Background: Elexacaftor-tezacaftor-ivacaftor (ETI) is a novel, highly effective CFTR modulator combination proven to enhance lung function and body weight in people with cystic fibrosis (pwCF) carrying a F508del mutation. Recently, we revealed significant reductions in abdominal symptoms (AS) in German, British, and Irish pwCF after 24-26 weeks of ETI using the CFAbd-Score, the first patient-reported outcome measure (PROM) specifically developed and validated for pwCF following FDA guidelines. Notably, many pwCF reported marked changes in their AS during the first days of the new treatment. To capture these immediate effects, we developed the CFAbd-day2day, a CF-specific GI-diary, following FDA and COSMIN guidelines. Aim: To prospectively capture the immediate dynamics of AS using the CFAbd-day2day 14 days before and 14-28 days after ETI initiation. In addition, we aim to provide validation steps of the novel PROM concerning sensitivity to changes. Methods: To develop the CFAbd-day2day, focus groups (community voice = pwCF and their proxies and CF specialists from different fields) were repeatedly consulted. Before and during the new ETI therapy, pwCF prospectively scored AS on a daily basis with the CFAbd-day2day. Results: Altogether, 45 pwCF attended in five CF centers prospectively completed the CFAbd-day2day before (mean ± sd:14 ± 7 days) and after (mean ± sd: 28 ± 23 days) ETI initiation. On the one hand, cumulative scores significantly decreased during the 3-4-week time frame after ETI initiation, compared to 2 weeks prior to therapy. On the other hand, many patients who revealed a relatively stable level of AS before ETI reported changes during the first days of treatment with the highly effective CFTR modulators. Factors like pain and flatulence increased in up to 21% of patients during the first 14 days of therapy, but they improved during days 15-27. Conclusion: The CFAbd-day2day, specifically developed and in the process of validation to prospectively capture GI symptoms in pwCF, provides new substantial insights into the dynamics of AS in pwCF receiving a new treatment with ETI. This novel tool is also helpful in prospectively monitoring patients with specific GI problems. International implementation and further validation steps of the diary are ongoing.

UNASSIGNED: Prognostics for patients with cancer is especially important for the supportive care of those who are terminally ill. We previously found that symptom scores as patient-reported outcomes (PROs)-such as dyspnea and fatigue scores-some biochemical parameters, the palliative performance scale (PPS) scores, and symptom clusters were useful prognostic factors; however, the predictability of a prognosis based on these factors remains unclear.
UNASSIGNED: To identify appropriate three-week survival predictive factor(s), in terms of performance, in patients who were terminally ill.
UNASSIGNED: We collected symptom scores as PROs using the Japanese version of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 15 Palliative Care (EORTC QLQ-C15-PAL).
UNASSIGNED: We used data from terminally ill patients with cancer who were hospitalized at the palliative care unit of the Higashisumiyoshi-Morimoto Hospital (Osaka, Japan) from June 2018 to December 2019 (n = 130), as well as additional data obtained from the same clinical study from January to March 2020 (n = 31).
UNASSIGNED: To evaluate predictive performance, indices such as sensitivity, specificity, positive predictive value, negative predictive value, and overall accuracy were calculated.
UNASSIGNED: We found that the presence of a symptom cluster showed high sensitivity but low specificity and that a higher PPS value (>30) showed high specificity but low sensitivity, suggesting that these factors could provide relevant information for survival prognosis (less than or equal to three weeks).
UNASSIGNED: Symptom clusters obtained from patients is important for effective supportive care of those who are terminally ill.

