Assessing Psychosocial Risk and Resilience to Support Readiness for Gene Therapy in Sickle Cell Disease: A Consensus Statement.

Importance: The introduction of gene therapies into the clinical care landscape for individuals living with sickle cell disease (SCD) represents a momentous achievement with the potential to rewrite the story of the world's most prevalent heritable blood disorder. This disease, which was first described in 1910 and did not see a US Food and Drug Administration-approved therapeutic until 1998, is poised to be among the first to realize the promise of gene therapy and genome editing. However, the future of these treatments now rests on how evidence of safety, outcomes, and acceptance in clinical practice unfolds in SCD.

Expecting more: the case for incorporating fertility services into comprehensive sickle cell disease care.

Assisted reproductive technologies (ART) are not yet systematically available to people with sickle cell disease or their parents. Fertility care for these groups requires addressing sickle cell disease-associated infertility risks, fertility preservation options, pregnancy possibilities and outcomes, and, when needed, infertility treatment. People with a chance of having a child with sickle cell disease can use in-vitro fertilisation with preimplantation genetic testing to conceive a child unaffected by sickle cell disease.

American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation.

Background: Sickle cell disease (SCD) is a life-limiting inherited hemoglobinopathy that results in significant complications and affects quality of life. Hematopoietic stem cell transplantation (HSCT) is currently the only curative intervention for SCD; however, guidelines are needed to inform how to apply HSCT in clinical practice.

Objective: These evidence-based guidelines of the American Society of Hematology (ASH) are intended to support patients, clinicians, and health professionals in their decisions about HSCT for SCD.

A national survey of functional septorhinoplasty surgery performed in the United Kingdom: a clinician end-user questionnaire to assess current practice and help inform future practice.

Purpose: The aim of this national survey is to assess the current practice of functional septorhinoplasty (SRP) surgery in the UK and better inform future practice.

Methods: An ENT-UK approved questionnaire was sent out to all 135 consultant members of the British Society of Facial Plastic Surgery (BSFPS). Data was collected on numbers of functional SRPs performed on the NHS, use of outcome measures, psychology and photography support, antibiotic use, referral base and consenting practice.

Effect of donor type and conditioning regimen intensity on allogeneic transplantation outcomes in patients with sickle cell disease: a retrospective multicentre, cohort study.

Background: Donors other than matched siblings and low-intensity conditioning regimens are increasingly used in haematopoietic stem cell transplantation. We aimed to compare the relative risk of donor type and conditioning regimen intensity on the transplantation outcomes of in patients with sickle cell disease.

Methods: For this retrospective cohort study, we collected data from 90 US centres reported to the Center for International Blood and Marrow Transplant Research.

Peptide hydrogel in vitro non-inflammatory potential.

Peptide-based hydrogels have attracted significant interest in recent years as these soft, highly hydrated materials can be engineered to mimic the cell niche with significant potential applications in the biomedical field. Their potential use in vivo in particular is dependent on their biocompatibility, including their potential to cause an inflammatory response. In this work, we investigated in vitro the inflammatory potential of a β-sheet forming peptide (FEFEFKFK; F: phenylalanine, E: glutamic acid; K: lysine) hydrogel by encapsulating murine monocytes within it (3D culture) and using the production of cytokines, IL-β, IL-6 and TNFα, as markers of inflammatory response.

Non-melanoma skin cancer: United Kingdom National Multidisciplinary Guidelines.

This is the official guideline endorsed by the specialty associations involved in the care of head and neck cancer patients in the UK. This paper provides consensus recommendations on the management of cutaneous basal cell carcinoma and squamous cell carcinoma in the head and neck region on the basis of current evidence. Recommendations • Royal College of Pathologists minimum datasets for NMSC should be adhered to in order to improve patient care and help work-force planning in pathology departments.

11 kGy gamma irradiated demineralized bone matrix enhances osteoclast activity.

Background: Demineralized bone matrix (DBM) allografts are widely used in orthopaedic clinics. However, the biological impact on its osteoinductivity after its sterilization process by gamma irradiation is not well studied. Furthermore, little is known about the relationship between residual calcium levels on osteoinductivity.

Current concepts and advances in the application of tissue engineering in otorhinolaryngology and head and neck surgery.

Objective: This paper reviews the progress in the rapidly expanding scientific discipline of tissue engineering, which may have an integral role in the future of otorhinolaryngology. This article seeks to inform on the current concepts and principles of tissue engineering, and describe the state of the art research and developments in this exciting field as applied to ENT and head and neck surgery.

Method: In order to carry out a comprehensive review of the literature spanning the past 30 years, a search of relevant publications was performed using the Web of Knowledge, Medline and PubMed databases.

