International Journal of Hematology

DOI: 10.1007/s12185-019-02760-5 Pages: 137-148

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

1. Fujita Health University School of Medicine, Department of Pediatrics

2. Nippon Medical School, Department of Pediatrics

3. Hokkaido Government

4. Tokyo Medical and Dental University, Department of Child Health and Development, Graduate School of Medical and Dental Sciences

5. Kyushu University, Department of Pediatrics, Graduate School of Medical Sciences

6. Ehime University Graduate School of Medicine, Department of Pediatrics

7. Children’s Cancer Center, National Center for Child Health and Development

8. Kyoto Prefectural University of Medicine, Graduate School of Medical Science, Department of Pediatrics

9. Uji-Tokushukai Medical Center, Department of Laboratory Medicine

10. Juntendo University School of Medicine, Department of Pediatrics

11. Iwate Medical University, Department of Pediatrics

12. Okayama University, Graduate School of Medicine, Department of Pediatric Hematology/Oncology

13. Osaka University Graduate School of Medicine, Department of Pediatrics

14. National Kyushu Cancer Center, Department of Pediatrics

15. Osaka Women’s and Children’s Hospital, Department of Hematology/Oncology

16. Tokyo Metropolitan Cancer and Infectious Disease Center Komagome Hospital, Division of Pediatrics, Tokyo Cancer Registry

17. Jichi Medical University of Medicine, Department of Pediatrics

Correspondence to:
Kazuko Kudo



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.

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