"Sickle cell disease" from_date:2012

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                            2025NHS England
                            Plerixafor use in patients with sickle cell disease (SCD) who are eligible for treatment with exagamglogene autotemcel (ages 12 years and above) NHS England » Plerixafor use in patients with sickle cell disease (SCD) who are eligible for treatment with exagamglogene autotemcel (ages 12 years and above) Skip to main content Cookies on the NHS England websiteWe’ve put some small files called * Publications * Statistics * Blogs * Events * Contact us Search Search Menu * About us * Our work * Commissioning * Get involved Plerixafor use in patients with sickle cell disease (SCD) who are eligible for treatment with exagamglogene autotemcel (ages 12 years and above)Document first published: 25 February 2025 Page updated: 27 February 2025 Topic: Specialised commissioning Publication type: Policy
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                            2024American College of Medical Genetics and Genomics
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                            NarrativeNarrative based
                            EvidenceEvidence based
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                            Casgevy (exagamglogene autotemcel) and Lyfgenia (lovotibeglogene autotemcel) for individuals 12 years and older with sickle cell disease (SCD) and recurrent vaso-occlusive crises (VOC): A therapeutics bulletin of the American College of Medical Genetics ACMG THERAPEUTICS BULLETINCasgevy (exagamglogene autotemcel) and Lyfgenia(lovotibeglogene autotemcel) for individuals 12 yearsand older with sickle cell disease (SCD) and recurrentvaso-occlusive crises (VOC): A therapeutics bulletin of theAmerican College of Medical Genetics and Genomics (ACMG)Harry Lesmana1,2,3, Sun Young Kim4,5, Andr ́es Morales Corado6, Sheri A. Poskanzer7;onbehalf of the ACMG Therapeutics Committee8,*ARTICLE INFOArticle history:Received 16 July 2024Accepted 18 July 2024Available online 10 September
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                            ASH Clinical Practice Guidelines on Sickle Cell Disease (SCD) 1 | 2019-2020 ASH Clinical Practice Guidelines on Sickle Cell Disease: What You Should KnowFor more information on the 2019-2020 ASH Clinical Practice Guidelines on Sickle Cell Disease, visit www.hematology.org/SCDguidelines.The American Society of Hematology (ASH) (www.hematology.org) is the world’s largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment, and prevention of disorders affecting the blood. For more than 60 years, the Society has led the development of hematology as a discipline by promoting research, patient care, education, training, and advocacy in hematology.2019-2021 ASH Clinical Practice Guidelines on Sickle Cell Disease (SCD): What You Should
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                            Black Americans with sickle cell disease (SCD) demonstrate accelerated epigenetic pace of aging compared to Black Americans without SCD. Sickle cell disease (SCD) is a chronic medical condition characterized by red blood cell sickling, vaso-occlusion, hemolytic anemia, and subsequently, end-organ damage and reduced survival. Because of this significant pathophysiology and early mortality, we hypothesized that patients with SCD are experiencing accelerated biological aging compared to individuals without SCD. We utilized the DunedinPACE measure to compare the epigenetic pace of aging in 131 Black Americans with SCD to 1391 Black American veterans without SCD. SCD patients displayed a significantly accelerated pace of aging (DunedinPACE mean difference of 0.057 points) compared to the veterans
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                            2024The Lancet. Haematology
                            Riociguat in patients with sickle cell disease and hypertension or proteinuria (STERIO-SCD): a randomised, double-blind, placebo controlled, phase 1-2 trial. Although nitric oxide based therapeutics have been shown in preclinical models to reduce vaso-occlusive events and improve cardiovascular function, a clinical trial of a phosphodiesterase 5 inhibitor increased rates of admission to hospital for pain. We aimed to examine if riociguat, a direct stimulator of the nitric oxide receptor soluble guanylate cyclase, causes similar increases in vaso-occlusive events. This was a phase 1-2, randomised, double blind, placebo-controlled trial. Eligible patients were 18 years or older, had confirmed sickle cell disease documented by haemoglobin electrophoresis or HPLC fractionation (haemoglobin SS, SC
