Treatment outcome and pattern of failure in hepatoblastoma treated with a consensus protocol in Hong Kong.


BACKGROUND AND AIM:We reviewed the results and pattern of failure of the consensus HB/HCC 1996 treatment protocol for pediatric hepatoblastoma (HB) in Hong Kong. The role of SIOPEL and Children's Hepatic tumors International Collaboration (CHIC) risk stratification was evaluated. METHODS:Patients enrolled on the protocol from 1996 to 2014 were included. PRETEXT staging, SIOPEL, and CHIC risk groups were retrospectively assigned. RESULTS:Sixty patients were enrolled with median age at diagnosis of 1.1 years and median follow-up time of 6.8 years. Alpha-fetoprotein (AFP) was raised (>100 ng/mL) in 58 (97%) patients. Five (8%) had metastases at presentation and 7 (12%) experienced tumor rupture prior to or during treatment. Twenty-nine patients (48%) received a first-line cisplatin, 5-fluorouracil, and vincristine regimen only while 23 (38%) also had alternative chemotherapeutic agents. Hepatic resection could be performed in 48 (80%) patients. Three (5%) patients underwent upfront liver transplantation. Five-year event-free survival and overall survival rates were 69.2% ± 6.1% and 77.6% ± 5.5% respectively. Among the 16 patients with relapse/progression, 9 had intrahepatic failure only, 5 had distant failure only, and 2 had combined local and distant failure. Predictors of inferior outcome included advanced Evans staging, disease involving both lobes, rupture, low AFP, and suboptimal response to first-line chemotherapy. Assigned in 44 patients, PRETEXT staging, SIOPEL, and CHIC risk groups significantly predicted EFS and OS. CONCLUSIONS:Although the consensus HB/HCC 1996 protocol led to cure in three-quarters of pediatric HB patients, an upfront risk stratification system is required to identify and improve the outcome of high-risk patients.


Pediatr Blood Cancer


Pediatric blood & cancer


Liu APY,Ip JJK,Leung AWK,Luk CW,Li CH,Ho KKH,Lo R,Chan EKW,Chan ACY,Chung PHY,Chiang AKS




Has Abstract


2019-01-01 00:00:00












  • Measuring the efficacy of a project for adolescents and young adults with cancer: A study from the Milan Youth Project.

    abstract:BACKGROUND:Various projects dedicated specifically to adolescents and young adults (AYA) with cancer have been developed in recent years. A critical aspect of such programs is the ability to demonstrate its value, and therefore how to measure desired outcomes. METHODS:A list of metrics to consider for demonstrating th...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Ferrari A,Silva M,Veneroni L,Magni C,Clerici CA,Meazza C,Terenziani M,Spreafico F,Chiaravalli S,Casanova M,Luksch R,Catania S,Schiavello E,Biassoni V,Podda M,Bergamaschi L,Puma N,Indini A,Proserpio T,Massimino M

    更新日期:2016-12-01 00:00:00

  • Salvage therapy for refractory hemophagocytic lymphohistiocytosis: A review of the published experience.

    abstract::Hemophagocytic lymphohistioytosis (HLH) is a severe, life-threatening hyperinflammatory disorder that requires prompt diagnosis and treatment. Approximately, 25-50% of patients with HLH fail to achieve remission with established regimens that include dexamethasone and etoposide, or methylprednisolone and antithymocyte...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,评审


    authors: Marsh RA,Jordan MB,Talano JA,Nichols KE,Kumar A,Naqvi A,Vaiselbuh SR,Histiocyte Society Salvage Therapy Working Group.

    更新日期:2017-04-01 00:00:00

  • Solitary plasmacytoma of the proximal tibia in an adolescent.

    abstract::This is the case report of a 14-year young female who was diagnosed with solitary bone plasmacytoma (SBP) of proximal tibia and was treated by local involved field radiotherapy. We present the clinical, radiological and pathological findings of the case and review of the available treatment options and prognosis of th...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Kumar P,Sharma SC,Saikia UN,Kumar N,Vyas S,Angurana SL

    更新日期:2011-01-01 00:00:00

  • Severe persistent bone marrow failure following therapy with 2-chlorodeoxyadenosine for relapsing juvenile xanthogranuloma of the brain.

    abstract::2-Chlorodeoxyadenosine (2-CdA) has been successfully used in children to treat refractory Langerhans cell histiocytosis and juvenile xanthogranuloma (JXG) as salvage therapy. Although 2-CdA is generally well-tolerated, with temporary myelosuppression as the primary dose-limiting toxicity, prolonged myelosuppressive, a...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Yamada K,Yasui M,Sawada A,Inoue M,Nakayama M,Kawa K

