Patients with non-idiopathic sudden sensorineural hearing loss show hearing improvement more often than patients with idiopathic sudden sensorineural hearing loss.

INTRODUCTION: To compare inpatient treated patients with idiopathic (ISSNHL) and non-idiopathic sudden sensorineural hearing loss (NISSNHL) regarding frequency, hearing loss, treatment and outcome. METHODS: All 574 inpatient patients (51% male, median age: 60 years) with ISSNHL and NISSNHL, who were treated in federal state Thuringia in 2011 and 2012, were included retrospectively. Univariate and multivariate statistical analyses were performed. RESULTS: ISSNHL was diagnosed in 490 patients (85%), NISSNHL in 84 patients (15%). 49% of these cases had hearing loss due to acute otitis media, 37% through varicella-zoster infection or Lyme disease, 10% through Menière disease and 7% due to other reasons. Patients with ISSNHL and NISSNHL showed no difference between age, gender, side of hearing loss, presence of tinnitus or vertigo and their comorbidities. 45% of patients with ISSNHL and 62% with NISSNHL had an outpatient treatment prior to inpatient treatment (p < 0.001). The mean interval between onset of hearing loss to inpatient treatment was shorter in ISSNHL (7.7 days) than in NISSNHL (8.9 days; p = 0.02). The initial hearing loss of the three most affected frequencies in pure-tone average (3PTAmax) scaled 72.9 dBHL ± 31.3 dBHL in ISSNHL and 67.4 dBHL ± 30.5 dBHL in NISSNHL. In the case of acute otitis media, 3PTAmax (59.7 dBHL ± 24.6 dBHL) was lower than in the case of varicella-zoster infection or Lyme disease (80.11 dBHL ± 34.19 dBHL; p = 0.015). Mean absolute hearing gain (Δ3PTAmaxabs) was 8.1 dB ± 18.8 dB in patients with ISSNHL, and not different in NISSNHL patients with 10.2 dB ± 17.6 dB. A Δ3PTAmaxabs ≥ 10 dB was reached in 34.3% of the patients with ISSNHL and to a significantly higher rate of 48.8% in NISSNHL patients (p = 0.011). CONCLUSIONS: ISSNHL and NISSNHL show no relevant baseline differences. ISSNHL tends to have a higher initial hearing loss. NISSHNL shows a better outcome than ISSNHL.

Inpatient treatment of patients with idiopathic sudden sensorineural hearing loss: a population-based healthcare research study.

PURPOSE: The aim was to determine inpatient treatment rates of idiopathic sudden sensorineural hearing loss (ISSNHL) with focus on diagnostics, treatment, and outcome. METHODS: A retrospective population-based study in the federal state Thuringia in 2011 and 2012 was performed on all 490 inpatients (51% females, median age: 60 years) treated for ISSNHL (Median duration: 7 days). The association between analyzed parameters and the probability of recovery was tested using univariable and multivariable statistics. RESULTS: The inpatient treatment rate for ISSNHL was 11.23 per 100,000. 172 patients (35%) had an outpatient treatment prior to inpatient treatment. For pure-tone audiometry of the three most affected frequencies (3PTAmax), the initial median hearing loss was 66.67 dB, the median absolute hearing gain ΔPTAabs was 10.0 dB, and the median relative hearing gain in relation with the contralateral side ΔPTArel contral was 30.86%. 51% of the patients reached a ΔPTAabs of ≥ 10 dB. About 2 of 5 patients recovered to a ΔPTArel contral ≥ 50% or reached ≤ 10 dB of contralateral ear. The multivariate analysis revealed that an ISSNHL on the left side [Hazard ratio (HR) = 1.6.88; confidence interval (CI) = 1.161-2.454], no down-sloping audiogram type (HR = 2.016; CI = 1.391-2.921), and no prior outpatient prednisolone treatment (HR = 2.374; CI = 1.505-3.745) were independent factors associated with better recovery (ΔPTAabs ≥ 10 dB). CONCLUSION: Inpatient treatment of ISSNHL is variable in daily practice. The population-based recovery rate was worse than reported in clinical trials. More standardization and clearer criteria for outpatient, inpatient, and salvage therapy are needed.

[Management of vestibular schwannomas]. / Management des Vestibularisschwannoms.

