International Journal of Hematology

DOI: 10.1007/s12185-018-02572-z Pages: 1-8

Outcomes in children with hemophagocytic lymphohistiocytosis treated using HLH-2004 protocol in Japan

1. Shinshu University Hospital, Division of Blood Transfusion

2. Shinshu University School of Medicine, Department of Pediatrics

3. National Hospital Organization Nagoya Medical Center, Clinical Research Center

4. Shinshu University Hospital, Department of Laboratory Medicine

5. Kyoto University Graduate School of Medicine, Department of Pediatrics

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

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

8. Jichi Medical University School of Medicine, Department of Pediatrics

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

10. Miyagi Children’s Hospital, Department of Hematology and Oncology

11. Kagoshima University Graduate School of Medical and Dental Sciences, Department of Pediatrics

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

Correspondence to:
Yozo Nakazawa
Tel: +81-263-37-2642
Email: yxnakaza@shinshu-u.ac.jp

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Abstract

Recent advances in intensive chemo- and immunotherapy have contributed to the outcome of hemophagocytic lymphohistiocytosis (HLH); however, the prognosis of HLH in children differs by HLH subtype. In Japan, secondary HLH, particularly Epstein–Barr virus-associated HLH (EBV-HLH), is the most common HLH subtype. The prognosis of HLH has improved in recent years. We here conducted a prospective study of 73 patients who were treated with HLH-2004 protocol in Japan. EBV-HLH, familial HLH (FHL), and HLH of unknown etiology were seen in 41, 9, and 23 patients, respectively. Patients with resistant or relapsed disease after HLH-2004 treatment and those with FHL received hematopoietic stem cell transplantation (HSCT). The induction rate after initial therapy was 58.9%, and the 3-year overall survival (OS) rate of all patients was 73.9% and differed significantly among those with EBV-HLH, FHL, and HLH of unknown etiology. Of the 17 patients who received HSCT, the 3-year OS rates of those with and without complete resolution before HSCT were 83.3% and 54.5%, respectively. Outcomes in children with HLH who were treated with the same protocol differed among HLH subtypes. Appropriate strategy for each subtype should be established in future studies.

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