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Hypophosphatemia

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https://www.readbyqxmd.com/read/29034009/a-multicenter-randomized-study-of-decitabine-as-epigenetic-priming-with-induction-chemotherapy-in-children-with-aml
#1
Lia Gore, Timothy J Triche, Jason E Farrar, Daniel Wai, Christophe Legendre, Gerald C Gooden, Winnie S Liang, John Carpten, David Lee, Frank Alvaro, Margaret E Macy, Carola Arndt, Philip Barnette, Todd Cooper, Laura Martin, Aru Narendran, Jessica Pollard, Soheil Meshinchi, Jessica Boklan, Robert J Arceci, Bodour Salhia
BACKGROUND: Decitabine is a deoxycytidine nucleoside derivative inhibitor of DNA-methyltransferases, which has been studied extensively and is approved for myelodysplastic syndrome in adults but with less focus in children. Accordingly, we conducted a phase 1 multicenter, randomized, open-label study to evaluate decitabine pre-treatment before standard induction therapy in children with newly diagnosed AML to assess safety and tolerability and explore a number of biologic endpoints. RESULTS: Twenty-four patients were fully assessable for all study objectives per protocol (10 in Arm A = epigenetic priming induction, 14 in Arm B = standard induction)...
2017: Clinical Epigenetics
https://www.readbyqxmd.com/read/29033620/comparative-safety-of-intravenous-ferumoxytol-versus-ferric-carboxymaltose-for-the-treatment-of-iron-deficiency-anemia-rationale-and-study-design-of-a-randomized-double-blind-study-with-a-focus-on-acute-hypersensitivity-reactions
#2
N Franklin Adkinson, William E Strauss, Kristine Bernard, Robert F Kaper, Iain C Macdougall, Julie S Krop
BACKGROUND: Intravenous (IV) iron is often used to treat iron deficiency anemia in patients who are unable to tolerate or are inadequately managed with oral iron. However, IV iron treatment has been associated with acute hypersensitivity reactions. The comparative risk of adverse events (AEs) with IV iron preparations has been assessed by a few randomized controlled trials, which are most often limited by small patient numbers, which lack statistical power to identify differences in low-frequency AE such as hypersensitivity reactions...
2017: Journal of Blood Medicine
https://www.readbyqxmd.com/read/29031590/association-between-iron-deficiency-anemia-and-hypophosphatemia
#3
Anuhya Kommalapati, Ryan Singhi, Sri Harsha Tella
No abstract text is available yet for this article.
October 11, 2017: American Journal of Medicine
https://www.readbyqxmd.com/read/29026610/selective-blood-sampling-for-fgf-23-in-tumor-induced-osteomalacia
#4
Hans-Christof Schober, Christian Kneitz, Franziska Fieber, Kathrin Hesse, Henry Schroeder
Tumor-induced osteomalacia (TIO) is caused by the hormone fibroblast growth factor 23 (FGF-23). It is mainly produced in the tissue of mesenchymal tumors. Patients with TIO frequently suffer from a chronic decompensated pain syndrome and/or muscle weakness with postural deformity. Despite the severity of the disease, the diagnosis is frequently established late. In some cases, it takes several years to establish the condition. This case report concerning a 68-year old woman demonstrates the selective blood sampling for FGF-23 as path-breaking diagnostics to confirm the diagnosis of a neuroendocrine tumor...
2017: Endocrinology, Diabetes & Metabolism Case Reports
https://www.readbyqxmd.com/read/29021995/tumor-induced-osteomalacia
#5
Pablo Florenzano, Rachel I Gafni, Michael T Collins
Tumor-induced osteomalacia (TIO) is a rare paraneoplastic syndrome clinically characterized by bone pain, fractures and muscle weakness. It is caused by tumoral overproduction of fibroblast growth factor 23 (FGF23) that acts primarily at the proximal renal tubule, decreasing phosphate reabsorption and 1α-hydroxylation of 25 hydroxyvitamin D, thus producing hypophosphatemia and osteomalacia. Lesions are typically small, benign mesenchymal tumors that may be found in bone or soft tissue, anywhere in the body...
December 2017: Bone Reports
https://www.readbyqxmd.com/read/28988377/phase-i-study-of-sorafenib-and-tipifarnib-for-recurrent-glioblastoma-nabtc-05-02
#6
Phioanh Leia Nghiemphu, Victoria Asuquo Ebiana, Patrick Wen, Mark Gilbert, Lauren E Abrey, F Lieberman, Lisa M DeAngelis, H Ian Robins, W K Alfred Yung, Susan Chang, Jan Drappatz, Minesh P Mehta, Victor A Levin, Kenneth Aldape, Janet E Dancey, J J Wright, Michael Prados, John Kuhn, Timothy F Cloughesy
Recurrent glioblastoma (GBM) has a very low 6-month progression free survival (PFS) with currently available treatments. Combination chemotherapy to target multiple cell signaling pathways is currently being investigated in order to improve prognosis for recurrent disease. The purpose of this phase I study was to determine the maximum tolerated dose (MTD) for the combination of tipifarnib and sorafenib for the treatment of recurrent GBM. Patients with pathologically proven WHO grade IV GBM and radiographically proven tumor recurrence were eligible for this study...
