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  1. Article ; Online: The prevalence of musculoskeletal pain and therapy needs in adults with Osteogenesis Imperfecta (OI) a cross-sectional analysis.

    Barlow, Sophie / Dove, Lucy / Jaggi, Anju / Keen, Richard / Bubbear, Judith

    BMC musculoskeletal disorders

    2022  Volume 23, Issue 1, Page(s) 485

    Abstract: Background: Osteogenesis Imperfecta affects approximately 1 in every 10,000 people. Musculoskeletal disorders and pain are common in adults with Osteogenesis Imperfecta, but specific knowledge of the problems people have is lacking. Access to therapy ... ...

    Abstract Background: Osteogenesis Imperfecta affects approximately 1 in every 10,000 people. Musculoskeletal disorders and pain are common in adults with Osteogenesis Imperfecta, but specific knowledge of the problems people have is lacking. Access to therapy services for adults with Osteogenesis Imperfecta is variable. We designed this analysis to better understand the musculoskeletal disorders and consequent therapy needs for adults with Osteogenesis Imperfecta.  METHODS: This study was a cross-sectional analysis of outpatients with Osteogenesis Imperfecta. Adults attending a newly established multidisciplinary clinic at a tertiary centre in 2019 were included. A highly specialist physiotherapist worked within the clinic to offer therapy input if required and to refer patients to appropriate therapy as needed. People over the age of 18 were included if they had a diagnosis of Osteogenesis Imperfecta. Data were collected over a five month period using routinely collected clinical information and patient reported outcomes.
    Results: Over five months 50 patients attended the clinic. Musculoskeletal pain was a significant feature reported by 84% of patients. Over 50% of patients reported persistent pain for longer than one year duration and the most common site of pain was in the spine (46%). No difference in pain between types of OI and age. Forty five per cent (n = 19) of patients reported moderate to severe problems with mobility on the EQ-5D with over half reporting problems with self-care and ability to carry out usual activities. Over 50% of patients in clinic also reported anxiety (EQ-5D). During the consultation 70% of patients received therapy input which was either advice in clinic or an onward referral to the appropriate service. The referral rate to specialist out-patient rehabilitation services at a tertiary centre was 30%.
    Conclusions: This analysis highlights the high prevalence of MSK pain in adults with OI and the effect on physical function and emotional wellbeing. This study demonstrates the diverse needs of the adult Osteogenesis Imperfecta population and the need for suitable multidisciplinary therapy services.
    MeSH term(s) Adult ; Cross-Sectional Studies ; Emotions ; Humans ; Middle Aged ; Musculoskeletal Pain/diagnosis ; Musculoskeletal Pain/epidemiology ; Musculoskeletal Pain/therapy ; Osteogenesis Imperfecta/complications ; Osteogenesis Imperfecta/epidemiology ; Osteogenesis Imperfecta/therapy ; Prevalence
    Language English
    Publishing date 2022-05-21
    Publishing country England
    Document type Journal Article
    ZDB-ID 2041355-5
    ISSN 1471-2474 ; 1471-2474
    ISSN (online) 1471-2474
    ISSN 1471-2474
    DOI 10.1186/s12891-022-05433-3
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  2. Article: Staged brachioradial artery to brachioradial vein arteriovenous fistula creation for hemodialysis access in three patients with a high origin of the radial artery.

    Lawrence, Zachary / Chivukula, Sitaram V / Farlow, Erin C / Keen, Richard R / Sheng, Neha

    Journal of vascular surgery cases and innovative techniques

    2021  Volume 7, Issue 2, Page(s) 271–274

    Abstract: When patients lack suitable superficial veins in the upper extremity to create an arteriovenous fistula, surgeons are faced with a decision between a synthetic graft or autologous fistula using deep veins, such as a brachial artery to brachial vein ... ...

    Abstract When patients lack suitable superficial veins in the upper extremity to create an arteriovenous fistula, surgeons are faced with a decision between a synthetic graft or autologous fistula using deep veins, such as a brachial artery to brachial vein arteriovenous fistula. In patients with a high radial artery origin (or brachioradial artery) and inadequate superficial veins, arteriovenous fistula creation will be even more challenging. In the present report, we describe a technique used in three such patients who underwent successful staged brachioradial artery to brachioradial vein arteriovenous fistula creation.
    Language English
    Publishing date 2021-02-04
    Publishing country United States
    Document type Case Reports
    ISSN 2468-4287
    ISSN 2468-4287
    DOI 10.1016/j.jvscit.2020.12.021
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  3. Article: Osteoporosis: strategies for prevention and management.

