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[PMID]:29301447
[Au] Autor:Richardson PG; Triplett BM; Ho VT; Chao N; Dignan FL; Maglio M; Mohty M
[Ad] Endereço:a Hematologic Oncology , Harvard Medical School, Jerome Lipper Multiple Myeloma Center, Dana-Farber Cancer Institute , Boston , MA , USA.
[Ti] Título:Defibrotide sodium for the treatment of hepatic veno-occlusive disease/sinusoidal obstruction syndrome.
[So] Source:Expert Rev Clin Pharmacol;11(2):113-124, 2018 Feb.
[Is] ISSN:1751-2441
[Cp] País de publicação:England
[La] Idioma:eng
[Ab] Resumo:INTRODUCTION: Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is an unpredictable condition associated with endothelial-cell damage due to conditioning for hematopoietic stem-cell transplantation (HSCT) or chemotherapy without HSCT. Mortality in patients with VOD/SOS and multi-organ dysfunction (MOD) may be >80%. Areas covered: Defibrotide is the only approved drug for the treatment of severe hepatic VOD/SOS after HSCT in the European Union and hepatic VOD/SOS with renal or pulmonary dysfunction in the United States. Its efficacy in patients with VOD/SOS with MOD post-HSCT was demonstrated in a clinical-trial program that included a historically controlled treatment study, a phase 2 trial, and a large T-IND expanded-access program that also included patients without MOD and who received chemotherapy without HSCT. Expert commentary: Defibrotide appears to protect endothelial cells and restore the thrombolytic-fibrinolytic balance. It addresses a significant clinical need and has demonstrated favorable Day +100 survival and overall adverse-event rates that seem similar to control groups receiving supportive care alone. Currently, defibrotide is under investigation for the prevention of VOD/SOS in high-risk pediatric and adult patients.
[Mh] Termos MeSH primário: Fibrinolíticos/uso terapêutico
Hepatopatia Veno-Oclusiva/tratamento farmacológico
Polidesoxirribonucleotídeos/uso terapêutico
[Mh] Termos MeSH secundário: Antineoplásicos/administração & dosagem
Antineoplásicos/efeitos adversos
Células Endoteliais/patologia
Transplante de Células-Tronco Hematopoéticas/efeitos adversos
Hepatopatia Veno-Oclusiva/etiologia
Hepatopatia Veno-Oclusiva/mortalidade
Seres Humanos
Índice de Gravidade de Doença
[Pt] Tipo de publicação:JOURNAL ARTICLE; REVIEW
[Nm] Nome de substância:
0 (Antineoplastic Agents); 0 (Fibrinolytic Agents); 0 (Polydeoxyribonucleotides); 438HCF2X0M (defibrotide)
[Em] Mês de entrada:1802
[Cu] Atualização por classe:180222
[Lr] Data última revisão:
180222
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:180106
[St] Status:MEDLINE
[do] DOI:10.1080/17512433.2018.1421943


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[PMID]:29069005
[Au] Autor:Mun JU; Cho HR; Bae SM; Park SK; Choi SL; Seo MS; Lim YS; Rn SHW; Kim YU
[Ad] Endereço:aDepartment of Orthopedic Surgery, Changwon Gyeongsang National University Hospital, Incheon bDepartment of Anesthesiology and Pain Medicine, Myongji Hospital, College of Medicine, Seonam University, Goyang cDepartment of Anesthesiology and Pain Medicine, Dongtan Sacred Heart Hospital, Hallym University College of Medicine, Incheon dDepartment of Anesthesiology and Pain Medicine, Catholic Kwandong University of Korea College of Medicine, International ST. Mary's Hospital, Incheon eDepartment of Nursing, Kyung-In Women's University, Incheon, Republic of Korea.
[Ti] Título:Effect of polydeoxyribonucleotide injection on pes anserine bursitis: A case report.
[So] Source:Medicine (Baltimore);96(43):e8330, 2017 Oct.
