudos Retrospectivos , Neoplasias Ósseas/diagnóstico por imagem , Neoplasias Ósseas/cirurgia , Neoplasias Ósseas/patologia , Biópsia com Agulha de Grande Calibre , Biópsia Guiada por Imagem/métodosRESUMO
BACKGROUND: Maintaining dose-dense, interval-compressed chemotherapy improves survival in patients with Ewing sarcoma but is limited by myelosuppression. Romiplostim is a thrombopoietin receptor agonist that may be useful in the treatment of chemotherapy-induced thrombocytopenia (CIT). METHODS: Patients aged between 3 and 33 years with Ewing sarcoma from 2010 to 2020 were reviewed. CIT was defined as a failure to achieve 75,000 platelets per microliter by day 21 after the start of any chemotherapy cycle. Fisher's exact test was used for univariate analysis and Pearson's correlation coefficient was used for the association between continuous variables. RESULTS: Twenty-seven out of 42 patients (64%) developed isolated CIT, delaying one to four chemotherapy cycles per patient. CIT occurred during consolidation therapy in 24/27(88.9%) and with ifosfamide/etoposide cycles in 24/27 (88.9%). Univariate analysis failed to identify risk factors for CIT. The use of radiation approached significance (p-value = .056). Ten patients received romiplostim. The median starting dose was 3 µg/kg (range 1-5). Doses were escalated weekly by 1-2 to 4-10 µg/kg and continued throughout chemotherapy. A higher romiplostim dose was associated with a higher change in average platelet counts from baseline, r = .73 (p = .04). No romiplostim-related adverse events were identified aside from mild headache. CONCLUSIONS: CIT is the primary reason for the inability to maintain treatment intensity in Ewing sarcoma. The concurrent use of romiplostim with chemotherapy was safe and feasible, and efficacy was associated with higher romiplostim doses.
Assuntos
Antineoplásicos , Tumores Neuroectodérmicos Primitivos Periféricos , Sarcoma de Ewing , Trombocitopenia , Adolescente , Adulto , Antineoplásicos/uso terapêutico , Criança , Pré-Escolar , Humanos , Receptores Fc/uso terapêutico , Proteínas Recombinantes de Fusão/efeitos adversos , Sarcoma de Ewing/tratamento farmacológico , Trombocitopenia/induzido quimicamente , Trombocitopenia/tratamento farmacológico , Trombopoetina/efeitos adversos , Resultado do Tratamento , Adulto JovemRESUMO
BACKGROUND: There are minimal data on long-term surgical outcomes of patients who have undergone resection for Wilms tumor (WT) and neuroblastoma (NB). METHODS: A retrospective review of patients in a long-term survivor clinic between the years 1967 and 2016 in a pediatric tertiary care hospital (>5 years posttreatment) was performed. RESULTS: Eighty-six survivors of WT and 86 survivors of NB who had ongoing follow-up in the survivors' clinic were identified. The median age at diagnosis was 2.5 years (range, 0.4-15.7 years) with a mean follow-up of 22.3 years (±10.4 years) for WT. The median age at diagnosis for patients with NB was 0.9 years (range, 0.1-8.6 months); mean follow-up of 21.7 years (±7.9 years). Twelve patients with WT (14.0%) had at least 1 repeat laparotomy, 11.1% for bowel obstruction, at a median of 3 months from initial surgery. Twelve patients (14.0%) with NB required laparotomy and 8.1% for bowel obstruction, at a median of 12 years after initial surgery. The incidence of hypertension in patients with WT who had undergone nephrectomy was not outside of population norms. Patients who underwent thoracotomy for a NB have a higher incidence of scoliosis and Horner syndrome. CONCLUSIONS: Small bowel obstruction requiring laparotomy is significantly higher than the literature norms for both tumor patient populations and typically occurs in the early postoperative period for patients with WT and remotely in patients with NB. The long-term surgical complications of patients who underwent resection for NB and WT clearly merit follow-up and patient education within multidisciplinary long-term survivorship clinics.
