Cediranib phase-II study in children with metastatic alveolar soft-part sarcoma (ASPS).

Abstract:

BACKGROUND:Alveolar soft-part sarcoma (ASPS), a rare vascular sarcoma with a clinically indolent course, frequently presents with metastases. Vascular endothelial growth factor (VEGF) is a promising therapeutic target. In a phase-II trial of the VEGF receptor inhibitor cediranib for adults with ASPS, the partial response (PR) rate (response evaluation criteria in solid tumors [RECIST] v1.0) was 35% (15/43; 95% confidence interval: 21-51%). We evaluated cediranib in the pediatric population. PROCEDURE:Patients <16 years old with metastatic, unresectable ASPS received cediranib at the pediatric maximum tolerated dose of 12 mg/m2 (≈70% of the fixed adult phase-II dose orally daily). Tumor response was assessed every two cycles (RECIST v1.0). A Simon two-stage optimal design (target response rate 35%, rule out 5%) was used. RESULTS:Seven patients (four females), with a median age of 13 years, (range 9-15), were enrolled on stage 1. The most frequent grade 2 or 3 adverse events were neutropenia, diarrhea, hypertension, fatigue, and proteinuria. The best response was stable disease (SD) (median cycle number = 34). Three patients were removed from the study treatment for disease progression (cycles 4, 5, and 36). Five of seven patients had SD for ≥14 months. Two patients with SD remain on study (34-57+ cycles). CONCLUSIONS:Cediranib did not reach the target response rate in this small pediatric cohort, in contrast to the adult 35% PR rate. The pediatric dosing was 30% lower compared to the adult dosing, which may have contributed to response differences. Prolonged SD was observed in five patients, but given the indolent nature of ASPS, SD cannot be clearly attributed to cediranib. Cediranib has an acceptable safety profile.

journal_name

Pediatr Blood Cancer

journal_title

Pediatric blood & cancer

authors

Cohen JW,Widemann BC,Derdak J,Dombi E,Goodwin A,Dompierre J,Onukwubiri U,Steinberg SM,O'Sullivan Coyne G,Kummar S,Chen AP,Glod J

doi

10.1002/pbc.27987

subject

Has Abstract

pub_date

2019-12-01 00:00:00

pages

e27987

issue

12

eissn

1545-5009

issn

1545-5017

journal_volume

66

pub_type

杂志文章,多中心研究
  • Influenza-associated morbidity in children with cancer.

    abstract:BACKGROUND:The clinical impact of influenza in children undergoing therapy for cancer is not well-described in the literature. PROCEDURE:Laboratory-documented influenza infection in pediatric oncology patients cared for in a single regional pediatric medical center between July 2000 and June 2005 was identified by rev...

    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章

    doi:10.1002/pbc.21472

    authors: Tasian SK,Park JR,Martin ET,Englund JA

    更新日期:2008-05-01 00:00:00

  • Insights on neoplastic stem cells from gel-based proteomics of childhood germ cell tumors.

    abstract:BACKGROUND:Childhood germ cell tumors (cGCTs), believed to arise from transformed primordial germ cells by an unknown mechanism, provide a unique model system for investigating cell signaling, pluripotency, and the microenvironment of neoplastic stem cells (NSCs) in vivo. This is the first report of proteomics of cGCTs...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.23282

    authors: Haskins WE,Eedala S,Jadhav YL,Labhan MS,Pericherla VC,Perlman EJ

    更新日期:2012-05-01 00:00:00

  • Metro-SMHOP 01: Metronomics combination with cyclophosphamide-etoposide and valproic acid for refractory and relapsing pediatric malignancies.

    abstract:BACKGROUND:In low- and middle-income countries, therapeutic options for advanced, refractory, or relapsing malignancies are limited due to local constraints such as cost of drugs, distance from oncology centers, and lack of availability of new anticancer drugs. Metronomics, which combines metronomic chemotherapy (MC) a...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.28508

    authors: El Kababri M,Benmiloud S,Cherkaoui S,El Houdzi J,Maani K,Ansari N,Khoubila N,Kili A,El Khorassani M,Madani A,Tazi MA,Ahid S,Hessissen L,Quessar A,Harif M,Khattab M,André N

