Patient-Reported Outcomes in Supportive Care

Patient-Reported Outcomes in Supportive Care

Patient-Reported Outcomes in Supportive Care Emma Bateman and Dorothy Keefe Traditionally, anticancer therapy has focused on eradication of neoplastic...

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Patient-Reported Outcomes in Supportive Care Emma Bateman and Dorothy Keefe Traditionally, anticancer therapy has focused on eradication of neoplastic tissue, predominantly by invasive and/or toxic treatments. In modern studies, patient-reported outcomes (PROs) have become more common, and give a true picture of toxicity. Increased consideration of subjective patient perspectives of anticancer treatments has allowed a notable shift within supportive oncology. Disparity exists between physician and patient perspectives of symptom severity, despite several common scoring methods. PROs are vital tools in the overall assessment of chronic illnesses, including cancer and associated treatments. Synergistic assessments of objective and subjective observations of symptoms and function are most accurate. PROs include information collected either in a clinic or by a diary system. Patient self-reporting, like any other assessment of health status, is not an absolute measure. Electronic data collection is an increasingly useful way to monitor PROs. Factors that influence quality of life (QOL) are predominantly subjective experiences, and can occur concurrently with pre-existing symptoms, which increases symptom burden. There are several validated systems for assessing PROs; some are concerned with specific conditions like mucositis (Oral Mucositis Weekly Questionnaire [OMDQ]), whereas others cover chronic illness in general (Patient-Reported Outcome Measurement Information System [PROMIS]). The PROMIS framework was developed by the National Health Institute (NHI) to standardize selfreported health measurements within chronic illnesses, including pain, fatigue and emotional distress. The general Assessment of Cancer Therapy (FACT-G) scale was developed to assess many different types of cancer; we will discuss use in oral mucositis as a model. There is more to measuring toxicity than the clinician’s objective view of the patient experience. There is still much to be done to validate all the necessary PRO tools so that we can competently measure both toxicity and toxicity-reduction strategies. Current systems to assess PROs continue to have a very positive impact on supportive oncology. Semin Oncol 38:358-361 © 2011 Elsevier Inc. All rights reserved.

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raditionally, anticancer therapy has focused on eradication of neoplastic tissue, predominantly by invasive and/or toxic treatments. The toxicities that patients needed to tolerate to achieve the longed-for cure were not always addressed in detail. More recently, the focus of treatment has broadened to include the side effects of cancer and cancer treatments; such supportive oncology is now a major focus of healthcare. When toxicity was first considered, the patient role in determining severity was limited; it was felt that “objective” measures must be used and that Mucositis Research Group, Discipline of Medicine, Faculty of Health Sciences, University of Adelaide, Adelaide, Australia. Statement of conflict of interest: the authors have no conflicts of interest. Address correspondence to Professor Dorothy Keefe, Level 4, East Wing, Royal Adelaide Hospital, North Terrace, Adelaide SA 5000, Australia. E-mail: [email protected] 0270-9295/ - see front matter © 2011 Elsevier Inc. All rights reserved. doi:10.1053/j.seminoncol.2011.03.003

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clinicians were more objective. It has been realized that patients are good at determining severity of symptoms. While patient-reported symptoms scores often parallel the severity measured by clinicians, patients suffer more than clinicians appreciate. In modern studies, patient reported outcomes (PROs) have become more common, and give a true picture of toxicity. Patient-reported toxicity also tracks reduction in quality of life (QOL). The expectation is that reduction in patient-reported toxicity would improve QOL. This has yet to be conclusively proven. A focus on the balance between quality and quantity of life associated with cancer treatments has developed. With funding from the National Cancer Institute (NCI) of clinical trials cancer cooperative groups in 1955, the objectives of cancer clinical trials were primarily to increase survival and decrease morbidity.1 It would be another three decades before measures of QOL were considered in the context of efficacy of anticancer treatments. In 1988, the NCI Cancer TherSeminars in Oncology, Vol 38, No 3, June 2011, pp 358-361

Patient-reported outcomes in supportive care

apy Evaluation Program (CTEP) modified their mission statement, declaring, “research aimed at improving survival and QOL for persons with cancer is of the highest priority.”2 Increased consideration of subjective patient perspectives of anticancer treatments has allowed a notable shift within supportive oncology. There is often incongruity between observable, clinical manifestations and patient perception of pain, nausea, vomiting and diarrhea, mucositis, and fatigue. All of them are hallmarks of antineoplastic therapies, and all affect QOL.3–5 Many “objective” evaluations by physicians and nurses underestimate pain and dysfunction.5,6 Disparity exists between physician and patient perspectives of symptom severity, despite several common scoring methods.7

