Cytopenia Associated With Worse Survival Outcomes in Patients With Myelofibrosis
Key Takeaways:
- In an analysis of the RUX-MF clinical trial, the presence of cytopenia was associated with worse patient outcomes and reduced ruxolitinib dosing in patients with myelofibrosis (MF).
- Patients with thrombocytopenia had the lowest overall survival (OS) medians, illustrating how this form of cytopenia has a greater impact on health outcomes.
- Cytopenia is also associated with increased symptom burden, as patients with cytopenia had higher symptom scores and fewer score reductions than patients without.
Cytopenia, particularly anemia and thrombocytopenia, is a common complication among patients with MF that is associated with poor survival outcomes.
Prior studies have indicated that ruxolitinib, although effective in managing disease progression and reducing splenomegaly, may negatively impact cytopenia.
Study Methods and Outcomes
Researchers conducted an analysis of the RUX-MF trial to evaluate cytopenia as a prognostic marker among patients with MF. Other aims of the study included comparing outcomes of persistent and treatment-emergent cytopenia, assessing the impact of hemoglobin improvement on survival outcomes, and examining how cytopenia impacted symptom burden.
The study included 879 patients with MF who were treated with ruxolitinib for at least 6 months. Patients were categorized into 1 of 4 groups:
- Never cytopenic: no cytopenia at baseline or after 6 months
- Treatment-emergent cytopenia: no cytopenia at baseline but developed cytopenia during 6-month study period
- Persistent cytopenia: cytopenia at baseline and 6-month follow-up
- Improved anemia: anemia at baseline, Hb >10 g/dL at 6-month follow-up
Patient Characteristics
Among the 879 patients included in the study, 357 (40.6%) had cytopenia at the beginning of the study. Of those, 301 (34.2%) had anemia, 32 (3.6%) had thrombocytopenia, and 24 (2.7%) at both.
At the 6-month follow-up, 508 patients (57.8%) presented with cytopenia. Overall, 317 patients (36.1%) were never cytopenic, 273 patients (31.1%) had treatment-emergent cytopenia, 235 patients (26.7%) had persistent cytopenia, and 54 patients had improved anemia.
Patients who were never cytopenic had higher doses of ruxolitinib than the other cohorts, and patients with treatment-emergent and persistent cytopenia had more frequent dose reductions.
Outcomes Based on Cytopenia
Patients with cytopenia at baseline had a shorter median OS than those without, 3.7 and 6.7 years, respectively. Median OS was similar between patients with anemia only (4.05 years), thrombocytopenia only (3.85 years), and both (4.41 years).
The never cytopenic cohort had the highest survival outcomes, with a median OS of 8.07 years. The treatment-emergent cytopenic group had a median OS of 5.11 years (patients with both anemia and thrombocytopenia). The persistent cytopenia cohort had the lowest survival outcomes, with a median OS of 3.68 years.
Within the treatment-emergent cytopenic cohort, investigators evaluated the effect of type of cytopenia on patient outcomes. Patients who only developed anemia had a median OS of 6.15 years. Thrombocytopenia was associated with lower survival, as patients with this type of cytopenia had a median OS of 4.33 years.
Among the patients who presented with anemia at baseline, 17.9% had Hb >10 g/dL at the 6-month follow-up without also developing thrombocytopenia. Patients in the improved anemia cohort had a median OS of 5.18 years, significantly higher than that of patients with persistent anemia.
The Impact of Cytopenia on Symptom Burden
At baseline, patients in the improved anemia group had the highest Total Symptom Score (TSS) at 35.3 out of all the cohorts. In comparison, baseline TSS was 27.1 among patients with persistent cytopenia, 26.8 among those with treatment-emergent cytopenia, and 23.0 among those who were never cytopenic.
After 6 months, the improved anemia cohort had a significantly lower TSS than patients with persistent anemia, 14.3 and 16.4, respectively, suggesting that hemoglobin recovery could improve quality of life.
Patients who were never cytopenic and who had improved anemia had the highest average TSS reductions, –63.5% and –57.8%, respectively. Patients in the treatment-emergent and persistent cytopenia groups had TSS reductions of –49.3% and –44.4%, respectively.
Using Cytopenia as a Prognosis Marker for Patients With MF
These findings demonstrate the association of cytopenia, particularly thrombocytopenia, with worse survival outcomes among patients with MF.
Cytopenia could be used as a risk stratification tool to signal suboptimal response or possible disease progression. Identifying cytopenia early may allow physicians to optimize treatment for MF.
The authors said, “These results underscore the evolving nature of hematologic risk during ruxolitinib therapy and reinforce the importance of continued blood count surveillance.”
Reference
Palandri F, Caocci G, Abruzzese E, et al. Survival and quality-of-life implications of cytopenia trajectories in ruxolitinib-treated myelofibrosis. Cancer. 2026;132(4):e70320. doi:10.1002/cncr.70320