[Is] ISSN:1536-5964
[Cp] País de publicação:United States
[La] Idioma:eng
[Ab] Resumo:RATIONALE: Pes anserine (PA) bursitis is an inflammatory condition of the medial knee. The PA bursa becomes more painful when infected, damaged, or irritated. Although various treatment options have been attempted to treat PA bursitis, optimal treatments are still debated. This study aims to investigate the effect of polydeoxyribonucleotide (PDRN) injection on reducing pain and inflammation in a patient presenting with PA bursitis. PATIENT CONCERNS: A 50-year-old female patient was admitted to our pain clinic with symptoms of tenderness and pain over the medial knee. Physical examination revealed the pain to be located over the proximal medial tibia at the insertion of the conjoined tendons of the PA. The knee had lost its range of movement and strength, and resisted knee flexion. DIAGNOSES: She was diagnosed as having PA bursitis. INTERVENTIONS: Ultrasound guided PA bursa injection was carried out. OUTCOMES: Follow-up for the patient was more than eight months. She showed good improvement in PA bursitis without any complications. LESSONS: This is the first successful report of successful PDRN injection for PA bursa.
[Mh] Termos MeSH primário: Artralgia
Bursite
Articulação do Joelho
Polidesoxirribonucleotídeos/administração & dosagem
[Mh] Termos MeSH secundário: Analgésicos/administração & dosagem
Anti-Inflamatórios/administração & dosagem
Artralgia/tratamento farmacológico
Artralgia/etiologia
Bolsa Sinovial/efeitos dos fármacos
Bursite/diagnóstico
Bursite/tratamento farmacológico
Bursite/etiologia
Bursite/fisiopatologia
Feminino
Seres Humanos
Injeções/métodos
Articulação do Joelho/diagnóstico por imagem
Articulação do Joelho/patologia
Meia-Idade
Resultado do Tratamento
Ultrassonografia/métodos
[Pt] Tipo de publicação:CASE REPORTS; JOURNAL ARTICLE
[Nm] Nome de substância:
0 (Analgesics); 0 (Anti-Inflammatory Agents); 0 (Polydeoxyribonucleotides)
[Em] Mês de entrada:1711
[Cu] Atualização por classe:171123
[Lr] Data última revisão:
171123
[Sb] Subgrupo de revista:AIM; IM
[Da] Data de entrada para processamento:171026
[St] Status:MEDLINE
[do] DOI:10.1097/MD.0000000000008330


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[PMID]:28859028
[Au] Autor:Kizilocak H; Dikme G; Özdemir N; Kurugoglu S; Adaletli I; Erkan T; Celkan T
[Ad] Endereço:*Pediatric Hematology-Oncology, Cerrahpasa Medical Faculty †Department of Radiology, Cerrahpasa Medical Faculty ‡Cerrahpasa Medical Faculty, Pediatric Gastroenterology, Istanbul University, Istanbul, Turkey.
[Ti] Título:Sinusoidal Obstruction Syndrome During Chemotherapy of Pediatric Cancers and its Successful Management With Defibrotide.
[So] Source:J Pediatr Hematol Oncol;39(7):e373-e376, 2017 Oct.
[Is] ISSN:1536-3678
[Cp] País de publicação:United States
[La] Idioma:eng
[Ab] Resumo:Sinusoidal obstruction syndrome (SOS) is a life-threatening complication generally occurring after hematopoietic stem cell transplantation. SOS after standard dose chemotherapy in malignancies is rare. Between the year 1995 and 2016, 414 patients were diagnosed with acute lymphoblastic leukemia and 113 patients were diagnosed with Wilms tumor in our institution. Among these patients, 4 patients with acute lymphoblastic leukemia (0.96%) and 2 patients with Wilms tumor (1.7%) developed SOS during treatment. SOS behaves like a local disseminated intravascular coagulation. Defibrotide has proved to be effective in SOS. In this article, we report our experience with defibrotide in SOS.
