Improving outcomes for high-risk ALL: translating new discoveries into clinical care.

Abstract:

:High-risk (HR) acute lymphoblastic leukemia (ALL) remains one of the greatest challenges in pediatric oncology. Relapsed ALL is a leading cause of death in young people, and further improvements in outcome will required the development of therapeutic approaches directed against rational therapeutic targets, as escalation of the intensity of existing therapies is limited by toxicity. This review summarizes advances in the biology and treatment of HR and relapsed ALL presented at a symposium at the 2010 American Society for Pediatric Hematology and Oncology Annual Meeting. Analysis of large patient cohorts has identified several factors associated with HR of relapse including older age, T-lineage disease, and persisting minimal residual disease (MRD) early in therapy. As the results of salvage therapy remain poor, new treatment approaches are needed. BCR-ABL1-positive (Ph+) ALL has historically had a very poor outcome, but recent studies have demonstrated the impressive improvements in treatment outcome with the use of tyrosine kinase inhibitors (TKIs). High-resolution genomic profiling of genetic alterations and gene expression has revolutionized our understanding of the genetic basis of ALL, and has identified several alterations associated with poor outcome, including mutations of the lymphoid transcription factor gene IKZF1 (IKAROS), activating mutations of Janus kinases, and rearrangement of the lymphoid cytokine receptor gene CRLF2. These data indicated that the genetic basis of HR-ALL is multifactorial, and have also provided a new potential therapeutic option directed at JAK inhibition.

journal_name

Pediatr Blood Cancer

journal_title

Pediatric blood & cancer

authors

Hunger SP,Raetz EA,Loh ML,Mullighan CG

doi

10.1002/pbc.22996

subject

Has Abstract

pub_date

2011-06-01 00:00:00

pages

984-93

issue

6

eissn

1545-5009

issn

1545-5017

journal_volume

56

pub_type

杂志文章,评审
  • A man-made disease: Fetal neonatal alloimmune thrombocytopenia due to incompatibility between oocyte donor and gestational mother.

    abstract::The incompatibility causing fetal and neonatal alloimmune thrombocytopenia (FNAIT) results from a fetus inheriting a paternal human platelet antigen (HPA), which is different from the maternal HPA. We present a unique case of FNAIT in a pregnancy involving an oocyte recipient mother with Turner syndrome. This is the f...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.26447

    authors: Barg AA,Ifrah AD,Strauss T,Simchen MJ,Orvieto R,Rosenberg N,Kenet G

    更新日期:2017-08-01 00:00:00

  • Could we use parent report as a valid proxy of child report on anxiety, depression, and distress? A systematic investigation of father-mother-child triads in children successfully treated for leukemia.

    abstract:BACKGROUND:Systematic assessment of emotional distress is recommended in after care. Yet, it is unclear if parent report may be used as a proxy of child report. The aim of this study was to assess agreements and differences and explore possible moderators of disagreement between child and parent ratings. METHODS:Sixty...

    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章

    doi:10.1002/pbc.26840

    authors: Abate C,Lippé S,Bertout L,Drouin S,Krajinovic M,Rondeau É,Sinnett D,Laverdière C,Sultan S

    更新日期:2018-02-01 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

    doi:10.1002/pbc.26076

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

    更新日期:2016-11-01 00:00:00

  • Increased prevalence of risk factors for cardiovascular disease in long-term survivors of acute lymphoblastic leukemia and Wilms tumor treated with radiotherapy.

    abstract:BACKGROUND:Only a few studies have assessed cardiovascular risk factors (CRFs) in childhood cancer survivors. We determined the prevalence of CRFs in long-term survivors of acute lymphoblastic leukemia (ALL) and Wilms tumor. PROCEDURE:Adult survivors of ALL and Wilms tumor treated with radiotherapy and chemotherapy (R...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.22518

    authors: Geenen MM,Bakker PJ,Kremer LC,Kastelein JJ,van Leeuwen FE

    更新日期:2010-10-01 00:00:00

  • Wilms tumor, AML and medulloblastoma in a child with cancer prone syndrome of total premature chromatid separation and Fanconi anemia.

    abstract::Wilms tumor (WT) is the most common primary renal tumor in childhood. The occurrence of WT in patients with growth retardation, mental retardation and central nervous system abnormalities in association with premature chromatid separation (PCS) and mosaic variegated aneuploidy has been previously described in only 10 ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.21966

    authors: Sari N,Akyuz C,Aktas D,Gumruk F,Orhan D,Alikasifoglu M,Aydin B,Alanay Y,Buyukpamukcu M