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                            ...............................................................................................................................52 Exagamglogene autotemcel for treating severe sickle cell disease in people 12 years andover (TA1044)© NICE 2025. All rights reserved. Subject to Notice of rights ( 3 of53 1 Recommendations 1.1 Exagamglogene autotemcel (exa-cel) is recommended with managed access as an option for treating sickle cell disease (SCD) in people 12 years and over: • who have: - recurrent vaso-occlusive crises (VOCs and people with SCD often feel stigmatised by healthcare professionals (see section 3.19). SCD mainly affects people from ethnic minority backgrounds. In the UK, most people with SCD are from Black African and Caribbean groups (see section 3.19). Exagamglogene autotemcel for treating severe sickle cell disease in people 12 years andover (TA1044)© NICE 2025. All rights reserved. Subject to Notice of rights
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                            , voxelotor is recommended. Voxelotor for treating haemolytic anaemia caused by sickle cell disease (TA981)© NICE 2024. All rights reserved. Subject to Notice of rights ( 5 of36 2 Information about voxelotor Marketing authorisation indication 2.1 Voxelotor (Oxbryta, Pfizer) is indicated for 'the treatment of haemolytic anaemia due to sickle cell disease (SCD) in adults and paediatric patients 12 years ), and responses from stakeholders. See the committee papers for full details of the evidence. The condition Details of condition 3.1 In sickle cell disease (SCD), a gene mutation causes red blood cells to become irreversibly sickle shaped. These cells are then broken down in a process called haemolysis, which causes haemolytic anaemia, resulting in low haemoglobin levels. The patient experts explained
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                            2025Prescrire
                            Exagamglogene autotemcel (Casgevy) in beta-thalassaemia or sickle-cell disease Prescrire IN ENGLISH - Spotlight ''Exagamglogene autotemcel (Casgevy°) in beta-thalassaemia or sickle-cell disease'', 1 March 2025 {1}##LOC[OK]## {1} ##LOC[OK]## ##LOC[Cancel]## {1}##LOC[OK]####LOC[Cancel]## Register online| Log in| My Prescrire Issue contents * Current issue * Last 12 issues * All issues Topics Subscription Rate * Subscribers: register online * Prescrire's other products * Free Special Edition * Sign up to receive the newsletter english.prescrire.org > Spotlight > 100 most recent > Exagamglogene autotemcel (Casgevy°) in beta-thalassaemia or sickle-cell disease SpotlightEvery month, the subjects in Prescrire’s Spotlight. 100 most recent: 1|10|20|30|40|50|60|70|80|90 Spotlight * 100 most recent
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                            2024Cochrane
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                            Antioxidant supplementation for sickle cell disease. Sickle cell disease (SCD) refers to a group of genetic disorders characterized by the presence of an abnormal haemoglobin molecule called haemoglobin S (HbS). When subjected to oxidative stress from low oxygen concentrations, HbS molecules form rigid polymers, giving the red cell the typical sickle shape. Antioxidants have been shown to reduce oxidative stress and improve outcomes in other diseases associated with oxidative stress. Therefore, it is important to review and synthesize the available evidence on the effect of antioxidants on the clinical outcomes of people with SCD. To assess the effectiveness and safety of antioxidant supplementation for improving health outcomes in people with SCD. We used standard, extensive Cochrane search
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                            2024Prescrire
                            Voxelotor (Oxbryta) in sickle-cell disease Register online | Log in | My PrescrireISSUE CONTENTSTOPICSABOUT PRESCRIREOFFERSenglish.prescrire.org > Spotlight > 100 most recent > Voxelotor (Oxbryta°) in sickle-cell diseaseSpotlightEvery month, the subjects in Prescrire’s Spotlight.100 most recent :  1 | 10 | 20 | 30 | 40 | 50 | 60 | 70 | 80 | 90Spotlight100 most recentArchivesVoxelotor (Oxbryta °) in sickle-cell disease Marketing Authorisations   Voxelotor binds to haemoglobin and increases its affinity for oxygen. It is claimed that this prevents sickling and premature destruction of red blood cells. A reduction in oxygen release by haemoglobin would be expected in its presence. Voxelotor has been authorised in the European Union for the treatment of haemolytic anaemia due to sickle-cell disease
