Treatment of the X-linked lymphoproliferative, Griscelli and Chédiak-Higashi syndromes by HLH directed therapy.

abstract：:Treatment with the nucleoside analog cytarabine has been shown to mimic changes in gene expression associated with downregulation of the EWS-FLI1 oncogene in Ewing sarcoma cell lines, selectively inhibit their growth in vitro, and cause tumor regression in athymic nude mice. For this report cytarabine was studied in v...

journal_title：Pediatric blood & cancer

authors： Houghton PJ,Morton CL,Kang M,Reynolds CP,Billups CA,Favours E,Payne-Turner D,Tucker C,Smith MA

更新日期：2010-12-01 00:00:00

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

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

更新日期：2006-10-15 00:00:00

abstract：BACKGROUND:Soft tissue sarcomas (STS) are a heterogeneous group of mesenchymal malignancies that occur throughout the lifespan. The impact of age on disease features and outcome is unclear. METHODS:We analyzed the clinical features and outcome of all STS cases registered between 1973 and 2006 in the SEER database. RE...

journal_title：Pediatric blood & cancer

authors： Ferrari A,Sultan I,Huang TT,Rodriguez-Galindo C,Shehadeh A,Meazza C,Ness KK,Casanova M,Spunt SL

更新日期：2011-12-01 00:00:00

abstract：:Squamous cell carcinoma of the anal canal in children is rare. To date, the etiology and outcome of this condition have been not fully understood. Here, we report an 11-year-old child with anal canal cancer who had concomitant disorders of sex development. Radiotherapy followed by salvage surgery achieved disease-free...

journal_title：Pediatric blood & cancer

authors： Watanabe T,Horikawa R,Masaki H,Yoshioka T,Matsumoto K,Kanamori Y

更新日期：2016-07-01 00:00:00

abstract：BACKGROUND:Thiotepa and carboplatin are known to be active in central nervous system tumors. Topotecan potentiates the anti-cancer effects of alkylators and crosses the blood-brain barrier. We present ten patients with recurrent or progressive central nervous system malignancies treated on a myeloablative regimen using...

journal_title：Pediatric blood & cancer

authors： Gilheeney SW,Khakoo Y,Souweidane M,Wolden S,Boulad F,Dunkel IJ

更新日期：2010-04-01 00:00:00

abstract：BACKGROUND:The awareness that adolescents can have cancer is probably insufficient, not only among teenagers and their families, but also among physicians, and adolescent patients are reportedly often referred to qualified cancer institutes after a considerable delay. PROCEDURE:A prospective series of 425 patients (28...

journal_title：Pediatric blood & cancer

authors： Veneroni L,Mariani L,Lo Vullo S,Favini F,Catania S,Vajna de Pava M,Massimino M,Ferrari A

更新日期：2013-04-01 00:00:00

abstract：:A male patient diagnosed with severe congenital protein C (PC) deficiency during the neonatal period was treated with long-term warfarin but frequently developed purpura fulminans and bleeding. At four years of age, edoxaban was initiated (direct oral anticoagulant [DOAC]). His d-dimer and fibrin/fibrinogen degradatio...

journal_title：Pediatric blood & cancer

authors： Watanabe K,Arakawa Y,Yanagi M,Isobe K,Mori M,Koh K

更新日期：2019-06-01 00:00:00

abstract：:Because of increasing survival rates in pediatric oncology, attention is focusing on cancer and its treatment-related side effects. Rehabilitation may reduce their impact. However, the literature does not provide strong evidence regarding rehabilitation pathways. Therefore, the Italian Association of Pediatric Hematol...

journal_title：Pediatric blood & cancer

authors： Rossi F,Ricci F,Botti S,Bertin D,Breggiè S,Casalaz R,Cervo M,Ciullini P,Coppo M,Cornelli A,Esposito M,Ferrarese M,Ghetti M,Longo L,Morri M,Naretto G,Orsini N,Fagioli F,AIEOP Rehabilitation Working Group Members.