PATIENT-REPORTED OUTCOMES PROs are vital tools in the overall assessment of chronic illnesses, including cancer and associated treatments.8,9 These PROs are defined as any aspect of an individual’s health status that comes directly from the patient, without influence, interpretation, or modification by any other observer.10 It has been shown that clinician-reported outcomes (CROs) of anticancer treatments may improve prediction of risk for adverse effects longitudinally, PROs are much better indicators of daily patient status.11 One problem associated with many side effects of cancer treatments is that symptoms like nausea, vomiting, and fatigue occur away from the clinical setting.12 In evidence-based, objective assessment of symptoms, this can lead to under-reporting of symptoms. Consequently, up-to-date patient reporting is invaluable to detect the severity and course of adverse symptoms, and to allow timely treatment. It has become evident that there are limitations of both CROs and PROs. Synergistic assessments of objective and subjective observations of symptoms and function are most accurate. They define the exact patient status, rather than traditional, evidence-based objective measurements used alone. This allows for better supportive care. PROs include information collected either in a clinic or by a diary system. These may include symptom and event logs, single-item outcome measures, instruments to measure health-related QOL, health status, adherence, and overall satisfaction with treatment.13 In some patient-reporting systems, caregivers contribute their independent observations of symptoms like nausea and vomiting.3 Observations by nursing staff are useful also, as they are more likely to have frequent patient contact. People often are more comfortable discussing adverse effects with a nurse rather than a clinician.6 Patient self-reporting, like any other assessment of health status, is not an absolute measure. The advantage of subjective perceptions for patient status mea-

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sures may be counteracted by this subjectivity. For example, perception of very high or low pain levels will be interpreted and reported differently by different patients.14 Compliance dependent on patient experience is another important issue; for example, the very sick may be unable to report their status.15 Another concern in assessing PROs is the burden of these measurements on the patient and research personnel.14 A balance between time requirements and precision ensures that additional stress is minimized. Electronic data collection is an increasingly useful way to monitor PROs. Access to these systems, as well as computer literacy (particularly in the elderly), may influence their effectiveness. Factors that influence QOL are predominantly subjective experiences, and can occur concurrently with pre-existing symptoms, which increases symptom burden.16 For example, copious quantities of oropharyngeal mucous with oral mucositis can cause nausea, vomiting, and fatigue independently of those symptoms caused directly by the anticancer therapy.15,17 Such increases in symptom burden may exacerbate declines in physical and psychological QOL, further compounding the adverse effects of antineoplastic therapy. Evaluation of PROs, particularly if performed early in a treatment regimen, may decrease symptom burden and increase QOL and recovery.

ASSESSMENT OF PATIENT-REPORTED OUTCOMES As with any assessment module to determine efficacy versus toxicity of a treatment, there needs to be regulation of systems using PROs, particularly standardized training.1,13,18,19 There are several validated systems for assessing PROs; some are concerned with specific conditions like mucositis (OMDQ), whereas others cover chronic illness in general (PROMIS).

Patient-Reported Outcomes Measurement Information System (PROMIS; National Health Institute) The PROMIS framework was developed by the National Health Institute (NHI) to standardize self-reported health measurements within chronic illnesses, including pain, fatigue, and emotional distress. Additional domains addressing cancer- and supportive oncology-specific assessments are being developed.20 –22

Functional Assessment of Cancer Therapy (FACT) System The general FACT (FACT-G) scale23 was developed to assess many different types of cancer. The nomenclature of each FACT system reflects each type of cancer; for example, FACT-L designates that to determine PROs in lung cancer.24 Each reflects symptoms and

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experiences that may be specific to particular cancer types.

PRO SYSTEMS FOR MUCOSITIS The painful and potentially life-threatening sequelae of mucositis are dose-limiting effects of antineoplastic therapies Complications of mucositis often interrupt treatment modules and further compromise the effectiveness of these treatments. Thus far, mucositis management is supportive; mucositis symptom control (such as pain, nausea, vomiting, diarrhea, and fatigue) may be improved by standardized PROs.