Assuntos
Neoplasias Renais , Neuroblastoma , Tumor de Wilms , Criança , Seguimentos , Humanos , Lactente , Neoplasias Renais/complicações , Neoplasias Renais/cirurgia , Neuroblastoma/complicações , Neuroblastoma/cirurgia , Estudos Retrospectivos , Resultado do Tratamento , Tumor de Wilms/complicações , Tumor de Wilms/cirurgiaRESUMO
OBJECTIVE: The current study examined the roles of constructive and dysfunctional problem-solving strategies in the relationships between illness uncertainty and adjustment outcomes (i.e., anxious, depressive, and posttraumatic stress symptoms) in caregivers of children newly diagnosed with cancer. METHODS: Two hundred thirty-eight caregivers of children (0-19 years of age) newly diagnosed with cancer (2-14 weeks since diagnosis) completed measures of illness uncertainty, problem-solving strategies, and symptoms of anxiety, depression, and posttraumatic stress. RESULTS: A mediation model path analysis assessed constructive and dysfunctional problem-solving strategies as mediators between illness uncertainty and caregiver anxious, depressive, and posttraumatic stress symptoms. Dysfunctional problem-solving scores partially mediated the relationships between illness uncertainty and anxious, depressive, and posttraumatic stress symptoms. Constructive problem-solving scores did not mediate these relationships. CONCLUSIONS: The current findings suggest that illness uncertainty and dysfunctional problem-solving strategies, but not constructive problem-solving strategies, may play a key role in the adjustment of caregivers of children newly diagnosed with cancer. Interventions aimed at managing illness uncertainty and mitigating the impact of dysfunctional problem-solving strategies may promote psychological adjustment.
Assuntos
Cuidadores , Neoplasias , Criança , Depressão , Ajustamento Emocional , Humanos , Lactente , Recém-Nascido , IncertezaRESUMO
Cardiac disease following radiation therapy represents a major consideration in the treatment of a variety of malignancies. Damage to the heart can manifest in a variety of pathologies including ischemic cardiac disease, cardiomyopathy, valvular dysfunction, arrhythmias, and pericarditis. This damage has been shown to directly relate to cardiac radiation dose and to stem from a range of cellular pathways that are often related to fibrosis. The importance of minimizing radiation dose to the heart is especially critical in the pediatric population and when treating disease sites adjacent to the heart. Proton therapy represents a promising approach to minimize dose to normal tissues such as the heart. The cardiac dosimetry reductions due to proton therapy have been demonstrated in multiple cancers and further long-term follow-up will determine the clinical significance of these reductions to cardiac structures. Future approaches using advanced techniques such as FLASH therapy could provide even further benefit by reducing post-radiation fibrosis.
Assuntos
Cardiopatias , Neoplasias , Terapia com Prótons , Cardiotoxicidade/etiologia , Criança , Cardiopatias/etiologia , Humanos , Neoplasias/radioterapia , Terapia com Prótons/efeitos adversos , Síndrome da Fibrose por RadiaçãoRESUMO
BACKGROUND: Adolescents and young adults (AYAs) with cancer have unique medical challenges compared with younger children and older adults. Dedicated centers have been established to deliver cancer therapy to the AYA population; many of these programs are located in pediatric hospitals. Outcomes of AYA patients on pediatric protocols are generally superior to those on adult protocols. Little is understood about the impact of care within a pediatric environment for surgical care of young adults. METHODS: A retrospective institutional review was performed of patients undergoing thoracic metastectomy between 2012 and 2017. Demographics, procedural factors, cost, and outcomes were analyzed. Patients were divided into two groups: > 18 and <18 years. RESULTS: Ninety-one procedures were performed: 61.5% (n = 56) were in patients <18 years old and 38.5% (n = 35) were > 18 years old. The median age was 6.5 years for <18 years old and 28 years for > 18 years old. Older patients had a significantly longer operative time on thoracoscopic cases; 91 versus 63 minutes. Fifty percent of the > 18 group had > 1 lesion resected compared with one lesion resected in 80.8% in <18 years old. No significant differences were found between the two groups in the duration of chest tube or length of stay. The AYA group demonstrated more "adult type" comorbidities. CONCLUSION: AYA patients have unique developmental and emotional challenges. Surgical intervention in this special population of patients cared for within a pediatric environment shows no significant difference in outcome compared with pediatric patients undergoing the same procedure. AYA patients with "adult type" comorbidities can safely undergo multidisciplinary care including surgery within a pediatric environment without the need to fragment care.