更新日期：2020-12-01 00:00:00

abstract：:Hepatic sinusoidal obstructive syndrome (HSOS) is a frequent complication in patients undergoing haematopoietic stem cell transplant (HSCT), and more rarely, in paediatric patients receiving conventional chemotherapy for solid tumours. Its diagnosis relies on a combination of clinical signs and symptoms such as hepato...

journal_title：Pediatric blood & cancer

authors： De Leonardis F,Koronica R,Bruno SD,Santoro N

更新日期：2014-05-01 00:00:00

abstract：:Osteoporosis and osteopenia are long-term side effects of bone marrow transplant (BMT). The purpose of this study was to determine the prevalence of bone mineral density (BMD) abnormalities in pediatric patients prior to BMT. Forty-four pediatric patients were evaluated with DEXA scans. The average Z-score was -0.37. ...

journal_title：Pediatric blood & cancer

authors： Klopfenstein KJ,Clayton J,Rosselet R,Kerlin B,Termuhlen A,Gross T

更新日期：2009-10-01 00:00:00

abstract：BACKGROUND:Image-guided percutaneous core needle biopsy (PCNB) is increasingly utilized to diagnose solid tumors. The objective of this study is to determine whether PCNB is adequate for modern biologic characterization of neuroblastoma. PROCEDURE:A multi-institutional retrospective study was performed by the Pediatri...

journal_title：Pediatric blood & cancer

authors： Overman RE,Kartal TT,Cunningham AJ,Fialkowski EA,Naik-Mathuria BJ,Vasudevan SA,Malek MM,Kalsi R,Le HD,Stafford LC,Lautz TB,Many BT,Jones RE,Bütter A,Davidson J,Williams A,Dasgupta R,Lewis J,Troutt M,Aldrink JH,Man

更新日期：2020-05-01 00:00:00

abstract：:Spermatogonial stem cells open novel strategies for preservation of testicular tissue and fertility preservation in boys and men exposed to gonadotoxic therapies. This review provides an update on the physiology of spermatogonial stem cells in rodent and primate testes. Species-specific differences must be considered ...

journal_title：Pediatric blood & cancer

authors： Schlatt S,Ehmcke J,Jahnukainen K

更新日期：2009-08-01 00:00:00

abstract：BACKGROUND:The high cost, coupled with the need for continuous infusion, renders Desferrioxamine (DFO), a non-feasible option for iron-chelation in a large majority of patients with β-thalassemia major in developing countries. Monotherapy with deferiprone (DFP) or deferasirox (DFX) may not always attain optimal control...

journal_title：Pediatric blood & cancer

authors： Totadri S,Bansal D,Bhatia P,Attri SV,Trehan A,Marwaha RK

更新日期：2015-09-01 00:00:00

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

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

更新日期：2009-05-01 00:00:00

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

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

abstract：:This review of the experience of high dose chemotherapy in patients with recurrent or refractory intracranial germ cell tumor confirms that sustained tumor control can be achieved with this modality both in germinoma and non-germinomatous germ cell tumors. Data from cooperative groups in Europe and North America sugge...

journal_title：Pediatric blood & cancer

authors： Bouffet E

更新日期：2010-04-01 00:00:00

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

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

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

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

更新日期：2018-12-01 00:00:00

abstract：:Germline or acquired mutations involving the GATA-binding protein gene (GATA2) have been linked to a variety of clinical conditions. In addition, patients harboring GATA2 mutations have a striking predisposition to develop myeloid malignancies, such as myelodysplastic syndrome or acute myeloid leukemia, but not acute ...

journal_title：Pediatric blood & cancer

authors： Esparza O,Xavier AC,Atkinson TP,Hill BC,Whelan K

更新日期：2019-06-01 00:00:00

abstract：BACKGROUND:Langerhans cell histiocytosis (LCH) is a rare proliferative disorder of pathological Langerhans cells, for which the aetiology and pathogenesis remain largely unknown. PROCEDURE:Information on the 101 children with LCH registered with the population-based Manchester Children's Tumour Registry (MCTR) between...

journal_title：Pediatric blood & cancer

authors： Alston RD,Tatevossian RG,McNally RJ,Kelsey A,Birch JM,Eden TO

更新日期：2007-05-01 00:00:00

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

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

abstract：BACKGROUND:Retinoblastoma occurs in approximately 4 per million children per year in the United States, usually before 2 years of age. In developed countries, 95% of children with tumor in one eye can be cured with enucleation, while children with tumor in both eyes require individualized therapy to preserve vision. Al...