October 7, 2017: Journal of Neuro-oncology
https://www.readbyqxmd.com/read/28982589/identification-of-a-novel-loss-of-function-phex-mutation-ala720ser-in-a-sporadic-case-of-adult-onset-hypophosphatemic-osteomalacia
#7
Katarzyna Goljanek-Whysall, Andreas Tridimas, Rachel McCormick, Nicki-Jayne Russell, Melissa Sloman, Alan Sorani, William D Fraser, Fadil M Hannan
Adults presenting with sporadic hypophosphatemia and elevations in circulating fibroblast growth factor-23 (FGF23) concentrations are usually investigated for an acquired disorder of FGF23 excess such as tumor induced osteomalacia (TIO). However, in some cases the underlying tumor is not detected, and such patients may harbor other causes of FGF23 excess. Indeed, coding-region and 3'UTR mutations of phosphate-regulating neutral endopeptidase (PHEX), which encodes a cell-surface protein that regulates circulating FGF23 concentrations, can lead to alterations in phosphate homeostasis, which are not detected until adulthood...
October 3, 2017: Bone
https://www.readbyqxmd.com/read/28977163/use-of-cinacalcet-and-sunitinib-to-treat-hypercalcaemia-due-to-a-pancreatic-neuroendocrine-tumor
#8
Hernan Valdes-Socin, Matilde Rubio Almanza, Mariana Tomé Fernández-Ladreda, Daniel Van Daele, Marc Polus, Marcela Chavez, Albert Beckers
Neuroendocrine tumors (NETs) can secrete hormones, including ectopic secretions, but they have been rarely associated with malignant hypercalcemia. A 52-year-old man with a history of diabetes mellitus was diagnosed with a pancreatic tumor. A pancreatic biopsy confirmed a well-differentiated pancreatic NET (pNET). The patient subsequently developed liver metastasis and hypercalcemia with high 1,25 OH vitamin D and suppressed parathyroid hormone (PTH) levels. Hypercalcemia was refractory to chemotherapy, intravenous saline fluids, diuretics, calcitonin and zoledronate...
September 18, 2017: Archives of Endocrinology and Metabolism
https://www.readbyqxmd.com/read/28954784/a-first-in-human-phase-1-study-of-the-anti-cancer-stem-cell-agent-ipafricept-omp-54f28-a-decoy-receptor-for-wnt-ligands-in-patients-with-advanced-solid-tumors
#9
Antonio Jimeno, Michael S Gordon, Rashmi Chugh, Wells Messersmith, David S Mendelson, Jakob Dupont, Robert J Stagg, Ann M Kapoun, Lu Xu, Shailaja Uttamsingh, Rainer Brachmann, David C Smith
PURPOSE: Wnt signaling is implicated in tumor cell de-differentiation and cancer stem cell function. Ipafricept (OMP-54F28) is a first-in-class recombinant fusion protein with the extracellular part of human Frizzled 8 receptor fused to a human IgG1 Fc fragment that binds Wnt ligands. This trial evaluated ipafricept in patients with solid tumors. EXPERIMENTAL DESIGN: A 3+3 design was used; ipafricept was given intravenously every 3 weeks. Objectives were determination of dose-limiting toxicities (DLTs), recommended phase 2 dose (RP2D), safety, pharmacokinetics (PK), immunogenicity, pharmacodynamics (PD), and preliminary efficacy...
September 27, 2017: Clinical Cancer Research: An Official Journal of the American Association for Cancer Research
https://www.readbyqxmd.com/read/28954742/outcomes-of-orthopedic-surgery-in-a-cohort-of-49-patients-with-x-linked-hypophosphatemic-rickets-xlhr
#10
A Gizard, A Rothenbuhler, Z Pejin, G Finidori, C Glorion, B de Billy, A Linglart, P Wicart
BACKGROUND: X-linked hypophosphatemic rickets (XLHR) is due to mutations in PHEX leading to unregulated production of FGF23 and hypophosphatemia. XLHR is characterized by leg bowing of variable severity. Phosphate supplements and oral vitamin analogs, partially or, in some cases, fully restore the limb straightness. Surgery is the alternative for severe or residual limb deformities. OBJECTIVE: To retrospectively assess the results of surgical limb correction in XLHR (osteotomies and bone alignment except for 3 transient hemiepiphysiodesis)...
