Treatment of Primary Myelofibrosis in Patients Aged ≤70 with Intermediate-2 or High-Risk Disease (IPSS/DIPSS/DIPSS+) Who Are Transplant Eligible

This protocol applies to patients with primary myelofibrosis classified as intermediate-2 or high-risk by IPSS, DIPSS, or DIPSS+, who are aged 70 years or younger and are considered transplant eligible. This subgroup warrants a distinct, structured treatment approach aimed at optimising the path to allogeneic haematopoietic stem cell transplantation.

Clinical Scenario

Patients aged ≤70 years with intermediate-2 or high-risk disease by established prognostic scoring (IPSS, DIPSS, or DIPSS+) should all be considered potential candidates for allogeneic haematopoietic stem cell transplantation (allo-HSCT). Transplant eligibility is a defining factor that shapes the treatment strategy in this population.

Treatment Approach — Partial Overview

The approach involves targeted therapy to maximise reduction of spleen size and control of symptoms before transplant proceeds. The full sequence of interventions, specific agent selection, and criteria for proceeding to transplant are detailed in the complete protocol.

Primary Goal Reduction of palpable splenomegaly to below 5 cm prior to transplant — associated with improved engraftment.

Instant Access to Structured Evidence-Based Regimens

References

All patients with intermediate-2 or high-risk disease according to IPSS, DIPSS or DIPSS+, and age ≤70 years, should be considered potential candidates for allo-HSCT.
JAK inhibitor to maximise spleen and symptom response.
Pretransplant therapy with JAKi or enrolment in a suitable clinical trial to maximise spleen response is warranted prior to allo-HSCT in those with bulky splenomegaly.
A large EBMT retrospective study suggested that splenomegaly should ideally be reduced to <5 cm palpable prior to allo-HSCT, and that this is associated with improved engraftment.

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