ISSN:1077-4114    

出版商:OVID    

年代:2003

数据来源: OVID

ISSN:1077-4114    

出版商:OVID    

年代:2003

数据来源: OVID

摘要:

ObjectivesTo describe features of patients with acute myeloid leukemia presenting with extramedullary leukemic tumors (EML).MethodsAmong 1,832 patients entered on Children's Cancer Group's chemotherapy trials with acute myeloid leukemia, 199 patients had EML, defined as any leukemic collection outside the bone marrow cavity. Three patient groups were denoted: group 1 (n = 109) with EML involving skin (with or without other sites of EML), group 2 (n = 90) with EML in sites other than skin, and group 3 (n = 1,633) without EML.ResultsThe incidence of EML was 10.9%. Group 1 patients tended to be younger, had higher white blood cell counts, were more often CNS positive, had FAB M4 or M5 subtypes, and possessed more abnormalities of chromosome 11 than group 3 patients. Group 2 patients were younger, more often had the FAB M2 subtype, and had a higher incidence of t(8;21)(q22;q22) abnormality than group 3, but had similar white blood cell counts and incidence of CNS positivity at diagnosis. For group 1 the 5-year event-free survival was 26%, significantly worse than for group 3 at 29%. Event-free survival was better for group 2 patients (5-year estimate 46%), which remained a favorable prognostic factor by multivariate analysis. The authors retrospectively determined whether 118 (59%) of the EML patients received localized radiotherapy to the site of EML: 42 did and 76 did not. There were no differences in estimated event-free survival between patients who did and did not receive radiotherapy.ConclusionsNon-skin (group 2) EML appeared to be an independent favorable prognostic factor. Localized radiotherapy to the site of EML at the end of induction chemotherapy did not improve outcome.

ISSN:1077-4114    

出版商:OVID    

年代:2003

数据来源: OVID

摘要:

PurposeTreatment of refractory neuroblastoma remains a significant clinical problem. Targeted radiotherapy with131I-MIBG has demonstrated antitumor activity in heavily pretreated neuroblastoma patients with recurrent disease. Response rates may be correlated with total radionuclide dose per kilogram body weight delivered, but higher dose levels are associated with protracted grade 4 hematologic toxicity. The optimal method for using single-agent131I-MIBG for patients with relapsed high-risk neuroblastoma has not been defined. This study was designed to retrospectively determine the clinical response to131I-MIBG therapy at submyeloablative doses in patients with refractory neuroblastoma and to describe the toxicities.Patients and MethodsA retrospective chart review of 20 patients with neuroblastoma treated with131I-MIBG at the Children's Hospital of Philadelphia from 1988 to 2000 was performed. Demographic data,131I-MIBG dose delivered, toxicities, and clinical responses were reviewed.ResultsA median dose of 9.5 mCi/kg of131I-MIBG was delivered in 32 courses to 20 patients. Three patients were treated in first complete response, and the remaining 17 patients for residual and/or progressive disease. The objective response rate to the first therapy was 31%, and the remaining patients achieved disease stabilization. In addition, 9 of 11 patients with pain at study entry had significant improvement. Disease response was not correlated with131I-MIBG dose delivered. No unanticipated toxicities were observed.ConclusionsSubmyeloablative-dose131I-MIBG is an effective and relatively nontoxic method for neuroblastoma disease palliation. Most patients show subjective improvement in pain and/or performance status. Increased availability and experience with131I-MIBG therapy would benefit a large number of children with end-stage neuroblastoma and no realistic hope for cure.

ISSN:1077-4114    

出版商:OVID    

年代:2003

数据来源: OVID

摘要:

PurposeTo evaluate local control, event-free survival, and overall survival for patients with parameningeal (PM) rhabdomyosarcoma (RMS) treated with intensive chemotherapy and delayed irradiation.Patients and MethodsThirteen consecutive patients with PM RMS were treated with an institutional protocol from 1992 to 1998 at the University of Washington/Children's Hospital and Regional Medical Center and Deaconess Medical Center. Patients received intensive chemotherapy consisting of vincristine, doxorubicin, cyclophosphamide, ifosfamide, and etoposide prior to radiotherapy. Irradiation was delayed, in contrast to current Intergroup Rhabdomyosarcoma Study Group (IRSG) recommendations.ResultsMedian follow-up was 39 months. Eleven patients had high-risk features, including five with intracranial extension. All patients responded to the intensive chemotherapy, with 38% exhibiting a complete response and the remaining 62% a partial response. Radiation was administered a median of 21 weeks from initiation of chemotherapy. The Kaplan-Meier estimate of 5-year local control was 92%, with event-free survival and overall survival rates of 83%.ConclusionsWith intensive induction chemotherapy, delayed irradiation for PM RMS does not compromise local control. Event-free survival and overall survival rates compare favorably with recently IRSG trials employing early irradiation. Delaying irradiation allows for intensification of chemotherapy and could permit response-based radiation volume and/or dose modifications, which could decrease treatment-related morbidity.

