CONTENTS:

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

Notes StudyThe goal of this study was to assess patient and physician thoughts towards current ITP treatments and treatment goals using data collected between February and 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 top three 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. 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.


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