Hildegard Emmermann¹, Angela Schulz²

¹ Centre for Health Ethics, Hanover

² Department of Paediatrics and Adolescent Medicine, University Medical Centre Hamburg-Eppendorf

Using the example of the life-limiting condition NCL, this paper examines, from an ethical perspective, which treatment and therapeutic decisions can help ensure that the remaining time is spent with quality of life and for the well-being of the child and their family.

Neuronal ceroid lipofuscinosis (NCL) is a rare, genetically determined metabolic disorder known as ‘paediatric dementia’. It usually begins in childhood and leads to the progressive death of nerve cells, resulting in blindness, epilepsy, loss of cognitive and motor skills, and early death (usually before the age of 30). NCL is currently incurable. The diagnosis is often preceded by a lengthy period of being passed from one doctor to another, meaning that appropriate therapeutic support is frequently delayed.

Time is a crucial factor in the treatment of patients with NCL and is closely linked to ethical considerations.

On the one hand, the timing of the diagnosis has a significant impact on the options for therapeutic interventions, measures to alleviate symptoms, and psychosocial support for those affected and their families.

On the other hand, the progressive and, as yet, largely incurable nature of the disease raises ethical considerations regarding treatment decisions, particularly with regard to the benefits, burdens and quality of life of patients.

Temporal aspects are also relevant when considering experimental therapies, prioritising limited resources and the early integration of palliative care approaches. Often, the decision to switch from curative to palliative care is accompanied by concerns on the part of the families involved that time-intensive care will no longer be guaranteed in future, to the detriment of the patients.

Against the backdrop of the progressive nature of the disease, anticipatory treatment planning is therefore becoming increasingly important in order to respect the presumed wishes of those affected and to ensure that decisions are made transparently and on an interdisciplinary basis.

Overall, considering the factor of time from an ethical perspective highlights the need for patient-centred, forward-looking and responsible care for people with NCL.