Abstract:
BACKGROUND:Patients with sickle cell disease (SCD) receiving chronic blood transfusions are at risk of developing iron overload and organ toxicity. Chelation therapy with either subcutaneous (SQ) desferrioxamine (DFO) or oral deferasirox is effective in preventing and reducing iron overload but poses significant challenges with patient compliance. Intravenous (IV) infusions of high dose DFO have been utilized in non-compliant patients with heavy iron overload in small case series. PROCEDURE:We review our experience of high dose IV DFO in 27 patients with SCD who had significant iron overload and were noncompliant with subcutaneous (SQ) DFO. All patients were treated in-hospital with DFO 15 mg/kg/hr IV for 48 hr every 2-4 weeks with a mean duration of 19.6 months. RESULTS:We observed a significant decrease in liver iron burden with high dose intermittent IV DFO. Histological examination of liver biopsies revealed a decrease in the grade of liver iron storage. Also there was significant improvement in liver enzymes (ALT, AST) after high dose IV DFO. No audiologic or ophthalmologic toxicity or acute or chronic pulmonary complications were observed. CONCLUSIONS:In our cohort of patients with SCD we observed a significant decrease in liver iron burden with high dose IV DFO. Our patients tolerated the therapy well without any major toxicity. This regimen is safe and may be an option for poorly compliant patients with significant iron overload.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Kalpatthi R,Peters B,Kane I,Holloman D,Rackoff E,Disco D,Jackson S,Laver JH,Abboud MRdoi
10.1002/pbc.22660subject
Has Abstractpub_date
2010-12-15 00:00:00pages
1338-42issue
7eissn
1545-5009issn
1545-5017journal_volume
55pub_type
杂志文章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:Progressive/recurrent high-grade and diffuse intrinsic pontine gliomas (DIPGs) are fatal. Treatments targeting molecular pathways critical for these cancers are needed. METHODS:We conducted a phase 1 study (rolling-six design) to establish the safety and maximum tolerated dose (MTD) of dasatinib, an oral pl...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27035
更新日期:2018-07-01 00:00:00
abstract:BACKGROUND:There are no tests to identify critically ill children at high risk of deep venous thrombosis (DVT). In this exploratory study, we aimed to identify proteins that are associated with incident DVT in critically ill adolescents. PROCEDURE:Plasma samples were obtained from critically ill adolescents within 24 ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.28159
更新日期:2020-04-01 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:Patient transition readiness self-assessment tools and investigation into patient and parent perceptions of the transition process from pediatric to adult care models have informed recognition of gaps in care, particularly for those with chronic disease. Implementation of a longitudinal transition process wi...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25807
更新日期:2016-03-01 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::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
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:Pegasparaginase (PEG) is important for treatment of Acute Lymphoblastic Leukemia (ALL). Despite conjugation to polyethylene glycol to reduce immunogenicity, allergic reactions still occur and may be severe. Traditionally, PEG is given via intramuscular (IM) injection but recent protocols have shown it can be...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23380
更新日期:2012-09-01 00:00:00
abstract:BACKGROUND:Glucocorticoids and asparaginase, used to treat acute lymphoblastic leukemia (ALL), can cause hypertriglyceridemia. We compared triglyceride levels, risk factors, and associated toxicities in two ALL trials at St. Jude Children's Research Hospital with identical glucocorticoid regimens, but different asparag...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28040
更新日期:2020-01-01 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:We report on the treatment of children and adolescents with acute lymphoblastic leukemia (ALL) in first relapse. The protocol focused on: (1) Intensive chemotherapy preceding allogeneic stem cell transplantation (SCT) in early bone marrow relapse; (2) Rotational chemotherapy in late relapse, without donor; (...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.22946
更新日期:2011-08-01 00:00:00
abstract:BACKGROUND:The aim was to evaluate self-reported reproductive characteristics and markers of ovarian function in a nationwide cohort of female survivors of childhood acute lymphoblastic leukemia (ALL), because prior investigations have produced conflicting data. PROCEDURE:Self-reported reproductive characteristics wer...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28894
更新日期:2021-01-18 00:00:00
abstract::Ependymoblastoma (EBL) is a rare malignant CNS tumor of early childhood, listed as a subgroup of primitive neuroectodermal tumors (PNET) in the 2007 WHO Classification of Tumours of the Central Nervous System. Histologically, EBL can be defined by multilayered, mitotically active "ependymoblastic" rosettes with centra...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24915
更新日期:2014-06-01 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::Osteosarcoma is the most common bone tumor in children and young adults, with few advances in survival and treatment, especially for metastatic disease, in the last 30 years. Recently, immunotherapy has begun to show promise in various adult cancers, but the utility of this approach for osteosarcoma remains relatively...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.27227
