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Cyclosporine A improves survival in lower-risk hypoplastic myelodysplastic syndromes: a single-center retrospective
Yumei Liu1,2,3, Liyan Yang1,2,3, Yansong Wei1
1Department of Hematology, Tianjin Medical University General Hospital, Tianjin, People's Republic of China.
Background:
Hypoplastic myelodysplastic syndrome (hMDS) is an uncommon MDS subtype, accounting for approximately 10-15% of cases, characterized by bone marrow hypocellularity, severe cytopenias, and features of immune dysregulation. Its clinical course overlaps with aplastic anemia, yet optimal management for lower-risk patients remains undefined, particularly in Asian populations.
Methods:
We retrospectively analyzed 48 patients with lower-risk hMDS (IPSS-R very low/low/intermediate) treated at Tianjin Medical University General Hospital from 2013 to 2021. Patients received cyclosporine A (CsA, n = 16), hypomethylating agents (HMAs, n = 10), low-dose HMAs (LD-HMAs, n = 11), or best supportive care (BSC, n = 11). Responses were assessed by modified IWG criteria, and survival was evaluated by Kaplan-Meier and Cox regression analyses.
Results:
Median age was 61 years (range 16-84), with 60.4% male. Somatic mutations were present in 64.6%, and PNH clones in 29.2%. Six-month overall response rates were 75.0% for CsA, 70.0% for HMAs, 54.5% for LD-HMAs, and 36.4% for BSC (p = 0.220). CsA achieved significantly longer complete remission (median 75 months) and hematologic improvement durations (p < 0.05). Median overall survival (OS)/progression-free survival (PFS) for CsA were 78.0/76.0 months, compared with 17.0/6.8 for HMAs, 31.0/15.0 for LD-HMAs, and 36.0/15.0 for BSC (p < 0.01). HMAs were associated with increased grade 3-4 infections.
Conclusion:
CsA provides superior response durability, survival outcomes, and safety compared with other approaches, supporting its use as first-line therapy in lower-risk hMDS. Integration of immune and molecular profiling may refine individualized treatment strategies.
