This page addresses the management of primary myelofibrosis in patients with anaemia and an inadequate serum erythropoietin level (< 500 mU/mL), specifically in the setting where a prior treatment course did not achieve the expected haemoglobin response.
The patient has primary myelofibrosis with anaemia and a serum erythropoietin level below 500 mU/mL — a threshold below which an anaemia treatment trial is generally considered warranted in routine clinical practice.
A trial of danazol (with or without ruxolitinib), given for a minimum period of around 6 months, did not achieve the required haemoglobin response assessed at 6 months. Failure to reach this haemoglobin endpoint triggers escalation to the current protocol.
Following danazol failure, the protocol considers several alternative agents depending on local availability — options span more than one therapeutic class, and may be used alone or in combination. The complete regimen, selection criteria, and sequencing are available through the full structured protocol below.
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.
Consider alternatives dependent on availability: Momelotinib IMIDs Luspatercept Combinations
For those failing ESA and/or danazol, a trial of immunomodulatory drugs (IMiDs; thalidomide, lenalidomide or pomalidomide) either alone or in combination with prednisolone may be merited, but access is difficult, response rates overall remain low and toxicity is not inconsiderable (Grade 2B).
Finally, luspatercept is an erythroid-maturing agent which demonstrated efficacy in a trial for MF-associated anaemia in cohorts both on and off ruxolitinib.
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