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 Registry (DBAR) of North America to assess the prevalence of various endocrinopathies. PROCEDURE:In a pilot study, retrospective data were collected for 12 patients with DBA. Subsequently, patients with DBA aged 1-39 years were recruited prospectively. Combined, 57 patients were studied; 38 chronically transfused, 12 glucocorticoid-dependent, and seven in remission. Data were collected on anthropometric measurements, systematic screening of pituitary, thyroid, parathyroid, adrenal, pancreatic, and gonadal function, and ferritin levels. Descriptive statistics were tabulated and group differences were assessed. RESULTS:Fifty-three percent of patients had ≥ 1 endocrine disorder, including adrenal insufficiency (32%), hypogonadism (29%), hypothyroidism (14%), growth hormone dysfunction (7%), diabetes mellitus (2%), and/or diabetes insipidus (2%). Ten of the 33 patients with available heights had height standard deviation less than -2. Low 25-hydroxy vitamin D (25(OH)D) levels were present in 50%. A small proportion also had osteopenia, osteoporosis, or hypercalciuria. Most with adrenal insufficiency were glucocorticoid dependent; other endocrinopathies were more common in chronically transfused patients. CONCLUSIONS:Endocrine dysfunction is common in DBA, as early as the teenage years. Although prevalence is highest in transfused patients, patients taking glucocorticoids or in remission also have endocrine dysfunction. Longitudinal studies are needed to better understand the etiology and true prevalence of these disorders.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Lahoti A,Harris YT,Speiser PW,Atsidaftos E,Lipton JM,Vlachos Adoi
10.1002/pbc.25780subject
Has Abstractpub_date
2016-02-01 00:00:00pages
306-12issue
2eissn
1545-5009issn
1545-5017journal_volume
63pub_type
杂志文章abstract:OBJECTIVE:To develop an objective tool for assessing disease activity in patients with Langerhans cell histiocytosis (LCH). METHOD:Scoring system was developed and applied to a database containing information on 612 patients. RESULTS:At diagnosis, the score distribution was highly asymmetrical: the score was between ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20160
更新日期:2004-12-01 00:00:00
abstract::Pediatric cancer programs in low-income countries (LIC) can improve outcomes. However, treatment must be tailored to the patient's living conditions and the availability of supportive care. In some cases, a more intense regimen will decrease survival since the increase in death from toxicity may exceed any decrease in...
journal_title:Pediatric blood & cancer
pub_type:
doi:10.1002/pbc.20989
更新日期:2007-04-01 00:00:00
abstract:BACKGROUND:Influenza is a health risk to children receiving chemotherapy for cancer. An absolute lymphocyte count (ALC) >1,000 cells/mm(3) has been associated with the ability to produce an immune response to influenza vaccine during chemotherapy. However, clinical efficacy of influenza vaccination during chemotherapy ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26110
更新日期:2016-11-01 00:00:00
abstract:BACKGROUND:Little is known about the visual outcome of children affected by an optic pathway glioma (OPG). PROCEDURES:We evaluated the long-term visual outcome of 32 consecutive children affected by OPG without neurofibromatosis type-1 referred to the Pediatric Department of Padua University and managed according to s...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.22748
更新日期:2010-12-01 00:00:00
abstract::In the present study, DNA sequencing of the genes SRGN, ARF6, AP3B1, and SH2D1A was performed in a well defined cohort of 18 families with familial hemophagocytic lymphohistiocytosis (FHL). A heterozygous nucleotide change (C > T) in the 3'untranslated region of the SRGN gene and a monoallelic 3-base pair deletion (c....
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21428
更新日期:2008-05-01 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
pub_type: 杂志文章
doi:10.1002/pbc.25712
更新日期:2016-01-01 00:00:00
abstract::Thrombocytopenia and autoimmune hemolytic anemia (Evans syndrome) with the presence of both warm and cold autoantibodies (mixed type) are rare in the pediatric age group. This condition may be associated with other autoimmune disorders and is notoriously difficult to treat. This case describes an adolescent male who p...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20312
更新日期:2005-09-01 00:00:00
abstract::Acute lymphoblastic leukemia (ALL) is the most common type of pediatric neoplasia. Highly heterogeneous, ALL includes several genetic subtypes with varying clinical outcome. Although, some features are well established as prognostic predictors, the details of the molecular mechanisms underlying different phenotypes ar...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.24167
更新日期:2012-10-01 00:00:00
abstract:BACKGROUND:More than 60% of children with acute myeloid leukemia (AML) become long-term survivors. Most are cured using chemotherapy without hematopoietic stem cell transplantation (HSCT). We report on pubertal development and compare self-reported parenthood among AML survivors and their siblings. PROCEDURE:We includ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24715
更新日期:2013-12-01 00:00:00
abstract::Progress has been made in resource-limited countries in treating acute lymphoblastic leukemia, but advances in solid malignancies have been slower. Multidisciplinary care coordination is challenging, assessing adherence to guidelines through quality improvement initiatives is essential. We characterized deviations fro...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26480
更新日期:2017-08-01 00:00:00
abstract:BACKGROUND:Patients with sickle cell anemia (SCA) experience a broad range of psychiatric disorders, placing them at risk for more complicated and longer hospitalizations for vaso-occlusive crises (VOC). The current study examined the frequency of psychiatric disorders in SCA patients (ages birth to 20 years) admitted ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23117
更新日期:2012-02-01 00:00:00
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
更新日期:2011-02-01 00:00:00
abstract:BACKGROUND:Physical fitness is an important determinant of quality of life (QOL) after hematopoietic stem cell transplantation. Cardiac function can influence exercise performance. The aim of this study was to assess these factors and their interrelationship. PROCEDURE:Children underwent cardiopulmonary exercise testi...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27499
