Browsing by Subject "patient-reported outcomes"

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  • Sousa-Pinto, Bernardo; Azevedo, Luis Filipe; Sa-Sousa, Ana; Vieira, Rafael Jose; Amaral, Rita; Klimek, Ludger; Czarlewski, Wienczyslawa; Anto, Josep M.; Bedbrook, Anna; Kvedariene, Violeta; Ventura, Maria Teresa; Ansotegui, Ignacio J.; Bergmann, Karl-Christian; Brussino, Luisa; Canonica, G. Walter; Cardona, Victoria; Carreiro-Martins, Pedro; Casale, Thomas; Cecchi, Lorenzo; Chivato, Tomas; Chu, Derek K.; Cingi, Cemal; Costa, Elisio M.; Cruz, Alvaro A.; De Feo, Giulia; Devillier, Philippe; Fokkens, Wytske J.; Gaga, Mina; Gemicioglu, Bilun; Haahtela, Tari; Ivancevich, Juan Carlos; Ispayeva, Zhanat; Jutel, Marek; Kuna, Piotr; Kaidashev, Igor; Kraxner, Helga; Larenas-Linnemann, Desiree E.; Laune, Daniel; Lipworth, Brian; Louis, Renaud; Makris, Michael; Monti, Riccardo; Morais-Almeida, Mario; Moesges, Ralph; Mullol, Joaquim; Odemyr, Mikaela; Okamoto, Yoshitaka; Papadopoulos, Nikolaos G.; Patella, Vincenzo; Nhan Pham-Thi; Regateiro, Frederico S.; Reitsma, Sietze; Rouadi, Philip W.; Samolinski, Boleslaw; Sova, Milan; Todo-Bom, Ana; Taborda-Barata, Luis; Tomazic, Peter Valentin; Toppila-Salmi, Sanna; Sastre, Joaquin; Tsiligianni, Ioanna; Valiulis, Arunas; Wallace, Dana; Waserman, Susan; Yorgancioglu, Arzu; Zidarn, Mihaela; Zuberbier, Torsten; Fonseca, Joao Almeida; Bousquet, Jean; Pfaar, Oliver (2022)
    Background Evidence regarding the effectiveness of allergen immunotherapy (AIT) on allergic rhinitis has been provided mostly by randomised controlled trials, with little data from real-life studies. Objective To compare the reported control of allergic rhinitis symptoms in three groups of users of the MASK-air(R) app: those receiving sublingual AIT (SLIT), those receiving subcutaneous AIT (SCIT), and those receiving no AIT. Methods We assessed the MASK-air(R) data of European users with self-reported grass pollen allergy, comparing the data reported by patients receiving SLIT, SCIT and no AIT. Outcome variables included the daily impact of allergy symptoms globally and on work (measured by visual analogue scales-VASs), and a combined symptom-medication score (CSMS). We applied Bayesian mixed-effects models, with clustering by patient, country and pollen season. Results We analysed a total of 42,756 days from 1,093 grass allergy patients, including 18,479 days of users under AIT. Compared to no AIT, SCIT was associated with similar VAS levels and CSMS. Compared to no AIT, SLIT-tablet was associated with lower values of VAS global allergy symptoms (average difference = 7.5 units out of 100; 95% credible interval [95%CrI] = -12.1;-2.8), lower VAS Work (average difference = 5.0; 95%CrI = -8.5;-1.5), and a lower CSMS (average difference = 3.7; 95%CrI = -9.3;2.2). When compared to SCIT, SLIT-tablet was associated with lower VAS global allergy symptoms (average difference = 10.2; 95%CrI = -17.2;-2.8), lower VAS Work (average difference = 7.8; 95%CrI = -15.1;0.2), and a lower CSMS (average difference = 9.3; 95%CrI = -18.5;0.2). Conclusion In patients with grass pollen allergy, SLIT-tablet, when compared to no AIT and to SCIT, is associated with lower reported symptom severity. Future longitudinal studies following internationally-harmonised standards for performing and reporting real-world data in AIT are needed to better understand its 'real-world' effectiveness.
