CONTENTS:

• A Statistical Crystal Ball: Predicting Pediatric Chronic ITP Before It Happens
• Experimental “Immune Reset” Leads to Remission in Severe ITP Case

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:

A Statistical Crystal Ball: Predicting Pediatric Chronic ITP Before It Happens

Taylor Kim^1,2, Kirsty Hillier^3,4, Mark Zobeck^5,6, Michele Lambert^7,8, Rachael Grace^9,10, and Amanda Grimes^5,6

When a child is diagnosed with ITP, it feels like there are more unanswered questions than clear anticipatory guidance provided by hematologists. Hematologists don’t always know what triggered ITP, cannot predict if there will be a major bleeding event, and are unable to determine if ITP will go away on its own or become a long standing, chronic condition. Without knowing the likely duration of disease for individual patients, children who do not have serious bleeding are monitored closely rather than being treated right away, even if their platelet count is very low.

Approximately 1 in 4 children with ITP go on to have chronic disease, where low platelet counts last longer than one year. For these children and their families, the “watch-and-wait” approach is problematic. It leads to ongoing uncertainty about whether the ITP will improve, and delays in starting treatments that could help raise the platelet count. With treatment withheld, chronic ITP patients suffer prolonged activity restrictions, poor quality of life and the ongoing worry about a possible bleeding event. Many available ITP treatments are only approved for those with persistent or chronic ITP, leading to months to years long delays in starting ITP directed medications. If hematologists could predict early on which children are likely to develop chronic ITP, they could begin treatment sooner and reduce the burden of ITP earlier. Children with chronic ITP are also more likely to have an underlying immune or autoimmune condition. Identifying those at higher risk sooner could lead to earlier screening for these conditions, and potentially lead to specific treatments that are targeted at the underlying condition.

Researchers have tried previously to identify clues that predict chronic ITP. Some individual factors, such as being older at diagnosis or having a higher starting platelet count, have been linked to a higher risk for chronic ITP. Our group has also found that certain lab results, like a positive direct antiglobulin test (DAT, a test that looks for antibodies against red blood cells) and abnormal immunoglobulin levels (immune proteins also known as “antibodies”), are associated with chronic disease. However, no single factor is strong enough on its own to reliably predict what will happen to children with ITP.

To address this, our team developed a new tool called the Chronic ITP Risk Model (cITP-RM: Chronic Immune Thrombocytopenia Purpura Risk Model). This model combines several pieces of information available at the time a child is diagnosed to estimate how likely that child is to ultimately develop chronic ITP. Using data from 866 children across several centers in the Pediatric ITP Consortium of North America (ICON: Pediatric ITP Consortium), the model includes age, sex, platelet count, lymphocyte count (a type of white blood cell), immunoglobulin levels, DAT results, and whether ITP is secondary to another condition. A complex statistical algorithm puts those factors together and provides a percent likelihood of developing chronic ITP.

The model is available online for use by hematologists, though it is important to emphasize that more research is needed before this tool can be used to definitively dictate clinical decisions. The Chronic ITP Risk Model takes a big step towards more personalized care, defining who can be safely observed and who would benefit from additional evaluation and earlier treatment.

Experimental “Immune Reset” Leads to Remission in Severe ITP Case

Researchers recently reported on a woman with severe immune thrombocytopenia (ITP) whose platelet counts returned to normal after an experimental treatment designed to reset the immune system. The case is especially notable because she was living with three serious autoimmune diseases at the same time. While this approach is still very early in research, it offers insight into how autoimmune conditions like ITP may one day be treated.

The patient, a 47-year-old woman in Germany, had chronic ITP that did not respond well to standard treatments. Her immune system was attacking her platelets, putting her at high risk for bleeding and requiring frequent medical care.

In addition to ITP, she had two other autoimmune conditions:

• Autoimmune hemolytic anemia (AIHA) – where the immune system destroys red blood cells
• Antiphospholipid syndrome (APS) – a condition that increases the risk of dangerous blood clots

Together, these conditions caused serious, ongoing health problems. She required frequent blood transfusions, blood-thinning medications, and repeated hospital care, and her quality of life was significantly affected.

Doctors treated her with a highly specialized therapy called CAR-T cell therapy, which is currently approved for certain cancers and is now being studied in a small number of autoimmune diseases.

In simple terms, this treatment works by:

• Collecting some of the patient’s own immune cells (called T cells)
• Re-engineering them in a laboratory to target harmful immune cells
• Returning those cells to the patient to stop the immune system from continuing its attack

In this case, the treatment targeted B cells, which are immune cells that produce antibodies. In ITP, harmful antibodies mistakenly target platelets and mark them for destruction.

Researchers describe this approach as an “immune reset”—allowing the immune system to rebuild itself without continuing the autoimmune attack.

The response was rapid and dramatic:

• Within days, she no longer needed transfusions
• Within weeks, her platelet counts returned to normal
• After about one month, she was in complete remission from ITP, AIHA, and antiphospholipid syndrome

More than a year later, she remained symptom-free and did not require ongoing treatment for any of the three autoimmune diseases.

This report describes one patient, not a cure. CAR-T therapy is complex, expensive, and can have serious side effects, which is why it is currently limited to research settings and only used in the most severe cases. However, the results are very promising. The scientists who worked on this trial noted that further controlled clinical trials are needed.

www.sciencealert.com/woman-with-3-autoimmune-diseases-enters-remission-after-immune-reset

PDSA will feature a session at this year’s ITP Conference in Salt Lake City this July focused on CAR-T therapy for ITP patients. In addition, the summer issue of The Platelet News will include an in-depth article explaining how this treatment works and exploring whether it may be considered the first potential “cure” for ITP. To access this information, be sure you are a PDSA member.

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