PDSA e-News: July 23, 2019

Long-term overuse of corticosteroids in the real-world treatment of patients with immune thrombocytopenia

Bottle with pillsSteroids like prednisone, prednisolone, and dexamethasone are typically the first-line therapy to treat ITP in both children and adults, although long-term use can come with serious side-effects. Many second-line treatments such as rituximab, IVIg, and TPO-RAs (eltrombopag and romiplostim) are available when initial steroid treatment is unsuccessful, however there is currently no official consensus on which treatment should be given next and when to change treatment from steroids to one of these second-line therapies. This study sought to examine real-world treatment patterns for ITP by analyzing two large USA databases Explorys and Marketscan. So-called lines of treatment were arbitrarily defined so that treatments could be categorized as to first, second, third etc. line of treatment. Repeated courses of steroids were seen in over 90% of patients, most commonly as three consecutive treatments of prednisone for the first three lines of treatment. Surprisingly, steroids were still included in over 50% of patients in 7th line. Initially this was shocking (it still is) but it is possible that some of this is a short course to stabilize the platelet count and/or risk of bleeding while a “true second line agent” is being initiated. The data base is being queried to see if this actually happens. In this analysis of electronic medical records, rituxan was given as a second line treatment in 12% of cases, romiplostim in 7% of cases, eltrombopag in 5% of cases, and splenectomy in 3% of cases. This study demonstrates the need for improved access to second-line treatment options to reduce the evident long-term steroid overuse. Eventually steroid use would ideally fall even in first-line to prevent this major issue in management of ITP.

Comment from PDSA medical advisors:
In this study by Cuker et al. conducted in the US where most therapeutic options are available, repetitive courses of corticosteroids were by far the most prevalent treatment for adults with ITP. As expected, all second line options were used to some extent, but in a remarkably small fraction of patients albeit the data were from 2011-17. The duration and intensity of corticosteroid usage is not provided and neither cost considerations of second line options nor patient preference is discussed. In some cases, corticosteroids may have been employed as a short-term measure while trying one or another second line agent. Nevertheless, there is no doubt that the side effects of repetitive and protracted courses of corticosteroids pose a significant clinical burden and the authors are correct to conclude that there is a need for improved options and better use of available options to reduce the unnecessary use of corticosteroids for this indication.



Spleen illustrationITP patient perceptions on splenectomy outcomes

Led by a group of ITP physician experts and patient advocacy organizations including PDSA, the ITP World Impact Survey (I-WISH) conducted a study to examine patient perception on splenectomy on ITP patients. After surveying 1507 ITP patients and 472 physicians, I-WISH found that physicians recommended splenectomy in 32% of patients with persistent, chronic, recurrent ITP. Undergoing a splenectomy has approximately a 60% success rate in treating ITP patients; patients who participated in this study had a much lower success rate of 36%, with 15% of those patients believing it was an undesirable as a treatment option, and 43% of surveyed patients regretting their splenectomy because it did not work. Top concerns of patients when deciding whether to undergo a splenectomy included the need for regular immunizations, risk of increased infections, carrying a splenectomy card for panel, GI inflammation, daily need for antibiotics, the risk of respiratory symptoms, clotting, and the risk of sepsis or fever requiring ER visits. One or more of these adverse events occurred in 78% of patients who received a splenectomy. Unfortunately, only half of patients felt they were made aware of the long-term complications of splenectomy before surgery, and one in four patients who received a splenectomy experienced other health issues following surgery. Over half of patients were worried about long-term side effects. Overall, this study highlighted the need for improved patient education on the potential adverse effects of splenectomy.



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