  • Homsy, S. Pauliina; Uimonen, Mikko M.; Lindford, Andrew J.; Repo, Jussi P.; Lassus, Patrik A. (2021)
    The FACE-Q Rhinoplasty module is a patient-reported outcome instrument developed for the assessment of primarily aesthetic outcomes of rhinoplasty. The aim of our study was to produce a Finnish version of the instrument and validate it for use in patients undergoing nasal reconstruction as well as those treated with a rhinoplasty. Finnish versions of the FACE-Q scales Satisfaction with Nose, Satisfaction with Nostrils and Adverse Effects: Nose, were translated following established guidelines. Patients undergoing nasal resection, reconstruction or rhinoplasty in Helsinki University Hospital plastic surgery department in 2009-2019 were identified using theatre records. A total of 240 Finnish-speaking patients 18-85 years old were approached with a postal survey questionnaire. The questionnaire included the translated FACE-Q modules and those for Satisfaction with Facial Appearance, Appearance-Related Psychosocial Distress and Satisfaction with Outcome, as well as the general health-related quality of life instrument 15 D. The FACE-Q scales translated readily to Finnish. Eighty-three patients (35%) responded to the survey. Most FACE-Q scales performed well with high internal consistency (Cronbach's alphas 0.87-0.92) and repeatability. Only the Adverse Effects: Nose scale displayed poor consistency and a floor effect with 18% of the patients reporting no adverse outcomes. Answers to the Appearance-Related Psychosocial Distress scale were skewed towards no experienced stress. Answers to the other scales were normally distributed with weak correlation with 15 D dimensions. The Finnish translations of the FACE-Q Rhinoplasty scales perform well at assessing a diverse group of patients including those undergoing nasal reconstruction as well as those undergoing rhinoplasty.
  • Cork, Michael J.; Eckert, Laurent; Simpson, Eric L.; Armstrong, April; Barbarot, Sebastien; Puig, Luis; Girolomoni, Giampiero; de Bruin-Weller, Marjolein; Wollenberg, Andreas; Kataoka, Yoko; Remitz, Anita; Beissert, Stefan; Mastey, Vera; Ardeleanu, Marius; Chen, Zhen; Gadkari, Abhijit; Chao, Jingdong (2020)
    Background: Atopic dermatitis (AD) profoundly affects quality of life (QoL). Dupilumab significantly improves clinical outcomes, is well tolerated, and approved to treat inadequately controlled moderate-to-severe AD in adults; however, its effect on patient-reported outcomes (PROs) is not fully characterized. Objective: To evaluate the impact of dupilumab on patient-reported AD symptoms and QoL. Methods: Pooled data were analyzed from two identically designed phase 3 studies, LIBERTY AD SOLO 1 (NCT02277743) and SOLO 2 (NCT02277769), assessing the following PROs: Peak Pruritus Numerical Rating Scale (NRS), Pruritus Categorical Scale, SCORing AD (SCORAD), Dermatology Life Quality Index (DLQI), Patient-Oriented Eczema Measure (POEM), Hospital Anxiety and Depression Scale (HADS), five-dimension EuroQoL questionnaire (EQ-5D), and patient-assessed disease status and treatment effectiveness. Results: Dupilumab rapidly improved (vs. placebo) Peak Pruritus NRS scores by day 2 (p <.05), anxiety and depression (HADS), and QoL (DLQI) by week 2, and maintained through week 16 (p <.0001). At week 16, more dupilumab-treated than placebo-treated patients reported improvement in SCORAD itch and sleep, and no pain/discomfort (EQ-5D) (p <.0001). Limitations: Cultural differences of translated PROs. Conclusion: Dupilumab had a significant, positive impact on AD symptoms, including itch, sleep, pain, anxiety and depression, and QoL in adults with moderate-to-severe AD.
  • Eriksson, Daniel; Karlsson, Linda; Eklund, Oskar; Dieperink, Hans; Honkanen, Eero; Melin, Jan; Selvig, Kristian; Lundberg, Johan (2017)
    Background. A limited number of studies have assessed health related quality of life (HRQoL) in autosomal dominant polycystic kidney disease (ADPKD). Results to date have been conflicting and studies have generally focused on patients with later stages of the disease. This study aimed to assess HRQoL in ADPKD across all stages of the disease, from patients with early chronic kidney disease (CKD) to patients with end-stage renal disease. Methods. A study involving cross-sectional patient-reported outcomes and retrospective clinical data was undertaken April December 2014 in Denmark, Finland, Norway and Sweden. Patients were enrolled into four mutually exclusive stages of the disease: CKD stages 1-3; CKD stages 4-5; transplant recipients; and dialysis patients. Results. Overall HRQoL was generally highest in patients with CKD stages 1-3, followed by transplant recipients, patients with CKD stages 4-5 and patients on dialysis. Progressive disease predominately had an impact on physical health, whereas mental health showed less variation between stages of the disease. A substantial loss in quality of life was observed as patients progressed to CKD stages 4-5. Conclusions. Later stages of ADPKD are associated with reduced physical health. The value of early treatment interventions that can delay progression of the disease should be considered.
