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1.
Ther Adv Hematol ; 14: 20406207231170708, 2023.
Artigo em Inglês | MEDLINE | ID: mdl-37151808

RESUMO

Background: Umbilical cord blood hematopoietic stem cells are commonly used for hematopoietic system reconstitution in recipients after umbilical cord blood transplantation (UCBT). However, the optimal conditioning regimen for UCBT remains a topic of debate. The exact impact of total body irradiation (TBI) as a part of conditioning regimens remains unknown. Objectives: The aim of this study was to evaluate the impacts of TBI on UCBT outcomes. Design: This was a multi-institution retrospective study. Methods: A retrospective analysis was conducted on the outcomes of 136 patients receiving UCBT. Sixty-nine patients received myeloablative conditioning (MAC), in which 33 underwent TBI and 36 did not, and 67 patients received reduced-intensity conditioning (RIC), in which 43 underwent TBI and 24 did not. Univariate and multivariate analyses were conducted to compare the outcomes and the post-transplant complications between patients who did and did not undergo TBI in the MAC subgroup and RIC subgroup, respectively. Results: In the RIC subgroup, patients who underwent TBI had superior overall survival (adjusted hazard ratio [aHR] = 0.25, 95% confidence interval [CI]: 0.09-0.66, p = 0.005) and progression-free survival (aHR = 0.26, 95% CI: 0.10-0.66, p = 0.005). However, in the MAC subgroup, there were no statistically significant differences between those receiving and not receiving TBI. Conclusion: In the setting of RIC in UCBT, TBI utilization can improve overall survival and progression-free survival. However, TBI does not show superiority in the MAC setting.

2.
Pediatr Blood Cancer ; 70(3): e30139, 2023 03.
Artigo em Inglês | MEDLINE | ID: mdl-36573296

RESUMO

BACKGROUND: Pediatric central nervous system (CNS) tumors are the leading cause of pediatric cancer mortality. Research addressing genomic biomarkers and clinical outcomes is needed to inform therapeutic decision-making. METHODS: We conducted a retrospective analysis of pediatric patients (age <21) diagnosed with a primary CNS tumor at four upstate New York hospitals from 2008 to 2021. Clinical and histopathologic data were identified from each patient, including genomic analysis of somatic mutations and tumor mutational burden (TMB) where available. These variables were each compared with overall survival using Cox regression analyses. Multivariable analysis was conducted to identify patient characteristics that may independently predict survival. RESULTS: We identified 119 patients. Common tumor types included low-grade glioma (N = 51), high-grade glioma (N = 29), and medulloblastoma (N = 11). Common driver mutations included TP53 inactivation (N = 16), BRAF-KIAA1549 fusion (N = 16), FGFR1 amplification (N = 12), BRAF V600E mutation (N = 12), NF1 loss (N = 12), and H3F3A K28M mutation (N = 6). Median TMB was one mutation/megabase (mut/Mb, range = 0-132). Overall survival was 79.9%. Variables associated with poorer survival on univariable analysis were higher TMB (p = .002, HR 4.97), high-grade tumors (p = .009, HR 84.3), and high-grade glioma histology (p = .021, HR 3.14). Multivariable analyses further identified TMB (p = .011, HR 4.46) and high-grade histology (p = .015, HR 5.28) as independently predictive of worse survival. Tumor progression was more common in high-TMB (N = 15, 44%) than in low-TMB tumors (N = 19, 35%). CONCLUSIONS: High TMB is correlated with higher rates of progression and death as compared to low-TMB tumors. These findings may help identify patients who may benefit from alternative treatments, such as immunotherapies.


Assuntos
Neoplasias do Sistema Nervoso Central , Neoplasias Cerebelares , Glioma , Humanos , Criança , Estudos Retrospectivos , Proteínas Proto-Oncogênicas B-raf/genética , Biomarcadores Tumorais/genética , Neoplasias do Sistema Nervoso Central/genética , Neoplasias do Sistema Nervoso Central/terapia , Glioma/genética , Glioma/terapia , Glioma/patologia , Mutação
3.
J Pediatr Hematol Oncol ; 44(1): e264-e267, 2022 01 01.
Artigo em Inglês | MEDLINE | ID: mdl-33843815

RESUMO

BACKGROUND: Infant acute myeloid leukemia is a rare but aggressive form of leukemia. OBSERVATION: We report 2 children who presented with hyperleukocytosis, subsequently diagnosed with infant acute myeloid leukemia, and both developed isolated central nervous system relapse while on chemotherapy. Both infants underwent successful bone marrow transplantation with myeloablative conditioning (thiotepa, busulfan, and cyclophosphamide) without radiation, followed by 12 empiric post-transplant lumbar punctures with intrathecal cytarabine. Both patients tolerated these therapies well, and are without infections, chronic graft-versus-host disease, or any post-transplant sequelae. CONCLUSION: Nonradiation-based conditioning followed by empiric central nervous system-directed intrathecal chemotherapy may be considered for high-risk infants with leukemia.