Current opinion on auricular reconstruction.

Purpose Of Review: To review the current practice in the field of auricular reconstruction and to highlight the recent advances reported in the medical literature.

Recent Findings: The majority of surgeons who perform auricular reconstruction continue to employ the well-established techniques developed by Brent and Nagata. Surgery takes between two and four stages, with the initial stage being construction of a framework of autogenous rib cartilage which is implanted into a subcutaneous pocket.

The role of nervus intermedius in side specific nasal responses.

Background: Nervus intermedius (NI) dysfunction is common in patients who have had vestibular schwannoma (VS) surgery. Such patients have a unilateral parasympathetic-denervated nasal cavity. A number of side-specific nasal reflexes have been demonstrated in normal individuals, including hand cold-water immersion.

A prospective audit to look at the number of plain sinus x-rays requested via the radiology department.

Chronic rhinosinusitis is currently diagnosed on history-taking with nasendoscopic confirmation. Sinus x-rays are insensitive and non-specific but are still requested, particularly by primary care physicians. The rate of sinus x-rays can be reduced by informing GPs of current best practice guidelines.

A natural obturator in hereditary haemorrhagic telangiectasia.

Objective: Most patients with hereditary haemorrhagic telangiectasia suffer with frequent episodes of epistaxis. The aim of this case report is to highlight the effect on epistaxis, occurring in hereditary haemorrhagic telangiectasia, when nasal airflow ceases.

Case Report: We present the interesting case of a patient with hereditary haemorrhagic telangiectasia who experienced cessation of her recurrent, refractory epistaxis through the development of coexisting polyp disease.

Non-auditory stimulation in adult cochlear implant users.

This is a retrospective case review of all 163 adults to have received multichannel cochlear implants on the Manchester University/Manchester Royal Infirmary programme between 1988 and 1998. The aims were to investigate the incidence of unwanted non-auditory effects of electrical stimulation (NAS) of the cochlea, and to try to identify any factors that seemed to be related to these effects. The effectiveness of programming strategies in eliminating unwanted effects was also studied.

Rhinitis due to food allergies: fact or fiction?

Food allergy is often thought to be a cause of rhinitis by patients, whose views are frequently influenced by information obtained from the Internet. The incidence of food allergy in children is 2-8 per cent, although a vast majority will grow out of it, and food allergy is actually very rare in adults. Food allergy usually presents with multi-system involvement, most commonly cutaneous and gastrointestinal symptoms.

Activated eosinophils in nasal polyps: a comparison of asthmatic and non-asthmatic patients.

Objectives: There is a recognized clinical association between nasal polyps and asthma. Nasal polyps and the airways of asthmatic patients demonstrate marked eosinophilia suggesting that this inflammatory cell may have a key role to play in both conditions. The objective of this study was to determine whether nasal polyps from patients with asthma had a greater density of activated eosinophils than patients with no associated respiratory disease.

Treatment of home oxygen induced rhinitis: an unusual use for a nasal obturator.

Patients requiring home oxygen can experience drying and crusting of nasal mucosa. On occasion it can be severe causing significant discomfort. In this report we present such a case.

Vascular endothelial growth factor in nasal polyps: a comparison of asthmatic and non-asthmatic patients.

The cause of nasal polyps remains unknown, although there is a well-recognized clinical association between nasal polyposis and asthma. The characteristic histological features of nasal polyps include large quantities of extracellular fluid. Vascular endothelial growth factor (VEGF) is a potent mediator of angiogenesis and vascular permeability.

The rhinological side-effects of systemic drugs.

Patients often present to otolaryngologists with nasal symptoms where no cause is apparent. A number of patients seen in outpatient departments are taking medication for other conditions and the adverse affects of these drugs may potentially be the source of these symptoms. In this short review, we present an overview of the more common drugs that may be responsible and outline the possible mechanisms where these are known.

Endoscopic bipolar diathermy in the management of epistaxis: an effective and cost-efficient treatment.

Epistaxis is one of the most frequently managed otorhinolaryngological emergencies. This prospective study over a 4-month period involved 38 adult patients presenting with epistaxis who underwent endoscopic bipolar diathermy under local anaesthesia. Thirty-four (89%) of the 38 adults were successfully treated and 28 (74%) of the patients did not require admission.

Informed consent for septal surgery: the evidence-base.

Informed consent is becoming an increasingly important issue in patient care. Inherent in this is the ability to accurately inform patients of the risks and complications involved in surgery. Nasal septal surgery is commonly performed by otolaryngologists and although often considered routine has a number of recognized complications that can have a significant effect on life quality.