IgE-mediated atopic diseases such as allergic rhinitis and rhinoconjunctivitis are common chronic diseases in the western world. Allergen immunotherapy (AIT) plays a fundamental role in the treatment of allergic patients by modulating the underlying immune mechanisms. Though this treatment is integrated in practice-patterns globally, many differences are found in the application of AIT on the national or international level due to heterogeneous methods, and clinical recommendations are given in different parts of the world. This review from authors in Europe and the United States highlights differences and similarities in important aspects of AIT application in the 2 global regions. First, the regulatory situation differs regarding marketing authorization and licensing. Secondly, differences are elaborated in manufacturing practices, marketing distribution and formulations of AIT products. Thirdly, clinical administration patterns in the current guidelines show similarities in indications and contraindications of AIT, but also are divergent in some practical aspects. Informing the readership on similarities, as well as differences of standards in AIT in the United States and Europe, the authors highlight the unmet need of thorough harmonization of standards of AIT, as it is the only disease modifying treatment option available for patients with allergic rhinitis and rhinoconjunctivitis.

[This corrects the article DOI: 10.3389/fphar.2022.877118.].

Psychological disturbances are frequent following COVID-19. However, there is not much information about whether pre-existing psychological disorders are associated with the severity and evolution of COVID-19. We aimed to explore the associations between regular psychotropic medication use (PM) before infection as a proxy for mood or anxiety disorders with COVID-19 recovery trajectories. We used data from the Predi-COVID study. We followed adults, tested positive for SARS-CoV-2 and collected demographics, clinical characteristics, comorbidities and daily symptoms 14 days after inclusion. We calculated a score based on 16 symptoms and modeled latent class trajectories. We performed polynomial logistic regression with PM as primary exposure and the different trajectories as outcome. We included 791 participants, 51% were men, and 5.3% reported regular PM before infection. We identified four trajectories characterizing recovery dynamics: \"Almost asymptomatic,\" \"Quick recovery,\" \"Slow recovery,\" and \"Persisting symptoms\". With a fully adjusted model for age, sex, socioeconomic, lifestyle and comorbidity, we observed associations between PM with the risks of being in more severe trajectories than \"Almost Asymptomatic\": \"Quick recovery\" (relative risk (95% confidence intervals) 3.1 (2.7, 3.4), \"Slow recovery\" 5.2 (3.0, 9.2), and \"Persisting symptoms\"11.7 (6.9, 19.6) trajectories. We observed a gradient of risk between PM before the infection and the risk of slow or no recovery in the first 14 days. These results suggest that a pre-existing psychological condition increases the risk of a poorer evolution of COVID-19 and may increase the risk of Long COVID. Our findings can help to personalize the care of people with COVID-19.

OBJECTIVE: Vulvar lichen sclerosus (LS) is a chronic, progressive, autoimmune dermatologic condition that causes cutaneous changes accompanied by pruritus and pain. There remains a small population with vulvar LS refractory to topical corticosteroids. Injection of platelet-rich plasma (PRP) has been reported to have positive effects on tissue repair. The aim of this pilot study was to evaluate changes in symptom scores during and after PRP vulvar infiltration.
METHODS: Three PRP infiltrations were administered to 28 female postmenopausal patients with biopsy-proved LS with unsatisfactory response to steroid therapy. Change in score according to the Clinical Scoring System for Vulvar Lichen Sclerosus (CSS) was measured on six occasions over the course of a year. We used growth curve modeling to measure change over the period of the study.
RESULTS: Women in our study experienced a statistically significant improvement in auto-assessed symptoms of vulvar lichen sclerosus, and this improvement appears to be maintained throughout the monitoring year.
CONCLUSIONS: Platelet-rich plasma may have a role in symptom relief in certain cases of patients with LS that do not respond to first-line therapy.

