Treatment of children with refractory anemia: the Japanese Childhood MDS Study Group trial (MDS99).

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 its safety and efficacy remains to be fully elucidated in childhood RA. PROCEDURE:Eleven children diagnosed with RA and enrolled on a prospective multicenter trial conducted by the Japanese Childhood MDS Study Group were eligible for analysis. If patients showed transfusion dependent or suffered from infection due to neutropenia, they received IST consisting of antithymocyte globulin (ATG), cyclosporine (CyA), and methylprednisolone (mPSL). RESULTS:Eight children received IST, 2 received only supportive therapy, and one underwent HSCT without IST. Five (63%) of eight children who received IST showed hematological response. Of note, one patient showed the disappearance of monosomy 7 after IST. Responders were significantly younger than non-responders (29 months vs. 140 months; P = 0.03). No severe adverse events related to IST were reported in this study. Of 6 children with chromosomal abnormalities who received IST, four showed hematological response. The probability of failure-free and overall survival at 5 years was 63 +/- 17% and 90 +/- 9% respectively. CONCLUSION:IST is likely to be a safe and effective modality for childhood RA.

journal_name

Pediatr Blood Cancer

journal_title

Pediatric blood & cancer

authors

Hasegawa D,Manabe A,Yagasaki H,Ohtsuka Y,Inoue M,Kikuchi A,Ohara A,Tsuchida M,Kojima S,Nakahata T,Japanese Childhood MDS Study Group.

doi

10.1002/pbc.22121

subject

Has Abstract

pub_date

2009-12-01 00:00:00

pages

1011-5

issue

6

eissn

1545-5009

issn

1545-5017

journal_volume

53

pub_type

临床试验,杂志文章,多中心研究
  • Stage 1 testing and pharmacodynamic evaluation of the HSP90 inhibitor alvespimycin (17-DMAG, KOS-1022) by the pediatric preclinical testing program.

    abstract:BACKGROUND:Alvespimycin (17-DMAG, KOS-1022), a potent small-molecule inhibitor of the protein chaperone Hsp90, is being developed as an anticancer agent because of the multiple Hsp90 client proteins involved in cancer cell growth and survival. PROCEDURES:Alvespimycin was tested against the in vitro panel of the Pediat...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.21508

    authors: Smith MA,Morton CL,Phelps DA,Kolb EA,Lock R,Carol H,Reynolds CP,Maris JM,Keir ST,Wu J,Houghton PJ

    更新日期:2008-07-01 00:00:00

  • TP53 codon 72 polymorphisms in favorable histology Wilms tumors.

    abstract::In Wilms tumor (WT), mutations in the gene encoding p53, TP53, are correlated with anaplasia; however TP53 variants have not been studied in favorable histology (FH) WTs. A single nucleotide polymorphism of TP53 encoding either arginine or proline at codon 72 is suggested to alter in vitro p53 behavior. Therefore, we ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.23315

    authors: Cost NG,Mitui M,Khokhar S,Wickiser JE,Baker LA,Rakheja D

    更新日期:2012-08-01 00:00:00

  • Risk prediction of fever in neutropenia in children with cancer: a step towards individually tailored supportive therapy?

    abstract:BACKGROUND:Fever in severe chemotherapy-induced neutropenia (FN) is the most frequent manifestation of a potentially lethal complication of current intensive chemotherapy regimens. This study aimed at establishing models predicting the risk of FN, and of FN with bacteremia, in pediatric cancer patients. METHODS:In a s...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.21726

    authors: Wicki S,Keisker A,Aebi C,Leibundgut K,Hirt A,Ammann RA

    更新日期:2008-12-01 00:00:00

  • A comparison of pediatric, adolescent, and adult testicular germ cell malignancy.

    abstract:BACKGROUND:Testicular germ cell tumors (T-GCTs) occur from infancy to adulthood, and are the most common solid tumor in adolescent and young adult males. Traditionally, pediatric T-GCTs were perceived as more indolent than adult T-GCTs. However, there are few studies comparing these groups and none that specifically ev...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.24773

    authors: Cost NG,Lubahn JD,Adibi M,Romman A,Wickiser JE,Raj GV,Sagalowsky AI,Margulis V

    更新日期:2014-03-01 00:00:00

  • Detection of cell-free RNA in children with neuroblastoma and comparison with that of whole blood cell RNA.