    Keen, Richard

    Best practice & research. Clinical rheumatology

    2007  Volume 21, Issue 1, Page(s) 109–122

    Abstract: Osteoporosis is a serious public health issue, affecting up to 1 in 2 women and 1 in 5 men over the age of 50 years. The common osteoporotic fractures occur at the spine, wrist and hip. For the patient affected by osteoporosis, these fractures are ... ...

    Abstract Osteoporosis is a serious public health issue, affecting up to 1 in 2 women and 1 in 5 men over the age of 50 years. The common osteoporotic fractures occur at the spine, wrist and hip. For the patient affected by osteoporosis, these fractures are associated with significant morbidity and, in the case of hip and spine fractures, an excess mortality. The treatment of osteoporotic fractures is also associated with a significant healthcare cost for society. Currently, measurement of bone mineral density using dual energy X-ray absorptiometry is the gold standard for the diagnosis of osteoporosis. In the future, however, assessment of fracture risk will be based on algorithms incorporating clinical risk factors and bone density measurements, where appropriate. The goal of treatment is to reduce the risk of future fracture. Patients at high risk for fracture should be assessed and screened to exclude secondary causes for osteoporosis. Bisphosphonates (alendronate, etidronate, ibandronate, risedronate) are the first-line therapy for the majority of patients and these treatments can be given either orally or intravenously. Alternative treatment options include strontium ranelate and raloxifene. Anabolic therapy with parathyroid hormone can be considered for patients with severe disease. These patients will often require referral for specialist assessment and monitoring. All patients at risk of developing osteoporosis should be given lifestyle advice regarding dietary intake of calcium and vitamin D and regular weight-bearing exercise.
    MeSH term(s) Calcitonin/therapeutic use ; Diphosphonates/therapeutic use ; Disease Management ; Female ; Fractures, Bone/complications ; Fractures, Bone/epidemiology ; Hormone Replacement Therapy ; Humans ; Life Style ; Organometallic Compounds/therapeutic use ; Osteoporosis/complications ; Osteoporosis/prevention & control ; Osteoporosis/therapy ; Parathyroid Hormone/therapeutic use ; Risk Factors ; Selective Estrogen Receptor Modulators/therapeutic use ; Thiophenes/therapeutic use
    Chemical Substances Diphosphonates ; Organometallic Compounds ; Parathyroid Hormone ; Selective Estrogen Receptor Modulators ; Thiophenes ; strontium ranelate (04NQ160FRU) ; Calcitonin (9007-12-9)
    Language English
    Publishing date 2007-02
    Publishing country Netherlands
    Document type Journal Article ; Review
    ZDB-ID 2052323-3
    ISSN 1521-6942
    ISSN 1521-6942
    DOI 10.1016/j.berh.2006.10.004
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  4. Article ; Online: "I Can't Take off My Shirt or Do My Own Hair"-A Qualitative Investigation of the Symptoms and Impact Experience of Children and Adolescents with Fibrodysplasia Ossificans Progressiva (FOP).

    Markowitz, Jessica T / Rofail, Diana / Vandenberg, Gerrit / Baldasaro, Jessica / Sanchez, Robert J / Pignolo, Robert J / Keen, Richard / Davis, Michelle / Marquis, Patrick

    Advances in therapy

    2022  Volume 39, Issue 6, Page(s) 2796–2805

    Abstract: Introduction: Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare, severely disabling, autosomal dominant, congenital disease characterized by progressive multi-focal heterotopic ossification (HO) of skeletal muscle, ligaments, tendons, and ... ...

