Abstract:
BACKGROUND:Although screening for familial retinoblastoma has been shown to be beneficial we suspected that such screening programs may be less than optimal in developing countries (DC). METHODS:Retrospective cohort study comparing patients with familial retinoblastoma from five centers in DC (Argentina, Brazil, Turkey, Jordan, and Venezuela) versus a reference center in the USA. RESULTS:Ninety-two (32 from the USA and 60 from DC) patients were included. Forty-one (44.6%) patients avoided enucleation, 42 (45.7%) had 1 eye removed, and 9 (9.8%) underwent bilateral enucleation. Eleven (11.9%) had major pathology risk factors at enucleation. There were no cases of metastatic disease at diagnosis. Detection via screening was significantly less common in DC than in the USA (23.3% vs. 71.8%, P < 0.0001). Patients in DC were diagnosed at a significantly later age and with more advanced intraocular disease that led to increased risk of bilateral enucleation. Patients detected by screening in DC were significantly younger at diagnosis, had less advanced intraocular disease, better ocular preservation rates and survival results than those whose retinoblastoma was not detected via early screening. Five-year pEFS was 0.92 for the patients treated in the USA and 0.81 for the patients in DC (P = 0.42). Seven events occurred (extraocular relapse four in patients from DC and second malignancies in three). CONCLUSIONS:Patients with familial retinoblastoma are less likely to be diagnosed by screening in DC and had higher morbidity and mortality caused by recurrent extraocular retinoblastoma.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Chantada GL,Dunkel IJ,Qaddoumi I,Antoneli CB,Totah A,Canturk S,Nawaiseh I,Fandiño A,Pífano I,Peksayar G,Ribeiro KB,Abramson DHdoi
10.1002/pbc.21970subject
Has Abstractpub_date
2009-09-01 00:00:00pages
338-42issue
3eissn
1545-5009issn
1545-5017journal_volume
53pub_type
杂志文章abstract:BACKGROUND:In the pediatric population, pathologic bleeding is often challenging to identify. The pediatric bleeding questionnaire (PBQ) was developed as a screening tool for von Willebrand disease (VWD) but was designed to be self-completed by children above 12 years of age. The study objective was to determine whethe...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27709
更新日期:2019-06-01 00:00:00
abstract:BACKGROUND:Based on the presumed clinical similarity between the two most severe sickle cell disease (SCD) genotypes, hemoglobin (Hb) Sβ0 thalassemia and HbSS, randomized controlled trials (RCTs) have included both genotypes. Our group has demonstrated that healthcare providers inadequately distinguish the two diagnose...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27352
更新日期:2018-11-01 00:00:00
abstract:BACKGROUND:Retinoblastoma (Rb) is the most common intraocular primary malignancy in children. In industrialised countries, the cure rate is about 95%. We present the results of a prospective study on the management of Rb in the paediatric oncology unit of Gabriel Touré Teaching Hospital and African Institute of Tropica...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27101
更新日期:2018-08-01 00:00:00
abstract::Pediatric renal cell carcinoma (RCC) is a rare cancer that can be associated with inherited diseases including tuberous sclerosis complex (TSC) caused by germline mutations in TSC1 or TSC2. Somatic mutations in TSC1 and TSC2 have also been reported in adult RCC, which predict response to mTOR inhibitors. Here, we pres...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26286
更新日期:2017-05-01 00:00:00
abstract::A 12-year-old boy in third remission of an acute lymphoblastic leukaemia developed infection of lung and paranasal sinuses with Aspergillus flavus in neutropenia. Because of the high risk of leukaemia-relapse bone marrow transplantation (BMT) from a matched unrelated donor was carried out despite invasive pulmonary as...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20302
更新日期:2005-06-15 00:00:00
abstract:INTRODUCTION:Our objectives were to design and validate methods to identify relapse and hematopoietic stem cell transplantation (HSCT) in children with acute lymphoblastic leukemia (ALL) using administrative data representing hospitalizations at US pediatric institutions. METHODS:We developed daily billing and ICD-9 c...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28315
更新日期:2020-05-11 00:00:00
abstract::The rejection rate in cord blood transplants for chronic Epstein-Bar virus-associated T or natural killer cell lymphoproliferative diseases using our standard reduced-intensity conditioning "LPAM140 regimen," which includes fludarabine, melphalan (LPAM), etoposide, and antithymocyte globulin, has been high. To ensure ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28536
