Investigation of diffusion, practical use, and utility of a medical device for Japanese hemophilia patients in a single institution

ConclusionThough personalized treatment can be formalized to some extent, it is expected that medical treatment skills and management by tacit knowledge are affected in individualized treatment based on standardized treatment. Medical institutions and medical staff are the main body sharing and deploying measures to disseminate and utilize medical devices, cooperating with them can be an effective management of standardized and individualized treatment.
Source: Personalized Medicine Universe - Category: Drugs & Pharmacology Source Type: research

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CONCLUSION: Haemophilic patients went to the Emergency Department for common paediatric causes, but also requested consultation on specific problems related to haemophilia, with musculoskeletal problems/injury or bleeding being the main issues. The paediatric Emergency Department is an indispensable component of haemophilia care. PMID: 31171477 [PubMed - as supplied by publisher]
Source: Anales de Pediatria - Category: Pediatrics Authors: Tags: An Pediatr (Barc) Source Type: research
This study sought to identify hemophilia carriers in C ôte d’Iv...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
In this study, the potential of PF-06741086 to restore thrombin generation in rare disease plasma was explored.Thrombin generation was measured in citrated platelet poor Factor XI (FXI), Factor V (FV), FVII, von Willebrand Factor (vWF) deficient (Type 1, 2A, 2B and 3) congenital donor plasma following the in vitro addition of PF-06741086 (0, 1, 10 or 100 nM) or a human IgG1 antibody; initiated with 1 pM TF and 4 µM phospholipid. FXI, FV, and FVII donors had less than 1% coagulation factor activity. Non-hemophilic plasma from healthy donors alone was also included in the analysis. In FXI deficient plasmas, a concentra...
Source: Blood - Category: Hematology Authors: Tags: 321. Blood Coagulation and Fibrinolytic Factors: Poster II Source Type: research
ConclusionsA pharmacometric approach guided the phase III dose selection of emicizumab in hemophilia A, without conducting a conventional dose-finding study. Phase III studies with the selected dosing regimens are currently ongoing. This case study indicates that a pharmacometric approach can substitute for a conventional dose-finding study in rare diseases and will streamline the drug development process.
Source: Clinical Pharmacokinetics - Category: Drugs & Pharmacology Source Type: research
CONCLUSIONS: Coordination with a hematologist and dose adjustment of the coagulation factor preparation to maintain a target level of coagulation factor activity facilitated a smooth postoperative course with perioperative control of bleeding during spinal surgery for patients with hemophilia. PMID: 29879771 [PubMed]
Source: Asian Spine Journal - Category: Orthopaedics Tags: Asian Spine J Source Type: research
ConclusionsA pharmacometric approach guided the phase III dose selection of emicizumab in hemophilia A, without conducting a conventional dose-finding study. Phase III studies with the selected dosing regimens are currently ongoing. This case study indicates that a pharmacometric approach can substitute for a conventional dose-finding study in rare diseases and will streamline the drug development process.
Source: Clinical Pharmacokinetics - Category: Drugs & Pharmacology Source Type: research
This article is protected by copyright. All rights reserved.
Source: Journal of the European Academy of Dermatology and Venereology - Category: Dermatology Authors: Tags: Letter to Editor Source Type: research
ConclusionThe findings highlight the need for more research on the shared psychological and social impact of living with a rare diagnosis across conditions, in order to identify risk factors and inform clinical practice. Individuals with rare diseases may face challenges that are different from those experienced in more common medical conditions. We performed a systematic review, including qualitative studies on adults, published between 2000 and 2016. The findings highlight the need for more research on the shared psychological and social impact of living with a rare diagnosis across conditions, in order to identify risk...
Source: Molecular Genetics & Genomic Medicine - Category: Genetics & Stem Cells Authors: Tags: Review Article Source Type: research
This article is protected by copyright. All rights reserved.
Source: American Journal of Hematology - Category: Hematology Authors: Tags: Critical Review Source Type: research
Abstract Rare diseases are defined as life-threatening or chronically debilitating diseases with a prevalence of less than one per 2000 according to the European Union or one per 1250 according to the USA. Congenital rare bleeding disorders RBD are reported in most populations, with incidence varying from 1 in 5000 (Hemophilia A), 1:30,000 (Hemophilia B) to much rarer (1:500,000 for FVII deficiency, 1-3 million for Prothrombin or FXIII deficiency). Acquired Hemophilia A is also a rare bleeding disorder with estimated frequency of 1 in million. Most RBDs are inherited as autosomal recessive (AR); however, heterozyg...
Source: Blood Cells, Molecules and Diseases - Category: Hematology Authors: Tags: Blood Cells Mol Dis Source Type: research
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