    更新日期:2020-09-01 00:00:00

  • Age dependency of primary tumor sites and metastases in patients with Ewing sarcoma.

    abstract:BACKGROUND:The median age of patients with Ewing sarcoma (EwS) at diagnosis is around 14-15 years. Older age is associated with a worse outcome. The correlation of age at diagnosis on sites of disease has not been fully described. OBJECTIVE:The goal of this study was to evaluate the differences in sites of primary tum...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.27251

    authors: Worch J,Ranft A,DuBois SG,Paulussen M,Juergens H,Dirksen U

    更新日期:2018-09-01 00:00:00

  • Hydroxyurea use among children with sickle cell anemia.

    abstract::This study describes hydroxyurea use among children ages 1 to 17 with sickle cell anemia (SCA) enrolled in at least one year of Medicaid in six states from 2005 to 2012. Administrative claims were used to summarize the number of days' supply of hydroxyurea dispensed by state and year. A total of 7963 children with SCA...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.27721

    authors: Reeves SL,Jary HK,Gondhi JP,Raphael JL,Lisabeth LD,Dombkowski KJ

    更新日期:2019-06-01 00:00:00

  • A teenage boy with late onset hemophagocytic lymphohistiocytosis with predominant neurologic disease and perforin deficiency.

    abstract::Familial hemophagocytic lymphohistiocytosis (FHLH) is an autosomal recessive disorder of cytotoxic cell function that results in abnormal proliferation of benign lymphocytes and histiocytes in response to infectious stimuli. FHLH generally occurs in very young children, and typically presents with fever, cytopenias, c...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.21438

    authors: Beaty AD,Weller C,Levy B,Vogler C,Ferguson WS,Bicknese A,Knutsen AP

    更新日期:2008-05-01 00:00:00

  • Neuropsychological sequelae in patients with neurodegenerative Langerhans cell histiocytosis.

    abstract:BACKGROUND:Patients with Langerhans cell histiocytosis (LCH) may develop neurodegeneration and significant CNS sequelae, affecting a significant proportion of the patients. We here aimed to investigate the neuropsychological consequences in more detail. METHODS:Using an extensive neuropsychological test battery, we ev...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.21656

    authors: Van't Hooft I,Gavhed D,Laurencikas E,Henter JI

    更新日期:2008-11-01 00:00:00

  • Paediatric malignant tumours of the gastrointestinal tract in the West Midlands, UK, 1957-2000: a large population based survey.

    abstract:BACKGROUND:To investigate the incidence, clinical characteristics and survival of malignant tumours of the gastrointestinal tract in children in the West Midlands in Britain over a 44-year span time, to identify any change over this period and to compare the data with the world literature. PROCEDURE:Retrospective popu...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.20086

    authors: Hameed R,Parkes S,Davies P,Morland BJ

    更新日期:2004-09-01 00:00:00

  • Adolescent and young adult oncology: transition of care.

    abstract::The fundamental purposes underlying formal health care transition from the pediatric to adult setting for young adult survivors of childhood cancer are to facilitate the continuous, medically and developmentally appropriate implementation of risk-based guidelines for the monitoring and management of late effects of ch...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,评审

    doi:10.1002/pbc.21455

    authors: Freyer DR,Brugieres L

    更新日期:2008-05-01 00:00:00

  • Impact of Whole-Body Radiation Dose on Response and Toxicity in Patients With Neuroblastoma After Therapy With 131 I-Metaiodobenzylguanidine (MIBG).

    abstract:BACKGROUND:(131) I-metaiodobenzylguanidine ((131) I-MIBG) is a targeted radiopharmaceutical for patients with neuroblastoma. Despite its tumor-specific uptake, the treatment with (131) I-MIBG results in whole-body radiation exposure. Our aim was to correlate whole-body radiation dose (WBD) from (131) I-MIBG with tumor ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.25816

    authors: Trieu M,DuBois SG,Pon E,Nardo L,Hawkins RA,Marachelian A,Twist CJ,Park JR,Matthay KK

    更新日期:2016-03-01 00:00:00

  • Severe neurologic side effects in patients being treated for hemophagocytic lymphohistiocytosis.