    更新日期:2012-02-01 00:00:00

  • Sequential administration of methotrexate and asparaginase in relapsed or refractory pediatric acute myeloid leukemia.

    abstract:BACKGROUND:The efficacy of combination chemotherapy with methotrexate (MTX) and asparaginase is not well known in relapsed and refractory acute leukemia after contemporary therapy. PROCEDURE:A retrospective study of pediatric patients with relapsed or refractory acute myeloid leukemia (AML) who received MTX and aspara...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Buaboonnam J,Cao X,Pauley JL,Pui CH,Ribeiro RC,Rubnitz JE,Inaba H

    更新日期:2013-07-01 00:00:00

  • Treatment of primary CNS lymphoma with high-dose methotrexate in immunocompetent pediatric patients.

    abstract::We report two cases of primary CNS lymphoma (PCNSL) treated with high-dose methotrexate. Though standard adult treatment of PCNSL incorporates whole-brain radiotherapy, the literature suggests it may be possible to delay or avoid radiotherapy and the associated increased risk of neurologic sequelae in pediatric patien...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Shah AC,Kelly DR,Nabors LB,Oakes WJ,Hilliard LM,Reddy AT

    更新日期:2010-12-01 00:00:00

  • Influence of pediatric cancer-related financial burden on parent distress and other stress-related symptoms.

    abstract:BACKGROUND:Pediatric cancer-induced financial burden is source of stress for parents, particularly mothers, single parents, and parents with lower incomes. This financial burden has been linked to poorer family quality of life (QOL) in terms of new onset material hardships, and could also affect individual QOL in terms...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Santacroce SJ,Kneipp SM

    更新日期:2020-03-01 00:00:00

  • Assessment of Financial Burden as a Standard of Care in Pediatric Oncology.

    abstract::Family financial hardship has emerged as a burden of pediatric cancer treatment with negative implications for family well-being. As part of an extensive project to create evidence-based standards for the psychosocial care of children with cancer, we performed a literature review of pediatric cancer-associated financi...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,评审


    authors: Pelletier W,Bona K

    更新日期:2015-12-01 00:00:00

  • Incidence of Langerhans cell histiocytosis in children: a population-based study.

    abstract:BACKGROUND:Langerhans cell histiocytosis is a rare disease of unknown etiology. We wanted to assess the population-based incidence of LCH in a well-defined cohort of children. METHODS:We identified all children <15-years old treated with LCH during the 10 years period 1992-2001 at the Department of Pediatrics, Karolin...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Stålemark H,Laurencikas E,Karis J,Gavhed D,Fadeel B,Henter JI

    更新日期:2008-07-01 00:00:00

  • Use of RNA interference to elucidate the effect of MYCN on cell cycle in neuroblastoma.

    abstract:BACKGROUND:MYCN amplification marks poor prognosis in neuroblastoma (NB) tumors. In evaluating the mechanisms by which retinoic acid (RA) or nerve growth factor (NGF) decrease cell number in MYCN amplified NB cells, we have identified a number of proteins whose expression either decreases (E2F, CDC2, CDK6, cyclin depen...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Woo CW,Tan F,Cassano H,Lee J,Lee KC,Thiele CJ

    更新日期:2008-02-01 00:00:00

  • Medulloblastoma in childhood: Impact of radiation technique upon the outcome of treatment.

    abstract:BACKGROUND:Medulloblastoma is an infratentorial primitive neuroectodermal tumour, diagnosed in paediatric population. The radiotherapy is an essential method of treatment for these tumours. The impact of technical quality of radiation therapy on survival was recently considered. In this study treatment-related variable...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Chojnacka M,Skowrońska-Gardas A

    更新日期:2004-02-01 00:00:00

  • Parental views on tissue banking in pediatric oncology patients.

    abstract:PURPOSE:Research using banked tissue is key to advancing risk-stratification and treatment of children with cancer. Knowledge of parental attitudes to ethical issues arising in tissue banking is very limited but essential in obtaining respectful consent. METHODS:One hundred parents of consecutively diagnosed children ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: McMurter B,Parker L,Fraser RB,Magee JF,Kozancyzn C,Fernandez CV

    更新日期:2011-12-15 00:00:00

  • A novel SAMD9 variant identified in patient with MIRAGE syndrome: Further defining syndromic phenotype and review of previous cases.

    abstract::We present here a case of MIRAGE syndrome due to novel variant (c.2318T>C) in the sterile α motif domain-containing protein 9 (SAMD9) gene. Previous reports have described the clinical phenotype, which includes myelodysplasia, recurrent infections, restriction of growth and development, adrenal insufficiency, genitour...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,评审


    authors: Perisa MP,Rose MJ,Varga E,Kamboj MK,Spencer JD,Bajwa RPS

    更新日期:2019-07-01 00:00:00

  • Risk factors for umbilical venous catheter-associated thrombosis in very low birth weight infants.

