Abstract
Introduction:
Patient reported outcomes (PROs) are recognised as critical components of clinical trials (CT) in the modern era and now mandated by many regulatory bodies. Several key guidance has been already published (Calvert 2013&2018). Historically only 46% of haematological cancer trials incorporated any PROs (Hadidi 2021). PROs results are also not reported in >50% of cancer CT despite being collected (Marandino 2023).
We analysed the use, type, timepoints and reporting of PROs within cooperative group blood cancer trials performed by the Australasian Leukaemia & Lymphoma Group (ALLG) to observe PRO usage patterns over 18 years.
Method:
We collected data from ALLG CT including CT protocols and relevant publications/conference abstracts from 2007-25. Data collected included general CT characteristics, PROs measures (PROMs), PROs collection time-points during the study. PROs reporting in randomised CT publications were assessed for completeness of reporting according to checklist from CONSORT-PRO guidance for randomised CTs in which PROs are a primary or secondary outcome. The items include: if PROs are defined as a primary/secondary outcome in abstract (1); if PROs' hypothesis and relevant domains are provided (2); if evidence of instrument validity and reliability is provided (3); if statistical approaches for dealing with missing data is stated (4); if PRO–specific limitations are mentioned in the discussion (5). Descriptive statistics were used for analysis.
Results:
65 consecutive phase I-III blood cancer CT were included. Trial details were as follows: 62% were phase II, 31% phase III & 17% phase I or I/II; 46% were randomised. 34% were international collaborations. Main CT diseases enrolled were: acute myeloblastic leukaemia-29%, non Hodgkin lymphoma-23%, multiple myeloma-20%, myelodysplastic syndrome-14%, Hodgkin lymphoma-8% and chronic myeloid leukaemia-6%. CT status at time of analysis: 45% (29/65) were completed, 26% (17/65) closed to recruitment, 29% (19/65) enrolling.
55/65 (85%) included at least one PRO and in 50 of 55, CT protocols incorporated PROs as endpoints/objectives (primary-1, secondary-45, exploratory-4). Most commonly used PROs measures (PROMs) were EORTC-QLQ-C30 (36/55, 65%), EQ-5D-3L/5L (20/55, 36%), FACT-Leu (7/55, 13%). 85% (47/55) CT clearly stated PROM planned analysis methods and 91% (50/55) included PROM collection timepoints. PROMs were collected at screening in 98% (49/50), on study 100% (50/50), end of treatment 60% (30/50), Follow up 56% (28/50).
39/55 CT incorporating PROs had publications with final or interim results (abstracts in all 39 [100%], full papers in 51% [20/39]). 32 of these CT were completed or closed to recruitment and PROs results were reported in publications or conference abstracts in 38% (12/32). Non-randomised CT reported PROs in 1 full manuscript and 4 abstracts, while randomised CT (14/32) reported PROs in 7 manuscripts.
PROs were reported in the same manuscript as primary CT results in 6/12 instances and in the other 6 as a separate publication. The seven full publications from randomised CT were assessed according to the CONSORT-PRO checklist: 4/5 CONSORT-PRO items were reported in 2 CT, 3/5 – in 2 CT, 2/5 – in 2 CT and 1/5 – in 1 CT.
Comparing 5-year brackets over time, PROs publications of ALLG CT have increased: 1 publication in 2007-2012, 2 publications in 2013-2018, 9 publications in 2019-2025.
Conclusion:
PROs have been incorporated into the majority of ALLG trials' protocols -higher than published data in industry trials. To date, reporting rate of PROs is in keeping with published data. Our data show the ALLG has responded to key guidance around PROs (CONSORT-PRO, 2013; SPIRIT-PRO, 2018) with a noticeable rise in PRO reporting. Ongoing work with investigators continues to prioritise ALLG trial PROs analysis and publication. Future studies should publish PRO endpoints alongside other endpoints in the main manuscript and ongoing trials or trials that have not yet reported PROs should maintain high quality of PROs reporting in accordance with CONSORT-PRO.
Acknowledgement: This research was funded by the ALLG and Haematology Society of Australia and New Zealand Clinical Trials Fellowship, allg.org.au. We also acknowledge all Principal Investigators, sites and patients and patients for their contribution to clinical trials.