[Mh] Termos MeSH primário: Hepatopatia Veno-Oclusiva/tratamento farmacológico
Neoplasias/complicações
Polidesoxirribonucleotídeos/uso terapêutico
[Mh] Termos MeSH secundário: Antineoplásicos/efeitos adversos
Criança
Hepatopatia Veno-Oclusiva/induzido quimicamente
Hepatopatia Veno-Oclusiva/etiologia
Seres Humanos
Neoplasias/tratamento farmacológico
Inibidores da Agregação de Plaquetas/uso terapêutico
Leucemia-Linfoma Linfoblástico de Células Precursoras/complicações
Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológico
Tumor de Wilms/complicações
Tumor de Wilms/tratamento farmacológico
[Pt] Tipo de publicação:JOURNAL ARTICLE
[Nm] Nome de substância:
0 (Antineoplastic Agents); 0 (Platelet Aggregation Inhibitors); 0 (Polydeoxyribonucleotides); 438HCF2X0M (defibrotide)
[Em] Mês de entrada:1710
[Cu] Atualização por classe:171025
[Lr] Data última revisão:
171025
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:170901
[St] Status:MEDLINE
[do] DOI:10.1097/MPH.0000000000000958


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[PMID]:28640096
[Au] Autor:Kang KN; Kim TW; Koh JW; Oh HB; Mun JU; Seo MS; Kim YU
[Ad] Endereço:aDepartment of Anesthesiology and Pain Medicine, National Police Hospital, Seoul bDepartment of Orthopaedic Surgery, Changwon Gyeongsang National University Hospital cDepartment of Anesthesiology and Pain Medicine, Catholic Kwandong University of Korea College of Medicine, International St. Mary's Hospital, Incheon, Republic of Korea.
[Ti] Título:Effect of transforaminal epidural polydeoxyribonucleotide injections on lumbosacral radiculopathy: A case report.
[So] Source:Medicine (Baltimore);96(25):e7174, 2017 Jun.
[Is] ISSN:1536-5964
[Cp] País de publicação:United States
[La] Idioma:eng
[Ab] Resumo:RATIONALE: Transforaminal epidural glucocorticoids administration is widely performed for the management of lumbosacral radiculopathy. However, it may worsen the condition of patients with type 2 diabetes mellitus (DM). Polydeoxyribonucleotide (PDRN) was recently noted as a substitute for glucocorticoids. PATIENT CONCERNS: A 44-year-old male patient was admitted to our pain clinic with symptoms of low back pain with severe pain and tingling sensation of left posterolateral leg. He had type 2 DM medicated with Glimepiride and Metformin. Blood glucose level was 367 mg/dL. He declined to use glucocorticoid. DIAGNOSES: He was diagnosed as left foraminal disc protrusion at L4-5, left subarticular disc protrusion at L5-S1. INTERVENTIONS: Fluoroscopically guided transforaminal epidural PDRN injections were carried out. OUTCOMES: The patient was followed up for more than 6 months and demonstrated good improvement in lumbosacral radiculopathy without any complications. LESSONS: This is the first successful report on epidural injection of PDRN.
[Mh] Termos MeSH primário: Anti-Inflamatórios não Esteroides/administração & dosagem
Deslocamento do Disco Intervertebral/tratamento farmacológico
Dor Lombar/tratamento farmacológico
Polidesoxirribonucleotídeos/administração & dosagem
Radiculopatia/tratamento farmacológico
[Mh] Termos MeSH secundário: Adulto
Seres Humanos
Injeções Epidurais
Deslocamento do Disco Intervertebral/complicações
Deslocamento do Disco Intervertebral/diagnóstico por imagem
Dor Lombar/diagnóstico por imagem
Dor Lombar/etiologia
Vértebras Lombares/diagnóstico por imagem
Masculino
Radiculopatia/diagnóstico por imagem
Radiculopatia/etiologia
Sacro/diagnóstico por imagem
[Pt] Tipo de publicação:CASE REPORTS; JOURNAL ARTICLE
[Nm] Nome de substância:
0 (Anti-Inflammatory Agents, Non-Steroidal); 0 (Polydeoxyribonucleotides)
[Em] Mês de entrada:1707
[Cu] Atualização por classe:170717
[Lr] Data última revisão:
170717
[Sb] Subgrupo de revista:AIM; IM
[Da] Data de entrada para processamento:170623
[St] Status:MEDLINE
[do] DOI:10.1097/MD.0000000000007174


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[PMID]:28600293
[Au] Autor:Ferdous F; Saski C; Bridges W; Burns M; Dunn H; Elliott K; Scott TR
[Ad] Endereço:Department of Animal and Veterinary Sciences, Clemson University, Clemson, SC 29634.
[Ti] Título:Bacterial and Viral Products Affect Differential Pattern Recognition Receptor Activation of Chicken Thrombocytes Evidenced through RNA Sequencing.
[So] Source:J Immunol;199(2):774-781, 2017 Jul 15.
