Histiocytic Neoplasms, Version 2.2021, NCCN Clinical Practice Guidelines in Oncology

Go, Ronald S.; Jacobsen, Eric; Baiocchi, Robert A.; Buhtoiarov, Ilia N.; Butler, Erin; Campbell, Patrick; Coulter, Don W.; Diamond, Eli L.; Flagg, Aron; Goodman, Aaron M.; Goyal, Gaurav; Gratzinger, Dita; Hendrie, Paul C.; Higman, Meghan A.; Hogarty, Michael D.; Janků, Filip; Karmali, Reem; Morgan, David; Raldow, Anne C; Stefanovic, Alexandra; Tantravahi, Srinivas K.; Walkovich, Kelly; Zhang, Ling; Bergman, Mary Anne; Darlow, Susan

doi:10.6004/jnccn.2021.0053

Cited by 36 publications

(43 citation statements)

References 144 publications

(355 reference statements)

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“…Patients with lesions of the facial bones or anterior or middle cranial fossae with intracranial tumor extension comprise a CNS-risk group. These patients have an increased risk of developing other CNS diseases and DI so systemic treatment is highly recommended [43].…”

Section: Unifocal Skeletal Involvementmentioning

confidence: 99%

“…For patients with multifocal bone LCH, bisphosphonates can be used as the initial treatment approach [42] and in case of no response radiation is the second step if 1-2 lesions while systemic therapy should be preferred in >2 lesions [43]. Chemotherapy is also frequently used.…”

Section: Single System Polyostotic Diseasementioning

confidence: 99%

“…The resolution of bone lesions is explained by the anti-osteoclastic effect of bisphosphonates that helps reduce the noxious inflammatory substances and other degrading cytokines in the active lesions [42]. Importantly, a beneficial effect of bisphosphonate treatment in co-existing non-osseous LCH lesions has also been reported [42]; moreover, indomethacin is also suggested as an alternative therapeutic approach for patients with primary and recurrent bone involvement [43].…”

Section: Single System Polyostotic Diseasementioning

confidence: 99%

“…For patients with bone disease within an MS symptomatic setting, first-line treatment options include chemotherapy with cytarabine, cladribine (2-CdA), methotrexate or methotrexate plus cytarabine, or hydroxyurea given that LCH shares both features of an inflammatory and neoplastic disease [43]. The combination of vinblastine and prednisone therapy has never been prospectively proven as effective among adults, in contrast with pediatric LCH [46,47].…”

Section: Multisystem Diseasementioning

confidence: 99%

“…In the same setting trametinib is also an option [53,54]. Finally, targeted therapy (e.g., crizotinib, pexidartinib, larotrectinib, entrectinib, sirolimus, selpercatinib) could be useful in certain circumstances where other gene mutations have been identified [43].…”

Section: Multisystem Diseasementioning

confidence: 99%

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Adult Langerhans Cell Histiocytosis and the Skeleton

Georgakopoulou

Anastasilakis

Makras

2022

JCM

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Langerhans cell histiocytosis (LCH) is a rare inflammatory neoplasia in which somatic mutations in components of the MAPK/ERK pathway have been identified. Osseous involvement is evident in approximately 80% of all patients and may present as a single osteolytic lesion, as a multi-ostotic single system disease or as part of multisystem disease. Both exogenous, such as treatment with glucocorticoids, and endogenous parameters, such as anterior pituitary hormone deficiencies and inflammatory cytokines, may severely affect bone metabolism in LCH. Computed tomography (CT) or magnetic resonance imaging (MRI) are usually required to precisely assess the degree of bone involvement; 18F-fluorodeoxyglucose (FDG) positron emission tomography—CT can both detect otherwise undetectable LCH lesions and differentiate metabolically active from inactive or resolved disease, while concomitantly being useful in the assessment of treatment response. Treatment of skeletal involvement may vary depending on location, extent, size, and symptoms of the disease from close observation and follow-up in unifocal single-system disease to chemotherapy and gene-targeted treatment in cases with multisystem involvement. In any case of osseous involvement, bisphosphonates might be considered as a treatment option especially if pain relief is urgently needed. Finally, a patient-specific approach is suggested to avoid unnecessary extensive surgical interventions and/or medical overtreatment.

