# Survival and quality‐of‐life implications of cytopenia trajectories in ruxolitinib‐treated myelofibrosis

**Authors:** Francesca Palandri, Giovanni Caocci, Elisabetta Abruzzese, Mario Tiribelli, Erika Morsia, Mirko Farina, Giulia Benevolo, Eloise Beggiato, Bruno Martino, Novella Pugliese, Alessia Tieghi, Monica Crugnola, Gianni Binotto, Francesco Cavazzini, Alessandra Iurlo, Alessandro Isidori, Alessandra Dedola, Emilia Scalzulli, Andrea Duminuco, Daniele Cattaneo, Roberto M. Lemoli, Costanza Bosi, Daniela Cilloni, Monica Bocchia, Fabrizio Pane, Chiara Sartor, Florian H. Heidel, Massimo Breccia, Filippo Branzanti, Giuseppe A. Palumbo, Massimiliano Bonifacio, Elena M. Elli

PMC · DOI: 10.1002/cncr.70320 · Cancer · 2026-02-13

## TL;DR

This study shows that changes in blood cell levels during ruxolitinib treatment for myelofibrosis strongly affect patient survival and quality of life.

## Contribution

The study introduces cytopenia trajectory monitoring as a novel tool for risk stratification and treatment optimization in myelofibrosis.

## Key findings

- Persistent cytopenia during treatment is linked to the worst survival outcomes (3.7 years median OS).
- Improved anemia identifies a subgroup with better survival (5.2 years median OS).
- Noncytopenic patients had the best survival (8.1 years median OS).

## Abstract

Cytopenia is a common complication in patients with myelofibrosis and may worsen during treatment with ruxolitinib.

The RUX‐MF multicenter study evaluated 879 patients treated with ruxolitinib for at least 6 months, categorizing them into four groups based on the evolution of cytopenia: never cytopenic, treatment‐emergent cytopenia, persistent cytopenia, and improved anemia.

At baseline, 40.6% of patients presented with cytopenia, increasing to 57.8% after 6 months. Baseline cytopenia was associated with significantly reduced median overall survival (OS) compared to noncytopenic patients (3.7 vs. 6.7 years). Prognosis varied notably across groups: patients who remained noncytopenic had the median best OS (8.1 years), whereas those with persistent cytopenia had the worst (3.7 years). Treatment‐emergent cytopenia was linked to intermediate outcomes (5.1 years), with isolated thrombocytopenia showing the poorest prognosis (4.3 years) and anemia a slightly better one (6.1 years). Patients with improved anemia had better survival than those with persistent anemia (5.2 vs. 3.5 years). Symptom response mirrored survival trends, with the best outcomes in noncytopenic and improved anemia groups.

These findings highlight the prognostic significance of cytopenia dynamics during ruxolitinib therapy and support the use of cytopenia trajectory monitoring as a valuable tool for risk stratification and treatment optimization in myelofibrosis.

Cytopenia trajectories during ruxolitinib therapy in myelofibrosis provide strong prognostic information, with persistent or treatment‐emergent cytopenias associated with inferior survival. Hemoglobin recovery identifies a small subgroup with improved survival and quality of life.

## Linked entities

- **Chemicals:** ruxolitinib (PubChem CID 17754772)
- **Diseases:** myelofibrosis (MONDO:0044903)

## Full-text entities

- **Diseases:** Cytopenia (MESH:D006402), anemia (MESH:D000740), isolated thrombocytopenia (MESH:C564052), myelofibrosis (MESH:D055728)
- **Chemicals:** ruxolitinib (MESH:C540383)
- **Species:** Homo sapiens (human, species) [taxon 9606]

## Full text

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## Figures

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## References

35 references — full list in the complete paper: https://tomesphere.com/paper/PMC12904337/full.md

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Source: https://tomesphere.com/paper/PMC12904337