Abstract
Background: Langerhans Cell Histiocytosis (LCH) is a childhood disorder of histiocytes that is generally treated with systemic chemotherapy. Spontaneous resolution has been previously reported in Single System LCH (SS-LCH), which is less aggressive than multisystem disease. However, there are no clear guidelines on which patients can be safely spared from systemic chemotherapy. Here, we propose a risk stratification framework based on disease quiescence as determined by clinical and biochemical features of inflammation, to identify low risk patients who may be potentially spared from chemotherapy through a conservative “wait-and-see” approach.Methods: Retrospective analysis in a single institution was conducted in children with SS-LCH, comparing features of inflammation and outcomes of those who received chemotherapy vs. those with quiescent disease, who were managed conservatively.Results: Of 44 children with SS-LCH, only patients without risk-organ involvement were considered for conservative management. A “wait-and-see” approach was adopted for patients with quiescent disease as defined by clinical and biochemical evidence of disease activity. Following 2 weeks of watchful observation, decisions were made to either start treatment or continue conservative management. Based on data collected at diagnosis, patients with quiescent disease had a lower mean platelet count 339 × 109/L (95%C.I: 285–393) vs. 482 × 109/L (95% C.I: 420–544) p < 0.01, a lower mean white cell count 9.3 × 109/L (95%C.I: 7.5–11.1) vs. 13.1 × 109/L (95%C.I: 11–15.2) p < 0.01 and lower Erythrocyte-Sedimentation-Rate (ESR) 8.2 mm/h (95%C.I: 5.4–11) vs. 53.7 mm/h (95%C.I: 11–96.3) p = 0.04, suggesting that these are potential biochemical markers of disease activity. Other features of disease quiescence noted were rapid progression, functional disability, presence of a skull depression rather a lump and the lack of fever.Conclusions: Further studies are required to validate our proposed framework to determine disease activity in SS-LCH. Within the limits of this current analysis, it appears that low-risk patients with clinically and biochemically quiescent SS-LCH, may potentially be spared from chemotherapy with good long-term outcomes.
Highlights
Langerhans Cell Histiocytosis (LCH) in childhood can present with a wide spectrum of clinical manifestations with varying treatment outcomes that is largely dependent on disease severity
Langerhans-cells were first described by Paul Langerhans in the 1868 [1] as epidermal cells—that we know as dendritic cells—which have the ability to migrate from the epidermis to lymph nodes where they present antigens to T-cells
In order to further distinguish a subgroup of patients quiescent SS-LCH who may be spared from systemic chemotherapy, a modified approach based on clinical and biochemical features of disease activity in addition to current guidelines may be helpful
Summary
Langerhans Cell Histiocytosis (LCH) in childhood can present with a wide spectrum of clinical manifestations with varying treatment outcomes that is largely dependent on disease severity. Observational studies of children with single-system-LCH (SS-LCH) report that some of these patients can be managed conservatively [4,5,6,7] but decisions as to who receives local or systemic treatment vs no treatment within this subgroup is still largely based on physician discretion [8]. Langerhans Cell Histiocytosis (LCH) is a childhood disorder of histiocytes that is generally treated with systemic chemotherapy. We propose a risk stratification framework based on disease quiescence as determined by clinical and biochemical features of inflammation, to identify low risk patients who may be potentially spared from chemotherapy through a conservative “wait-and-see” approach
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