Caroline, Jenn, and Melissa standing next to the ASH 2023 conference sign

This year’s 65th American Society of Hematology (ASH) Annual Meeting and Exposition was held in beautiful San Diego, California. Held December 9-12, 2023, the meeting attracted more than 30,000 clinicians, researchers, pharmaceutical company personnel and nonprofit organizations worldwide to share ground-breaking research in the field of hematology.

In this issue of the e-news, we will report on research presented in poster format at ASH that the PDSA staff and medical advisors were part of showcasing advanced knowledge on the patient experience and their quality of life living with ITP. Watch for additional ASH reports in the 2023 winter issue of The Platelet News, the PDSA quarterly newsletter.

Binary Gender Differences in Quality of Life and Social Supports Among Individuals with Immune Thrombocytopenia (ITP): Data from the ITP Natural History Study Registry

Silhouette of a person in a hoodie sitting and looking at the sunrise

Patients with ITP often report their quality-of-life (QoL) is negatively impacted by fatigue, mental health, and changes in their physical appearance. Research on impact of gender differences on QoL for ITP patients is limited. Using patient inputted registry data, this study highlights differences between men and women in social support and ITP disease impact on their overall health related QoL.

Data was collected using responses from three surveys within the ITP Natural History Study Registry up to July 3, 2023. Adult men and women were included in the study if they completed all three surveys. A total of 460 participants were included in the study: (78%) were females and 22% were males. Most participants were from the US and were between the ages of 18 and 100 years (average age was 47 years old).

Social Supports: Women and men reported similar levels of social support. Both men and women reported they often or always had some to listen to them, confide in, and had someone who understood them. There were no significant differences between both genders in terms of their social support.

Physical health: Women reported they experienced greater limitations in activities due to physical health compared to men and reported higher average fatigue levels and a greater frequency of feeling fatigued compared to men. These differences between men and women were statistically significant.

Emotional health: Women reported higher levels of negative mental health impacting their mood and ability to think compared to men and reported being bothered more by emotional problems such as feeling anxious, depressed, or irritable, compared to men. These differences were also statistically significant.

Overall health related QoL: Men reported their overall health related QoL as ‘very good’ or ‘excellent’ more often than women did, however this difference was not statistically significant.

Overall, this study demonstrates that adult females and males with ITP have differences in reported health related QoL. Both reported similar levels of social support, however despite this, females reported higher frequencies of being limited in doing various activities, feeling tired, mental health struggles, and emotional problems. This study offers novel insight into experiences between adult women and men with ITP. Comparisons with non-ITP participants would be helpful to include in future studies to investigate whether gender differences in QoL are in fact due to the ITP itself. Future treatment plans may consider which QoL factors are impacted most by ITP and could tailor management approaches accordingly.

Comments from PDSA Medical Advisors

In this survey of adults with ITP, females reported greater impacts on physical and emotional health than men. Although the survey detected important differences between men and women, the reasons for these differences were not evaluated. In addition, gender related differences have been identified in studies evaluating quality of life in other conditions and so it will be important for future studies to evaluate which of the gender related reported differences are related to ITP and which ones to other non-disease-specific factors.


The Lack of Tolerable Treatments Options That Can Induce Durable Responses without Fear of Relapse after Discontinuation: Results from the ITP World Impact Survey (I-WISh) 2.0

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The goal of this study was to assess patient and physician thoughts towards current ITP treatments and treatment goals using data collected between February – July 2022 from I-WISh 2.0, a survey, taken by both 1,018 patients and 431 physicians in 15 countries, including the USA.

Almost all participants reported they received more than one approved ITP treatment. Corticosteroids and thrombopoietin-receptor agonists (TPO-RAs) were the most common first and second-line treatments prescribed. About a third of the patient participants described taking medication daily was disruptive to their day-to-day living, and that they were bothered by needing to plan meals around dietary restrictions associated with medication use. One-fifth of patient participants were not happy with their platelet counts while taking their current medication and felt their medication did not adequately control their symptoms. Patient participants worried about both short term and long-term impacts of their treatments. Less than 10% of physician participants were dissatisfied with current treatment options and expressed concerns over limited treatment options, inability to achieve a stable remission for their patient, inability to reach treatment goals, perceived lack of efficacy (how well the drug works) were the most common reasons provided for the physician participant dissatisfaction.

When asked about treatment goals, most patient participants reported they wanted a treatment that offered a chance for them to go into remission or cure their ITP. Most patient participants reported they prefer treatments they do not need to take daily but have concerns about relapsing symptoms of ITP upon treatment discontinuation. Patient participants also expressed concerns with their medication suppressing their immune system and reported this is a factor in their treatment-based decision making. Physicians acknowledged the negative impact immunosuppressants including corticosteroids have on patients yet only a small number (12%) strongly agreed that treatments like steroids should be avoided, even though they acknowledged the negative effect.

Patient participants identified three top treatment goals, including: achieving healthy blood counts, improving their quality of life (QoL), and increasing their energy levels. In comparison, the top three treatment goals identified by physicians included reducing spontaneous bleeding, ensuring a healthy blood count, and improving the patient’s QoL.

Overall, the authors of the study reported that adult patients with ITP are not happy with their current treatment options because of a perceived lack of efficiency, burden of side effects both short and long term, and the need for daily use in many cases. Patients and physicians would like new ITP treatments that work for a longer duration and may even lead to a cure and will not cause a relapse in symptoms upon discontinuation. The authors reported there is an unmet need for well-tolerated ITP therapies that work to help ITP symptoms in a safe manner.

Comments from PDSA Medical Advisors

This study identified important patient goals of treatment, including: raising the platelet count, improving quality of life, and decreasing fatigue. Patients were mostly satisfied with their status. Treating physicians shared these goals of treatment and they too were mostly satisfied. This underlines the importance of measuring these three outcomes in clinical trials of treatments to evaluate whether treatments can achieve the outcomes of interest to patients and providers. Compared to the I-WISh 1.0. study, patients and physicians were more aligned in this study version.