Assuntos
Hospitais Pediátricos/estatística & dados numéricos , Neoplasias Pulmonares/mortalidade , Neoplasias/mortalidade , Procedimentos Cirúrgicos Torácicos/mortalidade , Adulto , Criança , Feminino , Seguimentos , Humanos , Neoplasias Pulmonares/secundário , Neoplasias Pulmonares/cirurgia , Masculino , Neoplasias/patologia , Neoplasias/cirurgia , Prognóstico , Estudos Retrospectivos , Taxa de SobrevidaRESUMO
Infantile myofibroma is the most common fibrous tumor of infancy. Despite the frequency of these tumors, the natural history is incompletely understood. We present two cases with a unique pattern of disease: solitary myofibromas with subsequent progression to diffuse myofibromatosis. Given the variable spectrum of disease and the corresponding difference in morbidity and potential mortality based on the extent of disease, we propose surveillance recommendations.
Assuntos
Miofibroma/fisiopatologia , Miofibromatose/patologia , Progressão da Doença , Feminino , Humanos , Recém-Nascido , Masculino , PrognósticoRESUMO
BACKGROUND: Survivors of Hodgkin lymphoma (HL) in childhood have an increased risk of subsequent malignant neoplasms (SMNs). Herein, the authors extended the follow-up of a previously reported Late Effects Study Group cohort and identified patients at highest risk for SMNs to create evidence for risk-based screening recommendations. METHODS: The standardized incidence ratio was calculated using rates from the Surveillance, Epidemiology, and End Results program as a reference. The risk of SMN was estimated using proportional subdistribution hazards regression. The cohort included 1136 patients who were diagnosed with HL before age 17 years between 1955 and 1986. The median length of follow-up was 26.6 years. RESULTS: In 162 patients, a total of 196 solid SMNs (sSMNs) were identified. Compared with the general population, the cohort was found to be at a 14-fold increased risk of developing an sSMN (95% confidence interval, 12.0-fold to 16.3-fold). The cumulative incidence of any sSMN was 26.4% at 40 years after a diagnosis of HL. Risk factors for breast cancer among females were an HL diagnosis between ages 10 years and 16 years and receipt of chest radiotherapy. Males treated with chest radiotherapy at age <10 years were found to be at highest risk of developing lung cancer. Survivors of HL who were treated with abdominal/pelvic radiotherapy and high-dose alkylating agents were found to be at highest risk of developing colorectal cancer and females exposed to neck radiotherapy at age <10 years were at highest risk of thyroid cancer. By age 50 years, the cumulative incidence of breast, lung, colorectal, and thyroid cancer was 45.3%, 4.2%, 9.5%, and 17.3%, respectively, among those at highest risk. CONCLUSIONS: Survivors of childhood HL remain at an increased risk of developing sSMNs. In the current study, subgroups of survivors of HL at highest risk of specific sSMNs were identified, and evidence for screening provided.
Assuntos
Doença de Hodgkin/terapia , Segunda Neoplasia Primária/epidemiologia , Adolescente , Adulto , Criança , Pré-Escolar , Tratamento Farmacológico , Feminino , Doença de Hodgkin/complicações , Doença de Hodgkin/epidemiologia , Humanos , Lactente , Recém-Nascido , Masculino , Pessoa de Meia-Idade , Vigilância da População , Radioterapia , Medição de RiscoRESUMO
Head and neck rhabdomyosarcoma lymph node staging is challenging due to varied patterns of lymphatic drainage and the suboptimal predictive value of available imaging modalities. Furthermore, regional relapse rates are unacceptably high, and the toxicity of empiric radiation is undesirable in the pediatric and young adult population. In an attempt to improve locoregional control without excess morbidity, we have adopted routine sentinel lymph node biopsy in head and neck rhabdomyosarcoma, which is safe and feasible in pediatric patients. Of six procedures reported here, pathologic findings led to intensification of regional and/or systemic therapy in two patients.