    Abstract Introduction: Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare, severely disabling, autosomal dominant, congenital disease characterized by progressive multi-focal heterotopic ossification (HO) of skeletal muscle, ligaments, tendons, and fascia. Past FOP studies have focused on the clinical aspects of the disease; therefore, there is a paucity of qualitative research on the patient experience. Our objective was to better understand the experience of children and adolescents living with FOP from their and their parents' perspectives.
    Methods: We conducted a qualitative research study comprising in-depth, open-ended interviews with children and adolescents with FOP and their parents. Semi-structured interviews were conducted via phone call or Microsoft Teams with parent-child dyads (n = 11), adolescents (n = 6), and two clinicians. Children/adolescents and their parents were asked open-ended questions to elicit their daily experience of FOP.
    Results: Concepts were organized into two major themes: symptoms of FOP and the impact of FOP on daily life. Symptoms of FOP reported by children/adolescents, parents, and clinicians were pain, swelling, redness, and stiffness. Functional impacts of flares and FOP in general included accommodations, mobility, activities of daily living, daily activities, and social activities. Impacts were attributed to the difficulties children and adolescents faced living with a disease that prohibited common activities.
    Conclusions: This research documented the experience of children and adolescents with FOP and its effects on their daily lives. It provides a conceptual model for further exploration of the symptoms and impacts important to children and adolescents with FOP and their parents. Children and adolescents and their parents offered novel insights into life with the disease that have not previously been discussed in published literature. Future studies should build upon our conceptual model to create a holistic view of the patient experience of FOP, to inform clinical practice, and the assessment of the patient experience in clinical trials for the disease.
    MeSH term(s) Activities of Daily Living ; Adolescent ; Arthrogryposis ; Hair ; Humans ; Myositis Ossificans/diagnosis ; Ossification, Heterotopic/diagnosis
    Language English
    Publishing date 2022-04-16
    Publishing country United States
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 632651-1
    ISSN 1865-8652 ; 0741-238X
    ISSN (online) 1865-8652
    ISSN 0741-238X
    DOI 10.1007/s12325-022-02096-3
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  5. Article ; Online: Aneurysm of the anterior tibial artery as a complication of infectious endocarditis.

    Terranella, Samantha L / Deal, Rebecca A / Farlow, Erin C / Keen, Richard R / Sheng, Neha

    Vascular

    2020  Volume 29, Issue 4, Page(s) 606–609

    Abstract: Objective: Mycotic aneurysms of the infrapopliteal vessels are rare, with few cases reported in the literature. Management strategies are diverse and should be tailored to the patient's presentation.: Methods: We describe the case of a 40-year-old ... ...

    Abstract Objective: Mycotic aneurysms of the infrapopliteal vessels are rare, with few cases reported in the literature. Management strategies are diverse and should be tailored to the patient's presentation.
    Methods: We describe the case of a 40-year-old male who presented with a painful left leg mass in the setting of bacteremia and infective endocarditis. Imaging revealed an aneurysm of the anterior tibial artery.
    Results: The patient was treated with antibiotics and open surgical repair with excision of the aneurysmal sac, ligation of the anterior tibial artery, and primary repair of the popliteal artery and tibioperoneal trunk.
    Conclusion: The epidemiology, pathophysiology, and clinical management of infrapopliteal aneurysms are briefly reviewed in this case study.
    MeSH term(s) Adult ; Aneurysm, Infected/diagnosis ; Aneurysm, Infected/microbiology ; Aneurysm, Infected/therapy ; Anti-Bacterial Agents/therapeutic use ; Cardiobacterium/isolation & purification ; Endocarditis, Bacterial/diagnosis ; Endocarditis, Bacterial/drug therapy ; Endocarditis, Bacterial/microbiology ; Gram-Negative Bacterial Infections/diagnosis ; Gram-Negative Bacterial Infections/drug therapy ; Gram-Negative Bacterial Infections/microbiology ; Humans ; Ligation ; Male ; Tibial Arteries/microbiology ; Treatment Outcome ; Vascular Surgical Procedures
    Chemical Substances Anti-Bacterial Agents
    Language English
    Publishing date 2020-11-11
    Publishing country England
    Document type Case Reports ; Journal Article ; Review
    ZDB-ID 2137151-9
    ISSN 1708-539X ; 1708-5381
    ISSN (online) 1708-539X
    ISSN 1708-5381
    DOI 10.1177/1708538120969463
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  6. Article: Casebook: osteoporosis.

    Keen, Richard

    The Practitioner

    2005  Volume 249, Issue 1674, Page(s) 639–643

    MeSH term(s) Aged ; Bone Density/physiology ; Female ; Humans ; Middle Aged ; Osteoporosis/diagnosis ; Osteoporosis/physiopathology ; Osteoporosis/therapy ; Referral and Consultation ; Risk Factors
    Language English
    Publishing date 2005-09
    Publishing country England
    Document type Journal Article ; Review
    ZDB-ID 124095-x
    ISSN 0032-6518
    ISSN 0032-6518
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  7. Article ; Online: Study methodology and insights from the palovarotene clinical development program in fibrodysplasia ossificans progressiva.

