scholarly journals Patient-reported complications, symptoms and experiences of living with X-linked hypophosphatemia across the life-course

2021 ◽  
Author(s):  
Moira Cheung ◽  
Angela J Rylands ◽  
Angela Williams ◽  
Karen Bailey ◽  
Judith Bubbear
Keyword(s):  
Life Course ◽  
Information Needs ◽  
Online Survey ◽  
Group Analysis ◽  
Clinical Signs ◽  
Signs And Symptoms ◽  
Appropriate Treatment ◽  
Patient Reported ◽  
Clinical Signs And Symptoms ◽  
The Life Course

Abstract Background X-linked hypophosphatemia (XLH) is a rare, genetic phosphate-wasting disease resulting in bone, muscular and dental problems, beginning in childhood and increasing in adulthood. This qualitative analysis aimed to explore patient-reported symptoms, complications and experiences of XLH over the life-course, using data from a large multinational online survey. Methods Responses to two open-ended questions from 209 adults and 86 children/adolescents (proxy report) with self-reported XLH were analyzed in eight age groups. Two researchers independently coded and analyzed the responses, using thematic analysis, with differences settled among a multi-disciplinary group. Six themes were identified, with age sub-group analysis conducted on the three most common, according to coding frequency. Results Within theme 1, ‘Clinical Signs and Symptoms of XLH’, ‘Pain’ was a dominant sub-theme across the life-course, but ‘Skeletal Pathology’ dominated the responses of children/adolescents. Within theme 2, ‘Impacts of Clinical Signs and Symptoms’, interference with ‘Physical Exertion’ and ‘Emotional Wellbeing’ (comprising depression/anxiety in adults and lack of self-esteem in children/adolescents) was reported across all ages. For theme 3, ‘Negative Treatment Experiences’, ‘Medication’ was problematic for children, with adults reporting lack of ‘Access to Appropriate Treatment’. Three further themes were identified: ‘Resilience’, ‘Positive Treatment Experiences’, and ‘Information Needs.’ Conclusion The multiple burdens imposed on people with XLH throughout their lifetime encompassed the physical, emotional and social, although the most challenging symptoms or complications differed between ages. Burden was further exacerbated by adult’s lack of access to appropriate treatment, illustrating the need for age-appropriate multidisciplinary care.

scholarly journals Correction to: Levoketoconazole improves clinical signs and symptoms and patient-reported outcomes in patients with Cushing’s syndrome

Pituitary ◽  
2020 ◽  
Author(s):  
Eliza B. Geer ◽  
Roberto Salvatori ◽  
Atanaska Elenkova ◽  
Maria Fleseriu ◽  
Rosario Pivonello ◽  
...  
Keyword(s):  
Cushing’S Syndrome ◽  
Patient Reported Outcomes ◽  
Clinical Signs ◽  
Signs And Symptoms ◽  
Cushing's Syndrome ◽  
Original Version ◽  
Patient Reported ◽  
Author Group ◽  
Clinical Signs And Symptoms

The original version of the article unfortunately contained an error in the first name and the surname of one of the authors in the author group. The last author name was incorrectly published as ‘F. Pecori Giraldi’ and the corrected name is ‘Francesca Pecori Giraldi’ (First name: Francesca; Surname: Pecori Giraldi).

scholarly journals Levoketoconazole improves clinical signs and symptoms and patient-reported outcomes in patients with Cushing’s syndrome

Pituitary ◽  
2020 ◽  
Author(s):  
Eliza B. Geer ◽  
Roberto Salvatori ◽  
Atanaska Elenkova ◽  
Maria Fleseriu ◽  
Rosario Pivonello ◽  
...  
Keyword(s):  
Cushing’S Syndrome ◽  
Patient Reported Outcomes ◽  
Clinical Signs ◽  
Maintenance Phase ◽  
Signs And Symptoms ◽  
Cushing's Syndrome ◽  
Baseline Score ◽  
Peripheral Edema ◽  
Patient Reported ◽  
Clinical Signs And Symptoms

