This protocol targets a defined subgroup of primary myelofibrosis: patients with symptomatic splenomegaly and/or prominent disease-related symptoms, classified as non-low risk, with a platelet count of ≥50×10⁹/L — a population for whom active therapeutic intervention is indicated.

Clinical Scenario

Symptomatic splenomegaly and/or constitutional symptoms; non-low-risk disease classification; platelet count ≥50×10⁹/L. The combination of symptomatic organ involvement and non-low-risk status defines the threshold for structured treatment selection.

Treatment Approach — Partial Overview

The approach in this setting includes approved targeted therapies — ruxolitinib is among the options considered — along with possible enrolment in clinical trials. The full sequence of options, decision criteria, and patient-specific considerations are contained in the complete structured protocol.

Response thresholds: The primary clinical goals are meaningful spleen volume reduction and a substantial reduction in total symptom burden, both assessed at week 24.

References

Symptomatic splenomegaly and/or symptoms
Non-low risk
PLT ≥50×10⁹/L
RUX, FEDR, or; Clinical trials
Spleen volume reduction equal to or greater than 35% (SVR35) and total symptom score reduction equal to or higher than 50% (TSS50) from baseline to week 24 were mostly the main end points.

DOI: 10.1182/blood.2022017423

View source ↗

Treatment of Primary Myelofibrosis with Symptomatic Splenomegaly in Non-Low-Risk Disease

References