November 2017: Endocrine Connections
https://www.readbyqxmd.com/read/28953654/a-case-of-osteomalacia-due-to-deranged-mineral-balance-caused-by-saccharated-ferric-oxide-and-short-bowel-syndrome-a-case-report
#11
Hiroshi Nomoto, Hideaki Miyoshi, Akinobu Nakamura, So Nagai, Naoyuki Kitao, Chikara Shimizu, Tatsuya Atsumi
RATIONALE: Saccharated ferric oxide has been shown to lead to elevation of fibroblast growth factor 23, hypophosphatemia, and, consequently, osteomalacia. Moreover, mineral imbalance is often observed in patients with short-bowel syndrome to some degree. PATIENT CONCERNS: A 62-year-old woman with short-bowel syndrome related with multiple resections of small intestines due to Crohn disease received regular intravenous administration of saccharated ferric oxide. Over the course of treatment, she was diagnosed with tetany, which was attributed to hypocalcemia...
September 2017: Medicine (Baltimore)
https://www.readbyqxmd.com/read/28943082/nutritional-rickets-in-taiwanese-children-experiences-at-a-single-center
#12
Li-Hsin Chuang, Yi-Ching Tung, Shih-Yao Liu, Cheng-Ting Lee, Huey-Ling Chen, Wen-Yu Tsai
BACKGROUND/PURPOSE: The number of children with nutritional rickets in Taiwan has increased over the last decade. The aim of this study was to present our experiences in the management of patients with this condition. PATIENTS AND METHODS: From 2011 to 2016, 10 children (3 boys and 7 girls) with nutritional rickets were enrolled in this study. Their clinical and biochemical data were analyzed. RESULTS: The median age of the 10 patients was 21 months (range, 12-25 months)...
September 21, 2017: Journal of the Formosan Medical Association, Taiwan Yi Zhi
https://www.readbyqxmd.com/read/28939740/immunomodulation-by-entinostat-in-renal-cell-carcinoma-patients-receiving-high-dose-interleukin-2-a-multicenter-single-arm-phase-1-2-trial-nci-ctep-7870
#13
Roberto Pili, David I Quinn, Hans Hammers, J Paul Monk, Saby George, Tanya Dorff, Thomas Olencki, Li Shen, Ashley R Orillion, Dominick Lamonica, Roberto A Salas Fragomeni, Zsolt Szabo, Alan D Hutson, Adrienne Groman, Susan M Perkins, Richard L Piekarz, Michael A Carducci
PURPOSE: Based on preclinical data suggesting that the class I selective HDAC inhibitor entinostat exerts a synergistic antitumor effect in combination with high dose interleukin 2 (IL-2) in a renal cell carcinoma model by down-regulating Foxp3 expression and function of regulatory T cells (Treg), we conducted a phase I/II clinical study with entinostat and high dose IL-2 in patients with metastatic clear cell renal cell carcinoma (ccRCC). EXPERIMENTAL DESIGN: Clear cell histology, no prior treatments, and being sufficiently fit to receive high dose IL-2 were the main eligibility criteria...
September 22, 2017: Clinical Cancer Research: An Official Journal of the American Association for Cancer Research
https://www.readbyqxmd.com/read/28939209/calcium-and-bone-metabolism-indices
#14
Lu Song
Calcium and inorganic phosphate are of critical importance for many body functions, thus the regulations of their plasma concentrations are tightly controlled by the concerted actions of reabsorption/excretion in the kidney, absorption in the intestines, and exchange from bone, the major reservoir for calcium and phosphate in the body. Parathyroid hormone (PTH) and 1,25-dihydroxyvitamin D (1,25(OH)2D) control calcium homeostasis, whereas PTH, 1,25(OH)2D, and bone-derived fibroblast growth factor 23 (FGF 23) control phosphate homeostasis...
2017: Advances in Clinical Chemistry
https://www.readbyqxmd.com/read/28938491/inhibition-of-fgfr-signaling-partially-rescues-hypophosphatemic-rickets-in-hmwfgf2-tg-male-mice
#15
Liping Xiao, Erxia Du, Collin Homer-Bouthiette, Marja M Hurley
Transgenic mice harboring high molecular weight fibroblast growth factor (FGF)2 isoforms (HMWTg) in osteoblast lineage cells phenocopy human X-linked hypophosphatemic rickets (XLH) and Hyp murine model of XLH demonstrating increased FGF23/FGF receptor signaling and hypophosphatemic rickets. Because HMWFGF2 was upregulated in bones of Hyp mice and abnormal FGF receptor (FGFR) signaling is important in XLH, HMWTg mice were used to examine the effect of the FGFR inhibitor NVP-BGJ398, now in clinical trials for cancer therapy, on hypophosphatemic rickets...
