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Clinical and pathological evaluation of cladribine treatment response in a case series of patients with xanthoma disseminatum

  • Therapy
  • Published:
European Journal of Dermatology

Abstract

Background

Xanthoma disseminatum (XD) is a rare form of non-Langerhans histiocytosis with extensive cutaneous involvement. There is a paucity of evidence-based recommendations for treatment decision-making. Previous case reports have established purine analogues, especially cladribine, as a hopeful first-line treatment option, but characterization of the clinical and pathological responses is lacking.

Objectives

To characterize the clinical and pathological responses to cladribine monotherapy based on serial examinations in XD patients.

Materials & Methods

We retrospectively studied the clinical, pathological and laboratory data in a cohort of five XD patients who received intravenous cladribine monotherapy with serial examinations in our hospital. Compared with baseline characteristics, changes in clinical features and pathological patterns were identified and analysed. We also conducted a literature review of reported cases of cladribine treatment in XD patients.

Results

Four male and one female patient were involved in the study. All patients demonstrated satisfactory clinical responses to cladribine monotherapy after 5 to 10 cycles. We observed a pathological shift in pattern from classic xanthogranuloma to transitional fibrohistiocytic infiltration during the treatment, and pathological responses heralded persistent clinical improvement. Other than afebrile neutropenia, no prominent adverse events were identified. Sustainable lesion clearance was achieved in all five patients during the follow-up period, ranging from 19 to 66 months.

Conclusion

Cladribine monotherapy is an effective and well-tolerated therapeutic option for XD patients. Pathological transformation is a signature of the clinical response and possibly unveils the underlying histiocyte biology of diseases in the xanthogranuloma family.

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Funding

Funding support: this research was funded by the National Natural Science Foundation of China (Grant No. 81773349) to Y.Z. and Beijing Tsinghua Changgung Hospital Fund (Grant No. 12017C1007) to E.Y.Z..

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Author notes

  1. These authors contributed to this work equally and should be regarded as co-first authors.

  2. These authors contributed to this work equally and should be regarded as co-corresponding authors.

Authors and Affiliations

  1. Department of Dermatology, Beijing Tsinghua Changgung Hospital, School of Clinical Medicine, Tsinghua University, Beijing, China

    Eray Yihui Zhou, Yalan Bi, Hsiaohan Tuan & Yi Zhao

  2. Department of Hematology and Oncology, Beijing Tsinghua Changgung Hospital, School of Clinical Medicine, Tsinghua University, Beijing, China

    Fan Yu, Yanying Wang, Yuehua Huang, Bianhong Wang, Jingxian Li & Lihong Li

  3. Department of Pathology, Beijing Tsinghua Changgung Hospital, School of Clinical Medicine, Tsinghua University, Beijing, China

    Hongfang Yin

  4. Photomedicine Laboratory, Institute of Precision Medicine, Tsinghua University, Beijing, China

    Yi Zhao

Authors
  1. Eray Yihui Zhou
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  2. Fan Yu
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  3. Yanying Wang
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  4. Yalan Bi
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  7. Bianhong Wang
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  8. Jingxian Li
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  9. Hongfang Yin
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  10. Lihong Li
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  11. Yi Zhao
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Corresponding authors

Correspondence to Eray Yihui Zhou, Lihong Li or Yi Zhao.

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Conflicts of interest: none.

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Cite this article

Zhou, E.Y., Yu, F., Wang, Y. et al. Clinical and pathological evaluation of cladribine treatment response in a case series of patients with xanthoma disseminatum. Eur J Dermatol 33, 270–279 (2023). https://doi.org/10.1684/ejd.2023.4502

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  • DOI: https://doi.org/10.1684/ejd.2023.4502

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