Clinician’s Knowledge and Awareness of Patient-Reported Outcomes in Immune Thrombocytopenia: Results of a National Survey (ITP NET, GIMEMA Working Group Anemia and Thrombocytopenia)

Background: Patient-reported outcomes (PROs) are gaining attention in hematological diseases. Nonetheless, clinicians' knowledge of PROs is not widespread. Immune thrombocytopenia (ITP) is an autoimmune hematological disease in which fatigue significantly impacts patients' quality of life (QoL).This national Italian study focused on assessing the knowledge of PROs among hematologists experienced in ITP within the ITP NET network.

Methods: From April 4 to 20, 2023, 63 hematologists from 49 Italian centers were contacted to complete a 63-item online survey covering physician's knowledge of QoL issues and the impact of QoL on physician behaviour, decision making and patient care.

Results: Thirty-nine hematologists (72% from Northern Italy), 72% female, with a mean age of 44 (SD 11), completed the survey. Median years of practice were 15 (IQR 9-24), and median years of experience treating ITP were 6 (IQR 4-16). In the previous 12 months, each respondent saw a median of 50 (IQR 20-150) ITP patients with a median of 40 (IQR 20-77) patients undergoing treatment. Knowledge of PROs was unspecific in 36% and absent in 10%. Thirty-one percent had experience only in research, 8% used PROs in clinical practice, and 15% had knowledge without use. Discrepancies included subsequent 51% reporting frequency of PRO use in research (28% once, 23% several times) and 56% reporting general use of QoL studies in their practice. Clinicians with >17 years of medical practice had more knowledge of PRO studies than those with ≤17 years of practice (72% vs. 38%, respectively; p=0.033), as those with >4 years ITP experience (64% vs. 27%, p=0.037). Physicians with larger ITP practices had more PRO experience in clinical trials (p=0.0345) and more recognition of the value of PROs in severely ill patients (100% vs. 73%, p=0.027), If QoL results were easier to understand, 93% of physicians working in northern Italy would be more likely to use them, versus 64% of physicians working in southern Italy (p=0.042).

Conclusions: There is a general lack of knowledge and use of PROs among hematologists, highlighting the need for further education and awareness. More experienced hematologists and those with larger ITP practices have better knowledge and use of PROs, suggesting that experience plays a crucial role in understanding and implementing PROs. The study serves as a foundation for future initiatives aimed at improving awareness and implementation of PROs, ultimately striving for more comprehensive and patient-focused care in ITP.