Opinion
Video
Risks versus Benefits: ITP Treatment Side Effects and the Influence on Patients’ Quality of Life
Author(s):
An expert discusses the importance of balancing efficacy with safety in ITP treatment, emphasizing cautious use of steroids, close monitoring of therapy-related side effects, and maintaining platelet counts just above bleeding thresholds to minimize long-term drug toxicity through personalized, informed care.
Managing treatment side effects is a critical part of ITP care, especially since many patients remain stable even with low platelet counts. Overtreating can do more harm than good. Steroids, a common first-line therapy, are highly effective but come with significant side effects—especially in older adults. These include insomnia, increased appetite, fluid retention, muscle weakness, and elevated blood sugar. Because of this, doses (e.g., 40 mg dexamethasone for 4 days) often need to be adjusted. Patients must be warned about these effects and monitored closely if steroids are used—whether for initial treatment or kept on hand for emergencies like travel.
Other therapies such as IVIG and rituximab also have distinct risks. As intravenous antibody-based treatments, both carry a risk of infusion-related reactions—particularly rituximab, which often causes reactions during the first infusion. Oral agents, while generally well tolerated, can also carry specific risks. Thrombopoietin receptor agonists (TPO-RAs), like romiplostim (subcutaneous) and eltrombopag (oral), can cause liver enzyme elevations, particularly in patients of East Asian ancestry, who may require lower starting doses. TPO-RAs have also been associated with reversible bone marrow reticulin fibrosis. Another oral option, fostamatinib, a spleen tyrosine kinase (Syk) inhibitor, may cause diarrhea and liver enzyme abnormalities.
For younger patients requiring long-term therapy, safety monitoring becomes even more important. Liver function, blood counts, and other labs should be tracked over time. Importantly, the goal of ITP treatment isn’t to normalize platelet levels, but to maintain a count above 20,000–30,000 without active bleeding. This allows clinicians to minimize drug exposure and long-term toxicities by adjusting doses to the lowest effective level. Educating patients about side effects and engaging them in ongoing risk-benefit discussions is key to safely managing chronic ITP.
Newsletter
Stay ahead of policy, cost, and value—subscribe to AJMC for expert insights at the intersection of clinical care and health economics.