更新日期:2020-09-01 00:00:00
abstract::A teenage boy presented with a CD30-positive anaplastic large cell lymphoma (ALCL) affecting his scapula and was successfully treated with chemotherapy. His clinical features and outcome were compared with other cases described in the literature. A further review of 11 ALCL cases with bony involvement treated in the U...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.20542
更新日期:2007-04-01 00:00:00
abstract::Children suffering from Acute Lymphoblastic Leukaemia (ALL) treated with asparaginase and corticosteroids are at risk of developing severe lipid abnormalities. The authors report the case of a 10-year-old male with extremely high plasma triglyceride concentrations (4,000 mg/dl) during the induction phase of ALL associ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20986
更新日期:2008-02-01 00:00:00
abstract::Ameloblastic fibro-odontosarcoma (AFOS) is an extremely rare malignant odontogenic tumor. Complete surgical excision is the treatment of choice. Deaths due to disease recurrence and/or progression are documented. Here, we report the case of a 15-year-old female with multiple recurrent AFOS. She responded to chemothera...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25627
更新日期:2015-11-01 00:00:00
abstract:PURPOSE:This pilot study was done to determine the feasibility and accuracy of University of Florida/National Cancer Institute (UF/NCI) phantoms and Monte Carlo (MC) retrospective dosimetry and had two aims: (1) to determine the anatomic accuracy of UF/NCI phantoms by comparing 3D organ doses in National Wilms Tumor St...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27395
更新日期:2018-12-01 00:00:00
abstract:OBJECTIVE:To investigate the effect of Helicobacter pylori eradication on platelet recovery in childhood chronic idiopathic thrombocytopenic purpura (ITP). PATIENTS AND METHODS:A multi-center randomized controlled trial was conducted. Patients aged 4-18 years, diagnosed with chronic ITP, defined by platelet count belo...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究,随机对照试验
doi:10.1002/pbc.21991
更新日期:2009-07-01 00:00:00
abstract:BACKGROUND:Sorafenib is an inhibitor of multiple kinases (e.g., VEGF receptors, PDGFR, FLT3, RET, BRAF, KIT) and is approved by FDA for treatment of two adult cancers. The activity of sorafenib was evaluated against the PPTP's in vitro and in vivo panels. PROCEDURES:Sorafenib was evaluated against the PPTP in vitro pa...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22712
更新日期:2010-12-01 00:00:00
abstract:BACKGROUND:Children with sickle cell disease (SCD) and abnormal transcranial Doppler (TCD) ultrasonography have a high risk of stroke, but this risk is greatly reduced when chronic transfusion therapy is administered. The change in TCD velocities during chronic transfusion therapy and rate and frequency of normalizatio...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22951
更新日期:2011-05-01 00:00:00
abstract::Progress has been made in resource-limited countries in treating acute lymphoblastic leukemia, but advances in solid malignancies have been slower. Multidisciplinary care coordination is challenging, assessing adherence to guidelines through quality improvement initiatives is essential. We characterized deviations fro...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26480
更新日期:2017-08-01 00:00:00
abstract::Medulloblastomas (MB) are classified in four subgroups: the well defined WNT and Sonic Hedgehog (SHH) subgroups, and the less defined groups 3 and 4. They occasionally occur in the context of a cancer predisposition syndrome. While germline APC mutations predispose to WNT MB, germline mutations in SUFU, PTCH1, and TP5...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24765
更新日期:2014-02-01 00:00:00
abstract:BACKGROUND:Little is known about the visual outcome of children affected by an optic pathway glioma (OPG). PROCEDURES:We evaluated the long-term visual outcome of 32 consecutive children affected by OPG without neurofibromatosis type-1 referred to the Pediatric Department of Padua University and managed according to s...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.22748
更新日期:2010-12-01 00:00:00
abstract::Granulocytic sarcoma (GS) is a localized tumor composed of immature myeloid cells. This extramedullary tumor can present before, concurrent with or after the diagnosis of acute myeloid leukemia. GS is extremely uncommon in acute promyelocytic leukemia (APL). As a proportion of patients never develop systemic disease, ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21190
更新日期:2008-03-01 00:00:00