[Is] ISSN:1550-6606
[Cp] País de publicação:United States
[La] Idioma:eng
[Ab] Resumo:It is now well understood that thrombocytes (nucleated platelets) express TLRs and respond to both bacterial and viral products. Release of proinflammatory molecules can be expected following relatively short exposure times to LPS, lipoteichoic acid (LTA), thymidine homopolymer phosphorothioate oligonucleotide [Poly(dT)], and polyinosinic-polycytidylic acid [Poly(I:C)]. This study reports the varied expressions of genes encoded for components of the TLR, nucleotide binding oligomerization domain-like receptor, and retinoic acid-inducible gene RIG-like receptor signaling pathways in response to the TLR ligands listed above. Highly sensitive RNA-sequencing technologies were used to analyze the complete transcriptome of thrombocytes treated with all four microbial products for a period of 1 h. A total of 14,326 gene transcripts were found in chicken thrombocytes across all ligand exposures. After 1 h of stimulation with ligands, 87, 138, 1013, and 22 genes were upregulated for LTA, LPS, Poly(dT), and Poly(I:C), and 12, 142, 249, and 16 genes were downregulated for LTA, LPS, Poly(dT), and Poly(I:C), respectively, with at least a 1-fold change relative to unexposed thrombocytes. Summarizations of biological processes, protein classes, and biochemical pathways reveal the role of chicken thrombocytes in proinflammatory responses linked to key signaling pathways. TLR, nucleotide binding oligomerization domain-like receptor, and retinoic acid-inducible gene RIG-like receptor pathways were mapped based on the transcriptome results with gene expression for common signal and proinflammatory mediators highlighted. The information reported in this study is useful for defining a limited set of proinflammatory molecules to evaluate in cases of either bacterial or viral disease monitoring.
[Mh] Termos MeSH primário: Antígenos de Bactérias/imunologia
Antígenos Virais/imunologia
Plaquetas/imunologia
Receptores de Reconhecimento de Padrão/genética
Receptores de Reconhecimento de Padrão/imunologia
[Mh] Termos MeSH secundário: Animais
Antígenos de Bactérias/química
Antígenos Virais/química
Plaquetas/efeitos dos fármacos
Galinhas
Proteína DEAD-box 58/imunologia
Proteína DEAD-box 58/metabolismo
Regulação para Baixo
Perfilação da Expressão Gênica
Inflamação
Lipopolissacarídeos/imunologia
Poli I-C/imunologia
Polidesoxirribonucleotídeos/imunologia
Receptores de Reconhecimento de Padrão/metabolismo
Análise de Sequência de RNA
Transdução de Sinais
Ácidos Teicoicos/imunologia
Receptores Toll-Like/imunologia
Receptores Toll-Like/metabolismo
Transcriptoma
Regulação para Cima
[Pt] Tipo de publicação:JOURNAL ARTICLE
[Nm] Nome de substância:
0 (Antigens, Bacterial); 0 (Antigens, Viral); 0 (Lipopolysaccharides); 0 (Polydeoxyribonucleotides); 0 (Receptors, Pattern Recognition); 0 (Teichoic Acids); 0 (Toll-Like Receptors); 0 (poly(dT)-oligo(dA)); 56411-57-5 (lipoteichoic acid); EC 3.6.1.- (Ddx58 protein, mouse); EC 3.6.4.13 (DEAD Box Protein 58); O84C90HH2L (Poly I-C)
[Em] Mês de entrada:1709
[Cu] Atualização por classe:171116
[Lr] Data última revisão:
171116
[Sb] Subgrupo de revista:AIM; IM
[Da] Data de entrada para processamento:170611
[St] Status:MEDLINE
[do] DOI:10.4049/jimmunol.1700189


  6 / 2428 MEDLINE  
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[PMID]:28592487
[Au] Autor:Vo T; Wang S; Poon GMK; Wilson WD
[Ad] Endereço:From the Department of Chemistry and.
[Ti] Título:Electrostatic control of DNA intersegmental translocation by the ETS transcription factor ETV6.
[So] Source:J Biol Chem;292(32):13187-13196, 2017 Aug 11.