更新日期:2019-01-01 00:00:00
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
更新日期:2014-02-01 00:00:00
abstract::Natural killer (NK) cell lymphoma of lung is very rare. We report a case of NK cell lymphoma presented in a 17-year-old male with bilateral pleural effusions and hemophagocytic lymphohistocytosis. Morphologic and immunohistochemical tests revealed an association of NK cell lymphoma with Epstein-Barr virus. A literatur...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21943
更新日期:2009-05-01 00:00:00
abstract::In Mexico, childhood cancer incidence and mortality have increased in the last decade. Through government actions since 2005, the Popular Medical Insurance (PMI) program for childhood cancer was created. The objective of PMI was to offer early cancer diagnosis, standardized treatment regimens, and numerous pediatric o...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.26366
更新日期:2017-06-01 00:00:00
abstract::Beckwith-Wiedemann syndrome (BWS) is a genetic overgrowth and cancer predisposition syndrome, associated with both benign and malignant adrenal findings. Literature review and an institutional case series elucidate the wide spectrum of adrenal findings in BWS patients. The altered expression of the 11p15 region is lik...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.26432
更新日期:2017-08-01 00:00:00
abstract::Minimal residual disease (MRD) is an independent predictor of relapse risk in children with leukemia and is widely used for risk-adapted treatment. This article summarizes current evidence supporting the use of MRD, including clinical significance, current international clinical practice, impact statement, and recomme...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,实务指引,评审
doi:10.1002/pbc.25939
更新日期:2016-06-01 00:00:00
abstract::Social media are powerful means of communication that can also have an important role in the healthcare sector. They are sometimes seen with diffidence in the healthcare setting, partly because they risk blurring professional boundaries. This issue is particularly relevant to relations between caregivers and adolescen...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26958
更新日期:2018-05-01 00:00:00
abstract:BACKGROUND:Outcomes for patients with oncologic disease and/or after hematopoietic stem cell transplant (HSCT) requiring intensive care unit admission have improved, but indications for and outcomes after extracorporeal membrane oxygenation (ECMO) support in this population are poorly characterized. PROCEDURE:We analy...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.28403
更新日期:2020-10-01 00:00:00
abstract:BACKGROUND:Neuroblastoma (NB) is cytogenetically characterized by a number of non-random events. However, knowledge is limited concerning the timing of occurrence and inter-action of many of these events. METHODS:Karyotypic patterns were obtained from a study group of 49 NB tumors that had been analyzed by conventiona...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20179
更新日期:2005-02-01 00:00:00
abstract::A 4-year-old male presented with rapid-onset cranial nerve palsy and ataxia. Brain magnetic resonance imaging (MRI) revealed a pontine mass lesion with discordant conventional and advanced imaging. A stereotactic core biopsy revealed glioblastoma with immunostaining suggestive of histone H3K27M and TP53 mutation, cons...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26416
更新日期:2017-08-01 00:00:00
abstract:BACKGROUND:The goals of this study included: (1) Identification of factors prognostic for event-free survival (EFS) and overall survival (OS), and (2) Definition of risk groups for risk adapted therapy in children with Hodgkin disease (HD). PROCEDURE:From 1991 to 2003, 69 children with newly diagnosed, untreated biops...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20487
更新日期:2005-10-15 00:00:00
abstract:BACKGROUND:Deaths during induction chemotherapy for pediatric acute lymphoblastic leukemia (ALL) account for one-tenth of ALL-associated mortality and half of ALL treatment-related mortality. We sought to ascertain patient- and hospital-level factors associated with induction mortality. PROCEDURE:We performed a retros...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24855
更新日期:2014-05-01 00:00:00
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
更新日期:2010-07-01 00:00:00
abstract:BACKGROUND:The efficacy of combination chemotherapy with methotrexate (MTX) and asparaginase is not well known in relapsed and refractory acute leukemia after contemporary therapy. PROCEDURE:A retrospective study of pediatric patients with relapsed or refractory acute myeloid leukemia (AML) who received MTX and aspara...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24470
更新日期:2013-07-01 00:00:00
abstract:BACKGROUND:We sought to determine the risk of bacteremia in a cohort of outpatient febrile nonneutropenic pediatric oncology patients and to assess clinical characteristics that may influence decisions regarding empiric antibiotic therapy. PROCEDURE:A single institution retrospective cohort study was performed of outp...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22264
更新日期:2010-01-01 00:00:00
abstract::We report the case of a drug interaction between methotrexate (MTX) and chloral hydrate (CH) observed in a child treated for acute leukemia. Significantly slower MTX clearance and increased MTX exposure occurred on the first three courses of a high-dose chemotherapy when co-administered with CH despite normal renal fu...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24393
更新日期:2013-03-01 00:00:00
abstract::Non-Hodgkin lymphoma (NHL) is a heterogeneous group of lymphoid malignancies with high incidence in adolescents and young adults (AYAs). The most common diseases include diffuse large B-cell lymphoma, anaplastic large cell lymphoma, Burkitt lymphoma, lymphoblastic lymphoma, and primary mediastinal large B-cell lymphom...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.27073
更新日期:2018-08-01 00:00:00
abstract:BACKGROUND:Osteopathy is one of the common initial symptoms of acute lymphocytic leukemia (ALL) in children and adolescents, but multiple osteolysis accompanied by hypercalcemia is rarely observed. PROCEDURE:We treated a 14-year-old female who had multiple osteolytic lesions and hypercalcemia at initial onset of ALL. ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.20357
更新日期:2005-09-01 00:00:00