Abstract Purpose The efficacy of levoketoconazole in treating hypercortisolism was demonstrated in an open-label phase 3 study (SONICS) of adults with endogenous Cushing’s syndrome (CS) and baseline mean urinary free cortisol (mUFC) ≥  1.5× ULN. Clinical signs and symptoms and patient-reported outcomes from the SONICS trial were evaluated in the current manuscript. Methods Patients titrated to an individualized therapeutic dose entered a 6-month maintenance phase. Secondary endpoints included investigator-graded clinical signs and symptoms of CS during the maintenance phase, and patient-reported quality of life (CushingQoL questionnaire) and depression symptoms (Beck Depression Inventory II [BDI-II]). Results Of 94 enrolled patients, 77 entered the maintenance phase following individualized dose titration. Significant mean improvements from baseline were noted at end of maintenance (Month 6) for acne, hirsutism (females only), and peripheral edema. These improvements were observed as early as Day 1 of maintenance for hirsutism (mean baseline score, 7.8; ∆ − 1.9; P < 0.0001), end of Month 1 for acne (mean baseline score, 2.8; ∆ − 1.2; P = 0.0481), and Month 4 for peripheral edema (mean baseline score, 1.0; ∆ − 0.5; P = 0.0052). Significant mean improvements from baseline were observed by Month 3 of maintenance for CushingQoL (mean baseline score, 44.3; ∆ + 6.9; P = 0.0018) and at Month 6 for BDI-II (mean baseline score, 17.1; ∆ − 4.3; P = 0.0043) scores. No significant mean improvement was identified in a composite score of 7 other clinical signs and symptoms. Conclusions Treatment with levoketoconazole was associated with sustained, meaningful improvements in QoL, depression, and certain clinical signs and symptoms characteristic of CS. ClinialTrials.gov identifier: NCT01838551.

scholarly journals Primary Manifestation of Sarcoidosis Involving the Gingivae

2012 ◽  
Vol 1 (2) ◽  
pp. 102-105 ◽  
Author(s):  
Catherine Saylor ◽  
Nabil Beaini ◽  
James Rokos ◽  
Keerthana Satheesh ◽  
Charles Cobb
Keyword(s):  
Clinical Signs ◽  
Signs And Symptoms ◽  
Definitive Diagnosis ◽  
Anatomical Location ◽  
Case Presentation ◽  
Histologic Analysis ◽  
Appropriate Treatment ◽  
Clinical Signs And Symptoms

ABSTRACT Sarcoidois of the gingiva is rarely the primary manifestation of the disease. The following case presentation highlights clinical signs and symptoms that are not uncommon or unusual except for the anatomical location of the initial expression. Appropriate treatment must be based on a definitive diagnosis which, in turn, requires a biopsy for histologic analysis. How to cite this article Saylor C, Beaini N, Rokos J, Satheesh K, Cobb C. Primary Manifestation of Sarcoidosis Involving the Gingivae. Int J Experiment Dent Sci 2012;1(2):102-105.

scholarly journals Successful treatment of herpes simplex-associated erythema multiforme with a combination of acyclovir and prednisone

2017 ◽  
Vol 2 (3) ◽  
pp. 191
Author(s):  
Arny E. Muryah ◽  
Irna Sufiawati
Keyword(s):  
Erythema Multiforme ◽  
Complete Blood Count ◽  
Leukocyte Adhesion ◽  
Clinical Success ◽  
Clinical Signs ◽  
Signs And Symptoms ◽  
The Body ◽  
Vascular Integrity ◽  
Patient Reported ◽  
Clinical Signs And Symptoms

Objective: Erythema Multiforme (EM) is an acute mucocutaneous hypersensitivity reaction triggered by certain infections and medications. EM induced by virus infection termed as Herpes- associated Erythema Multiforme (HAEM). The use of corticosteroids for treating HAEM has been a long debate issue. The aim of this case report is to present a patient with HAEM who was successfully treated with acyclovir combined with prednisone.Methods:A 31-years-old female, presented with a complaint of pain and diffuse ulcers in mouth. The patient reported recurrent episode of the ulcers of the mouth during the last two years. On extra oral examination, lips were crusted and bleeding. Intraoral examination revealed multiple diffuse ulcerations on mucosa. Nolesions were seen in other part of the body. Laboratory investigation revealed normal complete blood count and positive HSV-1 serology. The patient was diagnosed as HAEM.The patient was treated with acyclovir (1000 mg/day) and prednisone (10mg/day), topical mixture corticosteroid, chlorhexidine gluconate 0.2% and multivitaminduring seven days.Results:All lesions healed without any further clinical sequelae within 7 days.The clinical success of corticosteroids as effective anti-inflammatory agents is largely attributed to their ability to reduce the expression of pro-inflammatory genes, help to maintain vascular integrity and decrease the expression of leukocyte adhesion molecules.Conclusions:The addition of prednisone to acyclovir for HAEM resulted in a significant reduction of clinical signs and symptoms during the first week. The combination of acyclovir and corticosteroids may play an important role in the standard care for HAEM.

