Nationwide retrospective review of hematopoietic stem cell transplantation in children with refractory Langerhans cell histiocytosis

The Histiocytosis study group of the Japanese Society of Pediatric Hematology/Oncology

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Abstract

The efficacy of and indications for hematopoietic stem cell transplantation (HSCT) in pediatric Langerhans cell histiocytosis (LCH) remain undetermined. This retrospective study analyzed 30 children with refractory LCH who underwent HSCT in Japan between 1996 and 2014. Eleven patients received a myeloablative conditioning (MAC) regimen, while 19 patients received a reduced-intensity conditioning (RIC) regimen. Among the 26 patients with complete data, 23 patients had risk organ (RO) involvement during clinical course. Disease status at HSCT was no active disease (NAD) (4), active disease-regression (AD-r) (2), active disease-stable (AD-s) (4), and active disease-progressive (AD-p) (16). Seventeen of the 30 patients (57%) were alive with a median follow-up of 433 days (range 9–5307) after HSCT. Death occurred within 3 months after HSCT in eight of 13 patients. RIC and MAC patients were similar in both overall survival (OS) (56.8% vs. 63.6%, respectively, p = 0.789) and failure-free survival (56.8% vs. 54.6%, respectively, p = 0.938). Regarding disease status at HSCT, the six patients with NAD/AD-r experienced better outcomes than the 20 with AD-s/AD-p (5-year OS, 100% vs. 54.5%, respectively, p = 0.040). Disease state at the time of HSCT was the most important prognostic factor.

Original languageEnglish
Pages (from-to)137-148
Number of pages12
JournalInternational journal of hematology
Volume111
Issue number1
DOIs
Publication statusPublished - Jan 1 2020

Keywords

  • Hematopoietic stem cell transplantation (HSCT)
  • Reduced-intensity conditioning (RIC)
  • Refractory Langerhans cell histiocytosis (LCH)

ASJC Scopus subject areas

  • Hematology

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