    abstract:BACKGROUND:Since there is no validated assay to monitor disease in children with neuroblastoma (NB), we tested whether NB specific cell-free RNA could be detected in their plasma samples. Moreover, with the aim of reducing patients' discomfort, we compared this assay to a recently standardized procedure that uses a lar...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.22498

    authors: Corrias MV,Pistorio A,Cangemi G,Tripodi G,Carlini B,Scaruffi P,Fardin P,Garaventa A,Pistoia V,Haupt R

    更新日期:2010-07-01 00:00:00

  • Severe H1N1-associated acute respiratory failure in immunocompromised children.

    abstract:BACKGROUND:Severe pandemic influenza A (H1N1) infection can lead to acute respiratory failure (ARF) with associated high mortality. Children with malignancy may be at higher risk of H1N1-associated ARF because of underlying primary disease or immunosuppression associated with chemotherapy. PROCEDURE:We describe the cl...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.22973

    authors: Elbahlawan L,Gaur AH,Furman W,Jeha S,Woods T,Norris A,Morrison RR

    更新日期:2011-10-01 00:00:00

  • Improved outcome in childhood acute myeloid leukemia in Singapore with the MRC AML 10 protocol.

    abstract:BACKGROUND:The introduction of the United Kingdom Medical Research Council's 10th AML trial (MRC AML 10) protocol incorporating high-dose anthracycline therapy has improved outcome of children with acute myeloid leukemia (AML). In this study, we review the results of childhood AML therapy in a Singapore university hosp...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.20834

    authors: Tan RM,Quah TC,Aung L,Liang S,Kirk RC,Yeoh AE

    更新日期:2007-03-01 00:00:00

  • Hematopoietic stem cell transplant referral patterns for children with sickle cell disease vary among pediatric hematologist/oncologists' practice focus: A Sickle Cell Transplant Advocacy and Research Alliance (STAR) study.

    abstract:BACKGROUND:Hematopoietic stem cell transplantation (HSCT) provides a curative therapy for children severely affected by sickle cell disease (SCD). Rejection-free survival after matched sibling donor (MSD) HSCT is very high, but adoption of HSCT as a curative SCD therapy has been slow. In this study, we assess providers...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.28861

    authors: Meier ER,Abraham AA,Ngwube A,Janson IA,Guilcher GMT,Horan J,Kasow KA

    更新日期:2021-03-01 00:00:00

  • 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

  • An overview of mongenic and syndromic obesities in humans.

    abstract::Obesity is increasing in prevalence in the United States with over 65% of adults considered overweight and 16% of children with BMI > 95 percentile. The heritability of obesity is estimated between 40% and 70%, but the genetics of obesity for most individuals are complex and involve the interaction of multiple genes a...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.23372

    authors: Chung WK

    更新日期:2012-01-01 00:00:00

  • Pediatric cancer research: Surviving COVID-19.

    abstract::A diverse panel of pediatric cancer advocates and experts, whose collective experience spans the continuum of international academic medicine, industry, government research, and cancer advocacy, recently discussed challenges for pediatric cancer research in the context of coronavirus disease 2019 (COVID-19). Specifica...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,评审

    doi:10.1002/pbc.28435

    authors: Auletta JJ,Adamson PC,Agin JE,Kearns P,Kennedy S,Kieran MW,Ludwinski DM,Knox LJ,McKay K,Rhiner P,Thiele CJ,Cripe TP

    更新日期:2020-09-01 00:00:00

  • Changes in hepatitis B antibody status after chemotherapy in children with acute lymphoblastic leukemia.

    abstract:BACKGROUND:Children with cancer may be at an increased risk of infection with hepatitis B virus (HBV) when levels of hepatitis B antibodies are reduced owing to chemotherapy-induced immunosuppression. This study evaluated the changes in HBV antibody status and HBV infections after chemotherapy in children with acute ly...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.27904

    authors: Choi YB,Lee NH,Yi ES,Kim YJ,Koo HH

    更新日期:2019-12-01 00:00:00

  • Feasibility and accuracy of UF/NCI phantoms and Monte Carlo retrospective dosimetry in children treated on National Wilms Tumor Study protocols.

    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

    authors: Kalapurakal JA,Gopalakrishnan M,Mille M,Helenowski I,Peterson S,Rigsby C,Laurie F,Jung JW,Fitzgerald TJ,Lee C

    更新日期:2018-12-01 00:00:00

  • Full-dose ifosfamide can be safely administered to outpatients.

    abstract::This report compares a traditional full-dose ifosfamide administration modality (24-hr hyperhydration and mesna infusion) with a simplified 9-hr hyperhydration and mesna infusion for use in outpatients. Acute ifosfamide toxicity was the same, suggesting that ifosfamide could be safely administered to outpatients, redu...

