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 in high-risk patients. Replacement of cyclophosphamide with fludarabine has reduced toxicity and nonrelapse mortality (NRM), thus improving outcomes. We analyzed long-term data of our fludarabine-based myeloablative, reduced toxicity protocol, specifically in high-risk patients. METHODS:We retrospectively analyzed a cohort of 47 consecutive patients with TM undergoing allo-SCT from matched donors, using the fludarabine-based regimen (reduced toxicity regimen). The median age of the cohort was 10 years. Thirty-eight patients (80%) were in the high-risk and nine patients (20%) were in the low-risk category. The primary aim of this analysis was thalassemia-free survival (TFS). RESULTS:The rejection rate was 11% within high-risk patients with NRM of 2%. With a median follow-up period of 7 years (1-15 years), the 10-year TFS in the entire cohort was 87%, and the overall survival (OS) was 97%. The 10-year TFS and OS among the low-risk and high-risk groups were 90% versus 84%, respectively (P = 0.45) and 100% versus 96%, respectively (P = 0.5), and both subsets of patients did equally well. CONCLUSION:In conclusion, replacement of high-dose cyclophosphamide with fludarabine is well tolerated with minimal regimen-related toxicity and acceptable rejection rates, especially in high-risk patients.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Sheth V,Grisariu S,Avni B,Stepensky P,Ashkenazi M,Shapira MY,Or Rdoi
10.1002/pbc.27312subject
Has Abstractpub_date
2018-11-01 00:00:00pages
e27312issue
11eissn
1545-5009issn
1545-5017journal_volume
65pub_type
杂志文章abstract:BACKGROUND:Insomnia is common among adolescent and young adult (AYA) cancer survivors. Cognitive-behavioral therapy for insomnia (CBT-I) is considered the gold standard treatment. Standard CBT-I was designed for adults and not adapted to the unique medical, psychosocial, and developmental needs of AYA cancer survivors,...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28506
更新日期:2020-09-01 00:00:00
abstract::The commonly accepted definition of Fanconi anemia (FA) relying on DNA repair deficiency is submitted to a critical review starting from the early reports pointing to mitomycin C bioactivation and to the toxicity mechanisms of diepoxybutane and a group of nitrogen mustards causing DNA crosslinks in FA cells. A critica...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.25452
更新日期:2015-07-01 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:Symptoms of epidural compression (SEC) in children with neuroblastoma (particularly infants) may be misinterpreted, leading to delay in diagnosis. PATIENTS AND METHODS:Clinical, imaging and follow-up data of 34 infants with neuroblastoma and SEC diagnosed between 2000 and 2011 at Italian AIEOP centers were ...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.25028
更新日期:2014-08-01 00:00:00
abstract::Juvenile xanthogranuloma (JXG) is a disorder of disputed origin thought to be related to the dermal/interstitial macrophage. A 5-year-old female presented with an aggressive systemic JXG that developed 5 months after the diagnosis of T-cell acute lymphoblastic leukemia (T-ALL). Examination of the T-cell receptor gamma...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22756
更新日期:2011-05-01 00:00:00
abstract:BACKGROUND:Osteopenia has been reported in children surviving acute lymphoblastic leukemia and brain tumors, apparently as a consequence of therapy. It has been suggested that cranial irradiation may play a role in the development of this complication. In order to explore that possibility, we examined survivors of brai...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20512
更新日期:2006-03-01 00:00:00
abstract::Familial hemophagocytic lymphohistiocytosis (FHLH) is an autosomal recessive disorder of cytotoxic cell function that results in abnormal proliferation of benign lymphocytes and histiocytes in response to infectious stimuli. FHLH generally occurs in very young children, and typically presents with fever, cytopenias, c...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21438
更新日期:2008-05-01 00:00:00
abstract:BACKGROUND:Medication errors occur universally. Inappropriate administration of chemotherapy drugs can have adverse effects in cancer patients. Our objective was to assess the rate and type of medication errors in children with acute lymphoblastic leukemia (ALL) receiving oral chemotherapy in outpatient setting. PROCE...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25203
更新日期:2014-12-01 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:Although intra-retinal tumor has long been staged presurgically according to the Reese-Ellsworth (R-E) system, retinoblastoma differs from other pediatric neoplasms in never having had a widely accepted classification system that encompasses the entire spectrum of the disease. Comparisons among studies that ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.20606
更新日期:2006-11-01 00:00:00
abstract:BACKGROUND:Drug shortages require clinical teams to decide how to allocate drugs in limited supply among their patients. Ethical frameworks are invaluable for promoting rational approaches to drug distribution, but gaps remain between ethical theory and clinical application. The goal of this work was to explore how dec...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26331
更新日期:2017-07-01 00:00:00
abstract:BACKGROUND:Pain is the most common complication of sickle cell disease requiring emergency department (ED) visits and hospitalization. A Clinical Practice Guideline (CPG) to manage acute sickle cell pain offers clinicians a standardized approach for the provision of evidence-based, cost-effective care. After CPG implem...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21847
更新日期:2009-03-01 00:00:00
abstract::We aimed to provide recommendations on the infusion duration of anthracycline chemotherapy agents in children with cancer. This study also serves as a practice example of the essential steps that need to be taken when using a previously published systematic review to develop a high-quality clinical practice guideline....
