Successful Salvage Treosulfan-Based Megachemotherapy With Allogeneic Stem Cell Transplantation in Nonsyndromic, Therapy-Resistant Disseminated Juvenile Xanthogranuloma: A Case Report

Transplant Proc. 2020 Nov;52(9):2844-2848. doi: 10.1016/j.transproceed.2020.05.025. Epub 2020 Jul 16.

Abstract

Juvenile xanthogranuloma (JXG) is a rare histiocytic disorder classified as non-Langerhans cell histiocytosis; although it is usually a benign and self-limiting disease, it can be fatal in some cases, especially with systemic dissemination. We present a case report of a boy with therapy-resistant disseminated JXG who was treated with systemic chemotherapy and received 3 allogeneic hematopoietic stem cell transplantations (allo-HSCTs) from an unrelated donor. The post-transplant period was complicated by acute graft vs host disease and lymphoproliferative disease caused by Epstein-Barr virus. Currently, almost 7.5 years after the first transplantation, the boy is in complete remission with full donor chimerism and without symptoms of JXG. The presented data confirm rare observations that allo-HSCT can lead to durable remission of systemic JXG, which warrants its use in life-threatening, therapy-resistant subtypes of disease.

Publication types

  • Case Reports

MeSH terms

  • Antineoplastic Agents, Alkylating / therapeutic use
  • Busulfan / analogs & derivatives*
  • Busulfan / therapeutic use
  • Child, Preschool
  • Combined Modality Therapy / methods*
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Male
  • Remission Induction
  • Salvage Therapy / methods*
  • Xanthogranuloma, Juvenile / complications
  • Xanthogranuloma, Juvenile / therapy*

Substances

  • Antineoplastic Agents, Alkylating
  • treosulfan
  • Busulfan