Vestibular schwannomas are benign neoplasms originating from the Schwann cells of the vestibular vestibular nerve of the vestibulocochlear nerve, and rarely from the pars cochlearis. These are tumors that are in contact with the nerve but do not bind the fibers. Benign neoplasms of the Schwann cells of the auditory and equilibrium nerves can also occur primarily in the inner ear and are referred to as intralabyrinthine schwannomas (ILS). Vestibular schwannomas represent 6-7 % of all intracranial and 90 % of cerebellopontine angle tumors. Bilateral occurrence occurs in < 5 % of cases, and then corresponds to type 2 neurofibromatosis. The first symptom is often a unilateral hearing loss. It may then lead to balance disorders, tinnitus, facial paralysis and other impairments. Diagnosis is audiological, vestibular and imaging. Magnetic resonance imaging currently represents the gold standard. Management chooses between an observational strategy and surgery, depending on tumor size, age, and other factors. The possible access routes offer different advantages and disadvantages; the potential complications include the liquorrhoea. Radiation therapy is possible in special cases, and drug therapies are also being tested. In the rehabilitation of the hearing function, in addition to a CROS or BICROS restoration, the cochlear implant has been used with good success. The impact on quality of life is largely determined by hearing impairment, balance disorders, tinnitus, and possibly headache, which must be considered in patient consultation and long-term care.

Psychometric properties of the St George's Respiratory Questionnaire in patients with idiopathic pulmonary fibrosis.

The St George's Respiratory Questionnaire (SGRQ) has been used to measure health-related quality of life (HRQoL) in patients with idiopathic pulmonary fibrosis (IPF).This analysis evaluated the psychometric properties of the SGRQ using data from 428 patients with IPF who participated in a 12-month, randomised, placebo-controlled phase II trial of nintedanib.Internal consistency (Cronbach's α) was 0.91 for SGRQ total and >0.70 for domain scores. Test-retest reliability (intraclass correlation coefficients) was 0.77, 0.77, 0.69 and 0.66 for SGRQ total, activity, impact and symptoms scores, respectively. Construct validity of SGRQ total and domain scores was supported by weak to moderate cross-sectional correlations with the Medical Research Council dyspnoea scale (0.32-0.55), 6-min walk test distance (-0.25- -0.34), percentage predicted forced vital capacity (-0.11- -0.15) and measures of gas exchange (-0.26-0.03). There was some evidence that the SGRQ total score was sensitive to detecting change.The reliability, construct validity and responsiveness of the SGRQ in patients with IPF suggest that this is an acceptable measure of HRQoL in patients with IPF.

Evaluation of optimal masking levels in place-specific low-frequency chirp-evoked auditory brainstem responses.

The aim of the study is the experimental determination of the optimal required masking level for a given stimulus level when using a band limited "low-frequency chirp" in order to improve frequency and place specificity of auditory brainstem responses (ABRs). A low-frequency chirp (100-850 Hz) at stimulation levels between 40 and 80 dB normalized hearing level was presented to 12 normal hearing subjects. During presentation of each stimulus, the level of a high-pass noise with a low cutoff frequency of 1100 Hz was varied between 0 and 25 dB signal-to-noise ratio (SNR) by using 5 dB steps (at 0 dB SNR the same level of both the chirp and the masker in dB sound pressure level was presented). Measurements without masking were used as a reference. In all masking conditions, the latency of wave V was significantly increased compared to unmasked ABRs. The amplitude of wave V decreased when reaching the effective and therefore optimal masking level. Accordingly, in order to ensure place specificity of the ABR, ipsilateral masking is essential. At lower stimulus levels the SNR can be substantially increased (i.e., the masker level decreased) without loss of place specificity.

The accuracy of objective threshold determination at low frequencies: comparison of different auditory brainstem response (ABR) and auditory steady state response (ASSR) methods.