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                            2024Royal College of Emergency Medicine
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                            NarrativeNarrative based
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                            .................................................................................................................................... 17 Appendix 1 – Example of an SCD Clinical Pathway ....................................................................... 18 Appendix 2 – The ACT NOW* paradigm ........................................................................................ 19 RCEM Best Practice Guideline - Management of Acute Presentations of Sickle Cell Disease Page 5 This guideline has been developed to provide recommendations on the initial care of patients presenting to the emergency department (ED) with acute sickle cell disease (SCD) presentations and its potential complications. The aim is to increase Emergency Department (ED) awareness of SCD, optimise and standardise the management of such patients, and ensure equity of access to acute care and specialist services. The process of creating this guideline ha..
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                            statement Patients admitted on hydroxyurea shall not be given hydroxyurea prescription on discharge. Haematology is to prescribe in order to monitor blood work. 2.0 Definitions  Hydroxyurea (HU) is a chemotherapy medicine that has been used to treat many disorders, including sickle cell disease (SCD). Research has shown that patients with sickle cell disease who take hydroxyurea are admitted at the Hospital for Sick Children. Use of this guideline in any setting must be subject to the clinical judgment of those responsible for providing care. SickKids does not accept responsibility for the application of this guideline outside SickKids. Page 2 of 4 3.0 Guideline Pain assessment and consult completed of child with Sickle Cell Disease (SCD)Prior to starting hydroxyurea ensure that the following
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                            2025Austrian Institute of Health Technology Assessment
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                            Exagamglogene autotemcel (Exa-cel, Casgevy) for the treatment of beta-thalassaemia and severe sickle cell disease. Exagamglogene autotemcel (Exa-cel, Casgevy®) for the treatment of beta-thalassaemia and severe sickle cell disease - Repository of AIHTA GmbH English | Deutsch Atom RSS 1.0 RSS 2.0 * Simple search * Advanced search * Help * Services * Login * Browse * Type * Subject * Author / Editor * Institution * Year AIHTA - Publications - Search - Exagamglogene autotemcel (Exa-cel, Casgevy®) for the treatment of beta-thalassaemia and severe sickle cell disease Grössmann-Waniek, N. and Wild, C. and Malíková, E. and Wolf, S. and Fabian, D. and Sehic, O. and Zechmeister-Koss, I.(2025):Exagamglogene autotemcel (Exa-cel, Casgevy®) for the treatment of beta-thalassaemia and severe sickle cell disease
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                            2023Cochrane
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                            Interventions for chronic kidney disease in people with sickle cell disease. Sickle cell disease (SCD), one of the commonest severe monogenic disorders, is caused by the inheritance of two abnormal haemoglobin (beta-globin) genes. SCD can cause severe pain, significant end-organ damage, pulmonary complications, and premature death. Kidney disease is a frequent and potentially severe complication , outcomes are poor as a result of the progression to proteinuria and chronic kidney insufficiency. Up to 12% of people who develop sickle cell nephropathy will develop end-stage renal disease. This is an update of a review first published in 2017. To assess the effectiveness of any intervention for preventing or reducing kidney complications or chronic kidney disease in people with sickle cell disease
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                            2025CADTH - Reimbursement Review
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                            Exagamglogene Autotemcel (Casgevy) - sickle cell disease View of Exagamglogene Autotemcel (Casgevy) | Canadian Journal of Health Technologies Return to Article DetailsExagamglogene Autotemcel (Casgevy)
