Treatment of Primary Myelofibrosis with Anaemia When an Erythropoiesis-Stimulating Agent Did Not Work
This protocol addresses patients with primary myelofibrosis who have anaemia alongside an inadequate serum erythropoietin level, and whose haemoglobin did not respond to a prior erythropoiesis-stimulating agent trial.
Clinical Scenario
Primary myelofibrosis with anaemia and an inadequate serum erythropoietin level (<500 mU/mL). Routine practice supports an initial erythropoiesis-stimulating agent trial in this EPO range; when that trial fails, a different therapeutic approach is warranted.
Previous Treatment — Failure Condition
A 12-week trial of an erythropoiesis-stimulating agent (recombinant erythropoietin or darbepoetin) was completed. The required goal — a haemoglobin response at 3 months — was not achieved. This protocol is the recommended next step following that failure.
Treatment Approach
In this situation, a semisynthetic androgen-based therapy may be considered — given alone or in combination with another agent — over a defined monitoring period. The full regimen, combination rules, and assessment criteria are in the complete protocol.
Treatment Goal
The primary measure of success is a haemoglobin response (rise), evaluated at 6 months of treatment.
References
Serum EPO level. Inadequate. However routine practice would suggest that those with EPO levels <500 mU/mL likely merit an ESA trial for 12 weeks.
Danazol, a semisynthetic androgen, can demonstrate anaemia responses in up to 30% of patients.
If tolerated, a minimum of 12 weeks of therapy is warranted to assess if there is a response.
A trial of danazol, with or without ruxolitinib, can be considered initially for a period of 6 months including in patients who have failed a trial of ESA (Grade 1C).
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