    abstract:BACKGROUND:Hemophagocytic Lymphohistiocytosis (HLH) is characterized by uncontrolled inflammation that is generally fatal without immune modulating chemotherapy. At Texas Children's Hospital, we have observed significant central nervous system (CNS) toxicity in several patients treated for HLH according to the Histiocy...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.21838

    authors: Thompson PA,Allen CE,Horton T,Jones JY,Vinks AA,McClain KL

    更新日期:2009-05-01 00:00:00

  • Cooperating G6PD mutations associated with severe neonatal hyperbilirubinemia and cholestasis.

    abstract::We report a novel glucose-6-phosphate dehydrogenase (G6PD) mutation, which we propose to name G6PD Cincinnati (c.1037A > T, p.N346I), found in combination with G6PD Gastonia (c.637G > T, p.V213L) in an infant who presented with neonatal cholestasis. The G6PD Cincinnati mutation results in a non-conservative amino acid...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.22744

    authors: Mizukawa B,George A,Pushkaran S,Weckbach L,Kalinyak K,Heubi JE,Kalfa TA

    更新日期:2011-05-01 00:00:00

  • The strange case of the lost NRAS mutation in a child with juvenile myelomonocytic leukemia.

    abstract::Juvenile myelomonocytic leukemia (JMML) is a rare myelodysplastic/myeloproliferative disorder of early childhood characterized by mutations of the RAS-RAF-MAP kinase signaling pathway. We report the case of a child with a diagnosis of JMML carrying two mutations of NRAS gene (c.37G>C and c.38G>A) independently occurri...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.23401

    authors: De Filippi P,Zecca M,Novara F,Lisini D,Maserati E,Pasquali F,Rosti V,Carlo-Stella C,Zavras N,Cagioni C,Zuffardi O,Pagliara D,Danesino C,Locatelli F

    更新日期:2012-09-01 00:00:00

  • Carboplatin Hypersensitivity Reactions in Pediatric Low Grade Glioma Are Protocol Specific and Desensitization Shows Poor Efficacy.

    abstract:BACKGROUND:The use of carboplatin for the treatment of pediatric low grade gliomas (PLGG) is often limited by the development of carboplatin hypersensitivity. Reported rates of carboplatin hypersensitivity reactions vary between 6% and 32% in these patients. Here we report the frequency of carboplatin hypersensitivity ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.25686

    authors: Dodgshun AJ,Hansford JR,Cole T,Choo S,Sullivan MJ

    更新日期:2016-01-01 00:00:00

  • Plasma protein C is a useful clinical marker for hepatic veno-occlusive disease (VOD) in stem cell transplantation.

    abstract:BACKGROUND:Hepatic veno-occlusive disease (VOD) is one of the most serious complications in stem cell transplantation (SCT). Although plasma protein C activity decreases in VOD after SCT, the timeframe of plasma protein C activity decreases during SCT is not known. PROCEDURE:We examined levels of plasma protein C seri...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.22314

    authors: Iguchi A,Kobayashi R,Kaneda M,Kobayashi K

    更新日期:2010-03-01 00:00:00

  • CNS-directed therapy in young children with T-lineage acute lymphoblastic leukemia: High-dose methotrexate versus cranial irradiation.

    abstract:BACKGROUND:Prophylactic use of cranial radiation therapy (CRT) in young children with acute lymphoblastic leukemia (ALL) is associated with significant long-term morbidity. Therefore, current treatment protocols for pediatric B-precursor ALL have abandoned prophylactic CRT in favor of intrathecal chemotherapy, combined...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.10392

    authors: Nathan PC,Maze R,Spiegler B,Greenberg ML,Weitzman S,Hitzler JK

    更新日期:2004-01-01 00:00:00

  • A novel tissue-based ß-catenin gene and immunohistochemical analysis to exclude familial adenomatous polyposis among children with hepatoblastoma tumors.

    abstract:BACKGROUND:The Wnt/β-catenin pathway plays a central role in the pathogenesis of most hepatoblastomas (HBs), that is, up to 60-80% carry activating CTNNB1 mutations. HBs can however also be the first manifestation of familial adenomatous polyposis (FAP). As this is a severe disease, it is important for the patient and ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.26991

    authors: Dubbink HJ,Hollink IHIM,Avenca Valente C,Wang W,Liu P,Doukas M,van Noesel MM,Dinjens WNM,Wagner A,Smits R