[Is] ISSN:1083-351X
[Cp] País de publicação:United States
[La] Idioma:eng
[Ab] Resumo:To find their DNA target sites in complex solution environments containing excess heterogeneous DNA, sequence-specific DNA-binding proteins execute various translocation mechanisms known collectively as facilitated diffusion. For proteins harboring a single DNA contact surface, long-range translocation occurs by jumping between widely spaced DNA segments. We have configured biosensor-based surface plasmon resonance to directly measure the affinity and kinetics of this intersegmental jumping by the ETS-family transcription factor ETS variant 6 (ETV6). To isolate intersegmental target binding in a functionally defined manner, we pre-equilibrated ETV6 with excess salmon sperm DNA, a heterogeneous polymer, before exposing the nonspecifically bound protein to immobilized oligomeric DNA harboring a high-affinity ETV6 site. In this way, the mechanism of ETV6-target association could be toggled electrostatically through varying NaCl concentration in the bulk solution. Direct measurements of association and dissociation kinetics of the site-specific complex indicated that 1) freely diffusive binding by ETV6 proceeds through a nonspecific-like intermediate, 2) intersegmental jumping is rate-limited by dissociation from the nonspecific polymer, and 3) dissociation of the specific complex is independent of the history of complex formation. These results show that target searches by proteins with an ETS domain, such as ETV6, whose single DNA-binding domain cannot contact both source and destination sites simultaneously, are nonetheless strongly modulated by intersegmental jumping in heterogeneous site environments. Our findings establish biosensors as a general technique for directly and specifically measuring target site search by DNA-binding proteins via intersegmental translocation.
[Mh] Termos MeSH primário: DNA/metabolismo
Modelos Moleculares
Proteínas Proto-Oncogênicas c-ets/metabolismo
Proteínas Repressoras/metabolismo
[Mh] Termos MeSH secundário: Animais
Sítios de Ligação
Técnicas Biossensoriais
DNA/química
DNA de Cadeia Simples/química
DNA de Cadeia Simples/metabolismo
Motivo ETS
Difusão Facilitada
Seres Humanos
Cinética
Motivos de Nucleotídeos
Fragmentos de Peptídeos/química
Fragmentos de Peptídeos/genética
Fragmentos de Peptídeos/metabolismo
Polidesoxirribonucleotídeos/química
Polidesoxirribonucleotídeos/metabolismo
Conformação Proteica
Domínios e Motivos de Interação entre Proteínas
Proteínas Proto-Oncogênicas c-ets/química
Proteínas Proto-Oncogênicas c-ets/genética
Proteínas Recombinantes de Fusão/química
Proteínas Recombinantes de Fusão/metabolismo
Proteínas Recombinantes/química
Proteínas Recombinantes/metabolismo
Proteínas Repressoras/química
Proteínas Repressoras/genética
Elementos de Resposta
Salmão
Eletricidade Estática
Ressonância de Plasmônio de Superfície
Termodinâmica
[Pt] Tipo de publicação:COMPARATIVE STUDY; JOURNAL ARTICLE
[Nm] Nome de substância:
0 (DNA, Single-Stranded); 0 (ETS translocation variant 6 protein); 0 (Peptide Fragments); 0 (Polydeoxyribonucleotides); 0 (Proto-Oncogene Proteins c-ets); 0 (Recombinant Fusion Proteins); 0 (Recombinant Proteins); 0 (Repressor Proteins); 9007-49-2 (DNA)
[Em] Mês de entrada:1708
[Cu] Atualização por classe:170825
[Lr] Data última revisão:
170825
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:170609
[St] Status:MEDLINE
[do] DOI:10.1074/jbc.M117.792887


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[PMID]:28444784
[Au] Autor:Richardson PG; Smith AR; Triplett BM; Kernan NA; Grupp SA; Antin JH; Lehmann L; Miloslavsky M; Hume R; Hannah AL; Nejadnik B; Soiffer RJ
[Ad] Endereço:Jerome Lipper Multiple Myeloma Center, Division of Hematologic Malignancy, Department of Medical Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA.
[Ti] Título:Earlier defibrotide initiation post-diagnosis of veno-occlusive disease/sinusoidal obstruction syndrome improves Day +100 survival following haematopoietic stem cell transplantation.
[So] Source:Br J Haematol;178(1):112-118, 2017 Jul.