更新日期:2018-09-01 00:00:00
abstract:BACKGROUND:A lack of access to methotrexate levels is common in low- and middle-income countries (LMIC), relevant for 80% of children with cancer worldwide. We evaluated whether high-dose methotrexate (HD-MTX) can be administered safely with extended hydration and leucovorin rescue, with monitoring of serum creatinine ...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.27241
更新日期:2018-12-01 00:00:00
abstract:BACKGROUND:The death of a child is a devastating event that results in profound grief and significant psychosocial and physical morbidities in parents. The parental grief journey is a complex phenomenon necessitating the utilization of newer models of bereavement with a focus on relationships and exploration of parents...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26046
更新日期:2016-09-01 00:00:00
abstract:BACKGROUND:Pediatric papillary thyroid carcinoma (PTC) is clinically and biologically distinct from adult PTC. We sequenced a cohort of clinically annotated pediatric PTC cases enriched for high-risk tumors to identify genetic alterations of relevance for diagnosis and therapy. METHODS:Tumor DNA and RNA were extracted...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28741
更新日期:2021-01-01 00:00:00
abstract:BACKGROUND:To examine the practical applicability, internal consistency, and validity of the Bt-DUX, a disease-specific Health Related Quality of Life (HRQoL) instrument. The Bt-Dux was developed to examine patients' individual values of their life after a malignant bone tumor of the lower extremity at four domains (co...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.22078
更新日期:2009-09-01 00:00:00
abstract:BACKGROUND:We report a 13-year-old male with Diamond Blackfan anemia and short stature. He had a normal biochemical response to growth hormone (GH) stimulation, but his bone age was delayed, his insulin-like growth factor 1 (IGF-1) was low, and he had a poor growth velocity. He was started on daily GH injections. METH...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20075
更新日期:2004-10-01 00:00:00
abstract::Congenital hypotransferrinemia (OMIM 209300) is an extremely rare disorder of inherited iron metabolism. Since its description in 1961, only 16 cases have been reported. The defective gene and molecular defect causing this disorder and clinicolaboratory findings seen in the homozygous and heterozygous states have been...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26789
更新日期:2018-02-01 00:00:00
abstract:BACKGROUND:Cisplatin (CDDP) ototoxicity is a significant side effect of the current treatment of medulloblastoma (MB). Cumulative dose of CDDP and age are recognized risk factors for hearing loss, but inter-individual susceptibility limits our ability to identify patients at risk for hearing loss. We describe the kinet...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24307
更新日期:2013-02-01 00:00:00
abstract::Langerhans cell histiocytosis is known to mimic many other conditions. We present two patients where anti-tubercular therapy was instituted when clinical and radiological features suggested tuberculosis. The correct diagnosis of histiocytosis was reached only on further work-up including immunohistochemistry following...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21930
更新日期:2009-07-01 00:00:00
abstract:BACKGROUND:As pegylated asparaginase is becoming the preferred first-line asparaginase preparation in the chemotherapy regimens of childhood acute lymphoblastic leukemia (ALL), there is a need to evaluate this treatment. METHODS:The aim of this study was to evaluate the pharmacokinetics of prolonged upfront biweekly P...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,随机对照试验
doi:10.1002/pbc.26686
更新日期:2017-12-01 00:00:00
abstract::Immune-mediated hemolytic anemia is a well-recognized complication of hematopoietic stem cell transplantation. We report on a 6-year-old boy with X-linked adrenoleukodystrophy who developed severe delayed alloimmune hemolytic anemia associated with immune-mediated neutropenia and thrombocytopenia following major ABO i...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21187
更新日期:2008-04-01 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::Primary choriocarcinoma of the liver is an extremely rare childhood malignancy frequently associated with clinical instability at initial presentation. It often mimics other benign and malignant childhood liver tumors. Prompt diagnosis and initiation of treatment are necessary to attain a successful outcome. We descri...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20623
更新日期:2007-07-01 00:00:00
abstract:BACKGROUND:Hepatic veno-occlusive disease (VOD) is one of the most serious complications in stem cell transplantation (SCT). Although plasma protein C activity decreases in VOD after SCT, the timeframe of plasma protein C activity decreases during SCT is not known. PROCEDURE:We examined levels of plasma protein C seri...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.22314
更新日期:2010-03-01 00:00:00
abstract:BACKGROUND:Most relapses from Wilms' tumor occur within 2 years from diagnosis. This study aims to describe the incidence and outcome of patients who experienced a late recurrence (LR) more than 5 years after diagnosis across several clinical trials, and to develop evidence-based recommendations for follow-up surveilla...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.24604
更新日期:2013-10-01 00:00:00