Abstract:
OBJECTIVE:The objective of this single-center observational study was to determine the clinical and hematologic responses to intravenous ferric carboxymaltose (FCM) in a cohort of pediatric patients with poor response to oral iron therapy. The occurrence of adverse events was systematically recorded for up to 96 hours after infusion. STUDY DESIGN:A retrospective cohort of 144 consecutive patients aged 18 months to < 18 years with iron deficiency anemia (IDA) or iron deficiency (ID) without anemia was investigated. All patients had failed oral iron therapy. The assessments before and after FCM treatment followed a predefined protocol. RESULTS:One hundred of 117 (85 %) of patients with complete data achieved the target ferritin level ≥ 30 µg/L after a single FCM dose. Of 77 patients with IDA and complete data, 38 (49%) showed a complete hematological response within 6-12 weeks; a complete or partial response was achieved by 83%. Clinical symptoms improved in 85% of all patients. In 92% of patients (n = 133 /144), FCM infusion was uneventful. During the 96-hour follow-up, five patients reported potentially related symptoms. No serious adverse events occurred. CONCLUSION:The study confirms the safety and efficacy of FCM in children (aged 18 months and older) and adolescents unresponsive to oral therapy, in real-world experience. Single-dose FCM treatment was followed by clinical improvement with advantages of safety, compliance, and lower cost compared with previous generation parenteral iron preparations that had to be administered in fractionated sessions.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Ozsahin H,Schaeppi M,Bernimoulin M,Allard M,Guidard C,van den Ouweland Fdoi
10.1002/pbc.28614subject
Has Abstractpub_date
2020-10-01 00:00:00pages
e28614issue
10eissn
1545-5009issn
1545-5017journal_volume
67pub_type
杂志文章abstract::Germline DICER1 pathogenic variants predispose to numerous benign and malignant tumors. In this report, we describe DICER1 gene analysis in an adolescent diagnosed with multinodular goiter, ovarian Sertoli-Leydig cell tumor, and lung cyst. DICER1 mutational screening at the DNA level failed to detect any pathogenic va...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27005
更新日期:2018-06-01 00:00:00
abstract:INTRODUCTION:Understanding factors that affect the decisions of caregivers of African children to enroll their children in clinical trials would lead to more fully informed consent. METHODS:During the NOHARM study (NCT01976416), a placebo-controlled clinical trial of hydroxyurea for Ugandan children with sickle cell a...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27830
更新日期:2020-02-01 00:00:00
abstract::The Children's Oncology Group (COG) is a multi-institutional cooperative group dedicated to childhood cancer research that has helped to increase the survival of children with cancer through clinical trials. These clinical trials include a standardized regimen of imaging examinations performed prior to, during, and fo...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,实务指引
doi:10.1002/pbc.21596
更新日期:2008-08-01 00:00:00
abstract::Opportunities for cancer prevention in adolescents range from limitation of sun exposure to the use of human papillomavirus vaccines. Those who develop malignant disease experience longer waiting times for diagnosis and treatment than do children, especially when referred to adult treatment centers, and they are less ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.20634
更新日期:2006-03-01 00:00:00
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
更新日期:2016-12-01 00:00:00
abstract:BACKGROUND:The optimal dose and schedule of mycophenolate mofetil (MMF) in pediatric allogeneic stem cell transplant recipients remains to be determined. We previously reported safety and pharmacokinetics of MMF at 900 mg/m2 q6h dosing. This study was conducted to investigate the efficacy of tacrolimus plus q8h MMF dos...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.27091
更新日期:2018-08-01 00:00:00
abstract:BACKGROUND:Although weight loss and lack of linear growth occur in children with cancer, growth history is not included in research that aims to determine nutritional status in children newly diagnosed with cancer. Therefore, this study aimed to determine weight loss and lack of linear growth in this patient group. PR...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.25301
更新日期:2015-02-01 00:00:00
abstract:BACKGROUND:Conflicting data exist regarding whether low bone mineral density (BMD) is associated with Fanconi anemia (FA). The current study identified the frequency of low BMD in FA, expecting low BMD even in childhood and before HCT. PROCEDURE:Thirty-seven FA patients (18 prior HCT, 19 no prior HCT), participating i...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22956
更新日期:2011-12-01 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:BACKGROUND:Veno-occlusive disease (VOD) is a major complication following hematopoietic stem cell transplantation (HSCT). Its diagnosis is based on clinical criteria, which have a limited sensitivity. Increased plasminogen activator inhibitor-1 (PAI-1) levels have been suggested as a marker of VOD. We aimed to prospect...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23213
更新日期:2012-05-01 00:00:00
abstract::We report three cases of hemophagocytic lymphohistiocytosis (HLH) in infants within the first 6 weeks of life. Diagnosis of HLH was made early after symptoms started. All three cases were successfully treated with dexamethasone and none relapsed, indicating that not all cases of HLH in very young infants are familial....