Assuntos
Transplante de Medula Óssea , Neoplasias do Sistema Nervoso Central/terapia , Citarabina/administração & dosagem , Leucemia Mieloide Aguda/terapia , Agonistas Mieloablativos/administração & dosagem , Condicionamento Pré-Transplante , Aloenxertos , Feminino , Humanos , Lactente , Injeções Espinhais , Recidiva
4.
Neuro Oncol ; 22(12): 1862-1872, 2020 12 18.
Artigo em Inglês | MEDLINE | ID: mdl-32304218

RESUMO

BACKGROUND: "Head Start" III, was a prospective clinical trial using intensive induction followed by myeloablative chemotherapy and autologous hematopoietic cell rescue (AuHCR) to either avoid or reduce the dose/volume of irradiation in young children with medulloblastoma. METHODS: Following surgery, patients received 5 cycles of induction followed by myeloablative chemotherapy using carboplatin, thiotepa, and etoposide with AuHCR. Irradiation was reserved for children >6 years old at diagnosis or with residual tumor post-induction. RESULTS: Between 2003 and 2009, 92 children <10 years old with medulloblastoma were enrolled. Five-year event-free survival (EFS) and overall survival (OS) rates (±SE) were 46 ±â€…5% and 62 ±â€…5% for all patients, 61 ±â€…8% and 77 ±â€…7% for localized medulloblastoma, and 35 ±â€…7% and 52 ±â€…7% for disseminated patients. Nodular/desmoplastic (ND) medulloblastoma patients had 5-year EFS and OS (±SE) rates of 89 ±â€…6% and 89 ±â€…6% compared with 26 ±â€…6% and 53 ±â€…7% for classic and 38 ±â€…13% and 46 ±â€…14% for large-cell/anaplastic (LCA) medulloblastoma, respectively. In multivariate Cox regression analysis, histology was the only significant independent predictor of EFS after adjusting for stage, extent of resection, regimen, age, and sex (P <0.0001). Five-year irradiation-free EFS was 78 ±â€…8% for ND and 21 ±â€…5% for classic/LCA medulloblastoma patients. Myelosuppression was the most common toxicity, with 2 toxic deaths. Twenty-four survivors completed neurocognitive evaluation at a mean of 4.9 years post-diagnosis. IQ and memory scores were within average range overall, whereas processing speed and adaptive functioning were low-average. CONCLUSION: We report excellent survival and preservation of mean IQ and memory for young children with ND medulloblastoma using high-dose chemotherapy, with most patients surviving without irradiation.


Assuntos
Neoplasias Cerebelares , Intervenção Educacional Precoce , Meduloblastoma , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Carboplatina/uso terapêutico , Neoplasias Cerebelares/tratamento farmacológico , Criança , Pré-Escolar , Terapia Combinada , Feminino , Humanos , Masculino , Meduloblastoma/tratamento farmacológico , Estudos Prospectivos , Taxa de Sobrevida
5.
J Neuropathol Exp Neurol ; 78(12): 1089-1099, 2019 12 01.
Artigo em Inglês | MEDLINE | ID: mdl-31626289

RESUMO

ROS1 is a transmembrane receptor tyrosine kinase proto-oncogene that has been shown to have rearrangements with several genes in glioblastoma and other neoplasms, including intrachromosomal fusion with GOPC due to microdeletions at 6q22.1. ROS1 fusion events are important findings in these tumors, as they are potentially targetable alterations with newer tyrosine kinase inhibitors; however, whether these tumors represent a distinct entity remains unknown. In this report, we identify 3 cases of unusual pediatric glioma with GOPC-ROS1 fusion. We reviewed the clinical history, radiologic and histologic features, performed methylation analysis, whole genome copy number profiling, and next generation sequencing analysis for the detection of oncogenic mutation and fusion events to fully characterize the genetic and epigenetic alterations present in these tumors. Two of 3 tumors showed pilocytic features with focal expression of synaptophysin staining and variable high-grade histologic features; the third tumor aligned best with glioblastoma and showed no evidence of neuronal differentiation. Copy number profiling revealed chromosome 6q22 microdeletions corresponding to the GOPC-ROS1 fusion in all 3 cases and methylation profiling showed that the tumors did not cluster together as a single entity or within known methylation classes by t-Distributed Stochastic Neighbor Embedding.