OBJECTIVE: The hearing threshold at 500 Hz was estimated using five methods which are suitable for the low frequency range: Low-Chirp BERA (LCBERA), Notched-noise BERA (NNBERA), Narrow band CE-Chirp BERA (NBCBERA) and Narrow band CE-Chirp ASSR (NBCASSR) (40/90 Hz). The slope of the discrimination function of each method was used for determination of the most efficient method. The threshold values were compared and the corresponding odds ratios (OR) were calculated. DESIGN: All methods were applied to each subject. Stimulus levels were arranged individually. Response detection was carried out by visual inspection of the records in case of BERA and automatically in case of ASSR. Each individual series of recordings was converted to a dichotomous function indicating whether or not a response was discernible and a continuous method-specific discrimination function was constructed. This function was realised by a Boltzmann function whose slope in the inflection point serves as quality measure. Additionally, an OR evaluation was carried out in order to validate the significance of results. STUDY SAMPLE: Twenty five normal hearing adults (aged 18-30 years) were tested. RESULTS: LCBERA proved to have the highest reliability according to the slope of the Boltzmann function, the comparison of threshold values and OR. CONCLUSIONS: The LCBERA is recommended for use in routine clinical practice.

Using one filter stage of unsaturated/saturated vertical flow filters for nitrogen removal and footprint reduction of constructed wetlands.

French vertical flow constructed wetlands (VFCW) treating raw wastewater have been developed successfully over the last 30 years. Nevertheless, the two-stage VFCWs require a total filtration area of 2-2.5 m2/P.E. Therefore, implementing a one-stage system in which treatment performances reach standard requirements is of interest. Biho-Filter® is one of the solutions developed in France by Epur Nature. Biho-Filter® is a vertical flow system with an unsaturated layer at the top and a saturated layer at the bottom. The aim of this study was to assess this new configuration and to optimize its design and operating conditions. The hydraulic functioning and pollutant removal efficiency of three different Biho-Filter® plants commissioned between 2011 and 2012 were studied. Outlet concentrations of the most efficient Biho-Filter® configuration are 70 mg/L, 15 mg/L, 15 mg/L and 25 mg/L for chemical oxygen demand (COD), 5-day biological oxygen demand (BOD5), total suspended solids (TSS) and total Kjeldahl nitrogen (TKN), respectively. Up to 60% of total nitrogen is removed. Nitrification efficiency is mainly influenced by the height of the unsaturated zone and the recirculation rate. The optimum recirculation rate was found to be 100%. Denitrification in the saturated zone works at best with an influent COD/NO3-N ratio at the inflet of this zone larger than 2 and a hydraulic retention time longer than 0.75 days.

[Direct Microlaryngoscopy: A Population-Based Analysis on Outcome, Complications and Surgery Rates]. / Mikrolaryngoskopie: Eine populationsbezogene Analyse zu Outcome, Komplikationen und Operationsraten.

Although microlaryngoscopy is an integral part of surgical routine of otorhinolaryngologists, there is no population-based data published on surgery rates and efficiency of microlaryngoscopy country-wide or nation-wide. All 616 patients who underwent microlaryngoscopy 2011 in one of the eight ENT departments in Thuringia were analyzed according to patients' characteristics, therapy, complications and follow-up. The majority of admissions were performed because of a benign disease (60%) of the larynx, and in 33% related to a malignant disease or suspicion of a malignant disease. When a benign disease was suspected, it was confirmed 98% of cases. When a malignant tumor was suspected, it was confirmed in 51% of cases, i. e. ruled out in 49% of cases. Transient laryngeal edema (22%) and bleeding needing revision surgery (1%) were the most frequent or serve observed postoperative sequelae. Teeth damage occurred only in 2 cases (0.2%). A recurrence of the primary disease was observed in 14%. Longer surgery time was an independent predictor for postoperative bleeding and for postoperative laryngeal edema (p=0.050 and p=0.013, respectively). Revision surgery (p<0.0001) and a final diagnosis of a malignant disease (p=0.017) were independent predictors for recurrence of the primary disease. The overall incidence of microlaryngoscopy was 22.98/100000 population. The highest incidence was seen for patients 50-59 years of age with 39.76/100000. Benign diseases were the most frequent indication with 19.33/100000. This population-based analysis is showing that microlaryngoscopy is performed effectively and with low postoperative risks in daily routine of otorhinolaryngologists.

Treatment patterns, resource use and costs of idiopathic pulmonary fibrosis in Spain--results of a Delphi Panel.