    更新日期:2018-06-01 00:00:00

  • Female reproductive function after treatment of childhood acute lymphoblastic leukemia.

    abstract:BACKGROUND:The aim was to evaluate self-reported reproductive characteristics and markers of ovarian function in a nationwide cohort of female survivors of childhood acute lymphoblastic leukemia (ALL), because prior investigations have produced conflicting data. PROCEDURE:Self-reported reproductive characteristics wer...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.28894

    authors: Roshandel R,van Dijk M,Overbeek A,Kaspers G,Lambalk C,Beerendonk C,Bresters D,van der Heiden-van der Loo M,van den Heuvel-Eibrink M,Kremer L,Loonen J,van der Pal H,Ronckers C,Tissing W,Versluys B,van Leeuwen F,van den Ber

    更新日期:2021-01-18 00:00:00

  • A novel mutation of ABCG5 gene in a Turkish boy with phytosterolemia presenting with macrotrombocytopenia and stomatocytosis.

    abstract::Phytosterolemia is a rare autosomal recessive sterol storage disease caused by mutations in ABCG5 and ABCG8 genes. A 9-year-old Turkish boy who was presented with exclusively hematologic abnormalities had elevated plant sterol levels. Sequencing of ABCG5 and ABCG8 genes revealed a novel homozygous IVS10-1 G>T mutation...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.24934

    authors: Kaya Z,Niu DM,Yorulmaz A,Tekin A,Gürsel T

    更新日期:2014-08-01 00:00:00

  • Fournier's gangrene and scrotal ulcerations during all-trans-retinoic acid therapy for acute promyelocytic leukemia.

    abstract::Scrotal ulcers are a rare manifestation in patients with acute promyelocytic leukemia. Fournier's gangrene (FG) is even rarer. We describe three adolescents and young adults who developed scrotal ulcerations during induction with all-trans-retinoic acid. One patient developed FG. These lesions are predominantly seen i...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.21549

    authors: Naithani R,Kumar R,Mahapatra M

    更新日期:2008-08-01 00:00:00

  • A new clinical score for disease activity in Langerhans cell histiocytosis.

    abstract:OBJECTIVE:To develop an objective tool for assessing disease activity in patients with Langerhans cell histiocytosis (LCH). METHOD:Scoring system was developed and applied to a database containing information on 612 patients. RESULTS:At diagnosis, the score distribution was highly asymmetrical: the score was between ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.20160

    authors: Donadieu J,Piguet C,Bernard F,Barkaoui M,Ouache M,Bertrand Y,Ibrahim H,Emile JF,Hermine O,Tazi A,Genereau T,Thomas C

    更新日期:2004-12-01 00:00:00

  • The impact of illness perception and socio-clinico-demographic factors on perceived quality of life in children and adolescents with thalassemia intermedia.

    abstract:BACKGROUND/OBJECTIVES:β-Thalassemia intermedia (β-TI) accounts for up to one-fourth of β-thalassemia patients. Evaluating and improving quality of life (QOL) should be a goal in β-TI follow-up and management strategies. Patients' perceptions of their illness and its treatment may impact their QOL. This study aimed to e...

    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章

    doi:10.1002/pbc.27735

    authors: Atwa ZT,Wahed WYA

    更新日期:2019-07-01 00:00:00

  • Transcranial Doppler in hemoglobin SC disease.

    abstract:BACKGROUND:Stroke is a severe clinical disorder in sickle cell disease (SCD), and few studies have evaluated transcranial Doppler (TCD) flow velocities in hemoglobin SC disease (HbSC). The guidelines for stroke risk are based on evaluations in sickle cell anemia (SCA) or HbS/β thalassemia. PROCEDURE:In this study, we ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.26342

    authors: Vieira C,de Oliveira CN,de Figueiredo LA,Santiago RP,Adanho CS,Santana SS,Burak CL,Lyra IM,Goncalves MS

    更新日期:2017-05-01 00:00:00

  • Treatment of children with B-cell non-Hodgkin lymphoma in a low-income country.

