Allogeneic Hematopoietic Stem Cell Transplantation Negates Poor Outcomes in Myelodysplastic Syndrome With Myelofibrosis

In a recent study, patients with myelodysplastic syndrome (MDS) who have myelofibrosis (MF) showed a worse prognosis when MF was of greater severity. However, this pattern of severity influencing prognosis appeared negated with allogeneic hematopoietic stem cell transplantation (allo-HSCT). The study findings were reported in the journal Experimental Hematology & Oncology.

Patients with MDS and MF were evaluated in this retrospective analysis for clinical, treatment, and prognostic characteristics. Patients were grouped by MF severity into 2 categories, with MF of grade 0 or 1 (MF-0/1) characterizing 1 group and grade 2 or 3 MF (MF-2/3) characterizing the other group.

A total of 316 patients were included in the study, of whom 273 were in the MF-0/1 group, and 43 patients were in the MF-2/3 group. Patients in the MF-2/3 group showed a greater prevalence of complex karyotype, compared with the MF-0/1 group (P =.001). Overall response rates (ORRs) to cytoreduction treatment also differed by MF severity, with the MF-0/1 group having an ORR of 53.3% and the MF-2/3 group having an ORR of 16.7% (P =.017).

Allo-HSCT was used with 141 patients, including 121 of those in the MF-0/1 group and 20 in the MF-2/3 group. The MF-0/1 group had a median time to neutrophil reconstruction of 12 days (range, 7-34) and a median time to platelet reconstruction of 14 days (range, 8-68), for a platelet level of >20×109/L. These times were longer for the MF-2/3 group, with a median time to neutrophil reconstruction of 14 days (range, 10-45 days; P =.005) and a median time to platelet reconstruction of 18 days (range, 8-65; P =.045).

At day 30 following HSCT, there was no significant difference between MF-0/1 and MF-2/3 groups regarding cumulative incidence of either neutrophil engraftment (P =.107) or platelet engraftment (P =.303). Additionally, rates of nonrelapse mortality, relapse, and acute or chronic graft vs host disease did not significantly differ by MF severity group.

Differences in 2-year overall survival (OS) by MF severity were apparent when patients were evaluated based on receipt of allo-HSCT. With allo-HSCT, the 2-year OS rate was 68.5% (95% CI, 60.1%-76.9%) for patients in the MF-0/1 group, and it was 68.4% (95% CI, 47.4%-89.4%) for patients in the MF-2/3 group (P =.636). Without allo-HSCT, however, patients in the MF-0/1 group had a 2-year OS rate of 49.9% (95% CI, 40.7%-59.1%), compared with a rate of 19.2% (95% CI, 0%-39.6%) for patients with MF-2/3 (P =.002).

Multivariate analysis suggested that complex karyotype was associated with several worse outcomes after transplantation. These included relapse (hazard ratio [HR], 4.16; P =.006), OS (HR, 2.47; P =.009), and disease-free survival (HR, 2.16; P =.020).

The researchers who performed the study concluded that in patients with MDS, the presence of MF-2/3 was associated with a complex karyotype and poorer ORR with cytoreduction versus MF-0/1 status, and that patients with MF-2/3 status who had allo-HSCT also had a worse prognosis. “However, the adverse impact of MF on prognosis may be overcome by allo-HSCT,” the researchers wrote in their report.


Zeng X, Xuan L, Fan Z, et al. Allogeneic stem cell transplantation may overcome the adverse impact of myelofibrosis on the prognosis of myelodysplastic syndrome. Exp Hematol Oncol. 2021;10(1):44. doi:10.1186/s40164-021-00238-x