October 1, 2017: Endocrinology
https://www.readbyqxmd.com/read/28934935/treatment-and-outcomes-of-tumor-induced-osteomalacia-associated-with-phosphaturic-mesenchymal-tumors-retrospective-review-of-12-patients
#16
Qing-Yao Zuo, Hong Wang, Wei Li, Xiao-Hui Niu, Yan-Hong Huang, Jia Chen, Yu-Hua You, Bao-Yue Liu, Ai-Min Cui, Wei Deng
BACKGROUND: Tumor-induced osteomalacia (TIO) is a rare paraneoplastic syndrome characterized by severe hypophosphatemia and osteomalacia. Nonspecific symptoms make the diagnosis elusive. In addition, locating the responsible tumor(s) is challenging. The aim of this study was to investigate the clinical management and outcomes of TIO. METHODS: The clinical features, diagnostic procedures, treatment, and outcomes of 12 patients were reviewed retrospectively. RESULTS: The cohort comprised six men and six women (mean age 45...
September 21, 2017: BMC Musculoskeletal Disorders
https://www.readbyqxmd.com/read/28932769/microrna-expression-in-a-phosphaturic-mesenchymal-tumour
#17
Darrell Green, Irina Mohorianu, Isabelle Piec, Jeremy Turner, Clare Beadsmoore, Andoni Toms, Richard Ball, John Nolan, Iain McNamara, Tamas Dalmay, William D Fraser
Phosphaturic mesenchymal tumours are a heterogeneous set of bone and soft tissue neoplasms that can cause a number of paraneoplastic syndromes such as tumour induced osteomalacia. The term phosphaturic comes from the common finding that these tumours secrete high levels of fibroblast growth factor 23 which causes renal phosphate wasting leading to hypophosphatemia. Phosphaturic mesenchymal tumours are rare and diagnosis is difficult. A very active 68 year old male presented with bone pain and muscle weakness...
December 2017: Bone Reports
https://www.readbyqxmd.com/read/28904875/the-influence-of-hypophosphatemia-on-outcomes-of-low-and-high-intensity-continuous-renal-replacement-therapy-in-critically-ill-patients-with-acute-kidney-injury
#18
Soo Young Kim, Ye Na Kim, Ho Sik Shin, Yeonsoon Jung, Hark Rim
BACKGROUND: The purpose of this study was to assess the role of hypophosphatemia in major clinical outcomes of patients treated with low- or high-intensity continuous renal replacement therapy (CRRT). METHODS: We performed a retrospective analysis of data collected from 492 patients. We divided patients into two CRRT groups based on treatment intensity (greater than or equal to or less than 40 mL/kg/hour of effluent generation) and measured serum phosphate level daily during CRRT...
September 2017: Kidney Research and Clinical Practice
https://www.readbyqxmd.com/read/28880715/defective-mineralization-in-x-linked-hypophosphatemia-dental-pulp-cell-cultures
#19
B R Coyac, B Hoac, P Chafey, G Falgayrac, L Slimani, P S Rowe, G Penel, A Linglart, M D McKee, C Chaussain, C Bardet
X-linked hypophosphatemia (XLH) is a skeletal disease caused by inactivating mutations in the PHEX gene. Mutated or absent PHEX protein/enzyme leads to a decreased serum phosphate level, which cause mineralization defects in the skeleton and teeth (osteomalacia/odontomalacia). It is not yet altogether clear whether these manifestations are caused solely by insufficient circulating phosphate availability for mineralization or also by a direct, local intrinsic effect caused by impaired PHEX activity. Here, we evaluated the local role of PHEX in a 3-dimensional model of extracellular matrix (ECM) mineralization...
September 1, 2017: Journal of Dental Research
https://www.readbyqxmd.com/read/28874891/real-life-treatment-of-metastatic-colorectal-cancer-with-regorafenib-a-single-centre-review
#20
J Gotfrit, M Vickers, S Sud, T Asmis, C Cripps, R Goel, T Hsu, D Jonker, R Goodwin
BACKGROUND: Various tyrosine kinase signalling pathways affect the development and progression of colorectal cancer (crc). In clinical trials, regorafenib has been associated with a survival benefit in metastatic crc (mcrc). We assessed the safety and efficacy of regorafenib in real-world patients. METHODS: In a retrospective review of patients with mcrc treated with regorafenib at our institution from 2013 to 2015, patient demographics, treatment, and survival data were collected...
August 2017: Current Oncology
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