Long-term effects of Langerhans cell histiocytosis (LCH)

Author:  Dr. med. Anke Barnbrock, Prof. Dr. med. Thomas Lehrnbecher, Editor:  Maria Yiallouros, English Translation:  Dr. med. Gesche Riabowol (geb. Tallen), Last modification: 2025/08/19 https://kinderkrebsinfo.de/doi/e273018

Langerhans cell histiocytosis (LCH) mostly proceeds without complications, however, 30–40 % of patients may experience permanent damage to various organs. In particular bones, endocrine‎ glands, such as the pituitary gland‎, the brain‎ and, less often, the lungs and liver may be affected. Some of the problems may be present before or at the time of diagnosis, while others may not appear until years later. Therefore, structured follow-up care of patients into adulthood is crucial.

The most common long-term effects include hormone‎ deficiencies, hearing loss and musculoskeletal defects, while lung and liver problems and impaired brain function (neurocognitive problems) are much less common. In the case of chronic organ damage, the respective specialists should be contacted.

Hormone deficiencies due to damage to the pituitary gland

Depending on the part of the pituitary gland (posterior lobe or anterior lobe), different types of hormonal deficiencies can occur:

• Posterior pituitary gland: The most frequently observed hormone deficiency of the posterior pituitary gland in LCH, at approximately 30%, affects the so-called antidiuretic hormone (ADH) and leads to diabetes insipidus‎, which is a disturbance of the water-electrolyte‎ balance due to excessive excretion of urine. In this case, the kidneys are unable to concentrate urine and retain water. Excessive thirst (drinking up to 8-10 litres per day!) and frequent urination can be signs of diabetes insipidus. It can develop before, during, or after LCH diagnosis and is usually irreversible.
• Anterior pituitary gland: Growth hormone deficiency is one of the most common hormone deficiencies of the anterior pituitary gland (up to 10% of patients). However, it can also lead to delayed puberty‎ and, rarely, to a complete loss of function of the anterior lobe. Hence, monitoring growth, weight and development are crucial to identify deficits and initiate their management in a timely manner.

Bone damage

While disease manifestations in the skull and the long bones of the arms and legs usually heal without consequences, vertebral body involvement can lead to the occurrence of scoliosis and/or kyphosis. In order to prevent such long-term effects, the necessity of certain measures (e.g. orthopaedic corset, neck brace, physiotherapy/remedial gymnastics to strengthen the back muscles) should be discussed with orthopaedic surgeons and physiotherapists.

Damage to ears / jaw / gums

In patients with the disease involving the middle or inner ear and/or temporal bone, regular hearing tests are indicated until the disease has been successfully treated. Early detection allows the timely use of aids (such as hearing aids) and can prevent a potential deterioration of the symptoms.

Patients with affected jaw and gums should be carefully monitored with regards to tooth development. Co-assessment by an orthodontist/dentist is recommended for these patients.

Damage to the central nervous system (brain/spinal cord)

Patients with multisystem LCH have an increased risk of developing long-term neurological‎ damage („neurodegenerative LCH”). Magnetic resonance imaging (MRI) can be used to detect findings (imaging signs) indicative of neurodegenerative LCH in approximately 15% of patients, usually long after treatment. The cause of this is still largely unclear. However, it should be emphasised that only about one in four patients who show the typical changes of neurodegenerative LCH on imaging‎ go on to develop clinical symptoms, i.e. symptom‎s that are visible from the outside.

The signs of neurodegenerative LCH usually start slowly, but can increase rapidly over the course of the disease and may even lead to spastic paralysis. Typical symptoms include balance and articulation disorders or difficulty concentrating (including learning difficulties). In order to identify minor or (by means of magnetic resonance imaging‎) even non-detectable clinical (subclinical) changes as early as possible, regular neurophysiological and neuropsychological examinations are recommended for patients with imaging signs of neurodegenerative LCH [see neurophysiology‎, neuropsychology‎].