Background: The novel and highly effective CFTR modulator combination of elexacaftor-tezacaftor-ivacaftor (ETI) has been shown to improve lung function and body weight in people with Cystic Fibrosis (pwCF) carrying a F508del mutation. However, the impact of these modulators on gastrointestinal (GI) symptoms is relatively unknown. Therefore, the CFAbd-Score was developed and validated following FDA recommendations for development of a PROM including focus groups, multidisciplinary CF specialists, people with CF and their families. The aim of this study was to assess effects of ETI on GI symptoms using the CFAbd-Score. Methods: Gastrointestinal symptoms were prospectively assessed in pwCF using the CFAbd-Score before and up to 26 weeks during therapy. The CFAbd-Score was also administered to a healthy control (HC) group. The one-sided questionnaire includes 28 items grouped in five domains. Data analysis included calculation of scores with a weighting tool, developed according to FDA recommendations. Results: A total of 107 pwCF attended in four CF centres in Germany and four centres in the UK completed the CFAbd-Score on at least two occasions. Results were compared to those obtained from the questionnaire of 45 HCs. Despite differences in demographics, age and proportion of pancreatic insufficiency between German and UK patients, analyses based on linear mixed-effects models at week 24 of ETI therapy revealed that estimated marginal means (EMMs) of total CFAbd-Scores significantly reduced (mean ± SE: 14.9 ± 1.2→10.6 ± 1.4; p < 0.01). Also EMMs of all five domains significantly declined (\"pain\" 16.3 ± 1.6→10.2 ± 2.3, \"GERD\" 15.8 ± 1.8→8.2 ± 1.9, \"disorders of bowel movement\" 20.9 ± 1.5→16.0 ± 1.7, \"disorders of appetite\" 7.9 ± 1.1→2.6 ± 1.1 and \"quality of life impairment\" 10.1 ± 1.92→3.9 ± 1.9). However, during 24 weeks, CF participants\' symptoms mostly still did not reach the reference levels of HCs. Discussion: Using the CFAbd-Score, the first PROM specifically developed for assessment of CF-related abdominal symptoms, we demonstrate comprehensive improvements in GI symptoms after initiation of the highly effective modulator therapy ETI.

BACKGROUND: Myasthenia Gravis (MG) is a disorder of neuromuscular transmission bringing mild ocular weakness to severe generalized muscle weakness and disability. The conventional treatments have long-term side effects, and Chinese herbal medicines (CHM) have shown possible effect and safety for MG patients, but the existing evidence was not robust enough and the results were out of date.
METHODS: Searching for randomized controlled trials (RCTs) was conducted in 7 databases and clinical trial registries until July 2021. The ROB 2 tool was used to assess the study quality and GRADE was used to assess the quality of whole evidence. Meta-analyses were conducted and the results were presented as risk ratio (RR) or mean difference (MD) with 95% confidence interval (CI).
RESULTS: Nineteen RCTs (1283 participants) testing 13 kinds of CHM with adequate randomization were included and six RCTs investigating Compound Huangqi were included in the meta-analyses. In addition to conventional treatment, nine CHMs reduced symptom scores of MG. Compound Huangqi plus conventional treatment (pyridostigmine bromide or prednisone or both) reduced the symptom scores compared with conventional treatment (MD = -3.56, 95%CI -4.86 to -2.26). Less adverse events happened in the CHM groups (3/247 in the CHM groups, 52/245 in the control groups, RR = 0.13, 95%CI 0.06 to 0.30, 9 RCTs, a total of 492 participants). The effect on quality of life was inconsistent.
CONCLUSIONS: Nine CHMs could probably bring benefit for MG symptom improvement. Moderate to low certainty of evidence supported Compound Huangqi added-on conventional treatment probably bring extra benefit of improving MG symptoms. Adding CHMs could be safer than giving only conventional treatment.
BACKGROUND: The protocol was registered in PROSPERO (ID: 32718).

