Abstract:
BACKGROUND:A majority of Fanconi anemia (FA) patients will experience bone marrow failure (BMF) and androgen therapy (most often oxymetholone) may be utilized as a treatment to improve BMF-related cytopenias. However, oxymetholone is associated with toxicities making identification of other agents of interest. In this study we aimed to evaluate the toxicity profile and hematologic response in patients with FA who are treated with low-dose oxandrolone, a synthetic non-fluorinated anabolic steroid, similar to oxymetholone, with known dosing thresholds for virilization. PROCEDURE:A single arm, Phase I/II study was designed to treat patients on low-dose oxandrolone. If no toxicity or hematologic response was noted at 16 weeks, a single dose escalation was offered. Subjects were regularly assessed for toxicity, including determinations of virilization, behavioral changes, and liver and kidney function. At 32 weeks, those who demonstrated hematologic response were allowed to continue study treatment, and those without improvement were deemed non-responsive. RESULTS:Nine subjects completed the study and were followed for a median of 99 weeks (46-136 weeks). Three (33.3%) subjects developed mild sub-clinical virilization and continued treatment with a dose reduction. None (0%) had adverse behavioral changes. Two (22.2%) developed elevated liver function tests at 42 and 105 weeks. Seven (77.8%) subjects had a hematologic response. CONCLUSION:Oxandrolone appears to be well-tolerated, has limited toxicities at the administered doses in FA with patients, and may be an alternative androgen for the treatment of BMF in FA.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Rose SR,Kim MO,Korbee L,Wilson KA,Ris MD,Eyal O,Sherafat-Kazemzadeh R,Bollepalli S,Harris R,Jeng MR,Williams DA,Smith FOdoi
10.1002/pbc.24617subject
Has Abstractpub_date
2014-01-01 00:00:00pages
11-9issue
1eissn
1545-5009issn
1545-5017journal_volume
61pub_type
杂志文章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
更新日期:2008-11-01 00:00:00
abstract::Anaphylactic/anaphylactoid reaction to methotrexate (MTX) is uncommon. It may occur with the first dose (non-allergic reactions) or after a previous exposure to the drug (allergic or specific reactions). Desensitization has been shown effective in children with allergic-type reactions permitting the continuation of hi...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22616
更新日期:2010-09-01 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
pub_type: 杂志文章
doi:10.1002/pbc.21838
更新日期:2009-05-01 00:00:00
abstract:BACKGROUND:To evaluate outcomes of Group D eyes of bilateral retinoblastoma patients treated with primary chemoreduction and external beam radiation as salvage. PROCEDURE:Retrospective chart review of patients diagnosed with bilateral retinoblastoma and designated Group D in at least one eye from January 1, 2000 to De...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24303
更新日期:2013-04-01 00:00:00
abstract::Imatinib can be safely discontinued in adults with chronic myeloid leukemia (CML) where there is a prolonged complete molecular response (CMR). No data are available in the pediatric population. Six children with CML discontinued imatinib by themselves. Only three of them were in CMR but for <2 years. A significant in...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24521
更新日期:2014-02-01 00:00:00
abstract:BACKGROUND:Ewing sarcoma (ES), a highly aggressive tumor of children and young adults, is characterized most commonly by an 11;22 chromosomal translocation that fuses EWSR1 located at 22q12 with FLI1, coding for a member of the ETS family of transcription factors. Although genetic changes in ES have been extensively re...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.24526
更新日期:2013-09-01 00:00:00
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
更新日期:2018-06-01 00:00:00
abstract:BACKGROUND:Sickle cell disease (SCD) is characterized by frequent disease-related events that require acute care. It is unknown to what extent patients utilize multiple hospitals for acute care. We examined the continuity pattern of acute care visits to the hospital or emergency department. We hypothesized that among p...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24028
更新日期:2012-10-01 00:00:00
abstract:BACKGROUND:The presence of central nervous system (CNS) disease in pediatric acute myeloid leukemia (AML) is often thought to confer a worse prognosis. This study examined the outcome of children with AML who had CNS disease at diagnosis. METHODS:Patients enrolled on Children's Cancer Group protocols 2861, 2891, 2941,...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22511
更新日期:2010-09-01 00:00:00
abstract:BACKGROUND:Malignant peripheral nerve sheath tumor (MPNST) is an aggressive form of soft-tissue sarcoma (STS) in children. Despite intensive therapy, relatively few children with metastatic and unresectable disease survive beyond three years. RAS pathway activation is common in MPNST, suggesting MEK pathway inhibition ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28639
更新日期:2020-12-01 00:00:00
abstract:BACKGROUND:Children with hepatoblastoma (HB) are at risk of sarcopenia due to immobility, chemotherapy, and malnutrition. We hypothesized that children with HB have a low preoperative total psoas muscle area (tPMA), reflecting sarcopenia, which negatively impacts outcome. PROCEDURE:Retrospective study of children (1-1...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28862
更新日期:2021-01-12 00:00:00
abstract::Severe congenital neutropenia (SCN) is a bone marrow failure disease with an autosomal dominant inheritance from mutations in ELANE. Here, we report a 7-week-old Korean male with SCN. His elder sister died from pneumonia at 2 years. Direct sequencing of ELANE in the proband identified a heterozygous novel frameshift m...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25654
更新日期:2015-12-01 00:00:00
abstract::Although awareness of implicit bias and its influence on providers and patients is increasing, the effects of implicit bias on the field of pediatric hematology-oncology are less clear. This Special Report reviews the literature on implicit bias in pediatrics and medical oncology and further provides case examples and...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.28204
