In this issue, we share exciting developments in ITP presented at this year's 66th American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego, December 7-10, 2024. First, PDSA staff and members of the Pediatric ITP Consortium of North America (ICON) collaborated on research using data from our ITP Natural History Study Registry. Second, PDSA Medical Advisor, David Kuter, MD, highlights results from the LUNA clinical trial for a new drug on the horizon to treat adults and adolescents with ITP, called rilzabrutinib.

CONTENTS:


Complementary And Alternative Medicine (CAM) Use Reported Among Individuals with Immune Thrombocytopenia (ITP): Data from The ITP Natural History Study Registry

ASH conference sign

Complementary and Alternative Medicine (CAM) refers to “non-traditional” treatment strategies to improve one’s overall health. In this study, CAM use refers specifically to the use of at least one specialized diet and/or the use of at least one supplement for the treatment of immune thrombocytopenia (ITP) either alone, or in combination with traditional (or conventional) standard ITP therapies. Traditional standard therapies refer to first line ITP treatments such as steroids or Intravenous Immunoglobulin (IVIG), or second line ITP treatments such as thrombopoietin-receptor agonists (TPO-RAs), rituximab, and other medicines approved for patients living with ITP.

In this study, PDSA staff partnered with members of the pediatric ITP Consortium of North America (ICON) to describe the prevalence, associated characteristics, and reporting of CAM use among individuals of all ages with ITP based on survey data collected from the ITP Natural History Study Registry. The use of CAM in children and adults with ITP has not been well-studied or reported, which may in part be because patients may be reluctant to share CAM use with their health care provider.

Deidentified (anonymous) data was collected from the registry between February 2017-May 2024 and combined patient-reported data from five registry surveys including the participant profile survey, diagnosis survey, treatment survey, COVID-19 survey (for non-COVID related recent treatment history only), and the bleeding and hospitalization survey. Participants were included if they entered data for most of the five surveys. Data were analyzed with descriptive statistics.

A total of 788 participants were included. Most were female and had ITP versus another platelet disorder; most had chronic ITP. The age of participants ranged from less than 18 years (6%), to 18-59 (62%), to over 60 years (31%). Most participants were living in the US. Most participants had health insurance and reported currently receiving traditional standard treatments for their ITP.

Of the 788 participants, half of participants reported using both traditional therapies and at least one diet modification and/or at least one supplement to help treat their ITP; 44% reported using traditional therapies without CAM, and 6% had never used either CAM or traditional therapies. Interestingly, when participants were asked specifically if they had ever used a CAM to treat their ITP, without specifying this meant either using a modified diet or supplement, only 2% selected “yes”, suggesting that the term “CAM” perhaps is unfamiliar to participants, and should be defined within the registry. Among CAM users, less than a quarter reported they had discussed CAM use with their health care provider.

CAM users were more likely to see a naturopathic/holistic provider for their ITP management compared to non-CAM users. Dietary modifications included gluten-free, vegan, macrobiotic, and avoidance of quinine, inflammatory foods, aspartame, caffeine and alcohol diets. Almost all participants who reported modifying their diet to help treat their ITP also reported they used at least one supplement. The most common diets reported were gluten free and anti-inflammatory.

Supplements were reported in 389 participants, although the specific supplement was reported by only 87 participants and included melatonin, folic acid, papaya leaf extract, coenzyme Q10, chlorophyll, and vitamins C, D, K, and B12. Vitamin D was the predominant supplement reported by participants.

Among CAM users, 10% reported using a CAM within the first year of their ITP diagnosis; 30% within the first 5 years, and 53% within the first 9 years. CAM users tended to be older at ITP diagnosis and survey completion than non-CAM users and were more likely to be between the ages of 50-69 years compared to non-CAM users. CAM users had tried more standard ITP treatments and reported a higher education compared to those using traditional medication only. There was no difference in CAM use by country, race, health insurance, income level, employment, or hospitalization frequency.

In summary, CAM use is common among those living with ITP. Health care providers should inquire about CAM use by specifically asking about supplements and/or dietary modifications as the term “CAM” may not be understood, as shown in this study. Discussing CAM use with patients is important for evaluating drug-interactions with traditional therapies and the effect these interventions may have on ITP. Further studies should investigate the prevalence of CAM use in a wider ITP patient population and describe the reasons for, perceptions of, and types of CAM use in ITP.