Assuntos
Proteínas Adaptadoras de Transdução de Sinal/genética , Neoplasias Encefálicas/genética , Deleção Cromossômica , Cromossomos Humanos Par 6/genética , Glioma/genética , Proteínas da Matriz do Complexo de Golgi/genética , Proteínas de Fusão Oncogênica/genética , Proteínas Tirosina Quinases/genética , Proteínas Proto-Oncogênicas/genética , Encéfalo/patologia , Neoplasias Encefálicas/patologia , Carcinogênese , Criança , Pré-Escolar , Metilação de DNA , Epigênese Genética , Feminino , Glioma/patologia , Humanos , Masculino , Proto-Oncogene Mas
6.
J Neurooncol ; 140(3): 717-725, 2018 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-30392092

RESUMO

BACKGROUND: The dismal outcome in children with high-grade brainstem gliomas (BSG) accentuates the need for effective therapeutic strategies. We investigated the role of intensive, including marrow-ablative, chemotherapy regimens in the treatment of young children with newly-diagnosed high-grade BSG. METHODS: Between 1991-and-2002, 15 eligible children less than 10 years of age with a diagnosis of high-grade BSG were treated on "Head-Start" I and II protocols (HSI and HSII). Treatment included Induction with 4-5 cycles of one of three intensive chemotherapy regimens followed by Consolidation with one cycle of marrow-ablative chemotherapy (thiotepa, carboplatin and etoposide) with autologous hematopoietic cell rescue (AHCR). Irradiation was required for children over 6 years of age or for those with residual tumor at the end of Consolidation. RESULTS: We had two long-term survivors who were found retrospectively to harbor low-grade glial tumors and thus were not included in the survival analysis. Of the remaining 13 patients, the 1-year event-free (EFS) and overall (OS) survival for these children were 31% (95% CI 9-55%) and 38% (95% CI 14-63%), respectively. Median EFS and OS were 6.6 (95% CI 2.7, 12.7) and 8.7 months (95% CI 6.9, 20.9), respectively. Eight patients developed progressive disease during study treatment (seven during Induction and one at the end of Consolidation). Ten children received focal irradiation, five for residual tumor (three following Induction and two following Consolidation) and five due to disease progression. CONCLUSIONS: Children with high-grade BSG did not benefit from this intensive chemotherapy strategy administered prior to irradiation.


Assuntos
Neoplasias do Tronco Encefálico/tratamento farmacológico , Neoplasias do Tronco Encefálico/radioterapia , Quimioterapia de Consolidação , Glioma/tratamento farmacológico , Glioma/radioterapia , Quimioterapia de Indução , Protocolos de Quimioterapia Combinada Antineoplásica , Medula Óssea/efeitos dos fármacos , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Recém-Nascido , Estimativa de Kaplan-Meier , Masculino , Resultado do Tratamento
7.
Neuro Oncol ; 20(11): 1547-1555, 2018 10 09.
Artigo em Inglês | MEDLINE | ID: mdl-29741745

RESUMO

Background: Diagnosis of diffuse intrinsic pontine glioma (DIPG) has relied on imaging studies, since the appearance is pathognomonic, and surgical risk was felt to be high and unlikely to affect therapy. The DIPG Biology and Treatment Study (DIPG-BATS) reported here incorporated a surgical biopsy at presentation and stratified subjects to receive FDA-approved agents chosen on the basis of specific biologic targets. Methods: Subjects were eligible for the trial if the clinical features and imaging appearance of a newly diagnosed tumor were consistent with a DIPG. Surgical biopsies were performed after enrollment and prior to definitive treatment. All subjects were treated with conventional external beam radiotherapy with bevacizumab, and then stratified to receive bevacizumab with erlotinib or temozolomide, both agents, or neither agent, based on O6-methylguanine-DNA methyltransferase status and epidermal growth factor receptor expression. Whole-genome sequencing and RNA sequencing were performed but not used for treatment assignment. Results: Fifty-three patients were enrolled at 23 institutions, and 50 underwent biopsy. The median age was 6.4 years, with 24 male and 29 female subjects. Surgical biopsies were performed with a specified technique and no deaths were attributed to the procedure. Two subjects experienced grade 3 toxicities during the procedure (apnea, n = 1; hypertension, n = 1). One subject experienced a neurologic deficit (left hemiparesis) that did not fully recover. Of the 50 tumors biopsied, 46 provided sufficient tissue to perform the study assays (92%, two-stage exact binomial 90% CI: 83%-97%). Conclusions: Surgical biopsy of DIPGs is technically feasible, associated with acceptable risks, and can provide biologic data that can inform treatment decisions.


