Immune thrombocytopenia (ITP) is a rare acquired autoimmune bleeding disorder with an estimated prevalence of 1-5 per 10,000 population.1 It is characterized by low circulating platelets due to impaired platelet production and accelerated platelet destruction. Although some individuals with ITP remain asymptomatic, others experience symptoms such as spontaneous bruising or bleeding, with a heightened risk for serious bleeding events that may be fatal.
The highest ITP incidence rates are in children, young adults, and the elderly, though the disease can occur at any age. ITP may be classified as newly diagnosed (<3 months since diagnosis), persistent (3-12 months), or chronic (>12 months in duration).1 In a 2021 review article, Kruse et al reported that ITP spontaneously resolves in approximately 80% of children, while 20% of children and 80% of adults with ITP will develop chronic disease.2
Physicians treating ITP often focus on increasing platelet counts to reduce bleeding risk. However, numerous other components of the disease also can negatively affect patients’ health-related quality of life (QoL) in ITP.
“The impact of ITP on patients and families is grossly underappreciated,” said Taizo Nakano, MD, medical director of the Vascular Anomalies Center at Children’s Hospital Colorado and associate professor of pediatric hematology, oncology, and bone marrow transplantation at the University of Colorado Anschutz Medical Campus in Aurora. “Children and adolescents have strict contact precautions that take them out of organized sports and family activities.”
Disruption, Confusion, and Anxiety
Patients often experience “bleeding symptoms that are disruptive to their daily routine, time lost from work or school to attend physician visits, and medication side effects and administration,” according to Cindy Neunert, MD, MSCS, section head of pediatric hematology and assistant professor of hematology at Columbia University Medical Center in New York, and first author of the American Society of Hematology 2019 guidelines for ITP.3
Dr. Nakano adds that childcare providers and school staff often mistake the excessive bruising for abuse in children with ITP, resulting in further disruption.
Read more about ITP symptoms
Following the bleeding symptoms, fatigue is the second most common symptom which patients with ITP report.1 In a survey-based study published in 2022, 59% of patients with ITP reported fatigue.4
“There is likely some aspect of the immune response that causes significant, and sometimes debilitating fatigue, in patients with ITP,” explained Hanny Al-Samkari, MD, hematologist and clinical investigator at the Massachusetts General Hospital (MGH) and Harvard Medical School, assistant professor of medicine at Harvard Medical School, and associate director of the MGH Hereditary Hemorrhagic Telangiectasia Center of Excellence, all in Boston.
“Additionally, anxiety is very common. Patients often have anxiety about low platelet counts, anxiety about bleeding, and extra anxiety around planned surgical procedures,” he noted.
In the 2022 study, up to 61% of patients reported anxiety about unstable platelet counts, and more than one-half of patients indicated that ITP impacted their QoL via negative effects on social life, work and studies, concentration, and energy levels.4 Other findings have shown high levels of depression in patients with ITP.2
Dr. Al-Samkari also mentioned the burden and toxicity of the various treatment strategies used to treat ITP. “For example, corticosteroids are very effective in treating ITP but should be used only for short periods—with the exception of very low doses—given the considerable toxicity of long-term steroid use, and certain therapies require patients to come into the clinic weekly for blood tests and injections.” Such factors can also have a significant impact on QoL in this patient population.
Patient-Centered Care in ITP
There are multiple ways for physicians to address QoL issues in ITP and ensure that treatment plans are aligned with the goals and needs of patients.
Dr. Neunert advises structuring patient visits to include a direct conversation about health-related QoL: “There are questionnaires that can help with this, but it really involves making sure that each visit not only reviews bleeding symptoms and the platelet count but also how the patient is doing in their daily life,” she said. “An involved discussion about medications and side effects is important as well, as we never want a patient to be on a medication that has worse effects than the disease itself.”
Physicians should ask patients about fatigue, depression, and anxiety, Dr. Al-Samkari suggests, and he emphasized the importance of a shared decision-making approach in selecting therapies for chronic ITP treatment.
Read more about patient education on ITP
Dr. Nakano pointed to the need for individualized care in pediatric ITP specifically.
“Platelet counts can be dramatically low, but every individual is different and handles low platelets differently, and so a more personalized approach is important,” he explained. “When bruising turns to oozing or bleeding is when we likely initiate treatment to help diminish symptoms—we watch for and treat symptoms.”
In line with this approach, he encourages parents of patients with ITP to learn to monitor symptoms so they can gauge where platelet counts may be.
“As parents get better and better at this over time, we can rely less and less on blood draws, which greatly improves the patient experience,” he said. Ultimately, the “key to a good outcome is clear communication and ensuring the family has a great upfront understanding of their child’s condition and realistic expectations for the disease course,” he noted.
Among the various measures that could further improve QoL in patients with ITP are additional therapies for those refractory to currently available treatments. “Thankfully, many are under development right now—investigational therapies like efgartigimod, rilzabrutinib, and mezagitamab offer novel mechanisms to treat ITP,” Dr. Al-Samkari noted.5
Dr. Neunert cites the need for new tools such as easy-to-use questionnaires or decision aids that could help guide discussions with patients about QoL.
Dr. Takano and colleagues have implemented a quality improvement project at their institution to address misinformation regarding pediatric ITP. “Providers continue to treat based on platelet counts, reacting to numbers and not symptoms,” he stated “We are standardizing bleeding scores to ensure providers use the same scoring system to determine which children require treatment.”
They have also successfully reduced unnecessary hospital admissions of patients with ITP by advising providers and families that most cases can be addressed with supportive care and home monitoring, with timely clinic follow-up. He added that clinicians “still reflexively use [intravenous immunoglobulin] infusions for new-onset ITP, although most of these patients can be monitored clinically without intervention, saving them exposure to these medications that carry significant side effect profiles and cost.”
In the quest to improve treatment for patients with ITP, Colorado will host a medical summit focused on refractory ITP in 2023 with the support of a “generous donation,” Dr. Nakano said. “It’s an opportunity for national and international experts in the field to come together to develop the next generation of guidelines to help providers treat pediatric ITP.”
- Rovó A, Cantoni N, Samii K, et al. Real-world impact of primary immune thrombocytopenia and treatment with thrombopoietin receptor agonists on quality of life based on patient-reported experience: results from a questionnaire conducted in Switzerland, Austria, and Belgium. PLoS One. 2022;17(4):e0267342. doi:10.1371/journal.pone.0267342
- Kruse C, Kruse A, DiRaimo J. Immune thrombocytopenia: the patient’s perspective. Ann Blood. 2021;6:9. doi:10.21037/aob-20-57
- Neunert C, Terrell DR, Arnold DM, et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019;3(23):3829-3866. doi:10.1182/bloodadvances.2019000966
- Chakrabarti P, George B, Shanmukhaiah C, Sharma LM, Udupi S, Ghanima W. How do patients and physicians perceive immune thrombocytopenia (ITP) as a disease? Results from Indian analysis of ITP World Impact Survey (I-WISh). J Patient Rep Outcomes. 2022;6(1):24. doi:10.1186/s41687-022-00429-y
- Mingot-Castellano ME, Bastida JM, Caballero-Navarro G, et al; Grupo Español de PTI (GEPTI). Novel therapies to address unmet needs in ITP. Pharmaceuticals (Basel). 2022;15(7):779. doi:10.3390/ph15070779