Assuntos
Neoplasias de Cabeça e Pescoço/patologia , Rabdomiossarcoma/patologia , Biópsia de Linfonodo Sentinela/métodos , Linfonodo Sentinela/patologia , Adolescente , Adulto , Criança , Pré-Escolar , Feminino , Seguimentos , Neoplasias de Cabeça e Pescoço/cirurgia , Humanos , Lactente , Masculino , Prognóstico , Estudos Prospectivos , Rabdomiossarcoma/cirurgia , Linfonodo Sentinela/cirurgia , Adulto JovemRESUMO
BACKGROUND: Pediatric hematology/oncology (PHO) patients receiving therapy or undergoing hematopoietic stem cell transplantation (HSCT) often require a central line and are at risk for bloodstream infections (BSI). There are limited data describing outcomes of BSI in PHO and HSCT patients. METHODS: This is a multicenter (n = 17) retrospective analysis of outcomes of patients who developed a BSI. Centers involved participated in a quality improvement collaborative referred to as the Childhood Cancer and Blood Disorder Network within the Children's Hospital Association. The main outcome measures were all-cause mortality at 3, 10, and 30 days after positive culture date; transfer to the intensive care unit (ICU) within 48 hours of positive culture; and central line removal within seven days of the positive blood culture. RESULTS: Nine hundred fifty-seven BSI were included in the analysis. Three hundred fifty-four BSI (37%) were associated with at least one adverse outcome. All-cause mortality was 1% (n = 9), 3% (n = 26), and 6% (n = 57) at 3, 10, and 30 days after BSI, respectively. In the 165 BSI (17%) associated with admission to the ICU, the median ICU stay was four days (IQR 2-10). Twenty-one percent of all infections (n = 203) were associated with central line removal within seven days of positive blood culture. CONCLUSIONS: BSI in PHO and HSCT patients are associated with adverse outcomes. These data will assist in defining the impact of BSI in this population and demonstrate the need for quality improvement and research efforts to decrease them.
Assuntos
Bacteriemia/mortalidade , Infecções Relacionadas a Cateter/mortalidade , Cateterismo Venoso Central/mortalidade , Neoplasias Hematológicas/terapia , Transplante de Células-Tronco Hematopoéticas/mortalidade , Hospitalização/estatística & dados numéricos , Infecções/mortalidade , Adolescente , Bacteriemia/sangue , Bacteriemia/etiologia , Infecções Relacionadas a Cateter/sangue , Infecções Relacionadas a Cateter/etiologia , Cateterismo Venoso Central/efeitos adversos , Criança , Pré-Escolar , Feminino , Seguimentos , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Humanos , Infecções/sangue , Infecções/etiologia , Masculino , Prognóstico , Estudos Retrospectivos , Taxa de SobrevidaRESUMO
OPINION STATEMENT: The past 5 decades have seen significant improvements in outcomes for pediatric patients with cancer. Unfortunately, children and adolescents who have been treated for cancer are five to six times more likely to develop cardiovascular disease as a result of their therapies. Cardiovascular disease may manifest in a plethora of ways, from asymptomatic ventricular dysfunction to end-stage heart failure, hypertension, arrhythmia, valvular disease, early coronary artery disease, or peripheral vascular disease. A number of treatment modalities are implicated in pediatric and adult populations, including anthracyclines, radiation therapy, alkylating agents, targeted cancer therapies (small molecules and antibody therapies), antimetabolites, antimicrotubule agents, immunotherapy, interleukins, and chimeric antigen receptor T cells. For some therapies, such as anthracyclines, the mechanism of injury is elucidated, but for many others it is not. While a few protective strategies exist, in many cases, observation and close monitoring is the only defense against developing end-stage cardiovascular disease. Because of the variety of potential outcomes after cancer therapy, a one-size-fits-all approach is not appropriate. Rather, a good working relationship between oncology and cardiology to assess the risks and benefits of various therapies and planning for appropriate surveillance is the best model. When disease is identified, any of a number of therapies may be appropriate; however, in the pediatric and adolescent population supportive data are limited.