Assuntos
Neoplasias do Tronco Encefálico/patologia , Glioma/patologia , Imageamento por Ressonância Magnética/métodos , Adolescente , Biópsia , Neoplasias do Tronco Encefálico/cirurgia , Criança , Pré-Escolar , Estudos de Viabilidade , Feminino , Seguimentos , Glioma/cirurgia , Humanos , Masculino , Morbidade , Prognóstico , Estudos Prospectivos
8.
J Clin Oncol ; 36(19): 1963-1972, 2018 07 01.
Artigo em Inglês | MEDLINE | ID: mdl-29746225

RESUMO

Purpose Diffuse intrinsic pontine glioma (DIPG) is a brainstem malignancy with a median survival of < 1 year. The International and European Society for Pediatric Oncology DIPG Registries collaborated to compare clinical, radiologic, and histomolecular characteristics between short-term survivors (STSs) and long-term survivors (LTSs). Materials and Methods Data abstracted from registry databases included patients from North America, Australia, Germany, Austria, Switzerland, the Netherlands, Italy, France, the United Kingdom, and Croatia. Results Among 1,130 pediatric and young adults with radiographically confirmed DIPG, 122 (11%) were excluded. Of the 1,008 remaining patients, 101 (10%) were LTSs (survival ≥ 2 years). Median survival time was 11 months (interquartile range, 7.5 to 16 months), and 1-, 2-, 3-, 4-, and 5-year survival rates were 42.3% (95% CI, 38.1% to 44.1%), 9.6% (95% CI, 7.8% to 11.3%), 4.3% (95% CI, 3.2% to 5.8%), 3.2% (95% CI, 2.4% to 4.6%), and 2.2% (95% CI, 1.4% to 3.4%), respectively. LTSs, compared with STSs, more commonly presented at age < 3 or > 10 years (11% v 3% and 33% v 23%, respectively; P < .001) and with longer symptom duration ( P < .001). STSs, compared with LTSs, more commonly presented with cranial nerve palsy (83% v 73%, respectively; P = .008), ring enhancement (38% v 23%, respectively; P = .007), necrosis (42% v 26%, respectively; P = .009), and extrapontine extension (92% v 86%, respectively; P = .04). LTSs more commonly received systemic therapy at diagnosis (88% v 75% for STSs; P = .005). Biopsies and autopsies were performed in 299 patients (30%) and 77 patients (10%), respectively; 181 tumors (48%) were molecularly characterized. LTSs were more likely to harbor a HIST1H3B mutation (odds ratio, 1.28; 95% CI, 1.1 to 1.5; P = .002). Conclusion We report clinical, radiologic, and molecular factors that correlate with survival in children and young adults with DIPG, which are important for risk stratification in future clinical trials.


Assuntos
Neoplasias do Tronco Encefálico/diagnóstico , Sobreviventes de Câncer/estatística & dados numéricos , Glioma/diagnóstico , Adolescente , Adulto , Neoplasias do Tronco Encefálico/diagnóstico por imagem , Neoplasias do Tronco Encefálico/genética , Neoplasias do Tronco Encefálico/terapia , Criança , Pré-Escolar , Glioma/diagnóstico por imagem , Glioma/genética , Glioma/terapia , Humanos , Lactente , Recém-Nascido , Estimativa de Kaplan-Meier , Sistema de Registros , Adulto Jovem
9.
Am J Infect Control ; 42(11): 1157-60, 2014 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-25444262

RESUMO

BACKGROUND: Although several studies have estimated the attributable cost and length of stay (LOS) of central line-associated bloodstream infections (CLABSIs) in the pediatric intensive care unit setting, little is known about the attributable costs and LOS of CLABSIs in the vulnerable pediatric hematology/oncology population. METHODS: We studied a total of 1562 inpatient admissions for 291 pediatric hematology/oncology patients at a single tertiary care children's hospital in the mid-Atlantic region between January 2008 and May 2011. Costs were normalized to year 2011 dollars. Propensity score matching was used to estimate the effect of CLABSIs on total cost and LOS while controlling for other covariates. RESULTS: Sixty CLABSIs occurred during the 1562 admissions. Compared with the patients without a CLABSI, those who developed a CLABSI tended to be older (9.0 years vs 7.5 years; P = .026) and to have a tunneled catheter (46.7% vs 27.0%) and a peripherally inserted central catheter (20.0% vs 11.2%) as opposed to other types of catheters (P < .0001). Propensity score matching yielded matched groups without significant differences in patient characteristics. In the propensity score analysis, the attributable LOS of a CLABSI was 21.2 days (P < .0001), and the attributable cost of a CLABSI was $69,332 (P < .0001). CONCLUSIONS: Among pediatric hematology/oncology patients, CLABSI was associated with an additional LOS of 21 days and increased costs of nearly $70,000. These findings may inform decisions regarding the value of investing in efforts to prevent CLABSIs in this vulnerable population.


