Gene Therapy Could Be Big Advance Against Hemophilia

Title: Gene Therapy Could Be Big Advance Against HemophiliaCategory: Health NewsCreated: 11/18/2021 12:00:00 AMLast Editorial Review: 11/18/2021 12:00:00 AM
Source: MedicineNet Chronic Pain General - Category: Anesthesiology Source Type: news

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Conclusion We found that reported medication adherence can contribute to better health-related quality of life in patients with haemophilia. Since life with a chronic condition is demanding, it is an important finding that medication adherence to replacement therapy can improve life conditions for patients with haemophilia.
Source: International Journal of Clinical Pharmacy - Category: Drugs & Pharmacology Source Type: research
OSTEOMYELITIS IN CHILDREN: Osteomyelitis (OM) is defined as an infection of the bone marrow and adjacent osseous structures with potential surrounding soft tissue extent. It can occur at any age, generally in children 1 –16 years old. In children, it is predominantly caused by hematogenous spread of infection, typically bacterial. Generally, the metaphysis is involved. Transphyseal spread to the epiphysis and joint is rare but can occur in children younger than 18 months, or in older children with closed growth p lates, due to a different blood perfusion to the epiphysis. The first imaging modality of choice is conve...
Source: Journal of the Belgian Society of Radiology - Category: Radiology Source Type: research
DISCUSSION: Men with hemophilia described significant symptom burden and areas of ongoing need. Collaborative efforts between hematologists, emergency room physicians, and surgeons to establish hospital-specific testing, treatment and referral guidelines, and regular HTC audits may help address these care gaps, providing more person-centred, equitable care. Future work is required to implement these strategies and monitor their effects.PMID:34689399 | DOI:10.1111/jth.15570
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Source Type: research
Res Pract Thromb Haemost. 2021 Oct 11;5(7):e12602. doi: 10.1002/rth2.12602. eCollection 2021 Oct.ABSTRACTINTRODUCTION: In 2017, all people with severe hemophilia B in Ireland switched to recombinant factor IX Fc fusion protein concentrate (rFIXFc) prophylaxis. Patient-reported outcomes (PROs) and health-related quality of life (HRQoL) are important to evaluate with new treatments.AIMS: To assess HRQoL in people with severe hemophilia B and their experience after switching to rFIXFc prophylaxis.METHODS: Participants completed a Patient Reported Outcomes Burden and Experience (PROBE) questionnaire on initiation and following...
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Source Type: research
CONCLUSION: Switching from SHL to rFVIIIFc/rFIXFc resulted in short-term meaningful improvement in overall HRQoL and other PROMs in a small proportion. Longitudinal changes on PROMs are affected by ceiling effects and response shift, warranting further studies in instrument optimization in the era of EHL and nonfactor products.PMID:34667922 | PMC:PMC8505988 | DOI:10.1002/rth2.12601
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Source Type: research
Res Pract Thromb Haemost. 2021 Oct 11;5(7):e12602. doi: 10.1002/rth2.12602. eCollection 2021 Oct.ABSTRACTINTRODUCTION: In 2017, all people with severe hemophilia B in Ireland switched to recombinant factor IX Fc fusion protein concentrate (rFIXFc) prophylaxis. Patient-reported outcomes (PROs) and health-related quality of life (HRQoL) are important to evaluate with new treatments.AIMS: To assess HRQoL in people with severe hemophilia B and their experience after switching to rFIXFc prophylaxis.METHODS: Participants completed a Patient Reported Outcomes Burden and Experience (PROBE) questionnaire on initiation and following...
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Source Type: research
CONCLUSION: Switching from SHL to rFVIIIFc/rFIXFc resulted in short-term meaningful improvement in overall HRQoL and other PROMs in a small proportion. Longitudinal changes on PROMs are affected by ceiling effects and response shift, warranting further studies in instrument optimization in the era of EHL and nonfactor products.PMID:34667922 | PMC:PMC8505988 | DOI:10.1002/rth2.12601
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Source Type: research
J Thromb Haemost. 2021 Jul 1. doi: 10.1111/jth.15444. Online ahead of print.ABSTRACTHemophilia A and B are rare X-linked inherited bleeding disorders caused by complete or partial deficiency in or the absence of coagulation factors VIII and IX. Recurrent joint bleeding (hemarthrosis) is the most frequent clinical manifestation of severe hemophilia. Unless appropriately managed, even sub-clinical hemarthrosiscan lead to the development of hemophilic arthropathy, a disabling condition characterised by joint remodelling, chronic pain and a reduced quality of life, and eventually requires joint replacement. Given the lack of s...
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Source Type: research
Expert Rev Hematol. 2021 May 27. doi: 10.1080/17474086.2021.1935852. Online ahead of print.ABSTRACTINTRODUCTION: People with hemophilia (PWH) commonly experience acute and chronic musculoskeletal pain during childhood and young adulthood, but their treatment is often inadequate.AREAS COVERED: From September 1, 2020 to April 15, 2021, authors performed a literature search in PubMed and the Cochrane Library using "hemophilia AND pain" as keywords. Authors found 1082 articles, 51 of which were chosen because we considered them to be intimately connected with the topic of this report. Multimodal pain treatment, inclu...
Source: Expert Review of Hematology - Category: Hematology Authors: Source Type: research
Res Pract Thromb Haemost. 2021 Mar 6;5(4):e12488. doi: 10.1002/rth2.12488. eCollection 2021 May.ABSTRACTBACKGROUND: Patient-relevant health outcomes for persons with hemophilia should be identified and prioritized to optimize and individualize care for persons with hemophilia. Therefore, an international group of persons with hemophilia and multidisciplinary health care providers set out to identify a globally applicable standard set of health outcomes relevant to all individuals with hemophilia.METHODS: A systematic literature search was performed to identify possible health outcomes and risk adjustment variables. Persons...
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Source Type: research
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