Psychosocial and medical factors associated with intrathecal baclofen pump explants: A case series

1 Introduction

Intrathecal baclofen (ITB) therapy can be an effective treatment of spasticity [1], dystonia [2], and other dyskinesias [3] in children.Cerebral palsy (CP) is a condition that affects tone and movement due to non-progressive lesions of the brain acquired in infancy and is the most common cause of disability in children [4, 5]. ITB therapy, therefore, is most often used to treat spasticity and dystonia due to CP, but it can also be used to treat spasticity and dystonia in other conditions such as hereditary spastic paraplegia (HSP), spinal cord injury, and traumatic brain injury.

Gamma-aminobutyric acid (GABA) is an amino acid that serves as the primary inhibitory neurotransmitter in the brain and a major inhibitory neurotransmitter in the spinal cord [6]. Baclofen is a centrally-acting skeletal muscle relaxant that acts on the GABA_B receptors in the spinal cord [7]. The route of administration can be either oral or intrathecal. Because the intrathecal route does not have to cross the blood-brain barrier, lower doses of intrathecal baclofen are required to treat tone with potentially fewer systemic side effects.

While ITB therapy has the potential to decrease systemic side effects, it is also associated with important risks and care considerations. ITB pumps must be refilled regularly and consistently (e.g., every three to six months) to prevent baclofen withdrawal. Similarly, pump malfunction can lead to baclofen withdrawal. Symptoms of withdrawal may include increased spasticity, muscle breakdown, fever, high blood pressure, seizures, organ failure, and even death [8, 9]. As such, ITB pump management can be burdensome and stressful on the patient and their caregiver(s). This may be especially true for those with logistical or financial challenges (e.g., patients who live a significant distance from their medical provider, patients with inconsistent transportation access), emotional concerns (e.g., anxiety, needle phobia), or behavioral symptoms (e.g., self-harm behavior). In turn, these factors can lead to dissatisfaction with the pump and requests to abort ITB therapy.

Given that pump explantation may significantly impact a pediatric patient’s overall tone management and quality of life, it is essential to understand the factors that may lead patients and families to decide to abort ITB therapy and to address them before the pump is implanted. Although extant literature refers to the consideration of psychosocial, logistical, and financial variables before implantation [10], a standardized psychosocial assessment and pre-implantation education process in the pediatric population has yet to be identified within the literature. Thus, the aim of this study was to explore, via retrospective chart review of individual cases, the role that psychosocial barriers, expectations of treatment, and anticipatory guidance played in the decision to explant the ITB pump. It is the ultimate intention of this work to delineate best practices regarding multidisciplinary ITB pump pre-implantation assessments, education and goal setting.

2 Materials and methods

This Institutional Review Board (IRB)-approved study retrospectively assessed a series of patients at a single pediatric institution, who were treated with intrathecal baclofen between September 1, 2009, and October 1, 2020, and subsequently had the pump removed or attempted its removal. Participants met the following inclusion criteria for this study: between age 1 month to 26 years at the time of establishing care with the ITB team and must have been treated with an ITB pump.

All data collected as part of this study were recorded in the patient’s medical record. As such, there were no study-specific procedures performed on participants. Quantitative data were culled from patient medical records. A comprehensive list of collected variables is provided below. Additionally, qualitative data were collected from patient clinic visit notes, follow-up phone calls regarding the patient’s ITB pump, and any emergency room visits associated with the ITB pump.

Data were collected from the medical records of 30 participants who had been treated with intrathecal baclofen between September 1, 2009, and October 1, 2020. The participants were then divided into those who had pump removals and those who attempted pump removal. Specific variables were collected to further characterize those individuals.

Demographic variables collected include the following: patient age at initial clinic appointment with the ITB team, ITB pump trial, ITB pump implantation, and ITB pump explantation. Sex assigned at birth, race/ethnicity, and insurance coverage were collected.

Clinical variables (where applicable) collected include the following: medical diagnosis, tone classification, Gross Motor Function Classification Scale (GMFCS) level, medication regimen, identification of treatment goals, ITB trial information, pump-related complications, and ITB explantation information.

Psychosocial variables (where applicable) collected include the ITB screening information by Psychology, Physical Therapy and/or Occupational Therapy, identification of treatment goals, and overall candidacy for ITB pump treatment. Mood, anxiety, and behavioral symptoms, as well as logistical factors, were also collected.

Service utilization variables collected include the following: number of scheduled ITB-related clinic visits attended, number of ITB-related patient phone calls to nursing and social work, and number of ITB-related emergency room and urgent care visits.

Comparison between the groups showed no differences in the variables culled. A subsequent power analysis conducted using an unbalanced design of 1:2, chosen because ITB pumps are not frequently explanted once placed, demonstrated that for all the variables to achieve at least 80% power at 5% significance, a group size of between 56–204 and 112–408 would be needed to compare all the variables between cohorts that considered/underwent pump removal and those that did not consider pump removal, respectively. Given the limitation in sample size, a descriptive study using case studies was conducted.

3 Results

Thirty participants were identified who had ITB pumps. Participants ranged in age from 6 to 18 years old (average 11.8 years based on participant age at ITB pump implant). The sample was fairly evenly divided by sex (53% female assigned at birth; 47% male assigned at birth). Participants’ racial and ethnic breakdown was as follows: 87% White, 13% Black; 70% non-Hispanic White, 26% Hispanic/Latino, 3% Middle Eastern/North African. None of the subjects had a documented psychological assessment in their medical record.

Of the 30 participants with ITB pumps, six had them removed, and two had attempted pump removal. The majority of patients who had or attempted pump removal were diagnosed with CP (75%; N = 6). For the reviewed patients, the documented GMFCS ratings ranged from III to V, with 67% meeting criteria for GMFCS V (Table 1). Most patients were insured (87.5%) through private or public insurance plans (Table 1).