National Cancer Institute Common Terminology Criteria for Adverse Effects (NCI CTCAE) In the United States, the NCI CTCAE is the most commonly used assessor of mucositis.17 It is clinicianbased, and reports clinical and functional observations; one of the principal drawbacks is inter-assessor variability.25 Adjunct modules tailored to mucositis symptoms, like the M.D. Anderson Symptom Inventory–Head and Neck (MDASI-HN) module, more accurately reflect mucositis time-course and severity changes than the NCI CTCAE alone.17

Oral Mucositis Daily Questionnaire (OMDQ) A study on the effects of palifermin on mucositis used not only CROs, but also measurements of functionality and patient self-assessment (via OMDQ).26 Similar comparisons were made between clinicians, functionality assessments, and subjective observations regarding mucositis grade. It was shown that patients reported symptom onset, peak, and resolution 1 to 3 days earlier than clinicians.26,27 A weekly version (Oral Mucositis Weekly Questionnaire [OMWQ]) also has been evaluated in head and neck cancer, and showed good correlation between CROs and PROs.28 Similar observations of delayed symptom reporting by clinicans were observed, which may be attributed to symptoms occurring out of the clinical setting. This further underscores the importance of real-time PROs. The adult-validated OMDQ also has been used in a pediatric setting; an adaptation of this assessment evaluated mucositis symptoms in children.29

Patient-Reported Oral Mucositis Symptom (PROMS) The PROMS scale was developed and validated.30 This study compared the PROMS scale with FACT and clinician-rated oral mucositis (Visual Analogue Scale— Oral Mucositis Assessment Scale) and showed a high internal reliability and strong correlation with clinical assessments.

E. Bateman and D. Keefe

SUMMARY There is more to measuring toxicity than the clinician’s objective view of the patient experience. Gone are the days of ignoring toxicity in response to perceived powerlessness to prevent it. We now have a fuller comprehension of the vast difference between objective observation and subjective experience. This is particularly true regarding perception of pain, and allows more accurate evaluation of patient treatment and overall well-being. There is still much to be done to validate all the necessary PRO tools so that we can competently measure both toxicity and toxicity-reduction strategies. Future clinical trials of cancer treatments should all contain PROs for toxicity. Electronic capture of real-time symptoms would be an excellent step forward,31,32 and would enable clinicians to respond to patient symptoms in a more timely manner, hopefully (but not yet certainly) improving QOL. Additional population studies to fine-tune PRO assessments would allow more accurate interpretation, considering variables that may affect patient reporting. These would include age, gender, socioeconomic status, and geographic distance from clinics. It is possible that these modifications may influence future PRO assessment modules. Current systems to assess PROs continue to have a very positive impact on supportive oncology.

REFERENCES 1. Bruner DW, Bryan CJ, Aaronson N, et al. Issues and challenges with integrating patient-reported outcomes in clinical trials supported by the National Cancer Institutesponsored clinical trials networks. J Clin Oncol. 2007;25: 5051–7. 2. Clinical Trials Cooperative Group Program–Cancer Therapy Evaluation Program: guidelines. Bethesda, MD: National Cancer Institute, Division of Cancer Treatment; 1988. 3. Grunberg SM, Boutin N, Ireland A, et al. Impact of nausea/vomiting on quality of life as a visual analogue scalederived utility score. Support Care Cancer. 1996;4: 435–9. 4. Fromme EK, Eilers KM, Mori M, Hsieh YC, Beer TM. How accurate is clinician reporting of chemotherapy adverse effects? A comparison with patient-reported symptoms from the Quality-of-Life Questionnaire C30. J Clin Oncol. 2004;22:3485–90. 5. Vickers AJ, Salz T, Basch E, et al. Electronic patient self-assessment and management (SAM): a novel framework for cancer survivorship. BMC Med Inform Decis Mak. 2010;10:34. 6. Cirillo M, Venturini M, Ciccarelli L, et al. Clinician versus nurse symptom reporting using the National Cancer Institute-Common Terminology Criteria for Adverse Events during chemotherapy: results of a comparison based on patient’s self-reported questionnaire. Ann Oncol. 2009; 20:1929 –35. 7. Elting LS, Keefe DM, Sonis ST, et al. Patient-reported measurements of oral mucositis in head and neck cancer

Patient-reported outcomes in supportive care

8.

9.

10.

11.

12.

13.

14.

15.

16.

17.

18.

19.

