SickKids study shows following the latest care guidelines for blood disorder ITP in children benefits patients and the health system

A less intensive treatment approach for children with immune thrombocytopenia (ITP), a common blood disorder, has benefits for both patients and the health-care system.

The clinical benefits of managing ITP conservatively (e.g., observation) in some children is well known. A recent study published in the Journal of Pediatrics also demonstrates this internationally accepted protocol allows patients to avoid unnecessary treatments and allows hospitals to improve resource utilization. The study is based on a quality improvement initiative to promote an evidence-based and family-centred approach to the treatment of ITP at The Hospital for Sick Children (SickKids).

Immune thrombocytopenia in children is an acquired disorder resulting from an autoimmune breakdown of platelets, usually in response to a viral trigger. Treatment options for newly diagnosed, typical ITP include observation or active treatment with steroids or intravenous immunoglobulin (IVIG). Since 2011, evidence-based guidelines from the American Society of Hematology have recommended that, for most children presenting with no (or mild) bleeding, managing with IVIG or steroids does not result in improved outcomes compared with observation alone. Prior to the published guidelines, an audit at SickKids revealed that most children diagnosed with ITP between 2007 and 2009 were treated with IVIG.

“Given the lack of evidence for use of IVIG over other therapies, including observation or steroids, the side-effect profile, and expense of using treatments without any potential benefit, we saw a real opportunity to shift the management of newly diagnosed typical ITP to one that was both evidence-based and family-centered,” says Dr. Carolyn Beck, Staff Paediatrician and Inpatient Director, Paediatric Medicine, SickKids.

In 2013, a team led by Beck undertook a quality improvement initiative to decrease potentially unnecessary treatment options (including use of IVIG) while optimizing family satisfaction. Interventions included the development and dissemination of an evidence-informed institutional clinical management protocol for physicians who treat children with newly diagnosed ITP, and educational materials for patients and families.

Between 2007–2009 and 2013–2015, the initiative had decreased the baseline proportion of newly diagnosed patients receiving IVIG at SickKids from 88 per cent to 55 per cent. Length-of-stay for this patient population also decreased from 47 to 36 hours as a result of changing the treatment protocol. 

The management of children with newly diagnosed ITP was identified as part of SickKids' Choosing Wisely campaign, which launched in 2016. It is estimated that in health care as many as 30 per cent of all tests and treatments may be unnecessary, add no value, and in some cases can actually lead to harm. While the primary goal of the initiative was that of Choosing Wisely – to safely reduce tests and treatments that patients do not need – the impact on cost, in relation to outcomes, had not been formally evaluated.

Notably, there is considerable variation in costs associated with each treatment option for ITP; a single infusion of IVIG costs $1,100 for a 20 kg child (typically five or six years old), compared with less than $1 for a short course of the steroid prednisone. IVIG also requires significantly more supplies and human resources as it must be administered intravenously with in-hospital monitoring over several hours.   

“Given the challenges our health-care system is currently facing, it is important that we are continually looking for ways improve value while still maintaining the best possible care for our patients,” says Dr. Myla Moretti, Senior Research Associate and Health Economist, Ontario Child Health Support Unit, SickKids. “We undertook this evaluation to understand both the clinical and economic implications of removing sometimes unnecessary treatments, and we have found that doing less can be better for patients and the system.”

Using patient-level data, Moretti, together with Beck and her team, found that the newer treatment strategy was more cost-effective than the earlier treatment protocol, resulting in a cost savings of approximately $2,000 per patient treated, without adversely affecting patient outcomes. The results remained robust, and the strategy continued to be cost-effective through several sensitivity analyses.

“Our work has demonstrated how a shift in practice can be impactful from clinical and resource stewardship perspectives,” Beck notes. “IVIG need only be used when clinically indicated, such that our practice continues to be driven by evidence and an approach that is both family and resource-friendly.”