1. CoRPS London 26 & 27 October 2010 Center of Research on Psychology in Somatic diseases Understanding PRO in hematological disorders: Do we have a consensus? Floortje Mols Tilburg University & Comprehensive Cancer Center South The Netherlands

2. CoRPS Goal of guideline Goal of guideline: –Describe the importance of using PRO in hematology. –Provide an overview of PRO instruments that can be used in this field. –Discuss practical and clinical implications of using PRO in clinical practice and research. –Ultimate goal: support health care providers in selecting and implementing the most appropriate PRO instruments in clinical practice and research, in order to improve HRQOL and symptom management.

3. CoRPS Target group EHA guideline on PRO in hematology is intended for: –all health care professionals involved in the treatment and care of patients with hematological disorders such as: general practitioners, medical oncologists, radiotherapists, radiologists, psychologists, etc. –Also, this guideline can be used by researchers in this field.

4. CoRPS Definition of PRO I PRO is an umbrella term encompassing a number of parameters related to patient’s health status and perception of treatment side effects. PRO assessments introduce the patient’s perspective into the clinical research process via standardized self-report instruments that are scored by the patient. The use of PRO instruments is advised when measuring something that is best known to the patient or best measured from the patient’s perspective.

5. CoRPS Definition of PRO II “A measurement based on a report that comes directly from the patient about the status of a patient’s health condition without amendment or interpretation of the patient’s response by a clinician or anyone else.”

6. CoRPS Definition of quality of life I Quality of life is a multidimensional construct that consists of: –Physical functioning –Psychological functioning –Social functioning It is based on the patient’s perception, and changes over time.

7. CoRPS Definition of quality of life II QoL is not subjective in the usual sense of the term. It can be measured accurately in an individual, and in a group. It is “subjective” in that it derives from the human subject of research or clinical practice. Subjectivity and Objectivity

8. CoRPS Symptom vs. Sign Symptom: any subjective evidence of a disease, health condition, or treatment-related effect that can be noticed and known only by the patient. Sign: any objective evidence of a disease, health condition, or treatment-related effect that are usually observed and interpreted by the clinician but may be noticed and reported by the patient.

9. CoRPS What do we mean with PRO? In general: –Health status –(Health related) quality of life –Symptoms and signs –(Anxiety, depression, fatigue etc) Disease-specific: –Health status –(Health related) quality of life –Symptoms and signs –(Anxiety, depression, fatigue etc ) Although this looks identical, the associated PRO- instruments are different!!

10. CoRPS Definition of PRO instruments A PRO instrument: –Is defined as “any report coming directly from patients about a health condition and its treatment” and assesses how the patient “feels or functions with respect to his or her health condition”. –Can assess complex constructs but also more focused, one-dimensional constructs. –May include observable events, behaviours, feelings, or unobservable outcomes that are known only to the patient.

11. CoRPS Choice of PRO instruments Choice of instrument depends on: –Target population/condition –Measurement properties –Translation and cultural adaptation availability Clinical trials and research: adequacy of conceptual framework, administration mode, respondent and administrator burden. In clinical practice: recall period, patient burden, ease of interpretation. (Section III.B. FDA Guidance for Industry)

12. CoRPS Importance of PRO’s Information on side effects (symptoms/signs) can help physicians and patients in making informed treatment decisions. Efforts to develop reliable and valid PROs instruments have been successful, resulting in many well-validated instruments. FDA  Guidance for Industry on PROs. EMEA  Reflection paper on regulatory guidance for use of HRQOL measures in the evaluation of medicinal products. NIH  PROMIS

13. CoRPS PRO in hematology I Hematological disorders and its treatment (e.g. chemotherapy, radiotherapy, stem cell transplantation etc.) can have a major influence on patient-reported outcomes –QoL among Hodgkin’s lymphoma survivors is lower compared to an age-matched normative sample (Mols et al., EJC, 2006). –Non-Hodgkin lymphoma patients who had received chemotherapy (5-15 years ago) reported significantly worse psychological and social well-being and HRQOL than patients who had not received chemotherapy (Mols et al. Cancer, 2007).

14. CoRPS PRO in hematology II There is a paucity of studies on PRO in hematology, in contrast to studies on other (solid) tumors. A systematic review 1 on QOL among Hodgkin and non- Hodgkin lymphoma survivors revealed 20 studies published up until now and concluded that: –“More research on the longitudinal comparison between HL and NHL survivors and healthy controls should be performed in order to better understand the long term (side-) effects of treatment on HRQoL and possibilities to alleviate these.” (Oerlemans et al, submitted for publication)

15. CoRPS Place of PRO in Hematology Clinical Trails Clinical practice

16. CoRPS PRO in research/clinical trails PRO instruments can be used in research settings: –In prospective studies, PROs can describe the course of quality of life and possible side-effects, in large groups of patients with a certain disease, or patients who receive a specific treatment. –In comparative studies, PROs can be compared with healthy people to see whether cancer survivors get back to a similar level of PROs after diagnosis and treatment. Or, to see whether treatment A causes more side-effects, or a worse quality of life, compared to treatment B.

17. CoRPS PRO in clinical practice Use of PRO instruments in clinical practice can tell us: – if and how an individual patient is coping with his or her disease, treatment, and its’ possible side-effects. –PRO instruments can also be used repetitively, to see whether this changes over time, provided that the selected PRO instrument possess the necessary psychometric properties. –PRO’s can give an indication of a patients’ need for additional support (e.g., from a medical specialist, nurse, psychologist etc.)

18. CoRPS Questions?