BACKGROUND: Idiopathic pulmonary fibrosis (IPF) is a form of chronic fibrosing interstitial pneumonia characterized by progressive worsening of dyspnea and lung function, with a poor prognosis. The objective of this study was to determine treatment patterns, resource use and costs of managing Spanish patients with IPF. METHODS: A three-round Delphi consensus panel of 15 clinical experts was held between December 2012 and June 2013 using questionnaires to describe the management of patients with IPF. A cost analysis based on Delphi panel estimates was made from the Spanish National Health System (NHS) perspective, including the direct costs of IPF diagnosis and management. Unit costs were applied to Delphi panel estimates of health resource use. Univariate sensitivity analyses were made to evaluate uncertainties in parameters. RESULTS: The Delphi panel estimated that 20, 60 and 20% of IPF patients presented with stable disease, slow and rapid disease progression, respectively. The estimated annual cost per patient with stable disease, slow and rapid disease progression was €11,484, €20,978 and €57,759, respectively. This corresponds to a weighted average annual cost of €26,435 with itemized costs of €1,184 (4.5), €7,147 (27.0), €5,950 (22.5), €11,666 (44.1) and €488 (1.9%) for the diagnosis of IPF, treatment, monitoring, management of acute exacerbations and end-of-life care, respectively. The parameter that varied the annual cost per patient the most was resource use associated with acute exacerbations. CONCLUSIONS: The management of patients with IPF in Spain, especially patients with rapid disease progression, has a high economic impact on the NHS.

Cardiac comorbidity in head and neck cancer patients and its influence on cancer treatment selection and mortality: a prospective cohort study.

Comorbidity assessment and a profound cardiac examination were implemented into pre-treatment diagnostics to analyze their influence on head and neck squamous cell carcinoma (HNSCC) therapy selection and short-term mortality. Comorbidity was measured prospectively in 49 HNSCC patients using standard indices between 2012 and 2013. Cardiac examinations included electrocardiogram, echocardiography, and bicycle ergometry. Most patients had stage IV tumors (61 %), smoked (61 %), and showed alcohol abuse (57 %); 38 patients (78 %) received a multimodal therapy; 65 % had an adult comorbidity evaluation 27 index ≥2, 59 % a Charlson comorbidity index (CCI) ≥4, and 12 % a revised cardiac risk index (RCRI) ≥2. Additional cardiac examinations revealed moderate to severe cardiovascular pathologies in 32 % of the patients and led to recommendations for additional therapy in 4 patients (8 %) necessary only after cancer treatment. RCRI was associated with CCI and cardiac examinations (p = 0.009, p = 0.030). Chemotherapy, stroke history, and RCRI ≥2 were risk factors for early mortality within first 2 years after cancer therapy (p = 0.037; p = 0.012; p = 0.015). Although one-third of a strongly smoking and drinking patient cohort had relevant cardiac morbidity, extended unselected cardiac diagnostics had only low impact on HNSCC therapy selection. The risk of early mortality after HNSCC cancer treatment seems to be sufficiently reflected by the RCRI.

Magnitude of effect of asthma treatments on Asthma Quality of Life Questionnaire and Asthma Control Questionnaire scores: Systematic review and network meta-analysis.

BACKGROUND: The Asthma Quality of Life Questionnaire (AQLQ) and the Asthma Control Questionnaire (ACQ) are widely used in asthma research; however, in studies of newer asthma treatments, mean improvements in these measures compared with placebo arms do not exceed the minimal important difference (MID), particularly when a new treatment is added to current treatment. OBJECTIVE: We performed a systematic review and network meta-analysis to examine the magnitude of AQLQ and ACQ responses achieved with commonly used asthma drugs and factors influencing these end points in clinical trials. METHODS: A systematic literature search was conducted to identify blinded randomized controlled trials reporting AQLQ or ACQ results. Mixed treatment comparisons, combined with meta-regression, were then performed. RESULTS: Of the 64 randomized controlled trials (42,527 patients) identified, 54 included the AQLQ and 11 included the ACQ as end points. The presence of a run-in period, the nature of treatment during the run-in period, concurrent treatment during the treatment period, and instrument version significantly influenced the change in AQLQ score from baseline and whether it exceeded the MID. When compared with placebo, only inhaled corticosteroids (ICSs), with or without a long-acting ß-agonist, achieved the MID. The ACQ results were comparable with those of the AQLQ: no differences from placebo exceeded the MID, and ICS-based treatments provided the greatest improvements. CONCLUSION: The established within-patient MID for the ACQ and AQLQ is not achievable as a group-wise efficacy threshold between treatment arms in clinical studies in which controllers are added to ICS treatment. Thus in addition to reporting mean changes of the instruments, other measurement criteria should be considered, including responder analyses.