patients treated with radiotherapy with or without chemotherapy: demonstration of increased frequency, severity, resistance to palliation, and impact on quality of life. Cancer. 2008;113:2704 –13. Granda-Cameron C, Viola SR, Lynch MP, Polomano RC. Measuring patient-oriented outcomes in palliative care: functionality and quality of life. Clin J Oncol Nurs. 2008; 12:65–77. Wagner LI, Wenzel L, Shaw E, Cella D. Patient-reported outcomes in phase II cancer clinical trials: lessons learned and future directions. J Clin Oncol. 2007;25: 5058 – 62. Lipscomb J, Reeve BB, Clauser SB, et al. Patient-reported outcomes assessment in cancer trials: taking stock, moving forward. J Clin Oncol. 2007;25:5133– 40. Basch E, Jia X, Heller G, et al. Adverse symptom event reporting by patients vs clinicians: relationships with clinical outcomes. J Natl Cancer Inst. 2009;101: 1624 –32. Molassiotis A, Coventry PA, Stricker CT, et al. Validation and psychometric assessment of a short clinical scale to measure chemotherapy-induced nausea and vomiting: the MASCC antiemesis tool. J Pain Symptom Manage. 2007;34:148 –59. Willke RJ, Burke LB, Erickson P. Measuring treatment impact: a review of patient-reported outcomes and other efficacy endpoints in approved product labels. Control Clin Trials. 2004;25:535–52. Garcia SF, Cella D, Clauser SB, et al. Standardizing patient-reported outcomes assessment in cancer clinical trials: a patient-reported outcomes measurement information system initiative. J Clin Oncol. 2007;25:5106 –12. Patrick DL, Ferketich SL, Frame PS, et al. National Institutes of Health State-of-the-Science Conference Statement: Symptom Management in Cancer: Pain, Depression, and Fatigue, July 15–17, 2002. J Natl Cancer Inst. 2003;95:1110 –7. Cleeland CS. Symptom burden: multiple symptoms and their impact as patient-reported outcomes. J Natl Cancer Inst Monogr. 2007;37:16 –21. Rosenthal DI, Mendoza TR, Chambers MS, et al. The M.D. Anderson symptom inventory-head and neck module, a patient-reported outcome instrument, accurately predicts the severity of radiation-induced mucositis. Int J Radiat Oncol Biol Phys. 2008;72:1355– 61. Morris LA, Miller DW. The regulation of patient-reported outcome claims: need for a flexible standard. Value Health. 2002;5:372– 81. Lasch KE, Marquis P, Vigneux M, et al. PRO development: rigorous qualitative research as the crucial foundation. Qual Life Res. 2010;19:1087–96.

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20. Gershon RC, Rothrock N, Hanrahan R, Bass M, Cella D. The use of PROMIS and assessment center to deliver patient-reported outcome measures in clinical research. J Appl Meas. 2010;11:304 –14. 21. Cella D, Yount S, Rothrock N, et al. The Patient-Reported Outcomes Measurement Information System (PROMIS): progress of an NIH Roadmap cooperative group during its first two years. Med Care. 2007;45(Suppl 1):S3–11. 22. Cella D, Riley W, Stone A, et al. The Patient-Reported Outcomes Measurement Information System (PROMIS) developed and tested its first wave of adult self-reported health outcome item banks: 2005–2008. J Clin Epidemiol. 2010;63:1179 –94. 23. Cella DF, Tulsky DS, Gray G, et al. The Functional Assessment of Cancer Therapy scale: development and validation of the general measure. J Clin Oncol. 1993;11: 570 –9. 24. Temel JS, Greer JA, Muzikansky A, et al. Early palliative care for patients with metastatic non-small-cell lung cancer. N Engl J Med. 2010;363:733– 42. 25. Trotti A, Bentzen SM. The need for adverse effects reporting standards in oncology clinical trials. J Clin Oncol. 2004;22:19 –22. 26. Stiff PJ, Emmanouilides C, Bensinger WI, et al. Palifermin reduces patient-reported mouth and throat soreness and improves patient functioning in the hematopoietic stemcell transplantation setting. J Clin Oncol. 2006;24: 5186 –93. 27. Stiff PJ, Erder H, Bensinger WI, et al. Reliability and validity of a patient self-administered daily questionnaire to assess impact of oral mucositis (OM) on pain and daily functioning in patients undergoing autologous hematopoietic stem cell transplantation (HSCT). Bone Marrow Transplant. 2006;37:393– 401. 28. Epstein JB, Beaumont JL, Gwede CK, et al. Longitudinal evaluation of the oral mucositis weekly questionnairehead and neck cancer, a patient-reported outcomes questionnaire. Cancer. 2007;109:1914 –22. 29. Tomlinson D, Judd P, Hendershot E, Maloney AM, Sung L, Establishing literature-based items for an oral mucositis assessment tool in children. J Pediatr Oncol Nurs. 2008;25:139 – 47. 30. Kushner JA, Lawrence HP, Shoval I, et al. Development and validation of a Patient-Reported Oral Mucositis Symptom (PROMS) scale. J Can Dent Assoc. 2008;74:59. 31. Abernethy AP, Zafar SY, Uronis H, et al. Validation of the Patient Care Monitor (version 2.0): a review of system assessment instrument for cancer patients. J Pain Symptom Manage. 2010;40:545–58. 32. Basch E, Goldfarb S. Electronic patient-reported outcomes for collecting sensitive information from patients. J Support Oncol. 2009;7:98 –9.