This first-line protocol applies to patients with primary myelofibrosis who present with anemia and carry a del(5q) cytogenetic abnormality. The specific chromosomal deletion identifies a distinct subpopulation for whom a targeted treatment approach is indicated.
Both the presence of clinically significant anemia and confirmation of the del(5q) abnormality on cytogenetic assessment are central to this protocol's indication. Treatment selection is directly informed by this cytogenetic finding.
Management in this setting involves an oral immunomodulatory agent shown to be effective specifically in the context of the del(5q) abnormality. The complete regimen — including agent selection, sequencing, and monitoring — is available in the full structured protocol.
The primary objective is an anemia response: transfusion cessation for previously transfusion-dependent patients, or a hemoglobin increase of 2 g/dL or greater for transfusion-independent patients.
DOI: 10.1182/blood.2022017423