Long‐term outcome, clinical course and treatment approaches of paediatric langerhans cell histiocytosis: A greek reference centre report

Tzotzola, Vasiliki; Petrikkos, Loizos; Papadakis, Vassilios; Mitropoulou, Georgia; Kelaïdi, Charikleia; Dimitriadis, E.; Polychronopoulou, Sophia

doi:10.1111/apa.15743

Cited by 6 publications

(7 citation statements)

References 26 publications

(81 reference statements)

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“…In addition, no neurodegenerative LCH was observed in our adolescent series, which is consistent with the low incidence of CNS sequala previously reported in adult patients (3.8–10%) compared to that in paediatric patients[ 9 , 28 , 31 , 35 ]. Unlike the better EFS of paediatric SS-s LCH patients than that of MS patients[ 5 , 45 , 46 ], SS-s patients in our adolescent series had a similar EFS compared to MS patients (102.8 months vs. 71.8 months, p = 0.132), which is similar to that of adult patients[ 9 ].…”

Section: Discussionmentioning

confidence: 49%

Langerhans cell histiocytosis in adolescent patients: a single-centre retrospective study

Cai

Chen

Liu

et al. 2022

Orphanet J Rare Dis

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Background Langerhans cell histiocytosis (LCH) is a myeloid dendritic cell disorder frequently affecting children more than adults. The presentation of LCH varies with age, however, the clinical characteristics and genetic profiles of adolescent LCH remain elusive. To address the knowledge gap, we performed a single-centre retrospective study of 36 adolescent LCH patients aged between 14 and 17 years at Peking Union Medical College Hospital. Results At the time of diagnosis, 10 patients were classified as unifocal single system LCH (27.8%), 2 patients had pulmonary single system LCH (5.6%), 5 patients had multifocal single system LCH with bone involvement (13.9%), and 19 patients had multisystem LCH (52.8%). The most prevalent involvement in multisystem patients was the pituitary gland (78.9%), followed by the bone (42.1%), lung (42.1%), and lymph nodes (42.1%). Eight (42.1%) patients had risk organ involvement. BRAFN486_P490 was detected in 50% of patients who underwent next generation sequencing, and BRAFV600E was detected in one patient. Chemotherapies were the first line treatment in 24 patients. One patient died and thirteen patients relapsed during the follow-up. The estimated 5-year OS rate and EFS rate were 94.7% and 59.0%, respectively. Conclusions In this study, we report a large series of adolescent LCH patients. The clinical characteristics of adolescent LCH patients may be close to adult LCH. Compared with pediatric cases, adolescent LCH tends to have more pituitary lesions and pulmonary involvement, fewer skin and hematopoietic involvement, a higher frequency of BRAF deletion mutation, and a lower frequency of BRAFV600E mutation.

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Section: Discussionmentioning

confidence: 49%

Langerhans cell histiocytosis in adolescent patients: a single-centre retrospective study

Cai

Chen

Liu

et al. 2022

Orphanet J Rare Dis

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“…Skin lesions may resolve spontaneously or may need topical steroids, oral methotrexate, or thalidomide ( 5 ). These patients with localized disease usually have an excellent event-free survival (EFS) and overall survival (OS) of 80.6% and 100%, respectively ( 6 , 7 ).…”

Section: Introductionmentioning

confidence: 99%

“…Treatment with prednisone and vinblastine, according to LCH IV Protocol ( 8 ), represents the standard of care of MS-LCH with a 10-year OS and EFS rates of 49% and 79%, respectively ( 7 , 9 , 10 ). Several studies demonstrated that OS is significantly lower among patients younger than 2 years of age, who frequently show a RO involvement, elevated inflammatory markers, and resistance to standard treatment regimens ( 11 – 13 ).…”

Section: Introductionmentioning

confidence: 99%

Case Report: Early Association of Vemurafenib to Standard Chemotherapy in Multisystem Langerhans Cell Histiocytosis in a Newborn: Taking a Chance for a Better Outcome?

et al. 2021

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Langerhans cell histiocytosis (LCH) is due to aberrant monoclonal proliferation and accumulation of dendritic cells, ranging from a self-limiting local condition to a rapidly progressive multisystem disease with poor prognosis. Pathogenic cells originate from a myeloid-derived precursor characterized by an activation of the MAPK/ERK signaling pathway in about 70% of cases. In particular, BRAF V600E mutation is usually associated with a more severe clinical course and poor response to chemotherapy. We report on a newborn with multisystem LCH in life-threatening medical conditions. At diagnosis, the patient was successfully treated with the early association of BRAF inhibitor Vemurafenib to standard chemotherapy representing a new approach in first-line treatment. A rapid clinical improvement with a prompt fever regression from day 2 and complete resolution of skin lesions by week 2 were observed; laboratory data normalized as well. Vemurafenib was discontinued after 12 months of treatment. No signs of relapse occurred after 12 months of discontinuation. This case indicates that early combination of target therapy with standard treatment may induce rapid response and prolonged disease remission without significant toxicities in infants. This approach represents a valid and safe option as first-line treatment in multisystem disease, especially in high-risk patients.

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“…Skin lesions may resolve spontaneously or may be treated with topical steroids, oral methotrexate or thalidomide 5 . These patients with localized disease usually have excellent event-free survival (EFS) and overall survival (OS) of 80.6% and 100%, respectively 6,7 .…”

Section: Introductionmentioning

confidence: 99%

“…Treatment with prednisone and vinblastine, according to LCH IV Protocol 8 , represents the standard-of-care of MS-LCH, with a 10-year OS and EFS rates of 49% and 79% respectively 7,9,10 . Several studies demonstrated that OS is significantly lower among patients younger than 2 years of age, who frequently show a RO involvement, elevated inflammatory markers and resistance to standard treatment regimens [11][12][13] .…”

Section: Introductionmentioning

confidence: 99%

Early association of Vemurafenib to standard chemotherapy in multisystemic Langerhans cell histiocytosis in a newborn: take the chance for a better outcome?

Gaspari

Ruscio

Stocchi

et al. 2021

Preprint

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LCH is an aberrant monoclonal proliferation of dendritic cells, ranging from a self-limiting local condition to a rapidly progressive multisystemic disease. Pathogenic cells expressed, in almost 70% of cases, an activation of the MAPK/ERK signaling pathway, in particular BRAF V600E mutation. We report on a newborn with multisystemic disease diagnosed in life-threatening medical conditions, who was successfully treated with the early association of BRAF inhibitor Vemurafenib to chemotherapy. After 12 months, Vemurafenib was discontinued, without any signs of relapse. This case indicates that early combination of target therapy with standard treatment may induce rapid response and prolonged disease remission.

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Long‐term outcome, clinical course and treatment approaches of paediatric langerhans cell histiocytosis: A greek reference centre report

Cited by 6 publications

References 26 publications

Langerhans cell histiocytosis in adolescent patients: a single-centre retrospective study

Langerhans cell histiocytosis in adolescent patients: a single-centre retrospective study

Case Report: Early Association of Vemurafenib to Standard Chemotherapy in Multisystem Langerhans Cell Histiocytosis in a Newborn: Taking a Chance for a Better Outcome?

Early association of Vemurafenib to standard chemotherapy in multisystemic Langerhans cell histiocytosis in a newborn: take the chance for a better outcome?

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