This protocol addresses patients with primary myelofibrosis who have a suitable donor identified, are in good physiological condition with a good performance status and a non-high comorbidity index, yet carry a MF-related life expectancy of less than 5 years. These patients fall into intermediate-2 or high-risk disease categories by established prognostic scoring (DIPSS, MYSEC-PM, or MIPSS-70). Projected SCT-related survival exceeds 5 years, confirming appropriateness for transplant.
All of the following apply: a donor is available and SCT-related projected survival exceeds 5 years; physiological age and good performance status support transplant candidacy; the comorbidity index is not high; and MF-related life expectancy is below 5 years, placing the patient in the intermediate-2 or high-risk stratum under DIPSS, MYSEC-PM, or MIPSS-70.
A short course of targeted therapy — combined with anemia-oriented treatment — is used as bridge therapy ahead of transplant. The specific agents and full management algorithm are detailed in the complete protocol.
DOI: 10.1182/blood.2022017423
Donor available, and SCT-related survival higher than 5 years.
Physiological age, good PS, not-high CI.
MF-related life expectancy less than 5 years.
It is acceptable to select patients with an expected survival lower than 5 years (ie, intermediate-2 and high-risk DIPSS or MYSEC-PM/ high-risk MIPSS-70; Figure 1).
Short course of RUX/FEDR/PAC at MTD + anemia-oriented tx.
Patients on RUX who achieved clinical improvement at SCT had a lower risk of relapse (8% vs 19%) and better 2-year event-free survival (69% vs 54%) compared with those who did not.
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