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 dosing for acute graft versus host disease (GVHD) prophylaxis in a heterogeneous population of children, adolescent, young adult allogeneic stem cell transplant recipients, utilizing multiple allogeneic donor sources. PROCEDURE:GVHD prophylaxis consisted of tacrolimus 0.03-0.04 mg/kg/day intravenous continuous infusion or 0.12-0.16 mg/kg/day orally divided q8-12h and MMF 900 mg/m2 /dose (max. 1.5 g) or 15 mg/kg/dose intravenous/orally (age ≥18 years) q8h starting on Day +1. MMF was discontinued on Day +30 or Day +60 in the absence of acute GVHD. Thirty-five children, adolescents, and young adult allogeneic stem cell transplant recipients with malignant and nonmalignant disorders were enrolled. RESULTS:Kaplan-Meier probability of grade II-IV and grade III-IV acute GVHD was 22.8% (CI95 : 5.2-47.9 [where CI stands for confidence interval]) and 5.7% (CI95 : 0-48.9), respectively. Probability of extensive and limited chronic GVHD was 22.6% (CI95 : 3.4-52.2) and 12.2% (CI95 : 0.3-45.7), respectively. Probability of 1 year overall survival was 82% (CI95 : 64.1-99.8). Myeloablative conditioning was predictive of higher risk of acute GVHD in the univariate analysis (P = 0.01, hazard ratio = 6.6, CI95 : 0.91-48). CONCLUSION:This study demonstrated a low probability of acute and chronic GVHD in a diverse cohort of childhood, adolescent, and young adult allogeneic stem cell transplant recipients following MMF q8h plus tacrolimus prophylaxis.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Militano O,Ozkaynak MF,Mehta B,van deVen C,Hamby C,Cairo MSdoi
10.1002/pbc.27091subject
Has Abstractpub_date
2018-08-01 00:00:00pages
e27091issue
8eissn
1545-5009issn
1545-5017journal_volume
65pub_type
临床试验,杂志文章abstract:BACKGROUND:Infection in neutropenic children is a major cause of morbidity and mortality in children treated for cancer. In developing countries, children with cancer are often malnourished at diagnosis. In Blantyre, Malawi, children with Burkitt lymphoma are treated with a local protocol with limited toxicity. The aim...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22032
更新日期:2009-07-01 00:00:00
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
更新日期:2009-09-01 00:00:00
abstract::Malignant mesothelioma (MM) is a highly aggressive malignancy that is extremely rare in children. This case report documents a 7-year-old male without previous asbestos exposure with peritoneal MM that initially responded to chemotherapy with cisplatin and gemcitabine but ultimately metastasized to his chest. He was d...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24284
更新日期:2013-01-01 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:PURPOSE:About one-third of patients with rhabdomyosarcoma relapse despite appropriate treatment and experience a poor outcome. Little meaningful improvement in the outcome of this disease has been observed over the last 30 years. There is no clear international recommendation concerning the use of salvage chemotherapy ...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.25622
更新日期:2015-11-01 00:00:00
abstract:BACKGROUND:The identification of hemophagocytosis (HPC) in tissue or bone marrow (BM) represents only one of 5/8 criteria needed for the diagnosis of hemophagocytic lymphohistiocytosis (HLH). Yet, confirmation of HPC in bone marrow aspirates (BMA) is often relied upon to make therapeutic decisions. There is no standard...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21564
更新日期:2008-09-01 00:00:00
abstract::The Standards, Options, and Recommendations (SOR) project undertaken by the French National Federation of Cancer Centers (FNCLCC) to develop and disseminate clinical practice guidelines in oncology has now been taken over by the French National Cancer Institute. In 2007, the SOR updated the information related to the ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.21953
更新日期:2009-07-01 00:00:00
abstract::Wiskott-Aldrich syndrome (WAS) is characterized by primary immunodeficiency, thrombocytopenia and eczema. Patients with WAS have an increased risk to develop tumors. Non-Hodgkin lymphoma (NHL) represents the most common malignancy occurring in WAS-affected patients, diffuse-large-B-cell lymphoma is the most frequently...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23393
更新日期:2012-08-01 00:00:00