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                            2023Medical Services Advisory Committee
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                            Terms of Reference, visit the MSAC website 1. Purpose of application An application requesting the addition of sickle cell disease (SCD) to newborn bloodspot screening (NBS) was received from Australian Sickle Cell Advocacy Inc by the Department of Health and Aged Care. During the initial review stage, the Sickle Cell Disease Expert Working Group recommended that beta(β)-thalassaemia, Haemoglobin E-β deferred its advice regarding adding sickle cell disease (SCD) and beta (β)-thalassaemia to newborn bloodspot screening (NBS) programs. MSAC noted that SCD and β-thalassaemia are rare but severe conditions with high morbidity and mortality, especially SCD. MSAC considered that while adding these haemoglobinopathies to NBS would result in an earlier diagnosis than symptomatic presentation, the strength
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                            2023Royal College of Pathologists
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                            of autopsy practice, the evidence level for the majority of the material in this text is Grade D. Nonetheless, many of the clinicopathological scenarios that occur in sickle cell disease (SCD) and sickle trait persons have been investigated in Coroners’ courts, along with clinical expert witness contributions, and several have gone to High Court litigation for resolution on causality. Thus, the following ; pneumococcal antigen test if suspected. PGD 160823 20 V3 Final Appendix B Cardiac complications in sickle cell disease Researched and drafted by Dr Naoimh Herlihy. Cardiovascular causes are reported to account for up to 32% of deaths in SCD.28 Several autopsy series have reported cardiac pathologies including heart failure, myocardial ischaemia and infarction, sudden cardiac death and cardiac arrhythmias
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                            Effectiveness of a home-based therapeutic exercise program on lower back pain and functionality in Sickle Cell Disease (SCD) patients. To investigate the effectiveness of a home-based therapeutic exercise program on lower back pain and functionality of SCD patients. A Hematology and Transfusion Medicine Center, University of Campinas (HEMOCENTRO-UNICAMP). This was a prospective study , with a three-month follow-up of SCD patients with lower back pain. The lumbar spine functionality was evaluated by questionnaires, trunk flexion and extension analyses by fiber-optic-electrogoniometry and measurements of muscle strength of trunk flexor and extensors. The Intervention Group (IG) comprised 18 volunteers, median age 44y (28-58) and the control group (CG) comprised 15 volunteers, median age 42y
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                            Effects of home-based inspiratory muscle training on sickle cell disease (SCD) patients. Inspiratory muscle training (IMT) has been shown to be an efficient method of improving exercise tolerance and inspiratory and expiratory muscle strength in several diseases. The effects of IMT on patients with sickle cell anemia (SCD) are relatively unknown. Our study aimed to evaluate the effects of IMT on adult SCD patients, regarding respiratory muscle strength (RMS) variables, lung function, exercise tolerance, blood lactation concentration, limitation imposed by dyspnea during daily activities and impact of fatigue on the quality of life. This was a randomized single-blind study, with an IMT design comprising true load (TG) and sham load (SG) groups. Initial assessment included spirometry
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                            2022Cochrane
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                            Hydroxyurea (hydroxycarbamide) for sickle cell disease. Sickle cell disease (SCD) is one of the most common inherited diseases worldwide. It is associated with lifelong morbidity and a reduced life expectancy. Hydroxyurea (hydroxycarbamide), an oral chemotherapeutic drug, ameliorates some of the clinical problems of SCD, in particular that of pain, by raising foetal haemoglobin (HbF ). This is an update of a previously published Cochrane Review. The aims of this review are to determine through a review of randomised or quasi-randomised studies whether the use of hydroxyurea in people with SCD alters the pattern of acute events, including pain; prevents, delays or reverses organ dysfunction; alters mortality and quality of life; or is associated with adverse effects. In addition, we hoped