PDSA E-News: November 30, 2023



Trends in Outcomes and Racial Disparities in Adults with ITP

Doctor examining patient

Researchers in this study evaluated the trends in health outcomes in the US from 2010-2019 for adults with immune thrombocytopenia (ITP), particularly focusing on socioeconomic and racial disparities. They used a large national database for hospitalized patients that captured diagnosis information using billing codes. The researchers analyzed the number of admissions each year, mortality rate for hospitalized patients, average length of stay in the hospital, average hospital charges, and rates of bleeding events during hospital admission. This study grouped patients by sex (gender), race/ethnicity (White, Black, or Hispanic), and household income.

The authors found that the rates of hospital admission due to ITP increased from 392.2 per million adults in 2010 to 417.3 per million by 2019. The average age at hospital admission throughout this time was between 54 years to 56 years. In all years evaluated, most patients were female and white. The proportion of patients covered by Medicaid increased and the proportion of patients without insurance decreased over time.

Rates of mortality in the hospital stayed similar overtime. When patients were analyzed by household income, there was no difference in mortality rates. There was a decrease in mortality rate for White patients, whereas the mortality rates for Black and Hispanic patients did not change.

Hospital charges increased for all races analyzed, even after adjusting for inflation. This increase was greater for Black ($3128 USD/year) and Hispanic ($2925 USD/year) patients compared to White patients ($2159 USD/year).

When grouping all patients together, the length of hospital admission decreased overtime. This decrease in hospital stay was significant for the following patient groups: White, Black, female, and low-income patients.

Regarding inpatient recorded bleeding events, the rates of nosebleeds and melena (black stool) increased from 2010-2019, but the rates of intracranial hemorrhage (brain bleed) and hematemesis (vomiting blood) did not change. The increase in epistaxis and melena may relate to changes in admission criteria over time or to changes in billing codes.

While advances have been made, and while there have been decreases in deaths due to complications from ITP in patients who reported they identify as white, this has not been the case for those who reported they are of a non-white race. Future studies need to better understand barriers to care and how race may impact medical care, the natural history of disease, and response to available treatments. Racial and ethnic disparities exist in healthcare and are unlikely to be different for individuals with ITP.

https://link.springer.com/article/10.1007/s00277-023-05249-8

Comments from PDSA Medical Advisors

This article attempts to assess racial and ethnic disparities in outcomes of adults with ITP. The National Inpatient Database is used for this study and provides coverage of 97% of US hospital admissions. A major limitation of this analysis is the absence of consideration of relevant factors for admissions, such as comorbidities (other diseases that patients have) or primary versus secondary ITP, which impact length of hospital stay, mortality, and cost. Overall, the year-to-year variations appeared to be greater than the changes over the 10-year period. The differences, even if statistically significant, appear small.

This analysis demonstrates that rates of admissions and hospital costs for adults with ITP are rising over time. Differences were seen in rates of mortality and hospital charges between White, Black, and Hispanic individuals. These findings may reflect known racial health disparities including differences in access to health care, quality of care received, potential differences in health-seeking behaviors, and differences in social determinants of health. Improved collection of demographic information and data on social determinants of health may help to understand these differences and identify methods for decreasing risk and improving care.

 


Long-Term Use of Eltrombopag in Children with ITP

Blood examining female child

Eltrombopag (Promacta®/Revolade®) is a medication used to treat immune thrombocytopenia (ITP) and is part of a class of drugs known as thrombopoietin receptor agonists. Researchers in this study aimed to assess the long-term effects of eltrombopag use in children (aged 1-17 years) with chronic ITP. This study analyzed the medical history from 56 patients at 17 different centers in Italy between 2016 and 2022.

The study grouped patients into those who discontinued treatment because of stable platelet count (Group 1), discontinued treatment because it was ineffective (Group 2), and those who did not discontinue treatment (Group 3). Researchers found that the average duration of eltrombopag use in these patients was 40 months. However, when patients were further categorized, those in Group 1 had an average treatment duration of 34 months, while those in Group 2 had an average treatment duration of 13.5 months.

Some participants stopped eltrombopag permanently. This was due to unwanted side effects seen in one participant, ineffectiveness seen in ten patients, or achieving a stable platelet count seen in nine participants. Among those in Group 1 (discontinued due to stable platelet count), 67% patients achieved a platelet count greater than100,000 and 33% of patients achieved a platelet count greater than 50,000.

In terms of unwanted side effects, the small number of participants who experienced adverse effects reported headache, thrombocytosis (too many platelets) and microcytosis (small red blood cells). The thrombocytosis resolved in all patients after a short-term discontinuation of eltrombopag.

This study also found that patients in Group 2 (discontinued due to ineffectiveness) failed to achieve a stable platelet count in the first 6 months of treatment. Therefore, the researchers shared that the benefits of eltrombopag may be identifiable in responders in the first 6 months of initiation.

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10375288/

Comments from PDSA Medical Advisors

Thrombopoietin receptor agonists are recommended for children with ITP with non-life-threatening mucosal bleeding and/or diminished quality of life who do not respond to first line treatment. Eltrombopag is a daily oral thrombopoietin receptor agonist that was FDA approved for treatment of children with ITP in 2015. This report of 56 children with ITP treated with eltrombopag demonstrates that the effectiveness in raising the platelet count can be evaluated over several months. Eltrombopag was generally well tolerated in this study group. Eltrombopag is a known iron chelator (which means it can bind to iron in the body to remove it) which likely explains the microcytosis seen in a small number of children. Headaches and thrombocytosis were not infrequent.

Most children with ITP experience spontaneous remission or a rise in the baseline platelet count over time. In these children, eltrombopag may only be briefly needed to raise the platelet count and improve bleeding symptoms. The findings of this cohort study are consistent with prior trials and reports demonstrating that thrombopoietin receptor agonists are a generally effective and well-tolerated therapy in children with ITP.

 

 

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