Clinical advances in hemophilia management.

Abstract:

:Hemophilia is an excellent example in medicine where clinical translation of basic science discoveries has transformed the gloomy outlook of the disease. This review provides an overview of clinical advances in hemophilia management with a specific focus on the molecular heterogeneity of the disease and progress in management of patients with inhibitors. Novel therapeutics and the emerging ethical issues in the field of hemophilia are also discussed.

journal_name

Pediatr Blood Cancer

journal_title

Pediatric blood & cancer

authors

Sharathkumar AA,Carcao M

doi

10.1002/pbc.23193

subject

Has Abstract

pub_date

2011-12-01 00:00:00

pages

910-20

issue

6

eissn

1545-5009

issn

1545-5017

journal_volume

57

pub_type

杂志文章,评审
  • The impact of hyperglycemia on risk of infection and early death during induction therapy for acute lymphoblastic leukemia (ALL).

    abstract::Hyperglycemia during induction chemotherapy for childhood acute lymphoblastic leukemia (ALL) has been inconsistently associated with risk of infection. We investigated the incidence of hyperglycemia during induction for childhood ALL in a retrospective cohort study of 144 patients treated on a single national protocol...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.24689

    authors: Dare JM,Moppett JP,Shield JP,Hunt LP,Stevens MC

    更新日期:2013-12-01 00:00:00

  • Normal ovarian function and assessment of ovarian reserve in the survivor of childhood cancer.

    abstract::Increasingly young people survive cancer in childhood and as a result complications of its treatment are becoming more common and important. Premature ovarian failure is recognized as a complication of radiotherapy to a field that includes the pelvis and alkylating-agent-based chemotherapy. Young pre-pubertal girls ar...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,评审

    doi:10.1002/pbc.22012

    authors: Johnston RJ,Wallace WH

    更新日期:2009-08-01 00:00:00

  • Gender affects survival for medulloblastoma only in older children and adults: a study from the Surveillance Epidemiology and End Results Registry.

    abstract:BACKGROUND:Males have a higher incidence of medulloblastoma (MB) than females, but the effect of gender on survival is unclear. Studies have yielded conflicting results, possibly due to small sample sizes or differences in how researchers defined MB. We aimed to determine the effect of gender on survival in MB using a ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.21832

    authors: Curran EK,Sainani KL,Le GM,Propp JM,Fisher PG

    更新日期:2009-01-01 00:00:00

  • Histologic survey of neuroblastomas after intensive induction chemotherapy.

    abstract:BACKGROUND:Histology after intensive induction chemotherapy is expected to become a beacon indicating when and how extensively radical surgery and lymph node dissection should be performed in advanced neuroblastoma. A thorough histologic review of surgical specimens was undertaken. PROCEDURE:All specimens from 34 pati...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.20345

    authors: Tsuchida Y,Miyauchi J,Kuroiwa M,Suzuki N,Sakamoto J,Suzuki M,Shitara T

    更新日期:2005-10-15 00:00:00

  • Phase I study of oral cyclophosphamide and oral topotecan for children with recurrent or refractory solid tumors.

    abstract:BACKGROUND:To determine the maximum-tolerated duration and dose-limiting toxicity of a daily schedule of orally administered cyclophosphamide and topotecan in pediatric patients with recurrent or refractory malignant solid tumors. METHODS:Patients received oral cyclophosphamide (50 mg/m2/dose) in the morning followed ...

    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章

    doi:10.1002/pbc.10456

    authors: Bowers DC,Aquino VM,Leavey PJ,Bash RO,Journeycake JM,Tomlinson G,Mulne AF,Haynes HJ,Winick NJ

    更新日期:2004-01-01 00:00:00

  • Contributing factors and outcomes of treatment refusal in pediatric oncology in Germany.

    abstract:BACKGROUND:In Germany, about 1,800 new cases of pediatric cancer under 15 years of age are diagnosed each year and survival rates approach 80%. Although treatment is covered by health insurance and is thus available for all patients at no cost, treatment refusal and treatment discontinuation have been observed. However...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.26111

    authors: Zuzak TJ,Kameda G,Schütze T,Kaatsch P,Seifert G,Bailey R,Längler A

    更新日期:2016-10-01 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

  • Inflammatory myofibroblastic tumor as a second neoplasm after Wilms tumor.