    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章

    doi:10.1002/pbc.20958

    authors: Meazza C,Bisogno G,Casanova M,Zanetti I,Carli M,Ferrari A

    更新日期:2008-02-01 00:00:00

  • Evaluation of radiation dose to cardiac and pulmonary tissue among patients with stage IV Wilms tumor and pulmonary metastases.

    abstract:BACKGROUND:Most patients with stage IV Wilms tumor (WT) and pulmonary metastases are treated with surgery, local radiotherapy (RT), and whole-lung irradiation (WLI). The Children's Oncology Group is studying whether WLI should only be given if metastatic lung lesions persist following induction chemotherapy. We hypothe...

    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章

    doi:10.1002/pbc.25007

    authors: Farooqi A,Siddiqi A,Khan MK,Esiashvili N

    更新日期:2014-08-01 00:00:00

  • Hepatoblastoma in a child with neurofibromatosis type I.

    abstract::A major hallmark of NF1 is the development of benign tumors, including peripheral neurofibromas, plexiform neurofibromas, gliomas of the optic tract, other low grade gliomas, and pheochromocytomas. Hepatoblastoma have not been previously reported in patients with neurofibromatosis type 1. We present a case of a 9-mont...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.20663

    authors: Uçar C,Calişkan U,Toy H,Günel E

    更新日期:2007-09-01 00:00:00

  • Modifying bone mineral density, physical function, and quality of life in children with acute lymphoblastic leukemia.

    abstract:BACKGROUND:The early effects of childhood acute lymphoblastic leukemia (ALL) include decreased physical function, bone mineral density (BMD/g/cm2 ), and health-related quality of life (HRQL). We assessed the capacity of a physical therapy and motivation-based intervention, beginning after diagnosis and continuing throu...

    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.26929

    authors: Cox CL,Zhu L,Kaste SC,Srivastava K,Barnes L,Nathan PC,Wells RJ,Ness KK

    更新日期:2018-04-01 00:00:00

  • Bevacizumab for symptomatic radiation-induced tumor enlargement in pediatric low grade gliomas.

    abstract:BACKGROUND:Radiation therapy (RT)-induced effects in children treated for low grade glioma (LGG) can result in worsening of neurologic symptoms and clinical and radiographic deterioration. Treatment for radiation-induced tumor enlargement is based on symptom control and usually involves steroids. PROCEDURE:We conducte...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.25277

    authors: Foster KA,Ares WJ,Pollack IF,Jakacki RI

    更新日期:2015-02-01 00:00:00

  • Oral leukoplakia in patients with Fanconi anaemia without hematopoietic stem cell transplantation.

    abstract:BACKGROUND:Fanconi anaemia is a genetic disease characterized by congenital abnormalities, progressive bone marrow failure, and a higher predisposition of oral squamous cell carcinoma. The purpose of this study was to evaluate the prevalence of oral mucosa lesions in patients with Fanconi anaemia without hematopoietic ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.25417

    authors: Grein Cavalcanti L,Lyko KF,Araújo RL,Amenábar JM,Bonfim C,Torres-Pereira CC

    更新日期:2015-06-01 00:00:00

  • Reproductive intentions in mothers of young children with sickle cell disease.

    abstract:BACKGROUND:Sickle cell disease (SCD) is an autosomal recessive hemoglobinopathy associated with morbidity and mortality. We sought to understand family planning intentions of parents of young children with SCD including the awareness of three reproductive options (adoption, in vitro fertilization with egg/sperm donatio...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.28227

    authors: Schultz CL,Tchume-Johnson T,Jackson T,Enninful-Eghan H,Schapira MM,Smith-Whitley K

    更新日期:2020-05-01 00:00:00

  • Medulloblastoma in a child with down syndrome: long-term remission with multimodality treatment.

    abstract::A 4(3/4)-year-old male with Down syndrome (DS) presented with unsteady gait and fatigue. Neuroimaging revealed a cerebellar mass with concomitant obstructive hydrocephalus and additional metastatic lesions. He was successfully treated and is still in complete remission 5 years from diagnosis. The present case illustra...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.22109

    authors: Benesch M,Moser A,Sovinz P,Lackner H,Schwinger W,Eder H,Urban C

    更新日期:2009-12-01 00:00:00

  • Central nervous system disease in pediatric acute myeloid leukemia: A report from the Children's Oncology Group.

