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Langerhans cell histiocytosis: 23 years' paediatric experience highlights severe long-term sequelae
Allison Martin1, Susan Macmillan2, Dermot Murphy3
1Specialty Doctor in Neonatology, Paediatric Medicine, Wishaw General Hospital, UK.
Insights
Langerhans cell histiocytosis (LCH) is a rare, variable childhood disease. Multisystem LCH in young children requires intensive treatment and leads to long-term sequelae in over a third of patients.
Area of Science:
- Pediatric Oncology
- Hematology
- Rare Diseases
Background:
- Langerhans cell histiocytosis (LCH) is a rare clonal proliferative disorder.
- It primarily affects infants and young children, with a variable clinical course.
Purpose of the Study:
- To review the presentation and outcomes of pediatric patients diagnosed with Langerhans cell histiocytosis.
- To analyze disease classification, treatment strategies, and long-term sequelae over a 23-year period.
Main Methods:
- Retrospective analysis of 31 pediatric patients diagnosed with LCH between 1990 and 2012.
- Data collected included age at diagnosis, symptoms, disease classification, treatment, and long-term outcomes.
Main Results:
- The cohort included 17 boys and 14 girls, with a median age at diagnosis of 2 years 9 months.
- 18 patients had single-system disease and 13 had multisystem disease; 10 developed endocrine dysfunction.
- One patient died, and over a third experienced lasting sequelae.
Conclusions:
- LCH is a rare pediatric disease with a spectrum from self-limiting to life-threatening.
- Multisystem LCH in very young children necessitates intensive chemotherapy and lifelong monitoring.
- Endocrine dysfunction, hearing, neurological, and psychological issues are common long-term sequelae.
Purpose:
To review the presentation and outcome of patients with Langerhans cell histiocytosis attending The Royal Hospital for Sick Children, Glasgow over a 23-year period.
Method:
Thirty-one children were diagnosed with Langerhans cell histiocytosis between January 1990 and December 2012. Retrospective information from medical records was gathered on age at diagnosis, presenting symptoms, classification of disease, treatment and long-term outcome.
Results:
There were 17 boys and 14 girls; median age at diagnosis 2 years 9 months (interquartile range: 1 year 6 months to 4 years 4 months). Eleven were below 2 years and two were below 6 months of age at diagnosis. Eighteen (58%) children had single system disease of which four were multifocal; 13 (42%) had multisystem disease. Seventeen children improved with conservative treatment. Fourteen required steroids and dual agent chemotherapy; three required further chemotherapy. One child died. Two children had successfully treated relapses. Ten developed diabetes insipidus, seven were growth hormone deficient, two suffered from hypothyroidism and one panhypopituitarism. Median follow-up of the cohort was 8 years 10 months (interquartile range: 5 years 5 months to 12 years 7 months).
Conclusion:
Langerhans cell histiocytosis is a rare disease in infants and young children, with a variable course ranging from self-limiting to life threatening. In very young children (under 2 years of age), multisystem disease is more common, requiring intensive chemotherapy and lifelong follow-up. Lasting sequelae were identified in over a third of patients, including endocrine dysfunction, hearing difficulties, neurological and psychological problems.
