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BackgroundResearch on non-pharmacological treatments for fibromyalgia patients has demonstrated that exercise and education have positive effects on pain and disability1. However, the traditional approach of studying treatment effectiveness based exclusively on quantitative measures has been questioned. There is growing recognition of the importance of integrating patients` perspectives into clinical research. Outcome measures focused on patients` perceptions of improvement have been increasingly used, however they do not offer expanded definitions of what constitutes a “successful” outcome2. The further exploration of patients` perspectives about treatment benefits may contribute to the development of treatments that better match patients` needs.ObjectivesThe aim of this study was twofold: firstly, to examine the effectiveness of a group physiotherapy programme (incorporating exercise and education) on pain intensity, disability and global impression of change in fibromyalgia patients; and, to explore how patients, who achieved success in pain and/or disability (according to the score from the Patient Global Impression of Change Scale - PGIC), understand and make sense of the results.MethodsA sequential explanatory mixed methods approach, combining quantitative (1 st phase) and qualitative methods (2nd phase), was carried out. In the 1 st phase, the participants underwent an 8 week (3 times weekly) standardised group programme. Participants were assessed at baseline, 4 and 8 weeks later. Outcome measures included the Numeric Pain Rating Scale (NPRS), the Revised Fibromyalgia Impact Questionnaire (FIQR) and the PGIC. Participants who had scored ≥5 in the PGIC were invited to participate in a focus group. In the 2nd phase, 2 focus groups meetings were carried out to collect data. A semi-structured interview schedule was used and the sessions were audiotaped and transcribed verbatim.ResultsThirty-seven participants (females; 49.3±10.2 years) completed the 1 st phase. Analysis using SPSS revealed statistically significant improvements on pain intensity (mean SD change:−1.38±2.363,p=0.001) and disability (−21.577±21.02,p<0.001). In what concerns to participants` impression of change, 26 participants (70.3%) perceived substantial improvements on pain and disability (PGIC ≥5). From these, 12 accepted to participate in the focus groups. The qualitative analysis indicated that “doing more daily-life activities” and “taking less medication” were identified as the main reasons for the participants` improvements. According to these participants, the knowledge about strategies for self-management played a key role in their success.ConclusionsThe combination of data from both phases provided detailed information about the participants` perceptions regarding the key elements for achieving success with a physiotherapy programme. Further research on patients` perspectives regarding treatment effectiveness is recommended since it may contribute to the design of more effective and patient-centred treatments.References[1] Macfarlane...

Objectives: To apply indirect comparison methods to inform decision-making when standard of care has low level of evidence available. Methods: Simulated treatment comparison is one of the methods available to generate indirect comparison of data in different formats and levels of evidence. It is particularly helpful when patient level data exists for one set of data and there is the need to compare it with single-arm studies. In theses cases evidence is deemed ''unanchored'' due to a lack of a common comparator. We use the case of second line treatment of chronic immune thrombocytopenic purpura (ITP). Splenectomy has been historically recommended in guidelines mainly due to the lack of evidence for other second-line therapies for ITP. We use individual patient data from eltrombopag clinical trials and one of the only studies published in the literature reporting long term outcomes for splenectomy to generate an indirect comparison of eltrombopag and splenectomy in second line treatment of ITP. Results: A subset of patients (n=84) treated with eltrombopag but not subject to splenectomy in the randomised, phase 3 study RAISE (PMID:20739054) were indirectly compared with a large retrospective cohort of patients (n=233) who underwent splenectomy for ITP in the study by Vianelli et al (PMID:23144195). After controlling for gender, age, baseline platelet count and number of previous therapies, we estimate that the proportion of patients with ITP responding to second line therapy is higher with eltrombopag in comparison to splenectomy: complete response (platelet count >100x109/L) 68.6% vs 54.0%; partial response 18.6% vs 24.6%; no response (platelet count <30x109/L)) 12.8% vs 21.4%. Conclusions: Indirect comparison with simulated treatment comparison is useful for decision making when no head to head data exists between new treatment options and standard of care and also when standard of care has low level of evidence available. Disclosures No relevant conflicts of interest to declare.

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