Clinical Features and Treatment Outcomes of Langerhans Cell Histiocytosis

Abstract Purpose To analyze the genetic and clinical features of children with MAP2K1-mutated Langerhans cell histiocytosis (LCH). Methods We compared the clinical features of 37 children with MAP2K1-mutated LCH with those of the BRAFV600E mutation group (n = 133) and no known mutation group (n = 59) in the same period. Results We found 13 mutations of the MAP2K1 gene, which were mainly concentrated at p.53–62 and p.98–103. The most common mutation site was c.172_186del (12/37). Compared with the BRAFV600E mutation group, the patients with MAP2K1 mutations were mainly characterized by single system multiple bone involvement (P = 0.022), with later disease onset (P = 0.029) as well as less involvement of risk organs, especially liver (P = 0.024). There was no significant difference in clinical features compared with the no known mutation group. The 2-year progression-free survival rate of first-line treatment (ChiCTR1900025783, 07/09/2019) in MAP2K1-mutated patients was 65.6% ± 9.5%. The prognosis of patients with lung involvement was poor [HR (95% CI) = 6.312 (1.769–22.526), P = 0.005]. More progression or relapses could be found in patients with bony thorax involvement (8/17 vs. 2/20, P = 0.023), yet involvements in other sites of bones, such as craniofacial bone involvement (8/26 vs. 2/11, P = 0.688) and limb bone involvement (5/12 vs. 5/25, P = 0.240), were not correlated to disease progression or relapse. Conclusion The children with MAP2K1-mutated LCH have specific clinical features requiring clinical stratification and precise treatment. MAP2K1-mutated patients with lung involvement (especially with bony thorax involvement) had poor prognosis.

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Clinical Features and Treatment of Langerhans Cell Histiocytosis

Acta Paediatrica ◽

10.1111/apa.16014 ◽

2021 ◽

Author(s):

Carlos Rodriguez‐Galindo

Keyword(s):

Clinical Features ◽

Langerhans Cell Histiocytosis ◽

Langerhans Cell

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Clinical features and outcomes of adult Langerhans cell histiocytosis: a single-center experience

International Journal of Hematology ◽

10.1007/s12185-020-02892-z ◽

2020 ◽

Vol 112 (2) ◽

pp. 185-192

Author(s):

Masayuki Kobayashi ◽

Shohei Ando ◽

Toyotaka Kawamata ◽

Junya Makiyama ◽

Kazuaki Yokoyama ◽

...

Keyword(s):

Clinical Features ◽

Langerhans Cell Histiocytosis ◽

Langerhans Cell ◽

Single Center ◽

Single Center Experience

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Clinical presentation and prognostic analysis of adult patients with Langerhans cell histiocytosis with pulmonary involvement

BMC Cancer ◽

10.1186/s12885-020-07421-z ◽

2020 ◽

Vol 20 (1) ◽

Author(s):

Hui-lei Miao ◽

Ai-lin Zhao ◽

Ming-hui Duan ◽

Dao-bin Zhou ◽

Xin-xin Cao ◽

...

Keyword(s):

Pulmonary Function ◽

Clinical Features ◽

Langerhans Cell Histiocytosis ◽

Pulmonary Involvement ◽

Langerhans Cell ◽

Adult Patients ◽

Prognostic Analysis ◽

History Of ◽

Smoking Index

Abstract Background The study aimed to investigate the clinical features and prognosis factors of adult patients with Langerhans cell histiocytosis (LCH) with pulmonary involvement, especially multisystem (MS) LCH with pulmonary involvement. Methods We retrospectively analyzed the demographic materials, clinical features and treatment outcomes of 119 adult LCH patients with pulmonary involvement at our center from January 1990 to November 2019. Results Among 119 patients, 13 (10.9%) had single-system (SS) LCH, and 106 (89.1%) had MS-LCH with pulmonary involvement. SS-LCH patients had higher smoking rate (84.6% vs 52.8%, P = 0.026) and smoking index (300 vs 200, P = 0.019) than MS-LCH patients. The percentage of respiratory symptoms of SS-LCH patients was higher than MS-LCH patients (84.6% vs 53.8%, P = 0.034). Pulmonary function was impaired in 83.8% of the patients, and DLCO was the parameter most frequently impaired, accounting for 81.1%. The median DLCO was 65.1% predicted. Patients with pneumothorax had significantly worse DLCO (P = 0.022), FEV1 (P = 0.000) and FEV1/FVC (P = 0.000) than those without pneumothorax. During the follow-up, 72.4% of the patients had stable pulmonary function, and 13.8% showed improvements after chemotherapy. The estimated 3-year OS and EFS were 89.7 and 58.3%, respectively. Patients with a baseline FEV1 ≤ 55% predicted had worse OS. A history of pneumothorax indicated worse EFS and cytarabine based therapy predicted better EFS. Conclusions An FEV1 ≤ 55% predicted and a history of pneumothorax at diagnosis indicated a poor prognosis. Cytarabine based regimen may arrest the decline in pulmonary function in LCH patients with pulmonary involvement and improve EFS.

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