CONTENTS:

Each month, PDSA highlights interesting findings and insightful expert commentary to help enhance your understanding of ITP and partner more effectively with your care team. This month, we share the following:


Exploring the Lived Experience of Chronic ITP: Insights from a U.S. Patient Survey

For many individuals living with immune thrombocytopenia (ITP) for years, often decades, talk of platelet counts, treatment options, and “watch and wait” monitoring is second nature. Yet beyond the medical terms and therapeutic protocols lies a nuanced and often underrepresented patient experience that numbers alone can’t capture.

A U.S.-based survey conducted by the Platelet Disorder Support Association (PDSA) in collaboration with Sanofi was recently published in the esteemed British Journal of Haematology (BJH). The study aimed to better understand the lived experience of people with chronic ITP, highlighting what long-term patients identify as their most urgent needs: sustained disease control, improved quality of life, and meaningful support throughout their care journey.

The paper, titled “Patient survey in immune thrombocytopenia (ITP): Identifying unmet needs related to treatment and disease control in patients living in the United States,” included contributions from Dr. Nichola Cooper and Dr. Terry Gernsheimer (PDSA Medical Advisors), PDSA President and CEO Caroline Kruse, and longtime ITP patient and PDSA volunteer Sharon Deneen Morgan.

The survey included 80 adult respondents, 84% of whom had been diagnosed with ITP for more than 10 years. Despite their extensive experience navigating the disease, participants reported persistent challenges that extended beyond managing platelet counts. Notably, 73% of patients cited general fatigue and 71% reported physical fatigue as ongoing concerns, symptoms that were often more disruptive than thrombocytopenia itself. Many described a disconnect between clinical goals, such as achieving platelet stability, and the broader psychosocial and functional impacts of ITP. Emotional strain, treatment burden, and uncertainty during periods of disease monitoring were frequently mentioned as under-addressed in routine medical care.

Mental health emerged as a critical area of concern, particularly during “watch and wait” phases. Over half of respondents (54%) reported a decline in mental health during these periods, citing heightened anxiety and stress related to disease unpredictability. These findings suggest that while bleeding risk remains a central clinical focus, fatigue and psychological distress may be equally important determinants of patient well-being. The data underscores the need for a more comprehensive approach to ITP management, one that integrates patient-reported outcomes, prioritizes symptom relief, and acknowledges the cumulative toll of chronic disease.

Treatment history among long-term ITP patients revealed a high degree of therapeutic complexity and adaptation. Nearly all respondents (99%) had received multiple lines of therapy over the course of their disease management, with the most commonly reported treatments including corticosteroids (91%), intravenous immunoglobulin (IVIG, 74%), and thrombopoietin receptor agonists (TPO-RAs, 73%). Transitions between therapies were frequently prompted by suboptimal platelet response or adverse effects that compromised quality of life. While many participants reported experiencing clinical benefits from TPO-RAs, the need for ongoing dose adjustments and frequent monitoring contributed to treatment-related stress. Notably, 61% of patients had discontinued at least one therapy due to side effects, and 59% had done so due to lack of efficacy. These findings reflect a strong patient preference for treatment options that offer not only durable platelet stabilization but also meaningful relief from fatigue and other burdensome symptoms. The data suggest that therapeutic success, from the patient perspective, must encompass both hematologic control and improvements in overall well-being.

Even among patients with relatively stable platelet counts, the psychological burden of ITP remained significant. Nearly half of respondents (49%) reported fear of spontaneous bleeding, and 46% expressed concern about disease progression. These anxieties persisted despite active management, with many patients describing their condition as “uncontrolled,” a reflection not necessarily of platelet levels, but of the unpredictability and emotional toll of the disease. This uncertainty often led to heightened vigilance, with patients closely monitoring symptoms and laboratory results. While such self-management can be empowering, it also contributes to stress and a diminished peace of mind.

When asked to identify the most important aspects of their care, patients emphasized sustained platelet stability (61%) and greater involvement in treatment decisions (59%). These priorities reflect a desire for care that goes beyond the hematologic targets to address the broader impacts of ITP on daily functioning and emotional well­-being. Additionally, patients expressed interest in supportive resources: 54% wanted better access to educational materials, 45% sought psychological support, and 41% were interested in digital tools to help manage their condition. While most respondents felt their hematologists were knowledgeable (91%) and collaborative (84%), many still desired a more holistic approach, one that acknowledged lifestyle disruptions and mental health challenges alongside platelet counts.

Beyond the desire for greater involvement in treatment decisions, survey responses highlighted a broader need for care models that incorporate patient perspectives into clinical plans. Participants described instances where therapeutic choices were primarily driven by laboratory parameters, with limited consideration of symptom burden or psychological impact. These findings suggest that shared decision-making in chronic ITP should extend beyond selecting among treatment modalities to include ongoing dialogue about quality of life priorities, emotional well-being, and functional outcomes. Integrating patient-reported experiences into routine care may enhance alignment between clinical goals and individual needs, ultimately improving satisfaction and long-term disease management.

These findings reinforce a central truth familiar to long-term ITP patients: effective disease management encompasses more than the risk of serious bleeding or higher platelet counts. It requires a care model that integrates patient-reported outcomes, acknowledges the cumulative burden of chronic illness, and supports collaborative decision-making. As therapeutic options continue to evolve, so too must the clinical approach, toward one that is not only evidence-based but also responsive to the lived experience of patients, prioritizing both hematologic control and the broader dimensions of patient well-being. For the ITP community, shaping a future where care is not only clinically effective, but also compassionate and aligned with what matters most to those living with the disease.