Assuntos
Infecções Relacionadas a Cateter/economia , Neoplasias Hematológicas/complicações , Sepse/economia , Adolescente , Infecções Relacionadas a Cateter/epidemiologia , Criança , Pré-Escolar , Feminino , Custos de Cuidados de Saúde , Humanos , Lactente , Recém-Nascido , Tempo de Internação , Masculino , Sepse/epidemiologia , Centros de Atenção Terciária , Estados Unidos/epidemiologia
10.
Am J Med Qual ; 29(6): 484-90, 2014.
Artigo em Inglês | MEDLINE | ID: mdl-24226650

RESUMO

This study reports the results of an initiative to reduce central line-associated bloodstream infections (CLABSIs) among pediatric hematology/oncology patients, a population at increased risk for CLABSI. The study design was a pre-post comparison of a series of specific interventions over 40 months. Logistic regression was used to determine if the risk of developing CLABSI decreased in the postintervention period, after controlling for covariates. The overall CLABSI rate fell from 9 infections per 1000 line days at the beginning of the study to zero in a cohort of 291 patients encompassing 2107 admissions. Admissions during the intervention period had an 86% reduction in odds of developing a CLABSI, controlling for other factors. At the study team's institution, an initiative that standardized blood culturing techniques, lab draw times, line care techniques, and provided physician and nurse education was able to eliminate CLABSI among pediatric hematology/oncology patients.


Assuntos
Infecções Relacionadas a Cateter/prevenção & controle , Cateterismo Venoso Central/efeitos adversos , Infecção Hospitalar/prevenção & controle , Doenças Hematológicas/complicações , Neoplasias/complicações , Infecções Relacionadas a Cateter/epidemiologia , Cateterismo Venoso Central/métodos , Criança , Infecção Hospitalar/epidemiologia , Feminino , Doenças Hematológicas/terapia , Humanos , Masculino , Neoplasias/terapia , Melhoria de Qualidade , Fatores de Risco
11.
Pediatr Blood Cancer ; 61(4): 636-42, 2014 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-24123865

RESUMO

BACKGROUND: Preclinical models show that an antiangiogenic regimen at low-dose daily (metronomic) dosing may be effective against chemotherapy-resistant tumors. We undertook a prospective, open-label, single-arm, multi-institutional phase II study to evaluate the efficacy of a "5-drug" oral regimen in children with recurrent or progressive cancer. PROCEDURE: Patients ≤21 years old with recurrent or progressive tumors were eligible. Treatment consisted of continuous oral celecoxib, thalidomide, and fenofibrate, with alternating 21-day cycles of low-dose cyclophosphamide and etoposide. Primary endpoint was to assess, within eight disease strata, activity of the 5-drug regimen over 27 weeks. Blood and urine angiogenesis markers were assessed. RESULTS: One hundred one patients were enrolled; 97 began treatment. Median age was 10 years (range: 191 days-21 years); 47 (49%) were female. Disease strata included high-grade glioma (HGG, 21 patients), ependymoma (19), low-grade glioma (LGG, 12), bone tumors (12), medulloblastoma/primitive neuroectodermal tumor (PNET, 8), leukemia (4), neuroblastoma (3), and miscellaneous tumors (18). Treatment was generally well tolerated; most common toxicities were hematologic. Twenty-four (25%) patients completed 27 weeks therapy without progression, including HGG: 1 (5%), ependymoma: 7 (37%), LGG: 7 (58%), medulloblastoma/PNET: 1, neuroblastoma: 1, and miscellaneous tumors: 7 (39%). Best response was complete response (one patient with medulloblastoma), partial response (12), stable disease (36), progressive disease (47), and inevaluable (1). Baseline serum thrombospondin levels were significantly higher in patients successfully completing therapy than in those who progressed (P = 0.009). CONCLUSION: The 5-drug regimen was well tolerated. Clinical activity was demonstrated in some but not all tumor strata.


Assuntos
Inibidores da Angiogênese/uso terapêutico , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Recidiva Local de Neoplasia/tratamento farmacológico , Neoplasias/tratamento farmacológico , Neovascularização Patológica/tratamento farmacológico , Adolescente , Adulto , Celecoxib , Criança , Pré-Escolar , Ciclofosfamida/administração & dosagem , Etoposídeo/administração & dosagem , Feminino , Fenofibrato/administração & dosagem , Seguimentos , Humanos , Lactente , Masculino , Recidiva Local de Neoplasia/patologia , Estadiamento de Neoplasias , Neoplasias/patologia , Prognóstico , Estudos Prospectivos , Pirazóis/administração & dosagem , Sulfonamidas/administração & dosagem , Taxa de Sobrevida , Talidomida/administração & dosagem , Adulto Jovem
12.
Pediatr Blood Cancer ; 56(5): 799-804, 2011 May.
Artigo em Inglês | MEDLINE | ID: mdl-21370414