Comments from PDSA Medical Advisors:
The use of complementary and alternative medication (CAM) is clearly prevalent among ITP patients. This study indicates in a large number of patients that approximately half of ITP patients use it. As an aside, the study also demonstrates that there is not an accepted common name for “CAM” which makes it a little harder to assess. Unfortunately, the study is not able to specify which approaches were used most frequently: only 87 participants specified of the more than 350 who tried CAM.

Nonetheless, this is an important study demonstrating how widely used CAM are across demographics and locations. Further studies to see if it was possible to estimate the effects of different “CAM” would be important. However, even if this study were completed, positive results identified may be subject to the “polygenic” nature of ITP and thus limited to certain individuals.

Bottom line: it would be very helpful to know much more about the efficacy and safety of CAM and how best to use them. ITP patients and hematologists should be encouraged to communicate about them.

 

 

Results of LUNA’s Phase 3 Clinical Trial for Rilzabrutinib to Treat ITP

Medical researcher looking into a microscope

PDSA Medical Advisor, David Kuter, MD, from Massachusetts General Hospital and Harvard Medical School in Boston gave a prestigious oral plenary presentation, which is a highly featured talk, about a drug called rilzabrutinib and how it performed in the final phase (phase 3) of the clinical trial called LUNA. Rilzabrutinib is an oral ITP treatment belonging to a class of drugs known as a BTK inhibitor. It has been well studied across several immune-mediated diseases, including ITP, warm autoimmune hemolytic anemia, and even asthma, and has shown good results in earlier phases of clinical trials.

The LUNA 3 clinical trial is a multicenter site study that evaluates the safety and effectiveness of using rilzabrutinib to treat adults (over 18 years of age) and older pediatric patients (between 10-18 years of age) with persistent or chronic ITP compared to using a placebo (a sugar pill). Participants were unaware if they were receiving the placebo drug, or the treatment drug in the first 24 weeks of the study.

Participants in the treatment group received the drug for 24 weeks, and the placebo group received no treatment for 24 weeks. Then between 24-28 weeks, everyone received rilzabrutinib with the option of being following in a long-term extension trial depending on how well the drug worked. As of March 2024, there were a total of 202 participants who received rilzabrutinib, compared to 69 participants who received placebo.

Results showed that 65% of the participants receiving rilzabrutinib experienced a rise in their platelet count to over 50,000, or to over 30,000 with a doubling of their initial baseline platelet count compared to only 33% who received the placebo drug. A durable platelet count response was achieved by 23% of those in the treatment group compared to 0% of those taking the placebo pill. In the 24-28 week period of the trial which is referred to as the open label part of the study where every participant knowingly received rilzabrutinib, 29% were able to achieve a durable platelet count response. There were also other significant findings seen in the treatment group compared to the placebo group. Those receiving rilzabrutinib saw a rise in their platelet count on average within the first 15 days of receiving treatment and saw a reduction in bleeding symptoms and the need for rescue treatments by over 50%. The most common side effects reported included diarrhea, nausea, headache and abdominal pain. There were no serious adverse events or death as a result of taking rilzabrutinib.

Overall, rilzabrutinib was safe and effective, causing platelet counts to rise quickly maintaining a durable response in many, while reducing bleeding events and the need for rescue therapy, while improving fatigue and overall quality of life. The drug is currently under regulatory review in both the European union and the US Food and Drug Administration (FDA) with a target action date in the US of August 29, 2025.

Comments from PDSA Medical Advisors:
Dr. Kuter: “People living with immune thrombocytopenia who cannot tolerate or do not respond to medications aimed at raising platelet counts are at risk of uncontrolled bleeding and often endure side effects from steroids and other available therapies. A significant percentage of these patients also suffer from severe fatigue and an impaired quality of life. I’m encouraged by the robust therapeutic effects I’ve seen in patients of the LUNA 3 study across all aspects of the disease, including clinically meaningful and sustained improvements in platelet count, quality of life metrics, reduction in bleeding, and a favorable safety profile.”


Look out for more ASH updates in the upcoming Winter edition of The Platelet News!