Assuntos
Antineoplásicos/efeitos adversos , Cardiotoxicidade/etiologia , Cardiotoxicidade/terapia , Imunoterapia Adotiva/efeitos adversos , Imunoterapia/efeitos adversos , Neoplasias/terapia , Antineoplásicos/uso terapêutico , Cardiologia/métodos , Cardiologia/normas , Cardiotoxicidade/patologia , Criança , Gerenciamento Clínico , Humanos , Imunoterapia/métodos , Neoplasias/patologiaRESUMO
BACKGROUND: Desmoid tumors/aggressive fibromatosis (DT/AF) lack a reliably effective medical therapy. Surgical resection may be morbid and does not preclude recurrence. Radiation may carry severe late effects, particularly detrimental in young patients. At our institution, we recently observed promising results with pazopanib therapy for DT/AF in adolescent and young adult (AYA) patients. PROCEDURE: Retrospective single-institution chart review. RESULTS: Six DT/AF patients of 3-21 years with previously treated DT/AF received pazopanib; 31 DT/AF patients received established therapies only. In both groups, median age at diagnosis was 16 years, female patients comprised 50%, and most common DT/AF site was extremity. Established therapies showed few objective responses and most patients therefore received multiple therapies. Surgical resection had a 68% recurrence rate. Of eight patients who received vinblastine/methotrexate, only one had a partial response (PR) by RECIST 1.1 and five had stable disease (SD); 62.5% required additional therapy. Of seven patients who received sulindac/tamoxifen, none showed objective improvement. In contrast, pazopanib demonstrated best responses by RECIST of PR in two of seven and SD in six of seven tumors. A PR of 66% was observed in a patient who had failed multiple prior therapies. The mesenteric DT/AF also showed PR. Maximum volumetric decrease by T2-weighted magnetic resonance imaging (MRI) was 97%. Dramatically increased fibrosis was seen on T2-weighted MRI. Patients reported pain relief and improvement in function within 1 month. Except for one case of edema, all other toxicities responded to dose reduction without sacrificing objective treatment response. CONCLUSION: Pazopanib provides a promising, well-tolerated therapy for DT/AF in the AYA population and warrants further study.
Assuntos
Inibidores da Angiogênese/uso terapêutico , Fibromatose Agressiva/tratamento farmacológico , Recidiva Local de Neoplasia/tratamento farmacológico , Pirimidinas/uso terapêutico , Sulfonamidas/uso terapêutico , Adolescente , Adulto , Criança , Pré-Escolar , Feminino , Fibromatose Agressiva/patologia , Seguimentos , Humanos , Indazóis , Masculino , Recidiva Local de Neoplasia/patologia , Prognóstico , Estudos Retrospectivos , Adulto JovemRESUMO
BACKGROUND: Lymph node metastases are an important cause of treatment failure for pediatric and adolescent/young adult (AYA) sarcoma patients. Nodal sampling is recommended for certain sarcoma subtypes that have a predilection for lymphatic spread. Sentinel lymph node biopsy (SLNB) may improve the diagnostic yield of nodal sampling, particularly when single-photon emission computed tomography/computed tomography (SPECT-CT) is used to facilitate anatomic localization. Functional imaging with positron emission tomography/computed tomography (PET-CT) is increasingly used for sarcoma staging and is a less invasive alternative to SLNB. To assess the utility of these 2 staging methods, this study prospectively compared SLNB plus SPECT-CT with PET-CT for the identification of nodal metastases in pediatric and AYA patients. METHODS: Twenty-eight pediatric and AYA sarcoma patients underwent SLNB with SPECT-CT. The histological findings of the excised lymph nodes were then correlated with preoperative PET-CT imaging. RESULTS: A median of 2.4 sentinel nodes were sampled per patient. No wound infections or chronic lymphedema occurred. SLNB identified tumors in 7 of the 28 patients (25%), including 3 patients who had normal PET-CT imaging of the nodal basin. In contrast, PET-CT demonstrated hypermetabolic regional nodes in 14 patients, and this resulted in a positive predictive value of only 29%. The sensitivity and specificity of PET-CT for detecting histologically confirmed nodal metastases were only 57% and 52%, respectively. CONCLUSIONS: SLNB can safely guide the rational selection of nodes for biopsy in pediatric and AYA sarcoma patients and can identify therapy-changing nodal disease not appreciated with PET-CT. Cancer 2017;155-160. © 2016 American Cancer Society.