[Is] ISSN:1365-2141
[Cp] País de publicação:England
[La] Idioma:eng
[Ab] Resumo:Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a progressive, potentially fatal complication of conditioning for haematopoietic stem cell transplant (HSCT). The VOD/SOS pathophysiological cascade involves endothelial-cell activation and damage, and a prothrombotic-hypofibrinolytic state. Severe VOD/SOS (typically characterized by multi-organ dysfunction) may be associated with >80% mortality. Defibrotide is approved for treating severe hepatic VOD/SOS post-HSCT in the European Union, and for hepatic VOD/SOS with renal or pulmonary dysfunction post-HSCT in the United States. Previously, defibrotide (25 mg/kg/day in 4 divided doses for a recommended ≥21 days) was available through an expanded-access treatment protocol for patients with VOD/SOS. Data from this study were examined post-hoc to determine if the timing of defibrotide initiation post-VOD/SOS diagnosis affected Day +100 survival post-HSCT. Among 573 patients, defibrotide was started on the day of VOD/SOS diagnosis in approximately 30%, and within 7 days in >90%. The relationship between Day +100 survival and treatment initiation before/after specific days post-diagnosis showed superior survival when treatment was initiated closer to VOD/SOS diagnosis with a statistically significant trend over time for better outcomes with earlier treatment initiation (P < 0·001). These results suggest that initiation of defibrotide should not be delayed after diagnosis of VOD/SOS.
[Mh] Termos MeSH primário: Transplante de Células-Tronco Hematopoéticas/efeitos adversos
Hepatopatia Veno-Oclusiva/tratamento farmacológico
Inibidores da Agregação de Plaquetas/uso terapêutico
Polidesoxirribonucleotídeos/uso terapêutico
Condicionamento Pré-Transplante/efeitos adversos
[Mh] Termos MeSH secundário: Adolescente
Adulto
Idoso
Criança
Pré-Escolar
Esquema de Medicação
Feminino
Hepatopatia Veno-Oclusiva/etiologia
Seres Humanos
Lactente
Masculino
Meia-Idade
Inibidores da Agregação de Plaquetas/administração & dosagem
Polidesoxirribonucleotídeos/administração & dosagem
Estudos Prospectivos
Análise de Sobrevida
Condicionamento Pré-Transplante/métodos
Resultado do Tratamento
Adulto Jovem
[Pt] Tipo de publicação:JOURNAL ARTICLE
[Nm] Nome de substância:
0 (Platelet Aggregation Inhibitors); 0 (Polydeoxyribonucleotides); 438HCF2X0M (defibrotide)
[Em] Mês de entrada:1709
[Cu] Atualização por classe:171013
[Lr] Data última revisão:
171013
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:170427
[St] Status:MEDLINE
[do] DOI:10.1111/bjh.14727


  8 / 2428 MEDLINE  
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[PMID]:28008770
[Au] Autor:Veenstra DL; Guzauskas GF; Villa KF; Boudreau DM
[Ad] Endereço:a University of Washington , Seattle , WA , USA.
[Ti] Título:The budget impact and cost-effectiveness of defibrotide for treatment of veno-occlusive disease with multi-organ dysfunction in patients post-hematopoietic stem cell transplant.
[So] Source:J Med Econ;20(5):453-463, 2017 May.
[Is] ISSN:1941-837X
[Cp] País de publicação:England
[La] Idioma:eng
[Ab] Resumo:BACKGROUND: A Phase-3 study of defibrotide compared with historical controls demonstrated a 23% improvement in 100-day survival post-hematopoietic stem cell transplantation (HSCT) among patients with veno-occlusive disease with multi-organ dysfunction (VOD with MOD). AIM: To estimate the budget impact and cost-effectiveness of introducing defibrotide to a transplant center. METHODS: The authors developed a budget impact model from the perspective of a bone-marrow transplant center. It was estimated that 2.3% of adults and 4.2% of children would develop VOD with MOD following HSCT based on a retrospective hospital database analysis and the effect that treating patients with defibrotide would have on costs for adult and pediatric centers was estimated. A cost-utility analysis (CUA) was also developed to capture the long-term cost-effectiveness of defibrotide. Projected life expectancies in the two groups were estimated based on trial data, transplant registry data, studies of long-term survival among HSCT patients, and US population life-tables. RESULTS: There was an estimated 3% increase ($330,706) per year in total adult transplantation center costs associated with adopting defibrotide, and a <1% increase ($106,385) for pediatric transplant centers, assuming 100 transplants per year. In the CUA, the lifetime increase in cost per patient was $106,928, life expectancy increased by 3.74 years, and quality-adjusted life-years (QALYs) increased by 2.24. The incremental cost-effectiveness ratio (ICER) was $47,736 per QALY gained; 88% probability defibrotide was cost-effective at a $100,000/QALY threshold. CONCLUSION: The budget impact of defibrotide for a transplant center is relatively modest compared to the overall cost of transplantation. Defibrotide provides an important survival advantage for VOD with MOD patients, and the life years gained lead to defibrotide being highly cost-effective.