    abstract::We report two survivors of Wilms tumor (WT) who developed inflammatory myofibroblastic tumor (IMT). The first patient had IMT in the mainstem bronchus 1 year after WT therapy that included vincristine, dactinomycin, doxorubicin (VDA) and whole lung radiation therapy (XRT). The second patient had IMT in the cecum 7 yea...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.25345

    authors: Ortiz MV,Rossi CT,Hill DA,Guzzetta PC,Qureshi F,Dome JS

    更新日期:2015-06-01 00:00:00

  • Intermittent oral trimethoprim/sulfamethoxazole on two non-consecutive days per week is effective as Pneumocystis jiroveci pneumonia prophylaxis in pediatric patients receiving chemotherapy or hematopoietic stem cell transplantation.

    abstract::Pneumocystis jiroveci pneumonia (PCP) is a serious complication in patients receiving chemotherapy or hematopoietic stem cell transplantation. Current recommendations for trimethoprim-sulfamethoxazole (TMP-SMZ) dosing as PCP prophylaxis in immunocompromised patients are based on either daily dosing or dosing three con...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.21774

    authors: Ohata Y,Ohta H,Hashii Y,Tokimasa S,Ozono K,Hara J

    更新日期:2009-01-01 00:00:00

  • Intensity of CNS treatment for pediatric cancer: prediction of social outcomes in survivors.

    abstract:PURPOSE:To evaluate the association of central nervous system (CNS) treatment intensity and the social functioning of children who have completed treatment for leukemia, lymphoma, and solid tumors outside the CNS. Furthermore, we expected that these associations would be moderated by child age at diagnosis and gender. ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.21062

    authors: Vannatta K,Gerhardt CA,Wells RJ,Noll RB

    更新日期:2007-10-15 00:00:00

  • Asociación de Hemato-Oncología Pediátrica de Centro América (AHOPCA): a model for sustainable development in pediatric oncology.

    abstract::Bridging the survival gap for children with cancer, between those (the great majority) in low and middle income countries (LMIC) and their economically advantaged counterparts, is a challenge that has been addressed by twinning institutions in high income countries with centers in LMIC. The long-established partnershi...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,评审

    doi:10.1002/pbc.24802

    authors: Barr RD,Antillón Klussmann F,Baez F,Bonilla M,Moreno B,Navarrete M,Nieves R,Peña A,Conter V,De Alarcón P,Howard SC,Ribeiro RC,Rodriguez-Galindo C,Valsecchi MG,Biondi A,Velez G,Tognoni G,Cavalli F,Masera G

    更新日期:2014-02-01 00:00:00

  • Recurrent metastatic neuroblastoma followed by myelodysplastic syndrome: possible leukemogenic role of temozolomide.

    abstract::An 8-year old child had a pelvic MYCN-nonamplified neuroblastoma (NB) with retroperitoneal nodal extension. Multi-modality therapy achieved complete remission (CR). Small recurrences confined to left supraclavicular nodes were treated with surgery alone at 4.9, 6.5, 7.5, 9.5, and 12.9 years from diagnosis. Monitoring ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.21658

    authors: Kushner BH,Laquaglia MP,Kramer K,Modak S,Cheung NK

    更新日期:2008-10-01 00:00:00

  • Lung metastases in neuroblastoma at initial diagnosis: A report from the International Neuroblastoma Risk Group (INRG) project.

    abstract:BACKGROUND:Neuroblastoma is the most common extracranial pediatric solid cancer. Lung metastasis is rarely detected in children with newly diagnosed neuroblastoma. We aimed to describe the incidence, clinical characteristics, and outcome of patients with lung metastasis at initial diagnosis using a large international ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.21684

    authors: Dubois SG,London WB,Zhang Y,Matthay KK,Monclair T,Ambros PF,Cohn SL,Pearson A,Diller L

    更新日期:2008-11-01 00:00:00

  • Clinical practice guideline improves the treatment of sickle cell disease vasoocclusive pain.

    abstract:BACKGROUND:Pain is the most common complication of sickle cell disease requiring emergency department (ED) visits and hospitalization. A Clinical Practice Guideline (CPG) to manage acute sickle cell pain offers clinicians a standardized approach for the provision of evidence-based, cost-effective care. After CPG implem...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.21847

    authors: Morrissey LK,Shea JO,Kalish LA,Weiner DL,Branowicki P,Heeney MM

    更新日期:2009-03-01 00:00:00

  • Sickle-cell disease in California: a population-based description of emergency department utilization.