    abstract:BACKGROUND:Thrombosis in neonates is a rare but serious occurrence, usually associated with central catheterization. The objective of this study was to investigate the risk factors associated with catheter related thrombosis in very low birth weight (VLBW) infants. PROCEDURE:The present retrospective study was perform...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Narang S,Roy J,Stevens TP,Butler-O'Hara M,Mullen CA,D'Angio CT

    更新日期:2009-01-01 00:00:00

  • Circulating tumor DNA in neuroblastoma.

    abstract::As a sympathetic nervous system-derived tumor, aggressive neuroblastoma (NB) is currently attracting interest from researchers seeking diagnostic and prognostic markers via less invasive procedures. The analysis of circulating tumor DNA (ctDNA) in peripheral blood can provide genetic information from multiple tumor le...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,评审


    authors: Wei M,Ye M,Dong K,Dong R

    更新日期:2020-10-01 00:00:00

  • Initial testing (stage 1) of the multi-targeted kinase inhibitor sorafenib by the pediatric preclinical testing program.

    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


    authors: Keir ST,Maris JM,Lock R,Kolb EA,Gorlick R,Carol H,Morton CL,Reynolds CP,Kang MH,Watkins A,Houghton PJ,Smith MA

    更新日期:2010-12-01 00:00:00

  • Treatment of infantile choriocarcinoma of the liver.

    abstract::Primary choriocarcinoma of the liver is an extremely rare childhood malignancy frequently associated with clinical instability at initial presentation. It often mimics other benign and malignant childhood liver tumors. Prompt diagnosis and initiation of treatment are necessary to attain a successful outcome. We descri...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Yoon JM,Burns RC,Malogolowkin MH,Mascarenhas L

    更新日期:2007-07-01 00:00:00

  • Gene expression profile of idiopathic thrombocytopenic purpura (ITP).

    abstract::To search for novel mechanisms that contribute to the pathophysiology of idiopathic thrombocytopenic purpura (ITP), we determined the whole blood gene expression profile in five ITP patients and five control samples. Using DNA microarrays that contained 24,473 unique putative genes, we found 176 cDNAs that were strong...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Sood R,Wong W,Jeng M,Zehnder JL

    更新日期:2006-10-15 00:00:00

  • Primary Cutaneous Lymphomas in Children and Adolescents.

    abstract::Primary cutaneous lymphomas are rare in children and mostly represented by mycosis fungoides and CD30(+) lymphoproliferative disorders. Most pediatric cutaneous lymphomas have similar clinical/pathological features as their adult counterparts, particularly the T-cell subtypes. With regard to outcome, adult cutaneous m...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,评审


    authors: Ceppi F,Pope E,Ngan B,Abla O

    更新日期:2016-11-01 00:00:00

  • Comparison of gallium and PET scans at diagnosis and follow-up of pediatric patients with Hodgkin lymphoma.

    abstract:BACKGROUND:Positron emission tomography (PET) and gallium scans facilitate diagnosis and staging, evaluation of response to therapy, and monitoring for relapse in Hodgkin lymphoma (HL), but have not been compared in pediatric HL. PROCEDURE:We performed concurrent PET and gallium scans on 44 pediatric HL patients at di...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Hines-Thomas M,Kaste SC,Hudson MM,Howard SC,Liu WA,Wu J,Kun LE,Shulkin BL,Krasin MJ,Metzger ML

    更新日期:2008-08-01 00:00:00

  • High cumulative rate of secondary leukemia after continuous etoposide treatment for solid tumors in children and young adults.

    abstract:BACKGROUND:In a national pediatric case-control study, we observed a very high relative risk of leukemia in patients who had received continuous etoposide (CE) over 6 months or more, but we could not estimate the absolute risk. The purpose of the present study was to estimate this absolute risk after CE. PROCEDURES:We...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Le Deley MC,Vassal G,Taïbi A,Shamsaldin A,Leblanc T,Hartmann O

    更新日期:2005-07-01 00:00:00

  • Phase 1 trial, pharmacokinetics, and pharmacodynamics of dasatinib combined with crizotinib in children with recurrent or progressive high-grade and diffuse intrinsic pontine glioma.

    abstract:BACKGROUND:Progressive/recurrent high-grade and diffuse intrinsic pontine gliomas (DIPGs) are fatal. Treatments targeting molecular pathways critical for these cancers are needed. METHODS:We conducted a phase 1 study (rolling-six design) to establish the safety and maximum tolerated dose (MTD) of dasatinib, an oral pl...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Broniscer A,Jia S,Mandrell B,Hamideh D,Huang J,Onar-Thomas A,Gajjar A,Raimondi SC,Tatevossian RG,Stewart CF