The psychometric properties of the St George's Respiratory Questionnaire (SGRQ) in patients with idiopathic pulmonary fibrosis: a literature review.

Assessment of health-related quality of life (HRQL) is particularly important in patients with progressive and incurable diseases such as idiopathic pulmonary fibrosis (IPF). The St George's Respiratory Questionnaire (SGRQ) has frequently been used to measure HRQL in patients with IPF, but it was developed for patients with obstructive lung diseases. The aim of this review was to examine published data on the psychometric performance of the SGRQ in patients with IPF. A comprehensive search was conducted to identify studies reporting data on the internal consistency, construct validity, test-retest reliability, and interpretability of the SGRQ in patients with IPF, published up to August 2013. In total, data from 30 papers were reviewed. Internal consistency was moderate for the SGRQ symptoms score and excellent for the SGRQ activity, impact and total scores. Validity of the SGRQ symptoms, activity, impact and total scores was supported by moderate to strong correlations with other patient-reported outcome measures and with a measure of exercise capacity. Most correlations were moderately strong between SGRQ activity or total scores and forced or static vital capacity, the most commonly used marker of IPF severity. There was evidence that changes in SGRQ domain and total scores could detect within-subject improvement in health status, and differentiate groups of patients whose health status had improved, declined or remained unchanged. Although the SGRQ was not developed specifically for use with patients with IPF, on balance, its psychometric properties are adequate and suggest that it may be a useful measure of HRQL in this patient population. However, several questions remain unaddressed, and further research is needed to confirm the SGRQ's utility in IPF.

Lung transplantation in idiopathic pulmonary fibrosis: a systematic review of the literature.

BACKGROUND: Idiopathic pulmonary fibrosis (IPF) is a distinct form of interstitial pneumonia with unknown origin and poor prognosis. Current pharmacologic treatments are limited and lung transplantation is a viable option for appropriate patients. The aim of this review was to summarize lung transplantation survival in IPF patients overall, between single (SLT) vs. bilateral lung transplantation (BLT), pre- and post Lung Allocation Score (LAS), and summarize wait-list survival. METHODS: A systematic review of English-language studies published in Medline or Embase between 1990 and 2013 was performed. Eligible studies were those of observational design reporting survival post-lung transplantation or while on the wait list among IPF patients. RESULTS: Median survival post-transplantation among IPF patients is estimated at 4.5 years. From ISHLT and OPTN data, one year survival ranged from 75% - 81%; 3-year: 59% - 64%; and 5-year: 47% - 53%. Post-transplant survival is lower for IPF vs. other underlying pre-transplant diagnoses. The proportion of IPF patients receiving BLT has steadily increased over the last decade and a half. Unadjusted analyses suggest improved long-term survival for BLT vs. SLT; after adjustment for patient characteristics, the differences tend to disappear. IPF patients account for the largest proportion of patients on the wait list and while wait list time has decreased, the number of transplants for IPF patients has increased over time. OPTN data show that wait list mortality is higher for IPF patients vs. other diagnoses. The proportion of IPF patients who died while awaiting transplantation ranged from 14% to 67%. While later transplant year was associated with increased survival, no significant differences were noted pre vs. post LAS implementation; however a high LAS vs low LAS was associated with decreased one-year survival. CONCLUSIONS: IPF accounts for the largest proportion of patients awaiting lung transplants, and IPF is associated with higher wait-list and post-transplant mortality vs. other diagnoses. Improved BLT vs. SLT survival may be the result of selection bias. Survival pre- vs. post LAS appears to be similar except for IPF patients with high LAS, who have lower survival compared to pre-LAS. Data on post-transplant morbidity outcomes are sparse.

Proxy-reported questionnaires for young children with asthma: a structured review.

