Publications

  • Appraisal of patient-reported outcome measures in analogous diseases and recommendations for use in phase II and III clinical trials of pyruvate kinase deficiency.
    Salek, M-S. S., Ionova, T., Johns, J., & Oliva, E. N. (2018).
    Quality of Life Research. https://doi.org/10.1007/s11136-018-2025-y 
  • The Burden of Disease in Pyruvate Kinase Deficiency: Patients' Perception of the Impact on Health-Related Quality of Life.
    Grace, RF., Cohen, L., Egan, S., Wells, T., Witherspoon, B., Ryan, A., Salek, MS., Klaassen, RJ. (2018).
    European Journal of Haematology, 101(6), 758-765. https://doi.org/10.1111/ejh.13128 
  • Applying Modern Test Theory in PRO Measure Development: Rasch modelling.
    Kamudoni, P., Salek, M-S. S., & Johns, N. (2018).
    In Living with Chronic Disease: Measuring Important Patient-Reported Outcomes (pp. 91-113). Singapore: Adis. https://doi.org/10.1007/978-981-10-8414-0_5 
  • Approaches to the Development and Use of PRO Measures: A New Roadmap.
    Kamudoni, P., Johns, N., & Salek, M-S. S. (2018).
    In Living with Chronic Disease: Measuring Important Patient-Reported Outcomes (pp. 15-49). Singapore: Adis. https://doi.org/10.1007/978-981-10-8414-0_2 
  • Assessing the Performance of PRO Measures Against Expectations: Psychometric Evaluation.
    Kamudoni, P., Johns, N., & Salek, M-S. S. (2018).
    In Living with Chronic Disease: Measuring Important Patient-Reported Outcomes (pp. 115-140). Singapore: Adis. https://doi.org/10.1007/978-981-10-8414-0_6 
  • Conceptualisation and Qualitative Development of a PRO Measure.
    Kamudoni, P., Johns, N., & Salek, M-S. S. (2018).
    In Living with Chronic Disease: Measuring Important Patient-Reported Outcomes (pp. 53-73). Singapore: Adis. https://doi.org/10.1007/978-981-10-8414-0_3 
  • Content Validation by Patients and Experts: Is the PRO Measure Fit for Purpose?
    Kamudoni, P., Salek, M-S. S., & Johns, N. (2018).
    In Living with Chronic Disease: Measuring Important Patient-Reported Outcomes (pp. 75-90). Singapore: Adis. https://doi.org/10.1007/978-981-10-8414-0 
  • Integrating PRO Assessment in Clinical Trials, Routine Clinical Practice and Medicines Development Programmes.
    Kamudoni, P., Johns, N., & Salek, M-S. S. (2018).
    In Living with Chronic Disease: Measuring Important Patient-Reported Outcomes (pp. 143-165). Singapore: Adis. https://doi.org/10.1007/978-981-10-8414-0_7 
  • Evaluating Quality of Decision-Making Processes in Medicines' Development, Regulatory Review, and Health Technology Assessment: A Systematic Review of the Literature.
    Bujar, M., McAuslane, N., Walker, S. and Salek, S. (2018).
    Frontiers in Pharmacology, 8(189) 
  • The Reliability and Relevance of a Quality of Decision Making Instrument, Quality of Decision-Making Orientation Scheme (QoDoS), for Use During the Lifecycle of Medicines.
    Bujar M., McAuslane N., Walker S. and Salek, S (2018). Front. Pharmacol. 10:17. doi: 10.3389/fphar.2019.00017  
  • Observational study to develop a treatment–related patient-reported outcome measure in Gaucher disease (QOL-ONE PRO1G)
    Di Rocco, M., Cappellini, M.D., Carubbi, F., Alpa, M., Andria, G., Barbato, A., Burlina, A., Giona, F., Giuffrida, G., Roccatello, D., Borin, L.M., Spada, M., Papadia, F., S Linari, S., Salek, S., Ionova, T., Oliva, E.N. EHA Learning Center. 2018; 216144 Abstract: PB2398 
  • Presenting signs and patient co-variables in Gaucher disease: outcome of the Gaucher Earlier Diagnosis Consensus Delphi initiative.
    Mehta, A., Kuter, D.J., Salek, S.S., Belmatoug, N., Bembi, B., Bright, J., vom Dahl, S., Deodato, F., Di Rocco, M., Goker-Alpan, O., Hughes, D.A, Lukina, E.A., Machaczka, M., Mengel, E., Nagral, A., Nakamura, K., Narita, A., Oliveri, B., Pastores, G., Pérez-López, J., Ramaswami, U., Schwartz, I.V., Szer, J., Weinre, N.J., Zimran, A. ​
    Internal Medicine journal, 2018 https://doi.org/10.1111/imj.14156