    更新日期:2009-08-01 00:00:00

  • Comparison of PET-CT and conventional imaging in staging pediatric rhabdomyosarcoma.

    abstract:BACKGROUND:Over the past decade, PET-CT has been used to assess rhabdomyosarcoma (RMS) in children. However, the role of PET-CT in staging RMS is unknown. PROCEDURE:Thirty subjects with RMS, median age 7.3 years, underwent PET-CT before therapy. PET-CTs and conventional imaging (CI) were independently reviewed by two ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.24430

    authors: Federico SM,Spunt SL,Krasin MJ,Billup CA,Wu J,Shulkin B,Mandell G,McCarville MB

    更新日期:2013-07-01 00:00:00

  • Thymic carcinoma in children.

    abstract::Thymic epithelial neoplasms consist of thymomas, thymic carcinoids, and thymic carcinomas. Carcinomas are malignant tumors of the thymus characterized by obvious cytological anaplasia. They constitute only 4%-14% of thymic epithelial neoplams. Thymic carcinoma rarely occurs in children. Research in the English literat...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,评审

    doi:10.1002/pbc.20468

    authors: Yaris N,Nas Y,Cobanoglu U,Yavuz MN

    更新日期:2006-08-01 00:00:00

  • Phase 1 study of pomalidomide in children with recurrent, refractory, and progressive central nervous system tumors: A Pediatric Brain Tumor Consortium trial.

    abstract:BACKGROUND:Central nervous system (CNS) malignancies are the most common solid tumors among children, and novel therapies are needed to help improve survival. Pomalidomide is an immunomodulatory agent that displays antiangiogenic and cytotoxic activity, making it an appropriate candidate to explore in pediatric CNS tum...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.28756

    authors: Fangusaro J,Mitchell DA,Kocak M,Robinson GW,Baxter PA,Hwang EI,Huang J,Onar-Thomas A,Dunkel IJ,Fouladi M,Warren KE

    更新日期:2021-02-01 00:00:00

  • Clinical features and outcomes in patients with secondary Ewing sarcoma.

    abstract:BACKGROUND:Ewing sarcoma (EWS) is rarely diagnosed as a second malignancy. We sought to describe a cohort of patients with secondary EWS and investigate if patient characteristics and survival differ between patients with secondary and primary EWS. PROCEDURE:Patients with EWS or peripheral primitive neuroectodermal tu...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.24251

    authors: Applebaum MA,Goldsby R,Neuhaus J,DuBois SG

    更新日期:2013-04-01 00:00:00

  • Models of care for survivors of childhood cancer.

    abstract::With improvements in therapy for childhood cancer, the expectation that most childhood cancer patients will survive and enter adulthood is a reality. There is clear evidence that survivors are at risk for adverse health-related long-term sequelae associated with their cancer and its treatment, requiring appropriate he...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.20611

    authors: Friedman DL,Freyer DR,Levitt GA

    更新日期:2006-02-01 00:00:00

  • Oral health and dental anomalies in patients treated for leukemia in childhood and adolescence.

    abstract:BACKGROUND:This study was conducted to evaluate oral health and dental anomalies in children treated for acute lymphoblastic leukemia (ALL) and to compare results with those of a group of healthy children matched for sex and age. PROCEDURE:Fifty-six children treated for ALL and 56 healthy controls were examined for de...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.22108

    authors: Maciel JC,de Castro CG Jr,Brunetto AL,Di Leone LP,da Silveira HE

    更新日期:2009-09-01 00:00:00

  • Late effects of total body irradiation and hematopoietic stem cell transplant in children under 3 years of age.

    abstract:BACKGROUND:Total body irradiation (TBI) is an important component of hematopoietic stem cell transplant (SCT) for pediatric malignancies. With increasing survival rates, late effects of SCT become more important. Younger children may be at particular risk of late effects of radiation and SCT. METHODS:We retrospectivel...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.24252

    authors: Mulcahy Levy JM,Tello T,Giller R,Wilkening G,Quinones R,Keating AK,Liu AK

    更新日期:2013-04-01 00:00:00

  • Recognition of Li Fraumeni syndrome at diagnosis of a locally advanced extremity rhabdomyosarcoma.

    abstract::A contemporaneous presentation of a second breast cancer in a mother and an extremity rhabdomyosarcoma (RMS) in her daughter led to the diagnosis of the Li Fraumeni syndrome (LFS). Although the association between LFS and RMS in young patients is well recognised 1 there are no guidelines as to how this knowledge shoul...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.20795

    authors: Trahair T,Andrews L,Cohn RJ

    更新日期:2007-03-01 00:00:00

  • Ifosfamide and vinorelbine is an effective reinduction regimen in children with refractory/relapsed Hodgkin lymphoma, AHOD00P1: a children's oncology group report.