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,实务指引,评审
doi:10.1002/pbc.26867
更新日期:2018-02-01 00:00:00
abstract::For many children with cancer, participation in oncology camp programs is an important component of healing that offers opportunities for fun and can have substantial impacts on social and physical well-being. Optimal medical care and infectious screening for children attending oncology camp is critical to maximize sa...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28107
更新日期:2020-03-01 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
pub_type: 杂志文章
doi:10.1002/pbc.22102
更新日期:2009-10-01 00:00:00
abstract:BACKGROUND:Clinical characteristics and optimal treatment strategies for spindle cell/sclerosing rhabdomyosarcoma (ssRMS) have not been well established because of its rarity. PROCEDURE:Retrospective re-evaluation of sarcoma specimens (1997-2014) identified 16 ssRMSs (median age 20 years, range 7-39 years). Clinicopat...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25367
更新日期:2015-06-01 00:00:00
abstract:BACKGROUND:Extracranial malignant rhabdoid tumor (MRT) is a rare pediatric cancer with a poor prognosis. The kidney is the most common site. Isolated reports have shown improvements in patient survival, but no specific treatment regimen has shown efficacy over others. PROCEDURE:Retrospective review of patients diagnos...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.25093
更新日期:2014-08-01 00:00:00
abstract:BACKGROUND:In low- and middle-income countries, therapeutic options for advanced, refractory, or relapsing malignancies are limited due to local constraints such as cost of drugs, distance from oncology centers, and lack of availability of new anticancer drugs. Metronomics, which combines metronomic chemotherapy (MC) a...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28508
更新日期:2020-09-01 00:00:00
abstract::A case of invasive, keratinizing squamous cell carcinoma of the larynx in an 8-year-old female treated with laryngectomy is presented. Perinatal exposure to human papilloma virus and constitutional heterozygosity for a FANCC mutation were identified, though FANCC heterozygosity is not known to be cancer predisposing. ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26463
更新日期:2017-08-01 00:00:00
abstract:BACKGROUND:Better predictors of outcome would allow improved risk-adapted therapy for pediatric nonmetastatic osteosarcoma of the extremity. We investigated the predictive value of MR imaging-based measures of absolute and relative tumor size and volume at the time of diagnosis. We also assessed the relation of tumor s...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20077
更新日期:2004-12-01 00:00:00
abstract:INTRODUCTION:To describe the quality of life (QOL) of pediatric brain tumor survivors (PBTSs) prospectively and to identify potential medical, personal and family contextual factors associated with QOL. METHODS:Ninety-one PBTSs (8-16 years) who were off treatment and attending a regular classroom participated. Self- a...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26481
更新日期:2017-09-01 00:00:00
abstract:BACKGROUND:Low expression of HLA class II antigens has been associated with more aggressive disease in several human malignancies including adult adrenocortical tumors (ACT), but their clinical relevance in pediatric ACT needs to be investigated. PROCEDURE:This study analyzed the expression profile of three class II h...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25118
更新日期:2014-11-01 00:00:00
abstract::Infliximab-daclizumab was used to treat acute and chronic liver and gut graft-versus-host disease (GVHD) in two children after standard immunosuppressive therapy failed. Infliximab (10 mg/kg weekly, 4 doses) and daclizumab (1 mg/kg, days 1, 4, 8, 15, and 22) were given over 1 month. In case 1, grade 2 chronic GVHD of ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.20648
更新日期:2007-08-01 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
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.20132
更新日期:2004-12-01 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
pub_type: 杂志文章
doi:10.1002/pbc.25533
更新日期:2015-09-01 00:00:00
abstract:BACKGROUND:Advanced irradiation techniques, including intensity-modulated radiation therapy (IMRT), aim to limit irradiation to adjoining tissues by conforming beams to a well-defined volume. In intracranial germinomas, whole-ventricular IMRT decreases the volume of irradiation to surrounding parenchyma. This study exa...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28005
更新日期:2019-12-01 00:00:00
abstract:BACKGROUND:Ultrasound-guided (USG) cannulation of the brachiocephalic vein (BCV) is gaining worldwide consensus for central venous access in children. This study reports a 20-month experience with this approach in children. METHODS:All patients who underwent percutaneous USG central venous catheter (CVC) positioning i...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26202
更新日期:2017-02-01 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::Clinically apparent jaundice is unusual in patients with beta-thalassemia major. Co-inheritance of Gilbert syndrome has been reported to cause hyperbilirubinemia in these subjects. Crigler-Najjar syndrome is another rare disorder of bilirubin metabolism caused by mutation in the gene coding the enzyme UGT1A1. We repor...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22313
更新日期:2010-04-01 00:00:00
abstract:BACKGROUND:Few data exist regarding the clinical outcome of patients with Ewing sarcoma (EWS) treated with pencil beam scanning proton therapy (PT). We report the outcome of children, adolescents and young adults (AYA) treated with PT at the Paul Scherrer Institute. MATERIALS:Thirty-eight patients (median age, 9.9 yea...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26688
更新日期:2017-12-01 00:00:00