    Pignolo, Robert J / Al Mukaddam, Mona / Baujat, Geneviève / Brown, Matthew A / De Cunto, Carmen / Hsiao, Edward C / Keen, Richard / Le Quan Sang, Kim-Hanh / Grogan, Donna R / Marino, Rose / Strahs, Andrew R / Kaplan, Frederick S

    BMC medical research methodology

    2023  Volume 23, Issue 1, Page(s) 269

    Abstract: Background: The design of clinical trials in rare diseases is often complicated by a lack of real-world translational knowledge. Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder characterized by skeletal malformations and ... ...

    Abstract Background: The design of clinical trials in rare diseases is often complicated by a lack of real-world translational knowledge. Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder characterized by skeletal malformations and progressive heterotopic ossification (HO). Palovarotene is a selective retinoic acid receptor gamma agonist. Here, we describe the methodology of three studies in the palovarotene clinical development program in FOP and discuss insights that could inform future research, including endpoint suitability and the impact of trial design.
    Methods: PVO-1A-001 (NCT02322255) was a prospective, protocol-specified, longitudinal FOP natural history study (NHS). PVO-1A-201 (NCT02190747) was a randomized, double-blind, placebo-controlled phase II trial; PVO-1A-202 (NCT02279095) was its open-label extension. Trial designs, including treatment regimens and imaging assessments, were refined between PVO-1A-201 and PVO-‍1A-202, and within PVO-1A-202, based on emerging data as the studies progressed. Palovarotene doses were administered using a flare-up treatment regimen (higher dose for 2/4 weeks, followed by lower dose for 4/≥8 weeks; from flare-up onset), with or without accompanying chronic (daily) treatment. Flare-up and disease progression outcomes were assessed, including incidence and volume of new HO during flare-ups and/or annually, as well as other clinical, patient-reported, and exploratory outcomes. Safety was monitored throughout all studies.
    Results: Overall, 114 and 58 individuals with FOP were enrolled in the NHS and phase II trials, respectively. Results of the NHS and PVO-1A-201 were published in 2022; complete results of PVO-1A-202 will be publicly available in due course. Together the studies yielded important information on endpoint suitability, including that low-dose whole-body computed tomography was the optimum imaging modality for assessing HO progression annually and that long study durations are needed to detect substantial changes in functional and patient-reported outcomes.
    Conclusions: A flexible clinical development program is necessary for underexplored rare diseases to overcome the many challenges faced. Here, the NHS provided a longitudinal evaluation of FOP progression and interventional trials were based on emerging data. The studies described informed the design and endpoints implemented in the phase III MOVE trial (NCT03312634) and provide a foundation for future clinical trial development.
    Trial registration: NCT02322255 (registered 23/12/2014); NCT02190747 (registered 15/07/2014); NCT02279095 (registered 30/10/2014).
    MeSH term(s) Humans ; Myositis Ossificans/drug therapy ; Ossification, Heterotopic/drug therapy ; Prospective Studies ; Rare Diseases ; Randomized Controlled Trials as Topic ; Clinical Trials, Phase II as Topic
    Chemical Substances Palovarotene (28K6I5M16G)
    Language English
    Publishing date 2023-11-13
    Publishing country England
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 2041362-2
    ISSN 1471-2288 ; 1471-2288
    ISSN (online) 1471-2288
    ISSN 1471-2288
    DOI 10.1186/s12874-023-02080-7
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  8. Article ; Online: Anaerobic Infections of Autogenous Arteriovenous Fistulae.

    Coughlin, Julia / Fredericks, Charles / Keen, Richard / Farlow, Erin / Sheng, Neha

    The American surgeon

    2018  Volume 83, Issue 9, Page(s) e409–e410

    MeSH term(s) Aged, 80 and over ; Arteriovenous Shunt, Surgical/adverse effects ; Bacteria, Anaerobic/isolation & purification ; Bacteroides fragilis/isolation & purification ; Enterobacter cloacae/isolation & purification ; Enterococcus faecalis/isolation & purification ; Equipment Contamination ; Female ; Gram-Negative Bacterial Infections/etiology ; Humans ; Kidney Failure, Chronic/complications ; Kidney Failure, Chronic/therapy ; Klebsiella oxytoca/isolation & purification ; Male ; Middle Aged ; Renal Dialysis
    Language English
    Publishing date 2018-11-19
    Publishing country United States
    Document type Case Reports ; Journal Article
    ZDB-ID 202465-2
    ISSN 1555-9823 ; 0003-1348
    ISSN (online) 1555-9823
    ISSN 0003-1348
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  9. Article: Four patients with a clinically significant radial artery anomaly.