  • Mikkola, Heidi (Helsingin yliopisto, 2021)
    Pharmacotherapy plays a key role in the treatment of many conditions. Long-term medication therapy is an essential part of treatment in many common chronic conditions in Finland, such as diabetes, asthma, and cardiovascular diseases. However, medication therapy can be burdensome to patients and thus, influence their functional capacity and well-being. Therefore, patients’ lived experience of medication and its effect on their lives has a growing interest as a research area. The primary aim of this study was to test among Finnish patients with chronic conditions the consistency of a theoretical model ‘Patient’s Lived Experience with Medicine’ (PLEM) developed by a qualitative meta-synthesis by Mohammed et al. (2016). The secondary aim was to investigate the medication-related burden experienced by Finnish patients living with different chronic conditions. Based on the results of the content analysis of the focus group discussions, this study aimed to create a new, concise measure of medication-related burden to be utilized in a population-based online survey on medication use in Finland called Medicine Barometer (Lääkebarometri) by the Finnish Medicines Agency Fimea. The study was conducted as a qualitative focus group interview in summer 2020. Participated patients with chronic conditions (n=14) were recruited through four patient organizations. Focus group discussions (n=5) were held in groups of 2-3 people over Zoom. Deductive content analysis guided by PLEM model was used for data analysis. The measure of medication-related burden was formed on the grounds of the original PLEM model, content analysis of the interviews, research literature and expertise of the research group. The results of the focus group discussions (n=5) supported the functionality of the PLEM model. Study participants (n=14) described similar experiences of medication-related burden and beliefs and practices guiding the medication taking to those presented in the PLEM model. Burden caused by medication routines and healthcare system were most emphasized of the factors contributing to medication-related burden. As a new factor contributing to the burden, medication-related eco-anxiety emerged. The new measure for assessing medication-related burden consists of 13 items to be piloted by the Finnish Medicines Agency Fimea in the summer 2021. The experiences of the Finnish patients with chronic conditions are consistent with the PLEM model. Majority of the participants did not experience such burden from their medication that would significantly interfere with their daily lives. However, experiencing higher level of medication-related burden appeared to be related to independently modifying medication regimen or even neglecting the use of medicines. PLEM model and related patient interviews served as a solid foundation for developing items for the new measure to be piloted for assessing medication-related burden. The population-based survey will provide useful data for the further development of the measure and for researching the factors contributing to the burden.
  • Hammer, Hilde Berner; Hansen, Inger Marie Jensen; Järvinen, Pentti; Leirisalo-Repo, Marjatta; Ziegelasch, Michael; Agular, Birte; Terslev, Lene (2021)
    Objectives. Given that subjective variables might reduce remission by composite DAS (CDAS), the main objectives were to explore whether RA patients with mainly tender vs mainly swollen joints had differences in patient-reported outcome measures (PROMs), clinical or US assessments or in achieving remission defined by CDAS or US. Methods. In a Nordic multicentre study, RA patients initiating tocilizumab were assessed by PROMs, clinical, laboratory and US assessments (36 joints and 4 tendons) at baseline, 4, 12 and 24 weeks. Remission was defined according to clinical disease activity index (CDAI)/Boolean or no Doppler activity present. Tender-swollen joint differences (TSJDs) were calculated. Statistics exploring changes over time/differences between groups included Wilcoxon, Mann-Whitney, Kruskal-Wallis and Spearman tests. Results. One hundred and ten patients were included [mean (S.D.) age 55.6 (12.1) years, RA duration 8.7 (9.5) years]. All PROMs, clinical, laboratory and US scores decreased during follow-up (P < 0.001). During follow-up, tender joint counts were correlated primarily with PROMs [r = 0.24-0.56 (P < 0.05-0.001)] and swollen joint counts with US synovitis scores [r = 0.33-0.72 (P < 0.05-0.001)]. At 24 weeks, patients with TSJD > 0 had higher PROMs and CDAI (P < 0.05-0.001) but lower US synovitis scores (P < 0.05). Remission by CDAI/Boolean was seen in 26-34% and by Doppler 53%, but only 2-3% of patients with TSJD > 0 achieved CDAI/Boolean remission. Conclusion. Patients with more tender than swollen joints scored higher on subjective assessments but had less US synovitis. They seldom achieved CDAS remission despite many being in Doppler remission. If patients with predominantly tender joints do not reach CDAS remission, objective assessments of inflammation should be performed.