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Section: Unifocal Skeletal Involvementmentioning

confidence: 99%

Section: Single System Polyostotic Diseasementioning

confidence: 99%

Section: Single System Polyostotic Diseasementioning

confidence: 99%

Section: Multisystem Diseasementioning

confidence: 99%

Section: Multisystem Diseasementioning

confidence: 99%

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Adult Langerhans Cell Histiocytosis and the Skeleton

Georgakopoulou

Anastasilakis

Makras

2022

JCM

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Efficacy of denosumab monotherapy among adults with Langerhans cell histiocytosis: A prospective clinical trial

et al. 2023

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This phase IIb clinical trial evaluated the efficacy of a bimonthly treatment schedule (Q8W) with 4 subcutaneous doses of denosumab 120 mg among adults with Langerhans cell histiocytosis needing first‐line systemic therapy for either multifocal single‐system disease or multisystem disease without risk organ involvement. Two months after the last treatment administration, seven patients showed disease regression, one stable disease, one non‐active disease, and one disease progression. One year after treatment, progression was evident in two patients, while the remaining exhibited either a regression (three patients) or non‐active disease (five patients). No permanent sequalae developed during the study and no adverse events were adjudicated in treatment. In conclusion, four doses of denosumab 120 mg Q8W subcutaneously are an effective treatment option in Langerhans cell histiocytosis patients without risk organ involvement exhibiting a response rate of 80%. Further studies are needed to confirm its role as a disease modifying agent.

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Langerhans cell histiocytosis: NACHO update on progress, chaos, and opportunity on the path to rational cures

Bielamowicz,

Dimitrion,

Abla

et al. 2024

Cancer

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Langerhans cell histiocytosis (LCH) is a myeloid neoplastic disorder characterized by lesions with CD1a‐positive/Langerin (CD207)‐positive histiocytes and inflammatory infiltrate that can cause local tissue damage and systemic inflammation. Clinical presentations range from single lesions with minimal impact to life‐threatening disseminated disease. Therapy for systemic LCH has been established through serial trials empirically testing different chemotherapy agents and durations of therapy. However, fewer than 50% of patients who have disseminated disease are cured with the current standard‐of‐care vinblastine/prednisone/(mercaptopurine), and treatment failure is associated with long‐term morbidity, including the risk of LCH‐associated neurodegeneration. Historically, the nature of LCH—whether a reactive condition versus a neoplastic/malignant condition—was uncertain. Over the past 15 years, seminal discoveries have broadly defined LCH pathogenesis; specifically, activating mitogen‐activated protein kinase pathway mutations (most frequently, BRAFV600E) in myeloid precursors drive lesion formation. LCH therefore is a clonal neoplastic disorder, although secondary inflammatory features contribute to the disease. These paradigm‐changing insights offer a promise of rational cures for patients based on individual mutations, clonal reservoirs, and extent of disease. However, the pace of clinical trial development behind lags the kinetics of translational discovery. In this review, the authors discuss the current understanding of LCH biology, clinical characteristics, therapeutic strategies, and opportunities to improve outcomes for every patient through coordinated agent prioritization and clinical trial efforts.

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Histiocytic Neoplasms, Version 2.2021, NCCN Clinical Practice Guidelines in Oncology

Cited by 36 publications

References 144 publications

Adult Langerhans Cell Histiocytosis and the Skeleton

Adult Langerhans Cell Histiocytosis and the Skeleton

Efficacy of denosumab monotherapy among adults with Langerhans cell histiocytosis: A prospective clinical trial

Langerhans cell histiocytosis: NACHO update on progress, chaos, and opportunity on the path to rational cures

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