    abstract:BACKGROUND:We assessed the safety and efficacy of ifosfamide and vinorelbine (IV) as a less toxic and effective reinduction regimen for pediatric patients with relapsed or refractory Hodgkin Lymphoma. PROCEDURE:This multi-center Children's Oncology Group phase II pilot study enrolled patients <30 years of age with bio...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,多中心研究

    doi:10.1002/pbc.25205

    authors: Trippett TM,Schwartz CL,Guillerman RP,Gamis AS,Gardner S,Hogan S,London WB,Chen L,de Alarcon P

    更新日期:2015-01-01 00:00:00

  • Prolonged first-line PEG-asparaginase treatment in pediatric acute lymphoblastic leukemia in the NOPHO ALL2008 protocol-Pharmacokinetics and antibody formation.

    abstract:BACKGROUND:As pegylated asparaginase is becoming the preferred first-line asparaginase preparation in the chemotherapy regimens of childhood acute lymphoblastic leukemia (ALL), there is a need to evaluate this treatment. METHODS:The aim of this study was to evaluate the pharmacokinetics of prolonged upfront biweekly P...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,随机对照试验

    doi:10.1002/pbc.26686

    authors: Tram Henriksen L,Gottschalk Højfeldt S,Schmiegelow K,Frandsen TL,Skov Wehner P,Schrøder H,Klug Albertsen B,Nordic Society of Pediatric Hematology and Oncology, NOPHO Group.

    更新日期:2017-12-01 00:00:00

  • The importance of enteral nutrition to prevent or treat undernutrition in children undergoing treatment for cancer.

    abstract::Nutrition therapy is a therapeutic approach to treating medical conditions and symptoms via diet, which can be done by oral, enteral or parenteral routes. It is desirable to include nutritional interventions as a standard of care in pediatric cancer units (PCUs) at all levels of care. The interventions are dependent o...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,评审

    doi:10.1002/pbc.28378

    authors: Trehan A,Viani K,da Cruz LB,Sagastizado SZ,Ladas EJ

    更新日期:2020-06-01 00:00:00

  • Neuropsychological outcomes of a randomized trial of prednisone versus dexamethasone in acute lymphoblastic leukemia: findings from Dana-Farber Cancer Institute All Consortium Protocol 00-01.

    abstract:BACKGROUND:Dexamethasone is more efficacious than prednisone in the treatment of acute lymphoblastic leukemia (ALL), but has also been associated with greater toxicity. We compared neuropsychological outcomes for patients treated on DFCI ALL Consortium Protocol 00-01, which included a randomized comparison of the two s...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,随机对照试验

    doi:10.1002/pbc.24666

    authors: Waber DP,McCabe M,Sebree M,Forbes PW,Adams H,Alyman C,Sands SA,Robaey P,Romero I,Routhier MÈ,Girard JM,Sallan SE,Silverman LB

    更新日期:2013-11-01 00:00:00

  • Prognostic impact of diagnostic and treatment delays in children with osteosarcoma.

    abstract:BACKGROUND:The aim of this study is to evaluate the relationship between the latency to diagnosis (LD) and the time to completion of chemotherapy (TCC) with clinical outcomes in children with osteosarcoma. METHODS:We performed a retrospective analysis of all patients who received treatment for osteosarcoma in two tert...

    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章,多中心研究

    doi:10.1002/pbc.28180

    authors: Vasquez L,Silva J,Chavez S,Zapata A,Diaz R,Tarrillo F,Maza I,Sialer L,García J

    更新日期:2020-04-01 00:00:00

  • Pharmacokinetics and pharmacodynamics of intravenous epoetin alfa in children with cancer.

    abstract:BACKGROUND:Epoetin alfa (EPO, PROCRIT) pharmacokinetics and pharmacodynamics were evaluated in children with malignant solid tumors receiving chemotherapy. PROCEDURE:Children initially received IV EPO 600 IU/kg (max dose 40,000 IU) or placebo once weekly for 16 weeks. Dose was increased to 900 IU/kg (max dose 60,000 I...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,随机对照试验

    doi:10.1002/pbc.20685

    authors: Freeman BB 3rd,Hinds P,Iacono LC,Razzouk BI,Burghen E,Stewart CF

    更新日期:2006-10-15 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

  • Amifostine for children with medulloblastoma treated with cisplatin-based chemotherapy.

    abstract::In adult patients, amifostine appears to ameliorate cisplatin-related nephrotoxicity and ototoxicity. We assessed the safety and efficacy of amifostine in 11 children with newly diagnosed medulloblastoma/primitive neuroectodermal tumor treated with radiotherapy and vincristine, lomustine, and cisplatin. Amifostine was...

