The Need to Reframe Disease Control in Immune Thrombocytopenia (ITP)
No one living with immune thrombocytopenia (ITP) should have their condition defined by a single number.
Platelet counts have long guided how ITP is diagnosed, measured, and managed.1,2 While they remain a critical part of care, they do not always reflect the full experience of the disease. Many patients continue to face symptoms that affect how they feel and function each day, even when platelet counts appear stable and bleeding is minimal.3-5
That disconnect is becoming harder to ignore.
Understanding the burden beyond platelet counts
A growing body of research is reshaping how ITP is understood, not simply as a platelet deficiency, but as a disease of complex immune dysregulation, driven by a combination of immune imbalances and widespread inflammation.6-8 This broader understanding also helps explain why many patients continue to experience symptoms even when their platelet count is considered adequate.
“For me, it was never just about the numbers,” said Isabel, a patient living with ITP. “Even when my platelet counts looked okay, I was still dealing with exhaustion and moments where I couldn’t concentrate or keep up with everyday tasks. It’s something others don’t always see, but it has a real impact on daily life.”
In a recent cross-sectional study conducted in the US and the UK, 27% of adults with ITP reported notable fatigue despite having adequate platelet counts and minimal bleeding.9 Findings also suggest that around one in four people with chronic ITP experience clinically meaningful cognitive impairment, affecting memory, attention, and everyday functioning.10
“For many years, ITP has been viewed primarily through platelet counts, but that only tells part of the story,” said Alaa Hamed, Global Head of Medical Affairs, Rare Diseases. “What we’re increasingly seeing is that ITP reflects a broader disruption of the immune system, one that can affect patients in ways that go beyond what we traditionally measure. Recognizing that complexity is key to improving how we understand and manage the disease.”
This shift in mindset carries an important implication: if platelet counts alone do not capture the true burden of ITP, then disease control must be defined more broadly, and disease management approaches must become more comprehensive.3-5
Addressing ITP from the source
Guided by years of rare disease and immunology research, we call for reframing ITP as a disease of complex immune dysregulation – one that should be addressed through a care management approach known as multi-immune modulation.
As the name suggests, multi-immune modulation focuses on acknowledging that multiple facets of the immune system contribute to ITP disease expression, allowing us to more effectively target the imbalances and inflammation that exist throughout the patient’s body, while also decreasing platelet destruction.6,8 For patients, acknowledging the system-level pathology as opposed to just the symptom of low platelet counts could mean fewer rescue medications, improved daily functioning, and enhanced quality of life, because the root cause of the disease – and not just a single manifestation of it, is acknowledged.6,10
The term “multi” also points to something broader. Multi-immune modulation may have relevance across other diseases rooted in immune dysregulation such as warm autoimmune hemolytic anemia (wAIHA), Sickle Cell Disease (SCD), and IgG4 related disease. Through a wide range of studies, our team continues to explore opportunities to address longstanding unmet patient needs, and we remain hopeful about what the future of multi-immune modulation may mean for patients facing unmet needs.
What’s next for understanding and managing ITP
The evolving understanding of ITP marks more than a scientific reframing. It points toward a long-overdue change in how patient symptoms are interpreted, and how the disease is assessed and managed as a result.
As research advances, we believe this perspective can help drive more patient-centered care in ITP while also reshaping how immune-driven diseases are understood and managed more broadly.
Explore More
A Mother’s Story and Our Journey Living with ITP
Voices in ITP Care: The Power of Shared Decision-Making
How ITP Taught Me That My Voice Matters: My Journey from Silence to Advocacy
References
- Cooper N, Ghanima W. N Engl J Med. 2019;381(10):945-955.
- Kuter DJ, et al. N Engl J Med. 2022;386(15):1421-1431.
- Kuter DJ, et al. Br J Haematol. 2024;205(1):291-299.
- Cooper N, et al. Am J Hematol. 2021;96(2):188-198.
- van Dijk WEM, et al. TH Open. 2022;6(4):e387-e395.
- Andreescu M. Front Hematol. 2023;2:1191178.
- Lazarus AH, et al. Semin Hematol. 2013;50(0 1):S68-S70.
- Nelson VS, et al. Cells. 2021;10(11):3235.
- Kuter D, Panch S, Piatek C, Rodriguez MB, Daak A, Kissell J, et al. Fatigue in Adults with Immune Thrombocytopenia (ITP): A Cross-Sectional Observational Study. Presented at the European Hematology Association (EHA) 2026 Congress; Abstract EHA-3121.
- Cooper N, Panch S, Piatek C, et al. Cognitive Dysfunction in Adults with Immune Thrombocytopenia (ITP): A Cross-Sectional Observational Study. Presented at the European Hematology Association (EHA) 2026 Congress; Abstract EHA-3125