journal_title：Pediatric blood & cancer

authors： Lambert MP,Shields C,Meadows AT

更新日期：2008-02-01 00:00:00

abstract：:We describe a child with dyserythropoietic anemia, thrombocytosis, functional platelet defect, and megakaryocyte dysplasia. We show that (i) this constellation of hematopoietic abnormalities was due to a germline mutation within the 5' untranslated region (5'UTR) of globin transcription factor 1 (GATA1); (ii) the muta...

journal_title：Pediatric blood & cancer

authors： Zucker J,Temm C,Czader M,Nalepa G

更新日期：2016-05-01 00:00:00

abstract：BACKGROUND:Ispinesib is a highly specific inhibitor of kinesin spindle protein (KSP, HsEg5), a mitotic kinesin required for separation of the spindle poles. Here we report the activity of ispinesib against the in vitro and in vivo panels of the Pediatric Preclinical Testing Program (PPTP). PROCEDURES:Ispinesib was tes...

journal_title：Pediatric blood & cancer

authors： Carol H,Lock R,Houghton PJ,Morton CL,Kolb EA,Gorlick R,Reynolds CP,Maris JM,Keir ST,Billups CA,Smith MA

更新日期：2009-12-15 00:00:00

abstract：BACKGROUND:Hypogammaglobulinemia (hypo-IgG) is common early post-HSCT in children, occasionally necessitating long-term immunoglobulin (Ig) G replacement therapy. IgG replacement may not reduce mortality, although infectious complications are decreased PROCEDURE:Clinical data and samples from 86 children were analyzed...

journal_title：Pediatric blood & cancer

authors： Sundin M,Remberger M,Lindqvist H,Omazic B,Sundberg B,Uzunel M,Winiarski J

更新日期：2015-05-01 00:00:00

abstract：PURPOSE:To assess local control, event-free survival (EFS), and overall survival (OS) rates in 71 patients with localized, completely resected (Group I) alveolar rhabdomyosarcoma (ALV RMS) and their relation to radiation therapy (RT) on IRSG Protocols III and IV, 1984-1997. METHODS:Chart review and standard statistica...

journal_title：Pediatric blood & cancer

authors： Raney RB,Anderson JR,Brown KL,Huh WW,Maurer HM,Meyer WH,Parham DM,Rodeberg DA,Wolden SL,Donaldson SS,Soft-Tissue Sarcoma Committee of the Children's Oncology Group Arcadia California USA.

更新日期：2010-10-01 00:00:00

abstract：BACKGROUND:Accurate risk stratification of Langerhans cell histiocytosis (LCH) is essential as management can range from conservative in single system, low risk for central nervous system (CNS) involvement lesions to intensive chemotherapy for multisystem or high-risk disease. Additionally, being able to differentiate ...

journal_title：Pediatric blood & cancer

authors： Ferrell J,Sharp S,Kumar A,Jordan M,Picarsic J,Nelson A

更新日期：2021-01-14 00:00:00

abstract：BACKGROUND:Fever and neutropenia (F&N) is a pediatric oncology emergency due to the risk of disseminated infection. Quality improvement (QI) efforts to improve time to antibiotics for F&N in the emergency department have been documented, but the issue has not been studied in the established inpatient setting. PROCEDUR...

journal_title：Pediatric blood & cancer

authors： Green AL,Yi J,Bezler N,Pikman Y,Tubman VN,Obeng EA,O'Neil T,Mersereau R,Morrissey L,Billett AL

更新日期：2016-01-01 00:00:00

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

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

abstract：BACKGROUND:Severe congenital neutropenia is a rare disease, and autosomal dominantly inherited ELANE mutation is the most frequently observed genetic defect in the registries from North America and Western Europe. However, in eastern countries where consanguineous marriages are common, autosomal recessive forms might b...

journal_title：Pediatric blood & cancer

authors： Yılmaz Karapınar D,Patıroğlu T,Metin A,Çalışkan Ü,Celkan T,Yılmaz B,Karakaş Z,Karapınar TH,Akıncı B,Özkınay F,Onay H,Yeşilipek MA,Akar HH,Tüysüz G,Tokgöz H,Özdemir GN,Aslan Kıykım A,Karaman S,Kılınç Y,Oymak Y,Küpe

更新日期：2019-10-01 00:00:00