[Mh] Termos MeSH primário: Orçamentos/estatística & dados numéricos
Fibrinolíticos/economia
Hepatopatia Veno-Oclusiva/terapia
Insuficiência de Múltiplos Órgãos/terapia
Polidesoxirribonucleotídeos/economia
[Mh] Termos MeSH secundário: Análise Custo-Benefício
Fibrinolíticos/uso terapêutico
Transplante de Células-Tronco Hematopoéticas/efeitos adversos
Hepatopatia Veno-Oclusiva/economia
Hepatopatia Veno-Oclusiva/etiologia
Seres Humanos
Modelos Econométricos
Insuficiência de Múltiplos Órgãos/economia
Insuficiência de Múltiplos Órgãos/etiologia
Polidesoxirribonucleotídeos/uso terapêutico
Anos de Vida Ajustados por Qualidade de Vida
Estudos Retrospectivos
Análise de Sobrevida
Estados Unidos
[Pt] Tipo de publicação:CLINICAL TRIAL, PHASE III; JOURNAL ARTICLE
[Nm] Nome de substância:
0 (Fibrinolytic Agents); 0 (Polydeoxyribonucleotides); 438HCF2X0M (defibrotide)
[Em] Mês de entrada:1710
[Cu] Atualização por classe:171025
[Lr] Data última revisão:
171025
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:161224
[St] Status:MEDLINE
[do] DOI:10.1080/13696998.2016.1275652


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[PMID]:27914921
[Au] Autor:Yoon YC; Lee DH; Lee MY; Yoon SH
[Ad] Endereço:Department of Physical Medicine and Rehabilitation, Ajou University School of Medicine, Suwon, Republic of Korea.
[Ti] Título:Polydeoxyribonucleotide Injection in the Treatment of Chronic Supraspinatus Tendinopathy: A Case-Controlled, Retrospective, Comparative Study With 6-Month Follow-Up.
[So] Source:Arch Phys Med Rehabil;98(5):874-880, 2017 May.
[Is] ISSN:1532-821X
[Cp] País de publicação:United States
[La] Idioma:eng
[Ab] Resumo:OBJECTIVE: To determine the efficacy of polydeoxyribonucleotide (PDRN) injection for rotator cuff disease (RCD). DESIGN: Case-controlled, retrospective, comparative study. SETTING: Outpatient clinic at a university-affiliated tertiary care hospital. PARTICIPANTS: Patients (N=106) with chronic nontraumatic refractory RCD who were unresponsive to at least 1 month of conservative treatment: 55 patients received PDRN injection (PDRN group) and 51 continued conservative treatment (control group). INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: Shoulder Pain and Disability Index, score on a visual analog scale of the average shoulder pain level, number of analgesic ingestions per day, isometric strength of shoulder abductor, active range of motion (flexion, abduction, internal rotation, external rotation), and maximal tear size of tendon on ultrasonography at pretreatment and 3 and 6 months postinjection. RESULTS: There was no significant difference between the 2 groups in terms of age, sex, shoulder affected, duration of symptoms, and ultrasonographic findings at pretreatment. Compared with the control group, the treatment group showed a significant improvement in Shoulder Pain and Disability Index, visual analog scale score, and number of analgesic ingestions per day. However, there was no difference in isometric strength, active range of motion, and maximal tear size of tendon. No adverse events were reported. CONCLUSIONS: To our knowledge, this is the first study to assess the efficacy of PDRN injection for patients with RCD. The PDRN injection group showed improvement in pain and subjective disability in patients with RCD and continued to show improvement for 3 months thereafter; the PDRN injection can be an optional treatment for patients with chronic RCD who show no response to other treatments.