    abstract:BACKGROUND:Acute and chronic clinical manifestations of sickle-cell disease (SCD) lead to significant healthcare utilization, especially of the emergency department (ED). Limited population-level data are available in SCD with the ability to connect patients to visits, leaving us with minimal description of utilization...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,多中心研究

    doi:10.1002/pbc.22792

    authors: Wolfson JA,Schrager SM,Coates TD,Kipke MD

    更新日期:2011-03-01 00:00:00

  • Esophagitis associated with multimodality management of pediatric Ewing sarcoma of thorax.

    abstract:BACKGROUND:Ewing sarcoma of the thoracic spine and chest wall is frequently treated with concurrent chemotherapy and radiation therapy (RT). Treatment-related acute esophagitis can lead to hospitalization and treatment delays. The aim of this study was to analyze the incidence, risk factors, and management of esophagit...

    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章

    doi:10.1002/pbc.27006

    authors: Agarwal V,Logie N,Morris CG,Bradley JA,Rotondo RL,Bradfield SM,Indelicato DJ

    更新日期:2018-06-01 00:00:00

  • Internet-delivered insomnia intervention improves sleep and quality of life for adolescent and young adult cancer survivors.

    abstract:BACKGROUND:Insomnia is common among adolescent and young adult (AYA) cancer survivors. Cognitive-behavioral therapy for insomnia (CBT-I) is considered the gold standard treatment. Standard CBT-I was designed for adults and not adapted to the unique medical, psychosocial, and developmental needs of AYA cancer survivors,...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.28506

    authors: Zhou ES,Recklitis CJ

    更新日期:2020-09-01 00:00:00

  • Screening for vitamin D insufficiency in pediatric cancer survivors.

    abstract:BACKGROUND:Corticosteroids increase risk for decreased bone mineral density, which can be worsened by vitamin D insufficiency (VDI) or deficiency (VDD). PROCEDURE:In the Vanderbilt cancer survivorship clinic, we obtained screening total 25-hydroxy vitamin D levels (VDL) in 171 cancer survivors <23 years old who were t...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.24844

    authors: Esbenshade AJ,Sopfe J,Zhao Z,Li Z,Campbell K,Simmons JH,Friedman DL

    更新日期:2014-04-01 00:00:00

  • Reversible Fanconi syndrome in a pediatric patient on deferasirox.

    abstract::Deferasirox (Exjade®, Novartis) is a widely used oral iron chelator for the treatment of patients with iron overload due to chronic transfusion therapy for diseases such as β-thalassemia and sickle cell disease. Renal side effects of deferasirox are common and include non-progressive increases in serum creatinine, how...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.22711

    authors: Rheault MN,Bechtel H,Neglia JP,Kashtan CE

    更新日期:2011-04-01 00:00:00

  • Children with sickle cell disease migrating to the United States from sub-Saharan Africa.

    abstract::Sickle cell disease (SCD) is an inherited red blood cell disorder that occurs worldwide and results in significant morbidity and mortality. In addition to affected children who are born in the US, an increasing number of children with SCD are migrating to the US with their families or through international adoption. C...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.27000

    authors: Thornburg CD,Ware RE

    更新日期:2018-06-01 00:00:00

  • PARP1 expression in pediatric central nervous system tumors.

    abstract:BACKGROUND:Despite advances in therapy, outcome in many high-grade pediatric central nervous system (CNS) tumors remains poor. The focus of neuro-oncology research has thus turned towards identifying novel therapeutic targets. Poly(ADP-ribose) polymerase-1 (PARP1) is a DNA repair protein that has been studied in a vari...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.22141

    authors: Barton VN,Donson AM,Kleinschmidt-DeMasters BK,Gore L,Liu AK,Foreman NK

    更新日期:2009-12-15 00:00:00

  • Outcomes for pediatric patients with osteosarcoma treated with palliative radiotherapy.