OBJECTIVE: To observe clinical effects of Tiaoqi Jiangni Decoction combined with mosapride citrate tablets in the treatment of functional dyspepsia and its impact on quality of life.
METHODS: Seventy-six patients with functional dyspepsia were admitted to Cangzhou Central Hospital of Hebei Province from September 2019 to June 2020. They were selected and randomized into a treatment group and a control group (38 cases in each group). Both groups received mosapride citrate tablets (5 mg/time, 3 times/d). The treatment group was additionally given Tiaoqi Jiangni Decoction.
RESULTS: The total effective rate of the treatment group was higher than that of the control group (P < 0.05). After treatment, the sub-scores of each symptom and the overall score of traditional Chinese medicine (TCM) syndrome in the treatment group were significantly lower than those in the control group (all P < 0.05). The overall and sub-item scores of the Functional Digestive Disorders Quality of Life Questionnaire (FDDQL) of both groups saw an increase after treatment (all P < 0.05). There was no significant disparity in sleep and stress between the two groups. The treatment group presented a better performance in other aspects than the control group (all P < 0.05). Before treatment, the two groups showed no significant disparity in the gastric emptying rate of barium bar and mental and psychological state. The two indexes were observed with a rise after treatment, with superior results in the treatment group to those in the control group (P < 0.05). No adverse reactions were observed in the two groups. One month after treatment, the treatment group obtained a lower recurrence rate as compared to the control group (P < 0.05).
CONCLUSIONS: Tiaoqi Jiangni Decoction in combination with mosapride citrate tablets in the treatment of FD yields a significant clinical effect by substantially alleviating patients\' clinical symptoms and improving their quality of life, with no adverse reactions, high safety, and low recurrence rate, which merits further clinical application.
BACKGROUND: The name of the registry: Chinese Registry of Clinical Trials.
BACKGROUND: ChiCTR2100063542. Trial URL: http://www.chictr.org.cn/showproj.aspx?proj=6354283.

UNASSIGNED: To evaluate medium/long term outcomes and patient satisfaction through relief of symptoms and improved quality of life (QoL) after Jones tube conjunctivodacryocystorhinostomy (JT-CDCR) using the Naso Lacrimal Duct Obstruction symptom-score (NLDO-SS).
UNASSIGNED: We conducted a retrospective, non-comparative, multicentric study including patients with complete obstruction of the superior and inferior proximal lacrimal drainage system. All patients underwent JT-CDCR, and the patency of the tube was evaluated with saline irrigation and endoscopic examination. We assessed patient satisfaction and quality of life administering the NLDO-SS.
UNASSIGNED: We enrolled 16 patients, for a total of 21 eyes operated. The success rate for procedures was 81%. The success rate for single parameters was globally 95.9%; if considered separately, ocular symptoms and nasal symptoms were respectively 94.3% and 100%.
UNASSIGNED: JT-CDCR was a reliable procedure, able to solve symptoms in a majority of patients and guaranteed a good quality of life over a long period of time.
L’applicazione di un questionario sui sintomi dopo congiuntivodacriocistorinostomia: risultati.
UNASSIGNED: Valutare gli esiti a lungo termine e la soddisfazione del paziente, attraverso la risoluzione della sintomatologia e il miglioramento della qualità della vita (QoL) dopo intervento di congiuntivodacriocistorinostomua e posizionamento di Tubo di Jones CDCR-JT, mediante l’utilizzo del questionario “Naso Lacrimal Duct Obstruction Symptoms-Score (NLDO-SS)”.
UNASSIGNED: Abbiamo condotto uno studio retrospettivo, non comparativo, multicentrico, includendo pazienti con ostruzione completa della lacrimale prossimale superiore e inferiore. Tutti i pazienti sono stati sottoposti a JT-CDCR, la pervietà del tubo di Jones è stata valutata con irrigazione di soluzione fisiologia ed endoscopia trans-nasale. A ciascun paziente è stato poi somministrato il questionario NLDO-SS.
UNASSIGNED: Abbiamo arruolato 16 pazienti, per un totale di 21 occhi operati. Il tasso di successo delle procedure è stato dell’81%. Il tasso di successo per i singoli parametri è stato globalmente del 95,9%; se considerati separatamente, i sintomi oculari e quelli nasali erano rispettivamente del 94,3% e del 100%.
UNASSIGNED: L’intervento di JT-CDCR ha dimostrato di essere in grado di risolvere i sintomi nella maggior parte dei pazienti e di garantire una buona qualità di vita per un lungo periodo di tempo.