Commentary by Brian S. Carter, MD, FAAP
Feature Articles: Beernaert, K., Lovgren, M., Jeppesen, J., Werlauff, U., Rahbek, J., Sejersen, T., & Kreicbergs, U. (2019). Parents’ Experiences of Information and Decision Making in the Care of Their Child with Severe Spinal Muscular Atrophy: A Population Survey. J Child Neurol, 883073818822900.
Nelson, K. E., Rosella, L. C., Mahant, S., Cohen, E., & Guttmann, A. (2019). Survival and Health Care Use After Feeding Tube Placement in Children With Neurologic Impairment. Pediatrics, e20182863.
Jui-Chun, F., Wei-Wen, W. U., Miao-Ju, C., Shu-Yuan, L., & Su-Fen, C. (2019). The Long-term Care Experiences and Care Needs of Parents Caring for Children with Adrenoleukodystrophy. Journal of Nursing (Taiwan), 66(1), 27–37.
Other Articles Referenced: Kukulka K, Washington KT, Govindarajan R, Mehr DR. Stakeholder perspectives on the biopsychosocial and spiritual realities of living with ALS: implications for palliative care teams. Am J Hosp Palliat Med 2019.
In this month’s citation collection I was moved to see a number of references that are pertinent to pediatric palliative care clinicians – and others – that pertain to the child with a neurological condition. As we know, these children comprise a sizable portion of patients cared for in acute and chronic hospital settings, at home with home health or palliative care services, and at inpatient hospices. They may be arrested in usual childhood development, have a slowly degenerative course of neurological function, appear among the fragile young and decline even toward systemic dysfunction. How are decisions made for these children? Are there insights that might be gained prenatally – so as to engage families in conversations about an expected clinical course after birth? Can parental values be elicited that can help to direct discussions and decision-making? And, perhaps, seek stabilizing treatments, or procedures to sustain their fragile lives for highly valued and coveted time periods with families? Simple things might matter – like nutrition provision and a feeding gastrostomy.
Are there adult diseases that may give insight into decision-making? I have taken the liberty to include needs as voiced by stakeholders in one such parallel from the amyotrophic lateral sclerosis (ALS) population. See Kukulka et al.
But let’s look first at the paper by Beernaert et al. addressing parental decision making for their children with severe spinal muscular atrophy (SMA). The study comes from Denmark and was conducted in 2015, but covered a population of SMA patients born between 2003 and 2013, and their parents. The same investigators authored a population-based study in Sweden that explored the parents’ experiences of the care of their child with severe SMA. This current paper is truly worth a thorough read as it addresses a rare, but not uncommonly encountered disease for neonatologists, pediatricians, child neurologists, and geneticists as well as palliative care clinicians. Insights are given to parental experiences, clinical interventions and treatment options offered, and how decisions were made leading up to, and at, the end-of life. The majority of parents completing the survey were bereaved, but many were still caring for their child with SMA.
SMA is garnering increased interest in the era of nusinersen (Spinraza) which is administered intrathecal (IT), and the forthcoming gene transfer therapy (onasemnogene abeparvovec by AveXis and Novartis) which can be administered IV or IT. Much hopefulness has been generated by both of these therapies and working out patient selection (SMA Type 1 or Type 2), the logistics of which centers can participate in Phase III trials (hence, where patients might be referred), and measures by which to address the costliness of such treatment programs. Parent support groups may actually be clamoring for both treatments in the near future. Why? Well, as the paper by Beernaert et al. notes, it may likely be due to inconsistencies in helpful and understandable information (1/3 of their subjects had not received such); the surprising nature of not being told their child would have a shortened lifespan (26%) or was imminently dying (57%); although the location of their child’s death for bereaved parents was fulfilled >80% of the time in this Danish population – with a high proportion choosing for death to take place in the home.
During the study cohort’s inclusion period (2003-2013), pediatric palliative care was not available in Denmark. Even presently, it is uncommon. I have one personal friend, a neonatologist, from Denmark who has stated the idea seems foreign outside of pediatric oncology – clearly not one that has a standing even today in the Neonatal ICU. In Norway, the establishment of PC for children is also rather nascent (2012), as it is in Sweden (2015). Throughout the Nordic countries, then, it would appear that the provision of palliative care for children with any number of life-limiting conditions may receive palliative care – most often at home. In their statement regarding nusinersen and SMA, published in 2018, the American Academy of Neurology did not explicitly address the concomitant use of pediatric PC.
Kukulka et al. relate in their paper that a through a mixed methods methodology they were able to ascertain two distinct themes of stakeholder needs: biopsychosocial (falling within the longstanding construct of health care and its services), and spiritual and emotional well-being (what palliative care often adds to the biopsychosocial model – bringing with it inclusiveness of spirituality, meaning-making, and ongoing emotional support. The authors used the 40-item ALS Assessment Questionnaire (ALSAQ-40), in addition to semi-structured interviews with both patients and clinicians. This tool helped to quantify the frequency of concerns across 6 domains: the need for caregiving help; education on ALS & ALS care; social support; transportation and other resources to preserve independence, and the impact of ALS on spiritual and emotional well-being. To this reviewer, it would seem beneficial if in addition to the Revised Hammersmith Functional Motor Scale for SMA a similar tool to the ALSAQ-40 might be helpful in assessing parent needs in caring for their children.
Two additional papers address the child with a life-limiting neurologic condition. In Jui-Chun, F., Wei-Wen, W. U., Miao-Ju, C., Shu-Yuan, L., & Su-Fen, C., the care needs of parents whose children have adrenoleukodystophy (ALD) are also explored. In this qualitative study, 7 parents were interviewed, who divided their care needs into 3 distinct phases: 1) from pathogenesis to diagnosis, 2) a period of rapid degeneration and accompanying deterioration of physiologic functions, and 3) a final period from being bedridden until death. Each of these phases required varying support, from initialing integrating resources and care provision, on to a rapid acquisition of information and support about ALD, and finally to the establishment and provision of long-term care. Parents moved through individual experiences of initial shock, helplessness and chaos on to recognizable stages of grief and loss – finally accepting their reality and accompanying their children through even a limited life, without pain.
Nelson et al. present the evaluation of mortality following the placement of a feeding tube in a broad array of pediatrics neurological conditions. Their Canadian cohort ranged from 13 months of age through 17 years. Their overall cohort size (n=948) exceeds any previously reported. While inclusive of genetic, metabolic, malignancy or epilepsy-related diagnoses, the greatest preponderance of children in the study cohort had cerebral palsy, a degenerative CNS disease, or brain and spinal cord malformations (combined total here being 834 patients, 88% of the entire study cohort). Seventy-five percent of patients remained alive 5 years following their gastrostomy tube placement. There were no significant changes in periodic hospital days before or after the G-tube was placed, presumed to reflect the overall illness condition and frailty of this population of children with neurologic impairment.
So, what do we tell parents about the G-tube? It may be beneficial in a nutrition provision sense, and it certainly doesn’t hurt or negatively impact overall patient mortality. Understandably, however, this message must be conveyed with an explanation of the child’s neurologic condition, overall frailty, and clearly that the G-tube is not “life-saving”.