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Ruxolitinib‐combined doxorubicin‐etoposide‐methylprednisolone regimen as a salvage therapy for refractory/relapsed haemophagocytic lymphohistiocytosis: a single‐arm, multicentre, phase 2 trial
Author(s) -
Wang Jingshi,
Zhang Rui,
Wu Xiaoyan,
Li Fei,
Yang Haixia,
Liu Ligen,
Guo Haixia,
Zhang Xuejun,
Mai Huirong,
Li Hui,
Wang Zhao
Publication year - 2021
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.17331
Subject(s) - medicine , regimen , salvage therapy , etoposide , gastroenterology , refractory (planetary science) , methylprednisolone , surgery , phases of clinical research , chemotherapy , physics , astrobiology
Summary We performed a multicentre, non‐randomised trial (NCT03533790) to investigate the efficacy of ruxolitinib combined with the doxorubicin‐etoposide‐methylprednisolone (Ru‐DEP) regimen as a salvage therapy for refractory/relapsed haemophagocytic lymphohistiocytosis (HLH). All patients failing to achieve a complete or partial response 2 weeks after initial HLH‐94/HLH‐04 regimen or relapsed after remission were enrolled in the study between June 2018 and June 2019. The efficacy was evaluated 2 weeks after initiating Ru‐DEP salvage therapy. Fifty‐four eligible patients with refractory/relapsed (R/R) HLH were enrolled. One case could not be evaluated for efficacy. Excluding 12 patients who had previously received the DEP regimen, the overall response rate was 32 of 41 (78·0%) patients, with eight of 41 (19·5%) achieving complete response and 24 of 41 (58·5%) attaining a partial response. Of the R/R HLH patients who had previously received the DEP regimen, 7 of 12 (58·3%) achieved a partial response. Ferritin and soluble CD25 concentrations were significantly lower ( P < 0·05), while the platelet count increased significantly ( P = 0·034), and triglycerides decreased significantly ( P = 0·002) compared with those before treatment. The Ru‐DEP regimen may be a safe and effective salvage therapy, remaining effective in refractory/relapsed HLH following DEP treatment, especially in macrophage activation syndrome. In addition, the regimen can be considered for patients with contraindications to glucocorticoid, especially those with gastrointestinal bleeding.