abstract::As a sympathetic nervous system-derived tumor, aggressive neuroblastoma (NB) is currently attracting interest from researchers seeking diagnostic and prognostic markers via less invasive procedures. The analysis of circulating tumor DNA (ctDNA) in peripheral blood can provide genetic information from multiple tumor le...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.28311
更新日期:2020-10-01 00:00:00
abstract::Familial adenomatous polyposis (FAP) due to APC mutation is associated with an increased risk of hepatoblastoma. All cases of hepatoblastoma in patients with FAP reported in the literature were reviewed. One hundred and nine patients were identified. Thirty-five patients (of 49 with data) were diagnosed with hepatobla...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.27103
更新日期:2018-08-01 00:00:00
abstract::While encapsulated bacterial agents, particularly Streptococcus pneumoniae, are recognized as important microbes that are associated with serious illness in hosts with sickle cell disease (SCD), multiple pathogens are implicated in infectious manifestations of SCD. Variations in clinical practice have been an obstacle...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.25911
更新日期:2016-05-01 00:00:00
abstract:BACKGROUND:Fever in severe chemotherapy-induced neutropenia (FN) is the most frequent manifestation of a potentially lethal complication of current intensive chemotherapy regimens. This study aimed at establishing models predicting the risk of FN, and of FN with bacteremia, in pediatric cancer patients. METHODS:In a s...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21726
更新日期:2008-12-01 00:00:00
abstract::Neuroblastoma is the most common extracranial solid tumor of childhood, and the outcomes for children with high-risk and relapsed disease remain poor. However, new international strategies for risk stratification and for treatment based on novel tumor targets and including immunotherapy are being employed in attempts ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.24116
更新日期:2012-07-01 00:00:00
abstract::A 6-year-old female was admitted with abdominal pain and a mass in the right abdomen. Her lactose dehydrogenase level was 1,200 IU/L, and neuron specific enolase was 120 ng/ml. Computed tomography scan confirmed a large right renal mass with necrosis. A right radical nephrectomy was performed. The tumor was completely...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20831
更新日期:2008-01-01 00:00:00
abstract:BACKGROUND:Griscelli syndrome type 2 (GS2), the X-linked lymphoproliferative (XLP) and the Chédiak-Higashi (CHS) syndromes are diseases that all may develop hemophagocytic syndromes. We wanted to investigate whether the treatment protocols for hemophagocytic lymphohistiocytosis (HLH) can also be used for these syndrome...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21790
更新日期:2009-02-01 00:00:00
abstract::VPS45-associated severe congenital neutropenia (SCN) is a rare disorder characterized by life-threating infections, neutropenia, neutrophil and platelet dysfunction, poor response to filgrastim, and myelofibrosis with extramedullary hematopoiesis. We present a patient with SCN due to a homozygous c.1403C>T (p.P468L) m...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26571
更新日期:2017-09-01 00:00:00
abstract::Medulloblastomas (MB) are classified in four subgroups: the well defined WNT and Sonic Hedgehog (SHH) subgroups, and the less defined groups 3 and 4. They occasionally occur in the context of a cancer predisposition syndrome. While germline APC mutations predispose to WNT MB, germline mutations in SUFU, PTCH1, and TP5...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24765
更新日期:2014-02-01 00:00:00
abstract:BACKGROUND:Distinguishing alveolar rhabdomyosarcoma (ARMS) from embryonal rhabdomyosarcoma (ERMS) has historically been of prognostic and therapeutic importance. However, classification has been complicated by shifting histologic criteria required for an ARMS diagnosis. Children's Oncology Group (COG) studies after IRS...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26645
更新日期:2017-12-01 00:00:00
abstract:BACKGROUND:Children with sickle cell disease (SCD) are at increased risk of death from invasive bacterial infections. Emergent evaluation of fever allows early treatment of potentially fatal infections. Limited data exist regarding caregiver adherence to physician recommendations of prompt medical evaluation of fever i...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.25634
更新日期:2015-11-01 00:00:00
