Decreasing Parental Stress of Caregivers of Infants With Infantile Spasms by Using Telemedicine Technology

Infantile Spasms, historically call West Syndrome, is an infantile epileptic encephalopathy, typically occurring within the first two years of life. Infantile Spasms is often characterized by hypsarrhythmia on encephalogram (EEG) and developmental plateau or regression. It is estimated that Infantile Spasms affects 2 to 5 per 10,000 live births. The etiology of Infantile Spasms is varied and often unclear at the time of diagnosis. In almost half of cases it is linked to hypoxic-ischemic encephalopathy, chromosomal abnormalities, perinatal stroke, tuberous sclerosis complex, or periventricular leukomalacia or hemorrhage.

While historically treatment options have been widely variable, over the past several years three pharmacologic therapies have been adopted, including ACTH, oral corticosteroids (OCS), and vigabatrin.

ACTH was approved for use for multiple neurologic conditions in 1952 and specifically for Infantile Spasms in 2010. Unfortunately, as a hormone therapy, ACTH carries several potentially severe side effects including adrenal insufficiency, immunosuppression, electrolyte imbalances, and more commonly elevated blood pressure thought to lead to serious cardiac changes.

Complexity of ACTH therapy requires close monitoring. ACTH is administered intramuscularly twice a day for the initial two weeks with a complicated weaning schedule over the subsequent 2 weeks. Between the required monitoring and complicated administration regimen, ACTH therapy is often overwhelming and confusing for caregivers, and can lead to medication errors.

Unfortunately, primary care (PCP) monitoring for ACTH has proved to be inconsistent. Barriers for families have included: families without an identified PCP, families without the means to get to their PCP at the recommended intervals (rural location or inadequate transportation), PCP's without the correct equipment to monitor infants, limited office hours, and PCP with little comfort in managing side-effects when they present. This has resulted in disjointed care, and last-minute attempts to obtain needed monitoring in less-than-ideal settings (e.g., urgent care, hospital, neurology clinic) - all with additional expense to families.

Treatment with ACTH is associated with high direct and indirect costs. Treatment of Infantile Spasms with ACTH carries a significant financial burden for families. In August 2007, the cost for one vial of ACTH, then manufactured by Questcor Pharmaceuticals and now by Mallinckrodt Pharmaceuticals, increased from $1650 to $23,000. Current, non-discounted pricing is $34,400 per vial. The average therapy calls for 3-4 vials, with a total price tag between $103,200 and $137,600. Any potential for medication error further exacerbates costs of therapy.

Consumer-incurred costs of ACTH therapy have not previously been reported. Direct costs of ACTH therapy for caregivers include, at a minimum, the out-of-pocket expense for the drug as well as required PCP, neurology, and EEG follow-up. It is not uncommon, due to complications of monitoring, that caregivers sustain additional direct costs seeking monitoring in unplanned ways (e.g., urgent care/ED visits). In addition, prophylactic treatment with antibiotics and H2 blockers to combat potential side effects is necessary and adds to caregiver expense. Indirect costs include loss of productivity due to work absences and non-paid loss of productivity (e.g., homemaker). Expense of transportation and travel can also be significant.

Considering the potential severe side-effects of ACTH, the complicated medication regime, the rigorous required follow-up, the costs inherent in the therapy alone and those due to complications, it is hypothesized that parenting stress for parents of babies with IS is high. Support for these families is critical to successful ACTH therapy. Historically at Children's Hospital Colorado, there has been no structured support outside of the initial admission for diagnosis/start of therapy and follow-up neurology appointments. This study is intended to explore and compare parenting stress of parents/caregivers caring for babies with IS being treated with ACTH and secondarily, overall treatment satisfaction.

Remote biometric monitoring led by highly skilled registered nurses could offer a novel, feasible, cost-effective strategy to support families of infants with IS during treatment with ACTH. Although there is currently no published literature exploring the use of telehealth or remote monitoring in the specific treatment of Infantile Spasms, its successful use in other complex conditions underscores its potential for benefit. In addition to optimization of disease-related outcomes, remote monitoring has high potential to reduce financial burden and parenting stress on parent/caregivers.

This proposed study will utilize a prospective randomized clinical trial design. - Infants and caregivers of infants who meet the inclusion criteria will be invited to participate. They will be randomized to the intervention or usual care group in accordance with a random number generator program through the Biostatistics Core at CHCO. Randomization will be stratified by payor source.

The study hypotheses include:

1. Compared to those utilizing usual monitoring, parents/caregivers of infants with IS treated with ACTH utilizing nurse-led remote biometric monitoring will report less parenting stress at 2 and 4 weeks of treatment.
2. Compared to those utilizing usual monitoring, parents/caregivers of infants with IS treated with 4 weeks of ACTH utilizing nurse-led remote biometric monitoring will experience a decrease in direct (including travel) and indirect cost of care.
3. Compared to those utilizing usual monitoring, parents/caregivers of infants with IS treated with ACTH utilizing nurse-led remote biometric monitoring will experience greater overall satisfaction with treatment.

All caregivers will be given the standard education on Infantile Spasms, ACTH and ACTH administration.

The control group will continue to receive standard care for ACTH monitoring and follow-up, which includes:

1. Blood pressure monitoring by PCP recommended to occur a minimum of two times per week. Depending on the results and PCP's comfort in managing any aberrant results, the PCP may or may not share them with the patient's primary neurologist.
2. Nurse follow-up phone call at 1 week of therapy to assess spasm frequency, monitoring and medication adherence, side-effects, complications of therapy, validate the dosing schedule, confirm required follow-up appointments (Neurology and EEG), answer any questions, and provide support. Data collected during these calls is routed to the patient's primary neurologist for review.
3. EEG follow-up at 2 weeks of therapy - results are sent to primary neurologist.
4. Neurology appointment at 2 weeks of therapy

The intervention group will receive and be instructed on the use of a re-useable digital health kit that will include an electronic tablet with integrated monitoring and telehealth software and manual or automatic blood pressure machine with appropriate sized cuff. The software utilized is web-based and has the capability to integrate into an organization's electronic health record. It allows for the real-time monitoring by healthcare professionals and the establishment of alerts. Appropriate research personnel will be responsible for this monitoring. In addition to biometric monitoring, the software allows for individualized health and other surveys to gain immediate patient feedback. Finally, the tablet and software support video telemedicine visits in the patient home.

The following interventions will be utilized in the intervention group:

1. Caregivers will take blood pressure three times per week (ideally, Monday, Wednesday, Friday) and will uploaded into the monitoring software/tablet. A neurology nurse will monitor these results, transcribe them into the patient's medical record, and route them to the patient's primary neurologist for review and potential follow-up.
2. Caregivers will complete a twice-a-week side-effect and adherence survey in the software/tablet. The results of this survey will be transcribed into the patient's medical record and routed to the patient's primary neurologist for review and potential follow-up.
3. A neurology nurse will conduct a video telehealth visit with the caregiver to assess spasm frequency, monitoring and medication adherence, side-effects, complications of therapy, validate the dosing schedule, confirm required follow-up appointments (Neurology and EEG), answer any questions, and provide support between days 7-10 and again between days 21-24 of therapy. This will be documented as a telehealth nurse-visit and routed to the patient's primary neurologist for review and potential follow-up.
4. EEG follow-up at 2 weeks of therapy - results are sent to primary neurologist.
5. Neurology appointment at 2 weeks of therapy