更新日期:2020-05-01 00:00:00
abstract:BACKGROUND:Children with sickle cell disease (SCD) and moyamoya may benefit from indirect cerebral revascularization surgery in addition to chronic blood transfusion therapy for infarct prevention. We sought to compare overt and silent infarct recurrence rates in children with SCD undergoing revascularization. METHODS...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26022
更新日期:2016-08-01 00:00:00
abstract:BACKGROUND:Worldwide, hemoglobinopathies affect millions of children. Identification of hemoglobin disorders in most sub-Saharan African countries is delayed until clinical signs of the disease are present. Limited studies have been conducted to understand their prevalence and clinical presentation among newborns in re...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.26735
更新日期:2018-01-01 00:00:00
abstract:BACKGROUND:Information on the epidemiology of pediatric liver tumors in Latin America is limited. PURPOSE:To describe the incidence of liver tumors in a pediatric registry in Argentina according to geographic region, national trends over 16 years, and survival related to stage, age, sex, and care center. METHODS:Newl...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28583
更新日期:2020-11-01 00:00:00
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
更新日期:2008-05-01 00:00:00
abstract::A rare case of primary intestinal T-cell lymphoma (ITL) of an 8-year-old boy is reported. Medium- to large-sized tumor cells were betaF1+, CD3+, CD8+. TIA-1+, but CD4-, CD5-, CD30-, CD56-, CD20-, CD79a-, TdT-, consistent with an intraepithelial lymphocyte (IEL) origin. They showed monoclonal rearrangement of the T-cel...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22330
更新日期:2010-04-01 00:00:00
abstract::We describe here the outcomes of reduced-toxicity alternate-donor stem cell transplant (SCT) with posttransplant cyclophosphamide (PTCy) for primary immunodeficiency disorders (PIDs) in eight children (haploidentical-seven and matched unrelated donor-one). The conditioning was with serotherapy (alemtuzumab-3/rabbit-an...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26783
更新日期:2018-01-01 00:00:00
abstract::It is unclear if dosing intervals for Erwinase can be extended with intramuscular (i.m.) versus intravenous (i.v.) dosing. Children with acute lymphoblastic leukemia received Erwinase at 30 000-42 000 IU/m2 i.v. or i.m. I.m. Erwinase (n = 22) achieved activity above 0.1 IU/mL for longer than i.v. Erwinase (n = 33) (3....
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28244
更新日期:2020-07-01 00:00:00
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
更新日期:2015-01-01 00:00:00
abstract:INTRODUCTION:Thalassemia major (TM) is an inherited disorder caused by ineffective erythropoiesis. At the present time, allogeneic stem cell transplantation (allo-SCT) is a curative option. Conventional busulfan and cyclophosphamide based myeloablative conditioning regimens are limited by increased toxicity, especially...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27312
更新日期:2018-11-01 00:00:00
abstract:BACKGROUND:We investigated the spectrum, frequency, and risk factors for renal, gastrointestinal, and hepatic late adverse effects in survivors of childhood acute myeloid leukemia (AML) without relapse treated with chemotherapy alone according to three consecutive AML trials by the Nordic Society of Pediatric Hematolog...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25069
更新日期:2014-09-01 00:00:00
abstract::Both hepatoblastoma and hypoplastic kidneys are rare in children. A review of all patients with hepatoblastoma treated at our institution between 1993 and 2011 revealed three cases of hepatoblastoma occurring in children with hypoplastic kidneys and significantly impaired renal function. Two patients were treated with...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24936
更新日期:2014-08-01 00:00:00
abstract:BACKGROUND:Childhood cancer survivors (CCS) are at risk of developing long-term morbidity, which is likely to be presented to a primary care physician (PCP). Therefore, insight into CCS's PCP-based health care use is needed. We investigated the volume and underlying health problems of PCP-based health care use and the ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27774
更新日期:2019-08-01 00:00:00
abstract:BACKGROUND:Diamond-Blackfan anemia (DBA) is a rare inherited bone marrow failure syndrome. The mainstays of treatment involve chronic red cell transfusions, long-term glucocorticoid therapy, and stem cell transplantation. Systematic data concerning endocrine function in DBA are limited. We studied patients in the DBA R...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25780
更新日期:2016-02-01 00:00:00
abstract:BACKGROUND:A potential long-term complication of central venous catheter (CVC)-related deep vein thrombosis (DVT), both symptomatic and asymptomatic, is development of post-thrombotic syndrome (PTS) characterized by persistent pain, swelling, and skin changes. Signs and symptoms of PTS were reported after CVC removal. ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22459
更新日期:2010-07-15 00:00:00
abstract::Children with sickle cell anemia (SCA) have low bone mass though bone turnover has not been well described. In this study, growth and pubertal development were assessed twice, 1 year apart, in 80 young subjects with type-SS SCA, while whole body bone mineral content (BMC) and density where measured in a subset (n = 46...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21147
更新日期:2008-03-01 00:00:00
abstract:BACKGROUND:Numerous recent reports have described the use of recombinant factor VIIa (rFVIIa) in non-hemophilia bleeding situations for achievement of hemostasis. However, its use in clinical situations other than hemophilia patients with inhibitors has been complicated by some reports of thrombotic events. rFVIIa has ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.22160
更新日期:2009-12-01 00:00:00
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
更新日期:2020-09-01 00:00:00