Innovative sequential therapy in a rare case of refractory Erdheim-Chester disease with bilateral pulmonary involvement.
Zengyan Li, Zhiming Wu, Zelin Zhang et al.
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Case in brief
A 44-year-old man with Erdheim-Chester disease confirmed by lung biopsy presented with persistent fatigue, intermittent low-grade fever, and bilateral interstitial lung disease. Vincristine and prednisone followed by maintenance 6-mercaptopurine initially improved symptoms and imaging, but the disease progressed after six months. A second-line cytarabine-based regimen adapted from the Japan LCH Study Group-02 protocol was followed by sustained remission for over two years.
What made this case unusual
The case involved pulmonary-predominant ECD with bilateral interstitial lung disease and sustained remission after sequential cytotoxic chemotherapy, used as an alternative where targeted therapy was inaccessible.
Diagnostic / clinical pearl
In a patient with bilateral interstitial lung disease and systemic symptoms, lung biopsy may establish ECD; initial improvement on therapy does not exclude subsequent progression.
Why it matters
The reported response suggests sequential cytotoxic chemotherapy may be an option when targeted therapies such as BRAF inhibitors are unavailable, but broader studies are needed to assess its role.