    Babazadeh, Nasim / Zielsdorf, Shannon / Williams, Lauren / Farlow, Erin / Keen, Richard / Sheng, Neha

    Journal of vascular surgery cases and innovative techniques

    2019  Volume 5, Issue 2, Page(s) 104–106

    Abstract: The superficial radial artery is an anatomic variant in which the radial artery passes superficial to the tendons of the anatomic snuffbox. Cadaver studies have shown its incidence to be 0.5% to 1%. Four patients with this anatomic variation were ... ...

    Abstract The superficial radial artery is an anatomic variant in which the radial artery passes superficial to the tendons of the anatomic snuffbox. Cadaver studies have shown its incidence to be 0.5% to 1%. Four patients with this anatomic variation were encountered in our practice, and their clinical courses and anatomy are described. One patient presented with digital ischemia after catheter placement in the anomalous radial artery. Three patients with end-stage renal disease were found to have a superficial radial artery incidentally, and this was used for inflow in the creation of hemodialysis fistulas.
    Language English
    Publishing date 2019-04-28
    Publishing country United States
    Document type Case Reports
    ISSN 2468-4287
    ISSN 2468-4287
    DOI 10.1016/j.jvscit.2018.10.012
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  10. Article ; Online: Benefit of burosumab in adults with X-linked hypophosphataemia (XLH) is maintained with long-term treatment.

    Kamenicky, Peter / Briot, Karine / Brandi, Maria Luisa / Cohen-Solal, Martine / Crowley, Rachel K / Keen, Richard / Kolta, Sami / Lachmann, Robin H / Lecoq, Anne-Lise / Ralston, Stuart H / Walsh, Jennifer S / Rylands, Angela J / Williams, Angela / Sun, Wei / Nixon, Annabel / Nixon, Mark / Javaid, Muhammad K

    RMD open

    2023  Volume 9, Issue 1

    Abstract: Objectives: To report the impact of continued burosumab treatment on clinical laboratory tests of efficacy, patient-reported outcomes (PROs) and ambulatory function in adults with X-linked hypophosphataemia who continued from a 96-week phase 3 study ... ...

    Abstract Objectives: To report the impact of continued burosumab treatment on clinical laboratory tests of efficacy, patient-reported outcomes (PROs) and ambulatory function in adults with X-linked hypophosphataemia who continued from a 96-week phase 3 study into a 48-week open-label extension.
    Methods: Eligible participants from the phase 3 study continued on the burosumab regimen received at the end of the phase 3 study for a further 48 weeks (n=31). Some (not all) received compassionate burosumab treatment between the two studies (a period of 6-18 months). The primary efficacy outcome was fasting serum phosphate concentration; secondary outcomes were serum 1,25 dihydroxyvitamin D concentration, renal phosphate reabsorption, PROs and ambulatory function.
    Results: Improvements in fasting serum phosphate, serum 1,25 dihydroxyvitamin D and renal phosphate reabsorption at 96 weeks were maintained through the 48-week extension. Improvements were also maintained in stiffness and physical function measured using the Western Ontario and McMaster Universities Osteoarthritis Index, pain and fatigue endpoints measuring using the Brief Pain Inventory short-form and Brief Pain Inventory, respectively, and in ambulatory function (6-Minute Walk Test).A post-hoc exploratory analysis exploring outcomes in participants who discontinued burosumab treatment between the studies (n=7) and those who received at least one dose (n=23) indicated that the benefits of burosumab on clinical laboratory tests of efficacy, PROs and ambulatory function may be lost when treatment is interrupted but recover over time when treatment is reinstated.
    Conclusion: Continued treatment with burosumab appears necessary for sustained clinical benefit.
    Trial registration numbers: Phase 3: NCT02526160; open-label extension: NCT03920072.
    MeSH term(s) Adult ; Humans ; Antibodies, Monoclonal, Humanized/therapeutic use ; Familial Hypophosphatemic Rickets/drug therapy ; Pain ; Phosphates
    Chemical Substances Antibodies, Monoclonal, Humanized ; burosumab (G9WJT6RD29) ; Phosphates
    Language English
    Publishing date 2023-02-28
    Publishing country England
    Document type Clinical Trial, Phase III ; Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 2812592-7
    ISSN 2056-5933 ; 2056-5933
    ISSN (online) 2056-5933
    ISSN 2056-5933
    DOI 10.1136/rmdopen-2022-002676
    Database MEDical Literature Analysis and Retrieval System OnLINE

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