[Mh] Termos MeSH primário: Polidesoxirribonucleotídeos/uso terapêutico
Lesões do Manguito Rotador/tratamento farmacológico
Dor de Ombro/tratamento farmacológico
[Mh] Termos MeSH secundário: Adulto
Idoso
Estudos de Casos e Controles
Feminino
Hospitais Universitários
Seres Humanos
Injeções Intra-Articulares
Contração Isométrica/efeitos dos fármacos
Masculino
Meia-Idade
Força Muscular/efeitos dos fármacos
Músculo Esquelético/efeitos dos fármacos
Medição da Dor
Polidesoxirribonucleotídeos/administração & dosagem
Amplitude de Movimento Articular/efeitos dos fármacos
Estudos Retrospectivos
Lesões do Manguito Rotador/complicações
Dor de Ombro/etiologia
Ultrassonografia de Intervenção
[Pt] Tipo de publicação:COMPARATIVE STUDY; JOURNAL ARTICLE
[Nm] Nome de substância:
0 (Polydeoxyribonucleotides)
[Em] Mês de entrada:1707
[Cu] Atualização por classe:170724
[Lr] Data última revisão:
170724
[Sb] Subgrupo de revista:AIM; IM
[Da] Data de entrada para processamento:161205
[St] Status:MEDLINE


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[PMID]:27791262
[Au] Autor:Veronesi F; Dallari D; Sabbioni G; Carubbi C; Martini L; Fini M
[Ad] Endereço:Laboratory of Preclinical and Surgical Studies, Rizzoli Orthopedic Institute, Via Di Barbiano, Bologna, Italy.
[Ti] Título:Polydeoxyribonucleotides (PDRNs) From Skin to Musculoskeletal Tissue Regeneration via Adenosine A Receptor Involvement.
[So] Source:J Cell Physiol;232(9):2299-2307, 2017 Sep.
[Is] ISSN:1097-4652
[Cp] País de publicação:United States
[La] Idioma:eng
[Ab] Resumo:Polydeoxyribonucleotides (PDRNs) are low molecular weight DNA molecules of natural origin that stimulate cell migration and growth, extracellular matrix (ECM) protein production, and reduce inflammation. Most preclinical and clinical studies on tissue regeneration with PDRNs focused on skin, and only few are about musculoskeletal tissues. Starting from an overview on skin regeneration studies, through the analysis of in vitro, in vivo, and clinical studies (1990-2016), the present review aimed at defining the effects of PDRN and their mechanisms of action in the regeneration of musculoskeletal tissues. This would also help future researches in this area. A total of 29 studies were found by PubMed and www.webofknowledge.com searches: 20 were on skin (six in vitro, six in vivo, one vitro/vivo, seven clinical studies), while the other nine regarded bone (one in vitro, two in vivo, one clinical studies), cartilage (one in vitro, one vitro/vivo, two clinical studies), or tendon (one clinical study) tissues regeneration. PDRNs improved cell growth, tissue repair, ECM proteins, physical activity, and reduced pain and inflammation, through the activation of adenosine A receptor. PDRNs are currently used for bone, cartilage, and tendon diseases, with a great variability regarding the PDRN dosage to be used in clinical practice, while the dosage for skin regeneration is well established. PDRNs are usually administered from a minimum of three to a maximum of five times and they act trough the activation of A receptor. Further studies are advisable to confirm the effectiveness of PDRNs and to standardize the PDRN dose. J. Cell. Physiol. 232: 2299-2307, 2017. © 2016 Wiley Periodicals, Inc.
[Mh] Termos MeSH primário: Sistema Musculoesquelético/metabolismo
Polidesoxirribonucleotídeos/metabolismo
Receptores A2 de Adenosina/metabolismo
Regeneração
Pele/metabolismo
[Mh] Termos MeSH secundário: Animais
Remodelação Óssea
Movimento Celular
Proliferação Celular
Matriz Extracelular/metabolismo
Seres Humanos
Sistema Musculoesquelético/patologia
Sistema Musculoesquelético/fisiopatologia
Transdução de Sinais
Pele/patologia
Pele/fisiopatologia
Cicatrização
[Pt] Tipo de publicação:JOURNAL ARTICLE; REVIEW
[Nm] Nome de substância:
0 (Polydeoxyribonucleotides); 0 (Receptors, Adenosine A2)
[Em] Mês de entrada:1708
[Cu] Atualização por classe:170808
[Lr] Data última revisão:
170808
[Sb] Subgrupo de revista:IM
[Da] Data de entrada para processamento:161030
[St] Status:MEDLINE
[do] DOI:10.1002/jcp.25663



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