    abstract:BACKGROUND:The prognostic impact of central nervous system (CNS) involvement in children with acute myeloid leukemia (AML) has varied in past trials, and controversy exists over the degree of involvement requiring intensified CNS therapy. Two recent Children's Oncology Group protocols, AAML03P1 and AAML0531, directed a...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.26612

    authors: Johnston DL,Alonzo TA,Gerbing RB,Aplenc R,Woods WG,Meshinchi S,Gamis AS

    更新日期:2017-12-01 00:00:00

  • The Saint Siluan warning signs of cancer in children: impact of education in rural South Africa.

    abstract::Two thirds of children with cancer never reach a specialist centre for treatment in South Africa. The majority of those who present have advanced disease. A campaign was undertaken to educate the public and the primary health workers on the Saint Siluan early warning signs of cancer in children. There was a statistica...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.22853

    authors: Poyiadjis S,Wainwright L,Naidu G,Mackinnon D,Poole J

    更新日期:2011-02-01 00:00:00

  • Growth hormone therapy for short stature in Diamond Blackfan anemia.

    abstract:BACKGROUND:We report a 13-year-old male with Diamond Blackfan anemia and short stature. He had a normal biochemical response to growth hormone (GH) stimulation, but his bone age was delayed, his insulin-like growth factor 1 (IGF-1) was low, and he had a poor growth velocity. He was started on daily GH injections. METH...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.20075

    authors: Scott EG,Haider A,Hord J

    更新日期:2004-10-01 00:00:00

  • Defibrotide treatment but not prophylaxis is useful in hepatic sinusoidal obstruction syndrome in children undergoing autologous stem cell transplant following high-dose chemotherapy: A single-center experience from the Royal Marsden Hospital, UK.

    abstract:BACKGROUND:Hepatic sinusoidal obstruction syndrome (SOS) is a serious complication of autologous stem cell transplant (ASCT) in children with historically high mortality rates. Defibrotide has shown proven benefit in its treatment and may have a modest role in prevention. We report our experience with SOS in children u...

    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章

    doi:10.1002/pbc.28677

    authors: Roy Moulik N,Johnson I,Van Bruggen L,Petterson T,Mycroft J,Vaidya SJ

    更新日期:2020-11-01 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

  • Absence of platelet recovery following Helicobacter pylori eradication in childhood chronic idiopathic thrombocytopenic purpura: a multi-center randomized controlled trial.

    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

    authors: Treepongkaruna S,Sirachainan N,Kanjanapongkul S,Winaichatsak A,Sirithorn S,Sumritsopak R,Chuansumrit A

    更新日期:2009-07-01 00:00:00

  • Effect of transfusion therapy on transcranial Doppler ultrasonography velocities in children with sickle cell disease.

    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

    authors: Kwiatkowski JL,Yim E,Miller S,Adams RJ,STOP 2 Study Investigators.

    更新日期:2011-05-01 00:00:00

  • A phase II study of Campath-1H in children with relapsed or refractory acute lymphoblastic leukemia: a Children's Oncology Group report.

    abstract:BACKGROUND:Despite the increasing cure rates for children with acute lymphoblastic leukemia (ALL), patients who relapse continue to have poor prognosis. The Children's Oncology Group (COG) conducted a limited institution Phase II trial of Campath-1H, a monoclonal antibody that targets CD52 on leukemic cells, in childre...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,多中心研究

    doi:10.1002/pbc.22209

    authors: Angiolillo AL,Yu AL,Reaman G,Ingle AM,Secola R,Adamson PC

    更新日期:2009-12-01 00:00:00

  • Glucocorticoid-Induced Proliferation in Untreated Pediatric Acute Myeloid Leukemic Blasts.

    abstract::We evaluated the in vitro glucocorticoid (GC) responsiveness of 117 pediatric acute myeloid leukemia cells by considering GC resistance, GC-induced proliferation, and GC-induced differentiation. None of the samples was highly GC sensitive, and only 15% were intermediately sensitive. GC-induced differentiation was not ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.26011

    authors: Klein K,Haarman EG,de Haas V,Zwaan ChM,Creutzig U,Kaspers GL

    更新日期:2016-08-01 00:00:00