Clinical outcomes, survival, and predictors in lower-risk myelodysplastic syndrome patients treated with cyclosporine A.

INTRODUCTION: Therapeutic options to improve myelodysplastic syndrome (MDS)-related cytopenias in patients with lower-risk MDS are limited, and cyclosporin A (CSA) is an available option. METHODS: We retrospectively analysed the clinical data of 153 consecutive patients with lower-risk MDS at our institution from July 1997 to October 2017. Propensity score matching method was used to balance the influence of confounding factors between patients with MDS treated with CSA and other conventional treatments (excluding CSA), and 50 pairs of cases were successfully identified for the final analysis. We assessed response rates, progression-free survival (PFS), overall survival (OS), and factors affecting response and survival. RESULTS: Haematological improvement (HI) was observed in 35 (70%) patients treated with CSA and in 25 (50%) patients treated with conventional therapies (P < 0.05), respectively. Treatment with CSA was a favourable prognostic factor for HI in lower-risk MDS patients of both cohorts in univariate analysis [odds ratio (OR) 2.333, P < 0.05], but not in multivariate analysis. In the multivariate analysis, hypocellular marrow was the only independent prognostic factor for HI in the CSA group (OR 6.259, P < 0.05), and in the overall cohort (OR 3.102, P < 0.05).CSA treatment did not improve PFS or OS (P > 0.05). CONCLUSION: CSA is a safe treatment and can significantly improve cytopenias in a substantial proportion of patients with MDS, especially in individuals with hypocellular bone marrow. However, CSA is not associated with PFS or OS.

Case report: Ruxolitinib plus dexamethasone as first-line therapy in haemophagocytic lymphohistiocytosis.

Haemophagocytic lymphohistiocytosis (HLH) is a cytokine-driven inflammatory syndrome caused by uncontrolled hypersecretion of inflammatory cytokines. Conventional first-line treatment for HLH included HLH-94 and HLH-2004 regimens. However, quite a few patients do not respond to treatment or cannot tolerate intensive chemotherapy. We reported two cases of HLH, one caused by natural killer (NK)/T-cell lymphoma and another associated with missense variants in the perforin 1 gene. They both received the ruxolitinib plus dexamethasone protocol and had a rapid response to treatment without obvious adverse effects. Our report indicates that treatment with ruxolitinib plus dexamethasone might be a potential option for HLH, and clinical trials warrant further investigation. In addition, the detection of HLH-related genes is necessary for the identification of late-onset familial HLH in certain settings.