abstract:BACKGROUND:Dexamethasone is more efficacious than prednisone in the treatment of acute lymphoblastic leukemia (ALL), but has also been associated with greater toxicity. We compared neuropsychological outcomes for patients treated on DFCI ALL Consortium Protocol 00-01, which included a randomized comparison of the two s...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,随机对照试验
doi:10.1002/pbc.24666
更新日期:2013-11-01 00:00:00
abstract:BACKGROUND:Sickle cell disease (SCD) is an autosomal recessive hemoglobinopathy associated with morbidity and mortality. We sought to understand family planning intentions of parents of young children with SCD including the awareness of three reproductive options (adoption, in vitro fertilization with egg/sperm donatio...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28227
更新日期:2020-05-01 00:00:00
abstract:BACKGROUND:The hallmark of sickle cell disease (SCD) is recurrent, painful vaso-occlusive episodes (VOC) and is the most common reason for hospitalization in SCD patients. Narcotics, particularly morphine, along with fluid hydration are standard treatments for painful episodes but have been associated with the developm...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20403
更新日期:2005-10-15 00:00:00
abstract:BACKGROUND:Clinical outcomes of children with sickle cell disease (SCD) who undergo total or partial splenectomy (PS) are poorly defined. The purpose of this retrospective study was to initiate an Internet-based registry to facilitate analysis of clinical outcomes for these children. We hypothesized that both surgical ...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.24057
更新日期:2012-07-15 00:00:00
abstract:BACKGROUND:Diamond Blackfan Anemia (DBA) is a rare congenital, bone marrow failure syndrome characterized by normochromic macrocytic anemia, reticulocytopenia and absence or insufficiency of erythroid precursors in normocellular bone marrow, frequently associated with somatic malformations. Here, we present our finding...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25183
更新日期:2014-12-01 00:00:00
abstract:BACKGROUND:Outcomes for childhood brain tumors are now associated with a five-year survival rate of 75%. Endocrine effects of brain tumors are common, occurring in 43% of patients by 10 years from tumor diagnosis. Optimal timing of screening for endocrinopathies remains undefined. We aim to identify incidence and timin...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27631
更新日期:2019-05-01 00:00:00
abstract:PURPOSE:The Children's Oncology Group conducted a phase II trial of 21-day continuous infusion topotecan to determine the response rate in pediatric patients with recurrent or refractory malignant solid tumors. PROCEDURE:Patients with Ewing sarcoma family of tumors (ESFT), osteosarcoma (OS), soft tissue sarcomas (STS)...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20739
更新日期:2006-11-01 00:00:00
abstract:BACKGROUND:The transition from the pediatric setting to adult care is a well-described period of morbidity and mortality for persons with sickle cell disease (SCD). We sought to measure the feasibility and effectiveness of providing skill-based educational handouts on improving self-management and transition readiness ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27722
更新日期:2019-07-01 00:00:00
abstract:OBJECTIVE:Current guidelines recommend high-priority treatment of severe sickle cell disease (SCD) pain with opioids; however, patients with SCD have historically been undertreated. We used mixed methods to assess pediatric residents' perceptions toward opioid use in SCD pain management. METHODS:We distributed a surve...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27535
更新日期:2019-02-01 00:00:00
abstract:BACKGROUND:The early effects of childhood acute lymphoblastic leukemia (ALL) include decreased physical function, bone mineral density (BMD/g/cm2 ), and health-related quality of life (HRQL). We assessed the capacity of a physical therapy and motivation-based intervention, beginning after diagnosis and continuing throu...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究,随机对照试验
doi:10.1002/pbc.26929
更新日期:2018-04-01 00:00:00
abstract::Evidence of the laboratory benefits of hydroxyurea and its clinical efficacy in reducing acute vaso-occlusive events in adults and children with sickle cell anemia has accumulated for more than 15 years. A definitive clinical trial showing that hydroxyurea can also prevent organ damage might support widespread use of ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.22269
更新日期:2010-02-01 00:00:00