RESUMO

BACKGROUND: Children with acute lymphoblastic leukemia (ALL) are at risk for developing musculoskeletal complications during and after their medical treatment. The objective of this study was to examine the feasibility of an in-hospital physical therapy- and home exercise program during the first four phases of medical treatment, for children with newly diagnosed ALL. PROCEDURE: Nine patients, between the ages of 2-14 years old were enrolled within 2 weeks of diagnosis in the study. Each patient was evaluated at study entry, after each of the first four phases of therapy and each time patients were re-admitted to the hospital. Following the initial physical therapy evaluation an individualized home exercise program was developed, consisting of stretching, strengthening, and aerobic exercises. The following end points were measured at each evaluation: gross motor assessment as measured by gross motor function measure (GMFM), health-related quality of life as measured by the PedsQL and parent satisfaction questionnaire. RESULTS: This study was feasible with 98% of the evaluation sessions completed. The GMFM and PedsQL improved steadily throughout the study; however, the PedsQL slightly decreased from interim maintenance to delayed intensification. The parents reported being satisfied with the physical therapy program. CONCLUSION: We demonstrated that an in-hospital- and home exercise physical therapy program during the first four phases of medical treatments is feasible for children with ALL. Future randomized studies are needed to confirm whether an initial physical therapy program at diagnosis in children with ALL will limit functional impairments, improve overall fitness and increase health-related quality of life.


Assuntos
Terapia por Exercício , Exercício Físico , Modalidades de Fisioterapia , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Adolescente , Criança , Pré-Escolar , Estudos de Viabilidade , Feminino , Humanos , Masculino , Pais , Satisfação Pessoal , Resultado do Tratamento
13.
Pediatr Hematol Oncol ; 27(3): 179-87, 2010 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-20367261

RESUMO

Children with acute lymphoblastic leukemia (ALL) are at risk for developing musculoskeletal complications. Few studies have examined the role of physical therapy (PT) for addressing these complications. The study objective was to examine why and when in the medical treatment, children with ALL receive a referral to PT. Retrospective chart review of 35 children, diagnosed with ALL in 2006 and 2007 at Penn State Children's Hospital was carried out. A questionnaire was completed by 6 pediatric oncologists, to identify their referral patterns. The chart review demonstrated that 25 of the 35 patients had reports of musculoskeletal complications sometime during their treatment, but only 10 (30%) were referred to PT. The most common reason for referral was decreased functional mobility. Patients were referred evenly through all phases of therapy. Vincristine was reduced in 5 patients, the most common cause being peripheral neuropathy and foot drop. The majority (5/6) of the physicians reported that they "sometimes" refer patients to PT, through different phases of therapy, with 1/3 reporting all phases. The results show that although physicians identified the musculoskeletal complications, only a minority of patients were referred for PT. This supports the need for increasing the awareness of physicians about benefits of early integration of PT into the medical treatment.


Assuntos
Modalidades de Fisioterapia , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Encaminhamento e Consulta/estatística & dados numéricos , Adolescente , Adulto , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Masculino , Leucemia-Linfoma Linfoblástico de Células Precursoras/complicações , Estudos Retrospectivos
14.
Pediatr Blood Cancer ; 50(2): 312-8, 2008 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-17668858

RESUMO

BACKGROUND: Children with newly diagnosed supratentorial primitive neuroectodermal tumors (sPNET) have poor outcomes compared to medulloblastoma patients, despite similar treatments. In an effort to improve overall survival (OS) and event-free survival (EFS) and to decrease radiation exposure, the Head Start (HS) protocols treated children with newly diagnosed sPNET utilizing intensified induction chemotherapy (ICHT) followed by consolidation with myeloablative chemotherapy and autologous hematopoietic cell rescue (AuHCR). PROCEDURES: Between 1991 and 2002, 43 children with sPNET were prospectively treated on two serial studies (HS I and II). After maximal safe surgical resection, patients on HS I and patients with localized disease on HS II were treated with five cycles of ICHT (vincristine, cisplatin, cyclophosphamide, and etoposide). Patients on HS II with disseminated disease received high-dose methotrexate during ICHT. If the disease remained stable or in response, patients received a single cycle of high-dose myeloablative chemotherapy followed by AuHCR. RESULTS: Five-year EFS and OS were 39% (95%CI: 24%, 53%) and 49 (95%CI: 33%, 62%), respectively. Non-pineal sPNET patients faired significantly better than those patients with pineal sPNETs. Metastasis at diagnosis, age, and extent of resection were not significant prognostic factors. Sixty percent of survivors (12 of 20) are alive without exposure to radiation therapy. CONCLUSIONS: ICHT followed by AuHCR in young patients with newly diagnosed sPNET appears to not only provide an improved EFS and OS for patients who typically have a poor prognosis, but also it successfully permitted deferral and elimination of radiation therapy in a significant proportion of patients.


Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Transplante de Células-Tronco Hematopoéticas , Tumores Neuroectodérmicos Primitivos/terapia , Neoplasias Supratentoriais/terapia , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Carboplatina/administração & dosagem , Pré-Escolar , Cisplatino/administração & dosagem , Terapia Combinada , Ciclofosfamida/administração & dosagem , Intervalo Livre de Doença , Etoposídeo/administração & dosagem , Feminino , Fator Estimulador de Colônias de Granulócitos/administração & dosagem , Humanos , Masculino , Mesna/administração & dosagem , Metotrexato/administração & dosagem , Tumores Neuroectodérmicos Primitivos/tratamento farmacológico , Tumores Neuroectodérmicos Primitivos/radioterapia , Tumores Neuroectodérmicos Primitivos/cirurgia , Neoplasias Supratentoriais/tratamento farmacológico , Neoplasias Supratentoriais/radioterapia , Neoplasias Supratentoriais/cirurgia , Taxa de Sobrevida , Vincristina/administração & dosagem
15.
Pediatr Blood Cancer ; 50(5): 1056-7, 2008 May.
Artigo em Inglês | MEDLINE | ID: mdl-17828748

RESUMO

IPEX syndrome (immune deficiency, polyendocrinopathy, enteropathy, X-linked) is a disorder or regulatory T cell (Treg) function which can result in early death due to infection or complications related to autoimmunity. Therapeutic options for these patients can include allogeneic stem cell transplantation (SCT) or the use of immunosuppressive regimens to control the manifestations of autoimmunity. We report a patient with IPEX syndrome who was managed with rapamycin and subsequently developed EBV induced lymphoma.


Assuntos
Infecções por Vírus Epstein-Barr/virologia , Doenças Genéticas Ligadas ao Cromossomo X/complicações , Herpesvirus Humano 4/patogenicidade , Síndromes de Imunodeficiência/complicações , Linfoma/virologia , Poliendocrinopatias Autoimunes/complicações , Enteropatias Perdedoras de Proteínas/complicações , Criança , Infecções por Vírus Epstein-Barr/tratamento farmacológico , Doenças Genéticas Ligadas ao Cromossomo X/patologia , Doenças Genéticas Ligadas ao Cromossomo X/terapia , Humanos , Síndromes de Imunodeficiência/patologia , Síndromes de Imunodeficiência/terapia , Imunossupressores/uso terapêutico , Lactente , Linfoma/tratamento farmacológico , Masculino , Poliendocrinopatias Autoimunes/patologia , Poliendocrinopatias Autoimunes/terapia , Enteropatias Perdedoras de Proteínas/patologia , Enteropatias Perdedoras de Proteínas/terapia , Sirolimo/uso terapêutico , Síndrome
16.
Am J Surg Pathol ; 30(3): 328-36, 2006 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-16538052

RESUMO

Reported are 7 cases of posttransplant lymphoproliferative disorder (PTLD) arising in children who received umbilical cord blood transplantation (UCBT). There were 4 females and 3 males with a median age of 3 years (range, 1-16 years). All 7 patients received UCBT, including 1 patient who received multiple units and 1 transplanted under nonmyeloablative condition. The time interval from UCBT to PTLD averaged 4 months (range, 2 weeks to 9 months). Patients typically presented with high-stage disease with visceral organ involvement. Histology of the PTLDs showed monomorphic morphology in 5 cases and polymorphic morphology in the remaining 2 cases. Bone marrow biopsies were performed in 3 cases and were negative for PTLD. Epstein-Barr virus (EBV) was detected in the PTLD in all 7 patients by in situ hybridization. Evidence of past EBV infection was found in the recipients, but the EBV genome was not detected in the donor cord blood samples, suggesting that donor cord blood was not the source of EBV infection. The origin of the PTLD was investigated in 5 cases. PTLD was of host origin in 2 patients who failed engraftment and of donor origin in the remaining 3 patients who had complete engraftment. Four of 5 patients with monomorphic PTLD failed to demonstrate significant responses to rituximab and/or reduction of immunosuppression and died within 1 month after diagnosis. The remaining 2 patients with polymorphic PTLD showed complete response to therapy. One patient was alive 35 months after transplant, and the other patient died of infection 6 months after transplant. It is concluded that PTLD arising after UCBT in children occurs early after transplant and represents a serious EBV-related complication. PTLD may be of donor or recipient origin depending on engraftment status. Both monomorphic and polymorphic histology may be seen, and monomorphic histology appears to predict an unfavorable prognosis.


