Transplant underuse in intermediate-2/high-risk myelofibrosis
Current understanding
Real-world registry data show that a large fraction of transplant-eligible intermediate-2/high-risk MF patients never reach allo-HSCT despite guideline endorsement. In a Spanish national registry of 1649 MF patients diagnosed from 2010 onward, only 25% of transplant-eligible intermediate-2/high-risk patients were actually transplanted, and overall median survival was 6.6 years with 5-/10-year relative survival of 68%/44% and acute leukemia as the leading cause of death — quantifying the referral gap in the JAK inhibitor era.
Evidence log
- 2026-09-19 — Clinicopathologic Characteristics of SomaticMutations in Philadelphia Chromosome-Negative Myeloproliferative Neoplasms: A Systematic Review.: PRISMA-guided pooled synthesis finds TP53 mutations enriched in MF with multi-hit configuration driving a steep OS drop specifically in chronic-phase disease — arguing that TP53 (and multi-hit resolution) should be part of the referral trigger for the int-2/high-risk MF population currently under-transplanted, since the survival penalty is realized before overt AP/BP transformation. (novelty: 3)
- 2026-07-20 — Reduced Intensity Haploidentical Transplantation for the Treatment of Primary or Secondary Myelofibrosis: RIC haploidentical HCT protocols with peri-transplant JAK inhibition broaden donor availability for MF patients, potentially narrowing the referral gap for int-2/high-risk patients who currently miss transplant for lack of a matched donor. (novelty: 3)
- 2026-07-01 — Genomic profiling for decision-making in post-polycythemia vera and post-essential thrombocythemia myelofibrosis.: cross-connection with mysec-mpm-kmpm: Molecular/karyotype upstaging via MYSEC-mPM/kmPM may reclassify secondary MF patients into higher-risk strata where transplant is currently underused. (novelty: 3)
- 2026-07-18 — Acute Myeloid Leukemia Subtype-Specific Prognostic Value of NGS in the Setting of Intensive Chemotherapy.: Same Mayo series showing 0% 5-year transplant-censored survival for post-MPN AML after 7+3 quantifies the downstream cost of the int-2/high-risk MF referral gap: patients who progress to blast phase before transplant are essentially unsalvageable with intensive chemotherapy alone. (novelty: 3)
- 2026-06-30 — A systematic review of 47 published cases of extramedullary hematopoiesis in myelofibrosis: clinical patterns, treatment responses, and prognostic implications.: Systematic review of 47 atypical EMH cases in MF identifies extramedullary disease as an under-recognized marker of advanced MF with high short-term mortality, suggesting a specific clinical trigger (beyond DIPSS/MIPSS70) that should prompt transplant referral in the eligible population currently under-transplanted. (novelty: 3)
- 2026-08-04 — Survival outcomes and treatment patterns in myelofibrosis in the JAK inhibitor era.: cross-connection with dipss: DIPSS int-2/high-risk stratification defines the eligibility pool in which only ~25% actually reach transplant, linking the scoring system to a documented referral gap. (novelty: 3)
Open questions
Related
Contributing findings
New Drugs in Myelofibrosis: Incremental Progress in Symptom Management Without Disease Modification.
23-oct-2026
novelty 2
per-area 2
strengthens
Clinicopathologic Characteristics of SomaticMutations in Philadelphia Chromosome-Negative Myeloproliferative Neoplasms: A Systematic Review.
19-sep-2026
novelty 3
per-area 3
strengthens
Haplo-HSCT for Myelofibrosis
14-sep-2026
novelty 2
per-area 2
strengthens
You’re Not Alone: A Myelofibrosis Roadmap
31-aug-2026
novelty 1
per-area 1
mentions
Survival outcomes and treatment patterns in myelofibrosis in the JAK inhibitor era.
04-aug-2026
novelty 3
per-area 3
introduces
Reduced Intensity Haploidentical Transplantation for the Treatment of Primary or Secondary Myelofibrosis
20-jul-2026
novelty 3
per-area 2
mentions
Acute Myeloid Leukemia Subtype-Specific Prognostic Value of NGS in the Setting of Intensive Chemotherapy.
18-jul-2026
novelty 3
per-area 2
mentions
Study of Stem Cell Transplant vs. Non-Transplant Therapies in High-Risk Myelofibrosis
06-jul-2026
novelty 2
per-area 3
strengthens
A systematic review of 47 published cases of extramedullary hematopoiesis in myelofibrosis: clinical patterns, treatment responses, and prognostic implications.
30-jun-2026
novelty 3
per-area 3
strengthens
Primary Myelofibrosis (PMF)-The German ONKOPEDIA Guideline 2025.
21-jun-2026
novelty 2
per-area 2
mentions