Each month, PDSA highlights clinical advances being made to better understand ITP and provide expert care to patients. In this issue, we share current research about ITP presented at the European Hematology Association (EHA) Congress held in June. PDSA staff and medical advisors are proud to be authors on both research studies.
CONTENTS:
- Nighttime Symptoms are Associated with Fatigue in Children and Adolescents with Chronic ITP
- Patient and Physician Perceptions of the Burden of ITP and its Management: Results from I-WISh 2.0
Nighttime Symptoms are Associated with Fatigue in Children and Adolescents with Chronic ITP
The goal of this project was to use the PDSA ITP Natural History Study Patient Registry to identify factors that could impact fatigue in children (age 1-12) and adolescents (age 13-18) with chronic ITP. A second objective was to understand differences in nighttime symptoms between these two pediatric populations. The study included 28 children and 16 adolescents. The survey responses reflected their experience with fatigue and nighttime experiences in the past seven days.
The majority of both children (71.4%; 20/28) and adolescents (75%; 12/16) said they tired easily. More adolescents shared that they could not stop feeling sad compared to children, (25%; 4/16 vs. 11.5%; 3/36), felt pain at bedtime (42.9%; 6/14 vs. 22.2%; 4/18), and felt worried when going to bed (50%; 8/16 vs. 22%; 6/27).
Participants were then categorized depending on whether they received medication for ITP in the past six months. Sadness, pain, and anxiety at bedtime were reported less often in adolescents who were receiving ITP-directed medication compared to those who were not. In contrast, there were not differences in nighttime symptoms based on ITP medication in children.
Comments from PDSA Medical Advisors
This pilot study reports a unique look at fatigue, anxiety, and sadness in adolescents and children with ITP. The findings are striking in the degree of impairment seen in both of the two groups. As the study continues and the number of participants and duration of follow up increase, these data should prove to be very impactful. This study underlines the importance of discussing these symptoms in clinic visits and considering ways to address these issues.
Patient and Physician Perceptions of the Burden of ITP and its Management: Results from I-WISh 2.0
The global I-WISh survey was developed with patients, patient advocates, and ITP experts to study the impact of ITP on quality of life. The I-WISh 2.0 survey was completed between February and July 2022 by 1018 patients and 431 doctors, including PDSA members. The goal of this analysis was to capture the burden of ITP and the effect of the COVID-19 pandemic on the management of ITP.
Among the patients, 54% reported that their emotional well-being was strongly impacted by ITP; 77% of physicians also thought patients’ well-being was affected by their ITP. Mild to severe depression was experienced by 58% of patients, though only 16% received professional help for their ITP-related emotional struggles. Forty percent (40%) of patients worried about financial responsibilities when they were unable to work.
Fatigue was a symptom at the time of ITP diagnosis for the majority of patients (54%). Furthermore, 77% of patients mentioned they slept during the day at least occasionally. Many patients also felt that ITP affected their ability to concentrate on tasks (50%).
Regarding ITP medication, 73% of physicians considered patient perceptions when deciding on treatments. They also estimated that 15% of patients chose not to take any ITP medication, and 22% had difficulties following their treatment plan. Fifty-six percent (56%) of physicians felt that changes in diet due to ITP medication was a burden for their patients. The study also found that 71% of physicians reported their patients wanted to pause treatment if they experienced a sustained (or long-term) elevation in platelet count during treatment, but at the same time 67% of patients worried about relapsing if they stopped their medication.
Lastly, throughout the COVID-19 pandemic, 79% of physicians reported that how they managed their ITP patients was impacted, because of fewer patient visits and the need to focus on those with severe ITP. Additionally, 27% of physicians shared that the pandemic affected their treatment plans. For example, they had to prescribe certain medications for longer periods of time, avoided drugs that suppressed the immune system, and changed treatments to ones that patients could self-administer.
Comments from PDSA Medical Advisors
I-WISh 1.0 survey data were published in 2021. The findings were wide ranging and substantive, i.e. derived from 1500+ patients and 400+ physicians. I-WISh 2.0 attempts to extend these findings by focusing on specific issues identified in I-WISh 1.0, but not explored in depth. In this abstract, the findings on sleep disturbances and concentration in more than three quarters of patients was striking. The emotional impact and fatigue further emphasized previous findings. Overall, it is now very clear that there is a major systemic impact of ITP beyond issues related to bleeding. The underlying pathophysiology for fatigue in ITP remains obscure and deserves in-depth exploration.