    更新日期:2018-07-01 00:00:00

  • Feasibility of metronomic maintenance chemotherapy following high-dose chemotherapy for malignant central nervous system tumors.

    abstract:BACKGROUND:Children less than 5 years of age with malignant central nervous system (CNS) tumors, continue to have a high rate of morbidity and mortality following administration of conventional therapy. In an attempt to avoid the neurologic sequelae associated with craniospinal radiation, strategies such as high-dose c...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Choi LM,Rood B,Kamani N,La Fond D,Packer RJ,Santi MR,Macdonald TJ

    更新日期:2008-05-01 00:00:00

  • EBV lymphoproliferative disease of host origin after haploidentical stem cell transplantation.

    abstract::Post-transplant lymphoproliferative disease (PTLPD), due to the reactivation of Epstein-Barr virus (EBV), is a serious complication. The risk of the disorder increases with T-cell depletion methods, mismatched hematopoietic stem cell transplantation (HSCT), graft-versus-host disease (GVHD), and immunosuppression. In c...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Kasow KA,Leung W,Horwitz EM,Woodard P,Handgretinger R,Hale GA

    更新日期:2007-11-01 00:00:00

  • A proposal for an international retinoblastoma staging system.

    abstract:BACKGROUND:Although intra-retinal tumor has long been staged presurgically according to the Reese-Ellsworth (R-E) system, retinoblastoma differs from other pediatric neoplasms in never having had a widely accepted classification system that encompasses the entire spectrum of the disease. Comparisons among studies that ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,多中心研究


    authors: Chantada G,Doz F,Antoneli CB,Grundy R,Clare Stannard FF,Dunkel IJ,Grabowski E,Leal-Leal C,Rodríguez-Galindo C,Schvartzman E,Popovic MB,Kremens B,Meadows AT,Zucker JM

    更新日期:2006-11-01 00:00:00

  • Bone marrow aspiration technique may have an impact on therapy stratification in children with acute lymphoblastic leukaemia.

    abstract:BACKGROUND:Morphological evaluation of early response to chemotherapy and measurement of minimal residual disease by flow cytometry or PCR are being used for evaluation of prognosis and treatment stratification in children with acute lymphoblastic leukaemia (ALL). PROCEDURE:In a series of 14 consecutive bone marrow in...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Helgestad J,Rosthøj S,Johansen P,Varming K,Østergaard E

    更新日期:2011-08-01 00:00:00

  • Pheochromocytomas and paragangliomas in children: Data from the Italian Cooperative Study (TREP).

    abstract:BACKGROUND:Pheochromocytomas (PCs) are neuroendocrine tumors arising from the chromaffin cells of the adrenal gland, and paragangliomas (PGLs) are their extra-adrenal counterparts arising from ganglia along the sympathetic/parasympathetic chain. Surgery is the cornerstone of treatment. A sporatic or inherited germline ...

    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章,多中心研究


    authors: Virgone C,Andreetta M,Avanzini S,Chiaravalli S,De Pasquale D,Crocoli A,Inserra A,D'Angelo P,Alaggio R,Opocher G,Cecchetto G,Ferrari A,Bisogno G,Dall'Igna P

    更新日期:2020-08-01 00:00:00

  • Pilomyxoid astrocytoma treated successfully with vemurafenib.

    abstract::The BRAF V600E missense mutation is known to be present in a subset of central nervous system tumors. We report a patient with a BRAF V600E mutated pilomyxoid astrocytoma who failed multiple conventional chemotherapy regimens. Treatment with vemurafenib, a molecularly targeted therapy against the mutant BRAF V600E kin...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Skrypek M,Foreman N,Guillaume D,Moertel C

    更新日期:2014-11-01 00:00:00

  • Intravenous ferric carboxymaltose for iron deficiency anemia or iron deficiency without anemia after poor response to oral iron treatment: Benefits and risks in a cohort of 144 children and adolescents.

    abstract:OBJECTIVE:The objective of this single-center observational study was to determine the clinical and hematologic responses to intravenous ferric carboxymaltose (FCM) in a cohort of pediatric patients with poor response to oral iron therapy. The occurrence of adverse events was systematically recorded for up to 96 hours ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章


    authors: Ozsahin H,Schaeppi M,Bernimoulin M,Allard M,Guidard C,van den Ouweland F

    更新日期:2020-10-01 00:00:00

  • Validation of the school age self-administered pediatric bleeding questionnaire (Self-PBQ) in children aged 8-12 years.

    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


    authors: Bui J,Martyres D,James PD,Grabell J,Wu J,Steele M,Silva M,Rand ML,Blanchette VS,Barrowman N,Klaassen RJ

    更新日期:2019-06-01 00:00:00