PDSA e-News: July 22, 2020

In light of the COVID-19 global pandemic, the 25th Congress of the European Hematology Association (EHA) scheduled to take place in Frankfurt, Germany switched their annual meeting to a virtual meeting held online between June 11-21, 2020. PDSA staff and members of the PDSA’s medical advisory board participated in this prestigious meeting by submitting multiple abstracts that were accepted in e-poster format. The following summaries are from two insightful e-poster abstracts presented at EHA.


Long-Term Overuse of Corticosteroids in Patients with Immune Thrombocytopenia: A Real-World Analysis of a US Claims Database

Pills spilling from bottleMany treatments are available to manage ITP. There appears to be uniformity among clinicians when deciding on standard first line theray: steroids (whether dexamethasone or prednisone), however this isn’t always the case with regards to second line therapies.This study set out to determine what lines of therapy (LOT) are currently used to manage newly diagnosed adults with ITP in a real-time clinical setting, including how often first vs second line therapies are prescribed. Specifically, first line therapies are those such as corticosteroids, while second line therapies include Rituxan, TPOs, immunosuppressives, fostamatinib, and splenectomy. A line of therapy (LOT) was defined as a treatment plan that includes one or more complete cycles of a single medication, or combination of several medications, or a planned sequence of different therapies. Patients who met the study specific selection criteria were identified using the US IBM® MarketScan® database consisting of ITP treatment claims from commercial insurance and private Medicare between 2011-2017.

Second-line therapies were used less often than corticosteroids, regardless of LOT. Results also revealed that corticosteroids are not being used at the lowest possible dose for the shortest period of time and are given repeatedly at full therapueutic doses. We can only hope that this represents “the past”. The ASH Guidelines and the International Consensus report both strongly condemn this practice of overuse of steroids and recommend using steroids only for first line treatment and not for more than 6-12 weeks. Phrased differently, second line treatment, in patients requiring ongoing treatment should begin by the time of the disease has moved into the persistent state and patients should not continue prednisone for long periods of time. Further education about the outcomes associated with the use of second-line treatments are needed to enable hematologists to follow ITP expert recommendations and make informed treatment decisions to reduce corticosteroid overuse and toxicity in clinical practice. We believe that minimizing steroid use is already underway and that a major shift is happening in this area but further studies need to be done to verify this optimistic view.



Mental Health and Physical Function in Pediatric Immune Thrombocytopenia (ITP): Quality of Life Data from the Platelet Disorder Support Association (PDSA) Patient Registry

Tired child looking out windowHealth-related quality of life (HRQoL) studies among patients living with ITP have primarily focused on adults only. This study highlights the HRQoL of children living with ITP, specifically the impact of the disorder on mental health and physical function. Using data from PDSA’s Natural History Study Patient Registry, responses from 42 parents/caregivers were collected and analyzed. These responses were made on behalf of a child with ITP (under 18 years of age).

When participants were asked to reflect on the last seven days prior to survey completion, one of the striking findings was the amount of physical pain experienced. In fact, 42% reported attention difficulties due to pain, 6% reported attention difficulties due to chronic pain, 9% were unable to finish their schoolwork due to pain, and 36% experienced trouble falling asleep when they felt pain. When asked to rate the intensity of pain felt within the last seven days, 59% reported no pain and 39% reported a score from 1-5 on a ten-point likert scale, where 10 represented highest degree of pain. Overall, teens reported more pain than younger children. However, this difference was not found to be statistically significant likely due to a small sample size.

Over the seven days preceding survey completion, a high number reported fatigue (79%) and difficulties completing schoolwork (67.5%). Exercise fatigue occurred often (68%) and 62% reported fatigue interfered with their ability to engage in their usual daily routines. Approximately 17% indicated they could no longer participate in usual physical activities, and 12% reported they were unable to physically keep up with their peers. Many (54%) felt like something ‘awful’ was going to happen to them. Many (63%) felt lonely and almost half (46%) reported chronic sadness, and 76% felt generally unhappy.  When asked about peer relationships, 83% felt they were often/always accepted by their peers, and 80% had friends that helped each other. Most (68%) felt they could talk openly with their friends. Overall, teens reported being bothered by emotional problems (71.4%) more than younger children (64.7%). However, this difference was also not statistically significant.

Children and teens with ITP experience both physical and emotional consequences from their disorder, negatively impacting their daily quality of life. Living with a rare bleeding disorder often means limitations for physical activities, which was the case for a number of the participants. What was not expected was the finding that some participants experienced pain, which has not been reported before. High levels of anxiety, depression, and fatigue were disclosed despite very good social support systems in place. This could reflect the unpredictability of living with ITP. Further studies may clarify the nature and source of the pain, anxiety, depression, and fatigue reported in pediatric patients with ITP.


Comments from PDSA’s Medical Advisors:

This is an interesting article reflecting a substantial impairment in quality of life among a series of ITP patients. Certain parts of it seem very unusual in our experience. This raises the question of what has the past history of these patients been? Potentially, these participants may unusually have severe problems (emotionally and/or psychologically) or perhaps many patients have these problems but they usually go unrecognized.  The surprising part about the study is the intensity and frequency of the problems in these children. Other studies in children, for example recently from the ICON group, have described fatigue, depression, and impaired quality of life. In this study, using different scales, the authors have brought out a different sense of what is bothering their patients. In summary, this study adds a dimension to the fatigue, emotional and cognitive difficulties facing patients, in this case children, with ITP at any stage of their disease.


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