Assuntos
Fluordesoxiglucose F18/metabolismo , Metástase Linfática/diagnóstico , Metástase Linfática/patologia , Sarcoma/patologia , Linfonodo Sentinela/patologia , Linfonodo Sentinela/cirurgia , Adolescente , Adulto , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Excisão de Linfonodo/métodos , Linfocintigrafia/métodos , Masculino , Estadiamento de Neoplasias/métodos , Tomografia por Emissão de Pósitrons/métodos , Estudos Prospectivos , Sarcoma/metabolismo , Sarcoma/cirurgia , Sensibilidade e Especificidade , Linfonodo Sentinela/metabolismo , Biópsia de Linfonodo Sentinela/métodos , Tomografia Computadorizada por Raios X/métodos , Adulto JovemRESUMO
PURPOSE: Quality of life (QoL) has been increasingly emphasized in National Cancer Institute (NCI)-sponsored multisite clinical trials. Little is known about the outcomes of these trials in pediatric cancer. Objectives were to describe the proportion of Children's Oncology Group (COG) QoL studies that successfully accrued subjects and were analyzed, presented or published. METHODS: We conducted a survey to describe outcomes of COG QoL studies. We included studies that contained at least one QoL assessment and were closed to patient accrual at the time of survey dissemination. Respondents were the investigators most responsible for the QoL aim. RESULTS: Sixteen studies were included; response rate was 100%. Nine (56%) studies were embedded into a cancer treatment trial. Only 3 (19%) studies accrued their intended sample size. Seven (44%) studies were analyzed, 9 (56%) were presented, and 6 (38%) were published. CONCLUSIONS: NCI-sponsored pediatric QoL studies have high rates of failure to accrue. Many were not analyzed or disseminated. Using these data, strategies have been implemented to improve conduct in future trials. Monitoring of QoL studies is important to maximize the chances of study success.
Assuntos
Neoplasias/terapia , Qualidade de Vida , Pesquisa Biomédica , Criança , Ensaios Clínicos como Assunto , Feminino , Humanos , Masculino , National Cancer Institute (U.S.) , Pediatria , Inquéritos e Questionários , Estados UnidosRESUMO
BACKGROUND: The combination of vincristine, oral irinotecan, and temozolomide (VOIT regimen) has shown antitumor activity in a pediatric Phase I trial. To further potentiate synergy, we assessed the safety and feasibility of adding bevacizumab to VOIT for children and young adults with recurrent tumors. METHODS: Patients received vincristine (1.5 mg/m(2) on day 1), oral irinotecan (90 mg/m(2) on days 1-5), temozolomide (100-150 mg/m(2) on days 1-5), and bevacizumab (15 mg/kg on day 1) in 3-week cycles, which were repeated for up to six cycles. Cefixime prophylaxis was used to reduce irinotecan-associated diarrhea. RESULTS: Thirteen patients received 36 total cycles. Six of the first 10 patients required dose reductions due to toxicity during the first cycle (n = 3) or subsequent cycles (n = 3), and these grade 3 side effects included prolonged nausea, dehydration, anorexia, neuropathy, diarrhea, and abdominal pain, as well as prolonged grade 4 neutropenia. After reducing daily temozolomide to 100 mg/m(2) , three additional patients tolerated therapy well without the need for dose reductions. Toxicities attributed to bevacizumab were limited to grade 1 epistaxis (1) and grade 2 proteinuria (1). Tumor responses were seen in both patients with Ewing sarcoma. CONCLUSIONS: Reducing temozolomide from 150 to 100 mg/m(2) /day improved tolerability, and treatment with this lower temozolomide dose was feasible and convenient as outpatient therapy. Although responses were seen in Ewing sarcoma, the benefit of adding bevacizumab remains unclear.
Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/administração & dosagem , Neoplasias Encefálicas/tratamento farmacológico , Recidiva Local de Neoplasia/tratamento farmacológico , Sarcoma de Ewing/tratamento farmacológico , Administração Oral , Adolescente , Adulto , Antibacterianos/administração & dosagem , Antibacterianos/efeitos adversos , Anticorpos Monoclonais Humanizados/administração & dosagem , Anticorpos Monoclonais Humanizados/efeitos adversos , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Bevacizumab , Neoplasias Encefálicas/patologia , Camptotecina/administração & dosagem , Camptotecina/efeitos adversos , Camptotecina/análogos & derivados , Cefixima/administração & dosagem , Cefixima/efeitos adversos , Criança , Pré-Escolar , Dacarbazina/administração & dosagem , Dacarbazina/efeitos adversos , Dacarbazina/análogos & derivados , Feminino , Humanos , Lactente , Irinotecano , Masculino , Recidiva Local de Neoplasia/patologia , Projetos Piloto , Sarcoma de Ewing/patologia , Temozolomida , Vincristina/administração & dosagem , Vincristina/efeitos adversosRESUMO
BACKGROUND: Pediatric cancer survivors are at risk for multiple late effects including second malignancies, some a direct consequence of genetic susceptibility. Appropriate surveillance and management for the survivor and at-risk family members can often be established if the genetic predisposition is recognized and/or diagnosed. Numerous published guidelines outline which adult cancer patients and survivors should be referred for hereditary cancer risk assessment. In the pediatric oncology setting, minimal guidance exists for healthcare providers to determine which patients and families to refer for genetic evaluation. PROCEDURE: The aim of this project was to determine what percentage of childhood cancer survivors are appropriate for further evaluation in a hereditary cancer program or genetics clinic and characterize indications for referral. Participants included pediatric cancer survivors seen for follow-up in a large cancer survivor center. Medical and family histories were obtained and reviewed by a certified genetic counselor at the survivor's annual visit. Eligibility for genetics referral was determined based on personal/family medical history and published literature. RESULTS: Of 370 survivors of childhood cancer, 109 (29%) were considered eligible for genetics follow-up or referral. Family history of cancer is the most prevalent reason identified for eligibility for further genetics evaluation (61%) followed by tumor type (18%), medical history (16%), and family history of another condition (6%). CONCLUSIONS: This project provides evidence that inclusion of genetic evaluation is feasible and relevant in the care of childhood cancer survivors. Further study is warranted to determine optimal timing and clinical utility of this multidisciplinary and family-centered approach.
Assuntos
Predisposição Genética para Doença , Hospitais Pediátricos , Neoplasias/genética , Neoplasias/mortalidade , Sobreviventes/estatística & dados numéricos , Adolescente , Adulto , Criança , Pré-Escolar , Feminino , Seguimentos , Humanos , Masculino , Oncologia , Pessoa de Meia-Idade , Neoplasias/diagnóstico , Prognóstico , Medição de Risco , Fatores de Risco , Taxa de Sobrevida , Adulto JovemRESUMO
Background: Medulloblastoma is an aggressive central nervous system (CNS) tumor that occurs mostly in the pediatric population. Treatment often includes a combination of surgical resection, craniospinal irradiation (CSI), and chemotherapy. Children who receive standard photon CSI are at risk for cardiac toxicities including coronary artery disease, left ventricular scarring and dysfunction, valvular damage, and atherosclerosis. Current survivorship guidelines recommend routine echocardiogram (ECHO) surveillance. In this multi-institutional study, we describe markers of cardiac dysfunction in medulloblastoma survivors. Methods: A retrospective chart review of medulloblastoma patients who had photon beam CSI was followed by ECHO between 1980 and 2010 at Lurie Children's Hospital and Dana-Farber/Boston Children's Hospital. Results: During the 30-year study period, 168 medulloblastoma patient records were identified. Included in this study were the 75 patients who received CSI or spinal radiation and ECHO follow-up. The mean age at CSI was 8.6 years (range, 2.9-20), and the mean number of years between radiation therapy (RT) completion and first ECHO was 7.4 (range, 2-16). Mean ejection fraction (EF) was 60.0% and shortening fraction (SF) was 33.8%. Five patients (7%) had abnormal ECHO results: three with EF <50% and two with SF <28%. Conclusion: The majority of medulloblastoma patients who received CSI have relatively normal ECHOs post-treatment; however, 7% of patients had abnormal ECHOs. The implication of our study for medulloblastoma survivors is that further investigations are needed in this population with a more systematic, longitudinal assessment to determine predictors and screenings.