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21758
更新日期:2009-01-01 00:00:00
abstract:BACKGROUND:Juvenile xanthogranuloma (JXG) is the most common non-Langerhans cell histiocytosis in children. The mortality and morbidity of JXG with extracutaneous lesions remain unclear. METHODS:Data of patients aged < 18 years who were diagnosed with JXG between 2001 and 2010 were retrospectively collected through a ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28381
更新日期:2020-07-01 00:00:00
abstract::Torsades de Pointes (TdP) is a life-threatening ventricular arrhythmia that can be associated with metabolic abnormalities, exposure to arrhythmogenic medications, and congenital long-QT syndrome. This report describes a patient with ALL and multiple complications of therapy who developed TdP. The patient had no evide...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20712
更新日期:2007-12-01 00:00:00
abstract:INTRODUCTION:The National Heart, Lung, and Blood Institute guidelines for sickle cell disease (SCD) pain crisis management recommend opioids within 60 minutes of emergency department (ED) registration and every 30 minutes thereafter until acute pain is managed. These guidelines are based on expert opinion without publi...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28268
更新日期:2020-09-01 00:00:00
abstract:BACKGROUND:Neonatal leukemia characterized by early stem cell origin and extramedullary infiltration in the first 4 weeks of life is rare. We analyzed the features and outcome of neonatal leukemia in Japan to establish an appropriate treatment strategy for this rare disorder. PROCEDURE:Patients with infant leukemia re...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.20599
更新日期:2006-09-01 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::Antibody therapy has become standard of care for adult B cell lymphoma patients. It is a potentially less toxic and more targeted approach for lymphoma therapy and should therefore be applied to treat pediatric B cell lymphoma patients as well. In pediatric lymphoma patients, however, clinical experience with monoclon...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.25193
更新日期:2014-12-01 00:00:00
abstract::Juvenile myelomonocytic leukemia (JMML) has a poor prognosis in general, with hematopoietic stem cell transplant (HSCT) remaining the standard of care for cure. The hypomethylating agent, azacitidine, has been used as a bridging therapy to transplant. However, no patients have been treated with azacitidine without an ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27905
更新日期:2019-10-01 00:00:00
abstract::Embryonal rhabdomyosarcoma (ERMS) is the most common childhood sarcoma and is a component of the familial pleuropulmonary blastoma (PPB)-predisposition syndrome. Using the PPB model, we hypothesized that DICER1 mutations would be found in familial and sporadic forms of ERMS. Blood samples from four children with famil...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24020
更新日期:2012-09-01 00:00:00
abstract::Risk-adapted, response-based therapies for pediatric Hodgkin lymphoma have resulted in 5-year survival exceeding 90%. Although high-dose chemotherapy and autologous hematopoietic stem cell transplantation (AHSCT) are considered standard for most patients with relapsed or refractory Hodgkin lymphoma, a subset of childr...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.24851
更新日期:2014-04-01 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
pub_type: 杂志文章
doi:10.1002/pbc.25409
更新日期:2015-05-01 00:00:00
abstract:AIM:Osteopontin (OPN) has been investigated as a biomarker for cancer and nonmalignant diseases during the last decades. Data about OPN as a potential biomarker in childhood diseases are still sparse, and reference values are not available in children. We aimed to establish reference values for children from birth to y...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28272
更新日期:2020-07-01 00:00:00
abstract::With the increasing number of long-term survivors of childhood cancer, there continues to be a critical need for development and implementation of evidence-based recommendations for clinical follow-up. In order to establish and maintain health-related follow-up guidelines, it is important to recognize the attributes o...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20609
更新日期:2006-02-01 00:00:00
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
更新日期:2019-07-01 00:00:00
abstract::Acute gastrointestinal graft-versus-host disease (GVHD) refractory to first-line treatment with systemic corticosteroids results in increased morbidity and potential mortality. We retrospectively assessed the feasibility and efficacy of catheter-directed intra-arterial platelet infusion (IAPI) in two pediatric patient...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.25633
更新日期:2015-12-01 00:00:00
abstract:BACKGROUND:Approximately one-third of patients with osteosarcoma who have a complete response to their initial treatment can be expected to relapse. It is important to define what host, tumor, or treatment characteristics determine outcome after relapse. We present findings in 59 patients treated at our institution fro...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20580
更新日期:2006-09-01 00:00:00
abstract:BACKGROUND:Survival rates for patients with bone tumors have increased dramatically over the past few decades. Unfortunately, many patients face functional limitations resulting from disease management, but there is little evidence regarding physical functioning in adolescents with bone tumors. This lack is largely due...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.20383
更新日期:2005-10-15 00:00:00
abstract:PURPOSE:This study compared the relative incidence of treatment-related toxicities and the event-free and overall survival between Hispanic and non-Hispanic children undergoing therapy for acute lymphoblastic leukemia (ALL) on Dana-Farber Cancer Institute ALL Consortium protocol 05-001. PATIENTS AND METHODS:Secondary ...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.26871
更新日期:2018-03-01 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
pub_type: 杂志文章,评审
doi:10.1002/pbc.22376
更新日期:2010-04-01 00:00:00
abstract::Melanotic neuroectodermal tumor of infancy (MNTI) is a rare tumor arising mainly in the orofacial bones. Among more than 200 cases described in articles, only seven cases involved orbital regions. We present a case of newborn with recurrent MNTI in the lateral wall of right orbit. The patient underwent complete surgic...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21607
更新日期:2008-09-01 00:00:00