 


Watch Video: In ITP, How Do You Determine a Platelet Production versus Destruction Issue?


 


The 2026 ASH ITP Guidelines Are Here: What Do They Mean for You?

survey

The American Society of Hematology (ASH) has released updated guidelines for the management of adults with immune thrombocytopenia (ITP)—the first major update to the ASH ITP guidelines since 2019.

Over the past year, ASH brought together a group of experts to revise part of the ITP treatment guidelines. Before looking at the new recommendations, it is important to understand how the guidelines were developed. The guideline panel included adult and pediatric hematologists, patient representatives, and experts in guideline methodology. This broad group was designed to bring together medical expertise, the patient perspective, and a rigorous process for evaluating the available evidence.

A particularly important consideration is that the panel could only use evidence from studies involving the specific patient population being considered. The population was not simply "people with ITP." This version of the guidelines focused specifically on adults with newly diagnosed ITP—not people with chronic or refractory ITP.

This distinction is important when considering newer ITP treatments which have not yet been studied in people with newly diagnosed ITP. As a result, these newer treatments could not be included in those recommendations.

Another important consideration involved how treatment success was measured. When the panel began its work, it identified sustained response off treatment as one of the most important efficacy endpoints.

These details help explain why these recommendations take a different approach than previous guidelines. An enormous amount of time and effort went into developing the new guidelines, and every effort was made to identify and evaluate the usable evidence and reflect appropriate perspectives. The supplement to the Blood Advances publication provides additional details about the evidence and the process used to evaluate the different treatment options.

What’s Changed?

1. More treatment options are being considered earlier.

In the 2019 ASH ITP guidelines, corticosteroids were the recommended initial treatment for adults with newly diagnosed ITP who required treatment. The 2026 update expands the initial treatment approach, recommending that newly diagnosed patients who require treatment consider combining corticosteroids with either an anti-CD20 treatment (rituximab) or a thrombopoietin receptor agonist (TPO-RA), such as romiplostim (Nplate), eltrombopag (Promacta), or avatrombopag (Doptelet). The goal is to optimize outcomes while minimizing exposure to steroids and their potential toxicity. Of course, this assumes that the diagnosis of ITP is correct, since we know that low platelets can sometimes be caused by other things.

2. Steroids alone remain an option when certain treatments are unavailable.

The panel also considered what should happen when rituximab or a TPO-RA is not available. In that situation, the recommendation was that steroids alone are preferable to combining steroids with mycophenolate mofetil (MMF, CellCept). This recommendation was based in part on decreased quality of life reported in patients receiving MMF in a randomized trial involving newly diagnosed ITP and the lack of available long-term data to evaluate whether MMF leads to a higher rate of sustained response off treatment.

3. The approach to treatment after steroids is broader.

For patients who have completed a course of steroids and now need additional treatment because steroids did not work, stopped working, or caused unacceptable side effects, the guideline provides a broader range of options. TPO-RAs and rituximab are among the treatments considered, along with other therapies including a BTK inhibitor such as rilzabrutinib (Wayrilz), mycophenolate mofetil, or a SYK inhibitor such as fostamatinib (Tavalisse).

4. Some important 2019 recommendations have NOT changed.

For example, the platelet threshold for initiating treatment in newly diagnosed adults with little or no bleeding remains unchanged at 30,000/μL, as do the recommendations for hospitalization, and the duration and type of corticosteroid treatment.

How Does This Support Patient Care?

The new guidelines give patients and clinicians a more current framework for discussing treatment choices. Rather than the conversation being primarily about whether to continue steroids or move to one particular second-line approach, there is now a broader range of options that can be considered based on the patient's circumstances.

Take an Active Role in Your ITP Care

The 2026 ASH ITP Guidelines recognize the importance of shared decision-making—working together with your healthcare provider to make treatment decisions that take into account both the available medical evidence and what matters most to you.

To help you have those conversations, PDSA has created a new ITP Shared Decision-Making Toolkit, including a convenient Treatment Conversation Card that you can take with you to your next appointment.

The card helps you identify your treatment goals and priorities and gives you practical questions to ask about your treatment options, including potential benefits and side effects, treatment goals, costs, clinical trials, future options, and what to do if a treatment does not work or stops working.

Your ITP is unique to you. Your symptoms, quality of life, treatment preferences, and personal goals matter—not just your platelet count. The PDSA Shared Decision-Making Toolkit can help you prepare for your next appointment and work with your healthcare team to make treatment decisions that fit your individual needs and goals.

Explore the PDSA ITP Shared Decision-Making Toolkit and download your Treatment Conversation Card today: pdsa.org/shared-decision-making

ASH also created a decision-making tool for patients and providers considering initial medication choice based on the updated guidelines: itpaid.hematology.org/beginning-itp

PDSA would like to thank PDSA members Brenda Shy and Jessica VandeVelde for sharing the patient perspective and helping ensure that the voices and experiences of people living with ITP were represented in the development of the new ITP guidelines.

Thank you to PDSA Medical Advisors Donald Arnold, MD, James Bussel, MD, Rachael Grace, MD, and Michele Lambert, MD for contributing to this article. Drs. Arnold, Bussel and Grace were on the 2026 ASH ITP Guidelines committee.

 

 

 


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