Abstract
This study aimed to evaluate the uptake of primary ITP in RCT registries over the past 20 years.
Background: Immune thrombocytopenia is a complex autoimmune platelet disorder associated with bleeding symptoms,1,2 resulting in significant healthcare and financial burden.3 Randomized controlled trials (RCTs) are vital in assessing therapy interventions and guiding clinical practice,4 but inconsistencies in outcome reporting present challenges.7,8 To increase comparability in RCTs, a core outcome set (COS) was created for primary ITP in 2009 to standardize outcome measurements.
Methods: In June 2023, a search string was performed on ClinicalTrials.gov and International Clinical Trials Registry Platforms (ICTRP) databases to search for phase III/IV RCTs regarding primary ITP. Included criteria consisted of RCTs on subjects with primary ITP, study was registered five years prior to COS publication, and assessed their effectiveness of interventions. Screening and data extraction of included studies occurred in a masked duplicate manner. Data analysis focused on assessing uptake of COS before and after its publication using a time series analysis.
Results: Out of the 82 trials included, 39 (47.6%) followed the platelet response guidelines. However, a common alternative platelet count measurement was 50,000 x 109 L, with 35 (42.7%) trials reporting this value. The most reported outcome in our sample was adverse events, with 65/82 (79.3%) trials reporting it. All remaining outcomes were reported in less than 50% of studies.
Conclusion: The results of our study depict the increased uptake of the ITP COS since its publication in 2009 while simultaneously highlighting the continued lack of standardization among endpoints within ITP clinical trials. Furthermore, our analysis emphasizes the need for an updated COS that recognizes variability in clinical practice and considers outcomes from a patient perspective.
Background: Immune thrombocytopenia is a complex autoimmune platelet disorder associated with bleeding symptoms,1,2 resulting in significant healthcare and financial burden.3 Randomized controlled trials (RCTs) are vital in assessing therapy interventions and guiding clinical practice,4 but inconsistencies in outcome reporting present challenges.7,8 To increase comparability in RCTs, a core outcome set (COS) was created for primary ITP in 2009 to standardize outcome measurements.
Methods: In June 2023, a search string was performed on ClinicalTrials.gov and International Clinical Trials Registry Platforms (ICTRP) databases to search for phase III/IV RCTs regarding primary ITP. Included criteria consisted of RCTs on subjects with primary ITP, study was registered five years prior to COS publication, and assessed their effectiveness of interventions. Screening and data extraction of included studies occurred in a masked duplicate manner. Data analysis focused on assessing uptake of COS before and after its publication using a time series analysis.
Results: Out of the 82 trials included, 39 (47.6%) followed the platelet response guidelines. However, a common alternative platelet count measurement was 50,000 x 109 L, with 35 (42.7%) trials reporting this value. The most reported outcome in our sample was adverse events, with 65/82 (79.3%) trials reporting it. All remaining outcomes were reported in less than 50% of studies.
Conclusion: The results of our study depict the increased uptake of the ITP COS since its publication in 2009 while simultaneously highlighting the continued lack of standardization among endpoints within ITP clinical trials. Furthermore, our analysis emphasizes the need for an updated COS that recognizes variability in clinical practice and considers outcomes from a patient perspective.
| Original language | American English |
|---|---|
| State | Published - 21 Jul 2023 |
| Event | 7th Annual Joint Research Meeting: Biomedical, Biological, Neuroscience, Physiology, Forensics - Tandy Conference Center, Tulsa, United States Duration: 21 Jul 2023 → 21 Jul 2023 |
Conference
| Conference | 7th Annual Joint Research Meeting: Biomedical, Biological, Neuroscience, Physiology, Forensics |
|---|---|
| Abbreviated title | 7th Joint Annual Research Meeting |
| Country/Territory | United States |
| City | Tulsa |
| Period | 21/07/23 → 21/07/23 |