ISSN:1077-4114    

出版商:OVID    

年代:2003

数据来源: OVID

摘要:

PurposeThe authors' objectives were to compare height at diagnosis of children with bone tumors with that of Spanish reference children; to analyze the frequency of the genotypes for the polymorphisms of the vitamin D receptor (VDR), estrogen receptor (ER), and collagen I&agr;1 (COLI&agr;1) genes in patients and in healthy controls; and to test the relationship between the genetic markers and height.Patients and MethodsHeight and weight at diagnosis were measured in 58 osteosarcoma and 36 Ewing sarcoma patients and compared with standards published for Spanish reference children according to sex and age. For the molecular analysis, genetic polymorphisms of the VDR (Fok I, Apa I, and TaqI), ER (Pvu II and XbaI), and COLI&agr;1 (Msc I) genes were characterized in 72 osteosarcoma and 53 Ewing sarcomas and in a group of 143 healthy matched children.ResultsOsteosarcoma and Ewing sarcoma patients were significantly taller than Spanish reference children. Osteosarcoma patients showed a significantly higher frequency of theFfgenotype for the Fok I polymorphism (VDR gene) than the control group. The odds ratio for this genotype was 1.78, with an increased relative risk of 78% for heterozygousFfcarriers. Among Ewing sarcoma patients, this same genotype was significantly associated with lower height than homozygotes (FForff).ConclusionsChildren with bone cancer are significantly taller than the reference population, which may be influenced by the genotype for the Fok I polymorphism of the VDR gene.

ISSN:1077-4114    

出版商:OVID    

年代:2003

数据来源: OVID

ISSN:1077-4114    

出版商:OVID    

年代:2003

数据来源: OVID

摘要:

To better understand parental perceptions of the informed consent process in pediatric oncology clinical trials, 20 parents of newly diagnosed children at two pediatric cancer centers described their perceptions in a semi-structured interview. They recalled well the diagnosis, the general treatment plan, and the statistics of survival and/or cure, but the research nature of the clinical trials, particularly randomization, was not well understood. However, despite the need to assimilate a great deal of information, time pressure to make decisions, and reportedly high levels of distress during the discussions, parents expressed general satisfaction with the informed consent discussions with their pediatric oncology providers. However, half to two thirds of parents felt there had been inadequate discussion of alternatives to the proposed treatment and of the research nature of the protocol. While further study of the informed consent process should be conducted in larger, representative samples, the findings from this pilot study suggest that a goal of future informed consent interventions should be to improve parents' understanding of the research aspects of treatment. It is critical to parents' ability to provide informed consent that they feel satisfied that they know alternatives to proposed treatment and that they understand the randomization of treatments, which is the gold standard of clinical trials in pediatric oncology.

ISSN:1077-4114    

出版商:OVID    

年代:2003

数据来源: OVID

摘要:

Severe chronic neutropenia (SCN) is characterized by a profound neutropenia, which mostly presents during the neonatal period. The precise genetic basis of SCN remains elusive. Acquired somatic mutations involving the carboxy-terminus of the G-CSF receptor (G-CSFR) have been found, often in association with myelodysplastic syndrome. The authors describe a girl with SCN who did not respond to pharmacologic doses of filgrastim. Genetic analysis of bone marrow and germline cells revealed a 182-bp deletion in the extracellular domain of the G-CSFR. Co-precipitation studies showed an association between the wild-type and mutant G-CSFR, confirmed by their co-localization by confocal microscopy. Coexpression of the mutant receptor inhibited the wild-type response in Ba/F3 cells. These findings establish a novel constitutional defect in the G-CSFR that supports a partial dominant negative mechanism for receptor dysfunction in SCN.

ISSN:1077-4114    

出版商:OVID    

年代:2003

数据来源: OVID

摘要:

PurposeTo investigate the efficacy and side effects of two different intravenous immunoglobulin (IVIG) dose regimens for the initial treatment of childhood acute immune thrombocytopenic purpura (ITP).MethodsThirty-four consecutive patients with a clinical diagnosis of acute ITP and a platelet count below 20 × 109/L were randomized to receive either 1 g/kg body weight (n = 17; group A) or 0.3 g/kg body weight (n = 17; group B) IVIG per day for 2 consecutive days (total dose 2 g/kg and 0.6 g/kg).ResultsFifteen of the 17 patients (88.2%) in group A and 13 of the 17 patients (76.5%) in group B achieved a platelet count of more than 20 × 109/L within 72 hours. The increase in platelet counts on day 2 and 3 was more pronounced in the high-dose group. Two patients in the high-dose group and four in the low-dose group were non-responders. Chronic disease occurred in three patients receiving 2 g/kg IVIG and in five patients receiving 0.6 g/kg IVIG. Side effects of IVIG administration were more common in the high-dose group.ConclusionsThe present study showed that platelet counts increased more rapidly after high-dose IVIG administration within the first 72 hours, although a platelet count of more than 20 × 109/L can be achieved also with low-dose IVIG in most children with acute ITP. For patients with very low platelet counts, doses higher than 0.6 g/kg seem, therefore, to be more effective.

ISSN:1077-4114    

出版商:OVID    

年代:2003

数据来源: OVID