Assuntos
Transplante de Células-Tronco de Sangue do Cordão Umbilical/efeitos adversos , Transtornos Linfoproliferativos/etiologia , Transtornos Linfoproliferativos/patologia , Adolescente , Criança , Pré-Escolar , Infecções por Vírus Epstein-Barr/epidemiologia , Feminino , Citometria de Fluxo , Herpesvirus Humano 4/isolamento & purificação , Humanos , Imuno-Histoquímica , Hibridização in Situ Fluorescente , Lactente , Masculino , Reação em Cadeia da Polimerase , Proteínas de Ligação a RNA/análise , Proteínas Ribossômicas/análise
17.
Pediatr Emerg Care ; 21(5): 309-11, 2005 May.
Artigo em Inglês | MEDLINE | ID: mdl-15874813

RESUMO

OBJECTIVE: To report a case of idiopathic thrombocytopenic purpura (ITP) complicated by an intracranial hemorrhage (ICH) in a child with a previously undiagnosed arteriovenous malformation. CASE: We describe a child with known ITP who developed a severe headache, was evaluated in an emergency department of a community hospital, and was found by computer tomography (CT) scan to have an ICH. Despite treatment with platelets, corticosteroids, and intravenous immunoglobulin, she subsequently developed an acute change in mental status. A second CT scan showed that the hemorrhage had significantly increased in size despite treatment. The patient underwent an emergent splenectomy prior to a craniotomy to remove the hemorrhage. At the time of surgery, it was discovered that she had an arteriovenous malformation at the sight of the hemorrhage. Her recovery was unremarkable and she was discharged to home with no neurologic sequelae. CONCLUSIONS: ICH is a rare but life-threatening complication of ITP. Neurologic symptoms in a child with ITP should be quickly evaluated by CT scan. Most experts suggest careful observation for most cases of ITP. However, when neurologic symptoms occur, more aggressive treatment options must be used. Care of this child included an emergency splenectomy prior to her craniotomy. Pediatric emergency medicine practitioners must be aware of these neurologic symptoms and must not hesitate to involve pediatric surgeons and neurosurgeons in the care of the child. Prompt recognition and early intervention are the keys to improving outcomes when ICH complicates ITP.


Assuntos
Malformações Arteriovenosas Intracranianas/complicações , Hemorragias Intracranianas/etiologia , Púrpura Trombocitopênica Idiopática/complicações , Criança , Feminino , Humanos , Malformações Arteriovenosas Intracranianas/terapia , Hemorragias Intracranianas/terapia , Púrpura Trombocitopênica Idiopática/terapia
18.
Leuk Lymphoma ; 45(10): 1981-7, 2004 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-15370241

RESUMO

Epstein-Barr Virus (EBV) is associated with a number of tumors, including lymphomas in solid organ transplant (SOT) and hematopoietic stem cell transplant (HSCT) recipients, patients with the acquired immunodeficiency syndrome (AIDS), Burkitt's lymphoma, as well as a subset of patients with nasopharyngeal carcinoma (NPC) and Hodgkin's disease (HD). The types of latent EBV infections vary in these tumors, which influences the EBV antigens expressed and ultimately the immunogenicity of tumor cells. Not all EBV associated malignancies are directly related to altered cellular immunity, as is the case with EBV induced lymphoproliferations in immunocompromised patients. Treatment strategies have ranged from restoration of normal cellular immunity, which is generally successful in SOT and HSCT patients, anti-B cell monoclonal antibodies, and conventional chemotherapy and radiation. The fact that these tumors express EBV antigens for which many individuals have high circulating levels of protective cytotoxic T lymphocytes (CTL) has lead to investigation into the applicability of adoptive transfer of EBV specific T cells. Initial success with adoptive immunotherapy for HSCT and SOT patients has lead to current studies examining the feasibility and efficacy of this strategy for other EBV associated tumors, such as NPC and HD. We will review the pathogenesis of these disorders, current therapies, and future investigations aimed at targeting EBV antigen expression on these tumors.


Assuntos
Herpesvirus Humano 4/imunologia , Imunoterapia Adotiva/métodos , Linfoma/terapia , Antígenos Virais/imunologia , Antígenos Virais/uso terapêutico , Humanos , Linfoma/virologia , Neoplasias/terapia , Neoplasias/virologia , Linfócitos T Citotóxicos/imunologia , Linfócitos T Citotóxicos/transplante , Transplante
19.
Orbit ; 18(1): 17-24, 1999 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-12048694

RESUMO

A previously healthy 12-year-old boy presented with acute onset of proptosis of his left eye. CT scan demonstrated a mass involving the left orbit, left maxillary sinus, and left ethmoid sinus with extension through the cribriform plate into the anterior cranial fossa. Incisional biopsy of the mass revealed a precursor B-cell lymphoblastic lymphoma. Precursor B-cell lymphoblastic lymphoma is a rare type of non-Hodgkin's lymphoma seen exclusively in children and young adults. This is the first reported case of precursor B-cell lymphoblastic lymphoma presenting in the orbit. Treatment is primarily by systemic chemotherapy and is potentially curative. The principal role of the ophthalmologist is in diagnosis and monitoring of such patients. The clinical features and multidisciplinary diagnosis and management of childhood non-Hodgkin's lymphomas are reviewed.

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