Which proxy-reported outcome measures have been developed for use with children aged 6 years and younger to assess asthma symptoms, asthma control, and asthma-specific health-related quality of life, and do these questionnaires' measurement properties support their use as end-points in clinical trials? A two-phase literature search was conducted: 1) studies describing relevant questionnaires were identified, and the questionnaires were assessed against predefined criteria; 2) studies providing information on the measurement properties of questionnaires meeting the predefined inclusion criteria were identified. Literature sources included PubMed and EMBASE databases, scientific conference proceedings, a clinical trial registry, and a quality of life instrument database. The initial search of literature databases and conference abstracts identified 631 records. 20 paediatric asthma proxy-reported outcome instruments were identified; seven met the inclusion criteria: Childhood Asthma Control Test, Control de Asma en Niños Questionnaire, Pediatric Asthma Caregiver Diary, Pediatric Asthma Control Tool, PedsQL 3.0 Short-Form 22 Asthma Module, PedsQL Asthma Symptoms Scale, and Test for Respiratory and Asthma Control in Kids. Three proxy-reported outcome instruments were considered suitable for use as end-points in paediatric asthma clinical trials; the Pediatric Asthma Caregiver Diary possesses the strongest measurement properties of the three.

Development and Validation of a Remote Monitoring Tool for Assessment of Mild, Moderate, and Severe Infections in Inflammatory Bowel Disease.

BACKGROUND: Immunomodulators and biologics are cornerstones in the management of inflammatory bowel disease [IBD], but are associated with increased risk of infections. Post-marketing surveillance registries are pivotal to assess this risk, yet mainly focus on severe infections. Data on the prevalence of mild and moderate infections are scarce. We developed and validated a remote monitoring tool for real-world assessment of infections in IBD patients. METHODS: A 7-item Patient-Reported Infections Questionnaire [PRIQ] covering 15 infection categories was developed with a 3-month recall period. Infection severity was defined as mild [self-limiting or topical treatment], moderate [oral antibiotics, antivirals, or antifungals], or severe [hospitalisation or intravenous treatment]. Comprehensiveness and comprehensibility were ascertained through cognitive interviewing of 36 IBD outpatients. After implementation in the telemedicine platform myIBDcoach, a prospective, multicentre cohort study was performed between June 2020 and June 2021 in 584 patients, to assess diagnostic accuracy. Events were cross-checked with general practitioner and pharmacy data [gold standard]. Agreement was evaluated using linear-weighted kappa with cluster-bootstrapping to account for within-patient level correlation. RESULTS: Patient understanding was good and interviews did not result in reduction of PRIQ items. During validation, 584 IBD patients {57.8% female, mean age 48.6 (standard deviaton [SD]: 14.8), disease duration 12.6 years [SD: 10.9]} completed 1386 periodic assessments, reporting 1626 events. Linear-weighted kappa for agreement between PRIQ and gold standard was 0.92 (95% confidence interval [CI] 0.89-0.94). Sensitivity and specificity for infection [yes/no] were 93.9% [95% CI 91.8-96.0] and 98.5% [95% CI 97.5-99.4], respectively. CONCLUSIONS: The PRIQ is a valid and accurate remote monitoring tool to assess infections in IBD patients, providing means to personalise medicine based on adequate benefit-risk assessments.

Early mucosal healing with infliximab is associated with improved long-term clinical outcomes in ulcerative colitis.

BACKGROUND & AIMS: In the Active Ulcerative Colitis Trial (ACT)-1 and ACT-2, patients with ulcerative colitis treated with infliximab were more likely than those given placebo to have a clinical response, undergo remission, and have mucosal healing. We investigated the association between early improvement (based on endoscopy) and subsequent clinical outcome. METHODS: Patients underwent endoscopic evaluations at weeks 0, 8, 30, and 54 (ACT-1 only), and were categorized into 4 subgroups by week 8 (Mayo endoscopy subscore, 0-3). The association of week 8 endoscopy subscores, subsequent colectomy risk, symptoms and corticosteroid use outcomes were analyzed. Mucosal healing was defined as a Mayo endoscopy subscore of 0 (normal) or 1 (mild). RESULTS: Infliximab-treated patients with lower week 8 endoscopy subscores were less likely to progress to colectomy through 54 weeks of follow-up evaluation (P=.0004). This trend was not observed among patients given placebo (P=.47). Patients with lower endoscopy subscores achieved better symptomatic and corticosteroid use outcomes at weeks 30 and 54 (P<.0001, infliximab; P<.01, placebo). Among patients who achieved clinical response at week 8, trends in subsequent clinical outcomes by week 8 endoscopy subscores were generally consistent with that for the overall patient population; no trends were observed among patients who achieved clinical remission. CONCLUSIONS: The degree of mucosal healing after 8 weeks of infliximab was correlated with improved clinical outcomes including colectomy. Similar trends were observed for all outcomes except colectomy among the subgroup with clinical response at week 8. The degree of mucosal healing at week 8 among those in clinical remission did not predict subsequent disease course.