    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章

    doi:10.1002/pbc.20132

    authors: Fisher MJ,Lange BJ,Needle MN,Janss AJ,Shu HK,Adamson PC,Phillips PC

    更新日期:2004-12-01 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

    doi:10.1002/pbc.22752

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

    更新日期:2010-12-01 00:00:00

  • Quality of life in young patients after bone tumor surgery around the knee joint and comparison with healthy controls.

    abstract:BACKGROUND:This study aimed to compare the health related quality of life (HRQoL) of children and adolescents after malignant bone tumor surgery of the leg with healthy controls. PROCEDURE:Patients between 8 and 25 years old were cross-sectional recruited. Patients under 16 years of age received the TNO (Netherlands O...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,多中心研究

    doi:10.1002/pbc.22439

    authors: Bekkering WP,Vliet Vlieland TP,Koopman HM,Schaap GR,Schreuder HW,Beishuizen A,Tissing WJ,Hoogerbrugge PM,Anninga JK,Taminiau AH

    更新日期:2010-05-01 00:00:00

  • Validation of an algorithmic nutritional approach in children undergoing chemotherapy for cancer.

    abstract:BACKGROUND:Undernutrition impacts clinical outcome adversely in children with cancer. This study aimed to validate a nutritional algorithm with specific application to the low- and middle-income country (LMIC) setting. PROCEDURE:Fifty children with a new diagnosis of cancer were enrolled in this randomized interventio...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,随机对照试验

    doi:10.1002/pbc.27980

    authors: Totadri S,Trehan A,Mahajan D,Viani K,Barr R,Ladas EJ

    更新日期:2019-12-01 00:00:00

  • Parental Grief Following the Death of a Child from Cancer: The Ongoing Odyssey.

    abstract:BACKGROUND:The death of a child is a devastating event that results in profound grief and significant psychosocial and physical morbidities in parents. The parental grief journey is a complex phenomenon necessitating the utilization of newer models of bereavement with a focus on relationships and exploration of parents...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.26046

    authors: Snaman JM,Kaye EC,Torres C,Gibson D,Baker JN

    更新日期:2016-09-01 00:00:00

  • Improving the safety of high-dose methotrexate for children with hematologic cancers in settings without access to MTX levels using extended hydration and additional leucovorin.

    abstract:BACKGROUND:A lack of access to methotrexate levels is common in low- and middle-income countries (LMIC), relevant for 80% of children with cancer worldwide. We evaluated whether high-dose methotrexate (HD-MTX) can be administered safely with extended hydration and leucovorin rescue, with monitoring of serum creatinine ...

    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章

    doi:10.1002/pbc.27241

    authors: Vaishnavi K,Bansal D,Trehan A,Jain R,Attri SV

    更新日期:2018-12-01 00:00:00

  • Successful antifungal combination therapy with voriconazole and caspofungin.

    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

    authors: Schuster F,Moelter C,Schmid I,Graubner UB,Kammer B,Belohradsky BH,Führer M

    更新日期:2005-06-15 00:00:00

  • A family with Hoyeraal-Hreidarsson syndrome and four variants in two genes of the telomerase core complex.

    abstract::We describe an African American family with Hoyeraal-Hreidarrson syndrome (HHS) in which 2 TERT mutations (causing P530L and A880T amino acid changes) and two in the DKC1 variants (G486R and A487A) were segregating. Both genes are associated with dyskeratosis congenita and HHS. It was important to determine the import...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.24389

    authors: Vogiatzi P,Perdigones N,Mason PJ,Wilson DB,Bessler M

    更新日期:2013-06-01 00:00:00

  • Primary epithelial lung malignancies in the pediatric population.

    abstract:BACKGROUND:Primary epithelial lung malignancies are rare in childhood and adolescence. We reviewed the Memorial Sloan-Kettering Cancer Center experience with these tumors to better understand their histology, time to diagnosis, treatment, and outcome. PROCEDURE:A retrospective review was performed on all patients 21 y...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.20279

    authors: Lal DR,Clark I,Shalkow J,Downey RJ,Shorter NA,Klimstra DS,La Quaglia MP

    更新日期:2005-10-15 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

    doi:10.1002/pbc.28614

    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