Conjunctivitis

2022 ◽  
pp. 53-101
Keyword(s):  
Clinical Signs ◽  
Stable Condition ◽  
Signs And Symptoms ◽  
Cellular Infiltration ◽  
Ligneous Conjunctivitis ◽  
Appropriate Treatment ◽  
Before And After ◽  
Clinical Signs And Symptoms ◽  
Basic Approaches

Conjunctivitis represents an inflammation of conjunctiva with cellular infiltration, exudation, and vascular dilation. According to the course of the disease, conjunctivitis can be acute, hyperacute, and chronic. Morphologically, conjunctivitis can appear with papillary reaction, follicular reaction, cicatrizing, granulomatous and membranous changes. This chapter discusses all types of conjunctivitis, their clinical signs and symptoms, and basic approaches of treatment. This chapter includes before and after treatment photos of atypical inferiorly localized shield ulcer, Tularemia-associated Parinaud oculoglandular syndrome, and Stevens-Johnson disease. Pictures are included In the ligneous conjunctivitis patient's case taken at diagnosis as well as 10 years later, demonstrating stable condition with appropriate treatment throughout the period.

scholarly journals A Clinical Study to Evaluate the Effect of Amrita Guggulu and Guduchi Kwath in the Management of Vatarakta w.s.r. to Gout

AYUSHDHARA ◽  
2021 ◽  
pp. 3473-3481
Author(s):  
Vijay Chaudhary ◽  
Ankita Agrawal
Keyword(s):  
Clinical Signs ◽  
Visceral Obesity ◽  
Signs And Symptoms ◽  
Plain Water ◽  
Major Life ◽  
Lower Socioeconomic ◽  
Mild Improvement ◽  
Patient Reported ◽  
Clinical Signs And Symptoms ◽  
Trial Drug

In present time, lifestyle disorders are rapidly rising in our society. Over consumption of food, alcohol and fructose sweetened soft drinks in combination with a sedentary lifestyle has resulted in increased prevalence of visceral obesity, metabolic syndrome and gout in all socioeconomic groups of society including the lower socioeconomic status. Gout is one of the major life style disorders. The clinical manifestation and textual references of gout may be attributed with 'Vatarakta', also known as Vatashonita. Vata and Rakta are the Dosha and Dushya respectively which are primarily involved in the pathology of Vatarakta. Virudhaahar and Vihara aggravate Vata dosha and Rakta dhatu. Provocated Vata gets Aavrut with vitiated Rakta dhatu leading to the beginning of pathophysiological cascade of Vatarakta. The patients for this study were diagnosed based on Ayurvedic and modern parameters. Clinical signs and symptoms described in classical texts were considered for the diagnosis of Vatarakta, whereas serum uric acid was considered as investigation based diagnostic tool. Total 15 patients were registered in trial. The selected patients were given the trial drug i.e., Amrita guggulu one gram and Guduchi kwath 50ml twice a day with plain water after food. Out of 15 registered patients, 1 dropout from the trial, 4 patients (28.57%) showed marked improvement, 7 patients i.e., 50% showed moderate improvement and 3 patients 21.43% showed mild improvement. None of patient in present clinical trial remained unimproved or deteriorated during the clinical trials. In the present study, none of the patient reported any adverse effect to the trial drug during study and follow up period.

scholarly journals Dementia and Major Neurocognitive Disorders: Some Lessons Learned One Century after the first Alois Alzheimer’s Clinical Notes

Geriatrics ◽  
2021 ◽  
Vol 6 (1) ◽  
pp. 5
Author(s):  
Donatella Rita Petretto ◽  
Gian Pietro Carrogu ◽  
Luca Gaviano ◽  
Lorenzo Pili ◽  
Roberto Pili
Keyword(s):  
Young Woman ◽  
Clinical Signs ◽  
Signs And Symptoms ◽  
Neurocognitive Disorders ◽  
Lessons Learned ◽  
Alzheimer Dementia ◽  
Clinical Notes ◽  
Alois Alzheimer ◽  
Clinical Signs And Symptoms

Over 100 years ago, Alois Alzheimer presented the clinical signs and symptoms of what has been later called “Alzheimer Dementia” in a young woman whose name was Augustine Deter [...]

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