    abstract:BACKGROUND:Few studies have addressed the efficacy of palliative radiotherapy (RT) for pediatric osteosarcoma (OS), a disease generally considered to be radioresistant. We describe symptom relief, local control, and toxicity associated with palliative RT among children with OS. PROCEDURE:Patients diagnosed with OS at ...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.27967

    authors: Chen EL,Yoo CH,Gutkin PM,Merriott DJ,Avedian RS,Steffner RJ,Spunt SL,Pribnow AK,Million L,Donaldson SS,Hiniker SM

    更新日期:2020-01-01 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

  • Molecular characterization of a novel splice site mutation within the CYBB gene leading to X-linked chronic granulomatous disease.

    abstract::Chronic granulomatous disease (CGD) is a primary immunodeficiency that affects the oxidative mechanism of microbial killing of phagocytic cells. The defect is characterized by a lack or severely reduced superoxide anion (O2-) production by phagocytes. Seventy percent of CGD cases are X-linked (X-CGD) and they are caus...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.20204

    authors: Barese CN,Copelli SB,De Matteo E,Zandomeni R,Salgueiro F,Di Giovanni D,Heyworth P,Rivas EM

    更新日期:2005-04-01 00:00:00

  • Cytotoxic potential of IL-15-activated cytokine-induced killer cells against human neuroblastoma cells.

    abstract:BACKGROUND:Neuroblastoma (NB) is the most common solid extracranial tumor in childhood. Despite advances in therapy, the prognosis is poor and optimized therapies are urgently needed. Therefore, we investigated the antitumor potential of interleukin-15 (IL-15)-activated cytokine-induced killer (CIK) cells against diffe...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.26147

    authors: Cappel C,Huenecke S,Suemmerer A,Erben S,Rettinger E,Pfirrmann V,Heinze A,Zimmermann O,Klingebiel T,Ullrich E,Bader P,Bremm M

    更新日期:2016-12-01 00:00:00

  • Incidence of acute kidney injury among pediatric hematology/oncology patients receiving vancomycin in combination with piperacillin/tazobactam or cefepime.

    abstract::There is mounting evidence that combination of antibiotic therapy with vancomycin and piperacillin/tazobactam (pip/tazo) is associated with acute kidney injury (AKI). To determine whether vancomycin plus pip/tazo is associated with higher rates of AKI compared to vancomycin plus cefepime among pediatric hematology/onc...

    journal_title:Pediatric blood & cancer

    pub_type: 临床试验,杂志文章

    doi:10.1002/pbc.27750

    authors: Quach HT,Esbenshade AJ,Zhao Z,Banerjee R

    更新日期:2019-07-01 00:00:00

  • The Bt-DUX: development of a subjective measure of health-related quality of life in patients who underwent surgery for lower extremity malignant bone tumor.

    abstract:BACKGROUND:To examine the practical applicability, internal consistency, and validity of the Bt-DUX, a disease-specific Health Related Quality of Life (HRQoL) instrument. The Bt-Dux was developed to examine patients' individual values of their life after a malignant bone tumor of the lower extremity at four domains (co...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章,多中心研究

    doi:10.1002/pbc.22078

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

    更新日期:2009-09-01 00:00:00

  • Medication errors on oral chemotherapy in children with acute lymphoblastic leukemia in a developing country.

    abstract:BACKGROUND:Medication errors occur universally. Inappropriate administration of chemotherapy drugs can have adverse effects in cancer patients. Our objective was to assess the rate and type of medication errors in children with acute lymphoblastic leukemia (ALL) receiving oral chemotherapy in outpatient setting. PROCE...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.25203

    authors: Oberoi S,Trehan A,Marwaha RK

    更新日期:2014-12-01 00:00:00

  • Chemotherapy responsiveness in a patient with multiply relapsed ameloblastic fibro-odontosarcoma of the maxilla.

    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

    authors: Gatz SA,Thway K,Mandeville H,Kerawala C,MacVicar D,Chisholm J

    更新日期:2015-11-01 00:00:00

  • Familial retinoblastoma in developing countries.

    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, Turke...

    journal_title:Pediatric blood & cancer

    pub_type: 杂志文章

    doi:10.1002/pbc.21970

    authors: Chantada GL,Dunkel IJ,Qaddoumi I,Antoneli CB,Totah A,Canturk S,Nawaiseh I,Fandiño A,Pífano I,Peksayar G,Ribeiro KB,Abramson DH

    更新日期:2009-09-01 00:00:00