Translating the Science of Patient Reported Outcomes into Practice: Meaningfulness of HM-PRO Scores in Patients with Hematological Malignancies

Introduction: Patient-reported outcome (PRO) measures not only have been widely used in clinical research but also increasingly employed in daily clinical practice to understand the health outcomes of medical interventions. A novel hematological malignancy (HM) specific PRO tool, HM-PRO, has been recently developed for use in daily clinical practice. The HM-PRO is a composite measure consisting of two scales: Part A - measuring the 'impact on patients' quality of life (QoL); and Part B-measuring the effect of 'signs and symptoms' experienced by the patients. Both scales have linear scoring system ranging from 0 to 100, with higher scores representing greater impact on QoL and symptom burden. The assessment of the "meaningfulness" of HM-PRO scores is essential if clinicians are to be able to use the instrument to understand patient health outcomes to aid their clinical decision-making and encourage better patient engagement. One way of enhancing the clinical utility of scores on multi-item questionnaires is by investigating the importance (to patients and clinicians) of cross-sectional differences by anchoring those differences and changes to clinically familiar events that are related to patient well-being. The aims of the present study were to determine the relationship between the HM-PRO scores and a Global Question (GQ) measuring the impact on a patient's life from patients' perspective and to identify bands of HM-PRO scores equivalent to each GQ descriptor, reflecting patients' global rating of PROs.

Methods: In this multicenter cross-sectional study, 905 patients: male 486; mean age 64.3 (±12.4, years; mean time since diagnosis 4.6 (±5.2) years; with different HM's ( acute lymphoblastic leukemia n=29, acute myeloid leukemia n=67, aggressive non Hodgkin lymphoma n=54, chronic lymphocytic leukemia n=64, chronic myeloid leukemia n=45, Hodgkin lymphoma n=37, indolent non Hodgkin lymphoma n=41, myelodysplastic syndrome n=158, multiple myeloma n=296, and myeloproliferative neoplasm n=114); in different disease states (stable-399, remission-277, and progressing - 229) were recruited from seven secondary hospitals and five patient organizations in the UK. All patients were asked to complete the HM-PRO and answer the global question as an anchor. Anchor-based differences were determined cross-sectionally (differences between clinically-defined groups at one time point) to determine clinically important differences in scores. The data analysis was carried out using IBM SPSS 23, a statistical software.

Results: The mean HM-PRO score for Part A was 31.7 (±21.6) with median of 28.3 (IQR 13.6-46.6), for Part B was 20.9 (±14.2) with median of 17.6 (IQR 8.8 - 29.4), and the mean GQ score was 3.2 (±1.19) with range 1-5. The mean, mode, and median of the GQ scores for each HM-PRO score for both scales of HM-PRO were used to devise the bands (Figure 1) and intra-class correlation coefficient (ICC) was calculated for level of agreement. The set of scores proposed for adoption included: for Part A HM-PRO scores 0-7 = 'no impact' on patients' QoL (GQ=1, n=64), scores 8-25 = 'a small impact' on patients' QoL (GQ=2, n=133), scores 26-41 = 'moderate impact' on patients' QoL (GQ=3, n=97), scores 42-74 = 'very large impact' on patients' QoL (GQ=4, n=111), and scores 75-100 = 'extremely large impact' on patients' QoL (GQ=5, n=18), with ICC =0.80 (95% CI-0.77 - 0.83); for Part B HM-PRO scores 0-3 = 'no effect' of signs and symptoms on patient's life (GQ=1, n=56), scores 4-16 = 'a small effect' of signs and symptoms on patient's life (GQ=2, n=133), scores 17-29 = 'a moderate effect' of signs and symptoms on patient's life (GQ=3, n=122), scores 30-65 = 'very large effect' of signs and symptoms on patient's life (GQ=4, n=104), and scores 66-100 = 'extremely large effect' of signs and symptoms on patient's life (GQ=5, n=3), with ICC =0.75 (95% CI- 0.71-0.78), respectively (Table 1).

Conclusion: This study employed the anchor-based approach for devising a set of score banding for both Part A and Part B of HM-PRO. The proposed bands (Part A=0-7, 8-25, 26-41, 42-74, 75-100; Part B=0-3, 4-16, 17-29, 30-65, 66-100) had the highest agreement and number of patients in the individual bands. The proposed bands could be applied independent of gender and different age groups. The findings of this study will help the clinician and the care team to interpret the HM-PRO scores to aid their clinical decision-making process in daily routine practice.

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