abstract:BACKGROUND:Malignant peripheral nerve sheath tumor (MPNST) is an aggressive form of soft-tissue sarcoma (STS) in children. Despite intensive therapy, relatively few children with metastatic and unresectable disease survive beyond three years. RAS pathway activation is common in MPNST, suggesting MEK pathway inhibition ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28639
更新日期:2020-12-01 00:00:00
abstract:BACKGROUND:The identification of hemophagocytosis (HPC) in tissue or bone marrow (BM) represents only one of 5/8 criteria needed for the diagnosis of hemophagocytic lymphohistiocytosis (HLH). Yet, confirmation of HPC in bone marrow aspirates (BMA) is often relied upon to make therapeutic decisions. There is no standard...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21564
更新日期:2008-09-01 00:00:00
abstract:BACKGROUND:Although respiratory syncytial virus (RSV) is the leading cause of pediatric lower respiratory tract infections, the burden of RSV in children with sickle cell disease (SCD) is unknown. METHODS:We conducted a retrospective, nested, case-control study of children with SCD <18 years who had respiratory viral ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28759
更新日期:2021-01-01 00:00:00
abstract::Social media are powerful means of communication that can also have an important role in the healthcare sector. They are sometimes seen with diffidence in the healthcare setting, partly because they risk blurring professional boundaries. This issue is particularly relevant to relations between caregivers and adolescen...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26958
更新日期:2018-05-01 00:00:00
abstract::Global variations in the incidence of pediatric cancers have been described; however, the causes of such differences are not known. We investigated the relationship between the incidence of embryonal tumors and human development index on a global scale. Increasing incidence of neuroblastoma correlates significantly wi...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26244
更新日期:2017-02-01 00:00:00
abstract:BACKGROUND:The prevalence of nausea and vomiting after receipt of intrathecal methotrexate (IT-MTX) in pediatric oncology patients is unknown. METHODS:Patients (4-18 years) about to receive IT-MTX were eligible to participate in this prospective, observational study. Patients received antiemetics as prescribed by thei...
journal_title:Pediatric blood & cancer
pub_type: 评论,杂志文章
doi:10.1002/pbc.26603
更新日期:2017-10-01 00:00:00
abstract:BACKGROUND:The hallmark of sickle cell disease (SCD) is recurrent, painful vaso-occlusive episodes (VOC) and is the most common reason for hospitalization in SCD patients. Narcotics, particularly morphine, along with fluid hydration are standard treatments for painful episodes but have been associated with the developm...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20403
更新日期:2005-10-15 00:00:00
abstract:BACKGROUND:Although hematopoietic stem cell transplantation (HSCT) is offered as a curative therapy for pediatric myelodysplastic syndrome (MDS), it may cause severe complications and mortality. Several reports have shown the efficacy of immunosuppressive therapy (IST) in adult patients with refractory anemia (RA), but...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.22121
更新日期:2009-12-01 00:00:00
abstract:BACKGROUND:We sought to assess patterns of failure in pediatric patients with intracranial germ cell tumors (GCT) treated with intensity-modulated radiation therapy with dose painting (DP-IMRT). PROCEDURE:Between July 2007 and October 2013, 11 patients with localized GCT-five germinomas and six nongerminoma GCT (NGGCT...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25867
更新日期:2016-04-01 00:00:00
abstract::During ALL chemotherapy, a 4-year-old patient presented with febrile neutropenia and abdominal pain. Ultrasound examinations were repeatedly normal. Computerized tomography on day 7 demonstrated appendicitis and multiple hepatic foci identified as mucormycosis (Absidia corymbifera). Successful outcome was achieved by ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21918
更新日期:2009-07-01 00:00:00
abstract::Six patients with high-risk neuroblastoma underwent the second stem cell collection round with G-CSF (5 μg/kg/day) + plerixafor (0.24 mg/kg/day) because the amount of CD34(+) cells collected during the first collection round with G-CSF alone was insufficient. The number of CD34(+) cells collected in the second collect...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24506
更新日期:2013-08-01 00:00:00
abstract:BACKGROUND:Ewing sarcoma (ES) is a malignant tumor of bone and soft tissue of children and young adults. Patients with ES are treated with intensive chemotherapy regimens. We describe predictors of acute chemotherapy-associated toxicity in this population. PROCEDURE:In this retrospective cohort study, records of ES pa...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24031
更新日期:2012-10-01 00:00:00