Symptom Experience and Content Validity of the Psoriasis Symptom Scale (PSS) in Patients with Generalized Pustular Psoriasis (GPP).

INTRODUCTION: We sought to understand key symptoms of generalized pustular psoriasis (GPP) and to confirm the relevance to patients and content validity of the Psoriasis Symptom Scale (PSS) in GPP. METHODS: A targeted literature review and clinical expert interviews were conducted as background research. Patients were interviewed individually (involving concept elicitation and cognitive interviews), and a separate patient workshop was conducted to determine disease-specific symptoms of importance. RESULTS: Seven participants with moderate (n = 4), severe (n = 2), and mild (n = 1) GPP and clinician diagnosis were interviewed. During concept elicitation, all participants indicated that pustules may underlie other symptoms. Symptoms reported by all patients were pain, redness, itch, burning, and discomfort. The PSS symptoms of pain, itching, burning, and redness were reported by ≥ 86% of patients as most frequently experienced. Upon debriefing, the PSS was well understood. Relevance and importance of these symptoms was confirmed in the GPP patient workshop. CONCLUSION: Participant feedback found the PSS measure to be relevant and easy to understand. The symptoms included in the instrument, pain, redness, itch, and burning, were most frequently reported, important, and well understood by patients. Study results provided support for the content validity of the PSS for use as endpoints in GPP clinical trials.

Generalized pustular psoriasis (GPP) is a severe rare disease, including redness and boils that sometimes come with fever and other general symptoms. This study asked patients with GPP about their key symptoms, and whether the Psoriasis Symptom Scale (PSS) is relevant to them as patients. The PSS is a questionnaire with the symptoms pain, itching, burning, and redness. We searched the literature and interviewed clinical experts to guide the patient interviews. Patients were recruited through clinical sites and the National Psoriasis Foundation (NPF). The interviews discussed GPP symptoms and the PSS questionnaire. Patients with GPP were also asked about commonly experienced symptoms in a workshop. Most patients had moderate to severe GPP. Patients in both the interviews and workshop described experiencing pain, redness, itch, burning, and discomfort with their boils. During interviews, the patients said the PSS questionnaire was easy to understand. Patients in the workshop also found the PSS to be relevant and easy to understand. Patients agreed the symptoms in the PSS, pain, redness, itch, and burning, were common and important. Study results support the PSS for use with patients in clinical trials.

The clinical, humanistic, and economic burden of palmoplantar pustulosis: a structured review.

Introduction: Palmoplantar pustulosis (PPP) is a chronic, relapsing and refractory disease characterized by sterile pustules appearing on the palms and/or soles, accompanied by erythema, blistering, scales and/or keratinization. The overall burden of PPP in terms of its clinical impact, effect on patients and families, and economic consequences has not previously been investigated in a structured manner.Areas covered: A structured search focused on identification of studies in PPP using specific search terms in PubMed and EMBASE® from 2005 onwards, with additional back-referencing and pragmatic searches. Outcomes of interest included clinical burden, humanistic burden, and economic burden.Expert opinion: In cross-sectional studies, approximately 75% of all PPP patients suffer from active disease, with risk of relapse remaining constant over time. Patients' health-related quality of life is significantly impaired, as expected for a disease affecting hands and feet. Tools have been described that assess the clinical as well as patient-reported burden of PPP; their performance in larger cohorts and/or clinical trials remains to be investigated. The key data limitations identified include inconsistent definitions for characterizing remission/relapse, and limited humanistic and economic burden data; future studies are required to address these evidence gaps.