    abstract:BACKGROUND:An adapted LMB 96 derived protocol for B-cell non-Hodgkin lymphoma (NHL) was implemented at the pediatric oncology unit of the Children Welfare Teaching Hospital in Baghdad (Iraq) from 2000 to present. The purpose was to evaluate the feasibility and efficacy of this intensive therapeutic regimen in a limited...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.22905

    authors: Moleti ML,Al-Hadad SA,Al-Jadiry MF,Al-Darraji AF,Al-Saeed RM,De Vellis A,Piciocchi A,Uccini S,Foà R,Testi AM

    更新日期:2011-04-01 00:00:00

  • One-year results from a prospective randomized trial comparing phlebotomy with deferasirox for the treatment of iron overload in pediatric patients with thalassemia major following curative stem cell transplantation.

    abstract:BACKGROUND:Iron overload is well documented in patients with β-thalassemia major, and patients who have undergone hematopoietic stem cell transplantation (HSCT) remain at risk as a result of pre- and immediate post-HSCT transfusions. PROCEDURE:This is a prospective, randomized, 1-year clinical trial that compares the ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,随机对照试验

    doi:10.1002/pbc.26213

    authors: Inati A,Kahale M,Sbeiti N,Cappellini MD,Taher AT,Koussa S,Nasr TA,Musallam KM,Abbas HA,Porter JB

    更新日期:2017-01-01 00:00:00

  • Treatment of pharyngeal non-Hodgkin lymphoma in a patient with Wiskott-Aldrich syndrome.

    abstract::Wiskott-Aldrich syndrome (WAS) is characterized by primary immunodeficiency, thrombocytopenia and eczema. Patients with WAS have an increased risk to develop tumors. Non-Hodgkin lymphoma (NHL) represents the most common malignancy occurring in WAS-affected patients, diffuse-large-B-cell lymphoma is the most frequently...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.23393

    authors: Coccia P,Mastrangelo S,Ruggiero A,Scalzone M,Rosolen A,Maurizi P,Riccardi R

    更新日期:2012-08-01 00:00:00

  • Blood transfusion therapy is feasible in a clinical trial setting in children with sickle cell disease and silent cerebral infarcts.

    abstract:BACKGROUND:Silent cerebral infarcts are both morbid and progressive in children with sickle cell disease (SCD). While blood transfusion therapy is effective primary and secondary stroke prevention, the efficacy and acceptance of blood transfusion therapy for children with silent cerebral infarcts is unknown. The overal...

    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章

    doi:10.1002/pbc.21338

    authors: King AA,Noetzel M,White DA,McKinstry RC,Debaun MR

    更新日期:2008-03-01 00:00:00

  • Detectable minimal residual disease before allogeneic hematopoietic stem cell transplantation predicts extremely poor prognosis in children with acute lymphoblastic leukemia.

    abstract:BACKGROUND:The level of minimal residual disease (MRD) prior to allogeneic hematopoietic stem cell transplantation (HSCT) has been shown to be an independent prognostic factor for outcome of pediatric patients with high-risk acute lymphoblastic leukemia (ALL). Retrospective studies which used (semi-) quantitation of cl...

    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章

    doi:10.1002/pbc.20794

    authors: Sramkova L,Muzikova K,Fronkova E,Krejci O,Sedlacek P,Formankova R,Mejstrikova E,Stary J,Trka J

    更新日期:2007-01-01 00:00:00

  • Successful treatment of a patient with mixed warm and cold antibody mediated Evans syndrome and glucose intolerance.

    abstract::Thrombocytopenia and autoimmune hemolytic anemia (Evans syndrome) with the presence of both warm and cold autoantibodies (mixed type) are rare in the pediatric age group. This condition may be associated with other autoimmune disorders and is notoriously difficult to treat. This case describes an adolescent male who p...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.20312

    authors: Jubinsky PT,Rashid N

    更新日期:2005-09-01 00:00:00

  • A prospective study of admissions for febrile neutropenia in secondary paediatric units in South East England.

    abstract:BACKGROUND:The Paediatric Oncology Centres (POCs) treating childhood cancer in South East England produce unified supportive care guidelines for use in the secondary pediatric (shared care) units. This study evaluated the adherence to current guidelines for febrile neutropenia (FN) and documented outcome in terms of ba...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.21041

    authors: Duncan C,Chisholm JC,Freeman S,Riley U,Sharland M,Pritchard-Jones K

    更新日期:2007-10-15 00:00:00