In this case series, patients attempted and/or completed the removal of the ITB pump for numerous reasons. The most frequent reason for explantation was postoperative infection (Table 3), which often required significant medical intervention in the form of workup and revision surgeries (Table 2). Of those with infection, only one pursued pump reimplantation. Pump malfunction as well as perceived or actual lack of ITB pump effect were other important factors leading to attempt of/or completed explantation (Table 3), even in cases where available documentation noted positive response after a test dose (Cases 1–6). One patient (Patient 4) missed multiple appointments, causing their pump to run dry. However, this particular patient did not experience any negative effects of their pump running dry. Given the lack of withdrawal symptoms, which would be expected to occur if the pump was delivering medication as intended, the pump likely had malfunctioned (Table 3).

5 Discussion

In carefully selected patients, ITB therapy has the potential to make a meaningful difference in quality of life. While etiology, characterization, and distribution of tone should be considered when evaluating candidacy for ITB therapy, psychosocial and logistical considerations can be just as or even more important than clinical considerations [8, 10]. Proactively identifying barriers and complications is especially critical, as suboptimal ITB pump management is likely to lead to poor results and patient dissatisfaction with the therapy which may even lead to life-threatening complications [11].

Based on observations regarding the reasons patients in this series considered or had their pump removed, the following should be considered in the pre-implantation assessment and education process: risks and complications associated with ITB implantation, level of understanding of collaboratively created and realistic goals, level of understanding of the ongoing refill expectations, geographic availability of resources that may be needed in case complications or pump malfunctions arise, history of patient/family non-adherence, as well as logistical barriers such as transportation and work schedules that may preclude frequent follow-up. In addition, special considerations for maturing children should be addressed, including stability of family home/structure and the psychological and emotional changes that can accompany adolescence; this is particularly important as psychological distress may worsen the appearance of tone. Further, potential body habitus changes should be considered, as these may also lead to difficulties with refills. Finally, ensuring that families know that providers and plans may have to change when the child transitions out of the pediatric care setting should also be emphasized.

Standardizing the multidisciplinary preoperative psychosocial assessment and education process is a way to ensure a full and comprehensive evaluation of goals, expectations for treatment, and potential psychosocial barriers to successful treatment. Including a multidisciplinary team with various areas of expertise, such as physicians, advance practice providers, therapists, psychologists, social workers, and nurses allows for psychosocial considerations and families’ knowledge of goals and complications to be assessed from multiple points of view.

Goal setting should occur collaboratively between the patient, the patient’s family, and the medical team within the context of the preoperative assessment and should be tailored to the level of patient function. Care and comfort should be the goal setting focus for children who do not ambulate, while gait-associated activity goals should be targeted in the ambulatory population [10, 12]. Having the opportunity to assess patient/family understanding of treatment goals and limitations of treatment within the context of a child’s function is of utmost importance. Assessment of patient and family expectations and how these expectations have been formed is especially relevant as children with complex medical needs are more likely to receive care from multidisciplinary teams that may have variable familiarity with ITB therapy.

Given that ITB complications can arise throughout the course of the ITB life span [13], as was evident in the case series, education, anticipatory guidance, and contingency planning should also be conducted within the preoperative assessment setting. Families and caregivers should, therefore, be proactively aware of the potential problems that can arise, such as postoperative infections, programming errors, and mechanical dysfunction of the pump or catheter/tubing. The fact that these potential complications may also affect work attendance for caregivers, transportation logistics, and financial security should also be addressed. Additionally, a preoperative evaluation can help contextualize ITB therapy as a lifelong commitment with relation to insurance changes as well as adult treatment accessibility (e.g., distance between home and tertiary centers that are appropriately staffed to deal with ITB pumps; availability of adult providers), possibly minimizing the risk for explantation at the time of transition.

Furthermore, pediatric ITB pump management needs assessments should be ongoing, as the social and psychological environment can be dynamic throughout childhood. For example, family structure can change with divorce, marriage, the addition of family members with unique care needs, changes in parent/caregiver employment, and even caregiver turnover if the primary caregiver is hired through a nursing or caregiver agency. Insurance is also likely to change throughout a pediatric patient’s life, especially during transitions from pediatric age to adulthood. Patients may struggle to identify adult care providers who are willing to manage ITB pumps locally. If an adult care provider cannot be identified, patients may face pump explantation and an overall reduction in the quality of their symptom management. Finally, psychological well-being can also be dynamic, particularly during puberty and major school transitions. The care team should be aware that medications used for mood and psychiatric symptoms may also affect movement disorders [14] and body habitus [15].

Certain study limitations merit review. First, due to the small sample size, results may be incomplete, and there may be additional important variables that contribute to a patient’s risk for explantation. Second, and relatedly, this retrospective case series relied in part on patients’/families’ self-report of ITB challenges. As patients are sometimes motivated to try and ‘please’ their doctors, their self-report of relevant difficulties may not have been comprehensive.

It is hoped that the data from this study will be used to justify the benefit of establishing a formalized, multidisciplinary assessment and educational process before pediatric ITB pump implantation. Delineating best practice guidelines for the field will ideally improve treatment adherence and outcomes for those who do receive a pump while optimizing hospital service utilization.

6 Conclusions

This retrospective review cohort study indicated that, of 30 pediatric patients with ITB pumps reviewed, eight attempted and/or underwent pump explantation. Medical, psychosocial, and logistical factors contributed to a patient’s risk for explantation. Recognizing the critical importance of expectation and goal setting, proactive identification of barriers to treatment, contingency planning for medical complications, and transitional care variables, establishing a formalized, multidisciplinary assessment and education process before pediatric ITB pump implantation is indicated.