Abstract:
BACKGROUND:Prior reviews of phase I pediatric oncology trials involving primarily cytotoxic agents have reported objective response rates (ORRs) and toxic death rates of 7.9-9.6% and 0.5%, respectively. These data may not reflect safety and efficacy in phase I trials of molecularly targeted (targeted) drugs. METHODS:A systematic review of pediatric phase I solid tumor trials published in 1990-2013 was performed. The published reports were evaluated for patient characteristics, toxicity information, and response numbers. RESULTS:A total of 143 phase I pediatric clinical trials enrolling 3,896 children involving 53 targeted and 48 cytotoxic drugs were identified. A meta-analysis demonstrated that the ORR is 2.1-fold higher with cytotoxic drugs (0.066 vs. 0.031 per subject; P = 0.007). By contrast, the pooled estimate of the stable disease rate (SDR) is similar for cytotoxic and targeted drugs (0.2 vs. 0.23 per subject; P = 0.27). The pooled estimate of the dose-limiting toxicity rate is 1.8-fold larger with cytotoxic drugs (0.24 vs. 0.13 per subject; P = 0.0003). The hematologic grade 3-4 (G3/4) toxicity rate is 3.6-fold larger with cytotoxic drugs (0.43 vs. 0.12 per treatment course; P = 0.0001); however, the nonhematologic G3/4 toxicities and toxic deaths occur at similar rates for cytotoxic and targeted drugs. CONCLUSIONS:In phase I pediatric solid tumor trials, ORRs were significantly higher for cytotoxic versus targeted agents. SDRs were similar in targeted and cytotoxic drug trials. Patients treated with cytotoxic agents were more likely to experience hematologic G3/4 toxicities than those patients receiving targeted drugs.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Dorris K,Liu C,Li D,Hummel TR,Wang X,Perentesis J,Kim MO,Fouladi Mdoi
10.1002/pbc.26258subject
Has Abstractpub_date
2017-03-01 00:00:00issue
3eissn
1545-5009issn
1545-5017journal_volume
64pub_type
杂志文章,meta分析abstract::Hyperglycemia during induction chemotherapy for childhood acute lymphoblastic leukemia (ALL) has been inconsistently associated with risk of infection. We investigated the incidence of hyperglycemia during induction for childhood ALL in a retrospective cohort study of 144 patients treated on a single national protocol...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24689
更新日期:2013-12-01 00:00:00
abstract::Both hepatoblastoma and hypoplastic kidneys are rare in children. A review of all patients with hepatoblastoma treated at our institution between 1993 and 2011 revealed three cases of hepatoblastoma occurring in children with hypoplastic kidneys and significantly impaired renal function. Two patients were treated with...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24936
更新日期:2014-08-01 00:00:00
abstract::X-linked lymphoproliferative syndrome (XLP) is caused by mutations in SH2D1A, and is associated with overwhelming infectious mononucleosis, aplastic anemia, hypogammaglobulinemia, and B-cell lymphomas. However, the frequency of SH2D1A mutations in males who present with B NHL is unknown. Five cases of XLP were diagnos...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24525
更新日期:2013-09-01 00:00:00
abstract:BACKGROUND:The clinical impact of influenza in children undergoing therapy for cancer is not well-described in the literature. PROCEDURE:Laboratory-documented influenza infection in pediatric oncology patients cared for in a single regional pediatric medical center between July 2000 and June 2005 was identified by rev...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.21472
更新日期:2008-05-01 00:00:00
abstract::Almost all of what is known about neurologic and cognitive development in hemophilia derives from the Hemophilia Growth and Development Study, conducted during an era when treatment regimens and comorbidities differed significantly from the current environment. Results suggested hemophilia and human immunodeficiency v...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.28004
更新日期:2020-01-01 00:00:00
abstract::A male patient diagnosed with severe congenital protein C (PC) deficiency during the neonatal period was treated with long-term warfarin but frequently developed purpura fulminans and bleeding. At four years of age, edoxaban was initiated (direct oral anticoagulant [DOAC]). His d-dimer and fibrin/fibrinogen degradatio...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27686
更新日期:2019-06-01 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: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:Primary mediastinal germ cell tumors (GCTs) are rare in children and still represent a challenge for both adult and pediatric oncologists because of their worse outcome compared to their gonadal counterpart. PROCEDURE:Prospectively collected data concerning patients enrolled in the Italian Association of Pe...
journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/pbc.25895
更新日期:2016-05-01 00:00:00
abstract:BACKGROUND:Treatment abandonment (TxA) is a primary cause of therapy failure in children with cancer in low-/middle-income countries. We explored the absence of social support network (SSN), among other predictive factors, and TxA in children with cancer in Cali, Colombia. PROCEDURE:In this prospective cohort study, w...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25919
更新日期:2016-05-01 00:00:00
abstract:BACKGROUND:Nephrogenic rests (NRs) are abnormally persistent foci of embryonal cells, thought to be the precursor lesion of Wilms tumors (WTs). To date, their presence has not been systematically examined in WTs treated with preoperative chemotherapy. METHODS:A systematic analysis of the data on NRs in WTs treated wit...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.26547
更新日期:2017-11-01 00:00:00
abstract:BACKGROUND:(131) I-metaiodobenzylguanidine ((131) I-MIBG) is a targeted radiopharmaceutical for patients with neuroblastoma. Despite its tumor-specific uptake, the treatment with (131) I-MIBG results in whole-body radiation exposure. Our aim was to correlate whole-body radiation dose (WBD) from (131) I-MIBG with tumor ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25816
更新日期:2016-03-01 00:00:00
abstract::Acute immune thrombocytopenic purpura (ITP) occurs most commonly in young children who present with severe isolated thrombocytopenia and purpura. A marrow examination is not required unless glucocorticoids are used, lest treatment mask incipient acute lymphoblastic leukemia, but controversy exists here. The recommenda...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.21934
更新日期:2009-09-01 00:00:00
abstract::Survivors of childhood cancer are at risk for dental late effects. This systematic review summarizes associations between treatment exposures and dental late effects among survivors of childhood cancer. We included investigations with at least 20 study participants conducted for 2 or more years after completion of chi...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.24842
更新日期:2014-03-01 00:00:00
abstract:BACKGROUND:In Germany, about 1,800 new cases of pediatric cancer under 15 years of age are diagnosed each year and survival rates approach 80%. Although treatment is covered by health insurance and is thus available for all patients at no cost, treatment refusal and treatment discontinuation have been observed. However...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26111
更新日期:2016-10-01 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::High-risk (HR) acute lymphoblastic leukemia (ALL) remains one of the greatest challenges in pediatric oncology. Relapsed ALL is a leading cause of death in young people, and further improvements in outcome will required the development of therapeutic approaches directed against rational therapeutic targets, as escalat...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
doi:10.1002/pbc.22996
更新日期:2011-06-01 00:00:00
abstract::High dose acetaminophen (HDAC) with N-acetylcysteine (NAC) has been effective in adults with advanced malignancies. We report HDAC with NAC in a child with progressive hepatoblastoma, confirmed at biopsy of an unresectable hepatic mass. Alpha-fetoprotein (AFP) increased despite four courses of doxorubicin and one cour...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20330
更新日期:2005-08-01 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::Peripheral neuropathy is a well-known side effect of vincristine, a micro-tubule inhibitor commonly used to treat malignancies. Severe neurologic adverse events can occur in patients with Charcot-Marie-Tooth disease (CMT) treated with vincristine. Voriconazole is an antifungal agent used increasingly in children with ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.21786
更新日期:2009-02-01 00:00:00
abstract:PURPOSE:To assess the safety of delayed high dose intravenous (i.v.) sodium thiosulfate (STS) in a case series of 12 children with malignant brain tumors who were treated with intraarterial (i.a.) carboplatin in conjunction with blood-brain-barrier disruption (BBBD). METHODS:Twelve children ages 17 months-12 years und...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.20529
更新日期:2006-08-01 00:00:00
abstract:BACKGROUND:Burnout is a work-related syndrome consisting of emotional exhaustion, depersonalization, and diminished feelings of personal accomplishment. Physicians who care for patients with life-threatening illnesses are at high risk for developing burnout. This survey evaluates the prevalence of burnout among pediatr...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.23121
更新日期:2011-12-15 00:00:00
abstract:BACKGROUND:The prognostic significance of having extraskeletal (EES) versus skeletal Ewing sarcoma (ES) in the setting of modern chemotherapy protocols is unknown. The purpose of this study was to compare the clinical characteristics, biologic features, and outcomes for patients with EES and skeletal ES. METHODS:Patie...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26096
更新日期:2016-10-01 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::Germline or acquired mutations involving the GATA-binding protein gene (GATA2) have been linked to a variety of clinical conditions. In addition, patients harboring GATA2 mutations have a striking predisposition to develop myeloid malignancies, such as myelodysplastic syndrome or acute myeloid leukemia, but not acute ...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.27649
更新日期:2019-06-01 00:00:00
abstract::To characterize radiation necrosis following hypofractionated brainstem re-irradiation in pediatric patients, we reviewed 23 cases with 28 tumors invading or abutting brainstem and treated with hypofractionated re-irradiation from 2004 to 2014. Re-irradiation delivered total doses of 16-30 Gy in two to five fractions....
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26341
更新日期:2017-05-01 00:00:00
abstract:INTRODUCTION:Regret about loss is one of the most intense types of regret experienced in life. Little is known about the bereavement regret of parents whose child has died of cancer. Although knowledge about parents' experiences after their child's death is vital for supporting these families, parents' regret is mostly...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.28265
更新日期:2020-06-01 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:BACKGROUND:We sought to assess patterns of failure in pediatric patients with intracranial germ cell tumors (GCT) treated with intensity-modulated radiation therapy with dose painting (DP-IMRT). PROCEDURE:Between July 2007 and October 2013, 11 patients with localized GCT-five germinomas and six nongerminoma GCT (NGGCT...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25867
更新日期:2016-04-01 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